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Item 1. Business

2026-03-05 compared with 2025-03-03 · 267 added, 246 removed, 904 unchanged (36% of the section changed)

We are a clinical-stage biopharmaceutical company dedicated to the identification, development and commercialization of differentiated medicines to address the unmet medical needs of patients with cancers.

We seek to utilize our team’s deep drug development experience to maximize the potential of our lead development candidate, firmonertinib, and advance a pipeline of novel therapeutics, such as next-generation antibody drug conjugates, through approval and commercialization in patients suffering from cancer, with an initial focus on solid tumors.

Firmonertinib is currently being evaluated in multiple clinical trials across a range of epidermal growth factor receptor (EGFR) mutations (EGFRm) in non-small cell lung cancer (NSCLC), including a pivotal Phase 3 clinical trial in treatment naive, or first-line, patients with locally advanced or metastatic EGFRm NSCLC with exon 20 insertion mutations.

We received Breakthrough Therapy Designation for firmonertinib for this disease from the U.S.

We seek to utilize our team’s deep drug development experience to maximize the potential of our lead product candidate, firmonertinib, and advance a pipeline of novel therapeutics, such as next-generation antibody drug conjugates, including ARR-217 (MRG007), through approval and commercialization in patients suffering from cancer, with an initial focus on solid tumors.

Firmonertinib is currently being evaluated in multiple clinical trials across a range of epidermal growth factor receptor (EGFR) mutations (EGFRm) in non-small cell lung cancer (NSCLC).

We are conducting a pivotal Phase 3 clinical trial of firmonertinib in treatment naive, or first-line, patients with locally advanced or metastatic EGFRm NSCLC with exon 20 insertion mutations and a pivotal Phase 3 clinical trial of firmonertinib in first-line patients with locally advanced or metastatic EGFRm NSCLC with P-loop and-alpha-c-helix compressing (PACC) mutations.

We are conducting a Phase 1 clinical trial of ARR-217 in patients with unresectable locally advanced or metastatic solid tumors.

We received Breakthrough Therapy Designation (BTD) for firmonertinib for first line EGFRm NSCLC with exon 20 insertion from the U.S.

Food and Drug Administration (FDA) in October 2023 , and Orphan Drug Designation for treatment of NSCLC with EGFRm or human epidermal growth factor receptor 2 (HER2) mutations or human epidermal growth factor receptor 4 (HER4) mutations in February 2024 .

A product candidate can receive Breakthrough Therapy Designation if preliminary clinical evidence indicates that the product candidate, alone or in combination with one or more other drugs, may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.

For drugs that have been designated as Breakthrough Therapies, interaction and communication between the FDA and the sponsor can help to identify the most efficient path for development.

The receipt of a Breakthrough Therapy Designation for a product candidate may not result in a faster development process, review or approval compared to product candidates considered for approval under conventional FDA procedures and does not increase the likelihood that the product candidate will ultimately receive FDA approval for any indication.

A product candidate can receive BTD if preliminary clinical evidence indicates that the product candidate, alone or in combination with one or more other drugs, may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.

For drugs that have received BTD, interaction and communication between the FDA and the sponsor can help to identify the most efficient path for development, although BTD may not result in a faster development process, review or approval and does not increase the likelihood that the product candidate will ultimately receive FDA approval for any indication.

In 2021, we licensed from Shanghai Allist Pharmaceuticals, Co.

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The FDA has not approved firmonertinib for any use.

We selected firmonertinib for global development against nonclassical, or uncommon, mutations based on preliminary reductions in tumor size activity observed in seven out of ten patients in first-line treatment with EGFR exon 20 insertion mutations in the ongoing Phase 1b clinical trial, the FAVOUR trial, conducted by Allist in China, and preclinical activity in P-loop and-alpha-c-helix compressing (PACC) mutations, each a subtype of uncommon mutation.

We selected firmonertinib for global development against nonclassical, or uncommon, mutations based on preliminary reductions in tumor size observed in seven out of ten patients with EGFR exon 20 insertion mutations treated with firmonertinib in the ongoing Phase 1b clinical trial, the FAVOUR trial, conducted by Allist in China, and preclinical activity in PACC mutations, each a subtype of uncommon mutation.

In a subsequent interim data readout from the FAVOUR trial of firmonertinib administered at 240 mg once daily in first-line patients with locally advanced or metastatic EGFRm NSCLC with exon 20 insertion mutations, 79% of patients (n=22 out of 28 patients) were observed to experience a reduction in tumor size of at least 30%.

In a final analysis from the Phase 1b FURTHER trial of firmonertinib, which included a cohort of EGFRm NSCLC with PACC mutations, we observed 16.0 months median progression free survival (mPFS) with firmonertinib 240 mg in first-line, confirmed overall response rate (cORR) 68.2% (n=15 out of 22 1L patients at 240mg) and duration of response (DOR) 14.6 months, and confirmed CNS (central nervous system) responses with firmonertinib including complete responses (CRs).

As one of the most prevalent cancers in the world, lung cancer imposes a significant global burden on human health, and EGFRm NSCLC represents a significant proportion of those affected.

Despite progress in the therapeutic landscape for EGFRm NSCLC, many patients, particularly those with uncommon mutations, such as exon 20 insertions or PACC mutations, are underserved by existing treatments.

In an interim data readout from the FAVOUR trial of firmonertinib administered at 240 mg once daily in first-line patients with locally advanced or metastatic EGFRm NSCLC with exon 20 insertion mutations, 79% of patients (n=22 out of 28 patients) were observed to experience a reduction in tumor size of at least 30% from the baseline in a patient without evidence of progression as measured by Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 criteria.

Criteria in Solid Tumors (RECIST) 1.1 criteria.

This measurement of reduction is the threshold in this trial for a partial response and for inclusion in determination of the overall response rate (ORR), which is the primary endpoint of this trial.

In the same interim data readout, those 79% of patients were observed to experience a 15.2 month median duration of response (DOR).

In the same interim data readout, those 79% of patients were observed to experience a 15.2 month median DOR.

Interim results may not be indicative of final results;

however, we believe these interim clinical results underscore firmonertinib’s potential in patients whose tumors contain an uncommon EGFRm.

We are currently conducting a pivotal Phase 3 clinical trial, the FURVENT clinical trial, in first-line locally advanced or metastatic non-squamous EGFRm NSCLC patients with exon 20 insertion mutations and we expect topline data from this trial in 2025.

Additionally, we are conducting a Phase 1b clinical trial, the FURTHER clinical trial, in NSCLC patients with activating EGFRm, including a cohort with PACC mutations.

In September 2024, we reported 64% of patients (n=14 out of 22 1L patients at 240mg) were observed to experience a confirmed reduction in tumor size of at least 30% from the baseline without evidence of progression as measured by RECIST 1.1 criteria .

Additionally, we are conducting a pivotal Phase 3 clinical trial, the ALPACCA clinical trial, in first-line locally advanced or metastatic non-squamous EGFRm NSCLC patients PACC mutations.

In September 2025, our final Phase 1b PACC data reported 68.2% of patients (n=15 out of 22 1L patients at 240 mg) were observed to experience a confirmed reduction in tumor size of at least 30% from the baseline without evidence of progression as measured by RECIST 1.1 criteria .

The confirmed mPFS in these patients was 16.0 months.

Since our inception in 2021, we have assembled a robust oncology pipeline by leveraging our global network and our experience in business development transactions.

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(Alphamab), a wholly owned subsidiary of Alphamab Oncology, to discover, develop and commercialize novel ADCs for the treatment of cancers globally, with the exception of greater China, which includes mainland China, Hong Kong, Macau and Taiwan.

In January 2025, we entered into an exclusive license agreement with Lepu Biopharma Co.

(Lepu Biopharma) pursuant to which Lepu Biopharma granted us the right to develop and commercialize ARR-217 (MRG-007), an ADC for gastrointestinal cancers outside greater China, which includes mainland China, Hong Kong, Macau, and Taiwan.

In January 2025, we entered into an Exclusive License Agreement (the Lepu Biopharma Agreement) with Lepu Biopharma Co.

(Lepu Biopharma) pursuant to which Lepu Biopharma granted us the right to develop and commercialize ARR-217 (MRG007), a CD-H17-targeting ADC for gastrointestinal cancers outside greater China, which includes mainland China, Hong Kong, Macau, and Taiwan.

We plan to continue to engage in business development activities to source additional innovative therapeutics in areas currently underserved by approved therapies.

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Breakthrough Therapy Designation;

Antibody Drug Conjugate;

gastrointestinal tumors;

IND-enabling studies:

Investigational New Drug enabling studies;

new molecular entity;

First-line therapy;

Treatment naive and previously treated with non-TKI therapies;

Second-line or greater therapy;

SHP2 inhibitor.

The investigation of firmonertinib for the first-line treatment of NSCLC EGFR exon 20 insertion mutations is based on the ongoing FAVOUR Phase 1b study conducted by Allist and the ongoing FURVENT Phase 3 study.

These studies are not yet complete, and no Phase 2 study has been conducted for firmonertinib for the first-line treatment of NSCLC EGFR exon 20 insertion mutations.

The ongoing FURTHER Phase 1b study investigating firmonertinib for the treatment of EGFRm NSCLC includes cohorts with PACC mutations (first-line or greater) and exon 20 insertion mutations (second-line or greater).

The evaluation of firmonertinib in combination with SHP2i for the second-line or greater treatment of EGFRm NSCLC is based on the ongoing Phase 1b study in collaboration with InnoCare.

Global Ex-China:

Worldwide, except mainland China, Hong Kong, Macau and Taiwan.

Currently approved EGFR TKIs have achieved considerable commercial success and have become the standard of care for patients with NSCLC with classical EGFRm which comprise approximately 69% of all EGFRm NSCLC patients.

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Firmonertinib’s ability to cross the blood-brain barrier was also demonstrated in this clinical trial with a central nervous system (CNS) metastases specific ORR, which measured reduction of tumor size of at least 30% in brain metastases if present at the start of therapy, of 91% versus 65% for gefitinib.

Based on observed clinical activity against exon 20 insertions in the FAVOUR clinical trial and observed activity against PACC mutations in preclinical studies, we believe firmonertinib is potentially differentiated from third-generation EGFR TKIs approved for classical EGFRm NSCLC.

Based on observed clinical activity against exon 20 insertions in the FAVOUR clinical trial and observed activity against PACC mutations in the FURTHER clinical trial, we believe firmonertinib is potentially differentiated from third-generation EGFR TKIs approved for classical EGFRm NSCLC.

In the United States and the European Union, standard of care for first-line therapy in EGFRm NSCLC involving exon 20 insertion mutations is platinum-based chemotherapy with pemetrexed with or without amivantamab.

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Over 9% of EGFRm NSCLC patients are estimated to have exon 20 insertion mutations.

However, amivantimab lacks brain penetrance to effectively treat brain metastasis.

However, amivantamab appears to lack brain penetrance to effectively treat brain metastasis.

In March 2024 and July 2024, amivantamab in combination with chemotherapy, was approved in the United States and Europe, respectively, for first line EGFRm NSCLC patients with exon 20 insertion mutations.

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We believe firmonertinib, if approved, has the potential to become a chemotherapy-free oral regimen in first-line EGFRm NSCLC patients with exon 20 insertion mutations given the clinical data generated in this patient population to date.

Guidelines employing TKIs for the treatment of many of the PACC-specific EGFRm are not established and, as a result, chemotherapy is often used as the default course of therapy, offering limited efficacy and introducing chemotherapy-related toxicity.

Afatinib (GILOTRIF ® ), a second-generation TKI, is also used in some patients, but has a poor safety profile and is not brain penetrant.

Osimertinib is frequently used in some patients, but the activity of osimertinib is much less than in classical EGFRm NSCLC.

Guidelines employing TKIs for the treatment of many of the PACC EGFRm are not established and, as a result, chemotherapy is often used as the default course of therapy, offering limited efficacy and introducing chemotherapy-related toxicity.

Afatinib (GILOTRIF ® ), a second-generation TKI, is also used in some patients, but has an unfavorable safety profile and is not brain penetrant.

Osimertinib is frequently used in some patients, but the activity of osimertinib in such patients is less than its activity in patients with classical EGFRm NSCLC.

Over 12% of EGFRm NSCLC patients are estimated to have PACC mutations.

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*Allist sponsor;

** InnoCare sponsor;

First-Line Therapy;

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FURVENT — Our Ongoing Phase 3 Clinical Trial in First-Line Non-Squamous Locally Advanced or Metastatic EGFRm NSCLC Patients with Exon 20 Insertion Mutations

We have enrolled our target of 375 patients in FURVENT, a global, pivotal Phase 3 clinical trial of firmonertinib in first-line non-squamous locally advanced or metastatic NSCLC patients with exon 20 insertion mutations being conducted jointly with Allist.

We enrolled 398 patients in FURVENT, a global, pivotal Phase 3 clinical trial of firmonertinib in first-line non-squamous locally advanced or metastatic NSCLC patients with exon 20 insertion mutations being conducted jointly with Allist.

Our FURVENT clinical trial is designed to assess the safety and efficacy of firmonertinib administered at either 160 mg or 240 mg, once-daily as compared to platinum-based chemotherapy with pemetrexed, the current first-line standard of care.

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We enrolled 398 patients globally, including from sites in the United States, Europe and certain Asian countries including China.

We expect topline data from our FURVENT clinical trial in 2025.

FURTHER — Our Ongoing Phase 1b Clinical Trial in NSCLC Patients with EGFR Activating Mutations including PACC Mutations

We have completed enrolling patients in FURTHER, an ongoing, global Phase 1b dose escalation and expansion clinical trial being conducted jointly with Allist.

It is intended to evaluate the safety, pharmacokinetics and preliminary anti-tumor activity of once-daily firmonertinib when used in patients with NSCLC involving locally advanced or metastatic disease that have previously received systemic therapy and whose tumors contain EGFR activating mutations.

Anti-tumor activity is measured by confirmed complete response, defined as the disappearance of all target lesions, or partial response, defined as at least a 30% decrease in target lesions in the absence of a complete response, relative to the total number of patients.

The pharmacokinetics, adverse events, serious adverse events, and observed anti-tumor activity in the exon 20 patients of firmonertinib in the clinical trial to date are consistent with those observed in the FAVOUR clinical trial conducted in China.

The FURTHER clinical trial includes a cohort of 60 patients with a PACC EGFRm who are TKI treatment-naïve, and has been fully enrolled.

In September 2024, we reported 64% of patients (n=14 out of 22 patients) treated at the 240 mg dose were observed to experience a confirmed reduction in tumor size of at least 30% from the baseline without evidence of progression as measured by RECIST 1.1 criteria .

FAVOUR — Ongoing Phase 1b Clinical Trial in NSCLC Patients with EGFR Exon 20 Insertion Mutations

We expect topline data from our FURVENT clinical trial in mid-2026.

FAVOUR — Phase 1b Clinical Trial in NSCLC Patients with EGFR Exon 20 Insertion Mutations

The FAVOUR clinical trial is an ongoing 90-patient Phase 1b clinical trial being conducted by Allist in China that is intended to assess the safety and efficacy of firmonertinib in locally advanced or metastatic NSCLC patients who have EGFR exon 20 insertion mutations.

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As of June 15, 2023, treatment related serious adverse events (TRSAEs) were observed in 6 out of 86 of the treated patients and 2 out of 86 patients discontinued participation in the trial as a result of TRAEs.

SHP2 Combination Trial — Ongoing Phase 1b Clinical Trial in Classical EGFRm NSCLC Patients

Given the eventual emergence of resistance that occurs with EGFR TKIs we are also evaluating combination partner drugs with firmonertinib to treat or prevent resistance to third-generation EGFR TKIs.

We are evaluating the use of firmonertinib in combination with ICP-189, a SHP2 inhibitor (SHP2i), in collaboration with Beijing InnoCare Pharma Tech Co., Ltd.

SHP2 is a protein tyrosine phosphatase with an important role in the signaling pathways downstream of EGFR and other receptor tyrosine kinases (RTKs) that have been implicated in resistance such as the mesenchymal epithelial transition factor receptor (MET) tyrosine kinase.

We believe this therapeutic combination may not only further inhibit signaling pathways downstream from EGFR resulting in enhanced tumor shrinkage, but may also prevent the emergence of resistance due to signaling through other RTKs.

We have submitted an Investigational New Drug Application (IND) to the NMPA’s Center for Drug Evaluation for a Phase 1b trial designed to evaluate the safety, pharmacokinetics and preliminary efficacy of the firmonertinib-ICP- 189 combination in EGFRm NSCLC involving classical mutations and dosed the first patient in March 2024 and expect the potential proof of concept in second-line classical EGFRm NSCLC to become available in 2026.

FURTHER — Our Ongoing Phase 1b Clinical Trial in NSCLC Patients with EGFR Activating Mutations including PACC Mutations

We have completed enrolling patients in FURTHER, an ongoing, global Phase 1b dose escalation and expansion clinical trial being conducted jointly with Allist.

The FURTHER clinical trial includes a cohort of 60 patients with a PACC EGFRm who are TKI treatment-naïve, and has been fully enrolled.

In September 2025, we announced our final data from the FURTHER trial of firmonertinib in first-line patients with locally advanced or metastatic EGFRm NSCLC with PACC mutations.

In this final readout, patients treated with 240 mg of firmonertinib were observed to experience 16.0 months mPFS and 14.6 months median duration of response.

Further, 68.2% of patients treated in first-line at 240 mg and 43.5% of patients treated in first-line at 160 mg were observed to experience a reduction in tumor size of at least 30% from the baseline in a patient without evidence of progression as measured by RECIST 1.1 criteria, which measurement of reduction is the threshold in this trial for determination of the ORR.

In addition, 47% (n=8/17) of patients with brain metastases at baseline were observed to experience a confirmed response utilizing modified RECIST 1.1 and 42.9% (n=6/14) of first line patients with brain metastases at baseline were observed to experience a reduction in tumor size of at least 30% from the baseline in a patient without evidence of progression as measured by RECIST 1.1 criteria.

Firmonertinib was generally well-tolerated and consistent with interim safety results.

ALPACCA — Our Ongoing Phase 3 Clinical Trial in First-Line Non-Squamous Locally Advanced or Metastatic EGFRm NSCLC Patients with PACC Mutations

In December 2025, we announced the first patient dosed and plan to enroll 480 patients in ALPACCA, a global, pivotal Phase 3 clinical trial of firmonertinib in first-line non-squamous locally advanced or metastatic NSCLC patients with PACC mutations being conducted jointly with Allist.

Our ALPACCA clinical trial is designed to assess the safety and efficacy of firmonertinib administered at 240 mg, once-daily as compared to an investigator’s choice of osimertinib or afatinib.

The co-primary endpoints of this study are ORR and PFS.

The study design reflects design elements from the FDA’s Project FrontRunner where we have the potential to seek accelerated approval based on ORR and full approval based on PFS.

Planned Adjuvant Study in NSCLC Patients with Uncommon EGFRm, including Exon 20 Insertion and PACC Mutations

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We believe firmonertinib similarly has the potential to improve outcomes when administered as adjuvant therapy to EGFRm NSCLC patients with uncommon EGFRm, as these patients are not eligible for treatment with osimertinib.

We intend to participate in a global adjuvant study of firmonertinib in EGFRm NSCLC with uncommon mutations initiated in China based on results obtained from currently ongoing clinical trials.

We intend to participate in a global adjuvant study of firmonertinib in EGFRm NSCLC with uncommon mutations initiated in China, or to conduct a parallel study, depending on results obtained from currently ongoing clinical trials.

FURLONG — Completed Phase 3 Clinical Trial in Classical EGFRm First-Line NSCLC Patients

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Firmonertinib demonstrated clinically meaningful and statistically significant efficacy and a favorable safety profile as compared to the first-generation EGFR TKI gefitinib.

Importantly, firmonertinib exhibited superior efficacy in the treatment of CNS metastases, producing a confirmed CNS metastases specific ORR, which measured reduction in tumor size of at least 30% in brain metastases if present at the start of therapy, of 91% versus 65% for gefitinib among participants with measurable metastatic CNS disease, demonstrating firmonertinib’s ability to cross the blood-brain barrier.

versus 65% for gefitinib among participants with measurable metastatic CNS disease, demonstrating firmonertinib’s ability to cross the blood-brain barrier.

Our Antibody Drug Conjugate Collaborations

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We are using Aarvik’s proprietary multi-target, multivalent site-specific conjugation antibody platform to discover and develop ADCs with potentially improved activity and safety over single target bivalent ADCs.

We identified a preclinical candidate ARR-002 and have initiated IND-enabling activities.

We identified a preclinical candidate ARR-002 and have initiated Investigational New Drug (IND)-enabling activities.

We are also working with Alphamab to leverage their proprietary linker-payload platform and glycan-conjugation technology to discover, develop, and commercialize ADCs for the treatment of cancers.

Through our agreement with Lepu Biopharma, we are developing ARR-217, a novel ADC for the treatment of gastrointestinal cancers.

Pursuant to the Lepu Biopharma Agreement, we are developing ARR-217, a novel CDH17-targeting ADC for the treatment of gastrointestinal cancers, which is currently in Phase 1 clinical development.

Our Team and Approach

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Our other co-founder and President of Research and Development is Stuart Lutzker, M.D., Ph.D.

Lutzker joined from Genentech, Inc., where he served in a number of senior R&D roles.

Lutzker joined from Genentech, Inc., where he served in a number of senior Research and Development roles.

Most recently, Dr.

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We do not currently intend to conduct a Phase 2 trial in first-line non-squamous locally advanced or metastatic EGFRm NSCLC patients with exon 20 insertion mutations.

We have completed enrollment in our global, pivotal Phase 3 FURVENT clinical trial in first-line non-squamous locally advanced or metastatic EGFRm NSCLC patients with exon 20 insertion mutations and we expect topline data from this clinical trial in 2025.

● Continue to advance firmonertinib through the Phase 1b FURTHER clinical trial in EGFRm NSCLC to obtain proof of concept for the treatment of patients with PACC mutations.

● Continue to advance firmonertinib through the pivotal Phase 3 ALPACCA clinical trial in EGFRm NSCLC patients with PACC mutations.

We are also advancing the clinical development of firmonertinib as a potential treatment for EGFRm NSCLC patients with PACC mutations.

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Guidelines employing TKIs for many of the PACC-specific EGFR mutations are not established and, as a result, chemotherapy is often used as the default course of therapy, offering limited efficacy and introducing chemotherapy-related toxicity.

Osimertinib is frequently used in some patients, but the activity of osimertinib is much less than in classical EGFR mutant NSCLC.

Afatinib, a second-generation TKI, is also used in some patients, but has a poor safety profile and is not brain penetrant.

We are investigating the use of firmonertinib to treat a broad set of PACC mutations in a cohort in our FURTHER clinical trial based on firmonertinib’s observed activity against PACC mutations in preclinical studies together with the evaluation of firmonertinib in multiple clinical trials with an aggregate patient population of over 1000 patients, including over 450 patients outside China.

Other cohorts in the FURTHER trial include patients with EGFR exon 20 insertion mutations as second-line or greater therapy.

Osimertinib is frequently used in some patients, but the activity of osimertinib in such patients is much less than its activity in classical EGFR mutant NSCLC.

Afatinib, a second-generation TKI, is also used in some patients, but has an unfavorable safety profile and is not brain penetrant.

We are investigating the use of firmonertinib to treat a broad set of PACC mutations the ALPACCA clinical trial based on firmonertinib’s observed activity against PACC mutations in preclinical studies together with the evaluation of firmonertinib in multiple clinical trials with an aggregate patient population of over 1000 patients, including over 450 patients outside China.

We have initiated our pivotal Phase 3 ALPACCA study with our first patient dosed in December 2025.

● Evaluate the clinical benefit of treating early-stage disease with firmonertinib.

We also intend to participate in a global registrational Phase 3 clinical trial initiated in China to investigate the potential benefit of firmonertinib in the adjuvant setting in NSCLC patients with uncommon EGFRm that are not eligible for osimertinib such as exon 20 insertion and PACC mutations.

We intend to join the adjuvant study of firmonertinib once the EGFRm patient group is further defined based on ongoing clinical trials in the metastatic setting.

We have not yet sought alignment on the design of the adjuvant study with the FDA who may ask us to collect more clinical data prior to joining the adjuvant study.

We also intend to participate in a global registrational Phase 3 clinical trial initiated in China , or to conduct a parallel study, to investigate the potential benefit of firmonertinib in the adjuvant setting in NSCLC patients with uncommon EGFRm that are not eligible for osimertinib such as exon 20 insertion and PACC mutations.

We intend to join the adjuvant study of firmonertinib , or to conduct a parallel study, once the EGFRm patient group is further defined based on ongoing clinical trials in the metastatic setting.

● Employ combination strategies with firmonertinib to overcome and prevent resistance to EGFR TKI in NSCLC involving classical mutations.

Acquired resistance presents an inevitable challenge to longer-term EGFRm NSCLC management.

As such, we are evaluating the use of firmonertinib in combination with a signal

transduction inhibitor which we believe has the potential to both treat and prevent acquired resistance.

Our initial combination strategy involves the evaluation of firmonertinib together with a SHP2 inhibitor for use in treating NSCLC patients involving classical EGFRm that have progressed on prior EGFR TKI therapy and we have initiated a Phase 1b clinical trial designed to evaluate the safety, pharmacokinetics and preliminary efficacy of the combination with clinical proof of concept data expected in 2026 in a second-line therapy cohort.

As such, we are evaluating the use of firmonertinib in combination with other potential agents to extend patient benefit in EGFRm NSCLC patients.

Advance novel therapeutic product candidates for unmet medical needs, leveraging innovative platforms and technologies starting with ADCs.

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ADCs are a promising modality for treating cancer due to their ability to target chemotherapy directly to the tumor cells.

We have entered into a research collaboration with Aarvik to leverage its proprietary multi-target, multivalent, site-specific conjugation antibody platform to discover ADCs with potentially improved activity and safety over single target bivalent ADCs.

We identified a preclinical candidate, ARR-002, and have initiated IND-enabling activities.

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We are working with Alphamab to leverage their proprietary linker-payload platform and glycan-conjugation technology to identify novel ADCs for oncology indications globally, except greater China, which includes mainland China, Hong Kong, Macau and Taiwan where Alphamab retains the right to develop and commercialize ADCs.

We entered into a license agreement with Lepu Biopharma pursuant to which Lepu Biopharma granted us a right to develop and commercialize ARR-217, an antibody drug conjugate for gastrointestinal cancers outside greater China, which is mainland China, Hong Kong, Macau and Taiwan.

We entered into a license agreement with Lepu Biopharma pursuant to which Lepu Biopharma granted us a right to develop and commercialize ARR-217, an antibody drug conjugate targeting CDH17 for gastrointestinal cancers outside greater China, which is mainland China, Hong Kong, Macau and Taiwan.

ARR-217 is currently enrolling patients in a Phase 1 clinical study.

This is an open-label, multi-center, Phase 1 study to evaluate the safety, tolerability, efficacy, and pharmacokinetics of ARR-217 (MRG007) in patients with unresectable locally advanced or metastatic solid tumors.

Broaden our pipeline through expanded business development initiatives.

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We remain agnostic as to therapeutic modality, which we believe will expand our access to drug candidates with attractive therapeutic profiles.

In January 2025, we entered into an exclusive license agreement with Lepu Biopharma, pursuant to which Lepu Biopharma granted us a right to develop and commercialize ARR-217, an antibody drug conjugate for gastrointestinal cancers outside greater China, which is mainland China, Hong Kong, Macau and Taiwan.

● Track record of establishing collaborations and license agreements.

Since our inception in 2021, we have established two licensing agreements for oncology assets, two ADC collaborations and a clinical collaboration.

Since our inception in 2021, we have established four licensing agreements for oncology assets, including our lead product candidate firmonertinib, and the ADC product candidates ARR-217, ARR-002, ARR-421 and ARR-173.

We believe in continuing to build our pipeline through sourcing innovation globally and remaining nimble in developing such programs in collaboration with our partners.

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Substantially all NSCLC cases involving an EGFR mutation are non-squamous cell carcinomas.

Numerous EGFR mutations have been identified that cluster within EGFR kinase domain encoded by exons 18-21 in the EGFR gene.

Numerous EGFR mutations have been identified that cluster within the EGFR kinase domain encoded by exons 18-21 in the EGFR gene.

These mutations, which include amino acid insertions, deletions and substitutions, activate EGFR signaling within the tumor cell and can be categorized as either classical or uncommon mutations.

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The distribution of patients with EGFR-mutation positive NSCLC is presented below.

EGFRm are detected by numerous commercially available FDA-approved DNA tests, either PCR or NGS, utilizing either tumor tissue or blood samples and testing for EGFRm, as well as other activating gene mutations, is considered standard in the management of NSCLC as per national guidelines such as NCCN.

EGFRm are detected by numerous commercially available FDA-approved DNA tests, either Polymerase Chain Reaction (PCR) or Next Generation Sequencing (NGS), utilizing either tumor tissue or blood samples and testing for EGFRm, as well as other activating gene mutations, is considered standard in the management of NSCLC as per national guidelines such as National Comprehensive Cancer Network (NCCN).

We are working with a diagnostics company to develop an FDA-approved NGS test for confirming EGFR exon 20 insertion mutations in our clinical trial and if firmonertinib is approved, we believe it would be indicated for patients with EGFRm as detected by an FDA-approved DNA test.

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The ability to treat CNS metastases is of notable importance as patients with EGFR-activating mutations are particularly susceptible to the development of brain metastases, which can occur in up to 70% of patients during the course of their disease.

In 2024, amivantamab in combination with lazertinib was approved for the treatment of first-line NSCLC patients with EGFR Exon 19 deletions or Exon 21 L858R substitution mutations.

Amivantamab is a bispecific antibody and lazertinib is a third-generation

The key limitations of the current standard of care for patients with EGFRm NSCLC involving classical mutations is presented in the graphic below:

In 2024, amivantamab in combination with lazertinib was approved for the treatment of first-line NSCLC patients with EGFR exon 19 deletions

or exon 21 L858R substitution mutations.

Amivantamab is a bispecific antibody and lazertinib is a third-generation EGFR TKI.

The key limitations of therapies in EGFRm NSCLC are presented in the graphic below:

Table includes approved and disclosed registrational indications

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Firmonertinib is currently approved and commercially distributed by Allist in China as a first-line treatment of locally advanced or metastatic NSCLC patients with classical EGFRm as well as pre-treated patients with T790M mutations.

In addition, in an interim data readout from the FAVOUR trial of firmonertinib, reductions in tumor size have been observed in patients with exon 20 insertion mutations and activity in PACC mutations has been observed in an interm data readout from the FURTHER trial.

Exon 20 insertion mutations and PACC mutations are each a subtype of uncommon mutation.

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We anticipate that favorable safety data, if obtained for firmonertinib, could allow for the administration of firmonertinib at different doses required for optimal activity in different EGFRm patient populations.

Encouraging activity has been observed to date in both preclinical and clinical settings, including response in CNS metastases, in an interim data readout from our partner’s ongoing FAVOUR clinical trial in China with patients with exon 20 insertions, and in the ongoing FURTHER clinical trial in patients with PACC mutations globally.

Encouraging activity has been observed to date in both preclinical and clinical settings, including response in CNS metastases, in an interim data readout from our partner’s ongoing FAVOUR clinical trial in China with patients with exon 20 insertions, and in the FURTHER clinical trial in patients with PACC mutations globally.

Of particular note, firmonertinib exhibited elevated tissue distribution to the brain in preclinical studies.

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When mice were administered a single dose of either firmonertinib or osimertinib, they penetrated the blood-brain barrier to a similar extent.

We are currently conducting our FURVENT clinical trial, a global, pivotal Phase 3 clinical trial of firmonertinib in first-line non-squamous locally advanced or metastatic NSCLC patients with exon 20 insertion mutations and we expect topline data in 2025.

In addition, we are conducting our FURTHER trial, a Phase 1b dose escalation and expansion clinical trial in patients with EGFRm NSCLC, including a cohort with PACC mutations which has been fully enrolled, and we presented proof of concept data from this cohort in September 2024.

Subject to review of data and regulatory feedback, we intend to initiate a registrational clinical trial for NSCLC patients with PACC mutations.

Data from our partner’s completed and ongoing clinical trials and from our ongoing clinical trials are discussed below.

We are currently awaiting topline data from our FURVENT clinical trial, a global, pivotal Phase 3 clinical trial of firmonertinib in first-line non-squamous locally advanced or metastatic NSCLC patients with exon 20 insertion mutations and we expect topline data in mid-2026.

In addition, we are conducting our ALPACCA trial, a global, pivotal Phase 3 clinical trial of firmonertinib in first-line non-squamous locally advanced or metastatic NSCLC patients with PACC mutations.

Data from our clinical trials are discussed below.

FURVENT — Our ongoing Phase 3 Clinical Trial in First-Line Non-Squamous Locally Advanced or Metastatic EGFRm NSCLC Patients with Exon 20 Insertion Mutations

We have completed enrolling patients in FURVENT, a global, pivotal Phase 3 clinical trial of firmonertinib in first-line non-squamous locally advanced or metastatic EGFRm NSCLC patients with exon 20 insertion mutations being conducted jointly with Allist.

We designed a randomized clinical trial, integrating feedback from global regulatory agencies, that consists of three cohorts of 125 patients each, designed to evaluate the safety and efficacy of firmonertinib at two different dose levels, 160 mg and 240 mg, administered daily (QD) and compare therapeutic benefit to platinum-based chemotherapy cycles with pemetrexed, the clinical trial’s active control group.

We designed a randomized clinical trial, integrating feedback from global regulatory agencies, that consists of three cohorts, which enrolled 398 patients in total, designed to evaluate the safety and efficacy of firmonertinib at two different dose levels, 160 mg and 240 mg, administered daily (QD) and compare therapeutic benefit to platinum-based chemotherapy cycles with pemetrexed, the clinical trial’s active control group.

Participants in this clinical trial will continue to receive treatment until disease progression or discontinuation related to toxicity, with patients in the control group allowed to cross over into one of the two firmonertinib arms after disease progression.

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Secondary endpoints include ORR, overall survival (OS) and DOR, as well as brain-specific ORR and PFS in patients with brain metastases.

We are currently enrolling patients in this clinical trial and anticipate top-line data for the use of firmonertinib as first-line

therapy in patients with non-squamous locally advanced or metastatic EGFRm NSCLC with exon 20 insertion mutations in 2025.

We have completed enrolling patients in this clinical trial and anticipate top-line data for the use of

firmonertinib as first-line therapy in patients with non-squamous locally advanced or metastatic EGFRm NSCLC with exon 20 insertion mutations in mid-2026.

The FURVENT clinical trial’s study design is illustrated below:

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● Cohort Four is designed to enroll 60 patients with locally advanced or metastatic NSCLC with EGFR PACC mutations who are TKI-treatment naive.

Patients in Cohort Four are randomized into two groups, one group of up to 30 patients to receive 160 mg firmonertinib once-daily and the other group of up to 30 patients administered 240 mg firmonertinib once-daily.

Patients in Cohort Four are randomized into two groups, one group of up to 30 patients administered 160 mg firmonertinib once-daily and the other group of up to 30 patients administered 240 mg firmonertinib once-daily.

Pharmacokinetic data obtained in the first stage of the FURTHER clinical trial as of June 15, 2023 were consistent with initial pharmacokinetic data obtained in the FAVOUR clinical trial as of June 15, 2023 with similar steady-state levels of firmonertinib and its major metabolite, AST5902 (see figure below).

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The FURTHER clinical trial cohort 4 enrolled 60 patients with locally advanced or metastatic NSCLC with EGFR PACC mutations who are TKI-treatment naive.

Patients in Cohort 4 are randomized into two groups, one group of up to 30 patients to receive 160 mg firmonertinib once-daily and the other group of up to 30 patients administered 240 mg firmonertinib once-daily.

Patients in Cohort 4 are randomized into two groups, one group of up to 30 patients administered 160 mg firmonertinib once-daily and the other group of up to 30 patients administered 240 mg firmonertinib once-daily.

Patients with asymptomatic metastases involving the CNS are allowed to participate in the trial.

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Secondary endpoints include DOR sustained, disease controlled without progression of the disease (DCR), PFS, OS and safety.

DOR, PFS and OS are not yet being reported due to the data being immature as of the data cutoff date of June 20, 2024.

Follow-up evaluations with trial participants are conducted every six weeks.

The baseline characteristics of the 60 patients enrolled in the FURTHER clinical trial are

described in the following table.

As of June 20, 2024, initial interim data was available from the 45 patients who have had measurable disease at baseline by blinded independent central review committee (BICR), had received at least two tumor assessments, had progressive disease or death, or discontinued from treatment.

Follow-up evaluations with trial participants are

conducted every six weeks.

The baseline characteristics of the 60 patients enrolled in the FURTHER clinical trial are described in the following table.

As of June 3, 2025, the final data was available from the 45 patients who have had measurable disease at baseline by blinded independent central review committee (BICR), had received at least two tumor assessments, had progressive disease or death, or discontinued from treatment.

Of these 45 patients 22 are in the 240mg first line PACC patient group and 23 are in the 160mg first line PACC patient group.

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Confirmed ORRs by BICR (defined as a reduction in tumor size of at least 30% from the baseline in a patient without evidence of progression) were 68% among the 240mg cohort and 43% among the 160 mg cohort.

Confirmed ORRs by investigator review (defined as a reduction in tumor size of at least 30% from the baseline in a patient without evidence of progression) were 68% among the 240mg cohort and 52% among the 160 mg cohort.

The disease control rate (DCR) by BICR, which includes complete responses, partial responses and stable disease, was 100% and 91% for each of these respective trial cohorts, respectively.

The extent of change in tumor size was evaluated across patients and shown in the figure below.

Median tumor size reduction of 51% was observed in the 240 mg cohort and 34% was observed in the 160 mg cohort.

The DCR by BICR, which includes complete responses, partial responses and stable disease, was 100% and 91% for each of these respective trial cohorts, respectively.

The extent of change in tumor size was evaluated across patients and shown in the figure below taken from the final data cut of June 3, 2025.

Patients on average achieved greater tumor size reduction in the 240 mg dose versus the 160 mg dose.

Tumor size reduction was observed across the different PACC mutations (frequent, less frequent, single and compound mutations).

Tumor size reduction was observed across the different PACC mutations such as frequent (G719X, S78I), less frequent (other), single and compound mutations.

Tumor size reduction was observed in different PACC mutations with firmonertinib

Responses occurred rapidly with most patients having a partial response by the first tumor assessment at six weeks with responses ongoing in many patients as denoted in the figure below.

The confirmed median PFS as of the final data cut of June 3, 2025 was 16.0 months at the 240mg dose and 11.1 months at the 160mg dose.

The median duration of follow-up was 16.5 months.

Confirmed CNS responses were also observed in response evaluable patients as denoted in the figure below.

Firmonertinib was observed to be generally well tolerated and demonstrated safety results similar to prior firmonertinib trials.

The table below summarizes this safety and tolerability data collected in the FURTHER trial as a whole and in Cohort 4 specifically, as of July 5, 2024:

FAVOUR — Ongoing Phase 1b Clinical Trial in NSCLC Patients with EGFR Exon 20 Insertion Mutations

The table below summarizes this safety and tolerability data collected in the FURTHER trial as a whole and in Cohort 4 specifically, as of the final data cut date of June 3, 2025:

FAVOUR — Phase 1b Clinical Trial in NSCLC Patients with EGFR Exon 20 Insertion Mutations

The FAVOUR clinical trial is a 90-patient, Phase 1b clinical trial being conducted by Allist in China that is intended to assess the safety and efficacy of firmonertinib in locally advanced or metastatic NSCLC patients who have EGFR exon 20 insertion mutations.

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The primary endpoint of this trial is ORR as measured by the RECIST version 1.1 by BICR.

Secondary endpoints include DOR sustained, disease controlled without progression of the disease (DCR), PFS, overall survival (OS) and safety.

OS is not reported due to the

data being immature as of the data cutoff date of June 15, 2023.

Secondary endpoints include DOR, DCR, PFS,

overall survival (OS) and safety.

OS is not reported due to the data being immature as of the data cutoff date of June 15, 2023.

Follow-up evaluations with trial participants are conducted every six weeks.

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Median PFS was 10.7 months in the treatment-naïve patients, 7.0 months in the 240 mg previously treated cohort and 5.7 months in the 160 mg previously treated cohort.

The disease control rate (DCR), which includes complete responses, partial responses and stable disease, was 100%, 92% and 85% for each of these respective trial cohorts.

The DCR, which includes complete responses, partial responses and stable disease, was 100%, 92% and 85% for each of these respective trial cohorts.

These initial interim PFS and DOR data continue to mature with patients continuing in the clinical trial.

Interim data from clinical trials may change as more patient data becomes

available and are subject to audit and verification procedures that could result in material changes in the final data.

Interim data from clinical trials may change as more patient data becomes available and are subject to audit

and verification procedures that could result in material changes in the final data.

The following table describes these data.

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The majority of these AEs were grade 1 or 2 and commonly associated with EGFR TKIs as a class.

SHP2 Combination Trial — Ongoing Phase 1b Clinical Trial in Classical EGFRm NSCLC Patients

SHP2 is a protein tyrosine phosphatase that has an important role in the signaling pathway downstream of EGFR.

Activating SHP2 mutations have been observed in breast, lung and colorectal cancers, among others, which underscores the central role of SHP2 in tumor cells in transmitting growth signals to rat sarcoma RAS.

Besides transmitting growth signals from EGFR, SHP2 also participates in signal transduction processes of other receptor tyrosine kinase that can confer resistance to EGFR TKI such as MET.

In preclinical EGFRm NSCLC tumor xenograft models small molecular inhibitors of SHP2 can further potentiate the activity of EGFR TKI and overcome resistance conferred by activated MET signaling.

We therefore believe that the combination of firmonertinib with a potent and specific SHP2 inhibitor may further potentiate firmonertinib activity and prevent the outgrowth of resistant tumor cells.

We have therefore initiated a Phase 1b clinical trial combining firmonertinib with a SHP2 inhibitor ICP-189 being developed by InnoCare.

Preclinical studies with osimertinib demonstrated the encouraging activity of this combination in preventing resistance from

developing and we believe similar results will be obtained in preclinical studies with firmonertinib based on a common mechanism of action.

The Phase 1b trial is designed to evaluate the safety, pharmacokinetics and preliminary efficacy of the firmonertinib-ICP-189 combination in EGFRm NSCLC involving classical mutations and we dosed the first patient in March 2024.

An expansion cohort in patients previously treated with a third-generation EGFR TKI is initiated with potential proof of concept in second-line classical EGFRm NSCLC expected to become available in 2026 .

Planned Adjuvant Study in NSCLC Patients with Uncommon EGFRm, including Exon 20 Insertion and PACC Mutations

We intend to participate in a single multicenter, randomized, global pivotal Phase 3 clinical trial initiated in China to investigate the potential benefit of firmonertinib when administered to adult patients with Stage Ib to IIIb EGFRm NSCLC, after surgical resection.

We intend to participate in, or to conduct in parallel, a single multicenter, randomized, global pivotal Phase 3 clinical trial initiated in China to investigate the potential benefit of firmonertinib when administered to adult patients with Stage IIB to IIIB EGFRm NSCLC, after surgical resection.

Eligibility in this clinical trial is restricted to patients with uncommon EGFR mutations including PACC, exon 20 insertion and other uncommon EGFRm.

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Participants are to receive firmonertinib daily over a three-year period with the primary endpoint to be disease free survival with secondary endpoints being OS and safety.

We intend to participate in this clinical trial in 2026.

We intend to participate in, or to conduct in parallel, this clinical trial in 2026, depending on the timing and results of the FURVENT study.

FURLONG — Completed Phase 3 Clinical Trial in Classical EGFRm First-Line NSCLC Patients

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In addition, target expression tends to be heterogeneous within a tumor which can limit efficacy and/or restrict ADC use to only tumors that have high and mostly homogenous target expression.

Our Lepu Biopharma Antibody Drug Conjugate Collaboration

We also established an ADC collaboration with Lepu Biopharma in January 2025, to gain ex-Greater China, which includes mainland China, Hong Kong, Macau and Taiwan, rights to ARR-217, a CDH-17 targeted ADC, with the potential to treat gastrointestinal malignancies.

Our Aarvik Antibody Drug Conjugate Collaboration

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Our Lepu Biopharma Antibody Drug Conjugate Collaboration

We also established an ADC collaboration with Lepu Biopharma in January 2025, to gain ex-Greater China, which includes mainland China, Hong Kong, Macau and Taiwan, rights to ARR-217.

Licenses, Partnerships and Collaborations

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The parties have appointed a joint collaboration committee, comprised of representatives from both us and Allist (the Collaboration Committee), to oversee the parties’ development activities related to the Licensed Compound under the Allist License Agreement.

approvals in all major market countries in the Licensed Territory and (iii) perform all commercialization activities for the Licensed Product in each country in the Licensed Territory in which the Licensed Product has received regulatory approval, and we are solely responsible for all the expenses associated with the foregoing (i)-(iii).

Under the terms of the Allist License Agreement, we are required to use commercially reasonable efforts to (i) develop the Licensed Product in the Licensed Territory, (ii) prepare and submit regulatory filings and seek regulatory approvals in all major market countries in the Licensed Territory and (iii) perform all commercialization activities for the Licensed Product in each country in the Licensed Territory in which the Licensed Product has received regulatory approval, and we are solely responsible for all the expenses associated with the foregoing (i)-(iii).

We are also responsible for the manufacture and supply of the Licensed Product in the Licensed Territory, by ourselves or through a third-party manufacturer, for the purpose of conducting clinical trials, obtaining regulatory approval and commercializing the Licensed Product in the Licensed Territory.

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As additional consideration for the licenses and rights granted to us under the Allist License Agreement, we made an upfront, non-creditable and non-refundable cash payment of $40.0 million to Allist on the Effective Date (the Initial Payment).

We are obligated to make development and regulatory approval milestone payments to Allist upon the achievement of specific milestone events related to the Licensed Product in an aggregate amount up to $110.0 million, and commercial milestone payments to Allist upon the achievement of specified net sales thresholds of the Licensed Product in an aggregate amount up to $655.0 million.

In 2023, we made a $5.0 million payment to Allist after we met a clinical milestone under the Allist License Agreement.

We are obligated to make development and regulatory approval milestone payments to Allist upon the achievement of specific milestone events related to the Licensed Product in an aggregate amount up to $110.0 million, of which we have paid $5.0 million upon meeting certain clinical milestones, and commercial milestone payments to Allist upon the achievement of specified net sales thresholds of the Licensed Product in an aggregate amount up to $655.0 million.

We are also obligated to pay Allist tiered royalties ranging from high single digits to low mid-teens percentages on an incremental aggregated net sales basis on the net sales of any Licensed Products in the Licensed Territory made by or on behalf of us or our sublicensees.

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Pursuant to the Allist Collaboration Agreement, if either party or both parties wish to jointly conduct a Global Study, one or both parties (as the case may be) shall prepare and submit the proposed strategy, protocol design, budget, proposal for budget sharing and internal process timeline for such proposed Global Study to the Collaboration Committee that was appointed pursuant to the Allist License Agreement for its review at least 90 days in advance of the applicable protocol filing with the relevant regulatory authorities.

Upon the approval by the collaboration committee of a development plan for the proposed Global Study that includes the allocation of the sponsorship for the conduct of the proposed Global Study and other details for conducting the proposed Global Study as specified in the Allist Collaboration Agreement, the proposed Global Study shall be deemed to be a “Joint Global Study” and such development plan a “Joint Global Development Plan.” The parties have agreed in the Allist Collaboration Agreement that (i) we will be the sponsor of any Joint Global Study in the Licensed Territory (as defined in the Allist License Agreement), (ii) Allist will be the sponsor of any Joint Global Study in the PRC (as defined in the Allist License Agreement) and (iii) the Collaboration Committee shall designate the party that

will be the sponsor of any Joint Global Study in certain territories (Joint Territories).

Territory (as defined in the Allist License Agreement), (ii) Allist will be the sponsor of any Joint Global Study in the PRC (as defined in the Allist License Agreement) and (iii) the Collaboration Committee shall designate the party that will be the sponsor of any Joint Global Study in certain territories (Joint Territories).

The parties will mutually agree on the global regulatory strategy for each Joint Global Study.

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Under the Amended and Restated Aarvik Collaboration Agreement, we are required to pay Aarvik a collaboration execution fee and research fees as provided in the SOWs.

We have incurred $4.9 million of fees through December 31, 2024.

We further agreed to reimburse Aarvik for actual costs incurred in procuring other materials Aarvik will actually use in performing activities under the applicable SOWs.

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The Alphamab Agreement is subject to termination (i) by either party for customary purposes, including the material breach by or bankruptcy of the other party, (ii) by us for convenience, (iii) by Alphamab due to our challenge of certain patents held by Alphamab, or (iv) by Alphamab on a product-by-product basis due to our failure to carry out certain diligence obligations or in certain instances if there is a change of control of the Company.

Lepu Agreement

On January 21, 2025, ArriVent entered into an Exclusive License Agreement (the “Lepu Biopharma Agreement”) with Lepu Biopharma, pursuant to which Lepu Biopharma granted us a right to develop and commercialize ARR-217 (MRG007), an antibody drug conjugate for gastrointestinal cancers outside greater China, which is mainland China, Hong Kong, Macau and Taiwan.

Lepu Biopharma Agreement

On January 21, 2025, we entered into the Lepu Biopharma License Agreement with Lepu Biopharma, pursuant to which Lepu Biopharma granted us a right to develop and commercialize ARR-217 (MRG007), an antibody drug conjugate for gastrointestinal cancers, outside greater China, which is mainland China, Hong Kong, Macau and Taiwan.

Under the Lepu Biopharma Agreement, Lepu Biopharma granted to us:

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Currently, we rely on, and have agreements with, two third-party contract manufacturers, Zhejiang Raybow Pharmaceutical Company., Ltd.

(Raybow) and WuXi SynTheAll Pharmaceutical company, Ltd.

(Raybow) and SynTheAll Pharmaceutical Co., Ltd.

(WuXi STA) to supply the drug substance for firmonertinib to be used in on-going and planned clinical trials and with WuXi STA, with whom we have executed technology transfer related to the manufacture of drug product, to manufacture the clinical trial and initial commercial launch supplies of firmonertinib drug product.

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We intend to also maintain the current drug substance manufacturer as part of our supply chain strategy.

The BIOSECURE Act was introduced in 2024 but has not become law, and there remains potential for its reintroduction in the current congressional session.

If reintroduced and enacted, the legislation would prohibit federal agencies from entering into procurement contracts with an entity that uses biotechnology equipment or services from a biotechnology company of concern.

The National Defense Authorization Act for Fiscal Year 2026, enacted in December 2025, included a section titled “Prohibition on Contracting with Certain Biotechnology Providers,” aimed at discouraging federal contracting with certain biotechnology companies for biotechnology equipment or services in China and other countries of concern, also known as the BIOSECURE Act.

The statute prohibits federal executive agencies from procuring any biotechnology equipment or service from a biotechnology company of concern or contracting with any such company or any entity that procures or uses equipment or services from a biotechnology company of concern.

Such prohibitions may limit our ability to obtain federal government grants for research involving our products, if approved, or product candidates manufactured in China or to enter contracts to sell any such products, if approved, to the federal government.

Accordingly, we are taking measures to strengthen our supply chain in the event that Raybow, WuXi STA, or one of our other manufacturers is impacted.

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The biotechnology and pharmaceutical industries have made substantial investments in recent years into the rapid development of novel treatments for NSCLC.

We face substantial competition from multiple sources, including large and specialty pharmaceutical and biotechnology companies, academic research institutions and governmental agencies and public and private research institutions.

institutions.

Our competitors compete with us on the level of the technologies employed, or on the level of development of product candidates.

In addition, many small biotechnology companies have formed collaborations with large, established companies to (i) obtain support for their research, development and commercialization of products or (ii) combine several treatment approaches to develop longer lasting or more efficacious treatments that may potentially

directly compete with our current or future product candidates.

We anticipate that we will continue to face increasing competition as new therapies and combinations thereof, technologies and data emerge within the field of oncology and, furthermore, within the treatment of NSCLC.

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We will also seek to rely on regulatory protection afforded through inclusion in expedited development and review, data exclusivity, market exclusivity and patent term extensions where available.

Firmonertinib

Through our licensor Allist, we have sought and obtained patent protection in the United States and internationally related to firmonertinib.

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With regard to the firmonertinib molecule itself, we exclusively license from Allist two issued patents in the US, and one issued patent in each of, Canada, Europe, Japan and South Korea.

These patents are expected to expire in 2035, assuming payment of all appropriate maintenance, renewal, annuity or other governmental fees, and without taking potential patent term extensions or adjustments into account.

These patents are expected to expire as

indicated in the table below (composition of matter).

patents are reissue patents covering firmonertinib, other compounds represented by a general formula, and pharmaceutically acceptable salts of such compounds.

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With regard to crystalline forms, we also exclusively license from Allist one issued patent in each of the United States, Canada, Europe, Japan and South Korea.

patent covers two crystaline forms of mesylate salts of firmonertinib, as well as pharmaceutical compositions, methods for preparing such crystaline forms, and methods for treating a patient suffering from cancers.

Collectively, these patents are expected to expire in 2037, assuming payment of all appropriate maintenance, renewal, annuity or other governmental fees and without taking potential patent term extensions or adjustments into account.

patent covers two crystalline forms of mesylate salts of firmonertinib, as well as pharmaceutical compositions, methods for preparing such crystalline forms, and methods for treating a patient suffering from cancers.

Collectively, these patents are expected to expire as indicated in the table below (composition of matter).

With regards to methods of manufacturing firmonertinib, we exclusively license from Allist two pending patent applications in each of the United States and Europe.

These applications, if issued, would expire in 2043.

With regard to methods of use for firmonertinib, we exclusively license from Allist one Patent Cooperation Treaty (PCT) patent application and two families of national stage patent applications pending in multiple countries, including United States, Canada, Europe, Japan and South Korea.

The national stage patent application families relate to use of firmonertinib to treat disease, such as NSCLC, in patients (a) having exon 20 insertion mutations or (b) having an HER2 exon 20 insertion mutation and/or EGFR rare mutation, respectively, as well as pharmaceutical compositions containing therapeutically effective amounts of firmonertinib.

The PCT application relates to use of firmonertinib to treat disease, such as NSCLC, in patients having EGFR PACC mutations, as well as pharmaceutical compositions containing therapeutically effective amounts of firmonertinib.

or foreign patents issued from the national stage applications or the national stage filings of the PCT patent application are expected to expire in 2042 (pending national stage application) or 2043 (PCT patent application), assuming payment of all appropriate maintenance, renewal, annuity or other governmental fees and that national phase entries are timely made based upon the pending PCT applications and without taking potential patent term extensions or adjustments into account.

Aarvik has exclusively licensed us one PCT application relating to antibody scaffolds and ADCs comprising such scaffolds and two U.S.

provisional applications relating to antibody scaffolds and ADCs comprising such scaffolds, including ARR-002.

or foreign patents issued from national stage filings of the PCT patent application, and any U.S.

or foreign patents issued from the provisional application, are expected to expire in 2044 and 2045, respectively, without taking into account any patent term adjustments or extensions.

These applications, if issued, would expire as indicated in the table below (composition of matter).

With regard to methods of use for firmonertinib, we exclusively license from Allist three families of national stage patent applications pending in multiple countries, including United States, Canada, Europe, Japan and South Korea.

The national stage patent application families relate to use of firmonertinib to treat disease, such as NSCLC, in patients (a) having exon 20 insertion mutations or (b) having an HER2 exon 20 insertion mutation and/or EGFR rare mutation and (c) use of firmonertinib to treat disease, such as NSCLC, in patients having EGFR PACC mutations, , as well as pharmaceutical compositions containing therapeutically effective amounts of firmonertinib..

or foreign patents issued from the national stage applications are expected to expire as indicated in the table below (composition of matter).

Lepu Biopharma has exclusively licensed us two PCT applications relating to antibodies and ADCs, including ARR-217.

or foreign patents issued from such application are expected to expire in 2045, without taking into account any patent term adjustments or extensions.

or foreign patents issued from such PCT applications are expected to expire as indicated in the table below (composition of matter).

Aarvik has exclusively licensed us one PCT application relating to antibody scaffolds and ADCs comprising such scaffolds and one PCT application relating to antibody scaffolds and ADCs comprising such scaffolds, including ARR-002.

or foreign patents issued from national stage filings of the PCT patent applications, are expected to expire as indicated in the table below (composition of matter).

We are joint owners with Alphamab of a provisional application relating to the ARR-421 program and Alphamab has exclusively licensed us a PCT application relating to camptothecin derivatives and conjugates comprising the same.

or foreign patents issued from such applications are expected to expire as indicated in the table below (compositio n of matter).

We may file additional patent applications in support of current and new product candidates as well as new platform and core technologies.

Patent Expiration Dates* of Product Candidates Patents/Applications by Category

Product Candidate

Composition of Matter

Methods of Treatment

Manufacturing

Firmonertinib

*Expiration dates of granted patents assume payment of all appropriate maintenance, renewal, annuity or other governmental fees.

Expiration dates of pending patent applications assume grant of the patent applications.

The expiration dates in this table do not take into account potential patent term extensions or adjustments.

Actual expiration dates may vary based on interpretations of laws in the jurisdictions in which patents have granted.

Our commercial success will depend in part on obtaining and maintaining patent protection and trade secret protection of our current and future product candidates and the methods used to develop and manufacture them, as well as successfully defending any such patents against third-party challenges and operating without infringing on the proprietary rights of others.

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In the United States, a patent’s term may be lengthened by patent term adjustment, which compensates a patentee for administrative delays by the U.S.

Patent and Trademark Office (USPTO), in

examining and granting a patent, or may be shortened if a patent is terminally disclaimed over an earlier filed patent.

Patent and Trademark Office (USPTO), in examining and granting a patent, or may be shortened if a patent is terminally disclaimed over an earlier filed patent.

In the United States, the term of a patent that covers an FDA-approved drug may also be eligible for extension, which permits patent term restoration as compensation for the patent term lost during the FDA regulatory review process.

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non-provisional applications and PCT applications may claim the benefit of the priority date of earlier filed provisional applications, when applicable.

The PCT system allows a single application to be filed within 12 months of the original priority date of the patent application, and to designate all of the PCT member states in which national patent applications can later be pursued based on the international patent application filed under the PCT.

The PCT system allows a single application to be filed within 12 months of

the original priority date of the patent application, and to designate all of the PCT member states in which national patent applications can later be pursued based on the international patent application filed under the PCT.

The PCT searching authority performs a patentability search and issues a non-binding patentability opinion which can be used to evaluate the chances of success for the national applications in foreign countries prior to having to incur the filing fees.

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Third-party patents could require us to alter our development or commercial strategies, or our products or processes, obtain licenses or cease certain activities.

Our breach of any license agreements or our failure to obtain a license to proprietary rights required to develop or commercialize our future products may have a material adverse impact on us.

Our breach of any license agreements or our failure to obtain a license to proprietary rights required to develop or commercialize our future products may have a material

adverse impact on us.

If third parties prepare and file patent applications in the United States that also claim technology to which we have rights, we may have to participate in interference or derivation proceedings in the USPTO to determine priority of invention.

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FDA Approval Process

In the United States, pharmaceutical products are subject to extensive regulation by the FDA under the Federal Food, Drug, and Cosmetic Act (FDC Act), its implementing regulations and Biological products are regulated under the FDC Act, the Public Health Service Act (PHS Act), and their implementing regulations.

In the United States, pharmaceutical products are subject to extensive regulation by the FDA under the Federal Food, Drug, and Cosmetic Act (FDC Act) and its implementing regulations and while biological products are regulated under the FDC Act, the Public Health Service Act (PHS Act), and their implementing regulations.

Both drugs and biologics also are subject to other federal, state and local statutes and regulations.

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typically involves nonclinical laboratory, animal tests and formulation studies conducted according to good laboratory practices and other applicable regulations and guidance;

the submission to the FDA of an IND which must become effective before clinical testing may commence and adequate and well-controlled clinical trials to establish the safety and effectiveness of the drug for each indication for which FDA approval is sought.

the submission to the FDA of an IND which must become effective before clinical testing may commence;

and completion of adequate and well-controlled clinical trials to establish the safety and effectiveness of the drug for each indication for which FDA approval is sought.

Satisfaction of FDA pre-market approval requirements typically takes many years and the actual time required may vary substantially based upon the type, complexity and novelty of the product or disease.

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A 30-day waiting period after the submission of each IND is required prior to the commencement of clinical testing in humans.

If the FDA has not otherwise notified the sponsor of the IND within this 30-day period, then the clinical trial proposed in the IND may begin, unless the FDA, within the 30-day time period, imposes a clinical hold.

If the FDA has not otherwise notified the sponsor of the IND within this 30-day period, then the clinical trial proposed in

the IND may begin, unless the FDA, within the 30-day time period, imposes a clinical hold.

In such a case, the IND sponsor and the FDA must resolve any outstanding concerns before the clinical trial can begin.

Clinical holds also may be imposed by the FDA at any time before or during clinical trials due to safety concerns about ongoing or proposed clinical trials or non-compliance with specific FDA requirements, and the trials may not begin or continue until the FDA notifies the sponsor that the hold has been lifted.

Clinical trials involve the administration of the investigational new drug to healthy volunteers or patients under the supervision of one or more qualified investigators.

Clinical trials involve the administration of the investigational new drug or biologic to healthy volunteers or patients under the supervision of one or more qualified investigators.

Clinical trials must be conducted:

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The FDA or the sponsor may suspend a clinical trial at any time on various grounds, including a finding that the research subjects or patients are being exposed to an unacceptable health risk.

Similarly, an IRB may also require the clinical trial at the site to be halted, either temporarily or permanently (or impose

other conditions), for failure to comply with the IRB’s requirements or if the drug has been associated with unexpected serious harm to patients.

Similarly, an IRB may also require the clinical trial at the site to be halted, either temporarily or permanently (or impose other conditions), for failure to comply with the IRB’s requirements or if the drug has been associated with unexpected serious harm to patients.

In addition, some clinical trials are overseen by an independent group of qualified experts organized by the sponsor, known as a data safety monitoring board or committee.

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In most cases, the FDA requires two adequate and well-controlled clinical trials to demonstrate the efficacy of the therapeutic product candidate.

Results from a single adequate and well-controlled trial may be sufficient in rare instances, such as:

Results from a single adequate and well-controlled trial may be sufficient in certain instances, such as:

(i) where the study is a large multicenter trial demonstrating internal consistency and a statistically very persuasive finding of a clinically meaningful effect on mortality, irreversible morbidity or prevention of a disease with a potentially serious outcome and confirmation of the result in a second trial would be practically or ethically impossible;

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In addition, appropriate packaging must be selected and tested, and stability studies must be conducted to demonstrate that the product candidate does not undergo unacceptable deterioration over its shelf life.

Assuming successful completion of the required clinical testing, an NDA or BLA is prepared and submitted to the FDA.

FDA approval of the NDA or BLA is required before marketing of the product may begin in the U.S.

The NDA or BLA must include the results of all product development, nonclinical, clinical and other testing, a compilation of data relating to the product’s pharmacology, chemistry, manufacture and controls, along with proposed labeling and other relevant information.

The cost of preparing and submitting an NDA or BLA is substantial.

The submission of most prescription drug marketing applications is additionally subject to a substantial application user fee, and the applicant under an approved NDA or BLA is also subject to an annual program fee for each prescription product.

Assuming successful completion of the required clinical testing, an NDA/BLA is prepared and submitted to the FDA.

FDA approval of the NDA/BLA is required before marketing of the product may begin in the U.S.

The NDA/BLA must include the results of all product development, nonclinical, clinical and other testing, a compilation of data relating to the product’s pharmacology, chemistry, manufacture and controls, along with proposed labeling and other relevant information.

The cost of preparing and submitting an NDA/BLA is substantial.

The submission of most prescription drug marketing applications is additionally subject to a substantial application user fee, and the applicant under an approved NDA/BLA is also subject to an annual program fee for each prescription product.

These fees are typically increased annually.

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Congress is required to re-authorize the agency’s user fee programs every five years, and current legislative provisions supporting the prescription drug-specific program are set to expire on September 30, 2027.

The FDA has 60 days from its receipt of an NDA or BLA to determine whether the application will be filed based on the agency’s threshold determination that it is sufficiently complete to permit substantive review.

The FDA may request additional information rather than accept an NDA or BLA for filing.

In this event, the NDA or BLA must be resubmitted with the additional information.

The FDA has 60 days from its receipt of an NDA/BLA to determine whether the application will be filed based on the agency’s threshold determination that it is sufficiently complete to permit substantive review.

The FDA may request additional information rather than accept an NDA/BLA for filing, in which case the FDA may issue a Refuse to File letter.

In this event, the NDA/BLA must be resubmitted with the additional information.

The resubmitted application also is subject to review before the FDA accepts it for filing.

Once the submission is filed, the FDA begins an in-depth review to determine, among other things, whether a product is safe and effective for its intended use and whether its manufacturing is cGMP-compliant to assure and preserve the product’s identity, strength, quality and purity.

If the submission is filed, the FDA begins an in-depth review to determine, among other things, whether a product is safe and effective for its intended use and whether its manufacturing is cGMP-compliant to assure and preserve the product’s identity, strength, quality and purity.

Under the Prescription Drug User Fee Act (PDUFA) guidelines that are currently in effect, the FDA has a goal of ten months from the date of “filing” of a standard NDA for a new molecular entity, or an original BLA, to review and act on the submission.

This review typically takes twelve months from the date the NDA or BLA is submitted to FDA because the FDA has approximately two months to make a “filing” decision after it the application is submitted.

Most applications for priority review products are reviewed in six months of the date of the FDA’s filing determination.

This review typically takes twelve months from the date the NDA/BLA is submitted to FDA because the FDA has approximately two months to make a “filing” decision after it the application is submitted.

Most applications designated for priority review products are reviewed in six months of the date of the FDA’s filing determination.

Priority review can be applied to NDAs or BLAs for products that are designed to treat a serious condition, where the FDA determines the product may offer significant improvements in safety or effectiveness or provide a treatment where no adequate therapy exists.

The review process for both standard and priority review may be extended by the FDA on one occasion for three additional months to consider a “major amendment,” which may include certain late-submitted information, or information intended to clarify information already provided in the submission.

The FDA may also refer applications for novel drug products, or drug products that present difficult questions of safety or efficacy, to an independent advisory committee — typically a panel that includes clinicians and other scientific experts — for review, evaluation and a recommendation as to whether the application should be approved and under what conditions.

The FDA may also refer applications for novel drug/biologic products, or drug/biologic products that present difficult questions of safety or efficacy, to an independent advisory committee — typically a panel that includes clinicians and other scientific experts — for review, evaluation and a recommendation as to whether the application

should be approved and under what conditions.

The FDA is not bound by the recommendation of an advisory committee, but it generally follows such recommendations.

Before approving an NDA or BLA, the FDA may inspect one or more clinical sites to assure that the trials support the application were conducted compliance with GCP.

Before approving an NDA/BLANDA/BLA, the FDA may inspect one or more clinical sites to assure that the trials support the application were conducted compliance with GCP.

Additionally, the FDA will typically inspect the facility or the facilities at which the product is manufactured to assess compliance with cGMPs, to assure that the facilities, methods and controls are adequate to preserve the drug’s identity, strength, quality and purity and, for a biologic, its potency.

The FDA will not approve the product unless compliance with cGMP, is satisfactory and the NDA or BLA contains data that provide substantial evidence that the product is safe and effective in the indication sought.

After the FDA evaluates the NDA or BLA and the manufacturing facilities, it issues either an approval letter or a complete response letter (CRL).

The FDA will not approve the product unless compliance with cGMP, is satisfactory and the NDA/BLA contains data that provide substantial evidence that the product is safe and effective in the indication sought.

After the FDA evaluates the NDA/BLA and the manufacturing facilities, it issues either an approval letter or a complete response letter (CRL).

A CRL indicates that the review cycle of the application is complete, and the application will not be approved in its present form.

A CRL usually describes the specific deficiencies in the submission and may require additional clinical data, such as an additional clinical trial or other significant and time-consuming requirements related to clinical trials, nonclinical studies or manufacturing, in order for the FDA to reconsider the application.

If a CRL is issued, the sponsor must resubmit the NDA or BLA, addressing all of the deficiencies identified in the letter, or withdraw the application.

Even if such data and information are submitted, the FDA may decide that the NDA or BLA does not satisfy the criteria for approval.

In September 2025, the FDA began publishing CRLs soon after issuing them to the respective sponsors, breaking with long standing agency tradition of publishing CRLs with approval documentation after the product is approved.

If a CRL is issued, the sponsor must resubmit the NDA/BLA, addressing all of the deficiencies identified in the letter, or withdraw the application.

Even if such data and information are submitted, the FDA may decide that the NDA/BLA does not satisfy the criteria for approval.

The FDA has committed to reviewing such resubmissions in two or six months depending on the type of information included.

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If a product receives regulatory approval, the approval may be significantly limited to specific diseases and dosages or the indications for use may otherwise be limited, which could restrict the commercial value of the product.

As a condition of NDA or BLA approval, the FDA may also require a risk evaluation and mitigation strategy (REMS) to help ensure that the benefits of the drug outweigh the potential risks.

As a condition of NDA/BLA approval, the FDA may also require a risk evaluation and mitigation strategy (REMS) to help ensure that the benefits of the drug outweigh the potential risks.

If the FDA concludes a REMS is needed, the sponsor of the marketing application must submit a proposed REMS.

The FDA will not approve the NDA or BLA without an approved REMS, if required.

The FDA will not approve the NDA/BLA without an approved REMS, if required.

REMS can include medication guides, communication plans for healthcare professionals, and elements to assure safe use (ETASU).

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The requirement for a REMS can materially affect the potential market and profitability of the product.

Moreover, product approval may require substantial post-approval testing and surveillance to monitor the product’s

safety or efficacy.

Moreover, product approval may require substantial post-approval testing and surveillance to monitor the product’s safety or efficacy.

Once granted, product approvals may be withdrawn if compliance with regulatory standards is not maintained or problems are later identified.

Changes to some of the conditions established in an approved application, including changes in indications, labeling, or manufacturing processes or facilities, require submission and FDA approval of a new NDA or BLA or NDA/BLA supplement before the change can be implemented.

Changes to some of the conditions established in an approved application, including changes in indications, labeling, or manufacturing processes or facilities, require submission and FDA approval of a new NDA/BLA or NDA/BLA supplement before the change can be implemented.

A supplement seeking a new indication typically requires clinical data similar to that in the original application, and the FDA uses the same procedures and actions in reviewing efficacy supplements as it does in reviewing original NDAs and BLAs.

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Failure to timely register a covered clinical study or to submit study results as provided for in the law can give rise to civil monetary penalties and also prevent the non-compliant party from receiving future grant funds from the federal government.

The NIH’s Final Rule on ClinicalTrials.gov registration and reporting requirements became effective in 2017, and the government has begun enforcing those requirements against non-compliant clinical trial sponsors.

Pediatric Information

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Congress periodically considers enacting new incentives or mandates applicable to pediatric drug development, and the regulatory requirements applicable to pediatric drug developers may change in the future.

For example, bipartisan legislation introduced in 2023 in the House of Representatives would have increased funding for pediatric trials;

mandated that drugs for rare diseases be studied in children;

and granted FDA authority to assess penalties against companies that do not complete required pediatric studies.

For example, in February 2026, bipartisan legislation was signed into law and will grant FDA authority to assess penalties against companies that do not complete required pediatric studies.

Expedited Development and Review Programs & Accelerated Approval Pathway

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Specifically, investigational products are eligible for Fast Track designation if they are intended to treat a serious or life-threatening disease or condition and demonstrate the potential to address unmet medical needs for the disease or condition.

The sponsor of a Fast Track product candidate has opportunities for more frequent interactions with the applicable FDA review team during product development and, once an NDA or BLA is submitted, the application may be eligible for priority review.

With regard to a Fast Track product candidate, the FDA may consider for review sections of the marketing application on a rolling basis before the complete application is submitted, if the sponsor provides a schedule for the submission of the sections of the application, the FDA agrees to accept sections of the application and determines that the schedule is acceptable, and the sponsor pays any required user fees upon submission of the first section of the NDA or BLA.

The sponsor of a Fast Track product candidate has opportunities for more frequent interactions with the applicable FDA review team during product development and, once an NDA/BLA is submitted, the application may be eligible for priority review.

A product candidate intended to treat a serious or life-threatening disease or condition may also be eligible for breakthrough therapy designation to expedite its development and review.

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Any product candidate submitted to the FDA for approval, including a product candidate with a Fast Track designation or breakthrough designation, may also be eligible for other types of FDA programs intended to expedite development and review, such as priority review and accelerated approval.

An NDA or BLA is eligible for priority review if the product candidate is designed to treat a serious condition, and if approved, would provide a significant improvement in safety or efficacy compared to available therapies.

The FDA endeavors to review applications with priority review designations within six months of the filing date as compared to ten months for review of new molecular entity NDAs or original BLAs under its current PDUFA review goals.

An NDA/BLA is eligible for priority review if the product candidate is designed to treat a serious condition, and if approved, would provide a significant improvement in safety or efficacy compared to available therapies.

The FDA endeavors to review applications with

priority review designations within six months of the filing date as compared to ten months for review of new molecular entity NDAs or original BLAs under its current PDUFA review goals.

In 2025, the FDA created a new voucher program called the Commissioner’s National Priority Voucher (CNPV), with the goal of radically expediting therapeutic product review and approval processes.

The agency may award a CNPV to a company or a specific product candidate that demonstrates alignment with certain national health priorities.

The FDA aims to take action on a marketing application for which a CNPV is used within one to two months after the filing date.

In addition, a product candidate may be eligible for accelerated approval.

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In addition, the FDA currently requires as a condition of accelerated approval pre-approval of promotional materials, which could adversely impact the timing of the commercial launch of the product.

Fast Track designation, breakthrough therapy designation, priority review, and accelerated approval do not change the standards for approval but may expedite the development or approval process.

None of these FDA programs change the standards for approval but they may expedite the development or approval process.

Even if a product candidate qualifies for one or more of these programs, the FDA may later decide that the product no longer meets the conditions for qualification or decide that the time period for FDA review or approval will not be shortened.

Post-Approval Requirements

Once an NDA or BLA is approved, a product will be subject to pervasive and continuing regulation by the FDA, including, among other things, requirements relating to record-keeping, reporting of adverse experiences, periodic reporting, product sampling and distribution, and advertising and promotion of the product.

For instance, the FDA

closely regulates the post-approval marketing, labeling, advertising and promotion of drugs, including through its enforcement of standards and regulations for direct-to-consumer advertising, industry-sponsored scientific and educational activities and promotional activities involving the internet.

Once an NDA/BLA is approved, a product will be subject to pervasive and continuing regulation by the FDA, including, among other things, requirements relating to record-keeping, reporting of adverse experiences, periodic reporting, product sampling and distribution, and advertising and promotion of the product.

For instance, the FDA closely regulates the post-approval marketing, labeling, advertising and promotion of drugs, including through its enforcement of standards and regulations for direct-to-consumer advertising, industry-sponsored scientific and educational activities and promotional activities involving the internet.

Drugs may be marketed only for the approved indications and in accordance with the provisions of the approved labeling.

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Even if such trials are conducted, the FDA may not approve any expansion of the labeled indications for use in a timely fashion, or at all.

Adverse event reporting and submission of periodic reports are required following FDA approval of an NDA or BLA.

Adverse event reporting and submission of periodic reports are required following FDA approval of an NDA/BLA.

The FDA also may require post-marketing testing, known as Phase 4 testing (to gain additional experience from the treatment of patients in the intended therapeutic indication), REMS or surveillance to monitor the effects of an approved product, or FDA may place conditions on an approval that could restrict the distribution or use of the product.

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From time to time, new legislation and regulations may be implemented that could significantly change the statutory provisions governing the approval, manufacturing and marketing of products regulated by the FDA.

For example, the FDA released proposed regulations in February 2022 to amend the national standards for licensing of wholesale drug distributors by the states;

establish new minimum standards for state licensing third-party logistics providers;

and create a federal system for licensure for use in the absence of a state program, each of which is mandated by the DSCSA.

It is impossible to predict whether further legislative or regulatory changes will be enacted, or FDA regulations, guidance or interpretations changed or what the impact of such changes, if any, may be.

It is impossible to predict

whether further legislative or regulatory changes will be enacted, or FDA regulations, guidance or interpretations changed or what the impact of such changes, if any, may be.

Orphan Drug Designation

Under the Orphan Drug Act, the FDA may grant Orphan Drug Designation to a drug intended to treat a rare disease or condition, which is defined as one affecting fewer than 200,000 individuals in the United States or more than 200,000 individuals where there is no reasonable expectation that the product development cost will be recovered from product sales in the United States.

Orphan Drug Designation must be requested before submitting an NDA or BLA.

After the FDA grants orphan drug designation, the identity of the drug and its potential orphan use will be disclosed publicly by the FDA;

the posting will also indicate whether a drug is no longer designated as an orphan drug.

More than one product candidate may receive an Orphan Drug Designation for the same indication.

Orphan Drug Designation does not convey any advantage in or shorten the duration of the regulatory review and approval process.

Under PREA, submission of a pediatric assessment is not required for pediatric investigation of a product that has been granted Orphan Drug Designation.

However, in 2017, the scope of PREA was extended to require pediatric studies for products intended for the treatment of an adult cancer that are directed at a molecular target and that are determined to be substantially relevant to the growth or progression of a pediatric cancer.

In addition, the FDA finalized guidance in 2018 indicating that it does not expect to grant any additional Orphan Drug Designation to products for pediatric subpopulations of common diseases.

Nevertheless, the FDA intends to still grant Orphan Drug Designation to a drug or biologic that otherwise meets all other criteria for designation when it prevents, diagnoses or treats either (i) a rare disease that includes a rare pediatric subpopulation, (ii) a pediatric subpopulation that constitutes a valid orphan subset, or (iii) a rare disease that is in fact a different disease in the pediatric population as compared to the adult population.

If an orphan drug-designated product subsequently receives FDA approval for the disease for which it was designed, the product will be entitled to seven years of product exclusivity, which means that the FDA may not approve any other applications to market the same drug for the same indication, except in very limited circumstances (such as a showing of clinical superiority to the product with orphan exclusivity by means of greater effectiveness, greater safety or providing a major contribution to patient care or in instances of drug supply issues), for seven years.

Orphan exclusivity does not block the approval of a different drug or biologic for the same rare disease or condition, nor does it block the approval of the same drug or biologic for different conditions.

If a competitor obtains approval of the same drug, as defined by the FDA, or if our product candidate is determined to be the same drug as a competitor’s product for the same indication or disease, the competitor’s exclusivity could block the approval of our product candidate in the designated orphan indication for seven years, unless our product is demonstrated to be clinically superior to the competitor’s drug.

A product with Orphan Drug Designation may not receive orphan exclusivity if it is approved for a use that is broader than the indication for which it received orphan designation.

In addition, orphan drug exclusive marketing rights in the United States may be lost if the FDA later determines that the request for designation was materially defective or if the manufacturer is unable to assure sufficient quantities of the product to meet the needs of patients with the rare disease or condition.

FDA Regulation of Companion Diagnostics

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However, according to the guidance, if a diagnostic device and a drug are to be studied together to support their respective approvals, both products can be studied in the same investigational study, if the study meets both the requirements of applicable IDE regulations and the IND regulations.

The guidance provides that, depending on the

details of the study plan and degree of risk posed to subjects, a sponsor may seek to submit an IND alone, or both an IND and an IDE.

The guidance provides that, depending on the details of the study plan and degree of risk posed to subjects, a sponsor may seek to submit an IND alone, or both an IND and an IDE.

The FDA has generally required companion diagnostics intended to select the patients who will respond to cancer treatment to obtain approval of a PMA for that diagnostic simultaneously with approval of the therapeutic.

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In particular, for a diagnostic, the applicant must demonstrate that the diagnostic produces reproducible results when the same sample is tested multiple times by multiple users at multiple laboratories.

As part of the PMA review, the FDA will typically inspect the manufacturer’s facilities for compliance with the Quality System Regulation (QSR), which imposes elaborate testing, control, documentation and other quality assurance requirements.

The QSR is being replaced by a new final rule called the Quality Management System Regulation (QMSR), which is aimed at harmonizing the FDA’s medical device current good manufacturing practice regulations with the International Organization for Standardization standard for device quality management systems (ISO 13485:2016).

The effective date for the QMSR final rule is February 2, 2026.

Until then, device manufacturers are required to comply with the QSR.

As part of the PMA review, the FDA will typically inspect the manufacturer’s facilities for compliance with the Quality Management System Regulation (QMSR), which imposes elaborate testing, control, documentation and other quality assurance requirements.

The QSR was developed and implemented to harmonize the FDA’s previous medical device current good manufacturing practice regulations (referred to as the Quality System Regulation, or QSR) with the International Organization for Standardization standard for device quality management systems (ISO 13485:2016) and it became effective on February 2, 2026.

If the FDA’s evaluation of the PMA application is favorable, the FDA may issue an approvable letter requiring the applicant’s agreement to specific conditions, such as changes in labeling, or specific additional information, such as submission of final labeling, in order to secure final approval of the PMA.

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Once granted, PMA approval may be withdrawn by the FDA if compliance with post approval requirements, conditions of approval or other regulatory standards are not maintained, or problems are identified following initial marketing.

In November 2025, the FDA issued a proposed rule to reclassify certain nucleic acid-based test systems indicated for use with a corresponding approved oncology therapeutic product from Class III (PMA) into Class II, subject to 510(k) premarket notification with special controls.

This change, if finalized in 2026, will decrease the regulatory burden on industry because manufacturers of these kinds of in vitro diagnostic products will no longer have to submit a PMA and receive FDA approval before marketing the test.

After a device is commercialized, it remains subject to significant regulatory requirements.

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An ANDA provides for marketing of a drug product that has the same active ingredients in the same strengths and dosage form as the listed drug and has been shown through bioequivalence testing to be therapeutically equivalent to the listed drug.

Other than the requirement for bioequivalence testing, ANDA

applicants are not required to conduct, or submit results of, nonclinical or clinical tests to prove the safety or effectiveness of their drug product.

Other than the requirement for bioequivalence testing, ANDA applicants are not required to conduct, or submit results of, nonclinical or clinical tests to prove the safety or effectiveness of their drug product.

Drugs approved in this way are commonly referred to as “generic equivalents” to the listed drug and can often be substituted by pharmacists under prescriptions written for the original listed drug pursuant to each state’s laws on drug substitution.

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Regulatory exclusivity provisions under the FDC Act can delay the submission or the approval of certain marketing applications for small-molecule drug products.

Upon NDA approval of a new chemical entity (NCE), which is a drug that contains no active moiety (which is the molecule or ion responsible for the action of the drug substance) that has been approved by FDA in any other NDA, that NDA receives five years of non-patent data and marketing exclusivity within the United States during which FDA cannot accept for review any (i) ANDA seeking approval of a generic version of that drug, or (ii) any NDA submitted under Section 505(b)(2) (505(b)(2) NDA) of the Act (also referred to as a submitted by another company for another drug based on the same active moiety, regardless of whether the drug is intended for the same indication as the original innovative drug or for another indication, where the applicant does not own or have a legal right of reference to all the data required for approval.

own or have a legal right of reference to all the data required for approval.

An application referencing the protected NDA may be submitted one year before NCE exclusivity expires if a Paragraph IV certification is filed, i.e., certification of patent invalidity or non-infringement to one of the patents listed with the FDA by the innovator NDA holder.

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This amendment to the PHS Act, in part, attempts to minimize duplicative testing.

We expect that our ADC product candidates are regulated as biological products that will be submitted for approval pursuant to a BLA and will be subject to the BPCIA.

Since that time, the FDA has approved more than 80 biosimilars, including the first interchangeable biosimilars in 2021.

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(2) toxicity assessments;

and (3) one or more clinical studies to demonstrate safety, purity and potency in one or more appropriate conditions of use for which the reference product is approved.

and (3) for some biological products, one or more clinical studies to demonstrate safety, purity and potency in one or more appropriate conditions of use for which the reference product is approved.

In addition, the applicant must show that the biosimilar and reference products have the same mechanism of action for the conditions of use on the label, route of administration, dosage and strength, and the production facility must meet standards designed to assure product safety, purity and potency.

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If pediatric studies are performed and accepted by the FDA as responsive to a written request from FDA, as described above, the 12-year exclusivity period will be extended for an additional six months.

In addition, the FDA will not accept an application for a biosimilar or interchangeable product based on the reference biological product until four years after the date of first licensure of the reference product.

addition, the FDA will not accept an application for a biosimilar or interchangeable product based on the reference biological product until four years after the date of first licensure of the reference product.

“First licensure” typically means the initial date the particular product at issue was licensed in the United States.

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Patent Term Extension

After NDA or BLA approval, owners of relevant drug patents may apply for up to a five-year patent extension, under the Hatch-Waxman Amendments provisions that permit the extension of eligible patents as compensation for patent term lost during product development and FDA regulatory review process.

After NDA/BLA approval, owners of relevant drug patents may apply for up to a five-year patent extension, under the Hatch-Waxman Amendments provisions that permit the extension of eligible patents as compensation for patent term lost during product development and FDA regulatory review process.

The allowable patent term extension is calculated as half of the product’s testing phase (the time between IND application and NDA/BLA submission) and all of the review phase (the time between NDA/BLA submission and approval up to a maximum of five years).

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The USPTO director must determine that approval of the drug covered by the patent for which such interim patent extension is being sought is likely.

Interim patent extensions are not available for a drug for which an NDA or BLA has not been submitted.

Interim patent extensions are not available for a drug for which an NDA/BLA has not been submitted.

Regulation Outside of the United States

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Although the EU Clinical Trials Directive 2001/20/EC sought to harmonize the EU clinical trials regulatory framework, setting out common rules for the control and authorization of clinical trials in the EU, the EU Member States transposed and applied the provisions of the Directive differently.

This led to significant variations in the member state regimes.

This led to significant variations in the

member state regimes.

Under the previous regime, before a clinical trial could be initiated, a clinical trial application must have been approved in each of the EU countries where the trial was to be conducted by two distinct bodies:

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The extent to which ongoing clinical trials are governed by the Clinical Trials Regulation depends on when the Clinical Trials Regulation became applicable and on the duration of the individual clinical trial.

If a clinical trial continues for more than three years from the day on which the Clinical Trials Regulation became applicable the Clinical Trials Regulation will at that time begin to apply to

the clinical trial.

If a clinical trial continues for more than three years from the day on which the Clinical Trials Regulation became applicable the Clinical Trials Regulation will at that time begin to apply to the clinical trial.

In addition, use of the new EU-wide application procedure being implemented via the Clinical Trial Information System, became mandatory for new clinical trial application submissions as of February 1, 2023.

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Upon receiving an MA, reference product candidates generally receive eight years of data exclusivity and an additional two years of market exclusivity.

If granted, the data exclusivity period prevents generic applicants from relying on the nonclinical and clinical trial data contained in the dossier of the reference product when applying for a generic MA in the EU during a period of eight years from the date on which the reference product was first authorized in the EU.

If granted, the data exclusivity period prevents generic applicants from relying on the

nonclinical and clinical trial data contained in the dossier of the reference product when applying for a generic MA in the EU during a period of eight years from the date on which the reference product was first authorized in the EU.

The market exclusivity period prevents a successful generic applicant from commercializing its product in the EU until 10 years have elapsed from the initial MA of the reference product in the EU.

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In April 2023, the European Commission proposed widespread changes to the existing pharmaceutical legislation that would, among other things, alter the data exclusivity periods available to MA holders.

The proposed reforms must be reviewed and approval by the European Parliament and Council, as well as EU Member States, and in light of their controversial nature it is unclear whether they will be adopted as proposed or further revised.

As of December 2025, the three EU institutions, the European Commission, the European Parliament and the Council of the EU, are in the process of negotiating the final content of the new Directive and Regulation.

Once negotiations are complete, the European Parliament and the Council of the EU will vote on whether to approve the Directive and Regulation.

If adopted and implemented, these revisions will significantly change several aspects of drug development and approval in the European Union.

EU Post-Approval Requirements

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Moreover, although general requirements for advertising and promotion of medicinal products are established under EU directives, the details are governed by regulations in each Member State and can differ from one country to another.

In recent months, advertising and promotion by pharmaceutical companies in the EU and UK marketplaces have received heightened scrutiny from regulatory authorities and in some cases, fines have been issued.

In recent years, advertising and promotion by pharmaceutical companies in the EU and UK marketplaces have received heightened scrutiny from regulatory authorities and in some cases, fines have been issued.

Brexit and the Regulatory Framework in the United Kingdom (UK)

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In March 2023, the UK government and the European Commission reached agreement on a regulatory framework, referred to as the Windsor Framework.

The Windsor Framework will apply as of January 1, 2025 and changes the existing system for the regulation of pharmaceutical products in the UK.

Specifically, the MHRA will be responsible for approving all medicines intended to be marketed in the United Kingdom, while the EMA will no longer be involved in approving medicines intended for sale in Northern Ireland.

The Windsor Framework became effective January 1, 2025 and changed the system that was previously in effect under the Northern Ireland Protocol, including for the regulation of pharmaceutical products in the UK.

Specifically, the MHRA will be responsible for

approving all medicines intended to be marketed in the United Kingdom (including Northern Ireland), while the EMA will no longer be involved in approving medicines intended for sale in Northern Ireland.

Other Healthcare Laws

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Violations of this law are punishable by imprisonment, criminal fines, administrative civil money penalties and exclusion from participation in federal healthcare programs.

In addition, the

Patient Protection and Affordable Care Act (discussed further below), among other things, amended the intent requirement of the federal Anti-Kickback Statute and two of the five criminal healthcare fraud statutes created by the Health Insurance Portability and Accountability Act of 1996 (HIPAA).

In addition, the Patient Protection and Affordable Care Act (discussed further below), among other things, amended the intent requirement of the federal Anti-Kickback Statute and two of the five criminal healthcare fraud statutes created by the Health Insurance Portability and Accountability Act of 1996 (HIPAA).

A person or entity no longer needs to have actual knowledge of these two provisions in the statute or specific intent to violate them;

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Poison Prevention Packaging Act.

Manufacturing, sales, promotion and other activities also are potentially subject to federal and state consumer protection and unfair competition laws.

Manufacturing, sales, promotion and other activities are also potentially subject to federal and state consumer protection and unfair competition laws.

Also, many states have similar fraud and abuse statutes or regulations that apply to items and services reimbursed under Medicaid and other state programs, or, in several states, apply regardless of the payor.

Some state laws require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines, or the relevant compliance guidance promulgated by the federal government, in addition to requiring drug manufacturers to report information related to payments to physicians and other health care providers or marketing expenditures to the extent that those laws impose requirements that are more stringent than the Physician Payments Sunshine Act.

marketing expenditures to the extent that those laws impose requirements that are more stringent than the Physician Payments Sunshine Act.

Efforts to ensure that business arrangements with third parties comply with applicable state, federal and foreign healthcare laws and regulations involve substantial costs.

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Healthcare Reform and Potential Changes to Healthcare Laws

In the United States and some foreign jurisdictions, there have been, and continue to be, several legislative and regulatory changes and proposed changes regarding the healthcare system that could prevent or delay marketing approval of therapeutic product candidates, restrict or regulate post-approval activities, and affect the ability to profitably

sell therapeutic product candidates that obtain marketing approval.

The FDA’s and other regulatory authorities’ policies may change and additional government regulations may be enacted that could prevent, limit or delay marketing approval of our product candidates.

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Moreover, there has 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 legislation designed to, among other things, bring more transparency to product pricing, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement methodologies for drug products.

In August 2022, President Biden signed into the law the Inflation Reduction Act of 2022 (IRA).

For example, in August 2022, President Biden signed into the law the Inflation Reduction Act of 2022 (IRA).

Among other things, the IRA has multiple provisions that may impact the prices of drug and biological products that are both sold into the Medicare program and throughout the United States.

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This calculation is made on a drug product by drug product basis and the amount of the rebate owed to the federal government is directly dependent on the volume of a drug product that is paid for by Medicare Parts B or D.

Additionally, starting for payment year 2026, CMS is negotiating drug prices annually for a select number of single source Part D drugs without generic competition.

CMS will also negotiate drug prices for a select number of Part B drugs starting for payment year 2028.

Additionally, CMS is negotiating drug prices annually for a select number of single source Part D drugs

without generic competition and will also negotiate drug prices for a select number of Part B drugs starting for payment year 2028.

If a product is selected by CMS for negotiation, it is expected that the revenue generated from such product will decrease.

CMS has begun to implement these new authorities entering into a first set of agreements with pharmaceutical manufacturers to conduct price negotiations in October 2023 and ultimately announcing the first round of negotiated prices for the first 10 drugs in August 2024;

those negotiated “maximum fair prices” will be effective as of January 1, 2026 (payment year 2026).

CMS is currently initiating activities for the second year of the program and published the next 15 drugs selected for negotiation in January 2025.

However, their impact on the biopharmaceutical industry in the United States remains uncertain, in part because multiple large pharmaceutical companies and other stakeholders (e.g., the U.S.

CMS has begun to implement these new authorities by entering into agreements with pharmaceutical manufacturers to conduct price negotiations and ultimately announcing annual rounds of negotiated prices;

those negotiated “maximum fair prices” will be effective initially as of January 1, 2026 (payment year 2026), with subsequent rounds adding additional drugs with negotiated maximum fair prices effective ins subsequent years.

However, the IRA’s impact on the biopharmaceutical industry in the United States remains uncertain, in part because multiple large pharmaceutical companies and other stakeholders (e.g., the U.S.

Chamber of Commerce) have initiated federal lawsuits against CMS arguing the program is unconstitutional for a variety of reasons, among other complaints.

The outcome of such ongoing lawsuits, as well as potential legislative changes enacted by Congress or programmatic changes implemented at CMS by the Trump Administration, may impact the IRA drug price negotiation program in the future.

Separately, the Trump Administration announced the creation of a government website called TrumpRx, which will allow consumers to purchase certain drugs at reduced prices as negotiated between the drug manufacturers and the administration.

As of January 2026, the Trump Administration had secured deals with 16 major drug manufacturers to offer certain drugs at most-favored-nation prices.

Individual states in the United States have also increasingly passed legislation and implemented regulations designed to control pharmaceutical product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing.

In December 2020, the U.S.

Supreme Court held unanimously that federal law does not preempt the states’ ability to regulate pharmacy benefit managers (PBMs) and other members of the health care and pharmaceutical supply chain, an important decision that has led to

further and more aggressive efforts by states in this area.

The FTC in mid- 2022 also launched sweeping investigations into the practices of the PBM industry, and published interim reports with its findings in mid-2024 and January 2025, that could lead to additional federal and state legislative or regulatory proposals targeting such entities’ operations, pharmacy networks, or financial arrangements, including in the current 2025-2026 congressional session where PBM reform continues to be a bipartisan priority.

During the previous congressional session, numerous PBM reforms were considered in both the Senate and the House of Representatives;

they included diverse legislative proposals such as eliminating rebates;

divorcing service fees from the price of a drug, discount, or rebate;

prohibiting spread pricing;

limiting administrative fees;

requiring PBMs to report formulary placement rationale;

promoting transparency.

The FTC in mid-2022 also launched sweeping investigations into the practices of the PBM industry, and published interim reports with its findings in mid-2024 and January 2025, that could lead to additional federal and state legislative or regulatory proposals targeting such entities’ operations, pharmacy networks, or financial arrangements, as PBM reform continues to be a bipartisan priority.

In February 2026, President Trump signed into law several PBM regulatory reforms as part of a federal budget package, including but not limited to requirements for PBMs to pass back 100% of rebates and fees to commercial health plan sponsors;

to provide extensive informational disclosures related to patients’ coverage and benefits;

and to accept only bona fide service fees from drug companies when providing services under Medicare Part D.

The Department of Labor (DOL) also issued a proposed rule in January 2026 that would mandate specific PBM fee disclosures to self-insured plan fiduciaries under the Employment Retirement Income Security Act (ERISA).

If finalized as proposed, the DOL rule would also allow plan fiduciaries to audit those PBM disclosures to confirm accuracy.

Additional proposals and legislative changes aimed at PBMs and their business practices are likely to continue to be introduced and considered in Congress and by executive agencies.

Significant efforts to change the PBM industry as it currently exists in the U.S.

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Moreover, eligibility for reimbursement does not imply that any product will be paid for in all cases or at a rate that covers our costs, including research, development, manufacture, sale and distribution.

Interim payments for new products, if applicable, may also not be sufficient to cover our costs and may not be made permanent.

Interim payments for new products, if applicable, may also not be sufficient to cover

our costs and may not be made permanent.

Payment rates may vary according to the use of the product and the clinical setting in which it is used, may be based on payments allowed for lower cost products that are already reimbursed and may be incorporated into existing payments for other services.

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Additionally, the containment of healthcare costs has become a priority of federal and state governments and the prices of therapeutics have been a focus in this effort.

federal actions include initiatives incorporating “most favored nation” (international reference pricing) concepts for certain prescription drugs, as well as agency testing of new payment models that could tie Medicare reimbursement or manufacturer rebates to prices in specified reference countries.

The United States government, state legislatures and foreign governments have shown significant interest in implementing cost-containment programs, including price controls, restrictions on reimbursement and requirements for substitution of generic products.

In late 2025, the CMS proposed multiyear pilot models for Medicare Parts B and D that would require manufacturer rebates when U.S.

prices exceed prices in designated reference countries;

in parallel, the Trump Administration announced a series of manufacturer specific arrangements reflecting most favored nation style pricing commitments in certain programs and channels.

Adoption of price controls and cost-containment measures, and adoption of more restrictive policies in jurisdictions with existing controls and measures, could further limit our net revenue and results.

If these third-party payors do not consider our products to be cost-effective compared to other therapies, they may not cover our products after approval as a benefit under their plans or, if they do, the level of payment may not be sufficient to allow us to sell our products on a profitable basis.

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

Although the scope, timing and ultimate impact of these most favored nation style initiatives are uncertain and subject to rulemaking, program design and potential legal challenges, if implemented or expanded they could reduce reimbursement or net realized prices for affected products and adversely impact our margins and market access, particularly for products reimbursed under Medicare.

Further, if these third-party payors do not consider our products to be cost-effective compared to other therapies, they may not cover our products after approval as a benefit under their plans or, if they do, the level of payment may not be sufficient to allow us to sell our products on a profitable basis.

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

Moreover, in some foreign countries, the proposed pricing for a therapeutic product must be approved before it may be lawfully marketed.

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There can be no assurance that any country that has price controls or reimbursement limitations for pharmaceutical products will allow favorable reimbursement and pricing arrangements for any of our therapeutic candidates, if approved.

Historically,

therapeutic candidates launched in the European Union do not follow price structures of the United States and generally tend to be significantly lower.

Historically, therapeutic candidates launched in the European Union do not follow price structures of the United States and generally tend to be significantly lower.

Foreign Corrupt Practices Act

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The term “instrumentality” is broad and can include state-owned or state-controlled entities.

Importantly, United States authorities that enforce the FCPA, including the Department of Justice, deem most health care professionals and other employees of foreign hospitals, clinics, research facilities and medical schools in countries with public health care or public education systems to be “foreign officials” under the FCPA.

Importantly, U.S.

authorities that enforce the FCPA, including the Department of Justice, deem most health care professionals and other employees of foreign hospitals, clinics, research facilities and medical schools in countries with public health care or public education systems to be “foreign officials” under the FCPA.

When we interact with foreign health care professionals and researchers in testing and marketing our products abroad, we must have policies and procedures in place sufficient to prevent us and agents acting on our behalf from providing any bribe, gift or gratuity, including excessive or lavish meals, travel or entertainment in connection with marketing our future products and services or securing required permits and approvals such as those needed to initiate clinical trials in foreign jurisdictions.

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companies abroad.

It is unclear how this presidential directive may affect the biopharmaceutical industry as a whole or our business in particular.

On June 10, 2025, the DOJ issued guidelines for investigations and enforcement of the FCPA.

It is unclear how this presidential directive and DOJ guidelines may affect the biopharmaceutical industry as a whole or our business in particular.

Employees and Human Capital Resources

As of December 31, 2025, we had 77 employees, all of whom were full-time and 45 of whom were engaged in research and development activities.

Twenty-three of our employees hold Ph.D.

33 of our employees hold Ph.D.

None of our employees are represented by a labor union or covered under a collective bargaining agreement.

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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