Dyne Therapeutics gets BTD for DYNE-101 in Myotonic Dystrophy Type 1 (DM1)

Dyne Therapeutics gets BTD for DYNE-101 in Myotonic Dystrophy Type 1 (DM1)

By, Admin 19 June 2025

Overview

Dyne Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to DYNE-101, an investigational treatment for myotonic dystrophy type 1 (DM1).

The DYNE-101 trial

  • DYNE-101 is a novel therapeutic under investigation in the global Phase 1/2 ACHIEVE trial. 
  • It is composed of an antisense oligonucleotide (ASO) linked to a fragment antibody targeting transferrin receptor 1 (TfR1), facilitating delivery to muscle and the central nervous system. 
  • It aims to reduce toxic DMPK RNA and correct the abnormal splicing process seen in DM1. 

Myotonic dystrophy type 1 (DM1)

  • DM1 is a rare, inherited condition affecting muscle and nervous system function. 
  • It is caused by a CTG trinucleotide repeat expansion in the DMPK gene, leading to the accumulation of toxic RNA in the cell nucleus. 
  • This disrupts normal protein splicing and results in a broad range of symptoms including muscle stiffness, weakness, fatigue, heart and respiratory issues, and cognitive difficulties. 

Current treatment condition

  • There are currently no approved therapies that modify the course of the disease. 
  • DM1 is estimated to affect over 40,000 individuals in the U.S. and more than 74,000 in Europe.

The announcement follows a recent Type C meeting with the FDA and the availability of new long-term functional data. The company also shared its updated regulatory pathway, aiming for Accelerated Approval in the United States.

For submission of Accelerated Approval submission

  • Dyne plans to use these results, along with data from other parts of the ACHIEVE trial, to support a potential Accelerated Approval submission by the end of 2026. 
  • A confirmatory Phase 3 trial is planned to begin in the first quarter of 2026. 
  • The company is also exploring accelerated regulatory pathways in other countries.

Adverse effects- The treatment continues to demonstrate a favourable safety profile, with no serious adverse events related to the drug reported in 56 participants.

Investments till 2025

As of 31 March 2025, Dyne reported cash and investments totalling $677.5 million and expects to maintain operations into the fourth quarter of 2026.

BTD speeding up the drug development

  • Breakthrough Therapy Designation is intended to speed up the development of medicines showing early evidence of significant improvement over existing therapies. 
  • Benefits include increased guidance from senior FDA officials, frequent communications on trial design and regulatory issues, and eligibility for expedited reviews.

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