Merck Acquisition Expands Neurodegenerative Research, Treatments

Merck & Co. has purchased Calporta, a developer of neurodegenerative and lysosomal storage disorder treatments, for up to $576 million.

Calporta’s preclinical stage TRPML1 agonists—a substance that initiates a physiological response when combined with a receptor—are being studied for their potential to treat Niemann-Pick C Disease and other lysosomal storage diseases. The company is also studying how TRPML1 may potentially treat Alzheimer’s disease and Parkinson’s disease, among other neurodegenerative disorders.

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“Increasing evidence points to the accumulation of toxic proteins as a common mechanism in neurodegenerative conditions, such as Parkinson’s disease, amyotrophic lateral sclerosis (ALS), and Alzheimer’s,” says Fiona Marshall, vice president, neuroscience discovery in a recent statement. “We look forward to conducting further research to evaluate the potential of TRPML1 agonists to activate a natural clearance mechanism the brain employs to clear toxic proteins.”

The acquisition was made possible from a from a collective venture, COI Pharmaceuticals, created by Avalon Ventures and GlaxoSmithKline (GSK) to help develop 10 new life science startups in San Diego. Founded in 2013 COI previously funded each company launched with $10 million; Calporta received its $10 million when it launched in 2015.

“This agreement with Merck is an important milestone towards the rapid development of a novel therapeutic approach that could help millions of people with degenerative disorders caused by toxic accumulation of proteins, fats, or other cellular macromolecules,” says Sanford J. Madigan, CEO of Calporta and senior vice president. “I am proud of the Calporta team for their expertise, execution, and diligence in swiftly advancing a new scientific breakthrough toward the clinic.”

Alterations in TRPML1 function have been found in several neurodegenerative diseases and dystrophies, where intracellular accumulation of proteins or fats were shown as toxic to the cell. Activating TRPML1 with small molecules could help reestablish lysosomal processes and restore cellular function.

Jay Lichter, managing director of Avalon Ventures and president and CEO of COI Pharmaceuticals says that after reviewing thousands of research papers annually for early-stage, breakthrough discoveries with therapeutic potential, scientists have identified TRPML1 as an important target for improving lysosomal function in patients. “We saw the potential to treat a number of diseases by activating this ion channel,” says Lichter. “Now four years later, we have an agreement with Merck, an industry leader in biopharmaceutical research and development, which is key to advancing these therapies to clinical trials and patients.”