Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today is holding its first Investor Day and will provide an in-depth review of the Company's strategy and pipeline in rare diseases, including edasalonexent (CAT-1004) and other programs. Guest speakers will include Craig McDonald, M.D., UC Davis NeuroNEXT Program Director, University of California, and H. Lee Sweeney, Ph.D., Myology Institute Director, University of Florida. "At Catabasis, we have been executing relentlessly on our strategic plan and mission to bring hope and life-changing therapies to patients and their families suffering from rare diseases," said Jill C. Milne, Chief Executive Officer of Catabasis. "We expect to report top-line safety and efficacy results from the Phase 2 portion of the MoveDMD ® clinical trial in the first half of Q1 2017 after the JP Morgan conference. Assuming positive Phase 2 results, we anticipate initiating two additional clinical trials in DMD next year, including a pivotal Phase 3 trial. In addition to our work in DMD, we are also progressing the Catabasis rare disease pipeline. We look forward to initiating clinical work in an additional rare disease for edasalonexent where NF-kB plays an important role. Further, Catabasis recently expanded its rare disease pipeline by adding CAT-5571, an activator of autophagy, as a potential treatment of cystic fibrosis." Program Updates and Highlights: Edasalonexent (CAT-1004) for the Potential Treatment of DMD
- Top-line safety and efficacy results from the placebo-controlled portion of the MoveDMD Phase 2 trial are expected in the first half of Q1 2017.
- MoveDMD Phase 2 trial design: The primary efficacy end point is change in magnetic resonance imaging (MRI) T2 for the composite of five lower leg muscles for the pooled edasalonexent dose groups compared to placebo. Safety and tolerability will also be evaluated. Additional assessments are being measured, however the trial is not powered for statistical significance for these assessments. The additional assessments include timed function tests (10-meter walk/run, 4-stair climb and time to stand), muscle strength measures, the North Star Ambulatory Assessment (NSAA) and the pediatric outcomes data collection instrument (PODCI).
- The open-label extension for the MoveDMD trial, in which patients will continue on open-label edasalonexent for 36 weeks following completion of the 12-week, placebo-controlled portion of the trial, is ongoing. During the open-label extension, safety will be monitored and assessments including MRI, timed function tests, muscle strength measures, the NSAA and the PODCI, will be performed. Catabasis expects to report periodic results from the MoveDMD open-label extension in 2017.
- Assuming positive results from the Phase 2 MoveDMD clinical trial and supportive discussions with regulatory authorities, Catabasis intends to initiate a Phase 3 placebo-controlled pivotal clinical trial of edasalonexent in ambulatory boys with DMD aged 4 to 7 in H2 2017. The primary end point is expected to be one of the age-appropriate timed function tests included in the Phase 2 trial. The final design of the Phase 3 trial is expected to be informed by the results of the Phase 2 MoveDMD trial as well as the open-label extension data available prior to the initiation of the Phase 3 trial.
- Assuming positive results from the Phase 2 MoveDMD clinical trial, Catabasis also intends to initiate a clinical trial in non-ambulatory patients with DMD in H2 2017.
- There are additional diseases in which inhibiting NF-kB with edasalonexent may be beneficial. Catabasis expects to initiate a Phase 2 trial for an additional rare disease indication for edasalonexent in Q4 2017 or Q1 2018.
- Preclinical research with CAT-4001 has continued in diseases such as amyotrophic lateral sclerosis (ALS) and Friedreich's ataxia. Ongoing preclinical research for CAT-4001 is expected in 2017.
- CAT-5571 is a potential therapy to treat cystic fibrosis (CF). CAT-5571, an activator of autophagy, in combination with lumacaftor/ivacaftor, enhanced cell-surface trafficking and function of cystic fibrosis transmembrane conductance regulator (CFTR) in bronchial epithelial cells from CF patients with the F508del mutation. Catabasis also demonstrated that CAT-5571 enhanced the clearance of Pseudomonas aeruginosa infection in preclinical models of CF, irrespective of CFTR mutation status. Catabasis expects to initiate a Phase 1 trial with CAT-5571 for the potential treatment of CF in Q4 2017 or Q1 2018.