PPMD is excited to share that Avidity Biosciences has announced that they have received clearance from the FDA on their Investigational New Drug (IND) application for AOC 1044, an exon skipping therapy targeting those amenable to exon 44 skipping. With this clearance Avidity can move towards initiating their Phase I/II trial investigating AOC 1044 in participants with Duchenne amenable to exon 44 skipping this year.

We look forward to continued updates from Avidity as the program advances into the clinic.

Read the Press Release from Avidity Biosciences:

Avidity Biosciences Announces FDA Partial Clinical Hold on New Participant Enrollment in Phase 1/2 MARINA™ Trial
Participants currently enrolled in MARINA and MARINA-OLE™ trials may continue to be treated with AOC 1001

Avidity received Investigational New Drug (IND) clearance for FSHD and DMD studies from FDA; programs now advancing into the clinic

Company to host investor webcast today at 8:30 a.m. ET / 5:30 a.m. PT

SAN DIEGO, Sept. 27, 2022 /PRNewswire/ — Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the U.S. Food and Drug Administration (FDA) has placed a partial clinical hold on new participant enrollment in the 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. All participants in MARINA may roll over into the MARINA-OLE where they will receive AOC 1001 as planned. DM1 is an underrecognized, progressive and often fatal neuromuscular disease with no approved treatment options.

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. 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.

“The safety of participants enrolled in our clinical studies is our first priority. We are doing a thorough analysis and will work diligently with the FDA and the trial investigator to follow the progress of this participant and to resume new participant enrollment as soon as we can,” said Sarah Boyce, president and chief executive officer at Avidity. “We share the sense of urgency with the DM1 community for effective therapies and we remain confident that AOC 1001 has the potential to address important unmet needs of people living with DM1. We want to thank each participant in the study, their families and the investigators for their continued contributions.”

“We continue to look forward to the preliminary assessment of the MARINA study in Q4 and, with our two recent IND clearances, we are now advancing AOC 1020 for FSHD and AOC 1044 for DMD into the clinic this year as planned,” added Ms. Boyce.

Avidity remains on track to conduct a preliminary assessment of safety, tolerability and key biomarkers in approximately half of the study participants in the MARINA trial in the fourth quarter of 2022.

Avidity received IND clearance from the FDA to proceed with the clinical trial of AOC 1020 for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and AOC 1044 for the treatment of Duchenne muscular dystrophy (DMD) with mutations amenable to exon 44 skipping. These programs are now advancing into the clinic.