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Here are 1-3 brief analogies for PepGen (PEPG):

• An earlier-stage Sarepta Therapeutics, but focused on a novel delivery platform for genetic muscle diseases.
• Like an early-stage Vertex Pharmaceuticals, developing drugs for rare muscle disorders using a breakthrough delivery platform.
• A biotech company, similar to an early-stage Moderna, applying a breakthrough delivery platform for genetic muscle disorders.

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• PGN-EDO51: An investigational drug candidate currently in clinical trials for treating Duchenne muscular dystrophy patients amenable to exon 51 skipping.
• PGN-EDO53: A preclinical investigational drug candidate for Duchenne muscular dystrophy, designed to promote exon 53 skipping.
• Enhanced Delivery Oligonucleotide (EDO) Platform: A proprietary technology designed to improve the delivery and potency of therapeutic oligonucleotides for neuromuscular and neurological diseases.

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James McArthur, PHD. President and Chief Executive Officer

Dr. McArthur has over 25 years of industry experience and has cofounded five biotechnology companies, three of which were acquired. He also served as a Director for two companies through successful initial public offerings. He was the founding CEO of Imara, a public company developing therapies for sickle cell disease and beta-thalassemia, co-founder and President of R&D of Cydan, a rare disease accelerator, and founding CSO of Synovex/Adheron, a rheumatology therapy company acquired by Roche. Prior to these roles, he held senior research leadership positions in various gene and stem cell therapy companies. He was also a Venture Partner at RA Capital and an entrepreneur in residence at HealthCare Ventures. Dr. McArthur previously co-founded Vtesse and Tiburio, which were acquired or went public. He joined PepGen from RA Capital Management, where he served as a Venture Partner.

Noel Donnelly, MBA. Chief Financial Officer

Mr. Donnelly serves as the Chief Financial Officer at PepGen. Before joining PepGen, he was the CFO at EIP Pharma Inc., a private biotech company focused on neurodegenerative diseases. Prior to EIP Pharma, he spent 15 years at Shire PLC, a global biopharma acquired by Takeda Pharmaceuticals, where he held various leadership roles within both research and development (R&D) and finance functions. His positions at Shire included Vice President, R&D Business Operations, where he led strategic processes, portfolio prioritization, due diligence, and integrations, and Head of R&D Finance.

Kasra Kasraian, PHD. Chief Technology Officer

Dr. 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™. Before bluebird bio, Dr. Kasraian led the Technical Operation, Quality, and Regulatory CMC functions at Zafgen, Inc.. He also held scientific and leadership roles at Biogen, Wyeth Biotech, Pfizer Inc., and Genetics Institute.

Paul Streck, MD, MBA. Executive Vice President, Head of Research & Development

Dr. Streck serves as Executive Vice President, Head of Research & Development at PepGen. Prior to PepGen, he served as Chief Medical Officer at Albireo Pharma, a publicly traded rare disease biotechnology company acquired by Ipsen in March 2023. He was also Chief Medical Officer at Arena Pharmaceuticals, a publicly traded immuno-inflammatory biotechnology company acquired by Pfizer in March 2022. Earlier, he served as Chief Medical Officer at Alder Biopharmaceuticals, which was acquired by Lundbeck Pharmaceuticals in October 2020, and as the Chief Medical Officer of Insmed, where he advanced the company from clinical to commercial stage.

Afsaneh Mohebbi, PHD. Senior Vice President, Portfolio and Program Management

Dr. Mohebbi serves as Senior Vice President, Portfolio and Program Management at PepGen. She previously worked as a consultant to several small biotech companies, including PepGen since January 2024, assisting with portfolio and program management and cross-functional development activities. Her career includes leadership positions such as Vice President, Program Management, Rare Diseases at Agios Pharmaceuticals, and Head of Program and Alliance Management at BIND Therapeutics, which was acquired by Pfizer. She also held the role of Senior Program Executive, Rare Diseases at Shire Human Genetic Therapies.

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1. Financial Risks and Going Concern: PepGen has a history of significant operating losses and anticipates continued losses as it invests heavily in research and development. The company relies on external funding, such as equity offerings and collaborations, to sustain its operations and advance its clinical programs. Its existing cash resources are not expected to fund operations for an extended period, leading to substantial doubt about its ability to continue as a going concern without securing additional capital. This constant need for funding carries a risk of dilution for existing shareholders and may not always be available on favorable terms.
2. Clinical Development and Regulatory Risks: As a clinical-stage biotechnology company, PepGen's success is dependent on the successful, lengthy, and expensive process of developing and obtaining regulatory approval for its product candidates. The company has faced significant setbacks, including clinical holds by regulatory bodies like the FDA and Health Canada due to safety concerns. Notably, PepGen discontinued its PGN-EDO51 program for Duchenne muscular dystrophy (DMD) after it failed to meet expected dystrophin targets in clinical trials, leading to a substantial drop in stock price and a class-action lawsuit. The outcomes of preclinical and clinical studies are uncertain and not necessarily predictive of future results.
3. Intellectual Property and Market Competition Risks: PepGen's business is highly dependent on its proprietary Enhanced Delivery Oligonucleotide (EDO) platform and the exclusivity of its product candidates, which are protected by patents. The inability to secure, maintain, or defend its intellectual property could significantly harm its business. Additionally, the biotechnology sector is intensely competitive, with numerous larger and better-funded companies developing similar or alternative therapies for neuromuscular and neurological diseases. This competitive landscape poses a risk that other companies may develop superior treatments, or that PepGen's products, if approved, may not gain market acceptance or adequate reimbursement.

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The advancement and market penetration of gene therapies for Duchenne muscular dystrophy (DMD) represent a clear emerging threat to PepGen's core business. With Sarepta Therapeutics' Elevidys already approved and in the market, and other gene therapy programs from companies like Pfizer progressing through clinical trials, this modality offers a potentially one-time treatment approach, fundamentally differing from PepGen's chronic oligonucleotide therapies. This shift in treatment paradigm could capture significant market share and influence patient and physician preference away from chronic therapies, similar to how new technologies disrupted established markets in historical examples.

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PepGen Inc. (PEPG) focuses on developing oligonucleotide therapeutics for severe neuromuscular and neurologic diseases. The company's main product candidates include treatments for Myotonic Dystrophy Type 1 (DM1) and Duchenne Muscular Dystrophy (DMD). PepGen has strategically shifted its focus to its myotonic dystrophy type 1 (DM1) program (PGN-EDODM1) and discontinued its Duchenne muscular dystrophy (DMD) program (PGN-EDO51) to prioritize resources for the DM1 therapy.

Myotonic Dystrophy Type 1 (DM1)

The global market for Myotonic Dystrophy Type 1 (DM1) is projected to grow from USD 1,550 million in 2024 to USD 3,204 million by 2032, at a Compound Annual Growth Rate (CAGR) of 9.5% over the forecast period. Approximately 40,000 patients in the U.S. and 75,000 in Europe are affected by DM1, with over 115,000 patients globally facing progressive disability with no approved therapies to slow or reverse symptoms.

Duchenne Muscular Dystrophy (DMD)

While PepGen has discontinued its DMD program, the overall Duchenne Muscular Dystrophy (DMD) drugs market size was estimated at USD 4.1 billion globally in 2024 and is projected to reach approximately USD 19.46 billion by 2034, growing at a CAGR of 16.85% from 2025 to 2034. In the U.S. specifically, the DMD drugs market was valued at USD 1.32 billion in 2024 and is projected to reach around USD 6.38 billion by 2034, with a CAGR of 17.06% from 2025 to 2034. North America held the largest revenue share in the global DMD drugs market, accounting for 46% in 2024 and 45% in 2023.

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1. Advancement and Potential Commercialization of PGN-EDODM1 for Myotonic Dystrophy Type 1 (DM1): PepGen's lead program, PGN-EDODM1, a peptide-conjugated oligonucleotide candidate for DM1, is a significant driver. The company expects to report safety, 28-day splicing, and functional benefit data from the 15 mg/kg cohort of the FREEDOM-DM1 trial in the second half of 2025. Additionally, results from the 5 mg/kg cohort of the FREEDOM2 trial are anticipated in the first quarter of 2026. Positive clinical outcomes and progression towards later-stage trials and regulatory approval will be critical for future revenue.
2. Progression of PGN-EDO51 for Duchenne Muscular Dystrophy (DMD): Another key driver is the development of PGN-EDO51, an investigational candidate for the treatment of DMD, specifically targeting exon 51 skipping. PepGen anticipates reporting dystrophin production data from the 10 mg/kg cohort of the CONNECT1-EDO51 study in patients with DMD in the third quarter of 2025. Despite a clinical hold on the CONNECT2-EDO51 trial in the US, studies are ongoing in the United Kingdom and Canada, and successful progression in these trials will be vital for its eventual market entry and revenue generation.
3. Expansion of the EDO Platform into New Neuromuscular and Neurological Indications: PepGen's proprietary Enhanced Delivery Oligonucleotide (EDO) platform is designed to improve the uptake and activity of conjugated oligonucleotide therapeutics. The company aims to leverage this platform to generate a broader pipeline of therapeutic candidates for additional neuromuscular and neurological diseases. Success in advancing these discovery programs into clinical development will open up new market opportunities and serve as a long-term revenue driver.

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Share Issuance

• PepGen completed an underwritten public offering in September 2025, selling 35,937,500 shares of common stock at $3.20 per share, which generated aggregate gross proceeds of $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 general corporate purposes.
• In February 2024, the company undertook an offering of 7,530,000 shares of common stock, which was expected to yield approximately $76.4 million in net proceeds.
• PepGen became a public company with its common stock listed on Nasdaq under the symbol “PEPG” following its S-1 filing in April 2022.

Inbound Investments

• In September 2025, RA Capital Management, LLC acquired 9,375,000 shares of PepGen's stock for $30,000,000, signifying a substantial investment in the company.