PepGen Announces Q1 2024 Financial Results and Corporate Highlights

PepGen Inc., a clinical-stage biotechnology company listed on Nasdaq under the ticker PEPG, has reported its financial results and recent corporate achievements for the quarter ending March 31, 2024. The company is dedicated to developing advanced oligonucleotide therapies aimed at treating severe neuromuscular and neurological diseases.

Dr. James McArthur, President and CEO of PepGen, highlighted the company’s remarkable progress in the first quarter by advancing several clinical trials targeting Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1). He emphasized that the company is on course to achieve critical milestones in 2024, such as sharing preliminary data from the CONNECT1-EDO51 and FREEDOM-DM1 clinical trials and launching the FREEDOM2-DM1 Phase 2 clinical trial for patients with DM1.

PepGen’s lead investigational candidate, PGN-EDO51, is designed for the treatment of DMD. It employs the company's proprietary Enhanced Delivery Oligonucleotide (EDO) technology, which aims to skip exon 51 of the dystrophin transcript—a known therapeutic target for approximately 13% of DMD patients. In March 2024, the 5 mg/kg dose cohort for the Phase 2 CONNECT1-EDO51 clinical trial was fully enrolled. Preliminary data from this cohort, including initial safety, exon 51 skipping, and dystrophin production, are expected by mid-2024. CONNECT1-EDO51 is an open-label, multiple ascending dose (MAD) clinical trial involving around 10 male patients aged at least 8 years with DMD, conducted in Canada.

PepGen also received regulatory approval from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate the CONNECT2-EDO51 trial. This will be a Phase 2, randomized, double-blind, placebo-controlled MAD clinical trial, assessing PGN-EDO51 in about 20 male patients aged at least 6 years with DMD. PepGen plans to extend this study to the US and other countries, pending regulatory approvals. Data from CONNECT1 and CONNECT2 may support a future accelerated approval pathway, subject to regulatory feedback. PGN-EDO51 has been granted Orphan Drug and Rare Pediatric Disease Designations by the U.S. Food and Drug Administration (FDA) as of March 2024.

PepGen also presented nonclinical and clinical data concerning PGN-EDO51 at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in March 2024.

For DM1, PepGen is developing PGN-EDODM1, which also uses their proprietary EDO technology. This investigational therapy is designed to restore the normal splicing function of MBNL1, a crucial RNA splicing protein. DM1, a progressively disabling genetic disorder, affects an estimated 40,000 people in the U.S. and over 74,000 in Europe. Preliminary data from the Phase 1 FREEDOM-DM1 clinical trial, involving at least a 5 mg/kg dose cohort, is anticipated in the second half of 2024. This study evaluates the safety, splicing correction, and functional outcomes in approximately 24 adult DM1 patients across the U.S., Canada, and the UK. PepGen presented data on PGN-EDODM1 at both the 2024 IDMC-14 Meeting and the 2024 MDA Conference.

Another candidate, PGN-EDO53, is being developed for the treatment of DMD by targeting exon 53 of the dystrophin transcript, which is relevant for about 8% of DMD patients. PepGen is advancing this candidate through investigational new drug (IND) and clinical trial application (CTA) enabling nonclinical studies.

Financially, PepGen secured $9.9 million from its at-the-market offering program and $76.4 million from a public offering in February 2024. This influx of funds, totaling $86.3 million, is expected to extend the company's cash runway into 2026. As of March 31, 2024, the company reported cash, cash equivalents, and marketable securities of $175.2 million. Research and development expenses for the quarter were $14.7 million, up from $14.4 million in the same period last year, while general and administrative expenses increased to $5.1 million from $3.7 million. The net loss for the quarter was $18.0 million, compared to $16.3 million in the same period of 2023. PepGen had approximately 32.4 million shares outstanding as of March 31, 2024.

In summary, PepGen is making significant strides in the development of therapies for DMD and DM1, with several key milestones anticipated in the coming year. The company’s financial health has been bolstered by recent fundraising efforts, ensuring continued progress in their clinical and preclinical programs.