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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