Roche discontinues development of Duchenne drug RG6206

Swiss pharma giant Roche (ROG: SIX) has discontinued a Phase II/III trial of RG6206, an anti-myostatin adnectin, after a futility analysis showed that the drug was “highly unlikely” to demonstrate clinical benefit in patients with Duchenne muscular dystrophy (DMD).

In a letter to the Parent Project Muscular Dystrophy organization, Roche explained: “We are disappointed to announce that the clinical development program studying RG6206 (RO7239361) in Duchenne will be discontinued. This concerns both RG6206 studies: the open-label extension study of the Phase Ib/II THUNDERJET study, evaluating the safety and tolerability of RG6206 in ambulatory boys with DMD; and the Phase II/III SPITFIRE study, evaluating the efficacy, safety and tolerability of RG6206 in ambulatory boys with DMD. Our decision to close this program was made based on the results of a pre-planned interim data analysis of the Phase II/III SPITFIRE study, which indicated that RG6206 was highly unlikely to demonstrate clinical benefit as defined by meeting the primary endpoint (change from baseline in the North Star Ambulatory Assessment (NSAA) total score versus placebo). As you know, NSAA is a 17-item rating scale that is used to measure functional motor abilities in ambulant children with DMD. It was used to assess the treatment effects with RG6206 in the Phase II/III SPITFIRE study. No safety signals were observed in this analysis and the overall safety profile was similar to that seen in previous trials.”

Another myostatin inhibitor failure