RNA-focused biopharma Avidity Biosciences announced this week that the FDA has placed a clinical hold on participant enrollment in its phase 1/2 trial which intends to assess the efficacy of the company’s mAb drug candidate for patients with a type of muscular dystrophy.
The trial, called MARINA, has close to 40 participants enrolled, all adults with myotonic dystrophy type 1 (DM1). The genetic, multi-system disorder attacks the skeletal muscles as well as smooth muscles. In families with DM1 history, the repeat expansion can enlarge with each generation according to John Hopkins Medicine, frequently causing earlier onset and increased severity.
The drug candidate — AOC 1001 — works by helping to lower the levels of a disease-related mRNA called DMPK. The drug is a monoclonal antibody that successfully delivered siRNA to muscle cells affected in preclinical trials, and resulted in dose-dependent, durable reductions of the disease-related mRNA.
While participants that are already enrolled may continue being dosed, Avidity won’t bring new patients until the hold is resolved which was placed in response to a serious adverse event reported. In the recent press release, Avidity did not elaborate on the details of the adverse event but confirmed that the participant was a part of the cohort that received medication and not placebo.
The EMA has granted AOC 1001 Orphan Designation, as did the FDA, adding Fast Track Designation.
