– HARMONIA trial will assess multi-system efficacy, security and tolerability of z-basivarsen in DM1 –
– 48-week trial will enroll roughly 150 people, and first websites are actually open for enrollment –
– Major endpoint is the 5 instances sit to face (5xSTS) take a look at; secondary and exploratory endpoints will assess muscle perform, CNS manifestations, and patient- and clinician-reported outcomes –
– HARMONIA trial design and protocol aligned with FDA; trial meant to function confirmatory trial for conventional approval in the U.S. and help ex-U.S. advertising and marketing functions –
WALTHAM, Mass., March 08, 2026 (GLOBE NEWSWIRE) — Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage firm centered on delivering practical enchancment for individuals dwelling with genetically pushed neuromuscular ailments, as we speak introduced the initiation of the Phase 3 HARMONIA trial of zeleciment basivarsen (z-basivarsen, often known as DYNE-101), in people with myotonic dystrophy sort 1 (DM1). The design of the HARMONIA trial is being introduced on the 2026 Muscular Dystrophy Affiliation (MDA) Medical & Scientific Convention on Wednesday, March 11, 2026 at 9:30 a.m. ET. A corresponding poster can be out there in the Scientific Publications & Shows part of Dyne’s web site.
“We’re proud to be contributing to key advances in myotonic dystrophy scientific analysis with the initiation of a field-defining Phase 3 research designed to exhibit the broad potential advantages of z-basivarsen,” mentioned Doug Kerr, M.D., Ph.D., chief medical officer of Dyne. “Constructing on the continued registrational enlargement cohort of the Phase 1/2 ACHIEVE trial, which is using myotonia, as measured by video hand opening time, as an early indicator of scientific profit for potential U.S. Accelerated Approval, HARMONIA is a bigger and longer-term research using a clinically significant practical measure as the first endpoint. HARMONIA was designed to bolster the best-in-class potential of z-basivarsen based mostly on the differentiated capabilities of our FORCE platform to ship therapeutics to a broad vary of muscle programs in addition to the CNS.”
HARMONIA is a worldwide, randomized, placebo-controlled, double-blind, confirmatory Phase 3 trial designed to evaluate the multi-system efficacy, security, and tolerability of z-basivarsen administered intravenously to people with DM1. The trial will enroll roughly 150 members age 16 and older who might be randomized 1:1 to obtain 6.8 mg/kg of z-basivarsen or placebo each eight weeks (Q8W). The primary trial websites are activated and open to enrollment.
The first endpoint is the change from baseline in the 5 instances sit to face (5xSTS) take a look at at week 49. The 5xSTS take a look at is a dependable and responsive measure that displays key areas of DM1 impairment, together with decrease extremity energy, stability and trunk energy, that are important to performing day by day actions. Secondary endpoints embody video hand opening time, quantitative muscle testing, the 10-Meter Stroll/Run take a look at, the Myotonic Dystrophy Well being Index, and extra patient- and clinician-reported outcomes. The trial additionally features a broad set of exploratory endpoints designed to evaluate a number of domains of DM1 central nervous system (CNS) influence. Following the 48-week double-blind placebo-controlled remedy interval, sufferers might be eligible to enroll in a 24-week long-term extension.
Dyne has aligned with the U.S. Meals and Drug Administration (FDA) on the HARMONIA Phase 3 trial design and protocol. HARMONIA is meant to function a confirmatory trial to help conversion of Accelerated Approval to conventional approval in the U.S. and to help ex-U.S. advertising and marketing functions.
In regards to the HARMONIA Trial
HARMONIA is a worldwide, randomized, placebo-controlled, double-blind, confirmatory Phase 3 scientific trial evaluating the efficacy, security and tolerability of zeleciment basivarsen (z-basivarsen, often known as DYNE-101) in individuals dwelling with myotonic dystrophy sort 1 (DM1). The trial will enroll roughly 150 members age 16 and older who will obtain 6.8mg/kg of z-basivarsen or placebo as soon as each eight weeks for 48 weeks, and members who full the placebo-controlled interval could enter a long-term extension throughout which all will obtain 6.8mg/kg of z-basivarsen each eight weeks for as much as 24 further weeks. The first endpoint of HARMONIA is the change from baseline in the 5 instances sit to face (5xSTS) take a look at at week 49. The 5xSTS take a look at is a dependable and responsive measure that displays key areas of DM1 impairment, together with decrease extremity energy, stability and trunk energy, that are important to performing day by day actions. Secondary endpoints embody video hand opening time, quantitative muscle testing, the 10-Meter Stroll/Run take a look at, the Myotonic Dystrophy Well being Index, and extra patient- and clinician-reported outcomes. The trial additionally features a broad set of exploratory endpoints designed to evaluate a number of domains of DM1 central nervous system influence.
About zeleciment basivarsen (z-basivarsen, previously referred to as DYNE-101)
Z-basivarsen is an investigational therapeutic being evaluated in the Phase 1/2 world ACHIEVE scientific trial for individuals dwelling with DM1. Z-basivarsen consists of an antisense oligonucleotide (ASO) conjugated to an antigen-binding fragment (Fab) that binds to the transferrin receptor 1 (TfR1) to allow supply to muscle and the central nervous system. It’s designed to ship practical enchancment in people dwelling with DM1 by lowering poisonous nuclear DMPK RNA to launch splicing proteins and permit regular mRNA processing. Z-basivarsen has been granted Breakthrough Remedy, Orphan Drug and Quick Observe designations by the U.S. Meals and Drug Administration (FDA), in addition to Orphan Drug designation from the European Medicines Company (EMA) and the Ministry of Well being, Labour and Welfare (MHLW) in Japan for the remedy of DM1.
About Myotonic Dystrophy Type 1 (DM1)
Myotonic dystrophy sort 1 (DM1) is a uncommon, progressive, genetic neuromuscular illness with excessive morbidity and early mortality. DM1 impacts ~40,000 individuals in the U.S. and ~55,000 individuals in the EU. The severity of signs and price of development varies. Signs can start at any level in an affected particular person’s life, relying on the DM1 subtype. Grownup-onset DM1 signs usually seem between 20 to 40 years of age. DM1 is brought on by mutations in the DMPK gene, resulting in a widespread disruption of RNA splicing, referred to as spliceopathy, which drives the multi-system manifestations of the illness. Individuals expertise a broad spectrum of signs, together with: muscle weak point all through the physique, myotonia or problem enjoyable muscle tissue, extreme daytime sleepiness, fatigue, dysregulated sleep, cognitive impairments, cardiac arrhythmias, respiratory points and gastrointestinal dysfunction. Though the genetic trigger of DM1 is nicely understood, there are at present no accepted disease-modifying therapies for DM1.
About Dyne Therapeutics
Dyne Therapeutics is concentrated on delivering practical enchancment for individuals dwelling with genetically pushed neuromuscular ailments. We’re creating therapeutics that concentrate on muscle and the central nervous system (CNS) to deal with the basis trigger of illness. The corporate is advancing scientific packages for Duchenne muscular dystrophy (DMD) and myotonic dystrophy sort 1 (DM1) in addition to preclinical packages for facioscapulohumeral muscular dystrophy (FSHD), Pompe illness and a number of DMD mutations. At Dyne, we’re on a mission to ship practical enchancment for people, households and communities. Study extra at https://www.dyne-tx.com/, and observe us on X, LinkedIn and Fb.
Ahead-Trying Statements
This press launch accommodates forward-looking statements that contain substantial dangers and uncertainties. All statements, apart from statements of historic information, contained in this press launch, together with statements relating to Dyne’s technique, future operations, prospects and plans, goals of administration, the potential of the FORCE platform, the scientific potential of zeleciment basivarsen (z-basivarsen, often known as DYNE-101), the potential of video hand opening time to function an intermediate scientific endpoint for U.S. accelerated approval, and the aptitude of Dyne’s FORCE platform to ship therapeutics to a broad vary of muscle programs in addition to the central nervous system, represent forward-looking statements throughout the which means of The Personal Securities Litigation Reform Act of 1995. The phrases “anticipate,” “consider,” “proceed,” “might,” “estimate,” “anticipate,” “intend,” “could,” “would possibly,” “goal,” “ongoing,” “plan,” “predict,” “venture,” “potential,” “ought to,” “will” or “would,” or the unfavourable of these phrases, or different comparable terminology are meant to determine forward-looking statements, though not all forward-looking statements comprise these figuring out phrases. Dyne could not really obtain the plans, intentions or expectations disclosed in these forward-looking statements, and you shouldn’t place undue reliance on these forward-looking statements. Precise outcomes or occasions might differ materially from the plans, intentions and expectations disclosed in these forward-looking statements because of this of varied necessary components, together with: uncertainties inherent in the identification and growth of product candidates, together with the initiation and completion of preclinical research and scientific trials; uncertainties as to the provision and timing of outcomes from preclinical research and scientific trials; the timing of and Dyne’s skill to enroll sufferers in scientific trials; uncertainties as to the FDA’s and different regulatory authorities’ interpretation of the info from Dyne’s scientific trials and the regulatory approval course of; whether or not Dyne’s money sources might be adequate to fund its foreseeable and unforeseeable working bills and capital expenditure necessities; in addition to the dangers and uncertainties recognized in Dyne’s filings with the Securities and Alternate Fee (SEC), together with the Firm’s most up-to-date Type 10-Okay and in subsequent filings Dyne could make with the SEC. As well as, the forward-looking statements included in this press launch symbolize Dyne’s views as of the date of this press launch. Dyne anticipates that subsequent occasions and developments will trigger its views to vary. Nevertheless, whereas Dyne could elect to replace these forward-looking statements sooner or later in the long run, it particularly disclaims any obligation to take action. These forward-looking statements shouldn’t be relied upon as representing Dyne’s views as of any date subsequent to the date of this press launch.
Contacts:
Buyers
Mia Tobias
ir@dyne-tx.com
781-317-0353
Media
Stacy Nartker
snartker@dyne-tx.com
781-317-1938
(*1*)
#Dyne #Therapeutics #Announces #Initiation #Phase #HARMONIA #Trial #ZBasivarsen #Myotonic #Dystrophy #Type #DM1
