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Dyne Therapeutics Receives Orphan Drug Designation in Japan for Zeleciment Basivarsen (DYNE-101) for Myotonic Dystrophy Type 1

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- Zeleciment basivarsen (z-basivarsen) demonstrated sustained functional improvement across multiple...

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Dyne Therapeutics announced that z-basivarsen has shown sustained improvement in treating myotonic muscular dystrophy (DM1) in the ACHIEVE trial. The drug recently received Orphan Drug designation in Japan, highlighting its potential, and enrollment for the trial's expansion cohort is expected to complete in early Q2 2026, positioning Dyne for potential market entry.

Sentiment Rationale

Positive clinical trial results and regulatory designations historically correlate with share price increases in biotech firms, as seen with recent approvals and advancements in similar companies like Moderna.

Trading Thesis

Expect positive momentum for DYN as z-basivarsen progresses through trials and regulatory approval.

Market-Moving

  • Z-basivarsen's Orphan Drug designation could enhance market position and investor confidence.
  • Progress in the ACHIEVE trial may drive stock price higher ahead of regulatory discussions.
  • Completion of enrollment in early Q2 2026 could set the stage for future milestones.
  • Potential Accelerated Approval in the U.S. might create significant market opportunities.

Key Facts

  • Z-basivarsen shows promising results in ACHIEVE trial for DM1.
  • Japan grants Orphan Drug designation for z-basivarsen.
  • Enrollment for ACHIEVE expansion cohort expected by early Q2 2026.
  • Z-basivarsen targets root causes of myotonic muscular dystrophy.
  • No approved disease-modifying treatments for DM1 currently exist.

Companies Mentioned

  • U.S. Food and Drug Administration (FDA): Dyne's future approval chances hinge on FDA evaluation of z-basivarsen.
  • European Medicines Agency (EMA): EMA's designation reinforces the potential of z-basivarsen in Europe.

Corporate Developments

This article fits the category of 'Corporate Developments' as it discusses a key regulatory achievement and the ongoing clinical trial for Dyne Therapeutics' leading drug candidate, indicating significant progress in their drug development pipeline.

Dyne Therapeutics Secures Orphan Drug Designation in Japan for Zeleciment Basivarsen (DYNE-101) Targeting Myotonic Dystrophy Type 1

WALTHAM, Mass., January 20, 2026 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company dedicated to improving outcomes for individuals with genetically driven neuromuscular diseases, has announced that the Ministry of Health, Labour and Welfare (MHLW) in Japan has granted Orphan Drug designation for their investigational drug zeleciment basivarsen (z-basivarsen) for the treatment of myotonic dystrophy type 1 (DM1).

Clinical Advancements in the ACHIEVE Trial

Z-basivarsen is currently being evaluated in the Phase 1/2 ACHIEVE clinical trial where it has shown evidence of sustained functional improvements across multiple clinical metrics. Enrollment in the Registrational Expansion Cohort of this trial is expected to be completed by early Q2 2026.

Insights from Leadership

Doug Kerr, M.D., Ph.D., Chief Medical Officer of Dyne, stated, “By targeting the underlying biology of DM1, z-basivarsen has shown early and sustained improvements in myotonia, muscle strength, and function, with a favorable safety profile. This designation in Japan, alongside those already granted in the U.S. and Europe, underscores the urgent need for new therapies and highlights the potential of z-basivarsen to deliver meaningful functional improvement for people living with DM1.”

Understanding Orphan Drug Designation

The Orphan Drug designation in Japan is specifically aimed at treatments for rare diseases affecting fewer than 50,000 patients and addresses conditions with a high medical demand. Benefits of this designation include:

  • Subsidies for drug development costs
  • Potential market exclusivity for up to 10 years upon approval

In addition to the Japanese designation, z-basivarsen has received Breakthrough Therapy, Fast Track, and Orphan Drug designations from the U.S. FDA and Orphan Drug designation from the European Medicines Agency (EMA).

The ACHIEVE Clinical Trial Explained

The ACHIEVE clinical trial is a global, randomized, placebo-controlled, double-blind study aimed at evaluating the safety, tolerability, and efficacy of z-basivarsen (previously known as DYNE-101) in patients diagnosed with myotonic dystrophy type 1. The study’s multiple ascending dose (MAD) portion has led to the selection of a registrational dose of 6.8 mg/kg z-basivarsen administered every eight weeks.

The primary endpoint for the registrational expansion cohort is the change from baseline in middle finger myotonia, measured by video hand opening time (vHOT) at six months, compared to a placebo group. Additional details about the ACHIEVE trial can be found at clinicaltrials.gov.

About Zeleciment Basivarsen

Z-basivarsen is an investigational therapeutic featuring an antisense oligonucleotide (ASO) conjugated to an antigen-binding fragment (Fab) that binds to the transferrin receptor 1 (TfR1). This design aims to deliver functional improvement for individuals with DM1 by reducing toxic nuclear DMPK RNA, which is crucial for normal mRNA processing.

Impact of Myotonic Dystrophy Type 1

Myotonic dystrophy type 1 (DM1) is a rare, progressive genetic neuromuscular disease that affects approximately 40,000 individuals in the U.S. and ~55,000 in the EU. Symptoms can start between the ages of 20 and 40 and may include severe muscle weakness, myotonia (difficulty relaxing muscles), cognitive impairments, and other debilitating complications. Currently, there are no approved disease-modifying treatments specifically for DM1.

About Dyne Therapeutics

Dyne Therapeutics is on a mission to provide functional improvements for individuals suffering from genetically driven neuromuscular diseases. In addition to myotonic dystrophy type 1 (DM1), Dyne is advancing clinical programs for Duchenne muscular dystrophy (DMD) and is also pursuing preclinical programs for other related conditions. For more information about Dyne Therapeutics, visit dyne-tx.com.

Forward-Looking Statements

This press release contains forward-looking statements that involve substantial risks and uncertainties. These statements may include projections regarding Dyne’s strategy, potential benefits of z-basivarsen, and the anticipated timelines for clinical trial completion and regulatory submissions. Actual results may differ materially, and readers are cautioned not to place undue reliance on forward-looking statements.

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