Roche terminates Duchenne muscular dystrophy candidate due to poor clinical outlook
pharmafile | November 8, 2019 | News story | Medical Communications, Research and Development | Duchenne Muscular Dystrophy, Roche, pharma
Roche has revealed its decision to terminate further development of its anti-myostatin adnectin protein agent RG6206 after it became apparent that the candidate would not come to show clinical benefit in the treatment of Duchenne muscular dystrophy (DMD).
The firm shut down its Phase 2/3 trial investigating the drug following a futility analysis. The study was evaluating the drug in 166 ambulatory male patients between the ages of six and 11, measuring benefit according to the North Star Ambulatory Assessment (NSAA) total score.
The termination of the study also means that a Phase 1/2 open-label extension trial investigating the drug will also be shut down, the company confirmed.
“Our decision to close this programme was made based on the results of a pre-planned interim data analysis of the Phase 2/3 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),” the company said in a letter to the Duchenne community.
“We recognise this news is deeply disappointing for the Duchenne community, especially in view of the historical challenges in DMD drug development and the ongoing need for new treatment options to treat this devastating disease,” it continued. “While the science and large body of research gave us hope that RG6206 would have offered people living with DMD and their families a safe and effective treatment option, the results of the SPITFIRE study at this time lead us to the difficult conclusion that this approach will not be successful.”
Roche also confirmed that the full details from the study would be presented at an upcoming medical conference.
Matt Fellows
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