Catabasis hopes to ride Sarepta approval wave

Dive Brief:

  • Catabasis Pharmaceuticals announced Thursday it is teaming up with Sarepta Therapeutics on a joint research deal to explore a combination therapy for the treatment of Duchenne muscular dystrophy (DMD).
  • The two companies will test a pairing of an exon skipping therapy from the Sarepta pipeline along with an oral NF-kB inhibitor provided by Catabasis.
  • Work is expected to begin immediately and the companies intend to have an update on the progress of development in "one or two quarters," Catabasis CEO Jill Milne told BioPharma Dive in an interview. 

Dive Insight:

Fresh off the approval of the first DMD drug, Sarepta is exploring potential combinations for its other exon-skipping drug candidates. The approval of Exondys 51 (eteplirsen) has been mired by controversy, with looming questions remaining about the level of effectiveness of the drug — Sarepta is required to conduct further studies to gain full approval. Analysts have been criticizing the U.S. Food and Drug Administration's go-ahead for the drug, suggesting the decision was driven by patient advocacy rather than science. 

Similar to Exondys 51, all of the drugs in Sarepta's pipeline are exon-skipping drugs designed to "skip" over a faulty gene, allowing the body to make higher levels of the dystrophin protein which is characteristically missing in DMD patients.

Milne told BioPharma Dive that combining the two drugs makes "mechanistic sense," because the addition of the NF-kB inhibitor could further boost levels of dystrophin.

Catabasis has been exploring the potential of a combination for over a year, Milne said. The biotech's NF-kB inhibitor edasalonexent is currently in Phase 2 trials as a monotherapy and is expected to enter Phase 3 next year.

"There conversations had been ongoing prior to the approval [of Exondys 51]," said Milne. "With Sarepta being in the lead in this space, they were the logical partner."

The pair will split the work on the collaboration and Milne noted that the two companies are located just two blocks apart in Cambridge, MA., allowing their scientists to have easy access to each other. 

When asked about the negative headlines surrounding Sarepta, Milne admitted she'd seen them, but said that Catabasis was undeterred in its goal to provide strong treatment options for the boys with DMD. 

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Filed Under: Clinical Trials Mergers and Acquisitions / Deals