\nPepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today reported financial results for the quarter and year-ended December 31, 2025, and recent corporate highlights.<\/p>\n
\n\u201c2025 was a transformative year for PepGen as the Phase 1 FREEDOM trial delivered compelling data that set a new benchmark for splicing correction in DM1, reinforced our conviction in PGN-EDODM1 as a potential best-in-class treatment, and further demonstrated the differentiated potential of our EDO platform,” said James McArthur, PhD, President and Chief Executive Officer of PepGen. \u201cAfter a single dose, we observed robust target engagement beginning at the lowest dose of 5 mg\/kg \u2014 a finding that exceeded our expectations and underscored the potency of our drug candidate. PGN-EDODM1 was generally well-tolerated across doses, and based on the totality of FREEDOM data, we believe that doses of 10 mg\/kg could potentially offer a best-in-class treatment option for people living with this serious disease.\u201d<\/p>\n
\nDr. McArthur continued: \u201cAs we prepare for the 5 mg\/kg dose cohort readout from our ongoing randomized, placebo-controlled multiple ascending dose Phase 2 FREEDOM2 study later this quarter, we are focused on building upon the robust muscle oligonucleotide levels and splicing correction observed in FREEDOM, with the goal of translating sustained exposure into meaningful functional benefit. Following the Data Safety Monitoring Board\u2019s recommendation to dose escalate, we are actively dosing the 10 mg\/kg cohort in FREEDOM2 \u2013 four patients have been dosed, receiving up to two doses. PepGen is entering this new year with strong momentum and a clear path toward potentially delivering a meaningful new treatment for the DM1 community.\u201d<\/p>\n
Recent Program Updates<\/b><\/p>\n
PGN-EDODM1: Myotonic Dystrophy Type 1 (DM1)<\/span><\/b><\/p>\n 2026 MDA Conference Update<\/span><\/b><\/p>\n \nThe Company will present unblinded final results from FREEDOM-DM1 \u2014 its Phase 1, placebo-controlled single ascending dose study of PGN-EDODM1 in DM1 adult patients \u2014 in oral and poster presentations at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, being held March 8\u201311, 2026 in Orlando, Florida.<\/p>\n \nKey study findings include:<\/p>\n Efficacy:<\/b> PGN-EDODM1 demonstrated greater than dose-proportional increases in muscle tissue concentration and splicing correction across all doses at Day 28. Mean splicing correction reached unprecedented levels after a single dose, achieving 12.3%, 29.1%, and 53.7% at 5 mg\/kg (n=6), 10 mg\/kg (n=5), and 15 mg\/kg (n=6), respectively.<\/p>\n Safety:<\/b> PGN-EDODM1 was generally well-tolerated across all doses; treatment-emergent adverse events (TEAEs) were mild to moderate, transient, and required no intervention (except one patient in the 15 mg\/kg cohort, managed with oral antihistamines). No electrolyte-related TEAEs were observed, and magnesium levels remained within normal range across all doses. No renal biomarker-related TEAEs were observed at 5 or 10 mg\/kg. Post-unblinding, transient moderate albuminuria increases at the 15 mg\/kg dose and transient mild albuminuria increases at 10 mg\/kg were apparent \u2014 both resolving to normal within two to seven days with no clinical symptoms or need for intervention.<\/p>\n \nFollowing the conference, the presentations presented at the 2026 MDA Clinical & Scientific Conference will be available on PepGen\u2019s website under Scientific Publications.<\/p>\n Corporate Updates<\/span><\/b><\/p>\n Financial Results for the Three Months and Year Ended December 31, 2025<\/span><\/b><\/p>\n About PGN-EDODM1<\/b><\/p>\n \nPGN-EDODM1, PepGen’s investigational candidate in development for the treatment of DM1, utilizes the Company’s proprietary EDO technology to deliver a therapeutic oligonucleotide that is designed to restore the normal splicing function of MBNL1, a key RNA splicing protein. PGN-EDODM1 addresses the deleterious effects of cytosine-uracil-guanine (CUG) repeat expansion in the dystrophia myotonica protein kinase (DMPK) transcripts which sequester MBNL1, by binding to the pathogenic CUG trinucleotide repeat expansion present in the DMPK transcripts, and disrupting the binding between the CUG repeat expansion and MBNL1. PepGen believes this innovative therapeutic approach may have considerable advantages over oligonucleotide modalities that rely on knockdown or degradation of the DMPK transcripts as it will allow the DMPK transcripts to continue to perform their normal function within the cell, while also liberating MBNL1 to correct downstream mis-splicing events. The U.S. Food and Drug Administration has granted PGN-EDODM1 both Orphan Drug and Fast Track Designations for the treatment of patients with DM1. The European Medicines Agency (EMA) has recently granted Orphan Designation for PGN-EDODM1.<\/p>\n About PepGen<\/b><\/p>\n \nPepGen Inc. is a clinical-stage biotechnology company developing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases. PepGen\u2019s Enhanced Delivery Oligonucleotide (EDO) platform is founded on over a decade of research and development and leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics. Using these EDO peptides, the Company is generating a pipeline of oligonucleotide therapeutic candidates designed to target the root cause of serious diseases.<\/p>\n \nFor more information, please visit PepGen.com<\/i><\/a>. Follow PepGen on LinkedIn <\/i><\/a>and X<\/i><\/a>.<\/p>\n Forward-Looking Statements<\/b><\/p>\n \nThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by words such as \u201caims,\u201d \u201canticipates,\u201d \u201cbelieves,\u201d \u201ccould,\u201d \u201cestimates,\u201d \u201cexpects,\u201d \u201cforecasts,\u201d \u201cgoal,\u201d \u201cintends,\u201d \u201cmay,\u201d \u201cplans,\u201d \u201cpossible,\u201d \u201cpotential,\u201d \u201cseeks,\u201d \u201cwill,\u201d and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, statements regarding the therapeutic potential and safety profile of PGN-EDODM1 based on data from the 5, 10 and 15 mg\/kg cohorts of the FREEDOM-DM1 study, our expectations regarding the potential for significant correction of mis-splicing with more doses of PGN-EDODM1 over a longer treatment period to potentially provide improved functional benefit for patients with DM1, the design and conduct of clinical trials with our candidates, including expected timelines for the initial data report from our FREEDOM2-DM1 trial and the potential to extend dosing to the 12.5 mg\/kg dose level, the potential for any functional improvements that may result from robust splicing correction with PGN-EDODM1, dose-dependent increases in splicing suggesting that PGN-EDODM1 is getting into the muscle and effectively binding to the target, the potential for PGN-EDODM1 to offer a best-in-class treatment option, the potential for future patent term extension, forecasts relating to our cash runway, and ongoing and planned regulatory interactions and ongoing and planned regulatory interactions, including the potential timing and successful resolution of questions from the FDA relating to the partial clinical hold.<\/p>\n \nAny forward-looking statements in this press release are based on current expectations, estimates and projections only as of the date of this release and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to risks related to: delays or failure to successfully initiate or complete our ongoing and planned development activities for our product candidates, including PGN-EDODM1; our ability to enroll patients in our clinical trials, including FREEDOM2; that our interpretation of clinical and preclinical study results may be incorrect, or that we may not observe the levels of therapeutic activity in clinical testing that we anticipate based on prior clinical or preclinical results, including for PGN-EDODM1; our product candidates, including PGN-EDODM1, may not be safe and effective or otherwise demonstrate safety and efficacy in our clinical trials; adverse outcomes from our regulatory interactions, including delays in regulatory review, clearance to proceed with planned clinical studies or approval by regulatory authorities with respect to our investigational drug candidates, including release of the partial clinical hold placed by FDA on the FREEDOM2 study, or other regulatory feedback requiring modifications to our development programs, including release of the partial clinical hold or clearance to commence planned clinical studies of our product candidates, or other regulatory feedback requiring modifications to our development programs, including in each case with respect to our FREEDOM2 program; changes in regulatory framework that are out of our control; unexpected increases in the expenses associated with our development activities or other events that adversely impact our financial resources and cash runway; and our dependence on third parties for some or all aspects of our product manufacturing, research and preclinical and clinical testing. Additional risks concerning PepGen\u2019s programs and operations are described in our most recent reports filed with the SEC. PepGen explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.<\/p>\n \nThis release discusses PGN-EDODM1, an investigational therapy that has not been approved for use in any country, and is not intended to convey conclusions about its efficacy or safety. There is no guarantee that PGN-EDODM1 or any other investigational therapy will successfully complete clinical development or gain regulatory authority approval.<\/p>\n Condensed Consolidated Statements of Operations<\/b><\/p>\n \n(unaudited, in thousands)<\/p>\n<\/td>\n<\/tr>\n Twelve Months Ended Three Months Ended \n\u00a0<\/p>\n<\/td>\n 2025<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n 2024<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n 2025<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n 2024<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n Operating expenses:<\/b><\/p>\n<\/td>\n \nResearch and development<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n71,043<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n76,478<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n13,851<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n18,961<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nGeneral and administrative<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n22,569<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n21,261<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n5,854<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n5,384<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nTotal operating expenses<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n93,612<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n97,739<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n19,705<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n24,345<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nOperating loss<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(93,612<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(97,739<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(19,705<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(24,345<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n<\/tr>\n \nOther income (expense)<\/p>\n<\/td>\n \nInterest income<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n4,017<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n7,142<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n1,419<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n1,460<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nOther income (expense), net<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(11<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(1<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(5<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n26<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nTotal other income, net<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n4,006<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n7,141<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n1,414<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n1,486<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nNet loss before income tax<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(89,606<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(90,598<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(18,291<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(22,859<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n<\/tr>\n \nIncome tax (expense) benefit<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(49<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n617<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(49<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n617<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n Net loss<\/b><\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(89,655<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(89,981<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(18,340<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(22,242<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n<\/tr>\n \nNet loss per share, basic and diluted<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(2.12<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(2.85<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(0.27<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(0.68<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n<\/tr>\n \nWeighted-average common shares outstanding, basic and diluted<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n42,221,808<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n31,583,073<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n68,790,477<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n32,602,981<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n<\/table>\n\n
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