– Dosing Initiation in Multi-Dose Escalating Clinical Trial of DYNE-101 in DM1 Patients On Track for Mid-2022 –
– Execution of global clinical and regulatory strategy: clinical trial application approved in New Zealand and additional authorizations expected in several countries –
WALTHAM, Mass., July 12, 2022 (GLOBE NEWSWIRE) — Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on developing innovative therapies that transform the lives of people with genetic diseases, today announced that the New Zealand Medicines and Medical Devices has authorized its clinical trial application to initiate its phase 1/2 multiple escalating dose (MAD) clinical trial of DYNE-101 in patients with myotonic dystrophy type 1 (DM1). The Company also expects to receive regulatory clearance in other countries for DYNE-101. Dyne plans to begin dosing patients in its clinical trial of DYNE-101 in mid-2022.
“We are delighted to have received our first regulatory clearance for DYNE-101, an important milestone in our efforts to run our planned global clinical trial for people living with DM1. Additionally, we have aligned the clinical trial design, including our starting dose, with regulatory authorities in several countries, and are focused on initiating the study. Today’s DM1 update, coupled with the recent FDA clearance of DYNE-251 for DMD, reinforces our commitment to bringing our co-lead programs to clinic patients in mid-2022,” said Joshua Brumm, President and CEO of Dyne. “We thank the New Zealand Medicines and Medical Devices Safety Authority, the FDA and other global regulatory authorities for their partnership as we continue to advance our mission to provide life-changing therapies for people with cancer. serious muscle diseases.
Dyne plans to evaluate DYNE-101 in a global, randomized, placebo-controlled MAD clinical trial with a long-term extension study. The trial aims to enroll approximately 60 to 70 adult patients with DM1. The study will assess safety, tolerability, splicing index, pharmacokinetics and pharmacodynamics, as well as measures of muscle strength and function. Dyne plans to provide additional details regarding the trial design and data timing at the start of the assay.
About Myotonic Dystrophy Type 1 (DM1)
DM1 is a rare and progressive genetic disease that affects skeletal, cardiac and smooth muscle. It is an autosomal dominant monogenic disease caused by an abnormal expansion of trinucleotides in a region of the DMPK gene. This expansion of CTG repeats causes toxic RNA to cluster in the nucleus, forming nuclear foci and impairing the splicing of several proteins essential for normal cellular function. This altered splicing leads to a wide range of symptoms. People 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 more than 74,000 people in Europe, but there are currently no approved disease-modifying treatments.
DYNE-101 is Dyne’s product candidate developed for people with myotonic dystrophy type 1 (DM1). DYNE-101 consists of an antigen-binding fragment (Fab) antibody conjugated to an antisense oligonucleotide (ASO) to enable targeted delivery of muscle tissue to reduce toxic DMPK RNA in the nucleus, releasing splicing proteins, allowing normal processing and translation of normal protein mRNA, and potentially halting or reversing disease. Dyne has generated comprehensive preclinical data in support of its DM1 program, including reduction of nuclear foci and correction of splicing in patient cells, robust inactivation of toxic human nuclear DMPK RNA, and correction of splicing in a new in vivo model developed by Dyne, and reversal of myotonia after a single dose in a disease model. In non-human primates, DYNE-101 demonstrated a favorable safety profile and achieved improved muscle distribution as evidenced by a significant reduction in wild-type DMPK RNA.
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 limitations of delivery 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 , expected timelines for patient dosing in the DYNE-251 and DYNE-101 trial and planned trial design for the DYNE-101 trial and the status of other regulatory submissions for the DYNE-101 trial , constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 The words “anticipate”, “believe”, “continue”, “could”, “estimate”, “expect”, “intend of”, “may”, “could”, “goal”, “in progress”, “plan”, “predict”, “project”, “potential”, “should” or “would”, or the negative form of these terms , or other comparable terminology, are intended to identify 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 obtain regulatory approval, initiation and enrollment of 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 needs; 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