Dyne Therapeutics Announces FDA Approval of IND Application for DYNE-251 for the Treatment of … | Your money


– Initiation of Assay in Multi-Dose Escalating Clinical Trial in Patients with Exon 51-Skipping Mutations On Track for Mid-2022 –

WALTHAM, Mass., July 05, 2022 (GLOBE NEWSWIRE) — Dyne Therapeutics, Inc.. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative therapies that transform the lives of people with genetic diseases, today announced that the Food and Drug Administration (FDA) of the United States had lifted the clinical hold and authorized its New Drug Application (IND) study to initiate a clinical trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) susceptible to exon 51 skipping. company plans to begin dosing patients in a Phase 1/2 clinical trial evaluating DYNE-251 in mid-2022.

“Today marks an important milestone in our journey to build a DMD franchise to serve people around the world with potentially exon-skipping Duchenne mutations. Clearing our first IND is a significant achievement for Dyne, and we value the partnership with the FDA throughout this process. Our team has worked extensively with key opinion leaders, patient advocacy groups, and people with DMD to thoughtfully design and execute our global, multiple-escalating dose Phase 1/2 clinical trial of DYNE-251” , said Joshua Brumm, President and CEO. of Dyne. “We believe we are well placed to deliver on our commitment to begin dosing in both our DMD and DM1 programs in mid-2022. The entire Dyne team is proud of the progress we have made in advancing our mission and addressing the urgent need to bring new treatment options to people with serious muscle diseases.

Dyne plans to evaluate DYNE-251 in a global, randomized, placebo-controlled, multiple-escalating-dose (MAD) clinical trial with a long-term extension study. The trial aims to enroll 30-50 ambulant and non-ambulant males with Duchenne, ages 4-16 years, with mutations amenable to exon 51 skipping therapy. The study will assess the safety, tolerability, dystrophin expression as measured by Western Blot, pharmacokinetics and pharmacodynamics, and measures of muscle function. Dyne plans to provide additional details regarding the trial design and data timing at the start of the assay.

In addition to DYNE-251, Dyne is building a global DMD franchise with preclinical programs for patients with mutations that can skip other exons, including 53, 45 and 44.

About Duchenne Muscular Dystrophy (DMD) Duchenne is a rare disease caused by mutations in the gene that codes for dystrophin, a protein essential for normal muscle cell function. These mutations, the majority of which are deletions, result in a lack of the dystrophin protein and a progressive loss of muscle function. DMD occurs primarily in men and affects approximately 12,000 to 15,000 people in the United States and 25,000 in Europe. The loss of strength and function usually first appears in preschool boys and worsens with age. As the disease progresses, the severity of skeletal and cardiac muscle damage often results in patients losing their ability to walk completely in early adolescence and includes worsening cardiac and respiratory symptoms and loss of function. upper body in late adolescence. There is no cure for DMD and currently approved therapies offer limited benefit.

About DYNE-251 DYNE-251 is Dyne’s product candidate under development for people with Duchenne muscular dystrophy (DMD) at risk of exon 51 skipping. DYNE-251 consists of a phosphorodiamidate morpholino oligomer ( PMO) conjugated to an antibody fragment (Fab) that binds to transferrin receptor 1 (TfR1) which is highly expressed on muscle. It is designed to enable targeted delivery of muscle tissue and promote exon skipping in the nucleus, allowing muscle cells to create functional truncated dystrophin protein, with the aim of stopping or reversing disease progression . In preclinical studies with Dyne’s FORCE™ platform, robust and durable exon skipping and dystrophin expression was observed in the mdx mouse model in skeletal and cardiac muscle, along with reduced muscle damage and an increase in muscle function. DYNE-251 demonstrated a favorable safety profile and achieved impressive exon skipping in non-human primates, particularly in the heart and diaphragm, muscles that weaken over time leading to mortality in people with DMD.

About Dyne Therapeutics

Dyne Therapeutics is a clinical-stage muscle disease company focused on advancing innovative therapies that transform the lives of people with genetic diseases. With its proprietary FORCE™ platform, Dyne is developing modern oligonucleotide therapies designed to overcome the delivery limitations to muscle tissue seen with other approaches. Dyne has a broad portfolio of programs for serious muscle diseases, including candidates for Myotonic Dystrophy Type 1 (DM1), Duchenne Muscular Dystrophy (DMD) and Facioscapulohumeral Muscular Dystrophy (FSHD). For more information, please visit https://www.dyne-tx.com/and follow us on Twitter, LinkedIn and Facebook.

Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical fact, contained in this press release, including statements regarding Dyne’s strategy, future operations, outlook and plans, management objectives, potential of the FORCE platform , the anticipated timelines for patient dosing in the DYNE-251 and DYNE-101 trial and the anticipated trial design for the DYNE-251 trial, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act. from 1995. The words “anticipate”, “believe”, “continue”, “could”, “estimate”, “expect”, “intend”, “may”, “could”, “aim “, “in progress”, “plan”, “predict”, “project”, “potential”, “should” or “should” or the negative form of these terms, or other comparable terminology, are intended to identify statements forward-looking statements, although not all forward-looking statements contain these identifying words. Dyne may not actually achieve the plans, intentions or expectations disclosed in such forward-looking statements, and you should not place undue reliance on such forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements due to a variety of important factors, including: the uncertainties inherent in the identification and development of product candidates, including the launch and completion preclinical studies and clinical trials; uncertainties regarding the availability and timing of results from preclinical studies and clinical trials; the timing and ability of Dyne to initiate and enroll patients in clinical trials; whether the results of preclinical studies will be predictive of the results of preclinical studies and subsequent clinical trials; whether Dyne’s cash resources will be sufficient to fund the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements; uncertainties associated with the impact of the COVID-19 pandemic on Dyne’s business and operations; and the risks and uncertainties identified in Dyne’s filings with the Securities and Exchange Commission (SEC), including the Company’s most recent Form 10-Q and in Dyne’s subsequent filings with the SEC. In addition, the forward-looking statements included in this press release represent Dyne’s views as of the date of this press release. Dyne anticipates that subsequent events and developments will result in a change of opinion. However, while Dyne may choose to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be taken to represent the views of Dyne as of any date subsequent to the date of this press release.

Contact: Dyne Therapeutics Amy Reilly [email protected] 857-341-1203


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