Rycal ®

Clinical development of Rycal® (Ryanodine receptor calcium release channel stabilizer) compound: S48168 (also called ARM210) in the ambulant DMD population

Alexia Blesius, Servier/ARMGO

29th April 2017

Servier, in conjunction with ARMGO, is developing S48168 (also known as ARM210), a “Rycal®” that acts via stabilization of the interaction between Calstabins® and Ryanodine Receptors (RyRs), as a potential therapy for Duchenne muscular dystrophy. S48168 is a clinical stage compound currently being evaluated in a phase I healthy adult volunteer study, and initial data suggest an appropriate safety profile. The proposal is for a European-based, clinical program in ambulant boys aged 7-11 with genetically confirmed Duchenne muscular dystrophy.

The Committee welcomed the fact that the mechanism of action is one not currently targeted by other drugs or biologics in DMD and that drug activity would be independent of mutation. It is encouraging that the safety and tolerability profile appears good and that the pre-clinical validation of drug functional efficacy is derived from two independent laboratories.

The TACT report recommends continuing pre-clinical work to assess long-term efficacy and to look at potential cardiac benefits of this compound. The Committee also suggest changes to the study design and dose escalation part of the proposed trial. There are also recommendations on inclusion criteria and the selection of primary and secondary outcome measures.

To request a full copy of the report please contact the applicant Alexia Blesius

17 Oct 2017