CAT-1004, a novel anti-inflammatory agent for treatment of Duchenne Muscular Dystrophy

Joanne M. Donovan
Catabasis Pharmaceuticals, Inc.

28th October 2012

Catabasis Pharmaceuticals submitted a proposal to use CAT-1004 for a proof-of-concept phase 1/2 study of safety, pharmacokinetic and biomarkers of inflammation in steroid naïve Duchenne muscular dystrophy (DMD) patients.  The applicants propose the drug as useful in all DMD patients for an effective and safer control of inflammation versus glucocorticoids. The compound showed a good profile of safety and acceptable tolerability in adult healthy volunteers and type-2 diabetes patients, and demonstrated proof of concept in targeting NF-kB. TACT recommended that in addition to the current data that demonstrates CAT-1004 inhibits inflammation and inflammatory markers in murine and acutely in a canine model of DMD, Catabasis would complete the ongoing data set aboutefficacy and mechanism of action of CAT-1004 in both animalmodels before going to clinical trial. Also consistent with the presented plans, TACT underlined the importance to obtain theadditional safety / toxicology data in juvenile animals as well as PD /PK relationship to better appreciate the drug tissue concentration and / or salicylate plasma levels, in perspective of registration of this salicylate-containing drug for paediatric use. TACT considered the proposed clinical study of value for assessment of safety in the paediatric DMD population and an appropriate basis for planning a longer phase 2 efficacy study.However, TACT suggested reconsidering, with clinicians, the need for a number of secondary endpoints, balancing potentiallylimited short-term information against increasing the complexity of the study.

12 Apr 2017