Catabasis, Sarepta partner to explore combination drug for Duchenne muscular dystrophy

The two companies will contribute their respective expertise to study an exon skipping treatment developed by Sarepta, together with an oral NF-kB inhibition treatment developed by Catabasis in a mouse model of DMD.

Jill C. Milne, Ph.D., chief executive officer of Catabasis, said: "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. 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."

Edward Kaye, M.D., Sarepta's chief executive officer, said: "We recognize the extreme unmet medical need in DMD and are committed to determining the best treatment strategies for patients affected by Duchenne.

"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."

NF-kB inhibition and exon-skipping represent two novel investigational treatment strategies in Duchenne, each with the potential for disease-modifying effects when used as monotherapy.

The objective of the joint research is to study the safety and efficacy of combining these two treatment strategies using a mouse model of DMD, including evaluating the potential for additional or synergistic benefits.