Shares of Dyne Therapeutics DYN rose 10% on Wednesday after the company announced the initiation of the phase III FORZETTO study evaluating the safety and efficacy of zeleciment rostudirsen (z-rostudirsen/DYNE-251) in ambulatory male patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
DMD is a rare, progressive neuromuscular disorder caused by mutations in the DMD gene that lead to little or no production of dystrophin, a protein essential for muscle function. The disease usually begins in early childhood with muscle weakness and gradually worsens over time, impacting mobility and overall physical function.
Year to date, shares of DYN have declined 10.3% compared with the industry’s 8.6% fall.
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Key Details of DYN’s Phase III FORZETTO Study
The phase III FORZETTO study aims to enroll around 90 participants aged four to 18 years, who will receive either z-rostudirsen or a placebo every four weeks. The primary endpoint is the change from baseline in rise from floor (RFF) velocity at week 73, a key measure of muscle strength and motor function in DMD. Secondary endpoints include several measures of motor and respiratory function, along with patient-reported outcomes.
More on DYN’s Z-rostudirsen
Previously, in the registrational expansion cohort of the phase I/II DELIVER study, treatment with z-rostudirsen demonstrated clinically meaningful improvements in RFF velocity versus placebo at six months, with a favorable safety profile. The study met its primary endpoint.
The data are expected to support a planned biologics license application seeking potential accelerated approval in the United States. Z-rostudirsen is being evaluated in the ongoing long-term extension phase of the DELIVER study.
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 currently carries a Zacks Rank #3 (Hold).
Some top-ranked stocks in the biotech sector are Immunocore IMCR, Indivior Pharmaceuticals INDV and Liquidia Corporation LQDA, each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 60 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 14.9% 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%.
Over the past 60 days, estimates for Indivior Pharmaceuticals’ 2026 earnings per share have increased from $3.10 to $3.35. Over the same period, EPS estimates for 2027 have risen to $3.69 from $3.47. INDV shares have risen 4.8% 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 60 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 surged 79.1% 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%.
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Dyne Therapeutics, Inc. (DYN): Free Stock Analysis Report
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This article originally published on Zacks Investment Research (zacks.com).