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Item 1. Business

2026-02-26 compared with 2025-03-03 · 404 added, 290 removed, 695 unchanged (50% of the section changed)

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We are a biopharmaceutical company focused on discovering, developing, and commercializing potential best-in-class medicines for serious and rare diseases.

We target disease areas where marketed therapies often leave room for improvements in efficacy, safety, and/or dosing convenience.

We believe that first-generation medicines rarely represent optimal solutions, especially in rare disease areas, and that there is potential to develop differentiated, best-in-class medicines that could lead to improved patient outcomes, reduced side effects, improved quality of life, expanded market access, and augmented market competition.

Our business model is designed to identify and evaluate product opportunities in disease areas where trial data establishes proof-of-concept for a drug target in the clinic, but the competitive evolution of the product life cycle management and number of entrants appears incomplete.

We intend to prioritize indications where a fast-follower and a potentially differentiated drug candidate, or overall product profile, could create significant medical benefit for patients.

We are engineering product candidates to address unmet medical needs for patients and further advance drug innovation.

Our goal is to identify and evaluate product concepts leveraging clinically validated molecular targets using established therapeutic modalities.

We prioritize product concepts that are aligned with clinical and commercial hypotheses, which we expect will provide an attractive balance of risk and opportunity, thereby representing a compelling allocation of our resources.

We focus on advancing therapeutic proteins that we either in-license or discover internally, incorporating proprietary therapeutic protein and antibody discovery and optimization platforms to advance clinical candidates with unique characteristics.

We have built relevant expertise in protein and antibody discovery and engineering, biologics manufacturing, nonclinical and clinical development, and commercialization for thyroid eye disease (“TED”), development of anti-neonatal Fc receptor (“FcRn”) therapies, and nonclinical and clinical development for indications in rare and autoimmune diseases.

Our approach to rapidly discovering and developing novel therapeutics relies on our scientific expertise in evaluating pre-existing clinical proof-of-concept data for the drug targets we are pursuing, and opportunities to improve upon existing investigational and/or approved therapies.

This approach informs how we design, select, and develop our product candidates, including in critical areas such as pharmacokinetics, pharmacodynamics, clinical trial design, trial endpoints, and the selection and recruitment of patients.

We believe this strategy reduces the risks associated with discovering and developing novel therapeutics.

Development of Therapies to Treat Thyroid Eye Disease (TED)

We target therapeutic areas in which current treatments leave room for improvements in efficacy, safety, and/or dosing convenience.

We believe there is significant potential in these areas, for better medicines that address unmet needs, improve outcomes, and expand treatment options for patients.

We aim to develop differentiated, potential best-in-class medicines that could lead to improved patient outcomes, reduced side effects, improved quality of life, and expanded market access.

Our pipeline targets validated pathways and disease-driving mechanisms in autoimmune and rare diseases.

These include product candidates directed at the insulin-like growth factor 1 receptor (“IGF‑1R”) for the treatment of thyroid eye disease (“TED”), inhibitors of the neonatal Fc receptor (“FcRn”) with potential application across multiple autoimmune disorders, and a TSHR inhibitor program with potential in TED and Graves’ disease.

We develop therapeutics through internal research and discovery, as well as through in-licensing opportunities that align with our strategic focus.

Our capabilities span protein and antibody discovery and engineering, biologics manufacturing, nonclinical and clinical development, commercial planning, and commercialization in these therapeutic areas.

As we prepare for the anticipated launch of our first commercial product, if approved, we are building the infrastructure we believe is required to support a successful transition to a commercial organization.

This includes establishing sales and marketing, market access, patient services, and commercial operations functions, and expanding our medical, clinical, regulatory, quality, and supply chain and distribution capabilities.

Our commercial readiness efforts focus on enabling reliable access for patients, supporting physicians, and engaging effectively with payors.

Our strategy combines clear scientific, clinical, and commercial rationale with excellence in execution to rapidly discover, develop, and commercialize better medicines for patients.

We rely on our scientific, clinical, and commercial expertise to identify opportunities to improve upon existing investigational or approved therapies and to apply these insights to designing, selecting, developing, and commercializing potential best-in-class product candidates.

We bring potential improvements to critical areas such as molecular design, dose selection, pharmacokinetics, pharmacodynamics, clinical trial design, trial endpoints, and the selection and recruitment of patients.

We believe this strategy enables efficient product development and reduces the risk when developing novel therapeutics.

Development of IGF-1R Therapies to Treat Thyroid Eye Disease (TED)

We are developing therapies for the treatment of TED, a serious and debilitating rare autoimmune disease that causes inflammation within the orbit of the eye that can cause bulging of the eyes, redness and swelling, double vision, pain, and potential blindness.

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TED is a progressive disease consisting of an initial active phase (“active TED”), followed by a transition to a secondary chronic phase (“chronic TED”).

The only medicine approved by the FDA for TED is Tepezza® (teprotumumab), which is an intravenously administered monoclonal antibody that targets insulin-like growth factor 1 receptor (“IGF-1R”).

Tepezza is marketed in the United States (“U.S.”) by Horizon Therapeutics plc (“Horizon”), which was acquired by Amgen Inc.

(“Amgen”) in October 2023.

In 2024, Amgen gained approval for Tepezza in Japan and submitted a Marketing Authorization Application (“MAA”) to the European Medicines Agency (“EMA”).

The results from clinical trials of teprotumumab conducted by Horizon provide strong clinical validation linking the targeting of IGF-1R to clinical benefit in patients with TED.

However, clinical trials evaluating teprotumumab in patients with TED reported to date used a single dosing regimen, providing little guidance as to the optimal dosing required for clinical activity in TED.

We believe that there are multiple opportunities to develop fast-follower therapeutics that improve on teprotumumab’s features, including dosing schedule, route of administration, and safety profile.

We are developing two product candidates, veligrotug (formerly known as VRDN-001) for intravenous (“IV”) and VRDN-003 for subcutaneous (“SC”) administration, to treat patients who suffer from TED.

The only medicine approved by the FDA for TED is Tepezza® (teprotumumab), which is an intravenously administered monoclonal antibody that targets IGF-1R.

Tepezza is marketed in the United States (“U.S.”) by Amgen Inc.

Amgen gained approval for Tepezza in Japan in 2024 and from the European Commission in 2025.

We are developing two anti-IGF-1R product candidates, veligrotug for intravenous (“IV”) administration and elegrobart (formerly known as VRDN-003) for subcutaneous (“SC”) administration, to treat patients who suffer from TED.

Our most advanced program, veligrotug, is a differentiated humanized monoclonal antibody targeting IGF-1R intravenously administered for the treatment of TED.

In previously presented in vitro nonclinical data, we showed that veligrotug is a potentially differentiated full antagonist of IGF-1R, compared to teprotumumab’s incomplete antagonism of IGF-1R.

VRDN-003 has the same binding domain as veligrotug, and was engineered to have a longer half-life.

VRDN-003 is designed to be a low-volume, infrequently-dosed subcutaneous IGF-1R for TED, which we plan to launch commercially with an auto-injector to enable at-home patient self-

administration.

We believe VRDN-003 has the potential to be the best-in-class subcutaneous anti-IGF-1R product candidate by preserving the efficacy of anti-IGF-1Rs in TED, improving safety and maximizing convenience for patients.

We conducted phase 1/2 clinical trials of veligrotug in patients with active or chronic TED.

In the active TED portion of the phase 1/2 clinical trials, data reported from all three dose cohorts of veligrotug (n=21) showed significant and rapid improvement in both the signs and symptoms of TED after two infusions of veligrotug compared to placebo.

Across all veligrotug treated patients in the active TED trial, 71% were proptosis responders, 67% were overall responders, 62% achieved a clinical activity score (“CAS”) of 0 or 1, and 54% had complete resolution of their diplopia.

In the chronic TED portion of the phase 1/2 clinical trials, data reported from both dose cohorts of veligrotug (n=12) showed significant and rapid improvement in the signs and symptoms of TED after two infusions of veligrotug compared to placebo.

Across all veligrotug treated patients in the chronic TED trial, 42% were proptosis responders, 40% achieved a CAS of 0 or 1, and no patients had complete resolution of their diplopia.

In the phase 1/2 clinical trials of both active and chronic TED, veligrotug had a favorable safety profile and was well-tolerated by all patients treated in all dose cohorts.

We are conducting a global pivotal program for veligrotug, including evaluating its efficacy and safety in two global well-controlled phase 3 clinical trials, THRIVE and THRIVE-2, for the treatment of active and chronic TED, respectively.

THRIVE and THRIVE-2 are each designed to compare a five-dose IV treatment arm of veligrotug at 10 mg/kg, dosed three weeks apart, to placebo.

Elegrobart has the same binding domain as veligrotug, and was engineered to have a longer half-life.

Elegrobart is designed to be a low-volume, infrequently-dosed subcutaneous IGF-1R for TED, which we plan to launch commercially with an auto-injector to enable at-home patient self-administration.

We believe elegrobart has the potential to be the best-in-class anti-IGF-1R product candidate by preserving the efficacy of anti-IGF-1Rs in TED, improving safety, and maximizing convenience for patients with subcutaneous delivery.

We conducted a global pivotal clinical program for veligrotug, evaluating its efficacy and safety in two global well-controlled phase 3 clinical trials, THRIVE and THRIVE-2, for the treatment of active and chronic TED, respectively.

THRIVE-2 were each designed to compare a five-dose IV treatment arm of veligrotug at 10 mg/kg, dosed three weeks apart, to placebo.

This five-dose veligrotug regimen features fewer infusions and a shorter time per infusion compared to teprotumumab, the currently marketed IGF-1R inhibitor.

On September 10, 2024, we announced topline data from the THRIVE study, which enrolled 113 patients, randomized to veligrotug (n=75) and placebo (n=38).

THRIVE achieved all primary and secondary endpoints with a high level of statistical significance (p < 0.0001) and was generally well-tolerated, with no treatment-related serious adverse events (“SAEs”).

In September 2024, we announced topline data from the THRIVE study, which enrolled 113 patients, randomized to veligrotug (n=75) and placebo (n=38).

THRIVE achieved its primary and all secondary endpoints with a high level of statistical significance (p < 0.0001) and was generally well-tolerated, with no treatment-related serious adverse events (“SAEs”).

Veligrotug additionally showed a rapid onset of treatment effect, with the majority (53%) of veligrotug-treated patients achieving a proptosis response as early as three weeks.

On December 16, 2024, we announced topline data from the THRIVE-2 study, which enrolled 188 patients, randomized to veligrotug (n=125) and placebo (n=63).

THRIVE-2 achieved all primary and secondary endpoints with statistical significance and was generally well-tolerated.

Veligrotug continued to demonstrate a rapid onset of treatment effect, with a statistically significant proptosis response as early as three weeks and a statistically significant reduction and resolution of diplopia as early as six weeks.

In December 2024, we announced topline data from the THRIVE-2 study, which enrolled 188 patients, randomized to veligrotug (n=125) and placebo (n=63).

THRIVE-2 achieved its primary and all secondary endpoints with statistical significance and was generally well-tolerated.

Veligrotug demonstrated a rapid onset of treatment effect in THRIVE-2, with a statistically significant proptosis response as early as three weeks and a statistically significant reduction and resolution of diplopia as early as six weeks.

THRIVE-2 is the first global phase 3 study in patients with chronic TED to demonstrate a statistically significant and clinically meaningful diplopia responder rate and rate of diplopia complete resolution.

To meet the 300 patient standard safety database requirements for the veligrotug biologics license application (“BLA”), we are conducting our STRIVE clinical trial (safety database inclusive of patients from the THRIVE and THRIVE-2 trials).

STRIVE is a global study of veligrotug in TED patients that utilizes broad inclusion criteria (e.g., any severity or duration of disease) and is randomized 3:1 (10 mg/kg IV with an active control of 3 mg/kg IV).

In January 2025, we completed enrollment in STRIVE with a total of 231 patients, exceeding the enrollment target of 212 due to patient demand.

We have also completed enrollment of the open label extension study for non-responding patients in THRIVE and THRIVE-2.

We anticipate submitting a BLA for veligrotug in the second half of 2025 and an MAA to the EMA in the first half of 2026.

In addition to our intravenous veligrotug program, VRDN-003 is our subcutaneous product candidate currently in pivotal development in TED, which we selected in December 2023 following positive data in a phase 1 clinical trial in healthy volunteers.

The VRDN-003 phase 1 clinical study showed VRDN-003 to have a prolonged half-life of 40 to 50 days, which is four to five times that of veligrotug.

Because of the healthy volunteer data and the similarities between the veligrotug and VRDN-003 antibodies, we expect VRDN-003 to have similar clinical responses at the exposure levels of veligrotug that led to robust clinical activity in its clinical trials to date in TED.

Further, pharmacokinetic modeling of VRDN-003 based on the healthy volunteer data predicted that exposure levels of VRDN-003 could be achieved that are equivalent to exposure levels of veligrotug that produced clinically meaningful results with multiple dosing regimens of VRDN-003, i.e., subcutaneous injection every two, four, or eight weeks.

We are conducting a global pivotal program for VRDN-003, including evaluating its efficacy and safety in two global well-controlled phase 3 clinical trials, REVEAL-1 and REVEAL-2, for the treatment of active and chronic TED, respectively.

Both studies will evaluate subcutaneous VRDN-003 administered every four weeks or every eight weeks and will assess outcomes versus placebo.

Patient enrollment and dosing continues in both studies.

In addition, to enable BLA submission for VRDN-003, we have initiated a safety study to meet the 300 patient standard safety database requirement (to also include patients from the REVEAL-1 and REVEAL-2 trials) and plan to initiate an auto-injector study in 2025 to enable launching VRDN-003 in an auto-injector device, if approved.

We anticipate topline data for REVEAL-1 and REVEAL-2 in the first half of 2026, and we anticipate submitting a BLA for VRDN-003 for the treatment of TED by the end of 2026.

Veligrotug demonstrated durability at 52 weeks in THRIVE, showing that 70% of patients who were proptosis responders at week 15 maintained their response at week 52.

To meet the 300 patient safety database requirement for the veligrotug BLA, we are conducting STRIVE, a global phase 3 clinical trial.

STRIVE enrolled 231 TED patients, utilized broad inclusion criteria (e.g., any severity or duration of disease), and randomized patients 3:1 (10 mg/kg IV with an active control of 3 mg/kg IV).

We are also conducting an open label extension study for non-responding patients in THRIVE and THRIVE-2 which has completed enrollment.

In May 2025, the FDA granted Breakthrough Therapy designation to veligrotug.

We submitted a BLA for veligrotug to the FDA in October 2025, which was accepted for filing and granted Priority Review in December 2025 with a Prescription Drug User Free Act (“PDUFA”) target action date of June 30, 2026.

We additionally submitted an MAA to the EMA in January 2026.

We are also developing elegrobart, our subcutaneous anti-IGF-1R product candidate currently in pivotal clinical studies in TED, which we selected in December 2023 following positive data in a phase 1 clinical trial in healthy volunteers.

In its phase 1 clinical study in healthy volunteers, elegrobart was shown to have a prolonged half-life of 40 to 50 days, which is four to five times that of veligrotug.

Based on this data and the similarities between the veligrotug and elegrobart antibodies, we selected Q4W and Q8W dosing of elegrobart to advance to phase 3 pivotal studies.

PK modeling showed Q4W and Q8W subcutaneous elegrobart dosing could achieve the range of modeled veligrotug exposures based on a two-infusion phase 2 TED study at 3 mg/kg and 10 mg/kg IV, once every three weeks.

Both dosing regimens of veligrotug showed robust clinical activity.

We are conducting a global pivotal program for elegrobart, including evaluating its efficacy and safety in two global well-controlled phase 3 clinical trials, REVEAL-1 and REVEAL-2, for the treatment of active and chronic TED, respectively.

Both studies are evaluating elegrobart administered subcutaneously every four weeks or every eight weeks and will assess outcomes versus placebo.

In September 2025, we announced that REVEAL-1 and REVEAL-2 completed enrollment, enrolling 132 and 204 patients, respectively, each exceeding its target enrollments of 117 and 195 patients, respectively, due to demand.

67% of REVEAL-1 patients were enrolled from the U.S., and 56% of REVEAL-2 patients were enrolled from the U.S.

In addition, to enable BLA submission for elegrobart, we are conducting a safety study to meet the 300 patient safety database requirement (to also include patients from the REVEAL-1 and REVEAL-2 trials).

We completed enrollment of this safety study in October 2025, enrolling 321 patients, exceeding the target enrollment of 284 patients due to demand.

Additionally, we are conducting an auto-injector study to enable launching elegrobart in an auto-injector device, if approved.

We completed enrollment in the autoinjector study in December 2025, enrolling 87 patients, exceeding the target enrollment of 75 patients.

We anticipate topline data for REVEAL-1 in the first quarter of 2026 and REVEAL-2 in the second quarter of 2026.

Development of FcRn Inhibitors

In addition to developing therapies for TED, we are also developing a portfolio of engineered FcRn inhibitors, including VRDN-006 and VRDN-008.

We are also developing a portfolio of engineered FcRn inhibitors, including VRDN-006 and VRDN-008.

FcRn inhibitors have the potential to treat a broad array of autoimmune diseases, representing a possible significant commercial market opportunity.

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In our head-to-head NHP studies, VRDN-006 demonstrated comparable potency and IgG reductions to efgartigimod, which is the current standard of care in FcRn inhibition, as well as a similar safety profile.

We submitted an IND for VRDN-006 in December 2024, which cleared in January 2025, and we expect proof-of-concept IgG reduction data in healthy volunteers in the third quarter of 2025.

We submitted an IND for VRDN-006 in December 2024, which cleared

in January 2025.

In September 2025, we announced that data from an ongoing phase 1 clinical trial in healthy volunteers showed that VRDN-006 led to IgG reductions that are consistent with the FcRn inhibitor class, and that VRDN-006 was sparing of albumin and LDL and was generally well-tolerated with no dose-limiting toxicities or serious adverse events.

VRDN-008 is a half-life extended bispecific FcRn inhibitor comprising an Fc fragment and an albumin-binding domain designed to prolong IgG suppression and provide a potentially best-in-class subcutaneous option for patients.

In a single, high-dose, head-to-head study in NHPs, VRDN-008 demonstrated three times the half-life of efgartigimod.

Additionally, VRDN-008 showed a deeper and more sustained IgG reduction with peak IgG reductions that were 20% deeper than efgartigimod while not showing decreases in albumin or increases in LDL levels.

NHP studies are ongoing to generate additional data for VRDN-008.

Once completed, we plan to use the totality of VRDN-008’s NHP data to build a robust pharmacokinetic and pharmacodynamic model to predict potential human dosing regimens for VRDN-008.

We anticipate submitting an IND for VRDN-008 by the end of 2025.

Our mission is to create and advance new biologic medicines for patients suffering from serious and rare diseases that are underserved by today’s therapies.

Additionally, VRDN-008 showed a deeper and more sustained IgG reduction with peak IgG reductions that were 20% deeper than efgartigimod, and IgG levels returned to baseline 35 days after VRDN-008 dosing, more than twice as long as efgartigimod, which returned to baseline 14 days after dosing.

VRDN-008 spared albumin and LDL, consistent with efgartigimod.

We submitted an IND for VRDN-008 in December 2025 and received IND clearance from the FDA in January 2026.

We expect healthy volunteer data in the second half of 2026.

Development of TSHR Inhibitors

In January 2026, we announced that we are developing an anti-TSHR candidate with potential use in the treatment of Graves’ disease and TED.

This product candidate is a half‑life extended monoclonal antibody designed to inhibit activation of TSHR.

It is being developed for subcutaneous administration via autoinjector, with the goal of enabling extended dosing intervals intended to support patient convenience.

We anticipate submitting an IND for this program in the fourth quarter of 2026.

We believe inhibiting TSHR has the potential to treat both TED and Graves’ disease.

TED pathophysiology potentially stems from the activation of the TSHR and IGF-1R signaling complex on orbital fibroblasts, leading to hyaluronan secretion and expansion of orbital fat and muscle.

Autoantibodies that stimulate TSHR can activate pathways that promote inflammation, fibroblast proliferation, and tissue remodeling relevant to TED.

We believe inhibiting TSHR could complement the inhibition of IGF-1R in the treatment of TED.

In addition to TED, blocking TSHR could also be effective to treat Graves’ disease.

Graves’ disease is an autoimmune disease in which autoantibodies form against the TSHR, stimulating and activating the receptor.

These TSH receptor antibodies (“TRAb”) can drive a heightened activation of TSHR, resulting in excessive thyroid hormone production and hyperthyroidism.

Graves’ disease is one of the most prevalent autoimmune conditions, affecting more than 2 million people in the United States, and is the leading cause of hyperthyroidism.

Current treatments—including antithyroid drugs, radioactive iodine (“RAI”), and surgery—lower thyroid hormone levels but do not entirely address the underlying autoimmune drivers of the disease and are often associated with relapse or the development of permanent hypothyroidism.

Blocking TSHR activation through a TSHR antagonist represents a differentiated therapeutic approach aimed at targeting disease-driving mechanisms in TED and in Graves’ disease.

Our mission is to create and advance new medicines for patients suffering from serious and rare diseases that are underserved by today’s therapies.

Key elements of our business strategy are to:

• Evaluate opportunities to identify, engineer, and develop potential best-in-class therapeutic proteins and antibodies that optimize patient care.

Our pipeline of therapeutic programs represents a patient-centric model of innovation that leverages proven biology and protein and antibody technology to reduce research and development risk, while striving to address strategic gaps related to access, delivery, quality of life, efficacy, and/or safety and tolerability in targeted therapeutic areas.

Our multidisciplinary search process evaluates scientific and clinical validation of therapeutic targets, market potential, and feasibility of efficiently developing a competitive product.

• Our clinical development and commercial focus on thyroid eye disease (TED) :

• Identify, engineer, and develop potential best-in-class therapeutic proteins and antibodies that optimize patient care.

We develop therapeutics through internal research and discovery, as well as through in-licensing opportunities that align with our strategic focus.

We identify opportunities to advance potential best-in-class medicines that address unmet needs in autoimmune and rare diseases.

Our approach leverages proven biology on clinically validated targets and our internal capabilities such as protein and antibody engineering to reduce development risk while striving to address unmet needs for patients including improved clinical outcomes, reduced side effects, improved quality of life, and expanded market access.

• Advance our lead programs in thyroid eye disease (TED) :

◦ Seek marketing approval for veligrotug.

We are evaluating veligrotug in two pivotal global phase 3 clinical trials, THRIVE and THRIVE-2, for the treatment of active and chronic TED, respectively.

THRIVE and THRIVE-2 are each designed to compare a five-dose intravenous treatment arm of veligrotug at 10 mg/kg, dosed three weeks apart, to placebo.

We evaluated veligrotug in two pivotal global phase 3 clinical trials, THRIVE and THRIVE-2, for the treatment of active and chronic TED, respectively.

THRIVE and THRIVE-2 were each designed to compare a five-dose intravenous treatment arm of veligrotug at 10 mg/kg, dosed three weeks apart, to placebo.

This five-dose veligrotug regimen features fewer infusions and a shorter time per infusion compared to teprotumumab, the currently marketed IGF-1R inhibitor for TED.

We reported positive topline THRIVE and THRIVE-2 data in September 2024 and December 2024, respectively.

We expect that the THRIVE and THRIVE-2 phase 3 trials, together with a safety database comprising 300 treated patients (safety database inclusive of patients from the THRIVE and THRIVE-2 trials), will support global health authority registration for marketing approval in both active and chronic TED.

We expect to submit a BLA for veligrotug in the second half of 2025 and an MAA to the EMA for veligrotug in the first half of 2026.

◦ Rapidly develop subcutaneous VRDN-003 as our next generation, potential best-in-class IGF-1R antibody.

VRDN-003 is designed to be self-administered subcutaneously at home as an infrequent and low-volume injection to decrease the burden on TED patients.

VRDN-003 has the same binding domain as veligrotug and was engineered to have a longer half-life.

VRDN-003 is designed to maintain the efficacy of IGF-1Rs as demonstrated by the marketed product, teprotumumab, and by the clinical results for veligrotug in THRIVE and THRIVE-2, while improving on its safety and maximizing patient convenience.

We are evaluating VRDN-003 in two global phase 3 clinical trials, REVEAL-1 and REVEAL-2, for the treatment of active and chronic TED, respectively.

REVEAL-1 and REVEAL-2 are each designed to evaluate subcutaneous VRDN-003 administered every four weeks or every eight weeks and will assess outcomes

versus placebo.

We have initiated a safety study to meet the 300 patient standard safety database requirement (to also include patients from the REVEAL-1 and REVEAL-2 trials) and anticipate initiating an auto-injector study in 2025 to have an auto-injector device available at the time of commercial launch, if approved.

We expect to report topline REVEAL-1 and REVEAL-2 data in the first half of 2026.

We expect that the THRIVE and THRIVE-2 phase 3 trials, together with a safety database comprising 300 treated

patients (safety database inclusive of patients from the THRIVE and THRIVE-2 trials), will support global health authority registration for marketing approval in both active and chronic TED.

We submitted a BLA for veligrotug in October 2025, which was accepted for filing and granted Priority Review in December 2025 with a PDUFA target action date of June 30, 2026.

We also submitted an MAA to the EMA for veligrotug in January 2026.

◦ Rapidly develop subcutaneous elegrobart as our next generation, potential best-in-class IGF-1R antibody.

Elegrobart is designed to be self-administered subcutaneously at home as an infrequent and low-volume injection to decrease the burden on TED patients.

Elegrobart has the same binding domain as veligrotug and was engineered to have a longer half-life.

Elegrobart is designed to maintain the efficacy of IGF-1Rs as demonstrated by the marketed product, teprotumumab, and by the clinical results for veligrotug in THRIVE and THRIVE-2, with the potential to improve on its safety and maximize patient convenience.

We are evaluating elegrobart in two global phase 3 clinical trials, REVEAL-1 and REVEAL-2, for the treatment of active and chronic TED, respectively.

REVEAL-1 and REVEAL-2 are each designed to evaluate elegrobart administered subcutaneously every four weeks or every eight weeks and will assess outcomes versus placebo.

We are also conducting a safety study to meet the 300 patient safety database requirement (to also include patients from the REVEAL-1 and REVEAL-2 trials) and an auto-injector study which we believe will enable delivery via an auto-injector device at the time of commercial launch, if approved.

We expect to report topline REVEAL-1 and REVEAL-2 data in the first quarter and second quarter of 2026, respectively.

We expect that the REVEAL-1 and REVEAL-2 phase 3 trials, together with a safety database of 300 treated patients (safety database inclusive of patients from the REVEAL-1 and REVEAL-2 trials), will support global health authority registration for marketing approval in both active and chronic TED, respectively.

◦ Be a trusted partner in the care of TED patients.

We plan to cultivate a network across TED stakeholders to inform our patient-centric approach, including with patients and advocacy groups, key opinion leaders, research institutions, healthcare professionals and payers.

◦ Prepare for commercialization of veligrotug and VRDN-003, for the treatment of patients with TED.

We hold worldwide commercialization rights, excluding the greater area of China, to veligrotug and VRDN-003.

As a result, we have the flexibility to develop and potentially commercialize products ourselves, or alternatively to enter collaborations with industry partners.

◦ Commercially launch veligrotug, if approved, and prepare for the commercialization of elegrobart, for the treatment of patients with TED.

We hold worldwide commercialization rights, excluding Japan and the greater area of China, to veligrotug and elegrobart.

We are cultivating a network across TED stakeholders to launch our potential products with a patient-centric approach, including partnering with patients and advocacy groups, key opinion leaders, research institutions, healthcare professionals, and payers.

market, to plan for the anticipated launch of veligrotug, if approved, we are building the infrastructure required to support a successful transition to a commercial organization.

Our commercial readiness efforts focus on enabling reliable access for patients, supporting physicians, and engaging effectively with payors.

We believe these efforts to commercialize veligrotug will directly benefit the commercial launch of elegrobart.

Outside of the U.S.

in the regions where we have commercial rights, we have the flexibility to develop and potentially commercialize products ourselves, or alternatively to enter collaborations with industry partners.

• Advance our portfolio of FcRn inhibitors with the potential to treat a broad array of autoimmune disorders.

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Our multi-pronged engineering approach has resulted in a portfolio of FcRn-targeting molecules that leverage the clinically and commercially validated mechanism of FcRn inhibition while potentially addressing the limitations of current agents such as incomplete IgG suppression and safety.

◦ Generate VRDN-006 proof-of-concept IgG reduction data in healthy volunteers.

VRDN-006 is a highly selective Fc fragment that inhibits FcRn and is designed to be a convenient subcutaneous and self-administered option for patients.

VRDN-006 received IND clearance from the FDA in January 2025 and we expect proof-of-concept IgG reduction data in healthy volunteers in the third quarter of 2025.

◦ Advance VRDN-008 to the clinic.

◦ Complete VRDN-006 phase 1 study and communicate development plan.

We expect to communicate future development plans for VRDN-006 in 2026.

◦ Generate VRDN-008 proof-of-concept IgG reduction in healthy volunteers.

VRDN-008 is a half-life extended bispecific FcRn inhibitor comprising an Fc fragment and an albumin-binding domain designed to prolong IgG suppression as a subcutaneous, self-administered and potential best-in-class option for patients.

In single, high-dose head-to-head NHP studies, VRDN-008 demonstrated three times the half-life and showed a deeper and more sustained IgG reduction than efgartigimod.

NHP studies are ongoing to generate additional data for VRDN-008.

Once completed, we plan to use the totality of VRDN-008’s NHP data to build a robust pharmacokinetic and pharmacodynamic model to predict potential human dosing regimens for VRDN-008.

We anticipate submitting an IND for VRDN-008 by the end of 2025.

• Leverage our therapeutic protein, antibody, and multi-disciplinary search expertise to continue discovering and developing novel, best-in class product candidates.

We plan to continue to identify and advance novel product candidates and technologies to generate potential best-in-class protein and antibody therapeutics, either internally or through in-licensing.

We submitted an IND for VRDN-008 in December 2025 and received IND clearance from the FDA in January 2026.

We expect healthy volunteer data in the second half of 2026.

• Leverage our differentiated strategy, strong capabilities, and track record of execution to continue discovering and developing novel, potential best-in class product candidates.

We plan to continue to identify and advance novel product candidates and technologies to generate potential best-in-class therapeutics, including proteins and antibodies, either internally or through in-licensing.

◦ Advance our TSHR candidate to the clinic.

Our TSHR candidate is a potential best-in-class, half-life extended product candidate.

We anticipate submitting an IND for our TSHR program in the fourth quarter of 2026.

◦ Continue to identify and advance novel product candidates .

We plan to continue to identify and engineer, through internal discovery efforts, novel product candidates and technologies that have the potential to be best-in-class therapeutics.

On identifying such product candidates, we plan to advance and develop them to address unmet needs for patients.

◦ Identify external opportunities for potential in-licensing.

Monitor external opportunities and in-license potential assets that align with our strategic focus.

Thyroid Eye Disease (TED)

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Pathologies Leading to the Development of TED

TED develops in parallel with Graves’ Disease, an autoimmune disease in which antibodies form against the thyroid-stimulating hormone receptor (“TSHR”), which is present in the thyroid and other cells such as adipocytes and fibroblasts.

A close temporal relationship exists between the onset of Graves’ Disease and the onset of TED.

TED develops in parallel with Graves’ disease, an autoimmune disease in which autoantibodies form against the thyroid-stimulating hormone receptor (“TSHR”), which is present in the thyroid and other cells such as adipocytes and fibroblasts.

close temporal relationship exists between the onset of Graves’ Disease and the onset of TED.

Regardless of which condition occurs first, the other condition develops within 18 months in 80% of patients.

In addition to antibodies against TSHR, patients with TED also develop antibodies against IGF-1R.

Insulin-like growth factor 1 is a hormone similar in molecular structure to insulin with higher growth-promoting activity.

In addition to autoantibodies against TSHR, patients with TED may also develop autoantibodies against IGF-1R.

Insulin-like growth factor 1 (“IGF-1”) is a hormone similar in molecular structure to insulin with higher growth-promoting activity.

IGF-1R, the receptor for IGF-1, is highly expressed in fibrocytes, cells that are derived from the bone marrow and that have the potential to differentiate into either myofibroblasts or fat cells.

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One potential cause of TED is autoimmune antibodies against IGF-1R that lead to the activation of IGF-1R, resulting in increased proliferation, secretion of extracellular complex carbohydrates, and differentiation into fat cells.

These antibodies, and autoimmune antibodies to TSHR, can elicit an immune attack against the fibrocytes that surround the eye triggering the

development of TED.

These antibodies, and autoimmune antibodies to TSHR, can elicit an immune attack against the fibrocytes that surround the eye triggering the development of TED.

Inflammation associated with this attack combined with activation of IGF-1R leads to the wide spectrum of pathologies seen with this disease.

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The Tepezza labeling was updated in April 2023 to specify its use for the treatment of TED regardless of TED activity or duration.

In addition, the labeling was updated in its warnings and precautions, adverse reactions, and patient counseling information sections in December 2022 with additional information on hyperglycemia and in July 2023 to include hearing impairment, its potential severity, and guidance for monitoring.

In two randomized, double-blind placebo-controlled trials, infusions of teprotumumab every three weeks, for a total of eight doses, led to a greater than 2 mm decrease in proptosis in 71% and 83% of patients with active TED, respectively, compared to 20% and 10% with placebo.

Combined results from these two studies in patients with active TED showed that treatment with teprotumumab also led to a 53% decrease in diplopia compared to a 25% decrease when patients were treated with placebo control.

Since its initial approval in 2020, the label has been subsequently updated multiple times with additional information on administration, risks and adverse reactions.

Thus, these data show that targeting and blockade of IGF-1R in TED provides a clinically meaningful benefit and is a de-risked approach.

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We estimate approximately 190,000 TED patients in the United States with moderate to severe TED, which includes patients with either active and chronic TED, and a similar epidemiology in Europe.

In 2020, Horizon launched with a price of approximately $16,300 per vial of Tepezza which translates to a list price of approximately $375,000 based on patient weight for a six-month course of therapy.

Tepezza’s 2024 net sales in the U.S.

were approximately $1.8 billion, with the potential for additional revenue in the U.S.

Currently, each vial of Tepezza has an approximate price of $18,700, which translates to a list price of approximately $525,000 based on patient weight for a six-month course of therapy.

Tepezza’s 2025 net sales were approximately $1.9 billion with estimated single digit annual penetration of the addressable moderate to severe TED population.

We believe there is potential for additional revenue in the U.S.

with further penetration into the prevalent TED population and more convenient regimens and routes of administration.

With Tepezza now approved in multiple countries outside of the U.S., including Japan, and a regulatory submission completed in Europe, we believe there is potential for meaningful additional revenue outside of the U.S., which we believe will further support multiple entrants.

With Tepezza now approved in multiple countries outside of the U.S., including Japan and the European Union, we believe there is potential for meaningful additional revenue outside of the U.S., which we believe will further support multiple entrants.

Our Product Candidates

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This mechanism of action is clinically and commercially validated by the only FDA product approved for the treatment of TED, Tepezza.

Based on our ongoing THRIVE and THRIVE-2 phase 3 clinical trials, our goal is for veligrotug to be second to market in this class of medicine, with the opportunity to offer a differentiated IV product.

Based on the THRIVE and THRIVE-2 phase 3 clinical trials, our goal is for veligrotug to be second to market in this

class of medicine, with the opportunity to offer a differentiated IV product.

The FDA granted Breakthrough Therapy Designation to veligrotug in May 2025.

Further, the FDA accepted the veligrotug BLA for filing in December 2025 under Priority Review with a PDUFA target action date of June 30, 2026.

We have an exclusive license to the worldwide rights to develop and commercialize veligrotug for all non-oncology indications that do not use radiopharmaceuticals, including the treatment of patients with TED, from ImmunoGen, Inc.

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However, development in oncology was stopped in 2009 due to its failure to meet the primary efficacy endpoints in multiple myeloma.

As described below, we sublicensed the right to develop, manufacture and commercialize certain IGF-1R directed antibody products for non-oncology indications in the greater area of China to Zenas BioPharma (Cayman) Limited (now Zenas BioPharma, Inc., their successor in interest, “Zenas BioPharma”).

As described below, we licensed the right to develop, manufacture and commercialize certain IGF-1R directed antibody products for non-oncology indications in the greater area of China to Zenas BioPharma (Cayman) Limited (now Zenas BioPharma, Inc., their successor in interest, “Zenas BioPharma”).

In January 2025, Zenas BioPharma sublicensed their rights to Zai Lab (Hong King) Limited (“Zai Lab”).

As described below, in July 2025, we licensed the right to develop, manufacture under certain limited conditions, and commercialize veligrotug and elegrobart in Japan to Kissei Pharmaceuticals Co., Ltd.

Clinical Trials for veligrotug

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THRIVE enrolled 113 patients, randomized to veligrotug (n=75) and placebo (n=38).

The following activity was observed in veligrotug-treated patients at week fifteen (n=75):

The following activity was observed in veligrotug-treated patients at the week fifteen interim topline database lock (n=75):

• 70% proptosis responder rate (“PRR”) (64% placebo-adjusted, p < 0.0001), defined as a ≥2-millimeter (“mm”) reduction in proptosis from baseline in the study eye without worsening in the fellow eye (≥2 mm increase), as measured by exophthalmometry.

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A 5.5% placebo-adjusted rate of hearing impairment AEs was observed.

The vast majority of adverse events reported at the week fifteen topline readout had resolved by week fifty-two at final database lock.

Phase 3 Trial (THRIVE-2) of veligrotug in Patients with Chronic TED

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The mean time since onset of TED in patients treated with veligrotug was 69.8 months.

The following activity was observed in veligrotug-treated patients at week fifteen (n=125):

The following activity was observed in veligrotug-treated patients at the week fifteen interim topline database lock (n=125):

• 56% proptosis responder rate (“PRR”) (48% placebo-adjusted;

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A 9.6% placebo-adjusted rate of hearing impairment AEs was observed.

To meet the 300 patient standard safety database requirements for the veligrotug BLA, we are conducting STRIVE, a global clinical study of veligrotug in TED patients that utilizes broad inclusion criteria (e.g., any severity or duration of disease) and is randomized 3:1 (10 mg/kg IV with an active control of 3 mg/kg IV).

To meet the 300 patient safety database requirements for the veligrotug BLA, we are conducting STRIVE, a global clinical study of veligrotug in TED patients that utilizes broad inclusion criteria (e.g., any severity or duration of disease) and is randomized 3:1 (10 mg/kg IV with an active control of 3 mg/kg IV).

In January 2025, we completed enrollment in STRIVE with a total of 231 patients, exceeding the enrollment target of 212 due to patient demand.

We also completed enrollment of patients in the open label extension study for non-responding patients in THRIVE and THRIVE-2.

Together, THRIVE and THRIVE-2 evaluated veligrotug in the largest and broadest population of active and chronic TED patients studied to date in global phase 3 clinical trials.

In May 2025, we announced that veligrotug received Breakthrough Therapy Designation from the FDA for the treatment of TED.

The Breakthrough Therapy Designation request included data on veligrotug’s (i) consistent and robust improvement and resolution of diplopia in chronic TED, and (ii) rapid onset of proptosis response.

In May 2025, we announced positive long-term durability data from the phase 3 THRIVE trial following our final database lock, with 70% of patients who were proptosis responders at week fifteen maintaining their response at week fifty-two.

Maintenance of response is defined as responders at week fifteen who still had at least a 2-millimeter (mm) reduction in proptosis compared to baseline at week fifty-two, without worsening in the fellow eye (≥2 mm increase), as measured by exophthalmometry.

There were no changes to the safety profile in the follow-up period.

The vast majority of adverse events reported at the week fifteen topline readout had resolved by week fifty-two at final database lock.

Together, THRIVE and THRIVE-2 evaluated veligrotug in the largest and broadest population of active and chronic TED patients completed to date in global phase 3 clinical trials.

We believe that these robust and consistent clinical efficacy and safety results, after only five infusions of veligrotug, support a differentiated product profile and the potential for veligrotug to be the IV treatment-of-choice for all forms of active and chronic TED.

We expect that the THRIVE and THRIVE-2 phase 3 clinical trials, together with a safety database comprising 300 treated patients, will support global health authority registration for marketing approval in both active and chronic TED, respectively.

We expect to submit a BLA in the second half of 2025 and an MAA to the EMA in the first half of 2026.

VRDN-003, a potential best-in-class subcutaneously administered IGF-1R antibody

VRDN-003 is a monoclonal antibody that acts as a full antagonist of IGF-1R.

VRDN-003 utilizes the same binding domain as veligrotug and is engineered to extend its half-life, and therefore potentially enabling less frequent and more convenient dosing.

VRDN-003 is designed to maintain the clinical response of veligrotug IV while significantly increasing patient convenience.

We submitted a BLA for veligrotug to the FDA in October 2025, which was accepted for filing and granted Priority Review in December 2025 with a PDUFA target action date of June 30, 2026.

We additionally submitted an MAA to the EMA in January 2026.

Elegrobart, a potential best-in-class subcutaneously administered IGF-1R antibody

Elegrobart is a monoclonal antibody that acts as a full antagonist of IGF-1R.

Elegrobart utilizes the same binding domain as veligrotug and is engineered to extend its half-life, and therefore potentially enabling less frequent and more convenient dosing.

Elegrobart is designed to maintain the clinical response of veligrotug while significantly increasing patient convenience.

We believe a later-entrant subcutaneous therapy can convert meaningful portions of an IV market.

There is precedent that subcutaneous therapies can quickly command substantial market share even when launching several years after an incumbent IV.

Although we have designed VRDN-003 to have a superior dosing profile to its parent molecule, veligrotug, as well as to the currently marketed IV product, teprotumumab, market examples demonstrate that subcutaneous therapies have commanded market share even where such therapies have the same or worse dosing frequency than their IV counterparts.

Although we have designed elegrobart to have a superior dosing profile to veligrotug, as well as to the currently marketed IV product, teprotumumab, market examples demonstrate that subcutaneous therapies have commanded market share even where such therapies have the same or worse dosing frequency than their IV counterparts.

Further, subcutaneous offerings can also grow the overall market size for their class.

We believe VRDN-003 has significant potential to capture a meaningful proportion of the IV TED market and to grow the TED market as a convenient, less-frequent, low-volume subcutaneous anti-IGF-1R antibody that patients take at home.

We are conducting a global pivotal program for VRDN-003, including evaluating its efficacy and safety in two global well-controlled phase 3 pivotal clinical trials, REVEAL-1 and REVEAL-2, for the treatment of active and chronic TED, respectively.

We selected VRDN-003 for pivotal development in TED following positive data in a phase 1 clinical trial in healthy volunteers.

This study showed VRDN-003 to have a prolonged half-life of 40 to 50 days, which is four to five times that of its parent molecule, veligrotug.

Because of the similarities of veligrotug and VRDN-003, we expect VRDN-003 to have similar clinical responses at the exposure levels of veligrotug that led to robust clinical activity in its phase 2 clinical study in TED.

Further, pharmacokinetic modeling of VRDN-003 predicted that exposure levels of VRDN-003 could be achieved that are equivalent to exposure levels of veligrotug that produced clinically meaningful results with multiple dosing regimens of VRDN-003, i.e., subcutaneous injection every two, four, or eight weeks.

Patient enrollment and dosing continues in both REVEAL-1 and REVEAL-2.

Both studies will evaluate subcutaneous VRDN-003 administered every four weeks or every eight weeks and will assess outcomes versus placebo.

We believe elegrobart has significant potential to capture a meaningful proportion of the IV TED market and to grow the TED market with a convenient, less-frequent, low-volume subcutaneous anti-IGF-1R antibody auto-injector that patients take at home.

We are conducting a global pivotal program for elegrobart, including evaluating its efficacy and safety in two global well-controlled phase 3 pivotal clinical trials, REVEAL-1 and REVEAL-2, for the treatment of active and chronic TED, respectively.

We selected elegrobart for pivotal development in TED following positive data in a phase 1 clinical trial in healthy volunteers.

This study showed elegrobart to have a prolonged half-life of 40 to 50 days, which is four to five times that of veligrotug.

Because of the similarities of veligrotug and elegrobart, we anticipate elegrobart to have a favorable risk benefit profile in TED at similar exposure levels of veligrotug.

Our pharmacokinetic modeling of elegrobart predicted that exposure levels of elegrobart could be achieved that are equivalent to exposure levels of veligrotug that produced clinically meaningful results with multiple dosing regimens of elegrobart, i.e., subcutaneous injection every two, four, or eight weeks.

REVEAL-1 and REVEAL-2 are evaluating subcutaneous elegrobart administered every four weeks or every eight weeks and will assess outcomes versus placebo.

We expect that the REVEAL-1 and REVEAL-2 phase 3 trials, together with a safety database of 300 patients (safety database inclusive of patients from the REVEAL-1 and REVEAL-2 trials), will support global health authority registration for marketing approval in both active and chronic TED, respectively.

We also anticipate initiating an auto-injector study in 2025 to have an auto-injector device available at the time of commercial launch, if approved.

We anticipate topline data for REVEAL-1 and REVEAL-2 in the first half of 2026, and we anticipate submitting a BLA for VRDN-003 for the treatment of TED by the end of 2026.

Phase 1 Trial of VRDN-003 in Healthy Volunteers

We are also conducting an auto-injector study to support having an auto-injector device for elegrobart available at the time of commercial launch, if approved.

In September 2025, we announced completion of enrollment for both REVEAL-1 and REVEAL-2 clinical studies, with each study exceeding its enrollment target due to strong patient demand.

We anticipate topline data for REVEAL-1 and REVEAL-2 in the first quarter and second quarter of 2026, respectively.

Phase 1 Trial of Elegrobart in Healthy Volunteers

• Study Design :

VRDN-003 was dosed in four single dose cohorts of healthy volunteers at a concentration of 150 mg/ml receiving 5 mg/kg IV (n=4), 300 mg SC (n=6), 15 mg/kg IV (n=4), 600 mg SC (n=6) and a fifth cohort of two doses of VRDN-003 (n=4).

Elegrobart was dosed in four single dose cohorts of healthy volunteers at a concentration of 150 mg/ml receiving 5 mg/kg IV (n=4), 300 mg SC (n=6), 15 mg/kg IV (n=4), 600 mg SC (n=6) and a fifth cohort of two doses of elegrobart (n=4).

• Summary of Results :

• Extended Half Life :

VRDN-003 pharmacokinetics data showed an extended half-life of 40 to 50 days, which is a 4-to-5-fold increase over the half-life of veligrotug (which showed a half-life of 10 to 12 days).

Elegrobart pharmacokinetics data showed an extended half-life of 40 to 50 days, which is a 4-to-5-fold increase over the half-life of veligrotug (which showed a half-life of 10 to 12 days).

• Prolonged Pharmacodynamics (“PD”) :

Following a single subcutaneous dose of VRDN-003, IGF-1 serum levels increased approximately 4-fold at peak.

Following a single subcutaneous dose of elegrobart, IGF-1 serum levels increased approximately 4-fold at peak.

This was consistent with the increases in IGF-1 levels that have been shown in the clinic following a single dose of veligrotug SC and IV.

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• Well-Tolerated :

VRDN-003 was well tolerated in all subjects with no SAEs.

Elegrobart was well tolerated in all subjects with no SAEs.

All treatment-related, treatment-emergent adverse events were grade 1 (mild).

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• Dosing Flexibility for Pivotal Development :

VRDN-003 modeling demonstrated dosing flexibility for the program’s anticipated global pivotal development.

The modeling showed that dosing VRDN-003 every eight weeks, every four weeks, and every two weeks achieved a range of exposures levels that were seen for veligrotug after intravenous doses of 3 mg/kg, 10 mg/kg and 20 mg/kg, respectively.

Elegrobart modeling demonstrated dosing flexibility for the program’s anticipated global pivotal development.

The modeling showed that dosing elegrobart every eight weeks, every four weeks, and every two weeks achieved a range of exposures levels that were seen for veligrotug after intravenous doses of 3 mg/kg, 10 mg/kg and 20 mg/kg, respectively.

FcRn Inhibitor Portfolio:

VRDN-006 and VRDN-008

In October 2023, consistent with our vision to develop the next generation of best-in-class products for severe autoimmune and rare diseases, we unveiled VRDN-006 and VRDN-008 from our portfolio of engineered FcRn inhibitors.

In October 2023, consistent with our vision to develop the next generation of best-in-class products for autoimmune and rare diseases, we unveiled VRDN-006 and VRDN-008 from our portfolio of engineered FcRn inhibitors.

FcRn inhibitors have the potential to treat a broad array of autoimmune diseases, representing a possible significant commercial market opportunity.

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The first FDA-approved FcRn inhibitor, Vyvgart® (efgartigimod), was developed by argenx SE (“Argenx”) and is approved for myasthenia gravis and chronic inflammatory demyelinating polyneuropathy.

Argenx reported efgartigimod net sales of approximately $2.2 billion in 2024, and it is projected to generate close to $6 billion in annual sales by 2030.

Argenx reported preliminary efgartigimod net sales of approximately $4.15 billion in 2025, and it is projected to generate more than $9 billion in annual sales by 2030.

Furthermore, FcRn inhibitors have the potential to address additional sizable autoimmune indications such as myositis, membranous nephropathy, Graves’ disease, lupus nephritis, and Sjogren’s syndrome.

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VRDN-006 also shows a similar safety profile to efgartigimod in our head-to-head non-human primate study showing that, comparable to efgartigimod, it did not lower albumin or increase LDL levels.

VRDN-006 received IND clearance from the FDA in January 2025 and we expect proof-of-concept IgG reduction data in healthy volunteers in the third quarter of 2025.

In September 2025, we announced that data from an ongoing phase 1 clinical trial in healthy volunteers showed that VRDN-006 led to IgG reductions that are consistent with the FcRn inhibitor class, that VRDN-006 was sparing of albumin and LDL, and was generally well-tolerated with no dose-limiting toxicities or serious adverse events.

We expect to communicate future development plans for VRDN-006 in 2026.

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VRDN-008 also shows a similar safety profile to efgartigimod in our head-to-head non-human primate study showing that, comparable to efgartigimod, it did not lower albumin or increase LDL levels.

NHP studies are ongoing to generate additional data for VRDN-008.

Once completed, we plan to use the totality of VRDN-008’s NHP data to build a robust pharmacokinetic and pharmacodynamic model to enable the prediction of potential human dosing regimens for VRDN-008.

An IND for VRDN-008 was submitted in December 2025 and received IND clearance from the FDA in January 2026.

We expect healthy volunteer data in the second half of 2026.

In January 2026, we announced that we are developing an anti-TSHR candidate with potential use in the treatment of Graves’ disease and TED.

This product candidate is a half‑life extended monoclonal antibody designed to inhibit activation of TSHR.

It is being developed for subcutaneous administration via an autoinjector, with the goal of enabling extended dosing intervals intended to support patient convenience.

We anticipate submitting an IND for this program in the fourth quarter of 2026.

We believe inhibiting TSHR has the potential to treat both TED and Graves’ disease.

TED pathophysiology potentially stems from the activation of the TSHR and IGF-1R signaling complex on orbital fibroblasts, leading to hyaluronan secretion and expansion of orbital fat and muscle.

Autoantibodies that stimulate TSHR can activate pathways that promote inflammation, fibroblast proliferation, and tissue remodeling relevant to TED.

We believe inhibiting TSHR could complement the inhibition of IGF-1R in the treatment of TED.

In addition to TED, blocking TSHR could also be effective to treat Graves’ Disease.

Graves’ disease is an autoimmune disease in which autoantibodies form against the TSHR, stimulating and activating the receptor.

These TSH receptor antibodies (“TRAb”) can drive a heightened activation of TSHR, resulting in excessive thyroid hormone production and hyperthyroidism.

Graves’ disease is one of the most prevalent autoimmune conditions, affecting more than 2 million people in the United States, and is the leading cause of hyperthyroidism.

Intellectual Property

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We possess know-how and trade secrets relating to the development and commercialization of our product candidates, including related manufacturing processes.

As of January 15, 2025, our in-licensed and owned patent portfolio consists of approximately one U.S.

issued patent, approximately seventeen U.S.

pending patent applications, no patents issued in jurisdictions outside of the United States, one allowed patent in China, and approximately seventy-eight patent applications pending in jurisdictions outside of the United States (including approximately seven pending Patent Cooperation Treaty (PCT) applications) that, in many cases, are counterparts to the foregoing U.S.

As of January 2026, our in-licensed and owned patent portfolio consists of approximately three U.S.

issued patents, approximately twenty-two U.S.

pending patent applications, one issued patent in China, and approximately one hundred nineteen patent applications pending in jurisdictions outside of the United States (including approximately twelve pending Patent Cooperation Treaty (PCT) applications) that, in many cases, are counterparts to the foregoing U.S.

patents and patent applications.

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We may also pursue patent protection with respect to manufacturing and drug development processes and technology.

When available to expand

market exclusivity, our strategy is to obtain or license additional intellectual property related to core elements of technology and/or product candidates.

When available to expand market exclusivity, our strategy is to obtain or license additional intellectual property related to core elements of technology and/or product candidates.

Individual patents extend for varying periods of time, depending upon the date of filing of the patent application, the date of patent issuance, and the legal term of patents in the countries in which they are obtained.

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The duration of patents outside of the United States varies in accordance with provisions of applicable local law, but typically is also 20 years from the earliest non-provisional filing date.

Our patent issued as of January 15, 2025 is expected to expire no earlier than 2041.

Ours patent issued as of January 2026 are expected to expire no earlier than 2041.

If patents are issued on our patent applications pending as of January 2026, the resulting patents are projected to expire on dates ranging from 2041 to 2047.

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We face potential competition from many different sources, including larger and better-funded biotechnology and pharmaceutical companies.

In many cases, the companies with competing programs will have access to greater resources and expertise than we do and may be more advanced in those programs.

In many cases, companies with competing programs will have access to greater resources and expertise than we do and may be more advanced in those programs.

Amgen’s Tepezza is the only FDA-approved medication for TED.

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• Amgen is developing an on-body subcutaneous formulation of Tepezza in a phase 3 study and has an early-stage candidate AMG-732 in TED.

• Argenx is developing efgartigimod (Vyvgart), an antibody fragment to target the FcRn that is subcutaneously delivered and expected to be evaluated in a registrational phase 3 trial in patients with TED.

• Immunovant, Inc.

is developing a subcutaneous formulation of batoclimab in phase 3 studies in patients with active TED.

• Roche is developing satralizumab (Enspryng), an anti-1L-6R that is subcutaneously delivered and is being evaluated in a phase 3 trial in patients with active TED.

• Roche is developing satralizumab (Enspryng), an anti-1L-6R that is subcutaneously delivered and was studied in two phase 3 trial in patients with TED, one of which (SatraGO-1) failed to meet its primary endpoint.

• Sling Therapeutics, Inc.

is developing linsitinib, a small molecule IGF-1R inhibitor currently being evaluated in an ongoing phase 2b/3 study in patients with active TED and currently has plans to initiate a phase 3 trial.

• Tourmaline Bio is developing TOUR006, a subcutaneously delivered anti-IL-6, in an ongoing phase 2b clinical trial in patients with active TED.

• Lassen Therapeutics is developing LASN01, an intravenously delivered anti-1L-11R currently being evaluated in a phase 2 study in active TED.

• ACELYRIN, INC.

is developing lonigutamab (VB-421), a subcutaneously delivered anti-IGF-1R currently being evaluated in a phase 1/2 study in patients with TED.

On January 6, 2025, ACELYRIN reported partial data from the phase 1/2 study and announced plans to initiate two phase 3 studies in patients with TED in the first quarter of 2025.

On February 6, 2025, ACELYRIN announced a decision to re-evaluate the phase 3 development program for lonigutumab.

Argenx’s Vyvgart and UCB’s Rystiggo® are the only FDA-approved anti-FcRn therapies, each approved in generalized myasthenia gravis.

• Novartis (formerly Tourmaline Bio) is developing TOUR006, a subcutaneously delivered anti-IL-6, in an ongoing phase 2b clinical trial in patients with active TED.

• Lassen Therapeutics is developing LASN01, an intravenously delivered anti-1L-11R, which has completed a phase 2 study in active TED.

• Alumis (ACELYRIN, INC.) is developing lonigutamab (VB-421), a subcutaneously delivered anti-IGF-1R in TED.

As of January 2026, further development plans for lonigutamab are under evaluation.

Argenx’s Vyvgart, UCB’s Rystiggo®, and Johnson and Johnson’s Imaavy are the only FDA-approved anti-FcRn therapies, each approved in generalized myasthenia gravis (“gMG”).

A non-exhaustive list of other companies that are advancing therapies in clinical development for the treatment to target FcRn include:

• Immunovant, Inc.

is developing batoclimab (IMVT-1401/HBM9161) and IMVT-1402 with intentions to target generalized myasthenia gravis, CIDP, Graves’ disease, and Rheumatoid arthritis (“RA”).

Both are monoclonal antibodies targeting FcRn.

Batoclimab is currently being evaluated in ongoing phase 3 studies in TED and phase 2 studies in CIDP and Graves’ disease.

IMVT-1402 is currently being evaluated in a phase 2 study in patients with Graves’ disease and RA.

• Johnson and Johnson is developing nipocalimab, which is in ongoing phase 3 trials for a number of indications including generalized myasthenia gravis, CIDP, Sjogren’s disease and warm autoimmune hemolytic anemia.

Johnson and Johnson submitted a BLA for nipocalimab in generalized myasthenia gravis in August 2024.

• UCB continues to develop Rystiggo in an ongoing phase 3 study in Myelin Oligodendrocyte Glycoprotein Antibody-associated Disease.

is developing batoclimab (IMVT-1401/HBM9161) and IMVT-1402 with evaluations in various indications.

Both are monoclonal antibody fragments targeting FcRn.

Batoclimab is currently being evaluated in two ongoing phase 3 studies in TED, two phase 3 studies in gMG, a phase 2 study in chronic inflammatory demyelinating polyneuropathy (“CIDP”), and a phase 2 study in Graves’ disease.

IMVT-1402 is currently being evaluated in a phase 3 study for gMG, two phase 2b studies in Graves’ disease, one phase 2b study in CIDP, one phase 2b study in anti-citrullinated protein antibody positive difficult-to-treat rheumatoid arthritis, and one phase 2b study in Sjögren’s disease.

• Johnson and Johnson is developing nipocalimab (Imaavy), a full-length monoclonal antibody against FcRn, which is in clinical development for a number of indications including CIDP, Sjogren’s disease, systemic lupus erythematosus, and warm autoimmune hemolytic anemia.

• UCB continues to develop Rystiggo, a monoclonal antibody fragment that binds FcRn, in an ongoing phase 3 study in Myelin Oligodendrocyte Glycoprotein Antibody-associated Disease.

There are currently no FDA-approved anti–thyroid‑stimulating hormone receptor (“anti-TSHR”) therapies.

A non-exhaustive list of other companies that are advancing therapies in clinical development for the treatment to target TSHR include:

• GenSci’s subcutaneously administered monoclonal antibody, GenSci098, is in an ongoing phase 1 study in patients with TED.

As of January 2026, recruitment ongoing in China only and ex-China rights were licensed to Yarrow Bioscience in December 2025.

• AV7 Limited’s intravenous and intramuscularly administered monoclonal antibody, K1-70, is in an ongoing phase 2 study in active TED.

As of January 2026, ongoing study in Japan only with no registration in the U.S.

License Agreements

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(“Private Viridian”) entered a license agreement with Zenas BioPharma to license technology comprising certain materials, patent rights, and know-how to Zenas BioPharma.

On October 27, 2020, in connection with the closing of the Private Viridian acquisition, we became party to the license agreement with Zenas BioPharma.

In October 2020, in connection with the closing of the Private Viridian acquisition, we became party to the license agreement with Zenas BioPharma.

Since February 2021, we have entered into several letter agreements with Zenas BioPharma in which we agreed to provide assistance to Zenas BioPharma with certain development activities, including manufacturing.

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License Agreement with ImmunoGen, Inc.

On October 12, 2020, Private Viridian entered into a license agreement with ImmunoGen (the “ImmunoGen License Agreement”), under which we obtained rights to an exclusive, sublicensable, worldwide license to certain patents and other intellectual property rights to develop, manufacture, and commercialize certain products for non-oncology and non-radiopharmaceutical indications.

In October 2020, Private Viridian entered into a license agreement with ImmunoGen (the “ImmunoGen License Agreement”), under which we obtained rights to an exclusive, sublicensable, worldwide license to certain patents and other intellectual

property rights to develop, manufacture, and commercialize certain products for non-oncology and non-radiopharmaceutical indications.

In consideration for rights granted by ImmunoGen, we are obligated to make certain development milestone payments of up to $48.0 million.

In December 2021, we paid a $2.5 million milestone payment to ImmunoGen upon the submission of an IND application for veligrotug with the FDA.

In May 2022, we paid a $3.0 million milestone payment to ImmunoGen related to the first patient dosed in the clinical trial for veligrotug.

In December 2022, we recorded $10.0 million as research and development expense related to a milestone owed to ImmunoGen related to the first

patient dosed in a pivotal clinical trial for veligrotug, amount which was paid in January 2023, and which was included in accounts payable in the consolidated balance sheet as of December 31, 2022.

Additionally, if we successfully commercialize any product candidate subject to the ImmunoGen License Agreement, we are responsible for royalty payments equal to a percentage in the mid-single digits of net sales and commercial milestone payments of up to $95.0 million.

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If we fail to exercise an Option prior to expiration of the applicable Option Period, such Option for such Programs will terminate.

In consideration for Paragon’s grant of the Options to us, we paid to Paragon a non-refundable, non-creditable one-time fee of $2.5 million, which was recorded as research and development expense during the three months ended March 31, 2022.

In December 2022, we and Paragon entered into a first amendment to the Paragon Research Agreement, under which we obtained an additional limited license for the purpose of conducting certain activities.

In consideration for the rights and licenses obtained under the first amendment, we paid Paragon a non-refundable fee of $2.3 million (the “First Amendment Payment”), which was recorded as research and development expense during the three months ended December 31, 2022.

The non-refundable upfront fee and the First Amendment Payment are separate from any development costs or cost advance paid or owing with respect to the specified program.

In October 2023, we entered into a License Agreement with Paragon (the “Paragon License Agreement”) as a result of exercising our Option under the Paragon Research Agreement to obtain exclusive licenses to develop, manufacture and commercialize certain therapeutic proteins and antibodies and associated products.

In connection with the execution of the Paragon License Agreement, we made an initial payment of $5.3 million, which was recorded as research and development expense during the three months ended December 31, 2023.

As further described below, the Paragon License Agreement was amended and restated by the Amended and Restated License Agreement with Paragon in September 2024.

In January 2024, we entered into a letter agreement with Paragon pursuant to which Paragon agreed to continue to perform development activities under the existing Paragon Research Agreement and Paragon License Agreement, which we renewed in July 2024.

In consideration for the development activities to be conducted by Paragon, we agreed to reimburse Paragon for actual development costs incurred and agreed upon development fees in exchange for Paragon’s commitment of the necessary personnel and resources to perform these activities.

In September 2024, we entered into a second amendment to the Paragon Research Agreement to include additional development activities to be performed by Paragon.

Under the amended Paragon Research Agreement, we will be obligated to make a one-time non-refundable payment of $3.5 million to Paragon following the achievement of certain research and development objectives.

We achieved such objectives in February 2025 and the $3.5 million payment was recorded as research and development expense during the three months ended March 31, 2025.

In September 2024, we entered into the Amended and Restated License Agreement with Paragon (the “Amended Paragon License Agreement”) which amended and restated the Paragon License Agreement.

In connection with the execution of the

Amended Paragon License Agreement, we paid to Paragon a non-refundable fee of $4.0 million in September 2024, which was recorded as research and development expense during the three months ended September 30, 2024.

In connection with the execution of the Amended Paragon License Agreement, we paid to Paragon a non-refundable fee of $4.0 million in September 2024, which was recorded as research and development expense during the three months ended September 30, 2024.

In consideration for rights granted by Paragon, we are obligated to make certain future milestone payments of up to $16.0 million on a program-by-program basis upon the achievement of specified clinical or regulatory milestones, with total milestone payments under all programs not to exceed $40.0 million.

Additionally, if the Company develops a product utilizing certain intellectual property rights granted to it under the Amended Paragon License Agreement, the Company is obligated to pay Paragon potential additional future development milestone payments of up to $3.1 million and commercial milestone payments of up to $17.0 million with respect to such product.

If the Company successfully commercializes any product candidate subject to the Amended Paragon License Agreement, it is responsible for royalty payments equal to a percentage in the mid-single digits of such product’s net sales.

Additionally, if we develop a product utilizing certain intellectual property rights granted to it under the Amended Paragon License Agreement, we are obligated to pay Paragon potential additional future development milestone payments of up to $3.1 million and commercial milestone payments of up to $17.0 million with respect to such product.

If we successfully commercialize any product candidate subject to the Amended Paragon License Agreement, it is responsible for royalty payments equal to a percentage in the mid-single digits of such product’s net sales.

During the year ended December 31, 2025, we recorded $4.5 million in research and development costs related to the Paragon Research Agreement and Amended Paragon License Agreement (collectively the “Paragon Agreements”).

Collaboration and License Agreement with Kissei Pharmaceutical Co., Ltd.

In July 2025, we entered into a Collaboration and License Agreement with Kissei (the “Kissei Agreement”), pursuant to which we granted to Kissei an exclusive license to develop and commercialize products containing veligrotug and elegrobart for potential treatments, including treatment of TED, in Japan, and a non-exclusive license to manufacture such licensed products worldwide for use in Japan under certain limited circumstances.

The transaction price under the Kissei Agreement included a one-time, non-refundable and non-creditable upfront cash payment to us of $70.0 million.

Additionally, we are eligible to receive up to an additional $315.0 million of non-refundable milestone payments upon achieving specific milestone events during the contract term, as well as tiered royalty payments ranging from percentages in the twenties to the mid-thirties based on the annual net sales of any licensed products sold in Japan.

Kissei is obligated to make royalty payments to us for the royalty term as defined in the Kissei Agreement.

Kissei will be responsible for developing and seeking regulatory approval of the licensed products in Japan, subject to oversight from a joint steering committee.

Following regulatory approval, Kissei will be responsible for commercializing the licensed products in Japan.

Except in certain limited circumstances, we will be responsible for manufacturing and supplying the licensed products for Kissei’s developmental and commercial use in Japan.

The term of the Kissei Agreement will expire in its entirety upon the expiration of the royalty term for all licensed products and satisfaction of certain payment obligations as set forth in the Kissei Agreement, unless earlier terminated by the parties in accordance with the terms of the Collaboration Agreement.

Purchase and Sale Agreement

Purchase and Sale Agreement with DRI Healthcare Acquisitions LP

In October 2025, we entered into a Purchase and Sale Agreement of revenue participation right (the “DRI Purchase and Sale Agreement”) with DRI Healthcare Acquisitions LP (“DRI”), pursuant to which DRI purchased rights to certain revenue streams in the U.S.

from us in exchange for up to $300.0 million in consideration, including $55.0 million paid at signing and conditional payments consisting of:

(i) $25.0 million that is payable following the achievement of certain milestones with respect to our elegrobart pivotal phase 3 clinical trials, REVEAL-1 and REVEAL-2, on or before a specified date;

(ii) $75.0 million that is payable following receipt of marketing approval for veligrotug from the FDA on or before a specified date;

(iii) $15.0 million that is payable if the events set forth in the foregoing clauses (1) and (2) are met;

(iv) $50.0 million that is payable following receipt of marketing approval for elegrobart from the FDA on or before a specified date;

(v) at our election, $50.0 million that is payable following our achievement of net sales of certain products equal to or exceeding $1.1 billion on or before a specified date;

and (vi) an additional $30.0 million that may be payable to us at a time and pursuant to financial terms agreed upon by us and DRI at such time.

None of the milestones relates to the conditional payments have been achieved to date.

The DRI Purchase and Sale Agreement contains customary representations, warranties and indemnities of the Company and DRI and customary covenants on the part of the Company, as well as a limit on the amount of incurrence of certain types of indebtedness, which limit automatically terminates a certain period of time following receipt of marketing approval for veligrotug in the U.S.

The DRI Purchase and Sale Agreement requires us to pay tiered royalties to DRI based on net sales of veligrotug, elegrobart and certain other related products (the “Net Sales Royalties”).

The royalties consist of (i) 7.5% of annual U.S.

net sales up to and including $600 million, which royalties could increase to low-double digits if marketing approval for elegrobart is not received prior to a speciﬁed date, (ii) 0.8% of annual U.S.

net sales above $600 million and up to and including $900 million, (iii) 0.25% of annual U.S.

net sales above $900 million and up to $2 billion, and (iv) no royalty owed for annual U.S.

net sales in excess of $2 billion.

The DRI Purchase and Sale Agreement may only be terminated upon repayment by the Company of a certain multiplier of the consideration paid to us by DRI (less payments by the Company to DRI to date) on or prior to a certain date or repayment by an acquirer of the Company of a certain multiplier of the consideration paid by DRI to the Company (less payments by the Company to DRI to date) following a change of control of the Company.

Government Regulation

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The process of developing a biologic and obtaining regulatory approvals and compliance with federal, state, and local statutes and regulations, both pre- and post-approval, requires the expenditure of substantial time and financial resources.

Failure to comply with the applicable U.S.

Failure to comply with the

applicable U.S.

requirements at any time during the product development process, approval process or following approval may subject an applicant to delays in development or approval, administrative action and judicial sanctions .

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The IND automatically becomes effective 30 days after receipt by the FDA, unless the FDA, within the 30-day period, raises safety concerns or questions about the proposed clinical trial.

In such a case, the IND may be placed on clinical hold and the IND sponsor and the FDA must resolve any outstanding concerns or questions before the clinical trial can begin.

In such a case, the IND may be placed on clinical hold and the IND sponsor and the

FDA must resolve any outstanding concerns or questions before the clinical trial can begin.

Submission of an IND therefore may or may not result in FDA authorization to begin a clinical trial.

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Imposition of a clinical hold could cause significant delays or difficulties in completing planned clinical studies in a timely manner.

Some studies also include oversight by an independent group of qualified experts organized by the clinical study sponsor, known as a data safety monitoring board, which provides authorization for whether or not a study may move forward at designated check points based on access to certain data from the study and may halt the clinical trial if it determines that there is an unacceptable safety risk for subjects or other grounds, such as no demonstration of efficacy.

Some studies also include oversight by an independent group of qualified experts organized by the clinical study sponsor, known as a data safety monitoring board or a data monitoring committee, which provides authorization for whether or not a study may move forward at designated check points based on access to certain data from the study and may halt the clinical trial if it determines that there is an unacceptable safety risk for subjects or other grounds, such as no demonstration of efficacy.

For purposes of BLA approval, human clinical trials are typically conducted in three sequential phases that may overlap or be combined.

The investigational product is initially introduced into a limited population of healthy human subjects or, in some circumstances, patients with the target disease or condition.

These studies are designed to test the safety, dosage

tolerance, absorption, metabolism and distribution of the investigational product in humans, and the side effects associated with increasing doses.

These studies are designed to test the safety, dosage tolerance, absorption, metabolism and distribution of the investigational product in humans, and the side effects associated with increasing doses.

The investigational product is administered to a limited patient population with a specified disease or condition to evaluate the preliminary efficacy, optimal dosages and dosing schedule and to identify possible adverse side effects and safety risks.

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When the foreign clinical study is not conducted under an IND, the sponsor must ensure that the study complies with certain FDA regulatory requirements in order to use the study as support for an IND or application for marketing approval or licensure in the United States.

Specifically, the FDA requires that the study be conducted in accordance with GCP requirements intended to ensure the protection of human subjects and the quality and integrity of the study data, including review and approval by an independent ethics committee and use of proper procedures for obtaining informed consent from subjects, and that the FDA is able to validate the data from the study through an onsite inspection if the FDA deems such inspection necessary.

In December 2022, with the passage of the Food and Drug Omnibus Reform Act amendments to the FDCA, Congress added a requirement for sponsors to develop and submit a diversity action plan for each phase 3 clinical trial or any other “pivotal study” of a new drug or biological product.

These plans are meant to encourage the enrollment of more diverse patient populations in late-stage clinical trials of FDA-regulated products.

Specifically, diversity action plans must include the sponsor’s goals for enrollment, the underlying rationale for those goals, and an explanation of how the sponsor intends to meet them.

This requirement will apply with respect to clinical investigations for which enrollment commences 180 days after the publication of a final guidance by the FDA on diversity action plans.

FDA issued draft guidance addressing diversity action plans in June 2024, and the statute directs FDA to issue final guidance within nine months of closing the comment period on this draft guidance, which is expected to be in June 2025.

proper procedures for obtaining informed consent from subjects, and that the FDA is able to validate the data from the study through an onsite inspection if the FDA deems such inspection necessary.

Information about certain clinical trials must be submitted within specific timeframes to the NIH for public dissemination on its ClinicalTrials.gov website.

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In addition, under the Pediatric Research Equity Act (“PREA”), a BLA or certain supplements to a BLA must contain data that are adequate to assess the safety and effectiveness of the biological product candidate for the claimed indications in all relevant pediatric subpopulations and to support dosing and administration for each pediatric subpopulation for which the product is safe and effective, although deferrals or full or partial waivers may be available in some circumstances.

A sponsor who is planning

to submit a marketing application for a biological product that includes a new active ingredient, new indication, new dosage form, new dosing regimen or new route of administration must submit an initial pediatric study plan, within sixty days after an end-of-phase 2 meeting or as may be agreed between the sponsor and FDA.

A sponsor who is planning to submit a marketing application for a biological product that includes a new active ingredient, new indication, new dosage form, new dosing regimen or new route of administration must submit an initial pediatric study plan, within sixty days after an end-of-phase 2 meeting or as may be agreed between the sponsor and FDA.

The FDA must then review the information submitted, consult with the sponsor, and agree upon a final plan.

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Unless otherwise required by regulation, PREA does not apply to any biological product for an indication for which orphan designation has been granted.

Within 60 days following submission of the application, the FDA reviews a BLA submitted to determine if it is substantially complete before the agency accepts it for filing.

The cost of preparing and submitting a BLA is substantial.

Under the PDUFA, as amended, each BLA must be accompanied by an application fee.

For fiscal year 2026, the application fee for each BLA requiring clinical data is approximately $4.7 million.

The PDUFA also imposes an annual program fee for each approved prescription drug product, which has been set at approximately $442,000 for fiscal year 2026.

The FDA adjusts the PDUFA user fees on an annual basis.

Fee waivers, reductions and exceptions are available in certain circumstances.

Additionally, no application fees are assessed on BLAs for products designated as orphan drugs, unless the BLA also includes a non-orphan indication.

Within 60 days following submission of the application, the FDA reviews a BLA to determine if it is substantially complete before the agency accepts it for filing.

The FDA may refuse to file any BLA that it deems incomplete or not properly reviewable at the time of submission and may request additional information.

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Under the PHSA, the FDA may approve a BLA if it determines that the product is safe, pure, and potent and that the facility where the product will be manufactured meets standards designed to ensure that it continues to be safe, pure, and potent.

On the basis of the FDA’s evaluation of the application and accompanying information, including the results of the inspection of the manufacturing facilities and any FDA inspections of nonclinical and clinical trial sites to assure compliance with GLP or GCP, and the applicant to ensure compliance with GCP, the FDA may issue an approval letter or a complete response letter (“CRL”).To reach this determination, the FDA will evaluate whether the proposed new biologic’s benefits outweigh its potential risks to patients.

basis of the FDA’s evaluation of the application and accompanying information, including the results of the inspection of the manufacturing facilities and any FDA inspections of nonclinical and clinical trial sites to assure compliance with GLP or GCP, and the applicant to ensure compliance with GCP, the FDA may issue an approval letter or a complete response letter (“CRL”).To reach this determination, the FDA will evaluate whether the proposed new biologic’s benefits outweigh its potential risks to patients.

An approval letter authorizes commercial marketing of the product with specific prescribing information for specific indications.

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The FDA also may condition approval on, among other things, changes to proposed labeling, including a more limited indication or addition of warnings, precautions or contraindications, or implementation of testing and surveillance programs to monitor the product after commercialization.

The FDA may require one or more phase 4 post-market studies and surveillance to further assess and monitor the product’s safety and effectiveness after commercialization and may limit further marketing of the product based on the results of these post-marketing studies Once approved, the FDA may withdraw the product approval if

compliance with pre- and post-marketing requirements is not maintained or if problems occur after the product reaches the marketplace.

Orphan Drug Designation

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As a result, the scope of exclusivity has been narrow and protected only against competition from the same “use or indication” rather than the broader “disease or condition.” In the September 2021 case Catalyst Pharmaceuticals, Inc.

Becerra , a federal circuit court set aside the FDA’s narrow interpretation and ruled that orphan drug exclusivity covers the full scope of the orphan-designated disease or condition regardless of whether the drug obtains approval only for a narrower use.

Although the FDA announced in January 2023 that it will not apply the Catalyst decision beyond the facts at issue in that case, Catalyst could serve as a precedent for future challenges to FDA’s orphan drug-related decisions.

Becerra , a federal circuit court in the Eleventh Circuit set aside the FDA’s narrow interpretation and ruled that orphan drug exclusivity covers the full scope of the orphan-designated disease or condition regardless of whether the drug obtains approval only for a narrower use.

the FDA announced in January 2023 that it will not apply the Catalyst decision beyond the facts at issue in that case, in 2025 a federal district court in Neurelis, Inc.

Brenner struck down another FDA approval, adopting the same interpretation of orphan drug exclusivity as the Eleventh Circuit.

The FDA has appealed this decision to the U.S.

Court of Appeals for the D.C.

Legislation has been introduced, but has not been passed, that would codify the scope of orphan drug exclusivity set forth in the FDA’s regulations, rather than the interpretation adopted by the Eleventh Circuit in Catalyst .

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Even if designation is granted, FDA may later decide that a product candidate no longer meets the conditions for designation and the designation may be rescinded.

Separate from FDA’s priority review program, in 2025 the FDA created a new Commissioner’s National Priority Voucher (“CNPV”) pilot program.

A CNPV may be granted to products with significant potential to address certain national health priorities, which include:

(i) addressing a large unmet medical need, (ii) delivering innovative cures, (iii) onshoring drug development and manufacturing, (iv) increasing affordability, or (v) addressing a U.S.

public health crisis.

The FDA has said it will review marketing applications for drugs with a CNPV within approximately one to two months following filing of a complete application, though the agency retains full discretion to extend the review time if the data or application components submitted are insufficient or incomplete, if the results of pivotal trial(s) are ambiguous, or if the review is particularly complex.

In addition, applications filed with a CNPV will be evaluated by a multi-disciplinary review committee led by the FDA's Office of the Chief Medical and Scientific Officer, and the FDA will also provide enhanced communication with companies throughout the development and review process.

Additionally, the FDA may grant accelerated approval to a product for a serious or life-threatening condition that provides meaningful therapeutic advantage to patients over existing treatments based upon a determination that the product has an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit.

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As a condition of accelerated approval, the FDA will generally require the sponsor to perform adequate and well-controlled post-marketing clinical studies to verify and describe the anticipated effect on irreversible morbidity or mortality or other clinical benefit.

Under the Food and Drug Omnibus Reform Act of 2022, the FDA may require, as appropriate, that such studies be underway prior to approval or within a specific time period after the date of approval for a product granted accelerated approval.

accelerated approval.

Failure to conduct required post-approval studies with due diligence, failure to confirm a clinical benefit during the post-approval studies, or dissemination of false or misleading promotional materials would allow the FDA to withdraw the product approval on an expedited basis.

All promotional materials for therapeutic candidates approved under accelerated approval are subject to prior review by the FDA unless FDA informs the BLA holder otherwise.

The Consolidated Appropriations Act, 2023 strengthens the FDA’s authority to require and regulate post-approval studies of accelerated approval drugs and to expedite the rescission of accelerated approval based on these post-approval studies.

Regulation of Combination Products

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In reviewing the NDA or BLA for such a product, however, FDA reviewers would consult with their counterparts in the FDA’s Center for Devices and Radiological Health to ensure that the device component of the combination product met applicable requirements regarding safety, effectiveness, durability and performance.

In addition, under FDA regulations, combination products are subject to cGMP requirements applicable to both drugs and devices, including the Quality System Regulation applicable to medical devices.

In addition, under FDA regulations, combination products are subject to cGMP requirements applicable to both the drug or biologic constituent part and the device constituent part.

Post-Approval Requirements

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Biologic manufacturers and their subcontractors are required to register their establishments with the FDA and certain state agencies.

BLA holders and their contractors, including third-party manufacturers, are subject to periodic unannounced inspections by the FDA and certain state agencies for compliance with

ongoing regulatory requirements, including cGMPs and pharmacovigilance regulations.

BLA holders and their contractors, including third-party manufacturers, are subject to periodic unannounced inspections by the FDA and certain state agencies for compliance with ongoing regulatory requirements, including cGMPs and pharmacovigilance regulations.

Changes to the manufacturing process are strictly regulated, and, depending on the significance of the change, may require prior FDA approval before being implemented.

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The FDA approved the first interchangeable biosimilars in 2021.

However, in 2024, the agency updated its policies regarding interchangeable biosimilars to require fewer tests by the applicant to demonstrate interchangeability and to highlight that these products are not safer or more effective than biosimilars that have not been demonstrated “interchangeable” with their reference products.

However, in draft guidance issued in 2024, the agency updated its policies regarding interchangeable biosimilars to recommend fewer tests by the applicant to demonstrate interchangeability and to highlight that these products are not safer or more effective than biosimilars that have not been demonstrated “interchangeable” with their reference products.

In response to such scientific developments, it is possible that Congress could revisit and amend relevant provisions from the BPCIA as part of the upcoming legislative session.

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However, patent term restoration cannot extend the remaining term of a patent beyond a total of 14 years from the product’s approval date.

The patent term restoration period is generally one-half the time between the effective date of an IND and the submission date of an NDA or BLA, plus the time between the submission date and the approval of that application.

Only one patent applicable to an approved product is eligible for the extension and the application for the extension must be submitted prior to the expiration of the patent.

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The collection and use of personal health data and other personal data in the European Union (“EU”) is governed by the provisions of the European General Data Protection Regulation 2016/679 (“GDPR”), which became applicable in May 2018, and related data protection laws in individual EU Member States.

The GDPR imposes a number of strict obligations and restrictions on the ability to process, including collecting, analyzing and transferring, personal data of individuals, in particular with respect to health data from clinical trials and adverse event reporting.

The GDPR imposes a number of strict obligations and restrictions on the ability to process, including collecting, analyzing and transferring, personal data of individuals, in particular

with respect to health data from clinical trials and adverse event reporting.

The GDPR includes requirements relating to the legal basis of the processing (such as consent of the individuals to whom the personal data relates), the information provided to the individuals prior to processing their personal data, the notification obligations to the national data protection authorities, and the security and confidentiality of the personal data.

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Failure to comply with these obligations could lead to government enforcement actions and significant penalties against us, harm to our reputation, and adversely impact our business and operating results.

The uncertainty regarding the

interplay between different regulatory frameworks, such as the CTR and the GDPR, further adds to the complexity that we face with regard to data protection regulation.

The uncertainty regarding the interplay between different regulatory frameworks, such as the CTR and the GDPR, further adds to the complexity that we face with regard to data protection regulation.

With regard to the transfer of personal data from the EEA to the United Kingdom (“UK”), personal data may now freely flow from the EEA to the UK since the UK is deemed to have an adequate data protection level.

However, the adequacy decisions include a ‘sunset clause’ which entails that the decisions will automatically expire four years after their entry into force, and so this adequacy decision will be reviewed, and is expected – but not guaranteed - to be renewed, before or during June 2025.

Drug and Biologic Development Process in the EU

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Regardless of where they are conducted, all clinical trials included in applications for marketing authorization for human medicines in the EU / EEA must have been carried out in accordance with EU regulations.

This means that clinical trials conducted in the EU / EEA have to comply with EU clinical trial legislation but also that clinical trials conducted outside the

EU / EEA have to comply with the principles equivalent to those set out in the EEA, including adhering to GCPs and the ethical principles set out in the Declaration of Helsinki.

This means that clinical trials conducted in the EU / EEA have to comply with EU clinical trial legislation but also that clinical trials conducted outside the EU / EEA have to comply with the principles equivalent to those set out in the EEA, including adhering to GCPs and the ethical principles set out in the Declaration of Helsinki.

In accordance with the CTR, sponsors must submit a single CTA application through a centralized EU clinical trials portal, the Clinical Trials Information System (“CTIS”).

The CTA application will generally include the results of animal and in vitro studies assessing the toxicology, pharmacokinetics, pharmacology and pharmacodynamic characteristics of the product, chemistry, manufacturing and controls information, and any available human data or literature to support the use of the investigational product.

The sponsor must propose one NCA to act as reporting member state (“RMS”) to lead the validation and evaluation of the application.

This RMS will be responsible for consulting and coordinating with the NCAs of the other EU Member States, i.e., Concerned Member States (“CMS”).

One NCA acts as reporting member state (“RMS”) to lead the validation and evaluation of the application.

This RMS will be responsible for consulting and coordinating with the NCAs of the other EU Member States, i.e., Concerned Member States.

If an application is rejected, it may be amended and resubmitted through the CTIS.

If an approval is issued, the sponsor may start the clinical trial in all CMSs.

However, a CMS may in limited circumstances declare an “opt-out” from an approval and prevent the clinical trial from being conducted in such Member State.

Consistent with the position in the U.S., NCAs, RECs or the sponsor may suspend a clinical trial at any time on various grounds, including a finding that the subjects are being exposed to an unacceptable health risk or that the trial is unlikely to meet its stated objectives.

If an approval is issued, the sponsor may start the clinical trial in all Concerned Member States.

However, a Concerned Member State may in limited circumstances declare an “opt-out” from an approval and prevent the clinical trial from being conducted in such Member State.

During the development of a medicinal product, the EMA and NCAs provide the opportunity for dialogue and guidance on the development program.

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Advice is not legally binding with regard to any future MAA of the product concerned.

However, it is expected that the scientific

advice will be followed in the research and development program for the purpose of seeking product approval, unless any deviation from such advice is appropriately justified.

Drug Marketing Authorization

In the EU, medicinal products are subject to extensive pre- and post-market regulation by regulatory authorities at both the EU and national levels.

In the EU and EEA, after completion of all required testing, nonclinical studies, clinical trials and chemistry, manufacturing, and controls information can be included in a MAA requesting approval to market the product for one or more indications.

In the EU and EEA, after completion of all required testing, nonclinical studies, clinical trials and chemistry, manufacturing, and controls information can be included in an MAA requesting approval to market the product for one or more indications.

Pharmaceutical products may only be placed on the market after a marketing authorization has been obtained.

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centralized and national.

In December 2025, the EU Parliament and European Council agreed on major reforms to modernize EU pharmaceutical legislation, aiming to re-balance the promotion of innovation with improved patient access to safe, effective, and affordable medicines.

Key measures include a new exclusivity framework, enhanced incentives for orphan drugs and antibiotics, an expanded Bolar exemption, and a shortened regulatory assessment timeframe.

The reforms also introduce stricter controls on product availability and supply shortages.

It is expected that new EU pharmaceutical legislation will be fully applicable in 2028 following a two-year transition period.

Centralized Marketing Authorizations

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Upon request, the CHMP can reduce the time frame to 150 days if the applicant provides sufficient justification for an accelerated assessment.

The CHMP will provide a positive opinion regarding the application only if it meets certain quality, safety and efficacy

requirements.

The CHMP will provide a positive opinion regarding the application only if it meets certain quality, safety and efficacy requirements.

This opinion is then transmitted to the EC, which has the ultimate authority for granting marketing authorization within 67 days after receipt of the CHMP opinion.

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The decentralized procedure permits companies to file identical MAAs for a medicinal product to the NCAs of various EU Member States simultaneously.

The NCA of a single EU Member State, the reference member state, is appointed to review the application and provide an assessment report.

The NCA of a single EU Member State, the reference member state, is appointed to lead the review of the application and provide an assessment report.

The NCAs of the other Member States, the concerned member states, are subsequently required to grant a marketing authorization for their territories on the basis of this assessment.

The only exception to this is where the competent authority of a CMS considers that there are concerns of potential serious risk to public health, the disputed points are subject to a dispute resolution mechanism and may eventually be referred to the EC, whose decision is binding for all EU Member States.

The only exception to this is where the competent authority of a Concerned Member State considers that there are concerns of potential

serious risk to public health, the disputed points are subject to a dispute resolution mechanism and may eventually be referred to the EC, whose decision is binding for all EU Member States.

Risk Management Plan

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Even if a compound is considered to be a NAS and the marketing authorization applicant is able to gain the prescribed period of data exclusivity, another company could market a version of the medicinal product if such company can compile a full MAA based on its own complete set of chemistry, manufacturing, and controls information, nonclinical studies and clinical trials and obtain marketing authorization of its product.

Under the proposed reforms to EU pharmaceutical law, agreed in December 2025 by the European Parliament and the Council of the European Union (comprising representatives of all EU member state governments), a new framework for regulatory data and marketing exclusivity has been introduced.

The revised system provides for eight years of data exclusivity and one year of marketing exclusivity, with the possibility of extending total exclusivity to up to eleven years.

Extensions may be granted for medicines that address unmet medical needs, achieve commercial launch in all EU member states, or receive approval for a new clinically significant therapeutic indication.

Conditional Approval

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However, for such an approval to be granted a number of criteria should be fulfilled:

benefit/risk balance of the product is positive, (ii) it is likely that the applicant will be in a position to provide the comprehensive clinical data, (iii) unmet medical needs will be fulfilled by the grant of the marketing authorization and (iv) the benefit to public health of the immediate availability on the market of the medicinal product concerned outweighs the risk inherent in the fact that additional data are still required.

(i) the benefit/risk balance of the product is positive, (ii) it is likely that the applicant will be in a position to provide the comprehensive clinical data, (iii) unmet medical needs will be fulfilled by the grant of the marketing authorization and (iv) the benefit to public health of the immediate availability on the market of the medicinal product concerned outweighs the risk inherent in the fact that additional data are still required.

A conditional marketing authorization must be renewed annually.

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The EMA’s Committee for Orphan Medicinal Products (“COMP”) assesses orphan drug designation if the medicinal product is:

(1) intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union when the application is made, or without the incentives derived from orphan status, it is unlikely the medicinal product would generate sufficient return to justify the investment;

(1) intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition

affecting no more than five in 10,000 persons in the European Union when the application is made, or without the incentives derived from orphan status, it is unlikely the medicinal product would generate sufficient return to justify the investment;

and (2) there is no other satisfactory method approved in the EU of diagnosing, preventing, or treating the condition, or if such a method exists, the proposed medicinal product is a significant benefit to patients affected by the condition.

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Additionally, a marketing authorization may be granted to a similar medicinal product (orphan or not) for the same or overlapping indication subject to certain requirements.

Under the proposed reforms to EU pharmaceutical law referenced above, orphan medicinal products will benefit from a single period of nine years of market exclusivity.

This period may be extended to eleven years for orphan medicinal products intended for therapeutic areas where no treatment options currently exist.

The reforms also remove the previous system of staggered market exclusivity periods for orphan products granted for multiple indications for the same product.

PRIME Designation

In March 2016, the EMA launched an initiative to facilitate development of product candidates in indications, often rare, for which few or no satisfactory therapies currently exist in the EU.

The EMA has established an initiative to facilitate development of product candidates in indications, often rare, for which few or no satisfactory therapies currently exist in the EU.

The Priority Medicines (“PRIME”) scheme is intended to encourage drug development in areas of unmet medical need and provides accelerated assessment of products representing substantial innovation for them to be reviewed under the centralized procedure.

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Importantly, once a candidate medicine has been selected for the PRIME scheme, a dedicated contact point and rapporteur from the CHMP or from the Committee for Advanced Therapies (“CAT”) are appointed facilitating increased understanding of the product at EMA’s Committee level.

A kick-off meeting with the CHMP/CAT rapporteur initiates these relationships and includes a team of multidisciplinary experts to provide guidance on the overall development plan and

regulatory strategy.

A kick-off meeting with the CHMP/CAT rapporteur initiates these relationships and includes a team of multidisciplinary experts to provide guidance on the overall development plan and regulatory strategy.

PRIME eligibility does not change the standards for product approval, and there is no assurance that any such designation or eligibility will result in expedited review or approval.

Regulation of Combination Products in the EU

Unlike in the U.S., there is no single definition for a combination product.

In the EU, product containing a medical device and a medicinal product are either regulated as a medicinal product or a medical device and the primary mode of action governs the regulatory pathway.

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Similar to the position in the U.S., if a marketing authorization holder does not maintain compliance with applicable regulatory requirements, or if unfavorable signals derived from post-approval use of the medicinal product are identified, the relevant regulatory authority can impose various sanctions/remedial actions, including but not limited to:

the suspension or revocation of the underlying approvals;

the variation to, suspension or revocation of the underlying approvals;

the imposition of market recalls;

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the issuance of safety alerts including Dear Healthcare Professional letters;

and the imposition of criminal penalties.

and the imposition of criminal or financial penalties.

Regulation in the UK

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Until recently, certain aspects of EU pharmaceutical legislation applied in Northern Ireland by virtue of the Northern Ireland Protocol.

However, in accordance with the Windsor Framework, as of January 1, 2025, EU pharmaceutical law no longer applies in Northern Ireland.

However, in accordance with the Windsor Framework, as of January 1, 2025, the unified UK-wide licensing system applies in Northern Ireland.

The Windsor Framework sets out a long-term set of arrangements for the supply of medicines into Northern Ireland.

In particular, medicines need to be approved and licensed on a UK-wide basis by the UK’s Medicines and Healthcare products Regulatory Agency (the “MHRA”), with medicines using the same packaging and labeling across the UK.

The EMA has no role in approving or licensing new drugs for provision in Northern Ireland.

The EMA no longer has a role in approving or licensing new drugs for provision in Northern Ireland.

The European Union and the UK have agreed on a trade and cooperation agreement (“TCA”), which includes provisions affecting the life sciences sector (including on customs and tariffs).

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The MHRA has since been consulting on future regulations for medicines and medical devices in the UK.

delegated regulations under the UK's Medicines and Medical Devices Act 2021 have been adopted or are planned, significantly updating the medical device framework with stricter rules for Post-Market Surveillance, new pathways for innovative devices, changes to IVD rules, and upcoming core legislation expected in 2026, all aiming for alignment with international standards while maintaining UK competence to regulate such products.

The collection and use of personal health data and other personal data in the UK is governed by the provisions of the UK GDPR (as defined by section 3(10) (as supplemented by section 205(4)) of the Data Protection Act 2018 (the “DPA 2018”)), the DPA 2018, and related data protection laws in the UK.

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Other Regulations

Pharmaceutical companies are subject to additional healthcare regulation and enforcement by the federal government and by authorities in the states and foreign jurisdictions in which they conduct their business.

Pharmaceutical companies are subject to extensive healthcare regulation and enforcement by the federal government and by authorities in the states and foreign jurisdictions in which they conduct their business.

Such laws include, without limitation:

the federal Anti-Kickback Statute (“AKS”);

the federal False Claims Act (“FCA”);

the Health Insurance Portability and Accountability Act of 1996 (“HIPAA”) and similar foreign, federal and state fraud, abuse and transparency laws.

fraud and abuse laws such as the federal Anti-Kickback Statute (“AKS”) and the federal False Claims Act (“FCA”) in the U.S.

government pricing and price reporting laws, consumer protection laws and state licensure laws.

Some of these laws apply only when the manufacturer has a marketed product.

Fraud and abuse laws include a number of anti-kickback laws.

The AKS prohibits, among other things, persons and entities from knowingly and willfully soliciting, receiving, offering or paying remuneration, to induce, or in return for, either the referral of an individual, or the purchase or recommendation of an item or service for which payment may be made under any federal healthcare program.

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The government often takes the position that to violate the AKS, only one purpose of the remuneration need be to induce referrals, even if there are other legitimate purposes for the remuneration.

There are a number of statutory exceptions and regulatory safe harbors protecting some common activities from AKS prosecution, but they are drawn narrowly and practices that involve remuneration, such as consulting agreements, that may be alleged to be intended to induce prescribing, purchasing or recommending may be subject to scrutiny if they do not qualify for an exception or safe harbor.

Our practices may not in all cases meet all of the criteria for protection under a statutory exception or regulatory safe harbor.

There are a number of statutory exceptions and regulatory safe harbors protecting some common activities from prosecution, but protection is available only if all requirements are met.

Our practices, such as paying physicians for consulting services, may not in all cases meet all of the criteria for protection under a statutory exception or regulatory safe harbor.

Failure to meet all of the requirements of a particular applicable statutory exception or regulatory safe harbor does not make the conduct per se illegal under the AKS.

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A person or entity does not need to have actual knowledge of the statute or specific intent to violate it in order to have committed a violation.

Civil and criminal false claims laws, including the FCA, and civil monetary penalty laws, which can be enforced through civil whistleblower or qui tam actions, prohibit, among other things, individuals or entities from knowingly presenting, or causing to be presented, claims for payment of federal government funds, including in federal healthcare programs, that are false or

Other federal and state anti-kickback laws exist and, among other restrictions, prohibit certain payments related to referrals of patients to certain providers (such as clinical laboratories), applying to services reimbursed by private health plans as well as government health care programs.

Pharmaceutical and other healthcare companies have been prosecuted under these laws for engaging in a variety of different types of conduct that caused the submission of false claims to federal healthcare programs.

Under the AKS, for example, a claim resulting from a violation of the AKS is deemed to be a false or fraudulent claim for purposes of the FCA.

The FCA imposes mandatory treble damages and per-violation civil penalties up to approximately $25,000.

HIPAA created additional federal criminal statutes that prohibit, among other things, executing a scheme to defraud any healthcare benefit program, including private third-party payors, and making false statements relating to healthcare matters.

A person or entity does not need to have actual knowledge of the healthcare fraud statute implemented under HIPAA or specific intent to violate the statute in order to have committed a violation.

Biopharmaceutical manufacturers also are subject to federal and state price reporting laws.

Such laws require manufacturers to calculate and report pricing metrics to government programs, where such reported prices may be used in the calculation of reimbursement and/ or discounts on drug products.

Certain laws also may require biopharmaceutical manufacturers to offer products at discounted prices to specific government programs or specific purchasers as a condition for participation in certain government health benefit programs.

The FDCA addresses, among other things, the design, production, labeling, promotion, manufacturing, and testing of drugs, biologics and medical devices, and prohibits such acts as the introduction into interstate commerce of adulterated or misbranded drugs or devices.

The PHSA also prohibits the introduction into interstate commerce of unlicensed or mislabeled biological products.

federal Physician Payments Sunshine Act requires certain manufacturers of drugs, devices, biologics and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program, with specific exceptions, to annually report to CMS information related to payments or other transfers of value to various healthcare professionals including physicians, physician assistants, nurse practitioners, clinical nurse specialists, certified nurse anesthetists, certified nurse-midwives, and teaching hospitals, as well as ownership and investment interests held by physicians and their immediate family members.

Beginning on January 1, 2023, California Assembly Bill 1278 requires California physicians and surgeons to notify patients of Open Payments.

Laws and regulations have been enacted by the federal government and various states to regulate the sales and marketing practices of pharmaceutical manufacturers.

The laws and regulations generally limit financial interactions between manufacturers and health care providers;

require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the U.S.

federal government;

and/or require disclosure to the government and/or public of financial interactions (so-called “sunshine laws”).

For instance, the federal “sunshine” law implemented as Open Payments requires certain manufacturers of drugs, devices, biologics and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program, with specific exceptions, to annually report to the U.S.

Center for Medicare & Medicaid Services (“CMS”) information related to payments or other transfers of value to various healthcare professionals including physicians, physician assistants, nurse practitioners, clinical nurse specialists, certified nurse anesthetists, certified nurse-midwives, and teaching hospitals, as well as ownership and investment interests held by physicians and their immediate family members.

State and local laws may also require disclosure of pharmaceutical pricing information and marketing expenditures or licensure of sales representatives.

Manufacturers must also submit information to the FDA on the identity and quantity of drug samples requested and distributed by a manufacturer during each year.

New laws may be implemented.

For example, since January 1, 2023, California physicians and surgeons have had to notify patients of Open Payments where financial interactions with biopharmaceutical and medical device manufacturers are disclosed.

In addition, federal and state consumer protection and unfair competition laws broadly regulate our marketplace activities and activities that potentially harm consumers.

We are also subject to additional similar U.S.

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If our operations are found to be in violation of any of such laws or any other governmental regulations that apply, we may be subject to penalties, including, without limitation, civil, criminal and administrative penalties, damages, fines, exclusion from government-funded healthcare programs, such as Medicare and Medicaid or similar programs in other countries or jurisdictions, integrity oversight and reporting obligations to resolve allegations of non-compliance, disgorgement, individual imprisonment, contractual damages, reputational harm, diminished profits and the curtailment or restructuring of our operations.

For other countries outside of the EU, UK and United Stated such as countries in Eastern Europe, Latin America or Asia, the requirements governing the conduct of clinical trials, product licensing, pricing and reimbursement vary from country to country.

For other countries outside of the EU, UK and United States such as countries in Eastern Europe, Latin America or Asia, the requirements governing the conduct of clinical trials, product licensing, pricing and reimbursement vary from country to country.

In all cases, clinical trials must be conducted in accordance with GCP and the applicable regulatory requirements and the ethical principles that have their origin in the Declaration of Helsinki.

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and reporting of breaches of PHI to individuals and regulators.

A person or entity does not need to have actual knowledge of the healthcare fraud statute implemented under HIPAA or specific intent to violate the statute in order to have committed a violation.

Entities that are found to be in violation of HIPAA may be subject to significant civil, criminal, and administrative fines and other penalties and/or additional reporting and oversight obligations, for example, if required to enter into a resolution agreement and corrective action plan with the U.S.

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The HITECH Act also increased the civil and criminal penalties applicable to covered entities and business associates and gave state attorneys general new authority to file civil actions for damages or injunctions in federal courts to enforce HIPAA and seek attorneys’ fees and costs associated with pursuing federal civil actions.

To the extent that we submit electronic healthcare claims and payment transactions that do not comply with the electronic data transmission standards established under HIPAA and the HITECH Act, payments to us may be delayed or denied.

To the extent that submissions of electronic healthcare claims and payment transactions that do not comply with the electronic data transmission standards established under HIPAA and the HITECH Act, payments to us may be delayed or denied.

Even when HIPAA does not apply, according to the Federal Trade Commission, violating consumers’ privacy rights or failing to take appropriate steps to keep consumers’ personal information secure, including medical and health-related information, may constitute unfair or deceptive acts or practices in or affecting commerce in violation of Section 5(a) of the Federal Trade Commission Act.

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The CCPA applies to personal data of consumers, business contacts, and employees, and imposes obligations on certain businesses that do business in California, including to provide specific disclosures in privacy notices and rights to California residents in relation to their personal information.

Health information may fall under the CCPA’s definition of personal information where it identifies, relates to, describes, or is reasonably capable of being associated with or could reasonably be linked with a particular consumer or household—unless it is subject to HIPAA—and is included under a new category of personal information, “sensitive personal information,” which is offered greater protection.

offered greater protection.

In addition, almost 20 other states have now passed comprehensive privacy laws that have taken effect or will come into effect at various times over the next few years.

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In the United States, the pharmaceutical industry has been a particular focus of these efforts and has been significantly affected by federal and state legislative initiatives, including those designed to limit the pricing, coverage, and reimbursement of pharmaceutical and biopharmaceutical products, especially under government-funded health-care programs, and increased governmental control of drug pricing.

The ACA, which was enacted in March 2010, substantially changed the way healthcare is financed by both governmental and private insurers in the United States, and significantly affected the pharmaceutical industry.

Medicaid Drug Rebate Program are calculated for drugs that are inhaled, infused, instilled, implanted or injected, and annual fees based on pharmaceutical companies’ share of sales to federal health-care programs.

For example, the ACA, which was enacted in March 2010, substantially changed the way healthcare is financed by both governmental and private insurers in the United States, and significantly affected the pharmaceutical industry.

The ACA contains a number of provisions of particular import to the pharmaceutical and biotechnology industries, including, but not limited to, those governing enrollment in federal healthcare programs, altering certain requirements in the methodology by which rebates owed by manufacturers under the Medicaid Drug Rebate Program are calculated for drugs, and imposing annual fees based on pharmaceutical companies’ share of sales to federal health-care programs.

Since its enactment, there have been judicial and Congressional challenges to certain aspects of the ACA, and we expect there will be additional challenges and amendments to the ACA in the future.

Other legislative changes have been proposed and adopted since the ACA was enacted, including automatic aggregate reductions of Medicare payments to providers of 2% per fiscal year as part of the federal budget sequestration under the Budget Control Act of 2011.

These reductions went into effect in April 2013 and, due to subsequent legislative amendments, will remain in effect through 2030 with the exception of a temporary suspension from May 1, 2020 through December 31, 2020, unless additional action is taken by Congress.

In addition, the Bipartisan Budget Act of 2018, among other things, amended the Medicare Act (as amended by the ACA) to increase the point-of-sale discounts that manufacturers must agree to offer under the Medicare Part D coverage discount program from 50% to 70% off negotiated prices of applicable brand drugs to eligible beneficiaries during their coverage gap period, as a condition for the manufacturer’s outpatient drugs being covered under Medicare Part D.

Other legislative changes have been proposed and adopted since the ACA was enacted, , and in recent years, the pharmaceutical industry has been a particular focus of healthcare reform efforts and has been significantly affected by major legislative, administrative and executive initiatives.

For example, the Inflation Reduction Act of 2022 (“IRA”) included a number of changes relevant to drug prices in Medicare Parts B and D, including caps on Medicare Part D out-of-pocket costs, Medicare Part B and Part D inflationary rebates, a new Medicare Part D manufacturer discount drug program (replacing the previous coverage gap discount program) and a drug price negotiation program for certain high-spend Medicare Part B and D drugs.

The IRA has had and will likely continue to have a significant impact on the pharmaceutical industry.

Other recent reform initiatives have focused on drug pricing.

For example, President Trump issued Executive Orders targeting drug pricing, including to direct agencies to facilitate most favored nation drug pricing and direct to consumer purchasing initiatives.

In the wake of these Executive Orders and related executive initiatives, a number of pharmaceutical manufacturers have announced direct-to-consumer offerings with discounted prices and/or reached agreement with the federal government regarding pricing for drugs, including prices for Medicaid drugs and newly launched products.

A website sponsored by the federal government that is anticipated to offer pharmaceutical direct-to-consumer channels has also been announced and launched in February 2026.

Federal agencies are developing new drug pricing pilot programs, such as a voluntary Medicaid initiative which would authorize the federal government to negotiate Medicaid supplemental rebates with participating manufacturers on behalf of state Medicaid programs, in exchange for standardized coverage criteria for participating manufacturer drugs, and proposed Medicare Part B and Part D pilot models that, if finalized as proposed, would replace existing inflation-based Medicare rebates with rebates determined by reference to international drug prices.

Many of these reform initiatives would require additional legal and/or administrative action to implement and may be subject to legal challenge.

Other federal healthcare reform efforts or actions may affect access to healthcare coverage or the funding of health care benefits, although the full impact of such efforts or actions cannot be predicted.

At the state level, individual states are increasingly implementing initiatives designed to control pharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and measures to encourage importation from other countries and bulk purchasing.

For example, certain states have formed Prescription Drug Affordability Boards that assert authority to set reimbursement rates and/or drug pricing in the state.

These and other future state-level reform activities could negatively affect pricing, coverage and reimbursement for our products.

Other recent government actions also may affect prices or payments for prescription drugs.

For example, the Trump Administration’s recently announced tariff on branded or patented drugs may adversely impact our ability to realize an adequate return on the sale of drug products (if approved) that are imported from abroad or manufactured using products or materials imported from abroad.

The timeline for implementation of this tariff has not yet been finalized.

As another example, the Budget Control Act of 2011, provided for automatic aggregate reductions of Medicare payments to providers of 2% per fiscal year as part of the federal budget sequestration.

These reductions resulted in the imposition of reductions in Medicare (but not Medicaid) payments to providers in 2013 and will remain in effect into 2032, unless additional action is taken by Congress.

Any significant spending reductions affecting Medicare, Medicaid or other publicly funded or subsidized health programs that may be implemented and/or any significant taxes or fees that may be imposed on us could have an adverse impact on our results of operations.

Moreover, there has recently been heightened governmental scrutiny over the manner in which manufacturers set prices for their marketed products, which has resulted in several Congressional inquiries and proposed and enacted federal and state measures designed to, among other things, reduce the cost of prescription drugs, bring more transparency to product pricing, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement methodologies for drug products.

For example, in May 2019, CMS adopted a final rule allowing Medicare Advantage Plans the option to use step therapy for Part B drugs, permitting Medicare Part D plans to apply certain utilization controls to new starts of five of the six protected class drugs, and requiring the Explanation of Benefits for Part D beneficiaries to disclose drug price increases and lower cost therapeutic alternatives, which went into effect on January 1, 2021.

Notwithstanding the IRA, continued legislative and enforcement interest exists in the United States with respect to specialty drug pricing practices.

Specifically, we expect regulators to continue pushing for transparency to drug pricing, reducing the cost of prescription drugs under Medicare, reviewing the relationship between pricing and manufacturer patient programs, and reforming government program reimbursement methodologies for drugs.

Continued legislative and enforcement interest exists in the United States with respect to specialty drug pricing practices.

Specifically, we expect regulators to continue pushing for transparency to drug pricing, reducing the cost of prescription drugs under Medicare and other federal health care programs, reviewing the relationship between pricing and manufacturer patient programs, and reforming government program reimbursement methodologies for drugs.

The nature and extent of future healthcare reforms cannot be predicted.

There is uncertainty regarding the nature or impact of any drug pricing or broader health care or other reform implemented at the federal or state level and the extent to which such action may be subject to litigation or other challenges.

Ongoing efforts to contain or reduce costs of healthcare and/or impose price controls may adversely affect the demand for our product candidates, if approved, and our ability to achieve or maintain profitability.

Coverage and Reimbursement

Significant uncertainty exists as to the coverage and reimbursement status of any pharmaceutical or biological product for which we obtain regulatory approval.

Sales of any product, if approved, depend, in part, on the extent to which such product will be covered by third-party payors, such as federal, state, and foreign government healthcare programs, commercial insurance and managed healthcare organizations, and the level of reimbursement, if any, for such product by third-party payors.

Decisions regarding whether to cover any of our product candidates, if approved, the extent of coverage and amount of reimbursement to be provided are made on a plan-by-plan basis.

Further, no uniform policy for coverage and reimbursement exists in the United States, and coverage and reimbursement can differ significantly from payor to payor.

Third-party payors often rely upon Medicare coverage policy and payment limitations in setting their own reimbursement rates, but also have their own methods and approval process apart from Medicare determinations.

As a result, the coverage determination process is often a time-consuming and costly process that will require us to provide scientific and clinical support for the use of our product candidates to each payor separately, with no assurance that coverage and adequate reimbursement will be applied consistently or obtained in the first instance.

Third-party payors are increasingly challenging the prices charged for medical products and services, examining the medical necessity and reviewing the cost effectiveness of pharmaceutical or biological products, medical devices and medical services, in addition to questioning safety and efficacy.

and foreign markets, patients generally rely on third-party payors to reimburse all or part of the costs associated with their therapy.

Our ability to successfully commercialize our product candidates, if and when approved, will depend in part on the extent to which coverage and adequate reimbursement for these products and related treatments will be available from government healthcare programs, private health insurers and other organizations.

Within the U.S., no uniform policy for coverage and reimbursement exists, and coverage and reimbursement for drug products can differ significantly from payor to payor.

Significant uncertainty exists as to the coverage and reimbursement status of any pharmaceutical or biological product for which we may obtain regulatory approval.

Decisions regarding whether to cover any of our product candidates, if approved, the extent of coverage and amount of reimbursement to be provided are made on a payor-by-payor basis and coverage and reimbursement can differ significantly from payor to payor.

As a result, the coverage determination process is often a time-consuming and uncertain process with no assurance that coverage and adequate reimbursement will be applied consistently or obtained in the first instance.

Even if products are covered, payors may seek to control utilization of the products through various mechanisms.

Coverage of a product by a third-party payor does not mean that reimbursement will be adequate, and third-party payor reimbursement may not be sufficient to enable us to maintain price levels high enough to realize an appropriate return on our investment in product development.

Adoption of price controls and cost-containment measures, and adoption of more restrictive policies in jurisdictions with existing controls and measures, could further limit sales of any product that receives approval.

Decreases in third-party reimbursement for any product or a decision by a third-party not to cover a product could reduce physician usage and patient demand for the product.

For products administered under the supervision of a physician, obtaining coverage and adequate reimbursement may be particularly difficult because of the higher prices often associated with such drugs.

Additionally, separate reimbursement for the product itself or the treatment or procedure in which the product is used may not be available, which may impact physician utilization.

In addition, companion diagnostic tests require coverage and reimbursement separate and apart from the coverage and reimbursement for their companion pharmaceutical or biological products.

Similar challenges to obtaining coverage and reimbursement, applicable to pharmaceutical or biological products, will apply to companion diagnostics.

In addition, the U.S.

government, state legislatures and foreign governments have continued implementing cost-containment programs, including price controls, restrictions on coverage and reimbursement and requirements for substitution of generic products.

Recently, the U.S.

government passed the IRA, which includes provisions that authorize the U.S.

Department of Health and Human Services to negotiate prices of certain drugs with participating manufacturers in federal healthcare programs.

The IRA provides CMS with significant new authorities intended to curb drug costs and to encourage market competition.

For the first time, CMS will be able to directly negotiate prescription drug prices and to cap out-of-pocket costs.

Each year, CMS will select and negotiate a preset number of high-spend drugs and biologics that are covered under Medicare Part B and Part D that do not have generic or biosimilar competition.

These price negotiations will begin in 2023.

The IRA also provides a new “inflation rebate” covering Medicare patients that will take effect in 2023 and is intended to counter certain price increases in prescriptions drugs.

The inflation rebate provision will require drug manufacturers to pay a rebate to the federal government if the price for a drug or biologic under Medicare Part B and Part D increases faster than the rate of inflation.

To support biosimilar competition, beginning in October 2022, qualifying biosimilars may receive a Medicare Part B payment increase for a period of five years.

Separately, if a biologic drug for which no biosimilar exists delays a biosimilar’s market entry beyond two years, CMS will be authorized to subject the biologics manufacturer to price negotiations intended to ensure fair competition.

Notwithstanding these provisions, the IRA’s impact on commercialization and competition remains largely uncertain.

Net prices for products may be reduced by mandatory discounts or rebates required by government healthcare programs or private payors or by future laws, regulations, or guidance seeking to limit prescription drug prices.

Decreases in coverage and adequate third-party reimbursement from governmental healthcare programs, such as Medicare and Medicaid, and commercial payors for any product or a decision by a third-party not to cover a product could reduce physician usage and patient demand for the product.

For products administered under the supervision of a physician, inadequate reimbursement for the product itself or the treatment or procedure in which the product is used may adversely impact physician utilization.

The healthcare regulatory landscape can also be affected by election cycles and any resulting changes in healthcare policy priorities and broader industry response.

From time to time, the executive branch has issued executive orders aimed at reducing prescription drug prices, including policies that seek to link U.S.

drug prices to those paid in other countries.

Consistent with these objectives, CMS has proposed drug pricing models intended to reduce prescription drug costs that, if implemented, would require manufacturers to pay rebates on certain Medicare products when U.S.

prices for those products exceed benchmark prices based on prices paid in a set of economically comparable countries.

These changes, along with new demonstration modes adopted by the Center for Medicare and Medicaid Innovation, and other changes to current healthcare laws and reform measures that may be adopted in the future may significantly impact pricing, coverage, and reimbursement for any product candidates for which we obtain regulatory approval.

The full effect of these provisions on commercialization and competition remains uncertain.

We cannot be sure that adequate coverage and reimbursement will be available, or remain available, for any drug that we commercialize.

Coverage and reimbursement may impact the demand for, or the price of, our products and any product candidate for which we obtain marketing approval and limits on coverage and reimbursement may adversely affect our ability to successfully commercialize any product candidate for which we obtain marketing approval.

Manufacturing

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The agreement includes confidentiality and intellectual property provisions to protect our proprietary rights related to our product candidates.

We do not currently have arrangements for redundant supply and are working on building our supply chain to put these in place.

Any reduction or halt in supply from the CDMO could limit our ability to develop our product candidates until a replacement CDMO is found and qualified, although we believe that we have supply on hand that can partially support our current clinical trial programs until a replacement CDMO is secured.

We do not currently have arrangements for redundant supply and are working on building our supply chain robustness.

In light of our reliance on WuXi, we are taking several measures to strengthen our supply chain by moving certain CDMO activities outside of WuXi’s facilities.

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Sales and Marketing

We have not yet defined our sales, marketing, or product distribution strategy for our product candidates because our product candidates are still in development.

We have not yet fully defined our sales, marketing, or product distribution strategy for our product candidates because our product candidates are still in development.

Our commercial strategy may include the use of strategic partners, distributors, a contract sale force, or the establishment of our own commercial and specialty sales force.

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We are committed to equality, inclusion and diversity in the workplace.

As of December 31, 2024, approximately 24% of our workforce identify as members

of underrepresented ethnic communities and approximately 59% identify as female.

As of December 31, 2025, approximately 23% of our workforce identify as members of underrepresented ethnic communities and approximately 44% identify as female.

We strive to develop a diverse slate of candidates to interview for our open positions.

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The principal purpose of our equity incentive and annual bonus programs is to attract, retain and motivate personnel through the granting of stock-based compensation awards and cash-based performance bonus awards.

As a biopharmaceutical company, we recognize the importance of access to high quality healthcare and as such we cover 100% of our employees’ monthly healthcare premiums.

As a biopharmaceutical company, we recognize the importance of access to high quality healthcare and as such we cover a percentage of our employees’ monthly healthcare premiums.

We offer a package of competitive employee benefits, including 401(k) plan matching contributions and an employee stock purchase plan.

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as a Delaware limited liability company in January 2010 and subsequently incorporated as a Delaware corporation in June 2014.

On January 20, 2021, pursuant to a merger agreement under which miRagen Therapeutics, Inc.

In January 2021, pursuant to a merger agreement under which miRagen Therapeutics, Inc.

acquired Viridian Therapeutics, Inc., we changed our name from Miragen Therapeutics, Inc.

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The reports are also available at the SEC’s internet website at www.sec.gov.

A copy of our Corporate Governance Guidelines, Code of Business Conduct and Ethics, and the charters of the Audit Committee, Compensation Committee, Nominating and Corporate Governance Committee, and Science and Technology Committee are posted on our website, www.viridiantherapeutics.com, under “Corporate Governance.”

Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-K

Item 1. Business