Evotec and Celgene Partner to Screen Novel Therapies for ALS, Other Diseases

The pharmaceutical companies Evotec and Celgene will collaborate on a drug discovery and development program to identify and develop novel disease-modifying therapies for several neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS).

The partnership will allow the screening of drug compounds developed by Celgene using Evotec’s platform of induced pluripotent stem cells (iPSCs), a type of immature stem cell that can be generated directly from adult cells. Pluripotent stem cells can propagate indefinitely and form any other cell type in the body, replacing those lost to disease.

According to the agreement, Evotec will receive $45 million initially and Celgene holds exclusive options to license rights to the programs developed by Evotec. Celgene also can screen compounds from its proprietary CELMoD(R) library using Evotec’s iPSC platform to assess their effect on neurodegenerative diseases.

Evotec may receive additional payments up to $250 million in milestone payments and double-digit royalties on licensed programs.

“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,” Rupert Vessey, Celgene’s president of research and early development, 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. Screening for this activity in highly controlled cell-based screens developed by Evotec represents an excellent initial approach for drug discovery in neurodegenerative disorders,” he said.

Evotec developed its iPSC platform to industrialize drug screening using these cells in a fast, reproducible way and to achieve the highest industrial standards. Significant contributions to the platform have been made possible through previous collaboration with researchers from the Harvard Stem Cell Institute for the CureMotorNeuron project.

“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,” said Cord Dohrmann, chief scientific officer of Evotec. “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 [such as ALS] have largely failed,” he said.

“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,” Dohrmann added.