PepGen continues trials for DMD treatment, addresses safety concerns

BOSTON - PepGen Inc. (NASDAQ: PEPG), a biotechnology firm focused on oligonucleotide therapies for severe neuromuscular and neurological diseases, is progressing with its CONNECT clinical program for PGN-EDO51, a potential treatment for Duchenne muscular dystrophy (DMD). Trading near its 52-week low at $1.84 and with a market capitalization of $64.21 million, InvestingPro analysis suggests the stock is currently undervalued. The company has recently provided updates on the ongoing trials and regulatory interactions.

The CONNECT1-EDO51 Phase 2 trial in Canada has completed enrollment for the 10 mg/kg cohort, with participants continuing in a long-term study extension. PepGen anticipates sharing clinical data from this group by the end of 2025. Two participants in this cohort experienced asymptomatic hypomagnesemia, which resolved with oral magnesium supplementation. One of these patients had a dosing pause due to a decrease in kidney function, which is now improving.

Health Canada has allowed the trial to continue at current dosing levels but has requested additional information to address safety concerns before proceeding with higher doses or enrolling more participants. PepGen is collaborating with the agency to respond to its inquiries.

The CONNECT2-EDO51 Phase 2 trial, a multinational study, is underway in the United Kingdom (TADAWUL:4280). The U.S. FDA has placed a clinical hold on the trial’s initiation in the United States, requesting further supportive data for the dosing levels planned. The company is working to address the FDA’s questions, following authorization from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA).

Dr. Paul Streck, Head of R&D at PepGen, stated that all treatment-related adverse events in CONNECT1 have been mild and the safety profile of PGN-EDO51 appears favorable. The company remains vigilant regarding safety and looks forward to sharing data from its ongoing studies.

PGN-EDO51, designed to skip exon 51 in the dystrophin transcript, could potentially restore the production of a functional dystrophin protein for approximately 13% of DMD patients. The FDA has granted Orphan Drug and Rare Pediatric Disease Designations for this purpose. According to InvestingPro data, the company maintains a strong liquidity position with a current ratio of 8.47, though it’s currently experiencing rapid cash burn - one of 14 key insights available to Pro subscribers.

PepGen’s EDO technology is also being used to develop PGN-EDODM1 for myotonic dystrophy type 1 (DM1), with Orphan Drug and Fast Track Designations from the FDA. While the company holds more cash than debt on its balance sheet, InvestingPro analysis indicates volatile stock performance, with shares down nearly 89% over the past six months. Unlock comprehensive financial analysis and 12 additional expert insights with an InvestingPro subscription.

This information is based on a press release statement from PepGen Inc.

In other recent news, biotechnology company PepGen Inc. experienced a downgrade from BofA Securities, moving its rating from Neutral to Underperform. This follows a clinical hold placed by the U.S. Food and Drug Administration (FDA) on PepGen’s Investigational New Drug application for a Duchenne muscular dystrophy (DMD) treatment trial, known as CONNECT2-EDO51. Despite this, PepGen’s ongoing CONNECT1-EDO51 trial remains unaffected.

PepGen’s management is expected to receive an official letter regarding the FDA’s clinical hold within the next 30 days. In response to these developments, BofA Securities reassessed the value of PepGen’s EDO platform, lowering the pipeline plug value to $350 million from the previous $500 million, and adjusting the company’s operating expenses due to the anticipated delay in trial initiation.

On the corporate governance front, PepGen announced the retirement of Board Director Christopher Ashton, effective September 2024, and the appointment of Dr. Michelle Mellion as Chief Medical (TASE:PMCN) Officer and Dr. Hayley Parker as Senior Vice President, Global Regulatory Affairs. These are among the recent developments at PepGen.

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