Pfizer has teamed up with Cytoo to work on a Duchenne muscular dystrophy (DMD) target discovery platform. The early-stage alliance (PDF) comes a week after Pfizer halted development of domagrozumab in DMD because of lackluster midphase data.
Domagrozumab’s inability to move the needle in phase 2 added to the troubled history of DMD drug development. While DMD drugs have made it to market on both sides of the Atlantic, no candidate has generated overwhelming evidence of efficacy and others have failed altogether. Pfizer thinks a lack of basic information about the biology of DMD may be contributing to the challenge of treating the disease.
“Although the genetic cause of DMD has been known for years, little is known about the molecular functions that are affected in DMD muscles. Pfizer is committed to early stage DMD research and target identification,” John Murphy, VP of biology at Pfizer’s rare disease research unit, said in a statement.
Pfizer has responded to the need for insights into DMD by teaming up with Cytoo, a French drug discovery shop that has a myotube platform. The platform, MyoScreen, is an in vitro system that uses skeletal muscle cells to mimic the characteristics and metabolic functions of human muscle in vivo. Researchers can use the platform to analyze molecular disease mechanisms.
Working with Pfizer, Cytoo will adapt the platform to DMD using cells derived from people with the condition. In doing so, the partners think they may create a platform that can be used as a high-throughput target identification screen. If that happens, Pfizer has an option to license the platform to support its efforts to identify DMD targets.
Pfizer’s interest in the technology is a boost for Cytoo. The French company developed MyoScreen in 2015 and landed a partnership focused on the platform with Daiichi Sankyo later that year.