AOC1020 will be the first RNA therapy to be tried in FSHD
Avidity Biosciences announced today that it had received investigational new drug (IND) clearance from the U.S. Food and Drug Administration (FDA) for AOC 1020, its experimental therapy for facioscapulohumeral muscular dystrophy. This means the San Diego-based biopharmaceutical company is allowed to begin testing AOC 1020 in human clinical trials. Avidity said it had also received IND clearance for another program, AOC 1044, for Duchenne muscular dystrophy mutations amenable to exon 44 skipping.
This welcome news was included in a press release that also announced that the FDA has placed a “partial clinical hold” on new participant enrollment in Avidity’s Phase 1/2 MARINA™ clinical trial of AOC 1001 in adults with myotonic dystrophy type 1 (DM1). Close to 40 participants are currently enrolled in the MARINA and MARINA open label extension (MARINA-OLE™) trials. All participants, whether they are on AOC 1001 or placebo, may continue in their current dosing cohort although no additional participants may be enrolled until the partial clinical hold is resolved.
“The partial clinical hold is in response to a serious adverse event reported in a single participant in the 4mg/kg cohort of the MARINA study,” the company’s press release explained. “The safety of participants enrolled in clinical studies is Avidity’s first priority. Avidity is working closely with the FDA and the trial investigator to assess the cause of this event. The company is taking all necessary steps to resolve the partial clinical hold on new participant enrollment as quickly as possible.”
According to the FDA, a “serious adverse event” is any “undesirable experience associated with the use of a medical product in a patient,” which can include death, life-threatening complications, hospitalization, disability or permanent damage. The adverse event may or may not be due to the experimental drug, and when such an event occurs, the drug developer must pause the trial until it can determine the cause. If the adverse event is shown to not pose a risk to other patients, the trial may be allowed to proceed.
Avidity’s experimental treatments are a new type of RNA therapy based on Antibody Oligonucleotide Conjugates (AOCs™). Their approach uses antibodies that recognize and bind to proteins on the surface of muscles in order to delivery therapeutic RNA molecules into the muscle. For their FSHD therapy, Avidity’s AOC 1020 delivers a molecule that prevents the DUX4 gene from producing the toxic DUX4 protein. (See Dr. Jeff Statland’s talk about AOC-based FSHD therapies.)
The company has tested its drugs extensively in animals to minimize safety risks, but any drug, especially one that has never been tried in humans before, can cause side effects. This is why human clinical trials often start with small, homogenous groups of participants before proceeding into larger groups of patients in order to prioritize participant safety.
We will be keeping a close eye on the status of the MARINA study as well as the progress of AOC 1020 into FSHD clinical trials. Sign up for our e-alerts to stay informed.
Read the full Avidity press release here.