FDA puts PepGen’s oligonucleotide for myotonic dystrophy on clinical hold

The FDA imposed a clinical hold on PepGen’s IND for a drug for myotonic dystrophy type 1 (DM1), slowing down the company’s plans to bring a second neuromuscular program into the clinic.

PepGen did not specify what’s causing concern, saying only that the FDA will provide an official letter within 30 days stating the reasons behind the hold.

Spun out of Oxford University and backed by RA Capital, PepGen specializes in oligonucleotides — short strands of synthetic RNA or DNA molecules — that it delivers with special cell-penetrating peptides. By conjugating the peptides with oligonucleotides, the biotech promises to improve the uptake and activity of the resulting therapy.

Delivery is one of the challenges, if not the key issue, with oligonucleotide therapies, and multiple other biotechs have proposed their own solutions, including Vertex-partnered Entrada, Avidity (whose program has previously gone under clinical hold), Dyne and ARTHEx.

James McArthur

PepGen CEO James McArthur said the company will also continue to prepare for clinical testing outside the US while it works with the FDA to lift the hold.

Shares of PepGen $PGEN, which went public a year ago, fell almost 10% to $14 in pre-market trading.

The compound, PGN-EDODM1, is one of two lead programs at PepGen. The other, PGN-EDO51 for Duchenne muscular dystrophy, has been through a Phase I trial involving healthy volunteers and is slated to enter Phase II in Canada.

PGN-EDODM1 is engineered to correct the mis-splicing that is believed to lead to mutations in the DMPK gene, thereby targeting the underlying genetic cause of DM1.

Based on previous disclosure, PepGen had planned the Phase I trial as a placebo-controlled single-ascending dose study that will yield data on functional assessments, correction of mis-splicing and safety.

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