Biogen haemophilia spin-out to develop gene therapies and long-acting factors

By Dan Stanton contact

- Last updated on GMT

Image: iStock/jarun011
Image: iStock/jarun011

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The new entity will focus on a haemophilia pipeline utilising the XTEN half-life extension technology, bispecific antibodies and gene therapies.

Biogen announced yesterday it is planning to spin-out its haemophilia business into an independent, public firm based in Boston, Massachusetts by early next year.

Management said during a conference call this was the right time for a spin-out as Biogen’s haemophilia business has matured.

“It’s doing very well, it is profitable and can stand on its own, and it has a pipeline that needs to be developed aggressively, so it’s the right time,”​ said John Cox, the Biogen exec set to lead the new firm.

The firm also said Biogen will provide transition services to the new company, including manufacturing its therapies for the next three to five years.

‘Market leader in haemophilia’

The new company will be supported by Biogen’s existing marketed products Elocate (an antihemophilic Factor Fc fusion protein) and Alprolix (a Factor IX Fc Fusion Protein), Cox continued, and would help the spin-out “become the market leader in haemophilia.”

But to grow the business further it will be essential to build a novel pipeline based on “early work in gene therapies, even longer acting factors, and bispecific antibodies,” ​he told investors during a conference call.

In 2014, Biogen signed a licensing agreement with California-based biotech Amunix to access the XTEN half-life extension technology.

The platform uses hydrophilic, unstructured polypeptides which can be recombinantly fused or chemically conjugated to other peptides, proteins and small molecules, and - for haemophilia factor products - helps retain procoagulant activity, reducing infusion frequency and helping to protect patients from bleeding.

The new company plans to use this tech to accelerate the development of bispecific antibodies and hemophilia-related gene therapy programmes into the clinic over the next year.

Gene therapy

Biogen has several gene therapy programmes in the preclinical stage for haemophilia which will be transferred to the new company.

While Cox said the therapies “have a long way to go,”​ Biogen’s CEO George Scangos said such technologies would be important for both companies.

“We believe gene therapy has the potential to be a major therapeutic modality in the future, not only for haemophilia and other monogenic disorders but also for more complex disorders.”

Related topics: Drug Delivery, Mergers and Acquisitions

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