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PepGenF
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2026-08-06
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Earnings documents stored for PEPG.

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Investor releaseQuarter not tagged2026-08-06

PepGen Reports Second Quarter 2026 Financial Results and Recent Corporate Highlights

Business Wire
– DSMB recommends advancement of PGN-EDODM1 into the highest dose cohort in the FREEDOM2-DM1 study, as well as FREEDOM-OLE dose escalation from 5 mg/kg to 10 mg/kg – – 10 mg/kg FREEDOM2-DM1 cohort fully enrolled, with 7 of 8 participants having completed dosing; data expected in November – – Results from 12.5 mg/kg FREEDOM2-DM1 cohort expected in 1H 2027 – – Well-funded with $117.2M of cash as of June 30, 2026, sufficient to fund operations into the fourth quarter of 2027 – BOSTON, August 06, 2026--(BUSINESS WIRE)--PepGen 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 ended June 30, 2026 and recent corporate highlights. "During the second quarter, our team maintained a strong focus on execution as we advanced FREEDOM2 and continued preparing for our next clinical data readout," said James McArthur, PhD, President and Chief Executive Officer of PepGen. "The DSMB's recommendation to dose escalate in both FREEDOM2 and the open-label extension reflects the encouraging safety profile of PGN-EDODM1 following several months of repeat dosing. We look forward to initiating enrollment in the 12.5 mg/kg cohort shortly and sharing data from the 10 mg/kg cohort in November. We remain confident that repeat dosing has the potential to build on the clinical foundation established in the FREEDOM single-ascending dose trial as we continue evaluating the safety and benefits of PGN-EDODM1 for individuals living with DM1." Recent Program Updates PGN-EDODM1: Myotonic Dystrophy Type 1 (DM1) FREEDOM2 Phase 2 Multiple Ascending Dose (MAD) Randomized, Placebo-Controlled Clinical Trial of PGN-EDODM1: Corporate Updates In May 2026, PepGen presented a late-breaking oral and poster presentation featuring data from its PGN-EDODM1 program at the 15th International Myotonic Dystrophy Consortium (IDMC-15). The late-breaking oral session included analyses of individual patient mis-splicing data from the PGN-EDODM1 program, as well as natural history data characterizing mis-splicing in patients with DM1. Ahead of IDMC-15, PepGen's President and Chief Executive Officer, James McArthur, PhD, presented single and initial multiple ascending dose data for PGN-EDODM1 at the 6th Annual Ph…Read full document

– DSMB recommends advancement of PGN-EDODM1 into the highest dose cohort in the FREEDOM2-DM1 study, as well as FREEDOM-OLE dose escalation from 5 mg/kg to 10 mg/kg – – 10 mg/kg FREEDOM2-DM1 cohort fully enrolled, with 7 of 8 participants having completed dosing; data expected in November – – Results from 12.5 mg/kg FREEDOM2-DM1 cohort expected in 1H 2027 – – Well-funded with $117.2M of cash as of June 30, 2026, sufficient to fund operations into the fourth quarter of 2027 – BOSTON, August 06, 2026--(BUSINESS WIRE)--PepGen 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 ended June 30, 2026 and recent corporate highlights. "During the second quarter, our team maintained a strong focus on execution as we advanced FREEDOM2 and continued preparing for our next clinical data readout," said James McArthur, PhD, President and Chief Executive Officer of PepGen. "The DSMB's recommendation to dose escalate in both FREEDOM2 and the open-label extension reflects the encouraging safety profile of PGN-EDODM1 following several months of repeat dosing. We look forward to initiating enrollment in the 12.5 mg/kg cohort shortly and sharing data from the 10 mg/kg cohort in November. We remain confident that repeat dosing has the potential to build on the clinical foundation established in the FREEDOM single-ascending dose trial as we continue evaluating the safety and benefits of PGN-EDODM1 for individuals living with DM1." Recent Program Updates PGN-EDODM1: Myotonic Dystrophy Type 1 (DM1) FREEDOM2 Phase 2 Multiple Ascending Dose (MAD) Randomized, Placebo-Controlled Clinical Trial of PGN-EDODM1: Corporate Updates In May 2026, PepGen presented a late-breaking oral and poster presentation featuring data from its PGN-EDODM1 program at the 15th International Myotonic Dystrophy Consortium (IDMC-15). The late-breaking oral session included analyses of individual patient mis-splicing data from the PGN-EDODM1 program, as well as natural history data characterizing mis-splicing in patients with DM1. Ahead of IDMC-15, PepGen's President and Chief Executive Officer, James McArthur, PhD, presented single and initial multiple ascending dose data for PGN-EDODM1 at the 6th Annual Pharma Day, co-hosted by Euro-DyMA and the Myotonic Dystrophy Foundation. Presentation materials are available on PepGen’s website under Scientific Publications. Read the full release here. Financial Results for the Three Months Ended June 30, 2026 Cash, Cash Equivalents and Marketable Securities were $117.2 million as of June 30, 2026. Based on currently planned operations, PepGen believes that its existing cash, cash equivalents, and marketable securities will be sufficient to fund its operations into the fourth quarter of 2027. Research and Development Expenses were $12.5 million for the three months ended June 30, 2026, compared to $18.4 million for the same period in 2025. General and Administrative Expenses were $6.4 million for the three months ended June 30, 2026, compared to $5.5 million for the same period in 2025. Net Loss was $17.8 million, or $(0.26) basic and diluted net loss per share, for the three months ended June 30, 2026, compared to $23.1 million, or $(0.70) basic and diluted net loss per share, for the same period in 2025. PepGen had approximately 69.3 million shares outstanding on June 30, 2026. About PGN-EDODM1 PGN-EDODM1, PepGen's investigational candidate in development for the treatment of DM1, utilizes PepGen'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. About PepGenPepGen 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’s 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, PepGen is generating a pipeline of oligonucleotide therapeutic candidates designed to target the root cause of serious diseases. For more information, please visit PepGen.com. Follow PepGen on LinkedIn and X. Forward-Looking StatementsThis 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 "aims," "anticipates," "believes," "could," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," 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 whether repeat dosing has the potential to build on the clinical data from the FREEDOM trial, expected timelines for data reports from our FREEDOM2-DM1 trial, including the 10 mg/kg MAD cohort of FREEDOM2 trial and dose escalated 12.5 mg/kg FREEDOM2 trial as well as the OLE trial for PGN-EDODM1, initiation of the 12.5 mg/kg FREEDOM2 cohort of the FREEDOM2 study, forecasts relating to PepGen’s cash runway, and ongoing and planned regulatory timelines and interactions. Any 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 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 PGN-EDODM1 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’s 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. This 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. View source version on businesswire.com: https://www.businesswire.com/news/home/20260806833210/en/ Contacts Investor Contact Laurence WattsNew Street Investor [email protected] Media Contact Julia DeutschLyra Strategic Advisory, [email protected]

Investor releaseQuarter not tagged2026-05-13

PepGen Reports First Quarter 2026 Financial Results and Recent Corporate Highlights

Business Wire
– FREEDOM2-DM1 5 mg/kg cohort demonstrated favorable safety, splicing and vHOT data, with the totality of results supporting the potential of the ongoing 10 mg/kg dose cohort – – The FREEDOM2 10 mg/kg cohort is fully enrolled, with data on track for 2H 2026 – – Well-funded with $132.3M of cash as of March 31, 2026, sufficient to fund operations through FREEDOM2 12.5 mg/kg MAD readout and into 2H 2027 – BOSTON, May 12, 2026--(BUSINESS WIRE)--PepGen 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 and recent corporate highlights for the quarter ended March 31, 2026, and recent corporate highlights. "We made encouraging progress during the first quarter of 2026 in our FREEDOM2-DM1 trial. The 5 mg/kg cohort — the initial dose cohort — demonstrated a favorable tolerability profile and promising efficacy trends, reinforcing our confidence in PGN-EDODM1's potential at higher doses," said James McArthur, PhD, President and Chief Executive Officer of PepGen. "Enrollment in the 10 mg/kg cohort is now complete and we remain on track to report data in the second half of this year. With funding in place through the 10 and 12.5 mg/kg cohorts, we are well positioned to continue dose escalating and deliver on the promise of this program for the DM1 community." Recent Program Updates PGN-EDODM1: Myotonic Dystrophy Type 1 (DM1) FREEDOM2 Phase 2 Multiple Ascending Dose (MAD) Randomized, Placebo-Controlled Clinical Trial of PGN-EDODM1: PepGen reported promising topline results from the 5 mg/kg MAD cohort, demonstrating favorable safety, splicing and vHOT data. The Company believes the totality of safety and efficacy results support the potential of PGN-EDODM1 in the ongoing 10 mg/kg dose cohort. Read the full release here. The Company has fully enrolled the 10 mg/kg MAD cohort of FREEDOM2. PepGen expects to report data from this cohort in the second half of 2026, with data from the 12.5 mg/kg cohort anticipated in 2027. PepGen has currently enrolled 13 patients in the open label extension (OLE) at 5 mg/kg, including 6 patients from FREEDOM2, with no patient discontinuations. The Company has received regulatory clearance to initiate the FREEDOM2 trial in South Korea, Australia, and Ne…Read full document

– FREEDOM2-DM1 5 mg/kg cohort demonstrated favorable safety, splicing and vHOT data, with the totality of results supporting the potential of the ongoing 10 mg/kg dose cohort – – The FREEDOM2 10 mg/kg cohort is fully enrolled, with data on track for 2H 2026 – – Well-funded with $132.3M of cash as of March 31, 2026, sufficient to fund operations through FREEDOM2 12.5 mg/kg MAD readout and into 2H 2027 – BOSTON, May 12, 2026--(BUSINESS WIRE)--PepGen 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 and recent corporate highlights for the quarter ended March 31, 2026, and recent corporate highlights. "We made encouraging progress during the first quarter of 2026 in our FREEDOM2-DM1 trial. The 5 mg/kg cohort — the initial dose cohort — demonstrated a favorable tolerability profile and promising efficacy trends, reinforcing our confidence in PGN-EDODM1's potential at higher doses," said James McArthur, PhD, President and Chief Executive Officer of PepGen. "Enrollment in the 10 mg/kg cohort is now complete and we remain on track to report data in the second half of this year. With funding in place through the 10 and 12.5 mg/kg cohorts, we are well positioned to continue dose escalating and deliver on the promise of this program for the DM1 community." Recent Program Updates PGN-EDODM1: Myotonic Dystrophy Type 1 (DM1) FREEDOM2 Phase 2 Multiple Ascending Dose (MAD) Randomized, Placebo-Controlled Clinical Trial of PGN-EDODM1: PepGen reported promising topline results from the 5 mg/kg MAD cohort, demonstrating favorable safety, splicing and vHOT data. The Company believes the totality of safety and efficacy results support the potential of PGN-EDODM1 in the ongoing 10 mg/kg dose cohort. Read the full release here. The Company has fully enrolled the 10 mg/kg MAD cohort of FREEDOM2. PepGen expects to report data from this cohort in the second half of 2026, with data from the 12.5 mg/kg cohort anticipated in 2027. PepGen has currently enrolled 13 patients in the open label extension (OLE) at 5 mg/kg, including 6 patients from FREEDOM2, with no patient discontinuations. The Company has received regulatory clearance to initiate the FREEDOM2 trial in South Korea, Australia, and New Zealand. Sites are currently open and active in Canada, the UK and South Korea, with plans to open sites in New Zealand and Australia. The U.S. Food and Drug Administration (FDA) placed a partial clinical hold on the FREEDOM2 study. The partial clinical hold questions raised by the FDA relate to previously submitted preclinical pharmacology and toxicology studies. The timing of the ongoing FREEDOM2 clinical study has not been impacted. The Company continues to work with the FDA to address the Agency’s questions as quickly as possible. Financial Results for the Three Months Ended March 31, 2026 Cash, Cash Equivalents and Marketable Securities were $132.3 million as of March 31, 2026. Based on currently planned operations, the Company believes that its existing cash, cash equivalents, and marketable securities will be sufficient to fund its operations into the second half of 2027. Research and Development Expenses were $13.0 million for the three months ended March 31, 2026, compared to $25.4 million for the same period in 2025. General and Administrative Expenses were $5.9 million for the three months ended March 31, 2026, compared to $5.9 million for the same period in 2025. Net Loss was $17.8 million, or $(0.26) basic and diluted net loss per share, for the three months ended March 31, 2026, compared to $30.2 million, or $(0.92) basic and diluted net loss per share, for the same period in 2025. PepGen had approximately 69.2 million shares outstanding on March 31, 2026. About PGN-EDODM1 PGN-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. About Myotonic Dystrophy Type 1 (DM1) Myotonic dystrophy type 1 (DM1) is a rare, progressive, and highly variable genetic neuromuscular disease caused by an abnormal expansion of cytosine-thymine-guanine (CTG) repeats in the dystrophia myotonica protein kinase (DMPK) gene. DM1 affects over 115,000 individuals in the U.S. and EU and is characterized by widespread, multisystem symptoms that may include myotonia, progressive muscle weakness, fatigue, cardiac abnormalities, respiratory impairment, and cognitive dysfunction. The disease is driven by toxic RNA transcripts containing expanded cytosine-uracil-guanine (CUG) repeats that sequester muscleblind-like 1 (MBNL1), a key RNA splicing protein, leading to widespread mis-splicing across multiple tissues. There are currently no approved disease-modifying therapies for DM1, underscoring the significant unmet medical need for patients living with the disease. About PepGen PepGen 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’s 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. For more information, please visit PepGen.com. Follow PepGen on LinkedIn and X. Forward-Looking Statements This 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 "aims," "anticipates," "believes," "could," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," 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 single dose FREEDOM-DM1 study and data from 5 mg/kg cohort of the multi-dose FREEDOM2-DM1 study, expected timelines for data reports from our FREEDOM2-DM1 trial, forecasts relating to PepGen’s cash runway, and ongoing and planned regulatory interactions, including the potential timing and successful resolution of questions from the FDA relating to the partial clinical hold. Any 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 or approval by regulatory authorities with respect to our programs, including release of the partial clinical hold placed by the FDA and/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’s 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. This 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. View source version on businesswire.com: https://www.businesswire.com/news/home/20260512245445/en/ Contacts Investor Contact Laurence Watts New Street Investor Relations [email protected] Media Contact Julia Deutsch Lyra Strategic Advisory, LLC [email protected]

Investor releaseQuarter not tagged2026-03-31

PepGen Announces Topline Results from Lowest Dose (5 mg/kg) MAD Cohort in the Ongoing Phase 2 FREEDOM2 Study Demonstrating Favorable Safety, Splicing and vHOT Data

Business Wire
– PGN-EDODM1 was generally well-tolerated with all adverse events mild or moderate and no serious adverse events reported – – Mean splicing correction of 7.3% observed with PGN-EDODM1 (n=6) versus 6.8% with placebo (n=2); Excluding one outlier patient, treatment group demonstrated mean splicing correction of 22.9% (n=5) – – Promising trends observed in vHOT in the PGN-EDODM1 treated group – – Company on track to report clinical data from 10 mg/kg multiple dose cohort in 2H 2026 with sufficient cash expected to fund operations into 2H 2027– – Conference call scheduled today at 4:30 p.m. ET – BOSTON, March 30, 2026--(BUSINESS WIRE)--PepGen 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 announced promising clinical data from the 5 mg/kg multiple ascending dose cohort (MAD) of the ongoing Phase 2 FREEDOM2-DM1 trial in patients with myotonic dystrophy type 1 (DM1). The Company believes the totality of safety and efficacy results support the potential of the ongoing 10 mg/kg dose cohort, with clinical results expected in the second half of 2026. "We are excited to share data from the lowest dose cohort in our multiple ascending dose study of PGN-EDODM1 in patients with DM1," said Paul Streck, MD, EVP Research and Development. "Overall, we are encouraged by the favorable safety and tolerability profile, and the positive trends exhibited with splicing improvement and vHOT. We look forward to reporting data from the ongoing 10 mg/kg multiple ascending dose cohort of FREEDOM2, which is more than halfway enrolled and remains on track to read out in the second half of this year." FREEDOM2 Results for the 5 mg/kg (n=8) Dose Cohort FREEDOM2 is a Phase 2 MAD, randomized, placebo-controlled clinical trial evaluating PGN-EDODM1 in patients with DM1, with planned dose escalation up to 12.5 mg/kg. The 5 mg/kg cohort enrolled eight patients, who received PGN-EDODM1 or placebo (randomized 6:2) every four weeks over a 12-week period. Key endpoints include safety, splicing correction and functional outcome measures. The data cutoff was March 4, 2026. Splicing, Muscle Tissue Concentration and Functional Data: Patients (n=6) treated with PGN-EDODM1 at 5 mg/kg demonstrated a mean splicing correction of 7.3%, compa…Read full document

– PGN-EDODM1 was generally well-tolerated with all adverse events mild or moderate and no serious adverse events reported – – Mean splicing correction of 7.3% observed with PGN-EDODM1 (n=6) versus 6.8% with placebo (n=2); Excluding one outlier patient, treatment group demonstrated mean splicing correction of 22.9% (n=5) – – Promising trends observed in vHOT in the PGN-EDODM1 treated group – – Company on track to report clinical data from 10 mg/kg multiple dose cohort in 2H 2026 with sufficient cash expected to fund operations into 2H 2027– – Conference call scheduled today at 4:30 p.m. ET – BOSTON, March 30, 2026--(BUSINESS WIRE)--PepGen 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 announced promising clinical data from the 5 mg/kg multiple ascending dose cohort (MAD) of the ongoing Phase 2 FREEDOM2-DM1 trial in patients with myotonic dystrophy type 1 (DM1). The Company believes the totality of safety and efficacy results support the potential of the ongoing 10 mg/kg dose cohort, with clinical results expected in the second half of 2026. "We are excited to share data from the lowest dose cohort in our multiple ascending dose study of PGN-EDODM1 in patients with DM1," said Paul Streck, MD, EVP Research and Development. "Overall, we are encouraged by the favorable safety and tolerability profile, and the positive trends exhibited with splicing improvement and vHOT. We look forward to reporting data from the ongoing 10 mg/kg multiple ascending dose cohort of FREEDOM2, which is more than halfway enrolled and remains on track to read out in the second half of this year." FREEDOM2 Results for the 5 mg/kg (n=8) Dose Cohort FREEDOM2 is a Phase 2 MAD, randomized, placebo-controlled clinical trial evaluating PGN-EDODM1 in patients with DM1, with planned dose escalation up to 12.5 mg/kg. The 5 mg/kg cohort enrolled eight patients, who received PGN-EDODM1 or placebo (randomized 6:2) every four weeks over a 12-week period. Key endpoints include safety, splicing correction and functional outcome measures. The data cutoff was March 4, 2026. Splicing, Muscle Tissue Concentration and Functional Data: Patients (n=6) treated with PGN-EDODM1 at 5 mg/kg demonstrated a mean splicing correction of 7.3%, compared to 6.8% in placebo-treated patients (n=2). Excluding an outlier, patients showed a mean splicing correction of 22.9%. The outlier patient exhibited a worsening in splicing correction (70.8%), reducing the overall group mean to 7.3%. Middle finger vHOT in the treatment group showed a positive trend of improvement versus a worsening observed in the placebo group. Both returned to baseline at Week 16. Mean muscle tissue concentrations of PGN-EDODM1 available in 5 of 6 treated patients were 158 ng/g, as measured approximately one week after the fourth dose in the MAD study; one concentration readout remains pending. No meaningful improvements were observed in 10-meter walk/run test (10MWRT) or handgrip strength at the starting PGN-EDODM1 dose of 5 mg/kg. Safety and Tolerability Data: PGN-EDODM1 was generally well-tolerated, with no serious adverse events (SAEs), all related treatment emergent adverse events (TEAEs) reported as mild and non-related TEAEs reported as mild or moderate Nausea was the most common TEAE No treatment-related discontinuations No TEAEs related to renal function and no signs of cumulative toxicity The Company is actively dosing the 10 mg/kg MAD cohort of FREEDOM2, with 5 of 8 patients receiving up to three doses of PGN-EDODM1, and expects to report data in the second half of 2026. In addition, 12 patients have currently enrolled in the open label extension (OLE) at 5 mg/kg, including 5 patients from FREEDOM2. Conference Call Details To access the live audio webcast for today’s conference call beginning at 4:30 p.m. ET, please visit PepGen’s investor website at investors.pepgen.com. An archived replay of the webcast will be available on the Company’s website following the event. About PGN-EDODM1 PGN-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. About PepGen PepGen 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’s 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. For more information, please visit PepGen.com. Follow PepGen on LinkedIn and X. Forward-Looking Statements This 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 "aims," "anticipates," "believes," "could," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," 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 various data demonstrating promising results that support the safety and clinical potential of PGN-EDODM1 as a treatment for DM1 and expected timelines for the data report from the 10 mg/kg cohort of our FREEDOM2-DM1 trial, our expected cash runway, the design, initiation and conduct of clinical trials, including expected timelines for our FREEDOM2-DM1 trial, and ongoing and planned regulatory interactions. Any 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 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 for PGN-EDODM1; that 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 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’s 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. This 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. View source version on businesswire.com: https://www.businesswire.com/news/home/20260330100190/en/ Contacts Investor Contact Laurence Watts New Street Investor Relations [email protected] Media Contact Julia Deutsch Lyra Strategic Advisory [email protected]

Investor releaseQuarter not tagged2026-03-05

PepGen Reports Fourth Quarter and Year-End 2025 Financial Results and Recent Corporate Highlights

Business Wire
– FREEDOM2-DM1 5 mg/kg cohort data expected in Q1 2026 – – Patient dosing in the FREEDOM2 10 mg/kg cohort is proceeding in Canada and the UK, with data expected in 2H 2026 – – FREEDOM2 trial open in South Korea, Australia, and New Zealand – – Well-funded with $148.5M of cash as of December 31, 2025, sufficient to fund operations into the second half of 2027 – BOSTON, March 04, 2026--(BUSINESS WIRE)--PepGen 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. "2025 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. "After a single dose, we observed robust target engagement beginning at the lowest dose of 5 mg/kg — 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." Dr. McArthur continued: "As 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’s recommendation to dose escalate, we are actively dosing the 10 mg/kg cohort in FREEDOM2 – 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." Recent Program Updates PGN-EDODM1: Myotonic Dystrophy Type 1 (DM1) FREEDOM2 Phase 2 Multiple Ascending Dose (M…Read full document

– FREEDOM2-DM1 5 mg/kg cohort data expected in Q1 2026 – – Patient dosing in the FREEDOM2 10 mg/kg cohort is proceeding in Canada and the UK, with data expected in 2H 2026 – – FREEDOM2 trial open in South Korea, Australia, and New Zealand – – Well-funded with $148.5M of cash as of December 31, 2025, sufficient to fund operations into the second half of 2027 – BOSTON, March 04, 2026--(BUSINESS WIRE)--PepGen 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. "2025 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. "After a single dose, we observed robust target engagement beginning at the lowest dose of 5 mg/kg — 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." Dr. McArthur continued: "As 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’s recommendation to dose escalate, we are actively dosing the 10 mg/kg cohort in FREEDOM2 – 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." Recent Program Updates PGN-EDODM1: Myotonic Dystrophy Type 1 (DM1) FREEDOM2 Phase 2 Multiple Ascending Dose (MAD) Randomized, Placebo-Controlled Clinical Trial of PGN-EDODM1: The Company expects to report results from the 5 mg/kg cohort of the FREEDOM2 trial in the first quarter of 2026. After reviewing safety data from the 5 mg/kg cohort, the Data Safety Monitoring Board (DSMB) recommended escalating to the 10 mg/kg dose. PepGen has since dosed 50% of patients (4/8) in the 10 mg/kg cohort with up to two doses. The Company remains on track to report results from the 10 mg/kg cohort in the second half of 2026. Informed by the prior FREEDOM results, PepGen has amended the FREEDOM2 protocol to provide the option to dose up to 12.5 mg/kg. Patients from the FREEDOM and FREEDOM2 studies in Canada are continuing into the Open Label Extension (OLE) study. The Company has received regulatory clearance for the OLE study in the UK and intends to open the OLE study in all geographies where FREEDOM2 is open. The Company has recently received regulatory clearance to initiate the FREEDOM2 trial in South Korea, Australia, and New Zealand. Sites are currently enrolling in Canada, the UK and South Korea, with plans to open sites in New Zealand and Australia. The U.S. Food and Drug Administration (FDA) has placed a partial clinical hold on the FREEDOM2 study. The partial clinical hold questions raised by FDA relate to previously submitted preclinical pharmacology and toxicology studies. The partial clinical hold did not cite any questions regarding blinded clinical data from the Phase 1 FREEDOM study previously submitted to the FDA in order to initiate the FREEDOM2 study in the U.S. The Company is committed to working with the FDA to address the Agency’s questions as quickly as possible. Other FREEDOM Updates: In October 2025, PepGen’s data were featured in two oral presentations at the 30th Annual International Congress of the World Muscle Society (WMS). One presentation included previously reported positive FREEDOM-DM1 Phase 1 clinical data. In October 2025, the Company gave an oral presentation at the Oligonucleotide Therapeutics Society (OTS) Annual Meeting. The presentation reviewed the use of its Enhanced Delivery Oligonucleotide (EDO) Platform to develop a treatment for DM1 and included a review of the FREEDOM clinical data. 2026 MDA Conference Update The Company will present unblinded final results from FREEDOM-DM1 — its Phase 1, placebo-controlled single ascending dose study of PGN-EDODM1 in DM1 adult patients — in oral and poster presentations at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, being held March 8–11, 2026 in Orlando, Florida. Key study findings include: Efficacy: 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. Safety: 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 — both resolving to normal within two to seven days with no clinical symptoms or need for intervention. Following the conference, the presentations presented at the 2026 MDA Clinical & Scientific Conference will be available on PepGen’s website under Scientific Publications. Corporate Updates In December 2025, PepGen appointed Joseph Vittiglio, Esq., as Chief Business and Legal Officer. Mr. Vittiglio brings more than two decades of executive leadership experience across legal, compliance, corporate development, and corporate governance within public biotechnology companies. He joins PepGen after providing consulting support to the Company in 2025. In November 2025, the Company announced that the United States Patent and Trademark Office (USPTO) has issued the Company a new composition of matter patent covering PGN-EDODM1, which leverages PepGen’s proprietary EDO platform, including its unique peptide and linker chemistry. This newly issued composition of matter patent, which serves as a cornerstone of the Company’s expanding patient portfolio, is expected to provide exclusivity for PGN-EDODM1 in the United States into second half of 2042, with the possibility of patent term extension following a potential new drug application and approval by FDA of PGN-EDODM1. Financial Results for the Three Months and Year Ended December 31, 2025 Cash, Cash Equivalents and Marketable Securities were $148.5 million as of December 31, 2025. Based on currently planned operations, the Company believes that its existing cash, cash, equivalents, and marketable securities will be sufficient to fund its operations into the second half of 2027. Research and Development Expenses were $13.9 million for the three months ended December 31, 2025, compared to $19.0 million for the same period in 2024. Research and development expenses were $71.0 million for the year ended December 31, 2025, compared to $76.5 million for the same period in 2024. General and Administrative Expenses were $5.9 million for the three months ended December 31, 2025, compared to $5.4 million for the same period in 2024. General and administrative expenses were $22.6 million for the year ended December 31, 2025, compared to $21.3 million for the same period in 2024. Net Loss was $18.3 million, or $(0.27) basic and diluted net loss per share, for the three months ended December 31, 2025, compared to $22.2 million, or $(0.68) basic and diluted net loss per share, for the same period in 2024. Net loss was $89.7 million, or $(2.12) basic and diluted net loss per share, for the year ended December 31, 2025, compared to $90.0 million, or $(2.85) basic and diluted net loss per share, for the same period in 2024. PepGen had approximately 68,874,944 shares outstanding as of December 31, 2025. About PGN-EDODM1 PGN-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. About PepGen PepGen 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’s 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. For more information, please visit PepGen.com. Follow PepGen on LinkedIn and X. Forward-Looking Statements This 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 "aims," "anticipates," "believes," "could," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," 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. Any 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’s 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. This 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. View source version on businesswire.com: https://www.businesswire.com/news/home/20260304670027/en/ Contacts Investor Contact Laurence Watts New Street Investor Relations [email protected] Media Contact Julia Deutsch Lyra Strategic Advisory, LLC [email protected]

Investor releaseQuarter not tagged2025-11-12

PepGen Reports Third Quarter 2025 Financial Results and Recent Corporate Highlights

Business Wire
– FREEDOM-DM1 15 mg/kg cohort demonstrated the highest mean splicing correction reported to date in DM1 patients – – FREEDOM2-DM1 5 mg/kg cohort on track to readout in Q1 2026, with all patients enrolled and having received at least one dose – – Recent $115 million financing extends cash runway into the second half of 2027 – BOSTON, November 12, 2025--(BUSINESS WIRE)--PepGen 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 and recent corporate highlights for the quarter ended September 30, 2025. "This quarter was defined by the strong results from the 15 mg/kg cohort of our FREEDOM study, which showed unprecedented levels of splicing correction following a single dose," said James McArthur, PhD, President and Chief Executive Officer of PepGen. "With the key objectives of the FREEDOM study achieved, we are carrying this momentum forward as we advance our multiple ascending dose trial, FREEDOM2. Because mis-splicing is the underlying cause of DM1, we believe that the high levels of splicing correction we achieved have the potential to translate into meaningful functional improvements across key outcome measures, including myotonia and muscle weakness, over time; and we are eager to demonstrate this in FREEDOM2. Bolstered by our recent financing, we are now well positioned to advance FREEDOM2 and further validate the potential of PGN-EDODM1 to become a best-in-class treatment for individuals living with DM1. We look forward to sharing data from the 5 mg/kg multi-dose cohort in the first quarter of next year." Recent Program Updates PGN-EDODM1: Myotonic Dystrophy Type 1 (DM1) FREEDOM Phase 1 Single Ascending Dose (SAD) Randomized, Placebo-Controlled Clinical Trial of PGN-EDODM1: The Company reported topline results from the 15 mg/kg cohort of PGN-EDODM1, demonstrating a mean splicing correction of 53.7% following a single 15 mg/kg dose—substantially higher than any previously reported splicing correction in DM1 patients and with all patients showing improvement in splicing. PGN-EDODM1 was generally well tolerated at this dose, with drug-related adverse events mild or moderate in severity. For additional details, read the full press release here. FREEDOM2 Phase 2 Multi…Read full document

– FREEDOM-DM1 15 mg/kg cohort demonstrated the highest mean splicing correction reported to date in DM1 patients – – FREEDOM2-DM1 5 mg/kg cohort on track to readout in Q1 2026, with all patients enrolled and having received at least one dose – – Recent $115 million financing extends cash runway into the second half of 2027 – BOSTON, November 12, 2025--(BUSINESS WIRE)--PepGen 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 and recent corporate highlights for the quarter ended September 30, 2025. "This quarter was defined by the strong results from the 15 mg/kg cohort of our FREEDOM study, which showed unprecedented levels of splicing correction following a single dose," said James McArthur, PhD, President and Chief Executive Officer of PepGen. "With the key objectives of the FREEDOM study achieved, we are carrying this momentum forward as we advance our multiple ascending dose trial, FREEDOM2. Because mis-splicing is the underlying cause of DM1, we believe that the high levels of splicing correction we achieved have the potential to translate into meaningful functional improvements across key outcome measures, including myotonia and muscle weakness, over time; and we are eager to demonstrate this in FREEDOM2. Bolstered by our recent financing, we are now well positioned to advance FREEDOM2 and further validate the potential of PGN-EDODM1 to become a best-in-class treatment for individuals living with DM1. We look forward to sharing data from the 5 mg/kg multi-dose cohort in the first quarter of next year." Recent Program Updates PGN-EDODM1: Myotonic Dystrophy Type 1 (DM1) FREEDOM Phase 1 Single Ascending Dose (SAD) Randomized, Placebo-Controlled Clinical Trial of PGN-EDODM1: The Company reported topline results from the 15 mg/kg cohort of PGN-EDODM1, demonstrating a mean splicing correction of 53.7% following a single 15 mg/kg dose—substantially higher than any previously reported splicing correction in DM1 patients and with all patients showing improvement in splicing. PGN-EDODM1 was generally well tolerated at this dose, with drug-related adverse events mild or moderate in severity. For additional details, read the full press release here. FREEDOM2 Phase 2 Multiple Ascending Dose (MAD) Randomized, Placebo-Controlled Clinical Trial of PGN-EDODM1: All patients in the 5 mg/kg cohort of the FREEDOM2 trial have been enrolled and have received at least one dose. The Company remains on track to report results from the 5 mg/kg cohort in the first quarter of 2026 and expects to initiate dosing in the 10 mg/kg cohort during the same quarter. The Company remains on track to report results from the 10 mg/kg cohort in 2H 2026. Corporate Updates In September 2025, PepGen closed an underwritten public offering, raising $115 million in aggregate gross proceeds before deducting underwriting discounts, commissions, and offering expenses. Based on current plans, these proceeds extend the Company’s cash runway into the second half of 2027. In October 2025, the Company’s data were featured in two oral presentations at the 30th Annual International Congress of the World Muscle Society (WMS). One presentation included the previously reported positive FREEDOM-DM1 Phase 1 clinical data. The presentations presented are available on PepGen’s website under Scientific Publications. In October 2025, PepGen gave an oral presentation at the Oligonucleotide Therapeutics Society (OTS) Annual Meeting. The presentation reviewed the use of its Enhanced Delivery Oligonucleotide (EDO) Platform to develop a treatment for DM1 and included a review of the FREEDOM clinical data. The presentation presented is available on PepGen’s website under Scientific Publications. Financial Results for the Three Months Ended September 30, 2025 Cash, Cash Equivalents and Marketable Securities were $163.7 million as of September 30, 2025. Based on currently planned operations, the Company believes that its existing cash, cash equivalents, and marketable securities will be sufficient to fund its operations into the second half of 2027. Research and Development Expenses were $13.4 million for the three months ended September 30, 2025, compared to $17.7 million for the same period in 2024. General and Administrative Expenses were $5.2 million for the three months ended September 30, 2025, compared to $5.4 million for the same period in 2024. Net Loss was $18.0 million, or $(0.52) basic and diluted net loss per share, for the three months ended September 30, 2025, compared to $21.4 million, or $(0.66) basic and diluted net loss per share, for the same period in 2024. PepGen had approximately 68.7 million shares outstanding on September 30, 2025. About PGN-EDODM1 PGN-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 myotonic 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. About PepGen PepGen 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’s 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. For more information, please visit PepGen.com. Follow PepGen on LinkedIn and X. Forward-Looking Statements This 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 "aims," "anticipates," "believes," "could," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," 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, the potential for any functional improvements that may result from robust splicing correction with PGN-EDODM1, forecasts relating to our cash runway, and ongoing and planned regulatory interactions. Any 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: 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 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 FREEDOM and FREEDOM2 clinical trials; changes in regulatory framework that are out of our control; our ability to obtain, maintain and protect our intellectual property; our ability to enforce our patents against infringers and defend our patent portfolio against challenges from third parties; competition from others developing therapies for indications we are pursuing; 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’s 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. This 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. View source version on businesswire.com: https://www.businesswire.com/news/home/20251112398555/en/ Contacts Investor Contact Laurence Watts New Street Investor Relations [email protected] Media Contact Julia Deutsch Lyra Strategic Advisory, LLC [email protected]

Investor releaseQuarter not tagged2025-09-26

PepGen (PEPG) Skyrockets 121% on Stellar Experimental Treatment Trial Results

Insider Monkey

We recently published Massive Gains: 10 Stocks Investors Can’t Stop Buying. PepGen Inc. (NASDAQ:PEPG) is one of the top performers on Thursday. Shares of PepGen soared by 121.05 percent on Thursday to end at $5.88 apiece as investors cheered stellar results from its clinical trial of an experimental drug to treat myotonic dystrophy type 1 (DM1). In a statement, PepGen Inc. (NASDAQ:PEPG) said it saw a 53.7 percent correction in the key genetic error that drives the disease in patients taking 15 mg/kg of the drug candidate called PGN-EDODM1. It was higher than any previously reported splicing correction in DM1 patients. Image Point Fr/Shutterstock.com “We are delighted to report that the … clinical study achieved all of its key objectives, including unprecedented splicing correction following a single dose of PGN-EDODM1 at 15 mg/kg,” said Paul Streck, EVP for research and development. “Since mis-splicing is the underlying cause of DM1, we believe high levels of splicing correction have the potential to reverse the underlying molecular defects, and produce functional improvements in multiple outcome measures, including myotonia and muscle weakness, in repeat dose studies.” Following the results, PepGen Inc. (NASDAQ:PEPG) kicked off a $100 million public offering, covering the sale of 31.25 million shares at a price of $3.20 apiece. PepGen Inc. (NASDAQ:PEPG) said it plans to use the proceeds to fund its ongoing research and clinical development efforts, including the DM1 clinical trials, while the balance will be allocated for other general corporate purposes. While we acknowledge the potential of PEPG as an investment, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an extremely cheap AI stock that is also a major beneficiary of Trump tariffs and onshoring, see our free report on the best short-term AI stock. READ NEXT: 30 Stocks That Should Double in 3 Years and 11 Hidden AI Stocks to Buy Right Now. Disclosure: None. This article is originally published at Insider Monkey.

Investor releaseQuarter not tagged2025-08-07

PepGen Reports Second Quarter 2025 Financial Results and Recent Corporate Highlights

Business Wire
– Last patient dosed in 15 mg/kg cohort of FREEDOM-DM1; on track to report topline data from study in early Q4 2025 – – FREEDOM to conclude with the 15 mg/kg cohort based on splicing and safety data observed to date; clinical sites will begin transitioning to the Phase 2 multiple ascending dose study, FREEDOM2-DM1 – BOSTON, August 07, 2025--(BUSINESS WIRE)--PepGen 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 and recent corporate highlights for the quarter ended June 30, 2025. "This quarter, we made further progress in the development of our promising myotonic dystrophy type 1 program, PGN-EDODM1. With class-leading mean mis-splicing correction following a single 10 mg/kg dose, we believe PGN-EDODM1 has the potential to become a best-in-class treatment for patients with DM1," said James McArthur, PhD, President and CEO of PepGen. "Having recently completed patient dosing in the 15 mg/kg arm of our single ascending dose FREEDOM trial (PGN-EDODM1-101), we look forward to sharing topline data from this cohort in early Q4 2025. Furthermore, the efficacy and emerging safety profile of PGN-EDODM1 supports our decision to conclude the FREEDOM trial with the 15 mg/kg single dose cohort, with the additional benefit of being able to now transition clinical sites to the FREEDOM2 trial (PGN-EDODM1-102), our multiple ascending dose trial. Our FREEDOM2 trial is designed to demonstrate that sustained mis-splicing correction with multiple doses of PGN-EDODM1 can produce meaningful functional improvements in patients with DM1. We’re looking forward to reporting results from the initial dose cohort of FREEDOM2 in the first quarter of 2026." Recent Program Updates PGN-EDODM1: Myotonic Dystrophy Type 1 (DM1) FREEDOM Phase 1 Single Ascending Dose (SAD) Randomized, Placebo-Controlled Clinical Trial of PGN-EDODM1: The Company has completed patient dosing in the 15 mg/kg cohort of the FREEDOM trial and remains on track to report safety, 28-day splicing and functional benefit data from the 15 mg/kg cohort in early fourth quarter of 2025. Based on the robust splicing correction observed at the 5 and 10 mg/kg dose cohorts and the totality of the blinded safety data to date, the Company h…Read full document

– Last patient dosed in 15 mg/kg cohort of FREEDOM-DM1; on track to report topline data from study in early Q4 2025 – – FREEDOM to conclude with the 15 mg/kg cohort based on splicing and safety data observed to date; clinical sites will begin transitioning to the Phase 2 multiple ascending dose study, FREEDOM2-DM1 – BOSTON, August 07, 2025--(BUSINESS WIRE)--PepGen 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 and recent corporate highlights for the quarter ended June 30, 2025. "This quarter, we made further progress in the development of our promising myotonic dystrophy type 1 program, PGN-EDODM1. With class-leading mean mis-splicing correction following a single 10 mg/kg dose, we believe PGN-EDODM1 has the potential to become a best-in-class treatment for patients with DM1," said James McArthur, PhD, President and CEO of PepGen. "Having recently completed patient dosing in the 15 mg/kg arm of our single ascending dose FREEDOM trial (PGN-EDODM1-101), we look forward to sharing topline data from this cohort in early Q4 2025. Furthermore, the efficacy and emerging safety profile of PGN-EDODM1 supports our decision to conclude the FREEDOM trial with the 15 mg/kg single dose cohort, with the additional benefit of being able to now transition clinical sites to the FREEDOM2 trial (PGN-EDODM1-102), our multiple ascending dose trial. Our FREEDOM2 trial is designed to demonstrate that sustained mis-splicing correction with multiple doses of PGN-EDODM1 can produce meaningful functional improvements in patients with DM1. We’re looking forward to reporting results from the initial dose cohort of FREEDOM2 in the first quarter of 2026." Recent Program Updates PGN-EDODM1: Myotonic Dystrophy Type 1 (DM1) FREEDOM Phase 1 Single Ascending Dose (SAD) Randomized, Placebo-Controlled Clinical Trial of PGN-EDODM1: The Company has completed patient dosing in the 15 mg/kg cohort of the FREEDOM trial and remains on track to report safety, 28-day splicing and functional benefit data from the 15 mg/kg cohort in early fourth quarter of 2025. Based on the robust splicing correction observed at the 5 and 10 mg/kg dose cohorts and the totality of the blinded safety data to date, the Company has decided to conclude dose escalation in the FREEDOM trial with the 15 mg/kg cohort. PepGen will now redirect resources to the FREEDOM2 trial and begin transitioning open clinical sites to the multiple ascending dose (MAD) study. FREEDOM2 Phase 2 Multiple Ascending Dose (MAD) Randomized, Placebo-Controlled Clinical Trial of PGN-EDODM1: The Company expects to report results from the 5 mg/kg cohort of the FREEDOM2 trial in the first quarter of 2026. Corporate Updates In May 2025, PepGen appointed Kasra Kasraian, PhD, as Chief Technology Officer. Dr. Kasraian brings over 25 years of experience in product and process development, CMC strategy, and technical operations, spanning small and large molecules, as well as cell and gene therapies. In May 2025, the Company presented an oral presentation and two posters at the 2025 Myotonic Dystrophy Foundation (MDF) Conference. PepGen also presented at the 5th Edition of Euro-DyMA's Pharma Day, held in conjunction with the MDF Conference. These presentations highlighted the recently announced FREEDOM clinical data in DM1, as well as proof-of-mechanism preclinical results for PGN-EDODM1. Financial Results for the Three Months Ended June 30, 2025 Cash, Cash Equivalents and Marketable Securities were $74.7 million as of June 30, 2025. Based on currently planned operations, the Company believes that its existing cash, cash, equivalents, and marketable securities will be sufficient to fund its operations into the second quarter of 2026. Research and Development Expenses were $18.4 million for the three months ended June 30, 2025, compared to $25.1 million for the same period in 2024. General and Administrative Expenses were $5.5 million for the three months ended June 30, 2025, compared to $5.4 million for the same period in 2024. Net Loss was $23.1 million, or $(0.70) basic and diluted net loss per share, for the three months ended June 30, 2025, compared to $28.3 million, or $(0.87) basic and diluted net loss per share, for the same period in 2024. PepGen had approximately 32.8 million shares outstanding on June 30, 2025. About PGN-EDODM1 PGN-EDODM1, PepGen's investigational candidate in development for the treatment of myotonic dystrophy Type 1 (DM1), utilizes the Company's proprietary Enhanced Delivery Oligonucleotide (EDO) technology 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 dystrophia myotonic protein kinase (DMPK) transcripts which sequester MBNL1, by binding to the pathogenic CUG trinucleotide repeat expansion present in the DMPK transcripts, disrupting the binding between the CUG repeat expansion and MBNL1. DM1 is a progressively disabling, life-shortening genetic disorder. DM1 is estimated to affect 40,000 people in the United States, and over 74,000 people in Europe. 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. About PepGen PepGen 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’s 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, our goal is to generate a pipeline of oligonucleotide therapeutic candidates designed to target the root cause of serious diseases. For more information, please visit PepGen.com. Follow PepGen on LinkedIn and X. Forward-Looking Statements This 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 "aims," "anticipates," "believes," "could," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," 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 lead product candidate, including, based on early data, PGN-EDODM1, the magnitude of the cost savings from concluding the FREEDOM 1 trial with the 15 mg/kg cohort, the ability to complete enrollment and dose escalation in the FREEDOM2 trial, the expected timing for additional data reports from our FREEDOM Phase 1 trial and FREEDOM2 trial, ongoing and planned regulatory interactions, and our financial resources and expected cash runway. Any 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 current or future product candidates, including PGN-EDODM1; our ability to enroll patients in our clinical trials, including 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-EDODM1; our current and future 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 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 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’s 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. This release discusses our lead product candidate, 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. View source version on businesswire.com: https://www.businesswire.com/news/home/20250807282316/en/ Contacts Investor Contact Laurence Watts New Street Investor Relations [email protected] Media Contact Julia Deutsch Lyra Strategic Advisory, LLC [email protected]

Investor releaseQuarter not tagged2025-05-08

PepGen Reports First Quarter 2025 Financial Results and Recent Corporate Highlights

Business Wire
– Reported positive DM1 patient data with mean splicing correction of 29.1% following a single dose of PGN-EDODM1 at 10 mg/kg – – FREEDOM-DM1 data from 15 mg/kg cohort expected in the second half of 2025 – – CONNECT1-EDO51 data from 10 mg/kg cohort expected in the third quarter of 2025 – BOSTON, May 08, 2025--(BUSINESS WIRE)--PepGen 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 and recent corporate highlights for the quarter ended March 31, 2025. "Our EDO platform’s unique ability to efficiently deliver oligonucleotides into the nuclei of cells is the foundation of our differentiated pipeline, with promising clinical data from two programs underscoring what we believe to be the broad potential of our technology," said James McArthur, PhD, President and CEO of PepGen. "This is a pivotal and catalyst-rich 12 months for PepGen and for our DM1 and DMD clinical programs. Following the highly encouraging initial results from our FREEDOM-DM1 study reported in February, demonstrating potentially best-in-class splicing correction after a single therapeutic dose, we look forward to sharing data from the 15 mg/kg cohort in the second half of the year. We also expect to report dystrophin production data from the 10 mg/kg cohort of our CONNECT1-EDO51 study in patients with DMD in the third quarter. Collectively, we believe these data will build on the body of evidence supporting our platform’s promise. Additionally, during the first quarter, we have undertaken various measures to extend our cash runway while prioritizing delivering on our key clinical milestones." Recent Program Updates PGN-EDODM1: Myotonic Dystrophy Type 1 (DM1) Phase 1 FREEDOM-DM1 Single Ascending Dose (SAD) Randomized, Placebo-Controlled Clinical Trial of PGN-EDODM1: In February, the Company reported positive initial clinical data from the 5 mg/kg and 10 mg/kg dose cohorts from the ongoing FREEDOM trial. At day 28 following a single dose, PGN-EDODM1 was observed to have a favorable emerging safety profile and robust, dose-dependent splicing correction in the 5 and 10 mg/kg dose cohorts. PepGen expects to report safety, 28-day splicing and functional benefit data from the 15 mg/kg cohort during the second…Read full document

– Reported positive DM1 patient data with mean splicing correction of 29.1% following a single dose of PGN-EDODM1 at 10 mg/kg – – FREEDOM-DM1 data from 15 mg/kg cohort expected in the second half of 2025 – – CONNECT1-EDO51 data from 10 mg/kg cohort expected in the third quarter of 2025 – BOSTON, May 08, 2025--(BUSINESS WIRE)--PepGen 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 and recent corporate highlights for the quarter ended March 31, 2025. "Our EDO platform’s unique ability to efficiently deliver oligonucleotides into the nuclei of cells is the foundation of our differentiated pipeline, with promising clinical data from two programs underscoring what we believe to be the broad potential of our technology," said James McArthur, PhD, President and CEO of PepGen. "This is a pivotal and catalyst-rich 12 months for PepGen and for our DM1 and DMD clinical programs. Following the highly encouraging initial results from our FREEDOM-DM1 study reported in February, demonstrating potentially best-in-class splicing correction after a single therapeutic dose, we look forward to sharing data from the 15 mg/kg cohort in the second half of the year. We also expect to report dystrophin production data from the 10 mg/kg cohort of our CONNECT1-EDO51 study in patients with DMD in the third quarter. Collectively, we believe these data will build on the body of evidence supporting our platform’s promise. Additionally, during the first quarter, we have undertaken various measures to extend our cash runway while prioritizing delivering on our key clinical milestones." Recent Program Updates PGN-EDODM1: Myotonic Dystrophy Type 1 (DM1) Phase 1 FREEDOM-DM1 Single Ascending Dose (SAD) Randomized, Placebo-Controlled Clinical Trial of PGN-EDODM1: In February, the Company reported positive initial clinical data from the 5 mg/kg and 10 mg/kg dose cohorts from the ongoing FREEDOM trial. At day 28 following a single dose, PGN-EDODM1 was observed to have a favorable emerging safety profile and robust, dose-dependent splicing correction in the 5 and 10 mg/kg dose cohorts. PepGen expects to report safety, 28-day splicing and functional benefit data from the 15 mg/kg cohort during the second half of 2025. Phase 2 FREEDOM2-DM1 Multiple Ascending Dose (MAD) Randomized, Placebo-Controlled Clinical Trial of PGN-EDODM1: The Company expects to report results from the 5 mg/kg cohort of the FREEDOM2 trial in the first quarter of 2026. PGN-EDO51: Duchenne Muscular Dystrophy (DMD) Phase 2 CONNECT1-EDO51 Open-Label MAD Clinical Trial of PGN-EDO51: 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. Phase 2 CONNECT2-EDO51 MAD Clinical Trial of PGN-EDO51: 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. Corporate Updates In March 2025, PepGen announced that Lisa Wyman and Mitchell H. Finer, PhD, joined the Company’s Board of Directors. Each are industry veterans bringing decades of executive and operational experience in life sciences to the Company. In March 2025, the Company gave two oral presentations and presented five posters at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. The oral presentations featured data from the ongoing CONNECT1 clinical trial in DMD and the FREEDOM clinical trial in DM1. In May 2025, the Company made an oral presentation and presented two posters at the 2025 Myotonic Dystrophy Foundation (MDF) Conference. PepGen also presented at the 5th Edition of Euro-DyMA's Pharma Day, held in conjunction with the MDF Conference. These presentations highlighted the recently announced FREEDOM clinical data in DM1, as well as proof-of-mechanism preclinical results for PGN-EDODM1. Financial Results for the Three Months Ended March 31, 2025 Cash, Cash Equivalents and Marketable Securities were $97.8 million as of March 31, 2025. Based on currently planned operations, the Company believes that its existing cash, cash, equivalents, and marketable securities will be sufficient to fund its operations for at least 12 months from the date of this release. Research and Development Expenses were $25.4 million for the three months ended March 31, 2025, compared to $14.7 million for the same period in 2024. General and Administrative Expenses were $5.9 million for the three months ended March 31, 2025, compared to $5.1 million for the same period in 2024. Net Loss was $30.2 million, or $(0.92) basic and diluted net loss per share, for the three months ended March 31, 2025, compared to $18.0 million, or $(0.63) basic and diluted net loss per share, for the same period in 2024. PepGen had approximately 32.7 million shares outstanding on March 31, 2025. Upcoming Potential Milestones 3Q 2025: CONNECT1-EDO51 data from 10 mg/kg cohort expected 2H 2025: FREEDOM-DM1 data from 15 mg/kg cohort expected 1Q 2026: FREEDOM2-DM1 data from the 5 mg/kg cohort expected About PGN-EDODM1 PGN-EDODM1, PepGen's investigational candidate in development for the treatment of DM1, utilizes the Company's proprietary Enhanced Delivery Oligonucleotide (EDO) technology to deliver a therapeutic oligonucleotide that is designed to restore the normal splicing function of MBNL1, a key RNA splicing protein. DM1 is a progressively disabling, life-shortening genetic disorder. DM1 is estimated to affect 40,000 people in the United States, and over 74,000 people in Europe. 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. About PGN-EDO51 PGN-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 U.S. Food and Drug Administration 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. About PepGen PepGen 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’s 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. For more information, please visit PepGen.com. Follow PepGen on LinkedIn and X. Forward-Looking Statements This 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 "aims," "anticipates," "believes," "could," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," 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-EDODM1 and PGN-EDO51, the expected timing for additional data reports from our FREEDOM Phase 1 trial and CONNECT1 Phase 2 trial and initial data from our FREEDOM2 Phase 2 trial, ongoing and planned regulatory interactions, and our financial resources and expected cash runway. Any 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 and PGN-EDO51; our ability to enroll patients in our clinical trials, including FREEDOM, FREEDOM2 and CONNECT2; 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 and PGN-EDO51; our product candidates, including PGN-EDODM1 and PGN-EDO51, 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 FREEDOM, FREEDOM2, CONNECT1 and CONNECT2 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’s 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. This release discusses PGN-EDODM1 and PGN-EDO51, 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-EDODM1, PGN-EDO51 or any other investigational therapy will successfully complete clinical development or gain regulatory authority approval. View source version on businesswire.com: https://www.businesswire.com/news/home/20250508029516/en/ Contacts Investor Contact Laurence Watts New Street Investor Relations [email protected] Media Contact Julia Deutsch Lyra Strategic Advisory, LLC [email protected]

TranscriptFY2023 Q42024-03-07

FY2023 Q4 earnings call transcript

Earnings source - 35 paragraphs
Operator

Good afternoon, and welcome to PepGen's Fourth Quarter and Full Year 2023 Earnings Call to discuss its financial results and recent corporate developments. At this time all participants are on a listen-only mode. Following the manager's prepared remarks we will hold a question-and-answer session. [Operator Instructions] As a reminder, this call is being recorded today, Thursday, March 6, 2024. I would now like to turn the conference call over to Emiko Bryant, Chief of Staff of PepGen. Emiko, please go ahead.

Emiko Bryant

Thank you, operator. Good afternoon, everyone, and thank you for joining today's call. Earlier today, we released our financial results for the fourth quarter and full year 2023 and provided an update on recent corporate developments. The press release and our 10-K filed with the SEC this afternoon outlining our financial results are both available on our website at pepgen.com. Joining me on the call today are James McArthur, PhD, President and Chief Executive Officer; Dr. Michelle Mellion, Senior Vice President, Head of Clinical Development; and Noel Donnelly, Chief Financial Officer. As a reminder, we will be making forward-looking statements regarding our financial outlook in addition to regulatory and product development plans and research activities. These statements are subject to risks and uncertainties that may cause actual results to materially differ from those forecasted. A description of these risks can be found in our most recent 10-K on file with the SEC. PepGen does not undertake any obligation to publicly update its forward-looking statements as a result of new information, future events or changes in its expectations. I will now turn the call over to our CEO, James McArthur. James?

James McArthur

Thank you, Emiko, and good afternoon, everyone. PepGen made meaningful progress over the course of 2023, including the initiation of our first inpatient clinical trials evaluating programs derived from PepGen Enhanced Delivery Oligonucleotide, or EDO, cell-penetrating peptide platform in two neuromuscular diseases with high unmet medical need. Our lead clinical program, PGN-EDO51 or EDO51 is designed for the treatment of patients with Duchenne Muscular Dystrophy, also known as DMD, whose disease is amenable to an exon 51-skipping approach. As a reminder, an estimated 13% people with DMD or approximately 4,200 individuals in the U.S. and EU have a mutation amenable to an exon 51-skipping approach. Our second clinical program, PGN-EDODM1 or EDODM1 is designed for the treatment of Myotonic Dystrophy Type 1, or DM1. We estimate that DM1 affects more than 100,000 people in the U.S. and Europe for whom there are currently no approved disease-modifying treatments. Here at PepGen, developing potentially transformative medicines is the foundation of our mission and long-term vision for building the company. We believe that both our DMD and DM1 programs have the potential to be disease-modifying and meaningfully improve outcomes for patients. Our team is committed to advancing these important programs through the clinic with a sense of urgency to get them to the people living with these diseases as quickly as possible. We believe our recent stock offering puts us in a strong financial position for our team to execute on this commitment. The net proceeds from this offering, together with our existing cash and cash equivalent, extend our projected cash runway into 2026. Turning to the latest updates and highlights from our clinical programs, we are pleased to announce we have completed enrollment for cohort 1 in CONNECT1-EDO51, evaluating the 5 mg per kg dose in DMD patients in our first Phase II clinical trial. Following the review of the safety data of the 5 mg per kg dose cohort by our Data Safety Monitoring Board, and assuming an acceptable emerging safety profile, we plan to escalate to the second cohort at 10 mg per kg of EDO51. The same process will take place prior to advancing to cohort 3. In parallel, earlier this week, we announced we received clearance from the MHRA in the UK to initiate CONNECT2-EDO51, our Phase II study of EDO51 in people with DMD amenable to an exon 51-skipping therapy. We currently expect to be initiating dosing in cohort 1 evaluating the 5 mg per kg dose level in patients in the UK in the third quarter of 2024. Following CONNECT1's preliminary data readout for the 5 mg per kg cohort, we expect to open trial sites for CONNECT2 in other geographies, including the U.S., subject to regulatory authorizations. We anticipate reporting preliminary data for the CONNECT1 5 mg per kg cohort in mid-2024, including safety, exon skipping and dystrophin production. Based upon externally available data and using our own clinical and nonclinical work for internal modeling assumptions, PepGen expects treatment with EDO51 in DMD patients to produce high levels of dystrophin protein. At the 5 mg per kg dose level, we expect to see greater than 1% of normal levels of dystrophin protein above background levels in the CONNECT1-EDO51 trial as measured by Western Blot analysis following four repeat doses of EDO51 in DMD patients. For our 10 mg per kg dose cohort, if EDO51 were to achieve dystrophin levels of greater than 7%, this would be the highest level of dystrophin production achieved by a DMD exon-skipping therapy to date. Our modeling projections for this dose level suggests the possibility that we could potentially achieve greater than 9% of normal levels of dystrophin protein. The combined safety and dystrophin expression data package from CONNECT1 and CONNECT2 is designed to support a potential path towards accelerated approval, assuming alignment with regulatory authorities. Turning to our DM1 development program, we were pleased that the clinical hold on EDODM1 was lifted by the FDA in October 2023. Following discussions with regulatory authorities, we are advancing our EDODM1 therapy at the same dose level starting at 5 mg per kg across all countries, including the U.S., in the Phase I FREEDOM-DM1 clinical trial for people living with DM1. Just last month, EDODM1 was granted fast track designation by FDA. This designation is designed to facilitate the development, and expedite the review of potential therapies designed to treat serious diseases and conditions with clear unmet medical needs. Importantly, fast track designation allows for early and more frequent communication with the FDA, which can potentially lead to earlier drug approval and access for patients. In December 2023, PepGen announced the first patient was dosed in the Phase I FREEDOM-DM1, single ascending dose clinical trial, and we expect to report preliminary data, including safety, splicing correction and functional outcome measures from at least 5 mg per kg dose cohort in the second half of 2024. We expect both the 5 mg per kg dose and 10 mg per kg dose evaluated in the FREEDOM-DM1 clinical study to be pharmacologically active and believe that 10 mg per kg dose could exhibit meaningful splicing correction and myotonia correction. In addition to our FREEDOM-DM1 trial, we expect to open our FREEDOM2-DM1, placebo-controlled multiple ascending dose clinical trial in DM1 patients in the second half of 2024. An important differentiator of our PGN-EDODM1 is that it is designed to selectively target the pathogenic DMPK RNA with the CUG repeat expansion rather than degrading both the normal as well as pathogenic DMPK RNA. As a result of this selectivity, and based on our preclinical data, we believe that EDODM1 has the potential to achieve superior correction of splicing events at a well-tolerated dose levels, which could lead to improved functional outcomes for patients. In addition to our clinical programs, our research team continues to advance and evaluate our preclinical candidates in key areas of focus for neuromuscular and neurologic disorders. PGN-EDO53 is our lead preclinical program designed to skip exon 53 of the dystrophin transcript, a therapeutic target for approximately 8% of patients with DMD. We previously reported superior exon skipping in repeat dose studies in nonhuman primates, and our team is commencing IND and CTA-enabling studies in 2024. We look forward to providing more details as we progress. I will now turn the call over to Dr. Michelle Mellion, PepGen's Head of Clinical Development, to provide an in-depth review of the trial designs, of the ongoing clinical trials in DMD and DM1 that I just mentioned. Michelle?

Michelle Mellion

Thank you, James. Starting with our clinical trials in DMD, CONNECT1 is a Phase II 13-week open-label, multiple-ascending dose clinical trial that is enrolling both ambulatory and non-ambulatory boys and young men living with DMD amenable to an exon 51-skipping approach. Each of the DMD patients must be at least eight years of age to enroll and will provide a muscle biopsy pre-dose and on week 13. The dosing of EDO51 will occur once every four weeks for 12 weeks. We will evaluate safety data from three subjects in the 5 mg per kg dose cohort with the DSMB before progressing to the 10 mg per kg dose cohort. We will evaluate further dose escalations based upon the evaluation of safety data from prior dose cohorts. CONNECT2 is a multinational Phase II 26-week double-blind, placebo-controlled multiple ascending dose clinical trial that will enroll both ambulatory and non-ambulatory boys and young men living with DMD amenable to an exon 51-skipping therapy who are at least six years old. Participants will provide a muscle biopsy at baseline and then at week 25. ED051 will be administered every four weeks for six months. The DSMB will review the data before we proceed to the next dose cohort. In February, we received authorization from the MHRA for our CTA to initiate CONNECT2 in the UK and are planning to open the study in the EU and the U.S. later this year following regulatory clearance. Turning to our EDODM1 development plan, we are pleased to have dosed the first patient in our FREEDOM-DM1 global Phase I single ascending dose randomized, double-blind, placebo-controlled trial of EDODM1 in DM1 patients in December 2023. FREEDOM-DM1 will enroll a total of 24 DM1 patients randomized 3 to 1 in favor of drug versus placebo, evaluating 5, 10 and up to 20 mg per kg with dose escalation following review of safety data from prior dose cohorts. The subjects will provide a muscle biopsy at baseline followed by a single infusion of EDODM1 with muscle biopsies taken again at day 28 and week 16. Our FREEDOM-DM1 study will inform our planned multinational Phase II trial in DM1, a multiple ascending dose clinical trial that is designed to support potential regulatory approvals subject to alignment with regulatory authorities. We anticipate opening the Phase II trial of EDODM1 in the second half of 2024 following discussions with the regulators. With that review of PepGen's clinical development plans, I will now hand the line to Noel Donnelly, our Chief Financial Officer, to review our latest financial results. Noel?

Noel Donnelly

Thank you, Michelle. My comments will reflect the high-level financial results of our fourth quarter and full year 2023 period. More details are provided in this afternoon's financial results press release and in the corresponding SEC filing. As of December 31, 2023, PepGen held $110.4 million in cash and cash equivalents compared to $181.8 million on December 31, 2022. As James mentioned previously, on February 9, 2024, PepGen successfully completed an underwritten stock offering of 7.53 million common shares for gross proceeds of approximately $80 million. Based on our current operating plans, PepGen's current cash and cash equivalents, including the proceeds of the offering are expected to fund operations into 2026. Net loss for the fourth quarter of 2023 was $19.5 million. Our net loss for the full year 2023 was $78.6 million. Research and development expenses for the three months ended December 31, 2023, were $16.3 million. For the full year 2023, research and development expenses were $68.1 million. The increase in research and development expenses in the fourth quarter of 2023 compared to the fourth quarter of 2022 was primarily attributable to costs associated with the advancement of the company's PGN-ED051 and PGN-EDODM1 programs, including preclinical, clinical and manufacturing costs related to our ongoing and future clinical trials. General and administrative expenses were $4.5 million for the three months ended December 31, 2023. General and administrative expenses for the full year 2023 were $16.6 million. The increase in general and administrative expenses was primarily due to an increase in personnel-related costs. Finally, as of February 29, 2024, PepGen had approximately 32.4 million shares outstanding. And with that, I will turn the call back to James.

James McArthur

Thank you, Noel. I'm very proud of our team's ability to advance multiple clinical programs in 2023 and look forward to continuing the successful operational execution over the course of 2024 and beyond. This year is an important time for PepGen as we will have multiple upcoming clinical data readouts from our ongoing clinical trials in DMD and DM1 patients, making the first inpatient clinical data from our proprietary EDO platform. To quickly review our anticipated data announcements for the year. In the middle of 2024, we expect to report preliminary data from the 5 mg per kg dose cohort in CONNECT1-EDO51, the multiple ascending dose trial in boys with DMD. We plan to provide safety, exon 51-skipping and dystrophin production data. For the multinational FREEDOM-DM1 Phase I trial of EDODM1 in patients with DM1, we anticipate reporting preliminary safety, splicing correction and functional outcome measures from at least the 5 mg per kg dose cohort in the second half of 2024. With that, I will open the call for questions. Operator?

Operator

Thank you. [Operator Instructions] And our first question comes from Joseph Schwartz from Leerink Partners. Your line is now open.

Jenny Gonzalez-Armenta

Hi, everyone. This is Jenny on for Joe. I was just wondering if you could talk a little bit more about the mechanism of PGN-EDODM1? And if you expect a blocking risk approach to result in a more specific profile and how that might read through to splicing correction and any implications for safety? Thank you.

James McArthur

Thank you, Jenny. Our mechanism, unlike other approaches, which are seeking to degrade both the pathogenic DMPK as well as the nonpathogenic DMPK, is targeting the CUG repeat. And we've been able to demonstrate in cell-based models that we can liberate MBNL1 as well as reduce the number of toxic foci and nuclei of patient cells as well as dramatically correct this splicing in patient cells. And in those models, we're also similarly able to correct splicing and importantly, correct both the electrophysiologic as well as the observable myotonia in the mouse model. This is a very profound response, and we were able to do this at dose levels, which we were able to demonstrate achievable with our EDO051 technology in healthy volunteers, which employs the same EDO peptide and conjugation chemistry to the PMO. As such, we believe that we are specifically targeting the toxic species that drive this disease and coupling it to our EDO platform technology, which has been demonstrated to reduce the highest level of exon 51-skipping following a single dose in humans as well as robust delivery in nonhuman primates and mice coupled with excellent exon skipping. As such, we believe that at doses that we say are low risk (phon), we can achieve robust levels of splicing correction that will allow us to demonstrate correction of myotonia and improvement in strength. Although it's not been demonstrated, that [indiscernible] efficiency is a toxicologic challenge in this disease by indiscriminately knocking down DMPK, we believe that we will be able to avoid this by targeting the RNA species that is pathogenic and drives the fundamentals of this disease.

Operator

And thank you. One moment for our next question. And our next question comes from Paul Matteis from Stifel. Your line is now open.

James Condulis

This is James on for Paul. Thanks for taking our question. I just had one as it relates to the upcoming readout. Just as it relates to dystrophin specifically, there's been some discussions recently about different cuts of dystrophin, including an adjusted figure that accounts for muscle fat content. And you mentioned for your study at 5 mg per kg, you're looking for greater than 1% dystrophin in that. For 10 mg per kg, hoping to get above 9%, I guess, one, are these unadjusted dystrophin measures? And then two, just how much above 1% dystrophin do we need to see initial readout to have confidence that we can get to 9% plus at the next dose? Thanks so much.

James McArthur

Great. I appreciate the question. So you're correct that we are planning on reporting dystrophin levels above background. The reason why this is important is an individual enters the study with 0.5% endogenous levels of dystrophin and one can only elevate it by 0.3%. It's really a net of 0.3% gain of dystrophin. And so we are looking to see at least a 1% gain over background in terms of dystrophin levels at the 5 mg per kg dose level, but we have the possibility to go and see higher levels still. The reason why this 1% would give us confidence that at 10 mg per kg, we could produce 9% or better levels of dystrophin in patients is that when we looked in our nonhuman primate studies, we've observed that a single dose at a low level of 20 mg per kg, produces approximately 2% dystrophin. But following four monthly doses of EDO51 in nonhuman primates, this has increased to almost 35% exon skipping. I apologize, I misspoke. It was 2% exon skipping going to 35% exon skipping. Obviously, higher levels of exon skipping will produce higher levels of dystrophin production. But it gives us a good sense that going from low single-digit levels we have the potential to see much higher levels of exon skipping and dystrophin production. This is also supported, as I mentioned in the call by our modeling work based on both, primary work in cells in the mouse model, nonhuman primates. And of course, the work we have done in healthy volunteers.

James Condulis

Thanks very much.

Operator

And thank you. One moment for our next question. And our next question comes from Tazeen Ahmad from Bank of America. Your line is now open.

Tazeen Ahmad

Great. Thanks so much for taking my questions. Just two quick ones on DMD. For the 10 mg per kg cohort, have you already started the process of identifying patients for that? And based on your timeline for when to expect the 5 mg data, do you think it would be a reasonable expectation to expect data from the 10 mg cohort this year as well?

James McArthur

Thank you, Tazeen. So we see a lot of enthusiasm from our clinical investigators for the CONNECT1 clinical study and we anticipate seeing robust recruitment of this study. As I've mentioned, we've already recruited cohort 1 of 5 mg per kg cohort and we anticipate reporting out that data set mid- of this year. As soon as we have an update for you in terms of recruitment, we will give more guidance in terms of the timing of the 10-mg per kg data readout, but we anticipate being able to report that in a timely fashion based on the enthusiasm we're seeing for those patients as well as investigators in the study.

Operator

And thank you. One moment for our next question. And our next question comes from Laura Chico from Wedbush.

Laura Chico

Hey. Good afternoon, guys. Thanks very much for taking the questions. I have two for you. So with respect to CONNECT1 data. James, I'm wondering if you could spend a moment discussing a little bit more about the preliminary kinetics and the pace of exon skipping that you're seeing with initial doses. I think in the nonhuman primates, if I'm not mistaken, there was kind of a maximal effect or a plateauing, which occurred over time. I'm curious if you kind of anticipate seeing that in the patient samples. I guess what's the expectation that the magnitude of skipping can increase over time? And then I have a quick follow-up for you.

James McArthur

Terrific. I appreciate the question, Laura. You're correct. When we look at our nonhuman primate data for both our 51 as well as our 53 program, we got to observe some of the greatest increase that we've seen in terms of exon skipping is occurring from dose 1 to dose 2, a smaller level from dose 2 to dose 3 and a smaller increase still from dose 3 to dose 4, And it does appear that we are reaching these very high levels of exon skipping by dose 4, and there may not be incrementally that much more that one can achieve. As such, we do anticipate seeing better than 7% and very likely above 9% dystrophin production based on the level of exon skipping and the modeling work that we've done, based on the extrapolation we've done from prior work of other companies where we compare our single-dose exon skipping in humans to their single-dose exon skipping in humans. And then lastly, based on the very extensive nonhuman primate modeling work we've done looking at both single and multiple doses. So as such, we do expect to see very robust levels of exon skipping following four doses. And this will be reflective of what we're able to achieve, we believe, long term.

Laura Chico

Okay, that's helpful. And then maybe one quick question on DM1 and I'll hop back in the queue here. With respect to kind of the magnitude of splicing correction that you're looking for, I'm wondering if you could kind of share any more color around what you think would be a meaningful level and if you could just remind us with respect to FREEDOM1, what's the extent of functional assessments that we will be getting in the second half update? Thanks very much.

James McArthur

Let me first speak to the splicing correction work that we've done pre-clinically and then I'll hand it over to Michelle Mellion to speak to the outcome measures that we'll be looking at in Freedom1. So in terms of the preclinical work that we have conducted, we've observed that with higher and higher levels of splicing correction in the mouse model, which is granted an engineered animal model, we can go and see higher and higher levels of myotonia correction, both from the standpoint of the dragging the high lens [ph] as we observed in this model as well as measurement by electrophysiology. As such, if we're looking at 30%, 40% splicing correction at that level in the mouse model, we're beginning to see robust levels of correction of myotonia. And as we approach 60% at higher levels of splicing correction, we can begin to approach 70%, 80% correction of the myotonia. So we do believe it's important to be able to demonstrate better than 25%-plus splicing correction to see really robust changes in terms of the physiology and the pathology of this disease. With that I'll turn it over to Michelle to speak to the outcome metrics that we'll be looking at in Freedom1.

Michelle Mellion

So in FREEDOM 1, our clinical outcome measures include a full assessment of symptoms related to DM1. And this assessment includes the vHoT, which is an assessment of myotonia that I believe most are now familiar with as well as strength assessments of several different muscles, including risk and other functional outcome measures such as the 10-meter walk test and the [indiscernible] and these will be done at different endpoints or time points during the study to assess the impact of splicing correction on these assessments.

James McArthur

And I just want to add to what Michelle said. As was mentioned earlier, the FREEDOM1 clinical study is a single ascending dose clinical study and we expect both based on our work as well as the work of others that following a single dose, we can reasonably expect to see both robust splicing correction as well as changes in terms of myotonia. Some of the other assessments that Michelle mentioned that we will be looking at maintain multiple doses before we start seeing a meaningful change there. But we will get a very good sense from the data that we'll be presenting this year on the power of the EDODM1 molecule to go and really change the pathology of this disease.

Laura Chico

Thanks very much. Appreciate it.

Operator

And thank you. [Operator Instructions] One moment for our next question. And our next question comes from Ananda Ghosh from H.C. Wainwright & Company. Your line is now open.

Ananda Ghosh

Hi, James. Thanks for the opportunity. I had two questions, one on DMD and that is with respect to the recent SRP-5051 data. What does that data tell you about your program concerning the platform and the EDO51? And then I have one follow-up question on the DM1 program.

James McArthur

Thank you, Ananda. I appreciate the question. So we have done a cross-trial comparison of the ability of EDO51 to mediate exon 51 skipping in humans following a single dose. And there, we were able to observe six fold higher levels of exon 51 skipping compared to a single dose of the 5051 molecule at 20 mgs per kg. 20 mg per kg 5051 was relatively well tolerated at 10 mg per kg, ED051, demonstrated following the single dose in humans only grade 1 reversible transient adverse events. So very, very well tolerated drug. We anticipate, as we extrapolate forward that we could be producing six fold higher levels of exon skipping and potentially dystrophin than what was observed in both the three month Momentum A and the six month Momentum B clinical studies with 5051. This is what supports our contention that we have the potential to produce greater than 9% dystrophin levels in patients following four doses. And so we remain very confident, both based on the extrapolation in the cross-trial comparison to that clinical data, the work that we've done in animal modeling and nonhuman primates and the overall modeling work that has incorporated both mouse, nonhuman primate and human data with EDO051 that indeed, we'll be able to see better than 9% dystrophin in patients. If we could even achieve 7% dystrophin production with an exon skipping approach, this will be the highest level of dystrophin produced by any exon-skipping drug and projected to be produced by any exon-skipping drug. And so we would be -- feel that to be a huge success but we believe based on all the work we've done, we have the potential to be greater than to produce later than 9% dystrophin in patients.

Ananda Ghosh

Got it. Thanks. With respect to the DM1 program, there has been a debate in terms of what kind of primary endpoint the companies might see as the DM1 programs advance. And so there have been a lot of discussions around the vHoT. So what is your take about the agency's view on primary endpoint in the DM1 program? And my follow-up question to that is, is there a correlation between the splicing correction and the vHoT assay measurements?

James McArthur

Perhaps I could start off, and then I'll ask Michelle Mellion to add to my thoughts. So these conversations with regulators are ongoing and so we cannot speak to what would be the approval end points from our FREEDOM2 and beyond clinical studies. What we can say is that we believe that splicing is an important mechanistic underpinning of the EDODM1 program and with greater levels of slicing correction. We have, in animal models, seen greater levels of myotonia correction. And as such, we would expect to see with greater levels of slicing correction in patients, we would see greater levels of myotonia corrections and longer-term correction of many of the movement related topologies of this disease. So it is our anticipation that all of these will go into ultimately what would become an approvable endpoint. But let me allow Michelle to perhaps add to that.

Michelle Mellion

Yes, that's right, James. I believe that we will learn from our FREEDOM-DM1 study, which is our single ascending dose study about the potential impact on several of these intermediate endpoints such as vHoT and potentially at later time points grip and strength. And it is likely that looking at the totality of these endpoints taken together, the vHoT strength, grip and may be selected functional endpoints such as the 10 meter walk test, we'll be able to assess the full impact of placing correction over multiple doses in a future study on these endpoints to appreciate the potential impact on the disease that our therapies will have for this population.

Ananda Ghosh

Got it. Thanks very much. Much appreciated.

James McArthur

Thank you.

Operator

Thank you. And I am showing no further questions. I would now like to turn the call back over to James McArthur for closing remarks.

James McArthur

Thank you, operator, and thank you, everybody, for participating in today's call. If you have any questions or follow-up items, please do reach out to me or Noel. We look forward to connecting with all of you soon at upcoming investor conferences. Have a great evening. Thank you.

Operator

This concludes today's conference call. Thank you for participating. You may now disconnect.

As of 2026-08-08 • Updated weeklySource: Earnings sourceIngestion runbook