Catabasis Pursues Bifunctional Mutation-Agnostic Approach To DMD
Executive Summary
Using its safely metabolized and rationally targeted (SMART) linker platform, Catabasis has identified a NF-κB inhibitor – edasalonexent – as a potential Duchenne muscular dystrophy (DMD) treatment. Speaking at the 2017 Biotech Showcase, Dr Jill Milne, co-founder, president and CEO of Catabasis, describes the company’s ambitions to pursue a pivotal Phase III trial this year.
Jill Milne, co-founder and CEO of Catabasis Pharmaceuticals Inc., talks in this video interview to Scrip's Mike Ward about the company, which was founded around a proprietary chemistry platform technology. It is developing its lead product edasalonexent (CAT-1004) to target all patients with the condition regardless of their genetic mutations by targeting a pathway that is chronically activated in all patients, whereas Sarepta's approved product Exondys 51 (eteplirsen) targets only the 13% of patients who have an exon 51 mutation.
The CEO lauds the involvement of patient advocacy groups in helping shape regulatory guidance for Duchenne muscular dystrophy but says that the approval of Exondys 51 cannot be directly related to Catabasis' oral candidate and its own approval prospects.
Catabasis aims to engineer bifunctional product candidates composed of two bioactives held together by a proprietary linker to target disease pathways at more than one point, simultaneously.
The company's lead asset at the time of the interview was in Phase II; it subsequently announced that one portion of the trial had failed to meet its composite primary endpoint of the drug's effects assessed via magnetic resonance imaging T2 measures of leg muscles after 12 weeks, but said that an open label extension portion would continue, with an interim updated scheduled for Q2 2017. It declared that the biomarker, MRI T2, didn't work in boys with Duchenne's, and said it would stick with the four-step climb test or the 10-meter walk/run test in future. (Also see "Catabasis Refutes MRI T2 Biomarker In Duchenne Study" - Pink Sheet, 2 Feb, 2017.)
The company has additional preclinical programs in cystic fibrosis and rare neurodegenerative diseases such as amyotrophic lateral sclerosis and Friedreich's ataxia.
Scrip & EBD Group
This interview was recorded at EBD Group's Biotech Showcase in San Francisco in January 2017.
From the editors of Start-Up