Science Pool

The challenge of neurodegenerative diseases

In the context of an aging population, neurodegenerative conditions such as Parkinson´s disease or Alzheimer´s disease have become a major health problem in Western countries.
The global market size for neurodegenerative diseases drugs was estimated at USD 35 billion in 2018 and is projected to reach USD 63 billion by the end of 2026, exhibiting a CAGR of 7.2% (source: Fortune Business Insights).

Developing treatments for neurodegenerative diseases comes with a number of challenges: The underlying causes, diseases mechanisms and progression of disorders affecting the central nervous system have not yet been fully understood. This results in much higher drug failure rates as compared to other fields, making the development of novel therapeutics for neurodegenerative disease very time and cost intensive. Approved drugs only offer short-term improvement of the patients’ symptoms, so there is a huge unmet medical need for innovative therapies that slow down or ideally revert disease progression.

New treatment approaches urgently needed

In response to the high attrition rates, R&D efforts to unveil the mechanism of neurodegenerative diseases have gained increasing attention. Evotec has a strong commitment to developing novel therapeutic options in neurodegeneration for more than a decade. In our long-standing collaboration with Celgene (now Bristol Myers Squibb) we have set out to establish human induced pluripotent stem cell-based disease models to discover novel disease-modifying treatments for a broad range of neurodegenerative diseases.

What are induced pluripotent stem cells?
Induced pluripotent stem cells (also known as iPS cells or iPSCs) are a type of pluripotent stem cell that can be generated directly from the patient’s somatic cells through reprogramming. They can be propagated indefinitely and give rise to almost every cell type in the body (such as neurons, heart, pancreatic and liver cells) thereby presenting unprecedented opportunities to model human disease pathology.

Over the past decade, Evotec has built an industrialised iPSC infrastructure that represents one of the largest and most sophisticated iPSC platforms in the industry. It comprises multiple different cell types to investigate disease-relevant phenotypes, translatable biomarkers and therapeutic targets. Evotec’s iPSC platform has continuously been optimized for increased throughput, reproducibility and robustness to provide large-scale cultures of iPSC derived cells for disease modeling, drug discovery and cell therapy. Moreover, it is closely connected with our PanOmics and PanHunter platforms to determine molecular disease signatures that may aid in stratification of patients and clinical trial success.

Evotec’s iPSC platform has been developed in collaboration with top-tier academic and industrial partners such as the CHDI Foundation, the Harvard Stem Cell Institute, Centogene, CENSO Biotechnologies (now Axol Bioscience), Fraunhofer IME-SP, Reprocell, Pancella, the University of Tübingen – and more recently - Sartorius and Curexsys. The Company´s goal is to build a proprietary pipeline of first-in-class therapeutic agents for a broad range of different diseases with high medical need, including neurodegenerative disorders, to ultimately extend and improve the lives of millions of patients and their families worldwide.

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For more than a decade, Evotec has been closing strategic R&D collaborations in the area of induced pluripotent stem cells (iPSC), both with academic and industry partners.

The scope ranges from broad, long-term alliances to targeted research-driven collaborations. These partnerships support Evotec’s growing iPSC activities, strengthen the capabilities, and thus comprise a strong foundation for success within Evotec’s iPSC Lighthouse.

A brief overview of key references for iPSC partnerships is provided below.

Industry partnerships

In 2006, Evotec and CHDI Foundation, Inc. (“CHDI”) closed a strategic collaboration to advance drugs for the treatment of Huntington´s disease. The partnership builds on Evotec’s integrated neuroscience platform and its iPSC platform, among others, and was extended in 2018.

In 2016, Evotec and Celgene (now Bristol Myers Squibb) signed a broad R&D collaboration to develop disease-modifying treatments for neurodegenerative disorders based on Evotec's unique iPSC platform. The platform allows for systematic drug screening in patient-derived disease models. The partnership was expanded to include additional cell lines in 2018 and new cell types in 2019. Following the acquisition of Celgene by Bristol Myers Squibb, the agreement with Evotec was again expanded to further broaden the number of cell lines in 2020.

In 2020, Evotec formed an alliance with Sartorius and Curexsys to advance an iPSC-based exosome approach. The collaboration combines Evotec’s iPSC platform with Curexsys’ proprietary exosome isolation technology, while Sartorius will support Curexsys in setting up a GMP-compliant and scalable manufacturing platform.

Scientific collaborations

The first iPSC partnership was a collaboration with the Harvard Stem Cell Institute ('HSCI') in 2013 to identify compounds which prevent or halt the loss of motor neurons, a key symptom of amyotrophic lateral sclerosis ('ALS').

In 2017, Evotec entered into a research collaboration with the Center for Regenerative Therapies TU Dresden ("CRTD") to discover novel small molecule candidates for retinal diseases. Gola of the collaboration is to combine CRTD's expertise in stem cell-based retinal disease modelling with Evotec´s iPSC technology platform to generate promising drug candidates for potential clinical development.

In 2018, Evotec and Centogene signed an agreement for a global drug discovery collaboration to develop novel small molecules in rare hereditary metabolic diseases, which are generated by a joint high-throughput platform.

The collaboration was expanded into Gaucher´s disease in 2020, leveraging Evotec’s iPSC platform and broad drug discovery and development capabilities and Centogene´s proprietary rare disease platform, including iPSC lines, to generate novel treatment approaches for this orphan drug indication.

In 2021, Evotec and the Medical Center Hamburg-Eppendorf (“UKE”) signed a partnership for the development of a novel, innovative first-in-class cell therapy based on Engineered Heart Tissue for the treatment of heart failure. The goal is to produce human, clinical-grade heart muscle cells (cardiomyocytes) for implantation.

Evotec is continuously looking to expand its iPSC portfolio through industry and academic partnerships both within existing disease areas but also to expand into new disease areas. Reach out to us for questions around collaborations and partnerships.

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