Bayer Acquires AskBio and its Gene Therapy Program for Pompe Disease

Bayer reached an agreement to acquire Asklepios BioPharmaceutical (AskBio), including its clinical program of ACT-101, an experimental one-time gene therapy for late-onset Pompe disease.

AskBio, which will remain an independent entity, has a portfolio that comprises early and clinical stage gene therapies to treat neuromuscular, neurologic, cardiovascular, and metabolic diseases.

“As an emerging leader in the rapidly advancing field of gene therapies, the expertise and portfolio of AskBio supports us in establishing highly innovative treatment options for patients and further strengthens our portfolio,” Stefan Oelrich, member of Bayer’s board of management and president of Bayer’s pharmaceuticals division, said in a press release.

“We want to help patients whose medical needs are not yet met by today’s treatment options and we are looking forward to work together with the team at AskBio,” Oelrich added.

Sheila Mikhail, AskBio’s CEO and co-founder, said that with Bayer’s worldwide reach and therapy development expertise, the retention of AskBio’s independence, and both companies’ commitment to patients, they “are positioned to provide accelerated development of gene therapies to treat more patients who can benefit from them.”

By acquiring full rights to AskBio’s gene therapy platform, Bayer “significantly advances the establishment of a cell and gene therapy platform that can be at the forefront of breakthrough science, contributing to preventing or even curing diseases caused by gene defects and further driving company growth in the future,” said Werner Baumann, Bayer’s CEO.

Under the terms of the agreement, AskBio will receive an upfront $2 billion payment from Bayer and up to $2 billion upon achievement of development milestones. About 75% of the potential success-based milestone payments are expected to be due over the next five years.

AskBio’s lead research programs — focused on Pompe disease, Parkinson’s disease, and heart failure, are in early phases of clinical development.

ACT-101 (also known as ACTUS-101) is an investigational gene therapy developed by Actus Therapeutics, a member of AskBio, for the treatment of Pompe disease. It targets the underlying cause of the disorder by using a modified, harmless adeno-associated virus (AAV) to deliver a healthy copy of the GAA gene, which is mutated in Pompe patients.

The GAA gene provides instructions for making acid-alpha-glucosidase (GAA), an enzyme involved in breaking down a large sugar molecule, called glycogen,  used for energy storage. GAA deficiency causes a toxic accumulation of glycogen in multiple tissues, which mainly affects cardiac, respiratory, and skeletal muscles, as well as the central nervous system (spinal cord and brain).

ACT-101, given through a single injection directly into the bloodstream, specifically targets the liver to promote GAA production.

A Phase 1/2 clinical trial (NCT03533673) is evaluating the therapy’s safety and preliminary effectiveness in up to eight adults with late-onset Pompe disease.

Participants, recruited at Duke University in North Carolina, will be assigned randomly to receive one of two doses of ACT-101, along with their current enzyme replacement therapy (ERT) — Pompe’s only approved treatment to date.

The first participant was dosed in 2019. Depending on available results, the patients may suspend ERT.

The trial’s main goal is to assess ACT-101’s safety, while secondary goals include measuring GAA activity levels in the muscle and blood, the immune response against GAA, and changes in motor and lung function, as well as in quality of life.

ACTUS-101 received fast track designation from the U.S. Food and Drug Administration for the treatment of Pompe disease. This designation is meant to expedite the therapy’s clinical development, regulatory review, and entry into the market upon approval.

Early this year, AskBio launched AskFirst, a collaborative program designed to keep patients and families informed about the company’s gene therapy research and potential cures for conditions such as Pompe disease, and to support those enrolled in clinical trials.