Amathus Therapeutics signs strategic collaboration with Merck to Develop Treatments for Neurodegenerative Diseases

in Portfolio News

Cambridge, MA, 17, March 2021

Amathus Therapeutics announced today a strategic collaboration with Merck, known as MSD outside the United States and Canada, to develop novel small molecule therapeutic candidates for neurodegenerative diseases.

Edward Holson, President and CSO of Amathus added that Amathus will focus on exploiting genetically defined pathways and core pathologies in multiple disease indications, “the Amathus team has demonstrated activation of molecular chaperones is possible and the potential to address core mitochondrial dysfunction with specificity holds tremendous therapeutic potential. The Merck neuroscience team is an ideal partner to translate this novel approach into potential first-in-class, disease modifying treatments for patients with neurodegenerative diseases.”

Under the terms of the agreement, Amathus will be responsible for identifying and progressing novel chaperone activators through preclinical discovery. Merck has the option to acquire Amathus Therapeutics and its pipeline of mitochondrial targeted candidates for the treatment of neurodegenerative disorders and renal diseases. Amathus will receive an upfront payment from Merck and, upon Merck’s exercise of its option, will be eligible for milestone payments associated with the successful development of candidates in excess of $500 million per program. Upon exercise of its option, Merck will be solely responsible for clinical development and commercialization.

“We are excited to be working with the Amathus team to investigate the company’s novel mitochondrial targeted candidates for the treatment of neurodegenerative conditions,” said Fiona Marshall, Senior Vice President, Head of Discovery Sciences and Translational Medicine, Merck Research Laboratories. “External collaborations that supplement and complement our internal pipeline programs remain a cornerstone of Merck’s discovery strategy.”

About Amathus Therapeutics

Amathus Therapeutics is a biopharmaceutical company and leader in the field of small molecule modulators of organelle specific chaperones across a variety of genetically defined diseases including Parkinson’s Disease (PINK1) and Polycystic Kidney Disease (ADPKD). The company’s proprietary platform enables highly specific modulation of mitochondrial and endoplasmic reticulum chaperones to exploit cellular stress response pathways and rescue key cellular functions associated with disease. Amathus was founded by Sanofi Ventures based on licensed discoveries from the Icahn School of Medicine at Mount Sinai, Mount Sinai Health System faculty and the laboratory of Professor Yiannis A. Ioannou. Amathus Therapeutics is funded by Sanofi Ventures and the Dementia Discovery Fund. Mount Sinai and Mount Sinai faculty, including Yiannis A. Ioannou, have a financial interest in Amathus.


About DDF

The Dementia Discovery Fund (DDF) is a specialist venture capital fund that invests in projects and creates biotech companies to deliver high impact therapeutics for dementia - including Alzheimer’s Disease (AD), Parkinson’s Disease Dementia (PDD), Frontotemporal Dementia (FTD), Amyotrophic Lateral Sclerosis (ALS), and Huntington’s Disease (HD). By making meaningful and sustained investments which are actively managed, the DDF enables the development of therapeutics addressing one of the world’s largest unmet medical needs and the generation of significant returns for its investors. Established in 2015, it has raised funds from an influential group including leading pharmaceutical companies (Biogen, Eli Lilly and Co., GSK, Johnson & Johnson, Otsuka (Astex), Pfizer and Takeda), along with AARP, Aegon, Bill Gates, British Patient Capital, NFL Players Association, Quest Diagnostics, UnitedHealth Group, the UK Government’s Department of Health and Social Care, and the charity Alzheimer’s Research UK. The Fund is managed by SV Health Investors. Learn more at

About Sanofi Ventures

Sanofi Ventures is the corporate venture capital arm of Sanofi. Sanofi Ventures invests in early-stage biotech and digital health companies with innovative ideas and transformative new products and technologies of strategic interest to Sanofi. Among these areas are oncology, immunology, rare diseases, vaccines, potential cures in other core areas of Sanofi’s business footprint, and digital health solutions. For more information, visit


Media Contact:

Tandee Newman

Amathus Therapeutics, Inc.


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