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Dyne Hopes DM1 And DMD Mid-Stage Data Can Lead To Accelerated Approval

 
• By Joseph Haas

Phase I/II data for its ADC candidates in myotonic dystrophy type 1 and Duchenne muscular dystrophy indicate potential for biomarker-based accelerated approval in both diseases, Dyne asserts.

Expediting
Dyne plans to pursue accelerated approved in both DM1 and DMD • Source: Shutterstock

Dyne Therapeutics has reported new data from Phase I/II studies in myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy that it believes can position each candidate for US accelerated approval with best-in-class potential, and analysts see as validating the Waltham, MA-based company’s platform for oligonucleotide therapeutic.

Key Takeaways

  • Dyne reported new data from ongoing Phase I/II trials of its oligonucleotide conjugate therapies in DM1 and DMD that it thinks offer best-in-class potential.

  • The biotech will seek accelerated approval based on surrogate biomarker data in both indications

On 20 May, Dyne reported that DYNE-101, its oligonucleotide antibody-drug conjugate (ADC) therapy for DM1, demonstrated dose-dependent mean splicing correction which in theory can reduce toxic myotonic dystrophy protein kinase (DMPK) RNA in DM1 patients. Three-month data testing a 5

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