The companies will use Evotec’s induced pluripotent stem cell (iPSC) platform to develop the treatments. IPSC involves reprogramming body cells to act like embryonic stem cells. The reprogramming gives other cells the ability to regenerate or repair damaged and diseased tissue, as stem cells do.
Evotec has worked for five years with the Harvard Stem Cell Institute to develop its iPSC platform, which screens potential treatments in disease models derived from humans. A key goal is to see widespread healthcare-industry use of the screening devices.
The collaboration with Harvard started in 2013 with Professors Kevin Eggan, PhD, and Lee Rubin, PhD, trying to identify compounds that prevent or decrease the loss of motor neurons associated with amyotrophic lateral sclerosis (ALS). Their work has been a major underpinning of Evotec’s platform.
Additional features of the platform have come from a decade-long Huntington’s-disease partnership between Evotec and the CHDI Foundation.
“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 and is expected to lead to the discovery of more disease-relevant drug candidates but also more focused clinical development paths,” said Dr Cord Dohrmann, Evotec’s chief scientific officer. “This is particularly true for neurodegenerative diseases, a field that has proven intractable as novel therapeutics for Alzheimer’s disease, Parkinson’s disease, and motor neuron disease have largely failed.”
The five-year agreement between the companies requires Celgene to give Evotec an up-front payment of $45 million. Other terms could give Evotec up to $250 million in royalties and additional payments.
Celgene will hold global rights on programs that Evotec develops from Celgene’s compound library. The agreement also gives Celgene the right to use Evotec’s iPSC platform to screen compounds from Celgene’s proprietary CELMoD(R) library that could be used in neurodegenerative-disease treatment.
“Celgene perfectly complements and accelerates our business model and vision in bringing first-in-class therapeutics to patients with neurodegenerative diseases, where the burden for society is increasing dramatically,” “Dr. Werner Lanthaler, chief executive officer of Evotec, said in a press release.
“Recent breakthroughs in our understanding of the mechanism of action of the CELMoD(R) library may enable the discovery of other related compounds that can direct the degradation of proteins known to be neurotoxic,” said Dr. Rupert Vessey, executive vice president and head of Celgene’s research and early development program.”Screening for this activity in highly controlled cell-based screens developed by Evotec represents an excellent initial approach for drug discovery in neurodegenerative disorders.”