诺华DM1药物del-desiran三期HARBOR未达主要终点，拖累同病种Dyne Therapeutics盘前大跌

Dyne Therapeutics Inc. (DYN) shares are down 29.45% after Novartis reported that its Phase 3 HARBOR study of del-desiran in myotonic dystrophy type 1 (DM1) failed to meet its primary endpoint, weighing on shares of Dyne, which is developing z-basivarsen for the same disease.
Dyne Therapeutics announced that it will present additional one-year clinical data from the Phase 1/2 ACHIEVE trial of z-basivarsen (DYNE-101) for DM1 at upcoming medical meetings.
Additional One-Year ACHIEVE Data
The company will present the new data at the 31st Annual International Congress of the World Muscle Society (WMS), being held September 29 through October 3, 2026, in Hiroshima, Japan and virtually, and at the 2026 Annual Meeting of the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM), taking place September 29 through October 2 in Orlando, Florida and virtually.
Both presentations will include six- and 12-month data from a pooled dose group of 25 to 26 participants initially enrolled in the 3.4 mg/kg every four weeks, 5.4 mg/kg every eight weeks and 6.8 mg/kg every eight weeks cohorts of the multiple ascending dose portion of ACHIEVE, including participants originally assigned to placebo.
The company will also provide 12-month data from 41 to 46 participants in the ongoing END-DM1 natural history study who were propensity-matched to the ACHIEVE pooled-dose group, along with updated safety data from ACHIEVE participants.
Dyne also plans to provide the average baseline value for video hand opening time (vHOT) in the registrational expansion cohort (REC) of ACHIEVE, which includes 71 participants and remains blinded to treatment assignment.
Topline data from the ACHIEVE REC are on track for the first quarter of 2027.
WMS And AANEM Presentations
At WMS, the new ACHIEVE data will be presented in an oral presentation titled "Long-term functional improvement with zeleciment basivarsen in the phase 1/2 ACHIEVE trial" on September 30.
Additional WMS presentations will include the HARMONIA Phase 3 study design for z-basivarsen in DM1, as well as data from the DELIVER trial of z-rostudirsen and the FORZETTO Phase 3 study in DMD.
At AANEM, Dyne will present a poster titled "Zeleciment basivarsen targets the underlying cause of myotonic dystrophy type 1 to enable functional improvement in the Phase 1/2 ACHIEVE trial" on September 30 and October 1.
Additional AANEM presentations will cover the HARMONIA study design, increased dystrophin levels and functional improvement in the DELIVER trial, and trends in long-term clinical outcomes, including cardiopulmonary function, from z-rostudirsen.
ACHIEVE Trial
ACHIEVE is a global, randomized, placebo-controlled, double-blind Phase 1/2 clinical trial evaluating the safety, tolerability and efficacy of z-basivarsen in patients with DM1.
The multiple ascending dose portion of the study resulted in the selection of 6.8 mg/kg z-basivarsen administered every eight weeks as the registrational dose and regimen.
The registrational expansion cohort is fully enrolled and is intended to support potential regulatory submissions, including Accelerated Approval in the U.S.
Its primary endpoint is the change from baseline in middle-finger myotonia, measured by vHOT at six months, compared with placebo.
Z-Basivarsen For DM1
Z-basivarsen is an investigational therapeutic being evaluated in the global Phase 1/2 ACHIEVE trial and the global confirmatory Phase 3 HARMONIA trial for patients with DM1.
The therapy consists of an antisense oligonucleotide (ASO) conjugated to an antigen-binding fragment (Fab) that binds to transferrin receptor 1 (TfR1), enabling delivery to muscle and the central nervous system.
Z-basivarsen is designed to reduce toxic nuclear DMPK RNA, release splicing proteins and allow normal messenger RNA processing, with the goal of providing functional improvement for people living with DM1.
The candidate has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the U.S. Food and Drug Administration (FDA), as well as Orphan Drug designation from the European Medicines Agency (EMA) and Japan's Ministry of Health, Labour and Welfare.
Dyne anticipates submitting a Biologics License Application (BLA) for z-basivarsen in the third quarter of 2027.
Z-Rostudirsen For DMD
Dyne is also developing z-rostudirsen for patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
In mid-July 2026, the FDA accepted the company's BLA for z-rostudirsen, with a Prescription Drug User Fee Act (PDUFA) action date of January 21, 2027.
Z-rostudirsen continues to be evaluated in the Phase 3 FORZETTO trial for DMD and is also being advanced for DM1.
Broader Neuromuscular Pipeline
Dyne's broader pipeline includes clinical programs for DMD and DM1 and preclinical programs targeting facioscapulohumeral muscular dystrophy (FSHD), Pompe disease and additional DMD mutations.
The company is advancing DYNE-302 for FSHD, a disease associated with abnormal DUX4 expression. DYNE-302 has received FDA clearance to begin clinical development.
For DMD, Dyne's preclinical pipeline includes DYNE-253 targeting exon 53, DYNE-245 targeting exon 45, DYNE-244 targeting exon 44 and DYNE-255 targeting exon 55.
The company is also developing DYNE-401 as a preclinical program targeting the GAA gene for Pompe disease.
Dyne is leveraging its FORCE platform to deliver targeted therapies to skeletal muscle and the central nervous system, with the goal of addressing the underlying causes of genetically driven neuromuscular diseases.
Second-Quarter Financial Results
For the second quarter, Dyne recorded a net loss of $178.6 million, compared with a net loss of $110.9 million in the year-ago quarter.
The wider loss was primarily attributed to increased operating expenses associated with the final-stage clinical evaluation of the company's lead drug candidates.
Dyne is continuing to advance its late-stage clinical programs while preparing for potential regulatory submissions for z-rostudirsen and z-basivarsen.
The company expects the upcoming one-year ACHIEVE data to provide additional information on the durability of functional improvement and safety of z-basivarsen ahead of the planned first-quarter 2027 topline readout from the registrational expansion cohort.
DYN closed Friday's trade at $24.28, down 2.69%. In the premarket trading, the stock is trading down 29.82% at $17.04.
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