Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-101

2023-05-25

孤儿药寡核苷酸临床研究

- Initial Data from Global, Multiple Ascending Dose Phase 1/2 ACHIEVE Clinical Trial in Myotonic Dystrophy Type 1 Anticipated in the Second Half of 2023 -

Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that the European Medicines Agency (EMA) has granted orphan drug designation for DYNE-101. DYNE-101 is being evaluated in the Phase 1/2 ACHIEVE global clinical trial in adults with myotonic dystrophy type 1 (DM1).

“We are pleased to receive orphan drug designation from the EMA for DYNE-101, further supporting our efforts to develop a potentially transformative therapy for DM1,” said Wildon Farwell, M.D., MPH, chief medical officer of Dyne. “The DM1 community has waited far too long for a therapy that addresses the underlying cause of this devastating rare muscle disease. We are committed to advancing DYNE-101 as quickly as possible and anticipate our first clinical data readout from our ACHIEVE trial later this year.”

The EMA grants orphan drug designation to drugs and biologics intended for the treatment, diagnosis or prevention of rare, life-threatening or chronically debilitating diseases or conditions that affect fewer than five in 10,000 people in the European Union. Orphan designation allows companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and up to 10 years of market exclusivity in the European Union if approved.

About DYNE-101 and the ACHIEVE Trial

DYNE-101 is an investigational therapeutic being evaluated in the Phase 1/2 global ACHIEVE trial, for people living with myotonic dystrophy type 1 (DM1). The ACHIEVE clinical trial consists of a 24-week multiple ascending dose (MAD) randomized placebo-controlled period, a 24-week open-label extension and a 96-week long-term extension. The trial, which is designed to be registrational, is expected to enroll approximately 72 adult patients with DM1 who are 18 to 49 years of age. The primary endpoints are safety and tolerability with secondary endpoints of pharmacokinetics and pharmacodynamics, including change from baseline in splicing, as well as measures of muscle strength and function. Dyne anticipates reporting initial data from the MAD placebo-controlled portion of the ACHIEVE trial on safety, tolerability and splicing in the second half of 2023.

DYNE-101 consists of an antigen-binding fragment antibody (Fab) conjugated to an antisense oligonucleotide (ASO) and is designed to enable targeted muscle tissue delivery with the goal of reducing toxic DMPK RNA in the nucleus, releasing splicing proteins, allowing normal mRNA processing and translation of normal proteins, and potentially stopping or reversing the disease. Dyne has generated comprehensive preclinical data supporting its DM1 program, including reduction of nuclear foci and correction of splicing in patient cells, robust knockdown of toxic human nuclear DMPK RNA and correction of splicing in a novel in vivo model developed by Dyne, and reversal of myotonia in a disease model. In non-human primates, DYNE-101 demonstrated a favorable safety profile and achieved enhanced muscle distribution as evidenced by significant reduction in wild-type DMPK RNA.

About Myotonic Dystrophy Type 1 (DM1)

DM1 is a rare, progressive, genetic disease that affects skeletal, cardiac and smooth muscle. It is a monogenic, autosomal dominant disease caused by an abnormal trinucleotide expansion in a region of the DMPK gene. This expansion of CTG repeats causes toxic RNA to cluster in the nucleus, forming nuclear foci and altering the splicing of multiple proteins essential for normal cellular function. This altered splicing, or spliceopathy, results in a wide range of symptoms. People living with DM1 typically experience progressive weakness of major muscle groups, which can affect mobility, breathing, heart function, speech, digestion and vision as well as cognition. DM1 is estimated to affect more than 40,000 people in the United States and over 74,000 people in Europe, but there are currently no approved disease-modifying therapies.

About Dyne Therapeutics

Dyne Therapeutics is a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases. With its proprietary FORCE™ platform, Dyne is developing modern oligonucleotide therapeutics that are designed to overcome limitations in delivery to muscle tissue. Dyne has a broad pipeline for serious muscle diseases, including clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD) and a preclinical program for facioscapulohumeral muscular dystrophy (FSHD). For more information, please visit https://www.dyne-tx.com/, and follow us on Twitter, LinkedIn and Facebook.

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