Dyne Therapeutics (NASDAQ:DYN) executives outlined a slate of 2026 operational and regulatory milestones during a discussion with Stifel, emphasizing plans to transition toward a commercial-stage organization while advancing multiple neuromuscular disease programs.
Chief Financial Officer Erick Lucera, joined by Chief Scientific Officer Ranjan Batra, said the company views 2026 as a potential “breakout year,” following what he described as 2025 clinical validation of Dyne’s platform “in humans across a couple different diseases.” Lucera highlighted anticipated catalysts including completion of enrollment in Dyne’s registrational cohort for myotonic dystrophy type 1 (DM1), a planned biologics license application (BLA) filing for Duchenne muscular dystrophy (DMD), and advancing facioscapulohumeral muscular dystrophy (FSHD) into the clinic.
DMD program: DELIVER data and accelerated approval case
According to Lucera, dystrophin at six months was the primary endpoint, and the trial showed statistically significant increases in dystrophin expression, including “sevenfold improvement” on both adjusted muscle-adjusted and unadjusted measures. He said the dystrophin increases were “tenfold higher than the PMO that’s being used as standard of care,” while also noting the company’s dosing schedule is every four weeks.
On functional outcomes, Lucera said Dyne evaluated six functional endpoints spanning time to rise, ambulation, upper limb function, and respiratory measures. While the study was powered for dystrophin, he said two functional measures—time to rise velocity and 10-meter walk/run velocity—reached statistical significance with nominal p-values at or below 0.05. He also pointed to preservation of lung capacity at six months and described 24-month data as showing improvement across all six functional endpoints versus baseline.
Lucera also cited an optional biopsy component, noting that biopsies from four patients at more than 12 months showed adjusted dystrophin greater than 18% and unadjusted dystrophin at 9%. He characterized these findings as supportive of greater dystrophin and functional improvement with longer treatment duration.
On safety, Lucera said the profile remains favorable, referencing more than 1,400 doses administered and up to 36 months of follow-up.
Regulatory posture and safety database
In discussing the U.S. regulatory environment, Lucera said Dyne believes the components of its planned submission align with patterns seen in recent accelerated approvals, emphasizing the presence of a placebo-controlled data set and a larger sample size relative to some past filings. He also highlighted that Dyne has Breakthrough Therapy designation, which he said supports more frequent and higher-level interactions with FDA.
Lucera said the company remains confident that its BLA filing for DMD is on track for the second quarter. Asked about the safety database required, he said Dyne has data from 86 z-rostudirsen patients and described that safety database as “well in excess” of what other submissions have included. He added that the company expects Priority Review but noted it ultimately depends on FDA.
Commercial preparation for a potential DMD launch
Lucera said Dyne has been building out launch capabilities, including hiring Johanna Friedl-Naderer, who he said led the launch of Spinraza at Biogen, and building a commercial team with rare disease experience across reimbursement, marketing, and market research.
He framed DMD as a “well-defined market” with existing reimbursement infrastructure, well-characterized patients, and strong advocacy organizations. Lucera also argued the commercial opportunity could be capital efficient because the patient base is concentrated, stating that approximately 80% of DMD patients are treated in roughly 100 U.S. muscle centers, implying a smaller targeted sales footprint.
On manufacturing readiness, Lucera said Dyne has been building inventory and has dual sourcing for each component of its supply chain. He also referenced technical operations leadership from John Cox, noting his prior experience running tech ops at Biogen.
When asked about pricing and uptake relative to existing therapies, Lucera said it was too early to comment on pricing prior to a label, but asserted Dyne intends to bring a product with functional benefit and a more convenient dosing schedule versus products that are dosed weekly or monthly and “don’t have any functional benefits,” in his words.
Pipeline expansion: additional DMD exons, DM1 confirmatory study, and broader portfolio
Dyne executives said advancing additional DMD exon-skipping candidates is a key part of the company’s longer-term growth plan. Lucera described four additional exon programs in the “IND-enabling” phase and emphasized that Dyne’s approach leverages what he called a shared backbone—same linker, Fab, and oligochemistry—which he argued could reduce development risk versus typical preclinical programs.
Batra added that the additional exon programs are in IND-enabling studies and that Dyne aims to initiate GMP manufacturing and align progression of those programs with the anticipated approval timeline of z-rostudirsen. He said preclinical work has shown higher levels of exon skipping for these other exons compared with exon 51, which he described as the “hardest to skip.” The executives did not provide timelines for additional exon clinical starts.
For DM1, Batra discussed Dyne’s confirmatory Phase III plan, stating the company will use Five Times Sit-to-Stand as the primary endpoint. He said Dyne is using VHOT as an intermediate clinical endpoint in the registrational cohort, but not as the Phase III endpoint, and described Five Times Sit-to-Stand as measuring broader functional change. Batra said the confirmatory trial is designed for 150 patients at a 6.8 mg/kg dose every eight weeks (Q8W) and is powered to deliver statistical significance on that endpoint. He also noted central nervous system-related assessments, including fatigue, excessive daytime sleepiness, and cognition, along with additional secondary endpoints such as myotonia and other timed function tests.
Batra said Dyne has already observed improvement on Five Times Sit-to-Stand at six months that “deepens” at 12 months at the registrational dose, and that endpoint selection also reflected natural history and key opinion leader input. Dyne reiterated expectations to complete DM1 registrational cohort enrollment in the second quarter and report data in the first quarter of 2027.
Asked about other companies’ DM1 data expected later in the year, Batra cautioned against cross-trial comparisons and said Dyne’s mechanism differs, describing an antisense oligonucleotide designed to reach the nucleus and degrade pathogenic RNA foci by recruiting RNase H1. He also said Dyne has not observed increased anemia versus placebo or natural history in its trial, while referencing a paper reporting incidents of anemia in Avidity’s MARINA trial.
Lucera summarized Dyne’s broader portfolio as including a potential first-to-market DMD program followed by DM1, four additional DMD exon products, FSHD as the next program expected to enter the clinic, and a Pompe program—totaling eight products anticipated to be in the clinic over the next couple of years.
About Dyne Therapeutics (NASDAQ:DYN)
Dyne Therapeutics is a clinical-stage biotechnology company specializing in the development of localized gene regulation therapies for serious rare diseases. The company’s proprietary FORCE (Facilitated Orthogonal Receptor‐mediated Cargo Evaluation) platform is designed to enable targeted delivery of oligonucleotide and gene therapy modalities to skeletal and respiratory muscles. Dyne’s lead programs focus on Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy (FSHD), with preclinical and early clinical studies evaluating safety, tolerability and tissue specificity.
Since its founding in 2019 by Flagship Pioneering, Dyne has advanced multiple product candidates using its modular delivery approach, which couples engineered ligands with therapeutic payloads to improve uptake into muscle cells.
