Avidity’s DMD therapy shows functional improvements after one year

SAN DIEGO - Avidity Biosciences, Inc. (NASDAQ:RNA), a $6 billion market cap biotech company whose stock has surged over 55% in the past six months, reported Wednesday that its experimental therapy for Duchenne muscular dystrophy (DMD) demonstrated improvements in multiple functional measures after approximately one year of treatment. According to InvestingPro data, the company maintains a strong liquidity position with current assets significantly exceeding short-term obligations.

The company’s drug candidate, del-zota, is designed to help patients with DMD mutations amenable to exon 44 skipping. Data from the EXPLORE44 and EXPLORE44-OLE trials showed improvements in Time to Rise from Floor, 4-Stair Climb, Performance of Upper Limb, and 10-Meter Walk/Run Test compared to baseline and natural history.

According to the company, participants treated with del-zota showed statistically significant increases of approximately 25 percent of normal in dystrophin production. Creatine kinase levels, a marker of muscle damage, rapidly reduced by more than 80 percent compared to baseline and remained at near normal levels throughout the evaluation period of up to 16 months.

The trials included 17 participants who received del-zota for approximately one year. In contrast to natural history data showing decline, del-zota-treated patients improved in several measures, including a 2.1-second improvement in 4-Stair Climb and a 3.2-second improvement in Time to Rise from Floor.

The company reported that del-zota demonstrated a favorable long-term safety profile, with most treatment-emergent adverse events being mild or moderate. Common side effects included upper respiratory tract symptoms, diarrhea, fall, back pain, and headache.

"For the first time, we have data showing that sustained muscle protection leads to meaningful improvements across multiple key functional measures in DMD," said Sarah Boyce, President and Chief Executive Officer of Avidity, in the press release.

Avidity stated it remains on track to submit a Biologics License Application to the FDA for accelerated approval by the end of 2025.

DMD is a rare genetic condition characterized by progressive muscle damage due to the absence of dystrophin protein, affecting approximately one in 3,500 to 5,000 boys worldwide. With the stock trading near its 52-week high of $56, investors seeking deeper insights into Avidity’s valuation and growth prospects can access detailed financial analysis and Fair Value estimates through InvestingPro’s comprehensive research tools.

In other recent news, Avidity Biosciences has made significant strides in its clinical and regulatory efforts. The company announced the completion of enrollment for its global Phase 3 HARBOR clinical trial of delpacibart etedesiran for myotonic dystrophy type 1, with topline data expected in the second quarter of 2026. In addition, Avidity plans to submit marketing applications for this treatment in the United States, European Union, and Japan starting in the latter half of 2026. Furthermore, the U.S. Food and Drug Administration granted Breakthrough Therapy designation to Avidity’s delpacibart zotadirsen for treating Duchenne muscular dystrophy, aimed at expediting its development and review. This drug is currently being evaluated in the Phase 2 EXPLORE44 Open-Label Extension trial. Analyst firms have also shown confidence in Avidity’s prospects, with Goldman Sachs resuming coverage with a Buy rating and Cantor Fitzgerald maintaining an overweight rating. Both firms have expressed positive views on Avidity’s platform technology and leadership. These developments mark important progress for Avidity Biosciences in its pursuit of innovative treatments for rare diseases.

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