\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, 2024, and recent corporate highlights.<\/p>\n
\n\u201cToday, we reported initial results from FREEDOM-DM1, which showed robust splicing correction in patients with DM1 following a single dose of PGN-EDODM1. We believe these data contribute to the growing evidence of our novel EDO platform\u2019s potential to deliver the drug to the nucleus, the site of action,\u201d said James McArthur, PhD, President and CEO of PepGen. \u201cWe remain dedicated to our mission of developing life-changing therapeutics for patients with neuromuscular diseases and are committed to demonstrating the ability of our investigational candidates to address the root cause of disease for improved patient outcomes. We have numerous key data milestones expected this year and look forward to announcing clinical results from the 15 mg\/kg cohort of FREEDOM and 10 mg\/kg cohort of CONNECT1-EDO51.\u201d<\/p>\n
Recent Program Updates<\/b><\/p>\n
PGN-EDODM1: Myotonic Dystrophy Type 1 (DM1)<\/span><\/b><\/p>\n PGN-EDO51: Duchenne Muscular Dystrophy (DMD)<\/span><\/b><\/p>\n Financial Results for the Three and Twelve Months Ended December 31, 2024<\/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 is designed to directly address the deleterious effects of cytosine-uracil-guanine (CUG) repeat expansion in the DMPK<\/i> transcripts which sequester MBNL1, by binding to the pathogenic CUG trinucleotide repeat expansion present in the DMPK <\/i>transcripts, disrupting the binding between the CUG repeat expansion and MBNL1. We believe this mechanism will allow the DMPK <\/i>transcripts to continue performing its normal function within the cell, while also liberating MBNL1 to correct downstream mis-splicing events. We believe that this innovative therapeutic approach has considerable advantages over oligonucleotide modalities that rely on knockdown or degradation of the DMPK <\/i>transcripts. We believe that this therapeutic strategy positions us to potentially provide clinically meaningful benefits for DM1 patients while mitigating the risk of potential deleterious outcomes. The FDA has granted PGN-EDODM1 both Orphan Drug and Fast Track Designations for the treatment of patients with DM1.<\/p>\n About PGN-EDO51<\/b><\/p>\n \nPGN-EDO51, PepGen’s investigational candidate in development for the treatment of DMD, utilizes the Company’s proprietary EDO technology to deliver a therapeutic oligonucleotide that is designed to target the root cause of this devastating disease. PGN-EDO51 is designed to skip exon 51 of the dystrophin transcript, an established therapeutic target for approximately 13% of DMD patients, thereby aiming to restore the open reading frame and enabling the production of a truncated, yet functional dystrophin protein. The FDA has granted PGN-EDO51 both Orphan Drug and Rare Pediatric Disease Designations for the treatment of patients with DMD amenable to an exon-51 skipping approach.<\/p>\n About PepGen<\/b><\/p>\n \nPepGen is 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. PepGen\u2019s 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, we are 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 our product candidates, including, based on early data, PGN-EDO51 and PGN-EDODM1, the potential of our EDO platform to deliver higher levels of oligonucleotide to the nuclei, the design, initiation and conduct of clinical trials, including expected timelines for our CONNECT1 and CONNECT2 Phase 2 trials, our FREEDOM Phase 1 trial and our FREEDOM2 Phase 2 trial, the expected timing for additional data reports from our CONNECT1 Phase 2 trial, and our FREEDOM Phase 1 trial, ongoing and planned regulatory interactions, and our financial resources and expected cash runway.<\/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-EDO51 and PGN-EDODM1; our ability to enroll patients in our clinical trials, including CONNECT1, CONNECT2, FREEDOM and 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-EDO51 and PGN-EDODM1; our product candidates, including PGN-EDO51 and 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 or approval by regulatory authorities with respect to our programs, including 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 CONNECT1, CONNECT2, FREEDOM and FREEDOM2 clinical trials; 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 annual report on Form 10-K and quarterly report on Form 10-Q that are 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-EDO51 and PGN-EDODM1, investigational therapies that have not been approved for use in any country and is not intended to convey conclusions about their efficacy or safety. There is no guarantee that PGN-EDO51, 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<\/td>\n<\/tr>\n \n(unaudited, in thousands)<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n Twelve Months Ended \n\u00a0<\/p>\n<\/td>\n Three Months Ended \n\u00a0<\/p>\n<\/td>\n 2024<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n 2023<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n 2024<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n 2023<\/b><\/p>\n<\/td>\n<\/tr>\n Operating expenses:<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nResearch and development<\/p>\n<\/td>\n \n$ 76,478<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$68,126<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$18,961<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$ 16,300<\/p>\n<\/td>\n<\/tr>\n \nGeneral and administrative<\/p>\n<\/td>\n \n21,261<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n16,640<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n5,384<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n4,511<\/p>\n<\/td>\n<\/tr>\n \nTotal operating expenses<\/p>\n<\/td>\n \n$ 97,739<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$84,766<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$24,345<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$ 20,811<\/p>\n<\/td>\n<\/tr>\n \nOperating loss<\/p>\n<\/td>\n \n$(97,379)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$(84,766)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$(24,345)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$(20,811)<\/p>\n<\/td>\n<\/tr>\n \nOther income (expense)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nInterest income<\/p>\n<\/td>\n \n7,142<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n6,400<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n1,460<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n1,346<\/p>\n<\/td>\n<\/tr>\n \nOther income (expense), net<\/p>\n<\/td>\n \n(1)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(187)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n26<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n43<\/p>\n<\/td>\n<\/tr>\n \nTotal other income, net<\/p>\n<\/td>\n \n7,141<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n6,213<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n1,486<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n1,389<\/p>\n<\/td>\n<\/tr>\n \nNet loss before income tax<\/p>\n<\/td>\n \n$(90,598)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$(78,553)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$(22,859)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$(19,422)<\/p>\n<\/td>\n<\/tr>\n \nIncome tax (expense) benefit<\/p>\n<\/td>\n \n617<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(73)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n617<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(73)<\/p>\n<\/td>\n<\/tr>\n Net loss<\/b><\/p>\n<\/td>\n \n$(89,981)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$(78,626)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$(22,242)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$(19,495)<\/p>\n<\/td>\n<\/tr>\n \nNet loss per share, basic and diluted<\/p>\n<\/td>\n \n$(2.85)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$(3.30)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$(0.68)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$(0.82)<\/p>\n<\/td>\n<\/tr>\n \nWeighted-average common shares outstanding, basic and diluted<\/p>\n<\/td>\n \n31,583,073<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n23,796,000<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n32,602,981<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n23,816,919<\/p>\n<\/td>\n<\/tr>\n<\/table>\n Condensed Consolidated Balance Sheets<\/b><\/p>\n<\/td>\n<\/tr>\n \n(unaudited, in thousands)<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n December 31,<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n December 31,<\/b><\/p>\n<\/td>\n<\/tr>\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 2023<\/b><\/p>\n<\/td>\n<\/tr>\n Assets<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nCash, cash equivalents and marketable securities<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n120,191<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n110,407<\/p>\n<\/td>\n<\/tr>\n \nOther assets<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n30,692<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n32,645<\/p>\n<\/td>\n<\/tr>\n \nTotal assets<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n150,883<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n143,052<\/p>\n<\/td>\n<\/tr>\n Liabilities and stockholders\u2019 equity<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nLiabilities<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n32,263<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n34,631<\/p>\n<\/td>\n<\/tr>\n \nStockholders\u2019 equity<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n118,620<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n108,421<\/p>\n<\/td>\n<\/tr>\n \nTotal liabilities and stockholders\u2019 equity<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n150,883<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n143,052<\/p>\n<\/td>\n<\/tr>\n<\/table>\n\n
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December 31,<\/b><\/p>\n<\/td>\n\n \n
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