January 31, 2019

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AskBio: 1st Patient Dosed in Phase 1/2 Study for Pompe Disease


Initial clinical study to evaluate ACTUS-101 over 52 weeks that could improve quality of life for those who are affected with rare genetic disorder – Pompe Disease.

Actus Therapeutics, a privately held portfolio company of Asklepios BioPharmaceutical, Inc. (AskBio), today announced the dosing of the first patient in a phase 1/2 clinical study of ACTUS-101 in patients with Pompe Disease. Pompe Disease is a serious disease associated with high morbidity and often leads to premature death. There are variable rates of disease progression and different ages of onset characterized as infantile-onset and late-onset disease.

The initial clinical trial (ACT-CS101) is a study designed to assess the safety, bioactivity, and immune responses following treatment with a single infusion of ACTUS-101.

“This is an exciting milestone for our company but most importantly, if ACTUS-101 is successful, it could have a meaningful impact on the quality of life for those who suffer from Pompe Disease. Further, ACTUS 101 could replace enzyme replacement therapy (ERT) every other week with the potential to be a groundbreaking metabolic treatment for an unforgiving disease now showing up in an increasing number of patients,” said Sheila Mikhail, J.D., Chief Executive Officer and Co-Founder of AskBio.

Dr. Koeberl, Pediatric Medical Genetics Specialist at Duke University stated, “Preclinical data suggest that this gene therapy product may prove to continuously produce the GAA they (Pompe patients) are lacking. If enough GAA is produced, patients may be able to stop ERT entirely. This would improve their overall quality of life by eliminating the need for weekly or bi-weekly ERT treatments while also strengthening their outlook for living with Pompe.”

ACTUS-101 is delivered via intravenous injection and transduces the liver, leading to the continuous production of acid α-glucosidase (GAA). All study participants in this open label evaluation will receive ACTUS-101 while maintaining their current ERT with alglucosidase alfa. As the trial progresses, subjects who meet prespecified criteria for safety, transgene expression, and motor function will be eligible to suspend treatment with ERT.

“Our strategy at AskBio is to create transformative medicines for people with serious diseases through focusing on validated targets that address causal human biology,” added Mikhail. “ACTUS-101 is a next generation gene therapy, reflective of our commitment to discovering, developing, and manufacturing innovative AAV gene therapy candidates.”

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[Originally posted by CISION — January 231, 2019]