FDA lifts hold on PepGen's myotonic dystrophy drug; Takeda teams with cancer nonprofit
Plus, Scholar Rock’s entry into the weight loss space, new funding for Synerkine Pharma and its lead pain program, BioLineRx’s Asia deal, Hansa Biopharma’s Phase III delay, a bispecific deal around dermatology and VitaDAO creating its first biotech.
PepGen can now start the US portion of a Phase I study of its oligonucleotide candidate in myotonic dystrophy type 1 after the FDA lifted a clinical hold. The drug, PGN-EDODM1, is designed to correct the mis-splicing underlying the neuromuscular disease and was put on hold in May. After reviewing its preclinical safety data package, the FDA agreed with a proposed starting dose of 5 mg/kg, moving up to 10 mg/kg and 20 mg/kg, said the Boston biotech. The company kicked off the study in Canada in September and expects proof-of-concept data for the starter dose in mid-2024. — Amber Tong
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