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Item 1. Business

2025-03-13 compared with 2024-03-05 · 185 added, 138 removed, 601 unchanged (35% of the section changed)

We are a clinical-stage biopharmaceutical company focused on the development and commercialization of seralutinib for the treatment of pulmonary arterial hypertension, or PAH.

Our goal is to be an industry leader in, and to enhance the lives of patients living with, pulmonary hypertension, or PH.

To accomplish this goal, we have assembled a deeply

experienced and highly skilled group of industry veterans, scientists, clinicians and key opinion leaders from leading biotechnology and pharmaceutical companies, as well as leading academic centers from around the world.

We are a clinical-stage biopharmaceutical company focused on the development and commercialization of seralutinib for the treatment of pulmonary hypertension, or PH, including pulmonary arterial hypertension, or PAH, and PH

associated with interstitial lung disease, or PH-ILD.

Our goal is to be an industry leader in, and to enhance the lives of patients living with, PH.

To accomplish this goal, we have assembled a deeply experienced and highly skilled group of industry veterans, scientists, clinicians and key opinion leaders from leading biotechnology and pharmaceutical companies, as well as leading academic centers from around the world.

We intend to maintain a scientifically rigorous and inclusive corporate culture where employees strive to bring improved therapeutic options to patients.

Our founders and management team have held senior positions at leading biopharmaceutical companies and possess substantial experience and expertise across the spectrum of drug discovery, development and commercialization.

Faheem Hasnain is our Co-Founder and has served as our Chief Executive Officer since November 2020 and as our Chairman since our inception.

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Prior to Gossamer, Mr.

Smith was the National Sales Lead in charge of preparing for the potential launch of sotatercept for the treatment of PAH in the US at Merck & Co., or Merck.

Smith was the National Sales Lead in charge of preparing for the commercial launch of sotatercept for the treatment of PAH in the US at Merck & Co., or Merck.

Previously until 2018, Mr.

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Caryn Peterson, our Executive Vice President, Regulatory Affairs, has considerable experience and regulatory expertise as a Managing Director of Development & Strategic Consulting Associates, as well as management positions leading regulatory affairs at Syndax Pharmaceuticals and FeRx Incorporated.

We are a clinical-stage biopharmaceutical company focused on the development and commercialization of seralutinib for the treatment of PAH.

We are a clinical-stage biopharmaceutical company focused on the development and commercialization of seralutinib for the treatment of PH.

Our goal is to be an industry leader in, and to enhance the lives of patients living with, PH.

Critical components of our business strategy include:

• Complete the ongoing Phase 3 PROSERA Study of seralutinib in PAH and pursue regulatory approval.

We commenced the registrational Phase 3 PROSERA Study in PAH in the fourth quarter of 2023.

We expect to report topline data from the PROSERA study in the fourth quarter of 2025.

If the clinical trial is successful, we will seek marketing approval for seralutinib in the United States.

• Increase the impact of seralutinib by expanding development into PH indications of high unmet need.

We believe that not only does seralutinib offer potential as a therapeutic option for the treatment of PAH but also for the treatment of other rare PH indications of high unmet need, including PH-ILD.

To that end, we expect to activate clinical sites for a global registrational Phase 3 for the treatment of PH-ILD in the second half of 2025.

Additionally, we will continue to evaluate other potential related indications of high unmet need for further development of seralutinib.

• Leverage the clinical development and commercialization expertise of our world-class team.

Our executive management team and key scientific leaders have successfully discovered, developed and commercialized pharmaceuticals at both large and small biopharmaceutical companies.

Additionally, we have built experienced PH development and commercialization teams, with key team members selected from the leadership of such companies as Actelion, Johnson & Johnson, Merck and United Therapeutics.

• Increase the impact of seralutinib by expanding development into PH indications of high unmet need, beyond PAH.

We are actively pursuing clinical development plans for seralutinib in PH-ILD.

Additionally, we have built experienced PH development and commercialization teams, with key team members selected from the leadership of companies such as Actelion, Johnson & Johnson, Merck and United Therapeutics.

• Build our operational capabilities to successfully commercialize seralutinib for PH, if approved.

If we obtain regulatory approvals for seralutinib, we intend to build in-house sales and marketing capabilities to commercialize seralutinib in the United States and/or geographies for which we maintain seralutinib commercialization rights.

If we obtain regulatory approvals for seralutinib, we intend to build in-house sales and marketing capabilities to commercialize seralutinib in the United States, as part of our global collaboration and license agreement, or collaboration agreement, with Chiesi Farmaceutici S.p.A and Chiesi USA, Inc., or collectively, Chiesi.

Both PAH and PH-ILD patients are often treated by cardiologists and pulmonologists at national or regional centers of excellence.

concentrations of patients could allow us to commercialize seralutinib with a relatively small commercial footprint, if approved.

• Engage in partnerships, collaborations, licensing deals or other transactions.

Gossamer may seek to enter into strategic or financial transactions to gain access to additional financial, clinical or commercial resources to support the development and commercialization of seralutinib for PH.

These concentrations of patients could allow us to commercialize seralutinib with a relatively small commercial footprint, if approved.

Seralutinib (PDGFR, CSF1R and c-KIT Inhibitor)

Seralutinib, also known as GB002, is an investigational inhaled, small molecule, platelet-derived growth factor receptor, or PDGFR, colony-stimulating factor 1 receptor, or CSF1R, and c-KIT inhibitor, currently being evaluated in a Phase 3 clinical trial for the treatment of PAH.

In contrast to the three classes of marketed vasodilatory therapies for PAH, we believe that seralutinib has the potential to reverse pathological remodeling by addressing mechanisms that underlie PAH.

We believe that seralutinib has the potential to reverse pathological remodeling by addressing mechanisms that underlie PAH.

Inhaled seralutinib, which is designed to act on both isoforms of the PDGFR, α and β, as well as the CSF1R and c-KIT pathways, inhibited and reversed cellular overgrowth in lung blood vessels in multiple animal PAH models.

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We expect to report topline data from the PROSERA study in the fourth quarter of 2025.

In addition to PAH, we believe that seralutinib holds potential as a therapeutic option for the treatment of PH-ILD.

We are actively pursuing clinical development plans for seralutinib in PH-ILD.

In addition to PAH, we believe that seralutinib holds potential as a therapeutic option for the treatment of PH-ILD, and to that end, we expect to activate clinical sites for a global registrational Phase 3 for the treatment of PH-ILD in the second half of 2025.

In May 2024, we entered into a collaboration agreement with Chiesi.

Under the collaboration agreement, we will jointly develop seralutinib with Chiesi.

We will lead global development of seralutinib in PAH and PH-ILD and will lead potential commercialization for PAH and PH-ILD in the United States, with both parties contributing 50%of commercial efforts, including performing 50% of the commercialization activities.

Chiesi will lead global development in any additional indications and will lead potential commercialization in the United States in any additional indications.

Chiesi will also have the exclusive right to commercialize seralutinib outside of the United States.

We in-licensed seralutinib from Pulmokine, Inc.

in 2017 and retain worldwide rights.

The United States Food and Drug Administration, or FDA, and the European Medicines Agency, or EMA, have granted seralutinib orphan drug designation for the treatment of patients with PAH.

The United States Food and Drug Administration, or FDA, the European Commission, or EC, and the Pharmaceuticals and Medical Devices Agency, or PMDA, of Japan have granted seralutinib orphan drug designation for the treatment of patients with PAH.

The following table summarizes the current development plan for seralutinib in PH:

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Upregulated PDGFR signaling results in endothelial cell and fibroblast dysfunction and the proliferation and migration of smooth muscle cells.

This effect results in the overgrowth and occlusion of

blood vessels in the lung.

This effect results in the overgrowth and occlusion of blood vessels in the lung.

Kinase inhibitors with activity against the PDGFR pathway have shown the ability to reverse PAH in animal models.

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Novartis withdrew its supplemental regulatory applications in PAH in 2013 and, to our knowledge, did not pursue further development of imatinib in the indication.

Overview of Pulmonary Arterial Hypertension

PAH is a rare disease that is characterized by abnormally high blood pressure in the blood vessels carrying deoxygenated blood from the right side of the heart to the lungs and is progressive and often fatal.

Overview of PAH (WHO Group 1 Pulmonary Hypertension)

PAH, classified as World Health Organization, or WHO, Group 1 Pulmonary Hypertension, is a rare disease that is characterized by abnormally high blood pressure in the blood vessels carrying deoxygenated blood from the right side of the heart to the lungs and is progressive and often fatal.

Symptoms include shortness of breath at rest or with minimal exertion.

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Worsening symptoms, and thus higher numbered functional classes, are associated with higher mortality.

The four functional classes established by the World Health Organization are detailed below in Table 1.

The four functional classes established by the WHO are detailed below in Table 1.

PAH Functional Classes

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The true incidence and prevalence of PAH are unknown.

The estimated PAH patient population in the US is over 50,000 patients, and the estimated patient population in the EU is over 70,000 patients.

The estimated PAH patient population in the US is about 50,000 patients, and the estimated patient population in the EU is over 70,000 patients.

The number of diagnosed PAH patients continues to increase, and we believe this increase is likely due to enhanced awareness and diagnosis of the disease.

Total branded PAH drug sales worldwide in 2022 were approximately $5.9 billion.

Worldwide branded drug sales for PAH and PH-ILD therapies totaled over $7 billion in 2023.

Treatment Paradigm in PAH

Currently approved PAH therapies consist of three classes of vasodilators:

PDE5 inhibitors (and guanylate cyclase stimulators), ERAs, and prostanoids (and prostacyclin receptor agonists).

Currently approved PAH therapies consist of three classes of vasodilators and one activin ligand trap therapy.

The three classes of vasodilatory therapy are PDE5 inhibitors (and guanylate cyclase stimulators), ERAs, and prostanoids (and prostacyclin receptor agonists).

PDE5 inhibitors are often used in combination with ERAs as an early treatment strategy.

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Prostanoids are also commonly used to treat patients with evidence of right heart failure.

While some existing treatments have led to significant improvements in time to clinical worsening and other composite endpoints in PAH patients, none directly alter the underlying disease pathophysiology.

The vasodilation effects of approved PAH therapies, while capable of improving blood flow through the lungs, may eventually be overtaken by the worsening cellular proliferation and arterial remodeling underlying the condition.

The recent introduction of an activin ligand trap therapy (sotatercept) to the market has provided patients with an additional treatment option.

While some existing treatments have led to significant improvements in time to clinical worsening and other composite endpoints in PAH patients, there are no cures.

The effects of approved PAH therapies, while capable of improving blood flow through the lungs, may eventually be overtaken by the worsening cellular proliferation and arterial remodeling underlying the condition, given the progressive nature of the disease.

We believe an agent with the ability to safely reverse pathological remodeling could provide utility across functional classes and risk categories.

New investigational therapies, such as sotatercept (Merck), may impact the PAH treatment paradigm, if approved.

Sotatercept, a subcutaneously administered activin receptor type IIA-Fc fusion protein, is an investigational drug candidate that has been evaluated in a completed Phase 3 PAH clinical trial.

Marketing authorization for sotatercept for the treatment of PAH has been filed in both the US and the EU.

Seralutinib Product Differentiation in PAH

Overview of PH-ILD (WHO Group 3 Pulmonary Hypertension)

We believe that seralutinib has potential as a therapeutic treatment for PH-ILD, a subtype of WHO Group 3 Pulmonary Hypertension.

PH-ILD is a collection of progressive and often fatal forms of PH that affect the small airways of the

PH-ILD includes PH related to idiopathic pulmonary fibrosis and PH related connective tissue disease-associated interstitial lung disease, amongst other diseases.

These diseases are characterized by pulmonary vascular pathology associated with PH, in addition to thickening and scarring of the lung interstitium from interstitial lung disease.

While the prevalence of PH-ILD is unknown, we believe PH-ILD is at least as prevalent as PAH.

Patients living with PH-ILD generally have a poor disease prognosis and have an increased mortality rate, as compared to PAH patients.

There is only one FDA-approved treatment for PH-ILD, and there are no approved therapies in the EU.

Seralutinib Product Differentiation

Seralutinib is an inhaled kinase inhibitor designed to build on the evidence of efficacy seen in trials of imatinib while overcoming imatinib’s observed systemic safety and tolerability issues and improving on imatinib's kinase inhibitory profile.

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Additionally, seralutinib is multiple orders of magnitude more potent against CSF1R, as compared to imatinib.

We believe seralutinib has the potential to be a therapeutic option for PAH that may provide a:

• differentiated, anti-proliferative mechanism that addresses the underlying mechanisms of PAH;

We believe seralutinib has the potential to be a therapeutic option for PH that may provide a:

• differentiated, anti-proliferative mechanism that addresses the underlying mechanisms of both PAH and PH-ILD;

• more tolerable safety profile than systemic imatinib;

• convenient, simple and portable inhalation delivery system.

Summary of Preclinical Program in PAH

Summary of Preclinical Program

Seralutinib inhibits both PGDFR α and β, and it inhibited and reversed cell overgrowth in lung blood vessels in a PAH rat model, which replicates many features of human PAH, including the abnormal cell proliferation that can block the small vessels of the lung.

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There were no SAEs, and the most frequently reported AEs were mild-to-moderate cough and mild headache.

Systemic PK was characterized by low systemic exposure and rapid drug clearance in PAH patients, which was consistent with PK data from the Phase 1a studies in healthy volunteers.

Systemic PK was characterized by low systemic exposure and rapid drug clearance in PAH patients, which was consistent with PK data from the Phase 1a studies in healthy

Target engagement in PAH patients was demonstrated via whole blood CSF1R stabilization assay across all tested dose levels.

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Upon completion of the 24-week blinded portion of the Phase 2 TORREY Study, 73 of the 80 (91%) completing patients elected to rollover into an ongoing open-label extension trial.

In December 2023, we reported out interim results, including PVR results at Week 72.

The 27 seralutinib-continued patients who reported Week 72 PVR data showed a median PVR improvement from TORREY baseline of 146 dynes × seconds/cm 5 at Week 72, as compared to a median improvement of 89 dynes × seconds/cm 5 at Week 24.

18 of the 25 (72%) placebo-crossover patients who reached Week 72 (48 weeks on seralutinib) showed improvement in PVR at Week 72 as compared to TORREY baseline.

Additionally, further improvements in 6MWD, as compared to TORREY baseline, were observed, with greater improvements seen in patients with an elevated risk score.

Consistent with completed clinical trials, seralutinib has been generally well tolerated in the ongoing label extension study.

In the TORREY open-label continued-seralutinib group treated for 72 weeks continued improvement was noted in PVR and 6MWD, as compared to TORREY baseline.

Consistent with completed clinical trials, seralutinib has been generally well tolerated as of December 31, 2024, in the ongoing open-label extension study.

Summary of Ongoing PROSERA Phase 3 PAH Clinical Trial

In the fourth quarter of 2023, we commenced the registrational Phase 3 PROSERA Study, a randomized, double-blind, placebo-controlled, global clinical trial in PAH patients, after receiving input from global regulatory authorities, including the FDA and EMA.

We are enrolling approximately 350 Functional Class II and III PAH patients on stable background therapy.

Patients will be randomized in a 1:1 fashion to 90 mg twice daily seralutinib and placebo, and patients will receive blinded therapy for up to 48 weeks.

Patients will be randomized in a 1:1 fashion to 90 mg twice daily seralutinib or placebo, and patients will receive blinded therapy for up to 48 weeks.

Patients will remain on their background PAH therapies throughout the trial.

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In addition to secondary and exploratory endpoints, safety and tolerability will also be evaluated in the Phase 3 PROSERA Study.

We expect to report topline data from the PROSERA in the fourth quarter of 2025.

Plans for Future Development in PH-ILD

We believe that seralutinib has potential for development as a therapeutic treatment for PH-ILD.

A subgroup of WHO Group 3 Pulmonary Hypertension, PH-ILD is a collection of progressive and often fatal forms of pulmonary

hypertension that affect the small airways of the lungs.

PH-ILD includes PH related to idiopathic pulmonary fibrosis and PH related connective tissue disease-associated interstitial lung disease.

These diseases are characterized by pulmonary vascular pathology associated with pulmonary hypertension, in addition to thickening and scarring of the lung interstitium from interstitial lung disease.

We believe that seralutinib has the potential to improve the quality of life for PH-ILD patients.

There is only one FDA-approved treatment for PH-ILD, and there are no approved therapies in the EU.

We are actively pursuing clinical development plans for seralutinib in PH-ILD.

We expect to report topline data from the PROSERA study in the fourth quarter of 2025.

If the clinical trial is successful, we will seek marketing approval for seralutinib in the United States.

Summary of Planned Phase 3 Clinical Trial in PH-ILD

We expect to activate clinical sites for a global registrational Phase 3 for the treatment of PH-ILD in the second half of 2025.

The planned Phase 3 clinical trial will be a registrational, randomized, double-blind, placebo-controlled, global clinical trial in PH-ILD patients.

We have discussed the protocol design with global regulatory authorities and this will inform the final design of the Phase 3 clinical trial.

The primary endpoint of the trial will be change in 6MWD from baseline.

The biotechnology and pharmaceutical industries are characterized by rapid technological advancement, significant competition and an emphasis on intellectual property.

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Seralutinib is a PDGFR, CSF1R and c-KIT inhibitor initially targeted for PAH and PH-ILD patients.

We expect competition within the PAH indication will include prostanoids / prostacyclin receptor agonists, including Orenitram (United Therapeutics), Uptravi (Janssen), Tyvaso (United Therapeutics), and Remodulin (United Therapeutics).

We also may face some competition from products used in class I and II patients, such as the oral PDE5 inhibitors, including Revatio (Pfizer Inc.) and Adcirca (United Therapeutics);

We also may face some competition from products used in Functional Class I and II patients, such as the oral PDE5 inhibitors, including Revatio (Pfizer Inc.) and Adcirca (United Therapeutics);

the sGC stimulator Adempas (Bayer AG);

and oral ERAs, including Tracleer (Janssen), Letairis (Gilead Sciences, Inc.) and Opsumit (Janssen);

We believe that, if approved, seralutinib could be used alongside all three classes of approved therapies.

PAH is also an active indication for investigational drugs, and we may face competition in the future from CS1 (Cereno Scientific), KER-012 (Keros Therapeutics, Inc.), L606 (Pharmosa Biopharm Inc.), MK-5475 (Merck & Co., Inc.), ralinepag (Pfizer and United Therapeutics) and sotatercept (Merck).

Additionally, although not approved for the treatment of PAH, we may face competition from formulations of imatinib in development for the treatment of PAH, including those from Aerami Therapeutics, Aerovate Therapeutics and Tenax Therapeutics.

We expect to face competition from Tyvaso (United Therapeutics) within the PH-ILD indication, as it is the only approved therapy for PH-ILD in the US.

and combination PDE5 inhibitor / ERA therapies, such as Opsynvi (Janssen).

We believe that, if approved, seralutinib could be used alongside all classes of approved therapies.

PAH is also an active indication for investigational drugs, and we may face competition in the future from CS1 (Cereno Scientific), L606 (Liquidia / Pharmosa Biopharm Inc.), treprostinil palmitil (Insmed), ralinepag (United Therapeutics), and REGN13335 (Regeneron Pharmaceuticals, Inc.).

Additionally, although not approved for the treatment of PAH, we may face competition from formulations of imatinib, including the one in development from Tenax Therapeutics and Inhibikase Therapeutics.

We expect to face competition from Tyvaso (United Therapeutics) within the PH-ILD indication, as it is the only approved therapy for PH-ILD in the United States.

There are no approved therapies for PH-ILD in the EU.

PH-ILD is also an active indication for investigational drugs, and we may face competition in the future from L606 (Pharmosa Biopharm Inc.), MD-711 (Mochida Pharmaceutical Co., Ltd.), sirolimus (OrphAI Therapeutics) and treprostinil palmitil (Insmed, Inc.).

Additionally, although not approved for the treatment of PH-ILD, we may face competition from formulations of imatinib, including those from Aerami Therapeutics, Aerovate Therapeutics and Tenax Therapeutics.

PH-ILD is also an active indication for investigational drugs, and we may face competition in the future from L606 (Liquidia / Pharmosa Biopharm Inc.), sirolimus (OrphAI Therapeutics), treprostinil palmitil (Insmed, Inc.), MK-5475 (Merck), and mosliciguat (Pulmovant, Inc.).

There may be other earlier stage clinical programs that, if approved, would compete with seralutinib.

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Our success will be based in part on our ability to build and actively manage a portfolio of drugs that addresses unmet medical needs and creates value in patient therapy.

License Agreements

License and Collaboration Agreements

In October 2017, we entered into a license agreement, or the Pulmokine Agreement, with Pulmokine, Inc., under which we were granted an exclusive worldwide license and sublicense to certain intellectual property rights owned or controlled by Pulmokine, including intellectual property rights co-owned by Pulmokine and Gilead Sciences, to develop and commercialize seralutinib and certain backup compounds for the treatment, prevention and diagnosis of any and all disease or conditions.

On November 26, 2024, Pulmokine became a wholly-owned subsidiary of XOMA Royalty Corporation.

We also have the right to sublicense our rights under the Pulmokine Agreement, subject to certain conditions.

We are required to use commercially reasonable efforts to develop and commercialize at least one licensed product in the United States and in at least two countries in the European Union.

Under the terms of the Pulmokine Agreement, we made an upfront payment of $5.5 million and milestone payments of $5.0 million and $10.0 million to Pulmokine and are obligated to make future development and regulatory milestone payments of up to $48 million, commercial milestone payments of up to $45 million, and sales milestone payments of up to $190 million.

Under the terms of the Pulmokine Agreement, we made an upfront payment of $5.5 million and milestone payments of $5.0 million and are obligated to make future development and regulatory milestone payments of up to $48 million, which includes a payment of $5.0 million due upon the initiation of a Phase 3 clinical trial in a second indication, commercial milestone payments of up to $45 million, and sales milestone payments of up to $190 million.

In January 2024, the Company made a payment of $10.0 million in connection with the initiation of the Phase 3 clinical trial of seralutinib.

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Upon termination of the Gilead Agreement for any reason, our sublicense under the Pulmokine Agreement will survive provided that we did not cause a material breach that was the basis for such termination and we agree to be bound by the terms of the Gilead Agreement.

The Pulmokine Agreement also includes a sublicense to patents concerning methods for detecting pulmonary arterial hypertension owned by The Rensselaer Center for Translational Research, Inc., or Rensselaer, and licensed to Pulmokine in an exclusive license agreement, or the Rensselaer License.

Under the Rensselaer License, Pulmokine is required to use commercially reasonable efforts to develop and commercialize at least one licensed product covered by the Rensselaer patent rights, which obligation can be satisfied through our development efforts.

If such obligation is not satisfied by Pulmokine or us, or the Rensselaer License is otherwise terminated for any reason, our sublicense under the Pulmokine Agreement will, at our option, either terminate or, subject to Rensselaer’s approval and our acceptance of the provisions of the Rensselaer License, convert to a license directly between us and Rensselaer.

Upon termination of the Pulmokine Agreement for any reason, all rights and licenses granted to us under the agreement will terminate and revert to Pulmokine, and in the event of certain termination events, we would grant Pulmokine worldwide rights to the terminated program.

On May 3, 2024, we, GB002, Inc., our wholly-owned subsidiary, and Gossamer Bio 002 Ltd., our indirect wholly-owned subsidiary, entered into a collaboration agreement with Chiesi.

The collaboration is focused on the development and commercialization of seralutinib and licensed products including seralutinib and related licensed compounds, or Licensed Products, in the United States and the rest of the world, or the ROW Territory, for the treatment of PAH and PH-ILD and other indications, as may be permitted under the collaboration agreement.

Pursuant to the collaboration agreement, we granted exclusive, sublicensable (with our consent required in the United States for third party sublicenses) licenses to Chiesi under intellectual property rights controlled by us relating to Licensed Products, for the worldwide development, manufacture and commercialization of seralutinib and Licensed Products for therapeutic, prophylactic and diagnostic uses in humans and animals.

The licenses granted to Chiesi are subject to retained rights of our Company for the worldwide development and manufacture of seralutinib and Licensed Products, commercialization of Licensed Products in the United States, and performance of our obligations and exercise of our rights that may be set forth in the global development plan and U.S.

commercialization plan, in each case in accordance with the collaboration agreement.

Chiesi granted us a non-exclusive, sublicensable (with Chiesi’s consent required in the US Territory for third party sublicenses) licenses under certain practiced intellectual property rights relating to seralutinib and Licensed Products and arising intellectual property rights, in each case as controlled by Chiesi, for the worldwide development and manufacture of seralutinib and Licensed Product and a co-exclusive license (with Chiesi) to commercialize seralutinib and Licensed Products in the US Territory.

We agreed to use commercially reasonable efforts to conduct development and commercialization activities in relation to seralutinib and Licensed Products, under the global development plan and U.S.

commercialization plan in accordance with the timelines therein.

We will continue to lead global development of seralutinib in PAH and PH-ILD, and we and Chiesi will equally share the costs for the activities included in the global development plan for all Licensed Products, with the exception of the PROSERA Phase 3 study, which we will be solely responsible for conducting at our own cost and expense.

With respect to each country in the ROW Territory, such obligation to equally share such development costs shall end when regulatory approval is received for a Licensed Product in such country.

With respect to United States, the development costs incurred following regulatory approval shall continue to be shared equally.

We will lead commercialization for PAH and PH-ILD in the United States, with both parties contributing 50 percent of commercial efforts, including performing 50 percent of the commercialization activities.

Chiesi will lead commercialization in the United States in additional indications, and Chiesi will have the exclusive right to commercialize Licensed Products in the ROW Territory.

Chiesi further agreed to use commercially reasonable efforts to commercialize Licensed Product in certain specified countries in the ROW Territory following receipt of regulatory approvals.

Generally, we will have the right to lead in manufacturing commercial supply of seralutinib and Licensed Products for the United States for PAH and PH-ILD, and, subject to any of our existing obligations of to third party manufacturers, Chiesi will have the right to lead in manufacturing commercial supply of seralutinib and Licensed Products in the ROW Territory, in each case in accordance with the collaboration agreement.

Pursuant to the collaboration agreement, neither party nor its affiliates is permitted to develop or commercialize any compound or product throughout the term whose primary mechanism of action is inhibition of a tyrosine kinase for the treatment of PAH or PH-ILD in the United States or ROW Territory, subject to certain restrictions for the EU and UK.

In consideration and as reimbursement for our development costs, Chiesi agreed to pay us $160.0 million.

Additionally, we will be eligible to receive up to $146.0 million in regulatory milestones and $180.0 million in sales milestones.

In the United States, the parties agreed to share commercial profits and losses equally.

In the ROW Territory, Chiesi will pay us an escalating mid-to-high teens percentage royalty, subject to standard deductions, on net sales of Licensed Product for PAH and additional indications on a Licensed Product-by-Licensed Product and country-by-country basis with such payment obligations beginning on the first commercial sale of Licensed Product in such country and expiring on a country-by-country basis on the latest of (a) the expiration of a valid claim to our patent right in such country, (b) the expiration of regulatory exclusivity, and (c) the date that is 10 years after the first commercial sale of such Licensed Product in such country.

In addition, we granted to Chiesi an option to purchase directly from us, on one or more occasions, up to an aggregate number of shares of our common stock such that immediately following such issuance, Chiesi’s beneficial ownership of our common stock shall not exceed 9.9% of the total number of issued and outstanding shares of our common stock, or the Equity Option.

The Equity Option shall be exercisable by Chiesi, in whole or in part, at any time prior to the earliest to occur of the date on which (a) the last patient is last dosed in either (i) the PROSERA Phase 3 study for PAH or (ii) a Phase 3 clinical trial for the PH-ILD Indication, (b) any third party commences a tender offer or exchange offer for more than 50% of the outstanding shares of our common stock, and (c) we publicly announces our intent to consummate a GB002 change of control.

The purchase price of each share of our common stock subject to the Equity Option shall be equal to 107.5% of the daily volume-weighted average per share price of our common stock on The Nasdaq Stock Market over the 30-trading day period ending on and including the last trading day prior to the date on which Chiesi delivers an exercise notice to us;

provided that such purchase price shall be no less than $1.63 per share.

The shares of our common stock to be issued will be issued in a private placement in reliance on Section 4(a)(2) of the Securities Act of 1933, as amended, for transactions by an issuer not involving any public offering, pursuant to the terms of a stock issuance agreement to be entered into between us and Chiesi in connection with each such exercise of the Equity Option.

Unless earlier terminated, the collaboration agreement will remain in force until no Licensed Products are being developed or commercialized in the United States and in the ROW Territory, on a country-by-country basis, until no royalty terms are in effect for all countries.

Either party may terminate the collaboration agreement for the other party’s material breach, subject to a specified notice and cure periods, or due to an insolvency event of the other party.

In lieu of termination upon a party’s material breach due to non-payment of development costs within a specified time the non-breaching party may elect an alternative remedy which may involve modifications to their performance and payment obligations.

We have the right to terminate by providing written notice in the event Chiesi or its affiliates or sublicensee brings a patent challenge and Chiesi

does not take certain steps to withdraw from or cease supporting such challenge.

Chiesi may terminate the collaboration agreement for any reason upon prior written notice to us, subject to a notice period.

Manufacturing

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to preserve the confidentiality of our trade secrets;

to defend and enforce our proprietary rights, including any patents that we may

own in the future;

to defend and enforce our proprietary rights, including any patents that we may own in the future;

and to operate without infringing on the valid and enforceable patents and other proprietary rights of third parties.

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Patent application, which, if issued, is not due to expire before 2037, excluding any additional term for patent term extension;

and a number of patents and pending applications in other jurisdictions, including issued patents in Mexico and Russia, and pending applications in Australia, Brazil, Canada, China, the European Patent Convention, India, Japan, South Korea, and New Zealand, which, if issued, are not due to expire before 2037, excluding any additional term for patent term extension.

and a number of patents and pending applications in other jurisdictions, including issued patents in Mexico, Russia, Australia, India, Japan, South Korea, and New Zealand, and pending applications in, Brazil, Canada, China, the European Patent Convention, and New Zealand, which, if issued, are not due to expire before 2037, excluding any additional term for patent term extension.

These patents and patent applications are directed to method of use claims.

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patent applications which, if issued, are not due to expire before 2034, excluding any additional term for patent term extension;

and a number of patents and pending patent applications in other jurisdictions, including issued patents in Australia, Canada, China, the European Patent Convention and Japan, and pending applications in Australia, China, the European Patent Convention and Japan.

and a number of patents and pending patent applications in other jurisdictions, including issued patents in Australia, Canada, China, the European Patent Convention, Japan, and Hong Kong.

These patents and patent applications are directed to seralutinib compound, formulation and method of use claims.

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In the case of seralutinib, the primary mode of action is attributable to the drug component of the product, which means that the FDA’s Center for Drug Evaluation and Research has primary jurisdiction over the premarket development, review and approval.

Accordingly, we plan to investigate seralutinib through the IND framework and seek approval through the NDA pathway.

We do not anticipate that the FDA will require a separate medical device authorization for the device, but this could change during the course of its review of any marketing application that we may submit.

Accordingly, we plan to investigate seralutinib through the Investigational New Drug, or IND, framework and seek approval through the NDA pathway.

We do not anticipate

that the FDA will require a separate medical device authorization for the device, but this could change during the course of its review of any marketing application that we may submit.

Drug Development Process

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All clinical trials must be conducted under the supervision of one or more qualified investigators in accordance with GCP regulations, which among other things, include the requirement that all research subjects provide their informed consent in writing for their participation in any clinical trial.

Clinical Trials must be conducted under protocols detailing, among other things, the objectives of the trial, dosing procedures, subject selection and exclusion criteria and the safety and effectiveness criteria to be evaluated.

Clinical Trials must be conducted under protocols

detailing, among other things, the objectives of the trial, dosing procedures, subject selection and exclusion criteria and the safety and effectiveness criteria to be evaluated.

Each protocol must be submitted to the FDA as part of the IND as well as any subsequent protocol amendments.

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Information related to the product, patient population, phase of investigation, trial sites and investigators and other aspects of the clinical trial is then made public as part of the registration.

Sponsors are also obligated to

disclose the results of their clinical trials after completion.

Sponsors are also obligated to disclose the results of their clinical trials after completion.

Disclosure of the results of these trials can be delayed until the new product or new indication being studied has been approved.

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The manufacturing process must be capable of consistently producing quality batches of the product candidate and, among other things, the manufacturer must develop methods for testing the identity, strength, quality and purity of the final drug.

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.

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.

Regulation of Combination Products in the United States

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

Once filed, the FDA reviews an NDA 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.

strength, quality and purity.

Under the Prescription Drug User Fee Act, or 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 to review and act on the submission.

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If a product that has orphan designation subsequently receives the first FDA approval for the disease or condition for which it has such designation, the product is entitled to orphan product exclusivity, which means that the FDA may not approve any other applications to market the same drug for the same disease or condition for seven years, except in limited circumstances, such as a showing of clinical superiority to the product with orphan exclusivity or inability to manufacture the product in sufficient quantities.

The designation of such drug also entitles a party to financial incentives such as opportunities for grant funding toward clinical trial costs, tax advantages and user-fee waivers.

The designation of such drug also entitles a party to financial incentives such

as opportunities for grant funding toward clinical trial costs, tax advantages and user-fee waivers.

However, competitors, may receive approval of different products for the indication for which the orphan product has exclusivity or obtain approval for the same product but for a different indication for which the orphan product has exclusivity.

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The sponsor of a fast track product candidate has opportunities for more frequent interactions with the applicable FDA review team during development.

the FDA agrees to accept sections of the NDA and determines that the schedule is acceptable, and the sponsor pays any required user fees upon submission of the first section of the NDA.

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

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 drug products manufactured or distributed pursuant to FDA approvals will be subject to pervasive and continuing regulation by the FDA, including, among other things, record-keeping requirements, reporting of adverse experiences with the drug, providing the FDA with updated safety and efficacy information, drug sampling and distribution requirements, complying with certain electronic records and signature requirements, and complying with FDA promotion and advertising requirements.

in the product’s approved labeling (known as “off-label use”), industry-sponsored scientific and educational activities, and promotional activities involving the internet.

The FDA strictly regulates labeling, advertising, promotion and other types of information on products that are placed on the market and imposes requirements and restrictions on drug manufacturers, such as those related to direct-to-consumer advertising, the prohibition on promoting products for uses or in patient populations that are not described in the product’s approved labeling (known as “off-label use”), industry-sponsored scientific and educational activities, and promotional activities involving the internet.

Discovery of previously unknown problems or the failure to comply with the applicable regulatory requirements may result in restrictions on the marketing of a product or withdrawal of the product from the market as well as possible civil or criminal sanctions.

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Pediatric exclusivity provides for an additional six months of marketing exclusivity attached to an existing period of regulatory exclusivity or patent term if a sponsor conducts clinical trials in children in response to a written request from the FDA.

The issuance of a written request does not require the sponsor to undertake the described clinical trials.

issuance of a written request does not require the sponsor to undertake the described clinical trials.

In addition, orphan drug exclusivity, as described above, may offer a seven-year period of marketing exclusivity, except in certain circumstances.

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(6) created a new Patient-Centered Outcomes Research Institute to oversee, identify priorities in, and conduct comparative clinical effectiveness research, along with funding for such research;

(7) created a new Medicare Part D coverage gap discount program, in which manufacturers must agree to offer 50% (which was increased to 70% commencing January 1, 2019) point-of-sale discounts off negotiated prices of applicable brand drugs to eligible beneficiaries during their coverage gap period, as a condition for the manufacturer’s outpatient drugs to be covered under Medicare Part D;

(7) established a new Patient-Centered Outcomes Research Institute to oversee, identify priorities in, and conduct comparative clinical effectiveness research, along with funding for such research;

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Other legislative changes have been proposed and adopted since the ACA was enacted.

On August 2, 2011, the Budget Control Act of 2011 was signed into law, which, among other things, resulted in aggregate reductions of Medicare payments to providers, which went into effect on April 1, 2013 and, due to subsequent legislative amendments to the statute, will remain in effect through 2032, with the exception of a temporary suspension from May 1, 2020 through March 31, 2022, unless additional Congressional action is taken.

will remain in effect through 2032, with the exception of a temporary suspension from May 1, 2020 through March 31, 2022, unless additional Congressional action is taken.

On January 2, 2013, the American Taxpayer Relief Act of 2012 was signed into law, which, among other things, reduced Medicare payments to several providers, including hospitals, and increased the statute of limitations period for the government to recover overpayments to providers from three to five years.

On March 11, 2021, the American Rescue Plan Act of 2021 was signed into law, which eliminated the statutory Medicaid drug rebate cap, beginning January 1, 2024.

The rebate was previously capped at 100% of a drug’s average manufacturer price.

Moreover, there has recently been heightened governmental scrutiny over the manner in which manufacturers set prices for their marketed products, which has resulted in several Congressional inquiries and proposed and enacted federal and state 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.

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imposes rebates under Medicare Part B and Medicare Part D to penalize price increases that outpace inflation (first due in 2023);

and replaces the Part D coverage gap discount program with a new discounting program (beginning in 2025).

and replaces the Part D coverage gap discount program with a new manufacturer discounting program (which began in 2025).

The IRA permits the Secretary of HHS to implement many of these provisions through guidance, as opposed to regulation, for the initial years.

In August 2023, HHS announced the list of the first ten drugs that will be subject to price negotiations, although the Medicare drug price negotiation program is currently subject to legal

CMS has published the negotiated prices for the initial ten drugs, which will first be effective in 2026, and has published the list of the subsequent 15 drugs that will be subject to negotiation, although the Medicare drug price negotiation program is currently subject to legal challenges.

For that and other reasons, it is currently unclear how the IRA will be effectuated.

At the state level, legislatures have 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.

At the state level, legislatures have 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, drug price reporting and other transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing.

Some states have enacted legislation creating so-called prescription drug affordability boards, which ultimately may attempt to impose price limits on certain drugs in these states.

We expect that healthcare reform measures that may be adopted in the future may result in more rigorous coverage criteria, new payment methodologies and additional downward pressure on the price that we receive for any approved product.

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The federal civil and criminal false claims laws, including the civil False Claims Act, prohibit, among other things, any individual or entity from knowingly presenting, or causing to be presented, a false claim for payment to the federal government or knowingly making, using or causing to be made or used a false record or statement material to a false or fraudulent claim to the federal government.

In addition, the government may assert that a claim including items or services resulting from a violation of the federal Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the civil False Claims Act and the civil monetary penalties statute.

The federal Health Insurance Portability and Accountability Act of 1996, or HIPAA, created additional federal civil and criminal statutes that prohibit, among other things, knowingly and willfully executing a scheme to defraud any healthcare benefit program.

Similar to the federal Anti-Kickback Statute, A person or entity does not need to have actual knowledge of this statute or specific intent to violate it in order to have committed a violation.

The federal Physician Payments Sunshine Act requires certain manufacturers of drugs, devices, biologics and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program, with specific exceptions, to report annually to CMS information related to payments or other transfers of value made to physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors), certain non-physician providers including physician assistants and nurse practitioners, and teaching hospitals, and applicable manufacturers and applicable group purchasing organizations to report annually to CMS ownership and investment interests held by physicians (as defined by statute) and their immediate family members.

purchasing organizations to report annually to CMS ownership and investment interests held by physicians (as defined by statute) and their immediate family members.

Similar state, local and foreign laws and regulations may also restrict business practices in the biopharmaceutical industry, such as state anti-kickback and false claims laws, which may apply to business practices, including but not limited to, research, distribution, sales and marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental third-party payors, including private insurers, or by patients themselves;

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Additionally, there has been a recent trend of increased regulation of payments and transfers of value provided to healthcare professionals or entities and many EU member states have adopted national “Sunshine Acts” which impose reporting and transparency requirements (often on an annual basis), similar to the requirements in the United States, on pharmaceutical companies.

countries also mandate implementation of commercial compliance programs, or require disclosure of marketing expenditures and pricing information.

Certain countries also mandate implementation of commercial compliance programs, or require disclosure of marketing expenditures and pricing information.

Efforts to ensure compliance with applicable healthcare laws and regulations can involve substantial costs.

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Foreign Regulation

In order to market any product outside of the United States, we need to comply with numerous and varying regulatory requirements of other countries and jurisdictions regarding quality, safety and efficacy and governing, among other things, clinical trials, marketing authorization, commercial sales and distribution of our products.

Whether or not we obtain FDA approval for a product, we would need to obtain the necessary approvals by the comparable foreign regulatory authorities before we can commence clinical trials or marketing of the product in foreign countries and jurisdictions.

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Non-clinical studies are performed to demonstrate the health or environmental safety of new chemical or biological substances.

Non-clinical (pharmaco-toxicological) studies must be conducted in compliance with the principles of good laboratory practice, or GLP, as set forth in EU Directive 2004/10/EC (unless otherwise justified for certain particular medicinal products – e.g., radio-pharmaceutical precursors for radio-labelling purposes).

Non-clinical (pharmaco-toxicological) studies must be conducted in compliance with the principles of good laboratory practice, or GLP, as set forth in EU Directive 2004/10/EC (unless otherwise justified for certain particular medicinal products, e.g., radio-pharmaceutical precursors for radio-labeling purposes).

In particular, non-clinical studies, both in vitro and in vivo, must be planned, performed, monitored, recorded, reported and archived in accordance with the GLP principles, which define a set of rules and criteria for a quality system for the organizational process and the conditions for non-clinical studies.

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If the sponsor of the clinical trial is not established within the EU, it must appoint an EU entity to act as its legal representative.

must take out a clinical trial insurance policy, and in most EU countries, the sponsor is liable to provide ‘no fault’ compensation to any study subject injured in the clinical trial.

The sponsor must take out a clinical trial insurance policy, and in most EU countries, the sponsor is liable to provide ‘no fault’ compensation to any study subject injured in the clinical trial.

The regulatory landscape related to clinical trials in the EU has been subject to recent changes.

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Once the CTA is approved, clinical trial development may proceed.

The CTR foresees a three-year transition period.

The extent to which ongoing and new clinical trials will be governed by the CTR varies.

Clinical trials for which an application was submitted (i) prior to January 31, 2022 under the EU Clinical Trials Directive, or (ii) between January 31, 2022 and January 31, 2023 and for which the sponsor has opted for the application of the EU Clinical Trials Directive remain governed by said Directive until January 31, 2025.

After this date, all clinical trials (including those which are ongoing) will become subject to the provisions of the CTR.

The CTR transition period ended on January 31, 2025, and all clinical trials (and related applications) are now fully subject to the provisions of the CTR.

Medicines used in clinical trials must be manufactured in accordance with Good Manufacturing Practices , or GMP.

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Marketing Authorization

To market a medicinal product in the EU, we must obtain a marketing authorization, or MA.

To market a medicinal product in the EU, we must obtain a MA.

To obtain regulatory approval of an investigational medicinal product under EU regulatory systems, we must submit a MA application, or MAA.

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The centralized procedure is mandatory for certain types of products, such as:

(i) medicinal products derived from biotechnology medicinal products, (ii) designated orphan medicinal products, (iii) advanced therapy medicinal products, or ATMPs, and (iv) medicinal products containing a new active substance indicated for the treatment certain diseases, such as HIV/AIDS, cancer, neurodegenerative diseases, diabetes, auto-immune and other dysfunctions and viral diseases.

(i) medicinal products derived from biotechnology medicinal

products, (ii) designated orphan medicinal products, (iii) advanced therapy medicinal products, or ATMPs, and (iv) medicinal products containing a new active substance indicated for the treatment certain diseases, such as HIV/AIDS, cancer, neurodegenerative diseases, diabetes, auto-immune and other dysfunctions and viral diseases.

The centralized procedure is optional for products containing a new active substance not yet authorized in the EU, or for products that constitute a significant therapeutic, scientific or technical innovation or which are in the interest of public health in the EU.

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In the EU, new products authorized for marketing, or reference products, generally receive eight years of data exclusivity and an additional two years of market exclusivity upon MA.

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

product was first authorized in the EU.

During the additional two‑year period of market exclusivity, a generic or biosimilar MAA can be submitted, and the innovator’s data may be referenced, but no generic or biosimilar product can be marketed until 10 years have elapsed from the initial MA of the reference product in the EU.

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In the EU, MAAs for new medicinal products have to include the results of trials conducted in the pediatric population, in compliance with a pediatric investigation plan, or PIP, agreed with the EMA’s Pediatric Committee, or PDCO.

The PIP sets out the timing and measures proposed to generate data to support a pediatric indication of the drug for which marketing authorization is being sought.

The PIP sets out the timing and measures proposed to generate data to support a pediatric indication of the product candidate for which MA is being sought.

The PDCO can grant a deferral of the obligation to implement some or all of the measures of the PIP until there are sufficient data to demonstrate the efficacy and safety of the product in adults.

Further, the obligation to provide pediatric clinical trial data can be waived by the PDCO when these data is not needed or appropriate because the product is likely to be ineffective or unsafe in children, the disease or condition for which the product is intended occurs only in adult populations, or when the product does not represent a significant therapeutic benefit over existing treatments for pediatric patients.

Once the MA is obtained in all EU member states and study results are included in the product

information, even when negative, the product is eligible for six months’ supplementary protection certificate extension (if any is in effect at the time of approval) or, in the case of orphan pharmaceutical products, a two year extension of the orphan market exclusivity is granted.

Orphan drug designation

Once the MA is obtained in all EU member states and study results are included in the product information, even when negative, the product is eligible for six months’ supplementary protection certificate extension (if any is in effect at the time of approval) or, in the case of orphan medicinal products, a two year extension of the orphan market exclusivity is granted.

Orphan designation

The criteria for designating an “orphan medicinal product” in the EU are similar in principle to those in the United States.

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Similar to the United States, both MA holders and manufacturers of medicinal products are subject to comprehensive regulatory oversight by the EMA, the EC and/or the competent regulatory authorities of the member states.

The holder of a MA must establish and maintain a pharmacovigilance system and appoint an individual qualified person for pharmacovigilance, or QPPV, who is responsible for establishment and maintenance of that system, and oversees the safety profiles of medicinal products and any emerging safety concerns.

profiles of medicinal products and any emerging safety concerns.

Key obligations include expedited reporting of suspected serious adverse reactions and submission of periodic safety update reports, or PSURs.

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The aforementioned EU rules are generally applicable in the European Economic Area, or EEA, which consists of the 27 EU member states plus Norway, Liechtenstein and Iceland.

Failure to comply with EU and member state laws that apply to the conduct of clinical trials, manufacturing approval, MA of medicinal products and marketing of such products, both before and after grant of the MA, manufacturing of pharmaceutical products, statutory health insurance, bribery and anti-corruption or with other applicable regulatory

requirements may result in administrative, civil or criminal penalties.

These penalties could include delays or refusal to authorize the conduct of clinical trials, or to grant MA, product withdrawals and recalls, product seizures, suspension, withdrawal or variation of the MA, total or partial suspension of production, distribution, manufacturing or clinical trials, operating restrictions, injunctions, suspension of licenses, fines and criminal penalties.

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Brexit and the Regulatory Framework in the United Kingdom

Since the end of the Brexit transition period on January 1, 2021, Great Britain (England, Scotland and Wales) has not been directly subject to EU laws, however under the terms of the Ireland/Northern Ireland Protocol, EU laws generally apply to Northern Ireland.

It is currently unclear to what extent the Government of the United Kingdom, or UK, will seek to align its regulations with the EU.

The EU laws that have been transposed into UK law through secondary legislation remain applicable in Great Britain, however, new legislation such as the (EU) CTR is not applicable in Great Britain.

Since the end of the Brexit transition period on January 1, 2021, and the implementation of the Windsor Framework on January 1, 2025, the United Kingdom, or UK, has not generally been directly subject to EU laws with respect to medicinal products.

It is currently unclear to what extent the government of the UK will seek to align its regulations with the EU.

The EU laws that have been transposed into UK law through secondary legislation remain applicable in Great Britain (England, Scotland and Wales), or GB, however, new legislation such as the (EU) CTR is not applicable in GB.

Whilst the EU-UK Trade and Cooperation Agreement, or TCA, includes the mutual recognition of GMP inspections of manufacturing facilities for medicinal products and GMP documents issued, it does not contain wholesale mutual recognition of UK and EU pharmaceutical regulations and product standards.

There may be divergent local requirements in Great Britain from the EU in the future, which may impact clinical and development activities that occur in the UK in the future.

Similarly, clinical trial submissions in the UK will not be able to be bundled with those of EU member states within the EMA Clinical Trial Information System, or CTIS, adding further complexity, cost and potential risk to future clinical and development activity in the UK.

Significant political and economic uncertainty remains about how much the relationship between the UK and EU will differ as a result of the UK’s withdrawal.

The UK government has passed a new Medicines and Medical Devices Act 2021, which introduces delegated powers in favor of the Secretary of State or an ‘appropriate authority’ to amend or supplement existing regulations in the area of medicinal products and medical devices.

This allows new rules to be introduced in the future by way of secondary legislation,

which aims to allow flexibility in addressing regulatory gaps and future changes in the fields of human medicines, clinical trials and medical devices.

The UK Medicines and Medical Devices Act 2021 has introduced delegated powers in favor of the Secretary of State or an ‘appropriate authority’ to amend or supplement existing regulations in the area of medicinal products and medical devices.

This allows new rules to be introduced in the future by way of secondary legislation, which aims to allow flexibility in addressing regulatory gaps and future changes in the fields of human medicines, clinical trials and medical devices.

Since January 1, 2021, the Medicines and Healthcare products Regulatory Agency, or MHRA, has been the UK’s standalone medicines and medical devices regulator.

As a result of the Northern Ireland protocol, different rules will apply in Northern Ireland than in England, Wales, and Scotland, together, Great Britain, or GB;

broadly, Northern Ireland will continue to follow the EU regulatory regime, but its national competent authority will remain the MHRA.

The MHRA has introduced changes to national licensing procedures, including procedures to prioritize access to new medicines that will benefit patients, including a 150-day assessment and a rolling review procedure.

All existing EU MAs for centrally authorized products were automatically converted or grandfathered into UK MAs, effective in GB (only), free of charge on January 1, 2021, unless the MA holder has to opted-out.

In order to use the centralized procedure to obtain a MA that will be valid throughout the EEA, companies must be established in the EEA.

Therefore after Brexit, companies established in the UK can no longer cannot use the EU centralized procedure and instead an EEA entity must hold any centralized MAs.

In order to obtain a UK MA to commercialize products in the UK, an applicant must be established in the UK and must follow one of the UK national authorization procedures or one of the remaining post-Brexit international cooperation procedures to obtain an MA to commercialize products in the UK.

A new international recognition framework has been in place from January 1, 2024, whereby the MHRA will have regard to decisions on the approval of MAs made by the EMA and certain other regulators when determining an application for a new GB MA.

There is no pre-MA orphan designation.

Instead, the MHRA will review applications for orphan designation in parallel to the corresponding MA application.

The criteria are essentially the same, but have been tailored for the market, i.e., the prevalence of the condition in GB, rather than the EU, must not be more than five in 10,000.

Should an orphan designation be granted, the period or market exclusivity will be set from the date of first approval of the product in GB.

The UK regulatory framework in relation to clinical trials is derived from existing EU legislation (as implemented into UK law, through secondary legislation).

On January 17, 2022, the MHRA launched an eight-week consultation on reframing the UK legislation for clinical trials, which aims to streamline clinical trials approvals, enable innovation, enhance clinical trials transparency, enable greater risk proportionality, and promote patient and public involvement in clinical trials.

The MHRA responded to the consultation on March 21, 2023 and confirmed that it would bring forward changes to the legislation.

The final legal texts introduced by the UK Government will ultimately determine the extent to which whether the UK clinical trials framework aligns with or diverges from with the (EU) CTR.

As a result of the Northern Ireland Protocol, different rules applied in Northern Ireland than in GB;

broadly, Northern Ireland continued to follow the EU regulatory regime.

However, on January 1, 2025, a new arrangement called the “Windsor Framework” came into effect and reintegrated Northern Ireland under the regulatory authority of the MHRA with respect to medicinal products.

The Windsor Framework removes EU licensing processes, and EU labelling and serialization requirements in relation to Northern Ireland, and introduces a UK-wide licensing process for medicinal products.

MAs in the UK are governed by the Human Medicines Regulations (SI 2012/1916), as amended.

Under the terms of the Windsor Framework, these MAs became valid for the whole of the UK from January 1, 2025.

In order to use the centralized procedure to obtain an MA that will be valid throughout the EEA, companies must be established in the EEA.

Therefore, since Brexit, companies established in the UK can no longer use the EU centralized procedure and instead an EEA entity must hold any centralized MAs.

Applications are governed by the Human Medicines Regulations (SI 2012/1916), as amended, and are made electronically through the MHRA Submissions Portal.

The MHRA has introduced changes to national licensing procedures, including procedures to prioritize access to new medicinal products that will benefit patients, including a 150-day assessment (subject to clock-stops) and a rolling review procedure.

In addition, an international recognition procedure, or IRP, has been in place since January 1, 2024, whereby the MHRA will have regard to decisions on the approval of MAs made by the EMA and certain other regulators when determining an application for a new UK MA.

Pursuant to the IRP, the MHRA will take into account the expertise and decision-making of trusted regulatory partners (i.e.

the regulators in Australia, Canada, Switzerland, Singapore, Japan, the U.S.

The MHRA will conduct a targeted assessment of IRP applications but retain the authority to reject applications if the evidence provided is considered insufficiently robust.

The IRP allows medicinal products approved by such trusted regulatory partners that meet certain criteria to undergo a fast-tracked MHRA review to obtain and/or update an MA in the UK.

Applications should be decided within a maximum of 60 days if there are no major objections identified that cannot be resolved within such 60-day period and the approval from the trusted regulatory partner selected has been granted within the previous 2 years or if there are such major objections identified or such approval has not been granted within the previous 2 years within 110 days.

Applicants can submit initial MAAs to the IRP but the procedure can also be used throughout the lifecycle of a product for post-authorization procedures including line extensions, variations and renewals.

In the UK, the initial duration of an MA is five years and following renewal will be valid for an unlimited period unless the MHRA decides on justified grounds relating to pharmacovigilance, to proceed with only one additional 5-year renewal.

Any authorization which is not followed by the actual placing of the medicinal product on the market in the UK within 3 years shall cease to be in force.

There is no pre-MA orphan designation in the UK.

Instead, the MHRA reviews applications for orphan designation in parallel to the corresponding MA application.

The criteria are essentially the same, but have been tailored for the market, i.e., the prevalence of the condition in the UK, rather than the EU, must not be more than five in 10,000.

Should an orphan designation be granted, the period or market exclusivity will be set from the date of first approval of the product in the UK.

The UK regulatory framework in relation to clinical trials is derived from the now-repealed EU Clinical Trials Directive (as implemented into UK law, through the Medicines for Human Use (Clinical Trials) Regulations 2004, as amended).

The extent to which the regulation of clinical trials in the UK will mirror the (EU) CTR in the long term is not yet certain, however, on December 12, 2024, the UK government introduced a legislative proposal - the Medicines for Human Use (Clinical Trials) Amendment Regulations 2024 - that, if implemented, will replace the current regulatory framework for clinical trials in the UK.

The legislative proposal aims to provide a more flexible regime to make it easier to conduct clinical trials in the UK, increase the transparency of clinical trials conducted in the UK and make clinical trials more patient centered.

The UK government has provided the legislative proposal to the UK Parliament for its review and approval.

Once the legislative proposal is approved (with or without amendment), it will be adopted into UK law which is expected in early 2026.

Foreign Data Privacy and Security Laws

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The principal purposes of our equity and cash incentive plans are to attract, retain and motivate personnel through the granting of stock-based and cash-based compensation awards, in order to align our interests and the interests of our stockholders with those of our employees and consultants.

As of February 27, 2024, we had 135 full-time employees and no part-time employees.

As of March 6, 2025, we had 144 full-time employees and 1 part-time employee.

Of those 145 employees, 30, or 21%, have a Ph.D.

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and changed our name to Gossamer Bio, Inc.

Our principal executive offices are located at 3013 Science Park Road, San Diego, California 92121, and our telephone number is (858) 684-1300.

Our principal executive offices are located at 3115 Merryfield Row, Suite 120, San Diego, California 92121, and our telephone number is (858) 684-1300.

Available Information

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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