You could have forgiven Entrada Therapeutics for feeling it was riding high this month after banking a $224 million payment from Vertex for a preclinical myotonic dystrophy candidate. But the company has now been dragged back to Earth thanks to a clinical hold placed by the FDA on its program for another muscle disorder.
Specifically, the regulator placed a clinical hold notice on Entrada’s application to get its Duchenne muscular dystrophy (DMD) candidate ENTR-601-44 into human trials. The company’s release was sparse on details, saying the FDA’s hold letter is expected within 30 days and the biotech would provide an update when more information was available from the agency.
Boston-based Entrada made its name as a new challenger for the DMD market, raising $116 million in a private round and pulling off a $181.5 million IPO last year to fund ENTR-601-44. The therapy in question is based on technology for getting biologics across cell membranes to hit intracellular targets.
“The clinical hold on our ENTR-601-44 program is disappointing and we will work to address the FDA’s concerns regarding the IND,” CEO Dipal Doshi said in the release. “There are no approved Duchenne therapies for people with exon 44 skippable mutations and we are eager to resolve this hold and continue down the treatment development pathway.”
The news appeared enough to unnerve investors, with Entrada’s stock sinking over 30% in premarket trading to $13.50 a share from a Monday closing price of $19.89. The biotech has other programs for DMD in various stages of preclinical development, including a candidate for people with exon 45 skippable mutations.
While there are already a number of players in the DMD arena, it has proved to be a tricky indication with Astellas taking a $170 million hit in April to drop its gene therapies for the condition. Leading the pack is Sarepta; its CEO recently set out the company's DMD ambitions in the wake of filing for accelerated approval with the FDA in the third quarter.