RARE Daily

Amathus and Merck Enter into Neurodegenerative Disease Pact

March 17, 2021

Rare Daily Staff

Amathus Therapeutics has entered into a strategic collaboration with Merck to develop novel small molecule therapeutic candidates for neurodegenerative diseases.

Amathus focuses on small molecule modulators of organelle specific chaperones across a variety of genetically defined diseases including Parkinson’s disease and autosomal recessive Polycystic Kidney Disease (ADPKD), a rare genetic disorder characterized by the formation of fluid-filled sacs (cysts) in the kidneys. 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.

“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,” said Edward Holson, president and CSO of Amathus. “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 their 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.

Photo: Edward Holson, president and CSO of Amathus

Stay Connected

Sign up for updates straight to your inbox.