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US FDA places clinical hold on Astellas’ FORTIS trial of AT845 in adults with late-onset pompe disease

Tuesday, June 28, 2022, 12:00 Hrs  [IST]

Astellas Pharma Inc. announced that the US Food and Drug Administration (FDA) has placed a clinical hold on the FORTIS Ph1/2 trial following the occurrence of a serious adverse event (SAE) of peripheral sensory neuropathy in one of the trial participants. FORTIS is a clinical trial evaluating AT845, an investigational adeno-associated virus (AAV) gene replacement therapy in adults with late-onset pompe disease.

The FDA informed Astellas that it did not have sufficient information to assess the risks to subjects and requires additional information about the recently reported SAE. To date, the SAE has been classified by the site investigator as Grade 1 (mild in severity) and deemed serious due to medical significance. A written explanation for the basis of the hold will be issued by the FDA and sent to Astellas within the next 30 days.

Astellas is working with the site investigator to closely follow the patient's clinical course and will continue to gather and review all relevant data. All currently enrolled participants will continue to be monitored closely per the study protocol.

“Patient safety is our top priority, and we are working closely with the FDA to determine appropriate next steps,” explained Weston Miller, M.D., senior medical director, clinical development at Astellas Gene Therapies. “We remain committed to the safe and effective development of AT845 and will keep the scientific and patient communities informed with updates as we learn more.”

Astellas is focused on developing genetic medicines and working alongside its world-renowned partners to build a portfolio of potentially life-changing gene therapies. Astellas strives to identify, develop and deliver therapies for patients with genetic diseases who currently have few or no effective treatment options.

Astellas is reviewing potential financial impacts of this matter for the fiscal year ending March 31, 2023.

Pompe disease is a rare, severe, autosomal recessive metabolic disease characterized by progressive muscular degeneration. The overall incidence is estimated to be approximately 1 in 40,000 births, although frequency and disease progression varies with age of onset, ethnicity and geography. The disease is caused by mutations in the alpha-glucosidase (GAA) gene that prevent the production and function of a protein called acid alpha-glucosidase (GAA). GAA is responsible for metabolizing glycogen, and dysfunction or absence of this protein results in the accumulation of glycogen in tissues, primarily in the skeletal and cardiac muscles, where it causes damage to tissue structure and function. Currently, the only approved treatment for Pompe is enzyme replacement therapy (ERT), which is a chronic treatment delivered in bi-weekly infusions and relies solely on tissue uptake of GAA from plasma.

Astellas is developing AT845, a novel gene replacement therapy using an AAV8 vector under a muscle-specific promotor to deliver a functional copy of the GAA gene, for the treatment of LOPD. AT845 is being investigated to determine whether it can deliver a functional GAA gene that is efficiently transduced to express GAA directly in tissues affected by the disease, including skeletal and cardiac muscle.

FORTIS (NCT04174105) is a multicenter, open-label, ascending dose phase I/II first-in-human clinical trial to determine if AT845 is safe and tolerable in adults with LOPD. The primary endpoints of the trial are safety and tolerability, as well as efficacy measures, including change in muscle GAA protein expression and enzyme activity from baseline. Secondary endpoints evaluate improvements in respiratory, endurance and quality of life measures.

Astellas Pharma Inc. is a pharmaceutical company conducting business in more than 70 countries around the world.


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