Biotech Stock Pepgen Inc. (NASDAQ: PEPG) Makes NASDAQ Top Gainers List On Financial Results And Update

(MENAFN- Investor Ideas) Investorideas ( ), a go-to platform for big investing ideas for stock traders, including biotech Stocks reports on trading and news for 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.

The stock makes the nasdaq top percentage gainer list, currently trading at 2.7402, up 1.3702, gaining 100.0146% on volume of over 102 Million shares. The stock has a day's high of $4.48.

PepGen today reported financial results for the quarter and year-ended December 31, 2024, and recent corporate highlights.

"Today, we reported initial results from FREEDOM-DM1, which showed robust splicing correction in patients with DM1 following a single dose of PGN-EDODM1. We believe these data contribute to the growing evidence of our novel EDO platform's potential to deliver the drug to the nucleus, the site of action," said James McArthur, PhD, President and CEO of PepGen. "We remain dedicated to our mission of developing life-changing therapeutics for patients with neuromuscular diseases and are committed to demonstrating the ability of our investigational candidates to address the root cause of disease for improved patient outcomes. We have numerous key data milestones expected this year and look forward to announcing clinical results from the 15 mg/kg cohort of FREEDOM and 10 mg/kg cohort of CONNECT1-EDO51."

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:

The Company today reported positive initial clinical data, including mean splicing correction of 12.3% and 29.1% from evaluable participants in the 5 mg/kg (n=6) and 10 mg/kg (n=4)1 dose cohorts, respectively, and a favorable emerging safety profile in the ongoing FREEDOM study. The Company issued a separate press release this morning announcing these initial results and will host a webcast with a live question and answer session today at 8:00 a.m. ET.

The Company expects to report results from the 15 mg/kg cohort in the second half of 2025.

Phase 2 FREEDOM2-DM1 Multiple Ascending Dose (MAD) Randomized, Placebo-Controlled Clinical Trial of PGN-EDODM1:

The Company has opened the FREEDOM2 study and has started dosing participants in the 5 mg/kg dose cohort. The Company expects to report results from the 5 mg/kg cohort in the first quarter of 2026.

PGN-EDO51: Duchenne Muscular Dystrophy (DMD)

Phase 2 CONNECT1-EDO51 Open-Label MAD Clinical Trial of PGN-EDO51:

The 10 mg/kg cohort is fully enrolled (n=4) and participants in the 5 mg/kg cohort (n=3) are continuing in the long-term extension phase of the study. The Company expects to report clinical data from the 10 mg/kg cohort in the third quarter of 2025.

As previously reported on January 29, 2025, Health Canada has allowed continued dosing of participants in the 5 and 10 mg/kg cohorts with additional information requested to address its safety concerns before further dose escalation or enrollment of any additional participants at the current dose levels. The Company is working with Health Canada to address its questions.

Phase 2 CONNECT2-EDO51 MAD Clinical Trial of PGN-EDO51:

CONNECT2 is open in the United Kingdom. As previously reported, the Company received a clinical hold notice from the U.S. Food and Drug Administration (FDA) in December 2024 regarding its investigational new drug (IND) application to initiate the CONNECT2 study in the U.S. and is working to address its questions regarding supportive data for the dosing levels planned for the patient population.

Financial Results for the Three and Twelve Months Ended December 31, 2024

Cash, Cash Equivalents and Marketable Securities were $120.2 million as of December 31, 2024, which is anticipated to fund currently planned operations into 2026.

Research and Development Expenses were $19.0 million for the three months ended December 31, 2024, compared to $16.3 million for the same period in 2023. Research and development expenses were $76.5 million for the year ended December 31, 2024, compared to $68.1 million for the same period in 2023.

General and Administrative Expenses were $5.4 million for the three months ended December 31, 2024, compared to $4.5 million for the same period in 2023. General and administrative expenses were $21.3 million for the year ended December 31, 2024, compared to $16.6 million for the same period in 2023.

Net Loss was $22.2 million, or $(0.68) basic and diluted net loss per share, for the three months ended December 31, 2024, compared to $19.5 million, or $(0.82) basic and diluted net loss per share, for the same period in 2023. Net loss was $90.0 million, or $(2.85) basic and diluted net loss per share, for the year ended December 31, 2024, compared to $78.6 million, or $(3.30) basic and diluted net loss per share, for the same period in 2023. PepGen had approximately 32.6 million shares outstanding on December 31, 2024.

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