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Dosing and Enrollment in HT-100 Trial in Patients with DMD Suspended

Press Release: Dosing and Enrollment in HT-100 Trial Suspended

Background: Akashi Therapeutics has reported that dosing and new patient enrollment in the phase 1b/2a HALO trial, a study evaluating the experimental drug HT-100 in people with Duchenne muscular dystrophy (DMD), are being suspended following a medical emergency experienced by one of the trial participants. The participant, who was receiving the highest dose in the study, is reportedly experiencing serious, life-threatening health issues. Akashi is monitoring the medical situation closely and working with the U.S. Food and Drug Administration (FDA) to determine whether the patient’s health issues are related to HT-100. The company will provide additional information, including the impact on the future of this trial, once its investigation is complete.

Ht-100 is a small molecule drug designed to reduce fibrosis (scarring) and inflammation, and promote healthy muscle fiber regeneration in DMD.

Families of patients who had been participating or planning to participate in the HT-100 trial are encouraged to contact Akashi at, the principal investigator at their clinical site, or their physician with any questions or concerns.    

Statement from MDA Executive Vice President and Chief Medical and Scientific Officer Valerie Cwik, M.D.: “We are very saddened by this news. Our thoughts are with the trial participant and his family, and his recovery is our number one concern at this time. We will continue to be in contact with Akashi Therapeutics in the coming days as they work with the FDA to better understand the situation, including determining whether the drug in any way caused this medical emergency and what implications their findings will have for the future of the trial. The company will provide more information once the investigation is complete. MDA has led the search for treatments and cures for Duchenne and other neuromuscular diseases for more than 65 years. We have invested $209 million in DMD research alone, including support for Akashi Therapeutics through our MDA Venture Philanthropy program. Our community desperately needs and deserves treatment options, and we will continue to fund research that will ensure that safe and effective treatment options are made available as quickly as possible.”

About MDA

The Muscular Dystrophy Association is the world’s leading nonprofit health agency dedicated to saving and improving the lives of people with muscle disease, including muscular dystrophy, amyotrophic lateral sclerosis (ALS) and other neuromuscular diseases. It does so by funding worldwide research to find treatments and cures; by providing comprehensive health care services and support to MDA families nationwide; and by rallying communities to fight back through advocacy, fundraising and local engagement.

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