Dynacure Axes Investigational Study of CNM Treatment

2022-07-08

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Dynacure has ended its Unite-CNM Phase I/II trial on DYN101, an investigational therapy for the treatment of myotubular and centronuclear myopathies (CNM). In a letter addressed to patients, the company said it is "unable to continue dosing at the low study dose or increase to a dose level at which we would expect to see clinical efficacy." Dynacure noted that while the first cohort of trial participants, who received a single dose of DYN101, tolerated the drug well, those from the second cohort, who received a higher quantity at the lowest level possible to determine efficacy, experienced elevated liver enzyme levels. The researchers tried to adjust the dose based on the patients' actual to ideal body weight, but all six patients still showed elevated liver enzymes. Five out of the six also reported having low platelet counts. After reviewing the study with an independent data monitoring committee, it was decided that dosing should be paused. The patients who had already taken the investigational drug continued with their follow-up visits and did not demonstrate any exposure to further risk. "This is not the news we ever wanted to share, as we know how great your needs are for safe and effective treatments. As a company and as individuals, we are overwhelmed with disappointment," Chris Freitag, M.D., the chief medical officer of Dynacure, said in the letter. "Like all clinical research efforts into CNM, the learnings from Unite-CNM are hard-earned by these study participants and investigators. They will add to the world's growing understanding of CNM. As we continue to review the findings of Unite-CNM, we will stay connected with study investigators, researchers, and patient advocacy organizations around the world. We will fulfill our obligation of making study results public," Freitag added. Dynacure had a promising journey prior to this unfortunate news. In June 2021, the U.S. Food and Drug Administration accepted its Investigational New Drug application for DYN101 due to positive initial results from its UNITE-CNM study. In January, the FDA granted it a Fast Track Designation. "Dynacure is continuing to investigate new approaches to address CNM, but it is too early to comment on any efforts. We remain very grateful for the heroic efforts of the patient who participated in this study, and their families and caregivers who supported these difficult endeavors," the letter concluded. CNMs are rare, life-threatening disorders that affect skeletal muscles from birth and affect around 4,000 to 5,000 patients in Australia, the European Union, Japan and the U.S. People diagnosed with CNM start experiencing muscle weakness from birth to early adulthood, and many patients die within the first 18 months of life. Those who survive longer usually require intense and uninterrupted medical assistance. Current interventions are merely supportive and not curative.

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