FDA lifts hold on PepGen's myotonic dystrophy drug; Takeda teams with cancer nonprofit
2023-10-12
寡核苷酸临床3期临床2期
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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