Just one week after Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) drug became the first to gain US Food and Drug Administration (FDA) approval, the drug developer has teamed up with biopharmaceutical company Catabasis Pharmaceuticals. The two companies have announced a joint research project aimed at testing a combination therapy for the rare disease.
Sarepta’s exon skipping therapy and Catabasis’ oral NF-kB inhibitor will be combined as a possible DMD treatment. Jill Milne, CEO of Catabasis told BioPharma Dive that the project is expected to commence immediately, with development updates set to be released in just one or two quarters.
“We recognize the extreme unmet medical need in DMD and are committed to determining the best treatment strategies for patients affected by Duchenne,” said Dr. Edward Kaye, CEO of Sarepta. “We believe exon skipping has the potential to target the underlying genetic cause of the disease by restoring the mRNA reading frame to produce dystrophin in skeletal muscle. We are pleased to initiate activities with Catabasis to evaluate a potential combination treatment approach of exon-skipping and NF-kB inhibition in DMD.”
While FDA approval of Exondys 51 (eteplirsen) was a major win for Sarepta, concerns about the drug’s efficacy still remain. The regulatory approval was conditional on the biopharmaceutical company’s promise to conduct further clinical trials to prove the DMD drug’s benefit to patients.
All of the drugs in Sarepta’s pipeline are so-called exon-skipping drugs similar to Exondys 51. The therapeutics encourage gene expression machinery to skip those exons containing a DMD-causing mutation, so that more of the muscle-stabilizing protein dystrophin can be synthesized.
Catabasis’ NF-kB inhibitor, edasalonexent (CAT-1004), is currently being assessed in a Phase II clinical trial as a monotherapy. The company expects the drug to move through to Phase III sometime next year.
“We are excited to work with Sarepta on this joint research collaboration, which to our knowledge is the first time two companies are testing a combination of investigational therapies to treat Duchenne,” said Milne. “Although we believe edasalonexent (CAT-1004) has the potential to be a disease-modifying monotherapy, we think there is benefit to exploring innovative ways to make the most meaningful difference in this devastating disease. In addition to our continued development of edasalonexent, we are pleased to take the first step via this collaboration to determine if edasalonexent may be complementary to an exon-skipping treatment strategy in the treatment of DMD using a preclinical model.”