PepGen(PEPG) - 2025 Q1 - Quarterly Results

First Quarter 2025 Overview PepGen's Q1 2025 overview highlights positive initial clinical data for PGN-EDODM1 and upcoming data readouts for DM1 and DMD programs Headline Highlights PepGen reported positive initial clinical data for PGN-EDODM1, showing significant splicing correction, and outlined upcoming data readouts for both its DM1 and DMD programs in the second half of 2025 and first quarter of 2026 - Reported positive DM1 patient data with mean splicing correction of 29.1% following a single dose of PGN-EDODM1 at 10 mg/kg[1] - FREEDOM-DM1 data from the 15 mg/kg cohort is expected in the second half of 2025[1] - CONNECT1-EDO51 data from the 10 mg/kg cohort is expected in the third quarter of 2025[1] CEO Commentary The CEO highlighted the EDO platform's potential, emphasizing the promising clinical data from two programs and describing the next 12 months as pivotal for the DM1 and DMD clinical programs. The company also took steps to extend its cash runway - The EDO platform's unique ability to efficiently deliver oligonucleotides into cell nuclei is the foundation of a differentiated pipeline with promising clinical data[2] - The next 12 months are considered pivotal and catalyst-rich for PepGen and its DM1 and DMD clinical programs[2] - The company has undertaken various measures to extend its cash runway while prioritizing key clinical milestones[2] Recent Program Updates This section details recent clinical trial progress and anticipated milestones for PepGen's PGN-EDODM1 and PGN-EDO51 programs PGN-EDODM1: Myotonic Dystrophy Type 1 (DM1) PepGen reported positive initial data from its Phase 1 FREEDOM-DM1 trial, demonstrating a favorable safety profile and dose-dependent splicing correction. Further data from this program, including a Phase 2 trial, are anticipated in late 2025 and early 2026 Phase 1 FREEDOM-DM1 Single Ascending Dose (SAD) Trial Initial Phase 1 FREEDOM-DM1 trial data showed a favorable safety profile and robust, dose-dependent splicing correction, with further 15 mg/kg cohort data expected in H2 2025 - Positive initial clinical data from the 5 mg/kg and 10 mg/kg dose cohorts were reported in February, showing a favorable emerging safety profile and robust, dose-dependent splicing correction at day 28 following a single dose[3] - PepGen expects to report safety, 28-day splicing, and functional benefit data from the 15 mg/kg cohort during the second half of 2025[3] Phase 2 FREEDOM2-DM1 Multiple Ascending Dose (MAD) Trial Results from the 5 mg/kg cohort of the Phase 2 FREEDOM2-DM1 trial are expected in the first quarter of 2026 - The Company expects to report results from the 5 mg/kg cohort of the FREEDOM2 trial in the first quarter of 2026[3] PGN-EDO51: Duchenne Muscular Dystrophy (DMD) The company anticipates reporting dystrophin production data from the CONNECT1 trial in Q3 2025. The CONNECT2 trial has been voluntarily paused pending review of CONNECT1 results Phase 2 CONNECT1-EDO51 Open-Label MAD Clinical Trial PepGen expects to report additional safety and dystrophin production data from the 10 mg/kg cohort of the CONNECT1 trial in Q3 2025 - PepGen expects to report additional safety as well as dystrophin production data from the 10 mg/kg cohort of the CONNECT1 trial in DMD patients in the third quarter of 2025[7] Phase 2 CONNECT2-EDO51 MAD Clinical Trial The Phase 2 CONNECT2 trial for DMD has been voluntarily paused pending review of results from the CONNECT1 trial's 10 mg/kg cohort - In March 2025, the Company announced its voluntary decision to pause the Phase 2 CONNECT2 trial in DMD patients until it can review results from the 10 mg/kg cohort in the ongoing Phase 2 CONNECT1 trial[7] Corporate Updates This section covers recent corporate developments, including board appointments, conference presentations, and upcoming key milestones Board of Directors Appointments PepGen strengthened its Board of Directors with the appointment of two industry veterans in March 2025 - In March 2025, Lisa Wyman and Mitchell H. Finer, PhD, joined the Company's Board of Directors, bringing decades of executive and operational experience in life sciences[7] Conference Presentations PepGen actively participated in key industry conferences, presenting data from its clinical trials and preclinical results for PGN-EDODM1 - In March 2025, the Company gave two oral presentations and presented five posters at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, featuring data from CONNECT1 (DMD) and FREEDOM (DM1)[7] - In May 2025, the Company made an oral presentation and presented two posters at the 2025 Myotonic Dystrophy Foundation (MDF) Conference, highlighting recent FREEDOM clinical data in DM1 and preclinical results for PGN-EDODM1[7] Upcoming Potential Milestones The company has several key data readouts anticipated in the coming quarters for its DM1 and DMD programs - Upcoming Milestones[7] - 3Q 2025: CONNECT1-EDO51 data from 10 mg/kg cohort expected[7] - 2H 2025: FREEDOM-DM1 data from 15 mg/kg cohort expected[7] - 1Q 2026: FREEDOM2-DM1 data from the 5 mg/kg cohort expected[7] Financial Results for the Three Months Ended March 31, 2025 This section presents PepGen's financial performance for Q1 2025, including cash position, operating expenses, and net loss Key Financial Highlights PepGen reported a cash position of $97.8 million as of March 31, 2025, sufficient to fund operations for at least 12 months. The company experienced increased R&D and G&A expenses, leading to a higher net loss compared to the prior year - Cash, Cash Equivalents and Marketable Securities were $97.8 million as of March 31, 2025[7] - Existing cash, cash equivalents, and marketable securities are believed to be sufficient to fund operations for at least 12 months from the release date[7] Financial Performance (Q1 2025 vs Q1 2024) | Metric | Q1 2025 (USD) | Q1 2024 (USD) | Change (%) | | :-------------------------------- | :------ | :------ | :----- | | Research and Development Expenses | $25.4M | $14.7M | +72.8% | | General and Administrative Expenses | $5.9M | $5.1M | +15.7% | | Net Loss | $(30.2)M | $(18.0)M | +67.8% | | Net Loss per share (basic & diluted) | $(0.92) | $(0.63) | +46.0% | Condensed Consolidated Statements of Operations The condensed consolidated statements of operations detail the company's operating expenses, other income, and net loss for the three months ended March 31, 2025, compared to the same period in 2024 Condensed Consolidated Statements of Operations (unaudited, in thousands) | | | Three Months | Ended | | | --- | --- | --- | --- | --- | | | | March | 31, | | | | | 2025 | | 2024 | | Operating expenses: | | | | | | Research and development | $ | 25,378 | $ | 14,732 | | General and administrative | | 5,943 | | 5,066 | | Total operating expenses | $ | 31,321 | $ | 19,798 | | Operating loss | $ | (31,321) | $ | (19,798) | | Other income (expense) | | | | | | Interest income | | 1,122 | | 1,735 | | Other (expense) income, net | | (3) | | 43 | | Total other income, net | | 1,119 | | 1,778 | | Net loss before income tax | $ | (30,202) | $ | (18,020) | | Income tax expense | | — | | — | | Net loss | $ | (30,202) | $ | (18,020) | | Net loss per share, basic and diluted | $ | (0.92) | $ | (0.63) | | Weighted-average common stock outstanding, basic and diluted | | 32,674,720 | | 28,656,401 | Condensed Consolidated Balance Sheets The condensed consolidated balance sheets provide a snapshot of PepGen's financial position as of March 31, 2025, compared to December 31, 2024, showing changes in assets, liabilities, and stockholders' equity Condensed Consolidated Balance Sheets (unaudited, in thousands) | | March | 31, 2025 | | December 31, | | --- | --- | --- | --- | --- | | | | (unaudited) | | | | | | | | 2024 | | Assets | | | | | | Cash, cash equivalents and marketable securities | $ | 97,781 | $ | 120,191 | | Other assets | | 29,612 | | 30,692 | | Total assets | $ | 127,393 | $ | 150,883 | | Liabilities and stockholders' equity | | | | | | Liabilities | $ | 35,588 | $ | 32,263 | | Stockholders' equity | | 91,805 | | 118,620 | | Total liabilities and stockholders' equity | $ | 127,393 | $ | 150,883 | About PepGen and Product Candidates This section provides an overview of PepGen, its proprietary EDO platform, and its investigational product candidates, PGN-EDODM1 and PGN-EDO51 About PGN-EDODM1 PGN-EDODM1 is PepGen's investigational candidate for Myotonic Dystrophy Type 1 (DM1), utilizing EDO technology to restore normal splicing function. It has received Orphan Drug and Fast Track Designations from the FDA - PGN-EDODM1 is an investigational candidate for DM1, utilizing PepGen's proprietary Enhanced Delivery Oligonucleotide (EDO) technology to restore the normal splicing function of MBNL1[8] - DM1 is a progressively disabling, life-shortening genetic disorder estimated to affect 40,000 people in the United States and over 74,000 in Europe[8] - PGN-EDODM1 has been granted both Orphan Drug and Fast Track Designations by the U.S. FDA for the treatment of DM1[8] About PGN-EDO51 PGN-EDO51 is PepGen's investigational candidate for Duchenne Muscular Dystrophy (DMD), designed to skip exon 51 using EDO technology to restore dystrophin production. It holds Orphan Drug and Rare Pediatric Disease Designations - PGN-EDO51 is an investigational candidate for DMD, utilizing EDO technology to target the root cause by skipping exon 51 of the dystrophin transcript[9] - It is designed to restore the open reading frame and enable the production of a truncated, yet functional dystrophin protein for approximately 13% of DMD patients[9] - PGN-EDO51 has been granted both Orphan Drug and Rare Pediatric Disease Designations by the U.S. FDA for DMD amenable to an exon-51 skipping approach[9] About PepGen PepGen is a clinical-stage biotechnology company focused on developing next-generation oligonucleotide therapies for severe neuromuscular and neurological diseases, leveraging its proprietary EDO platform - PepGen is a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies to transform the treatment of severe neuromuscular and neurological diseases[10] - The company's EDO platform leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics, forming the basis of its pipeline[10] Legal Disclosures and Contact Information This section provides important legal disclaimers regarding forward-looking statements and investigational therapies, along with contact information Forward-Looking Statements This section outlines the forward-looking nature of certain statements in the press release, emphasizing that they are based on current expectations and subject to various risks and uncertainties that could cause actual results to differ materially - The press release contains forward-looking statements regarding therapeutic potential, safety profile, expected timing for data reports, regulatory interactions, and financial resources[12] - These statements are based on current expectations and are subject to risks and uncertainties, including delays in development, patient enrollment, interpretation of results, safety/efficacy concerns, adverse regulatory outcomes, increased expenses, and dependence on third parties[13][14] - PepGen explicitly disclaims any obligation to update forward-looking statements except as required by law[14] Investigational Therapies Disclaimer A disclaimer stating that PGN-EDODM1 and PGN-EDO51 are investigational therapies not yet approved, and there is no guarantee of their successful clinical development or regulatory approval - PGN-EDODM1 and PGN-EDO51 are investigational therapies not approved for use in any country, and this release is not intended to convey conclusions about their efficacy or safety[15] - There is no guarantee that any investigational therapy will successfully complete clinical development or gain regulatory authority approval[15] Investor and Media Contacts Contact information for investor and media inquiries - Investor Contact: Laurence Watts, New Street Investor Relations, laurence@newstreetir.com[15] - Media Contact: Julia Deutsch, Lyra Strategic Advisory, LLC, Jdeutsch@lyraadvisory.com[15]