Evotec, Celgene partner to identify neurodegenerative disease candidates

Initial disease areas of focus will include Amyotrophic lateral sclerosis, Alzheimer's disease, Parkinson's disease, and multiple other neurodegenerative disorders.

Evotec has built an industrialised iPSC infrastructure that represents one of the largest and most sophisticated iPSC platforms in the industry.

Evotec's iPSC platform has been developed over the last five years with the goal to industrialise iPSC-based drug screening in terms of throughput, reproducibility and robustness to reach the highest industrial standards.

This effort was enabled by a research collaboration and licence agreement with Harvard University involving world-leading scientists at the Harvard Stem Cell Institute.

In particular, a collaboration termed CureMotorNeuron that was initiated in 2013 with the laboratories of Professors Kevin Eggan, PhD, and Lee Rubin, PhD, resulted in significant contributions to the platform.

Additional aspects of the platform were built up through Evotec's more than 10-year collaboration with the CHDI Foundation in the field of Huntington's disease.

Under the terms of the agreement, Evotec will receive an upfront payment of $45m. Celgene holds exclusive options to in-license worldwide rights to Evotec programmes developed from the company's compound library.

Evotec may be eligible to receive up to $250m in milestones as well as up to low double-digit royalties on in-licensed programmes.

As part of the collaboration, Celgene may also elect to screen compounds from its proprietary CELMoD(R) library using Evotec's iPSC platform to evaluate activity in models of neurodegenerative diseases. The initial term of the collaboration is five years.

Evotec CEO Dr Werner Lanthaler said: "We are very excited about the opportunity to collaborate with Celgene, a medical innovation leader in the industry. 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."

Celgene EVP and research and early development president Dr Rupert Vessey said: "We are very pleased to enter into our first neurodegeneration collaboration with Evotec and look forward to the screening of their compound libraries using their proprietary iPSC platform.

“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. Screening for this activity in highly controlled cell-based screens developed by Evotec represents an excellent initial approach for drug discovery in neurodegenerative disorders."

Evotec chief scientific officer Dr Cord Dohrmann said: "The fact that many promising drug candidates fail during clinical development highlights the limited predictive and translational value of pre-clinical disease models commonly used during the drug discovery process.

“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 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."