Catabasis Pharmaceuticals announced it will present data on CAT-1004 at the XIV International Conference on Duchenne and Becker Muscular Dystrophy to be held in Rome, Italy, on Feb. 13-14. Joanne Donovan, chief medical officer of Catabasis, will talk about the drug, now in clinical testing, and the design of the MoveDMD trial.
Catabasis Pharmaceuticals, Inc. is a biopharmaceutical company developing therapies for patients with rare diseases. The company recently announced a partnership with the Muscular Dystrophy Association (MDA) for the second part of the MoveDMD study, which will investigate the efficacy and safety of CAT-1004.
The drug is a small molecule intended for oral use in patients with Duchenne muscular dystrophy (DMD) irrespective of the underlying mutation. The drug blocks NF-κB, a key inflammatory response mediator that is overactive in DMD, driving inflammation and fibrosis to further worsen muscle degeneration and hinder re-generation.
CAT-1004 has been shown to slow degeneration and improve re-generation in animal models of the disease. Improved muscle function in skeletal, diaphragm and cardiac muscles was observed in these preclinical studies.
The FDA has granted CAT-1004 orphan drug status as well as fast track and rare pediatric disease designations. The European Commission also designated it an orphan medicinal product. Such designations help to advance a drug’s development.
“We are currently conducting the MoveDMD trial of CAT-1004 in 4-7 year-old boys affected by Duchenne and have previously reported safety, tolerability and reduction in NF-kB activity in Phase 1 trials,” Catabasis reported in a press release.
MoveDMD is a Phase 1/2 clinical trial of CAT-1004. Part A of the trial evaluated the drug’s safety, tolerability and pharmacokinetics (how the drug behaves in the body). At study start, Catabasis collected data using magnetic resonance imaging (MRI) from muscles of the lower and upper legs, and on physical function and muscle strength. Boys included in Part A of the trial will also be asked to participate in Part B, if eligible, which will evaluate the safety and efficacy of CAT-1004 in DMD over a 12-week treatment period. The protocol of this second part is not finalized.