Bayer Expands into Gene Therapy with Acquisition of Asklepios for Up to $4 Billion

Rare Daily Staff

Bayer made a strong commitment to gene therapy medicines, announcing it will acquire Asklepios BioPharmaceutical for $2 billion upfront and up to another $2 billion in success-based milestone payments.

“With this acquisition, 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, chairman of the Board and CEO of Bayer.

Asklepios (AskBio) specializes in the research, development, and manufacturing of gene therapies across different therapeutic areas, and has a pipeline of experimental preclinical and clinical stage candidates that includes treatments for rare neuromuscular, central nervous system, cardiovascular, and metabolic diseases. Lead programs include treatments for Pompe disease, Parkinson’s disease, and congestive heart failure. The company also has several active collaborations, including with Pfizer in Duchenne muscular dystrophy and with Takeda Pharmaceutical in hemophilia.

Founded in 2001 by Jude Samulski, the first scientist to clone AAV, along with Xiao, who was the first to develop a miniaturized dystrophin gene enabling the advancement of gene therapy for Duchenne muscular dystrophy, and company CEO Sheila Mikhail, AskBio offers proprietary AAV technology, best-in-class manufacturing capabilities, and a robust pipeline of in-house clinical programs.

An early innovator in the space, the company holds more than 500 patents in areas such as AAV production, chimeric vectors, and self-complementary DNA. The company has generated hundreds of proprietary third generation AAV capsids and promoters that offer differentiation through potentially improved efficacy, immune response and tissue and organ specificity. AskBio’s engineered viral vectors are able to target particular tissues, de-target other tissues, and minimize the effects of neutralizing antibodies. The platform also holds promise to also tackle polygenetic indications, thereby also helping a larger number of patients.

“Our innovation in capsid re-engineering and promoter design, coupled with our scaled manufacturing processes, gives us the tools to provide gene therapy solutions to more people suffering from a wider spectrum of disease that is not being adequately treated today,” said Jude Samulski, chief scientific officer and co-founder of AskBio.

Under the terms of the buyout, Bayer will own full rights to AskBio’s gene therapy platform, including a broad intellectual property portfolio and an established contract development and manufacturing organization laying the foundation for future partnerships in the area of adeno-associated virus (AAV) therapies.

Bayer says the deal will strengthen Bayer’s emerging cell and gene therapy business and commitment to the field and complements the Big Pharma’s 2019 acquisition of BlueRock Therapeutics. Bayer will pay an upfront consideration of $2 billion and potential success-based milestone payments of up to $2 billion. Some 75 percent of the potential success-based milestone payments are expected to be due during the course of the next five years and the remaining amount late thereafter.

AskBio will operate as an independent company on an arm’s-length basis. Bayer’s newly established Cell and Gene Therapy unit will bundle Bayer’s activities in this area moving forward in order to establish an innovation ecosystem for the participating partners within the Bayer organization.

Through the acquisition, Bayer will add an industry-leading AAV-based gene therapy platform to its portfolio that has already demonstrated applicability across different therapeutic areas. Besides multiple clinical-stage assets for indications with high unmet need, the acquisition includes a state-of-the-art gene therapy technology platform as well as an existing gene therapy-manufacturing platform.

“With Bayer‘s worldwide reach and translational expertise, especially in pathway diseases, our combined cultures of scientific advancement and commitment to patients, along with the retention of AskBio’s independent structure, Bayer and AskBio are positioned to provide accelerated development of gene therapies to treat more patients who can benefit from them,” said Sheila Mikhail, CEO and co-founder of AskBio.

Closing of the transaction is contingent on customary closing conditions, including receipt of the required regulatory approvals, and is expected to take place during the fourth quarter of 2020.

Photo: Sheila Mikhail, CEO and co-founder of AskBio