Catabasis Pharmaceuticals and the Muscular Dystrophy Association (MDA) will partner to support the Part B portion of the Phase I/II MoveDMD clinical trial of CAT-1004, a drug candidate for patients with Duchenne muscular dystrophy (DMD).
Under the collaboration, MDA agreed to provide funding for transportation to participants who need to travel to take part in Part B, which is expected to launch in the first half of this year.
Participants will be randomized to take CAT-1004 or a placebo for 12 weeks. All participants in the placebo group will be crossed over to receive CAT-1004 after 12 weeks, so that all trial patients would receive CAT-1004 for 12 weeks.
MoveDMD is a two-part clinical trial investigating the safety and efficacy of CAT-1004 in DMD via any confirmed mutation in boys ages four to seven.
Part A of the trial (NCT02439216) has been completed, with Catabasis recently reporting positive top-line results for safety, tolerability, and pharmacokinetics following seven days of treatment with CAT-1004. In addition, the company collected data at baseline on the muscles of the lower and upper legs using MRI, physical function (including timed function tests), and muscle strength.
Boys who participated in Part A will be asked to join Part B, along with additional participants to be enrolled. Catabasis said it is now identifying additional patients who are interested in participating in Part B, whose enrollment criteria are expected to be similar to those for Part A.
CAT-1004 is an oral small-molecule drug that, according to Catabasis, has potential to be a disease-modifying therapy for Duchenne regardless of the underlying dystrophin mutation.
“The DMD community desperately needs and deserves treatment options that will help families by allowing for everyday freedoms that many take for granted such as fastening a button without assistance, moving into a more comfortable position independently, or just being able to hug the people you love,” MDA evp and chief medical and scientific officer Valerie Cwik, M.D., said in a statement.
In Phase I trials, CAT-1004 inhibited activated nuclear factor kappa-light-chain-enhancer of activated B cells (NF-kB), a protein chronically activated in DMD as well as multiple other skeletal muscle disorders. The drug candidate was well tolerated with no observed safety concerns in adults, Catabasis said. CAT-1004 also inhibited NF-kB, reduced muscle degeneration and increased muscle regeneration in animal models of DMD.
The FDA has granted CAT-1004 orphan drug, fast track and rare pediatric disease designations for DMD, while the European Commission has granted CAT-1004 orphan medicinal product designation for DMD.
Catabasis develops therapeutics based on its Safely Metabolized And Rationally Targeted (SMART) linker technology platform, which focuses on simultaneously modulating multiple targets in one or more related disease pathways.