#United States #San Diego #RNA Therapeutics #Avidity Biosciences #muscle diseases

Avidity Biosciences Sets the Stage for Major Drug Approvals in 2025

Avidity Biosciences, a pioneering biopharmaceutical firm based in San Diego, California, is preparing for a landmark year in 2025 as it aims to submit its first Biologics License Application (BLA) for delpacibart zotadirsen (del-zota). This submission is targeted for individuals suffering from Duchenne muscular dystrophy (DMD) mutations suitable for exon 44 skipping (DMD44).

The company has confirmed an accelerated regulatory pathway for del-zota, deepening its commitment to serving patients affected by rare muscle diseases. A BLA submission at the end of 2025 is part of Avidity's broader strategy which includes preparing for multiple product launches in related conditions such as myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy (FSHD). These efforts are a significant leap towards fulfilling the unmet medical needs of these communities, which are often left with limited treatment options.

Expanding Leadership to Drive Growth

To bolster its capacity for managing the upcoming expansion, Avidity has restructured its leadership team. Eric Mosbrooker has been promoted to Chief Commercial Officer (CCO), overseeing global product launches, while Charles Calderaro III steps in as Chief Technical Officer (CTO) to streamline technical development and operations. Kat Lange has also been appointed Chief Business Officer (CBO) to lead the investor relations and business development initiatives.

In a statement, Sarah Boyce, Avidity's President and CEO, expressed enthusiasm about the imminent transition into a commercially driven organization. With the accelerated approval path for del-zota confirmed, the focus is now on executing a rapid timeline to ensure that patients can access these therapies as soon as possible.

Future Clinical and Regulatory Highlights

Avidity's roadmap for 2025 includes several key clinical and regulatory milestones. For the del-zota program, the company is preparing for a presentation of the topline data from the ongoing EXPLORE44™ trial in Q1, alongside critical analyses from the EXPLORE44-OLE™ trial by Q4. The company anticipates that the data generated could robustly support the BLA submission.

For delpacibart etedesiran (del-desiran), aimed at DM1, further data from the Phase 1/2 MARINA® trial will be showcased in Q1. Meanwhile, enrollment for the Phase 3 HARBOR™ trial is set to conclude by mid-2025. This drug could see market application submissions starting in 2026, primarily in the U.S. and EU markets. In the case of delpacibart braxlosiran (del-brax) targeting FSHD, Avidity has received regulatory alignment on the global Phase 3 trial design, with critical data set to be released in Q2 2025.

Connecting with the Community

As Avidity progresses through these crucial stages, the company is also committed to establishing robust medical affairs and commercial teams dedicated to addressing the needs of the rare disease communities it serves. This approach will not only support current product launches but will also continue to broaden Avidity's pipeline of Antibody Oligonucleotide Conjugates (AOCs™) targeting neuromuscular diseases and precision cardiology.

As Avidity prepares for a presentation at the upcoming 43rd Annual J.P. Morgan Healthcare Conference set for January 14, 2025, the biopharmaceutical landscape is keenly watching how these developments will unfold.

The promise of new therapies through Avidity Biosciences could signify a shift in treatment paradigms for patients grappling with devastating conditions caused by rare muscular diseases. With its innovative approach to RNA therapeutics, Avidity aims to make a substantial impact in the biotech industry, reshaping the narrative for patients who are in desperate need of viable treatment options.