The company also has filed a similar application with the European Medicines Agency (EMA). A decision is is expected within weeks.
Sarconeos, now renamed Myoda, works by activating the MAS receptor and, in an animal model of DMD, the candidate therapy led to significant improvements in exercise tolerance and muscle strength. It also reduced muscle fibrosis. Overall, the therapy has the potential to halt the disease’s progression and delay the loss of mobility.
The clinical program of Sarconeos includes two studies — a Phase 1/2 study (MYODA-PK) expected to start in 2018, and a Phase 2/3 (MYODA-INT) scheduled for 2019.
The MYODA-PK study, performed in centers in Europe, will recruit children with Duchenne to test Myoda’s safety and the therapy’s pharmacokinetics (the movement of the drug into, through, and out of the body) and pharmacodynamics (the effect of a drug within the body).
The trial aims to recruit 24 boys with Duchenne myopathy (muscle disease), aged 2 to 18 years, and will have two phases: a single-ascending dose (SAD) and multiple-ascending dose (MAD). The objective is to assess the optimal dose for further testing in the Phase 2/3 trial, MYODA-INT.
The MYODA-INT study will continue to evaluate Sarconeos’ safety but also its efficacy. The trial aims to enroll about 60 ambulant boys with Duchenne myopathy. Participants will be randomized to a placebo (control) or Myoda for at least six months.
The primary objective will be to measure participants’ functional motor capacities, according to the North Star Ambulatory Assessment (NSAA).
This trial will be performed in centers in Europe and the United States and the results will precede Biophytis’ application for the therapy’s approval.
“The orphan drug designation that has just been granted by the FDA is a significant milestone for our team and the development of Sarconeos [Myoda] in Duchenne Myopathy, a particularly severe pediatric indication. We hope to obtain this designation in Europe as well in the coming weeks. We will then be able to conduct the MYODA clinical program. This program will consist of two studies: a pharmacokinetic phase 1/2a MYODA-PK study, which could begin in 2018 and an efficacy phase 2/3 MYODA-INT study, which could start in 2019,” Stanislas Veillet, CEO of Biophytis, said in a press release.
Orphan drug status aims to encourage therapies for rare and serious diseases, through benefits such as seven years of market exclusivity and exemption from FDA application fees.