- Days after Boston-based Entrada Therapeutics announced a deal with Vertex Pharmaceuticals to develop drugs for a rare muscle disorder, the Food and Drug Administration has paused the smaller company’s plans to start human trials of a treatment for another disease.
- The regulators’ decision doesn’t appear to involve research covered by the Vertex deal, which is focused on a condition known as myotonic dystrophy type 1, or DM1. Instead, the halted trial involves a preclinical drug for Duchenne muscular dystrophy.
- In Duchenne, Entrada is a late entrant in a competitive field, with many other developers, like Sarepta Therapeutics, further along. Still, it raised $182 million in an initial public offering last year to fund its work.
The cash from the Vertex deal is expected to extend the company’s runway into 2025, buying Entrada time to produce data for its lead candidates. Part of its pitch in Duchenne is that its lead drug targets a group of patients ineligible to be treated by Sarepta’s currently approved therapies.
“[Entrada’s] program isn’t as advanced as some competitors, so they’ll want to be able to move rapidly if their human data looks good,” Evercore analysts Jon Miller and Umer Raffat wrote in a Dec. 8 note following the Vertex deal.
Duchenne is a genetic disorder that affects the muscles, leading to progressive weakness and disability. There’s no curative treatment yet available for the condition, which is caused by defects in the gene that produces a key protein. But drugs from Sarepta and others are approved on the basis of their ability to increase forms of that protein, known as dystrophin.
According to Entrada, preclinical data for the program that’s now on hold, dubbed ENTR-601-44, helped improve dystrophin production in mice.
“The clinical hold on our ENTR-601-44 program is disappointing and we will work to address the FDA’s concerns regarding the IND,” Dipal Doshi, Entrada’s CEO, said in a statement. “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.”
While the Vertex deal gave Entrada a boost, the hold is still a blow for a company that hoped to soon get clearance for human testing.
Entrada raised $59 million in a Series A round in 2018, three years prior to its Wall Street debut. The terms of Entrada’s $250 million deal with Vertex included a $26 million equity investment, and as much as $485 million in additional payments contingent on hitting certain milestones.
Shares of Entrada fell 20% Tuesday, to $15.96 apiece.