Think of it as a clinical-stage Sarepta Therapeutics (SRPT), but focused on developing next-generation treatments for Duchenne muscular dystrophy (DMD).

An emerging biotech, similar to an earlier version of Sarepta, targeting severe neuromuscular diseases with advanced oligonucleotide therapies.

• PGN-EDO51: A lead EDO peptide therapeutic candidate in Phase I clinical trials for Duchenne muscular dystrophy (DMD).
• PGN-EDODM1: An EDO peptide-conjugated PMO being developed for the treatment of myotonic dystrophy type 1.
• PGN-EDO53, PGN-EDO45, and PGN-EDO44: Additional EDO therapeutic candidates in development for the treatment of Duchenne muscular dystrophy (DMD).

As a clinical-stage biotechnology company, PepGen Inc. (PEPG) is focused on the research and development of therapeutic candidates for severe neuromuscular and neurologic diseases. At this stage, the company's product candidates are in clinical trials (e.g., PGN-EDO51 in Phase I) and have not yet been commercialized.

Consequently, PepGen Inc. does not currently have major commercial customers, whether they be other companies or individuals. Its primary activities revolve around advancing its drug pipeline through development and clinical trials.

James McArthur, PhD, President and Chief Executive Officer

Dr. James McArthur has over 25 years of industry experience, having cofounded five biotechnology companies, three of which were acquired. He served as a Director for two companies through successful initial public offerings. He was the founding CEO of Imara, a public company, and co-founder and President of R&D of Cydan, a rare disease accelerator. Dr. McArthur was also a founding CSO of Synovex/Adheron, a rheumatology therapy company acquired by Roche. He co-founded Vtesse and Tiburio. Prior to joining PepGen, he served as a Venture Partner at RA Capital Management, which led PepGen's Series A financing.

Noel Donnelly, MBA, Chief Financial Officer

Mr. Noel Donnelly previously served as the CFO at EIP Pharma Inc., a small private biotech in the neurodegenerative disease space. He spent 15 years at Shire PLC in various leadership roles within both research and development (R&D) and finance functions, most recently as Vice President, R&D Business Operations. In that role, he established and led key strategic processes and teams, including R&D strategy, portfolio prioritization, due diligence, and integrations.

Michelle Mellion, M.D., Chief Medical Officer

Dr. Michelle Mellion leads PepGen's medical initiatives as Chief Medical Officer. Prior to this role, she was Senior Vice President, Head of Clinical Development at PepGen. Dr. Mellion has extensive experience in clinical development and neurology, having led the development of the FSHD program at Fulcrum Therapeutics through Phase 2 clinical trials. She also held leadership positions at Vertex Pharmaceuticals and Biogen. Before transitioning to industry, she was an Attending Neurologist in the Muscular Dystrophy Association (MDA) clinic and an Assistant Professor of Neurology at Brown University.

Kasra Kasraian, PhD, Chief Technology Officer

Dr. Kasra Kasraian joined PepGen from bluebird bio, where he held various roles of increasing responsibility, including Senior Vice President, Quality, Regulatory Affairs, and CMC Sciences, and Senior Vice President, Technical Development and Operations. At bluebird bio, he played a central role in the approval and launch of three gene therapy products (SKYSONA™, ZYNTEGLO™, and LYFGENIA™). Earlier in his career, Dr. Kasraian led the Technical Operation, Quality, and Regulatory CMC functions at Zafgen, Inc..

Joseph Vittiglio, Esq., Chief Business and Legal Officer

Mr. Joseph Vittiglio brings over two decades of executive leadership experience in legal, compliance, corporate development, and governance within public biotechnology companies. Before joining PepGen, he served as Chief Business and Legal Officer and Corporate Secretary at bluebird bio, where he supported the approval and launch of three U.S. gene therapy products and completed over $400 million in financings. He was also Chief Business and Legal Officer at Finch Therapeutics, guiding the company's IPO and building its public-company governance and compliance functions. Previously, he was Chief Business Officer and General Counsel at AMAG Pharmaceuticals.

The most significant risk to PepGen's business is the Clinical Trial and Regulatory Approval Risk. As a clinical-stage company, its success is highly dependent on its product candidates successfully navigating lengthy, costly, and uncertain preclinical and clinical development processes, and ultimately receiving regulatory approval. PepGen has a history of regulatory challenges, including a recent partial clinical hold placed by the U.S. Food and Drug Administration (FDA) on its FREEDOM2-DM1 Phase 2 trial for PGN-EDODM1, its lead candidate for myotonic dystrophy type 1 (DM1). This hold was due to concerns regarding preclinical pharmacology and toxicology studies, and the company has previously experienced similar regulatory interruptions. Additionally, PepGen voluntarily discontinued the development of PGN-EDO51 for Duchenne muscular dystrophy in May 2025, after the FDA had previously issued a clinical hold on its Phase 2 trial. There is no guarantee that any of PepGen's investigational therapies will successfully complete clinical development or gain regulatory authority approval.

A second key risk is Financial Risk and the Need for Additional Capital. PepGen has a history of operating losses and anticipates incurring further losses as it continues its research and development activities. With no approved products generating revenue, the company relies heavily on external funding sources, such as equity offerings and private placements, to finance its operations. While the company currently has cash reserves to fund operations into the second half of 2027, it will require substantial additional capital beyond this period. Failure to secure this funding on favorable terms could materially harm the business and may lead to further dilution for existing shareholders.

Finally, PepGen faces Product Pipeline Concentration Risk. Following the discontinuation of PGN-EDO51, the company's product pipeline is now heavily concentrated on PGN-EDODM1 for myotonic dystrophy type 1. This high degree of dependence on a single lead product candidate means that any significant setback or failure in the development, clinical trials, or regulatory approval of PGN-EDODM1 would have a disproportionately adverse impact on PepGen's future prospects and overall business viability.

PepGen Inc. focuses on developing therapies for severe neuromuscular and neurologic diseases, with lead product candidates targeting Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1).

Addressable Markets:

• PGN-EDO51 for Duchenne Muscular Dystrophy (DMD): The global Duchenne muscular dystrophy drugs market was valued at approximately USD 2.3 billion in 2023 and is projected to reach USD 5.5 billion by 2032, demonstrating a Compound Annual Growth Rate (CAGR) of 9.96% during this period. Another estimate indicates the global Duchenne muscular dystrophy treatment market size was valued at USD 4.63 billion in 2025 and is projected to grow to USD 19.18 billion by 2034, exhibiting a CAGR of 17.1%. The Duchenne muscular dystrophy market across the seven major markets (7MM), which include the US, France, Germany, Italy, Spain, the UK, and Japan, is projected to grow from USD 2.3 billion in 2023 to USD 5.2 billion by 2033. The United States is expected to maintain its dominant position in the DMD market, commanding 84.8% of the total market share in the 7MM. The U.S. market alone for Duchenne muscular dystrophy was approximately USD 1.9 billion in 2023.
• PGN-EDODM1 for Myotonic Dystrophy Type 1 (DM1): The global Myotonic Dystrophy Type 1 (DM1) market is projected to grow from USD 1,550 million in 2024 to USD 3,204 million by 2032, with a CAGR of 9.5%. Another forecast estimates the global myotonic dystrophy treatment market to reach approximately USD 2,789.35 million by 2033, increasing at a CAGR of 12.3% between 2023 and 2033. The Myotonic Dystrophy therapeutics market is projected to grow at a robust CAGR of 18.4% from 2020 to 2034. In 2023, the United States accounted for nearly 54,068 diagnosed prevalent cases of Myotonic Dystrophy, out of approximately 105,362 cases in the 7MM.

PepGen Inc. (PEPG) is a clinical-stage biotechnology company primarily focused on developing oligonucleotide therapeutics for severe neuromuscular and neurologic diseases. Following the discontinuation of its Duchenne muscular dystrophy (DMD) programs in May 2025, the company has concentrated its resources on its promising myotonic dystrophy type 1 (DM1) program. Expected drivers of future revenue growth over the next 2-3 years are intrinsically linked to the successful progression and potential commercialization of its lead candidate, PGN-EDODM1, and the broader validation of its Enhanced Delivery Oligonucleotide (EDO) platform.

1. Successful Clinical Development and Regulatory Approval of PGN-EDODM1 for Myotonic Dystrophy Type 1 (DM1): The primary driver of future revenue growth for PepGen hinges on the successful advancement and eventual regulatory approval of PGN-EDODM1. The company is currently conducting the FREEDOM2 Phase 2 clinical trial for PGN-EDODM1 in patients with DM1, with data from the 5 mg/kg cohort expected in the first quarter of 2026 and from the 10 mg/kg cohort in the second half of 2026. Positive outcomes from these trials are crucial for moving towards later-stage studies and ultimately seeking marketing authorization. PGN-EDODM1 has already received Orphan Drug and Fast Track Designations in the U.S., as well as Orphan Drug Designation in the EU, which could expedite its development and review process.
2. Market Penetration in the Myotonic Dystrophy Type 1 (DM1) Therapeutic Area: The myotonic dystrophy type 1 (DM1) market represents a significant opportunity for PepGen. There are currently no approved disease-modifying therapies that address the underlying cause of DM1, positioning PGN-EDODM1 as a potential first-in-class treatment. A successful launch and uptake of PGN-EDODM1 could establish PepGen as a leader in this underserved market, which affects over 115,000 patients in the U.S. and EU. The company's unique EDO platform, designed for enhanced delivery and splicing correction, could offer a significant competitive advantage.
3. Validation and Expansion of the Enhanced Delivery Oligonucleotide (EDO) Platform: While PGN-EDODM1 is the immediate focus, its success would validate PepGen's proprietary EDO platform, which is engineered to optimize tissue penetration, cellular uptake, and nuclear delivery of oligonucleotide therapeutics. The EDO platform is also being explored for other therapeutic candidates, such as PGN-EDO53, PGN-EDO45, and PGN-EDO44 for DMD (though DMD programs are currently discontinued, the platform itself is still a long-term asset). Success with PGN-EDODM1 could de-risk the platform and potentially lead to the development of additional product candidates or attract collaboration opportunities, thereby expanding future revenue streams beyond DM1.

Share Issuance

• In September 2025, PepGen completed an underwritten public offering of 35,937,500 shares of its common stock at $3.20 per share, raising approximately $115 million. The proceeds are intended to fund ongoing research and clinical development efforts, including the FREEDOM-DM1 and FREEDOM2-DM1 clinical trials, as well as for working capital and other general corporate purposes.
• The number of shares outstanding for PepGen increased by 33.69% in the year leading up to March 7, 2026.
• As of December 31, 2025, PepGen had approximately 68,874,944 shares outstanding.

Capital Expenditures

• PepGen's capital expenditures for the latest trailing twelve months (TTM) stood at $264,000.
• Over the past five years, the company's average capital expenditure has been $1.59 million.
• The highest capital expenditure recorded by PepGen over the past five years was $3.75 million in 2022.