Celgene pushes into brain diseases with a little help from Evotec

Celgene's has made no secret of its plans to expand beyond its heartland in cancer, and it revealed another part of the plan yesterday. Neurodegenerative diseases are now in its sights, thanks to a five-year alliance with German biotech Evotec. 

Ever ambitious, Celgene is taking aim at some of the biggest targets, with Alzheimer's, Parkinson's and amyotrophic lateral sclerosis (ALS)—amongst others—all on the hit list. 

In the last few years Celgene has already moved beyond its main focus in hematological cancers, with the addition of Abraxane (paclitaxel protein-bound) for solid tumors and psoriatic arthritis treatment Otezla (apremilast). And it has been steadily acquiring pipeline projects outside cancer, including ozanimod for multiple sclerosis and mongersen (GED-0301) for Crohn's disease.

Under the terms of the new deal, Celgene is paying Evotec $45 million upfront to tap into the firm's induced pluripotent stem cell (iPSC) platform, a platform which can be used to create models of human diseases for drug screening purposes. Evotec also stands to receive up to $250 million in milestone payments plus royalties on each drug candidate that Celgene chooses to in-license.

The collaboration will draw on collaborative research between Evotec and scientists at the Harvard Stem Cell Institute focused on identify compounds that prevent or slow down the loss of motor neurons, the key characteristic of ALS. 

That three-year-old project involves using iPSCs harvested from ALS patients to create assays against which compounds could be screened, and in the meantime the platform has been extended to include assays for other neurodegenerative disorders, including Huntington's disease.

"The use of patient-derived disease models for drug screening represents a paradigm shift as it places the testing of human disease relevance at the front end of the drug discovery process," according to Evotec's chief scientific officer Dr Cord Dohrmann.

He said many promising drug candidates fail during clinical development because of the "limited predictive and translational value" of currently-used preclinical disease models.

Evotec's compound library will initially be used for the screens, but Celgene said it will also be able to screen compounds from its own CELMoD library.

"Recent breakthroughs in our understanding of the mechanism of action of the CELMoD library may enable the discovery of other related compounds that can direct the degradation of proteins known to be neurotoxic," commented Celgene's president of research and early development Dr Rupert Vessey.

The latest deal comes as contract research and drug discovery specialist Evotec is going through a purple patch, with revenues rising 30 per cent in the first nine months of the year thanks to hitting R&D milestones in partnerships with Bayer, Boehringer Ingelheim and Padlock (now part of Bristol-Myers Squibb).

The company has also signed a number of new deals over the past few months, including an antibiotic development partnership with U.K. consortium Antibiotics Research UK, and has been adding to its platforms with hot technologies such as CRISPR/Cas9