Asklepios BioPharma Launches New Biotech Actus Therapeutics

Asklepios BioPharmaceutical, which goes by AskBio, is forming a new biotech portfolio company, Actus Therapeutics.

AskBio was formed in 2003 by Richard Jude Samulski and Sheila Mikhail to leverage the gene therapy resources in the Research Triangle Park area. It has created numerous spinout companies while pursuing its own gene therapies. Others include NanoCor Therapeutics, partnered with Medtronic to develop treatments for heart failure, Chatham Therapeutics, which sold to Baxter to develop hemophilia treatments, and Bamboo Therapeutics which sold to Pfizer to develop therapies for rare neuromuscular diseases and muscular dystrophy.

Actus Therapeutics is built on AskBio’s gene therapy platform and will focus on multiple rare genetic diseases. It will initially work on a gene therapy for Pompe Diseases based on the research of Dwight Koeberl, a professor of pediatrics and a medical genetics specialist at Duke University.

The company will develop targeted low-dose gene therapies. AskBio’s gene therapy program using adeno-associated virus vectors (AAVs) are effective at lower doses, and because of their cell-type specificity, potentially have fewer systemic side effects.

“AskBio is focused on expanding our current gene therapy portfolio to help patients and families with rare genetic diseases that might be treated through our proprietary gene-therapy platform,” said Sheila Mikhail, president and chief executive officer of both AskBio and Actus, in a statement. “We continue to advance the efficacy of our vectors as well as our manufacturing platform, and we are excited to be working with Dr. Dwight Koeberl and his team at Duke, with the aim of developing a new therapy for Pompe Disease.”

The new company is recruiting 20 adults with late-onset Pompe Disease for a clinical trial.

Pompe Disease is an inherited disease which is the result of glycogen buildup in cells. The accumulation of the complex sugar in certain organs and tissues, particularly muscles, causes abnormal function. There are three different types of the disease that vary in severity and age of onset. They are classic infantile-onset, non-class infantile-onset, and late-onset. Overall, the disease can lead to muscle weakness, poor muscle tone, enlarged liver, and heart defects, breathing difficulties, and liver disease. Infantile-onset is often fatal in the first year of life. Non-classic infantile-onset children also usually die very young. Late-onset can lead to respiratory failure.

On Sept. 26, AskBio’s NanoCor Therapeutics announced it had published a large animal study in support of its lead candidate, Carfostin. In the publication, heart function improved by up to 25 percent after a single gene therapy regimen in a pig model. The data were published in the Journal of the American College of Cardiology, titled, “Protein Phosphatase Inhibitor Gene Therapy in a Swine Model of Nonischemic Heart Failure.”

“In this study of large animals with heart failure, the investigators have shown that the treatment NanoCor will soon be testing in humans appears promising,” Mikhail, also president and chief executive officer of NanoCor, said in a statement at the time. “There are currently no gene therapy treatment options approved for late stage congestive heart failure. We are excited to move Carfostin forward and make a new treatment option available.”