Avidity's DMD Therapy Shows Promise, Paving Way for FDA Submission

Avidity Biosciences has reported promising results from two mid-stage trials of its investigational exon-44-skipping therapy for Duchenne muscular dystrophy (DMD), potentially clearing the path for a biologics license application (BLA) to the FDA. The therapy, delpacibart zotadirsen (del-zota), demonstrated significant functional improvements in patients after one year of treatment, reversing disease progression in key metrics.

Clinical Trial Results Demonstrate Efficacy

The Phase I/II EXPLORE44 and EXPLORE44-OLE studies enrolled a total of 26 patients with DMD amenable to exon 44 skipping, with 16 participants continuing to the open-label extension study. Key findings from the trials include:

• A 25% increase in normal dystrophin production, with dystrophin concentrations restored to up to 58% of normal levels
• An over 80% reduction in creatine kinase (CK) levels, a marker of muscle damage, maintained at near-normal levels through 16 months of follow-up
• Functional benefits correlated with biomarker improvements, including a 2.1-second improvement in the four-stair climb test, compared to a 2.7-second decline in natural history controls
• Improved outcomes in the 10-meter walk/run, time to rise from floor, and North Star Ambulatory Assessment tests

Regulatory and Commercial Outlook

Avidity aims to complete a BLA submission by the end of 2025, seeking accelerated approval for del-zota. The company is also preparing a confirmatory trial to support full global approval. Analysts at BMO Capital Markets expect a "smooth" approval process, citing the lack of other approved exon-44 skipping treatments.

If approved, del-zota could transform Avidity into a commercial-stage company by 2026. The addressable U.S. patient population is estimated at around 1,000 individuals.

Industry Implications and Potential Acquisition Interest

The positive results from del-zota's trials may heighten Avidity's appeal to potential acquirers. In August, reports surfaced that Novartis had approached Avidity with a buyout offer, although no deal has been finalized. This development comes amid a series of recent acquisitions by Novartis, including the $1.4 billion takeover of Tourmaline Bio and a $5.2 billion commitment to expand its agreement with China's Argo Biopharma.

As Avidity progresses towards a potential FDA submission, the pharmaceutical industry will be watching closely to see how this promising therapy could reshape the landscape of DMD treatment and potentially spark further M&A activity in the sector.