2022-12-20| Trials & Approvals

Entrada Hit With Clinical Hold on IND Application for Muscular Dystrophy Therapy

by Reed Slater
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Just weeks after snagging a $224 million deal with Vertex for myotonic dystrophy type 1 therapy development, the FDA placed another one of Entrada Therapeutics’ development programs, ENTR-601-44, on clinical hold after the company submitted the therapy for an Investigational New Drug (IND) Application. 

A Hitch in ENTR-601-44’s Clinical Development

Entrada designed ENTR-601-44 to treat Duchenne Muscular Dystrophy (DMD), an aggressive muscle-degenerating disease with limited treatment options. The therapy uses Entrada’s proprietary Endosomal Escape Vehicle (EEV™)-Oligonucleotides platform to skip a missing or mutated exon 44. According to the company, the drug could treat up to 7.5% of individuals with DMD who are exon 44 skipping amenable. 

Despite the drug’s promise, the FDA placed the program on clinical hold after Entrada submitted the drug’s IND Application. Though the press release is scant on details, the company said the FDA would send an official clinical hold letter within 30 days. Entrada said it would provide updates when it receives the official letter. 

Disappointed, Entrada’s President and CEO, Dipal Doshi, said, “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.”

Even with the company’s positive outlook on resolving the issue, the clinical hold scared many Entrada shareholders off, with the company’s stock dropping 30% after the announcement. 

Related Article: Sarepta Submits BLA for First-Ever Duchenne Muscular Dystrophy Gene Therapy

DMD Therapies Throughout the Industry

Several companies throughout biotech are trying to tackle DMD with varying degrees of success. Entrada is not the only one struggling, though, with safety and efficacy concerns surrounding the space. 

In April, Astellas cut three preclinical DMD gene therapies based on preclinical study data. Astellas made that cut, along with many more that the company said resulted in over $390 million in losses. 

On a brighter note, in October, Sarepta submitted a Biologics License Application (BLA) for its DMD gene therapy, SRP-9001. If approved, the drug would mark the first-ever gene therapy for DMD. Sarepta is aiming for accelerated approval from the FDA and has already enrolled participants in a confirmatory trial for the therapy. 

Even with emerging technologies, DMD is proving a difficult disease to treat, but that is not stopping several companies from trying. The clinical hold on Entrada’s ENTR-601-44 is hopefully just a bump in the road for the promising therapy. Once the FDA issues an official letter, the company can build a blueprint for resolving the issues and moving forward in the therapy’s development. 

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