Dyne Therapeutics DYN announced the submission of a biologics license application (BLA) to the FDA seeking accelerated approval for zeleciment rostudirsen (z-rostudirsen/DYNE-251). The investigational therapy has been developed for ambulatory patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. Following the announcement, DYN’s shares rose 4.5% on Tuesday.
The proposed treatment regimen involves a 20 mg/kg intravenous dose administered once every four weeks.
The company has requested a Priority Review from the regulatory authority which could reduce the review timeline to six months compared with 10 months under standard review, following the FDA’s 60-day filing review period. If approved on schedule, the company expects to launch z-rostudirsen in the United States in the first quarter of 2027.
DMD is a rare, progressive neuromuscular disorder caused by mutations in the DMD gene, which result in little or no dystrophin production, a protein essential for muscle function. The disease usually begins in early childhood with muscle weakness and gradually worsens, impacting mobility and overall physical function.
Year to date, shares of DYN have declined 7.3% compared with the industry’s 6.4% fall.
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DYN Submits BLA Based on Phase I/II DELIVER Study
The BLA submission is supported by positive data from the registrational expansion cohort of the phase I/II DELIVER study which met its primary endpoint. The study demonstrated that treatment with z-rostudirsen led to a statistically significant increase in dystrophin production and clinically meaningful improvements in rise from floor (RFF) velocity versus placebo at six months, with a favorable safety profile. RFF is a key measure of muscle strength and motor function in DMD. At present, z-rostudirsen is being evaluated in the ongoing long-term extension phase of the DELIVER study.
Dyne Therapeutics initiated a global phase III FORZETTO study evaluating z-rostudirsen in DMD patients earlier this month. The primary endpoint is the change from baseline in RFF velocity. Secondary endpoints include several measures of motor and respiratory function, along with patient-reported outcomes.
Per management, Dyne has reached an agreement with the FDA on the design and protocol of the FORZETTO study, which is intended to serve as the confirmatory study supporting potential conversion of accelerated approval to full approval in the United States, as well as future regulatory filings in international markets.
Z-rostudirsen received several regulatory designations, including Breakthrough Therapy, Fast Track, Rare Pediatric Disease and Orphan Drug status from global health authorities.
Besides z-rostudirsen, Dyne is advancing additional preclinical DMD programs such as DYNE-253, DYNE-245, DYNE-244 and DYNE-255 targeting multiple exon-skipping mutations in DMD.
DYN’s Zacks Rank & Stocks to Consider
Dyne Therapeutics currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Indivior Pharmaceuticals INDV and Liquidia Corporation LQDA, each currently sporting a Zacks Rank #1 (Strong Buy) and Immunocore IMCR, which carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 30 days, estimates for Indivior Pharmaceuticals’ 2026 earnings per share have increased from $3.00 to $3.35. Over the same period, EPS estimates for 2027 have risen to $3.33 from $3.47. INDV shares have risen 5.4% year to date.
Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 65.44%.
Over the past 30 days, estimates for Liquidia’s 2026 earnings per share have increased to $2.97 from $1.50. Over the same period, EPS estimates for 2027 have risen to $4.81 from $2.91. LQDA shares have gained 74.3% year to date.
Liquidia’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 54.40%.
Over the past 30 days, estimates for Immunocore’s 2026 loss per share have narrowed from 97 cents to 16 cents. Over the same period, estimates for 2027 have improved from a loss of 39 cents to earnings of 11 cents per share. IMCR shares have lost 15.3% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 46.66%.
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