Date: 2016-09-29
Type of information: R&D agreement
Compound: exon skipping treatment and oral NF-kB inhibition treatment, edasalonexent (CAT-1004)
Company: Sarepta Therapeutics (USA - MA) Catabasis Pharmaceuticals (USA - MA)
Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases
Type agreement: R&D
Action mechanism: Edasalonexent (CAT-1004) is an oral small molecule that has the potential to be a disease-modifying therapy for all patients affected by Duchenne muscular dystrophy (DMD or Duchenne), regardless of the underlying mutation. Edasalonexent inhibits NF-kB, a protein that is activated in Duchenne and drives inflammation and fibrosis, muscle degeneration and suppresses muscle regeneration. In animal models of DMD, edasalonexent inhibited NF-kB, reduced muscle degeneration and improved muscle regeneration and function, and beneficial effects were observed in skeletal, diaphragm and cardiac muscle. The FDA has granted orphan drug, fast track and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD.
Disease: Duchenne muscular dystrophy
Details:
Financial terms:
Latest news: * On September 29, 2016, Catabasis Pharmaceuticals and Sarepta Therapeutics announced a joint research collaboration to explore a combination drug treatment approach for Duchenne muscular dystrophy (DMD). 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.
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.
