PepGen receives U.S. patent for myotonic dystrophy drug candidate

BOSTON - PepGen Inc. (NASDAQ:PEPG) has secured a new composition of matter patent from the United States Patent and Trademark Office for its myotonic dystrophy type 1 (DM1) drug candidate, PGN-EDODM1, the clinical-stage biotechnology company announced in a press release. The company, currently valued at approximately $320 million, has seen its shares surge over 200% in the past six months, according to InvestingPro data.

The patent (No. 12,465,646) covers PepGen's novel PGN-EDODM1 molecule, which utilizes the company's proprietary Enhanced Delivery Oligonucleotide (EDO) platform. According to the company, the patent is expected to provide exclusivity in the United States until the second half of 2042, with the possibility of extension following potential FDA approval. This development comes as PepGen maintains a strong financial position, with InvestingPro analysis showing the company holds more cash than debt on its balance sheet.

PGN-EDODM1 is designed to treat myotonic dystrophy type 1, a severe neuromuscular disease that currently has no approved therapies targeting its underlying cause. The drug candidate aims to restore normal splicing function of MBNL1, a key RNA splicing protein, by binding to pathogenic repeat expansions in DMPK transcripts.

"This patent protection underscores the novelty and differentiation of our peptide-conjugated oligonucleotide candidate," said James McArthur, President and CEO of PepGen, in the statement.

The FDA has previously granted PGN-EDODM1 both Orphan Drug and Fast Track Designations for the treatment of patients with DM1.

PepGen's EDO platform leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics. The company is developing a pipeline of oligonucleotide therapeutic candidates targeting severe neuromuscular and neurological diseases.

The newly issued patent complements PepGen's expanding global patent portfolio, which includes multiple issued and pending patents, according to the company's statement. While the company's stock has shown strong momentum with a 23% gain year-to-date, investors should be aware that PepGen exhibits high price volatility and is not expected to be profitable this year. InvestingPro offers additional insights with 11 more tips on PepGen's financial health and investment potential.

In other recent news, PepGen Inc. has made significant strides in its financial and clinical endeavors. The company successfully raised $115 million through a public offering, selling 35,937,500 shares of common stock at $3.20 per share, with Leerink Partners and Stifel as joint bookrunning managers. This funding is intended to support ongoing clinical trials. Additionally, PepGen reported notable results from its FREEDOM-DM1 Phase 1 study, achieving a 53.7% mean splicing correction in patients with myotonic dystrophy type 1, marking the highest level of splicing correction disclosed for DM1 patients to date.

In light of these developments, H.C. Wainwright raised its price target for PepGen to $18 from $12, maintaining a Buy rating. This adjustment follows a previous increase from $8 to $12, which was also based on the promising results from the FREEDOM-DM1 study. The firm noted that the recent acquisition of Avidity by Novartis has positively impacted sentiment in the neuromuscular oligonucleotide sector. PepGen's ability to secure substantial funding and demonstrate clinical progress highlights its ongoing efforts in the biotechnology field.

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