Rare Daily Staff

Merck has acquired Calporta Therapeutics, a biotech developing selective small molecule drugs targeting lysosomal storage and neurodegenerative disorders, including Alzheimer’s disease and Parkinson’s disease.

Merck acquired Calporta for total potential consideration of up to $576 million, including an upfront payment and contingent milestone payments, said COI Pharmaceuticals, established in 2013 as an early venture-pharma entity that provides the intellectual capital to launch companies, a fully-equipped R&D infrastructure and industry mentors prepared to leverage their expertise to help entrepreneurial scientists succeed. Calporta operates under the COI family of companies.

Calporta develops selective small molecule agonists to TRPML1, (transient receptor potential cation channel, mucolipin subfamily), an important lysosomal ion channel and a known regulator of autophagy. The lysosome is a cellular compartment involved in the break down and recycling of cellular waste products, such as fats, proteins, and other macromolecules. Alterations in TRPML1 function have been implicated in several neurodegenerative diseases and dystrophies, where intracellular accumulation of proteins or fats has been shown to be toxic to the cell. Activating TRPML1 signaling with small molecules could reestablish lysosomal processes and restore cellular function. Calporta’s preclinical stage TRPML1 agonists are being evaluated for their potential to treat various lysosomal storage and neurodegenerative disorders, including Alzheimer’s and Parkinson’s disease.

“We review thousands of research papers annually for early-stage, breakthrough discoveries that have important therapeutic potential,” said Jay Lichter, managing director of Avalon Ventures and president and CEO of COI Pharmaceuticals. “As early as 2014, our scientists identified TRPML1 as an important target for improving lysosomal function. We saw the potential to treat a number of diseases by activating this ion channel, and we launched Calporta in early 2015. 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.”

“Increasing evidence points to the accumulation of toxic proteins as a common mechanism in neurodegenerative conditions such as Parkinson’s disease, amyotrophic lateral sclerosis and Alzheimer’s,” said Fiona Marshall, vice president, neuroscience discovery Merck Research Laboratories. “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.”

Photo: Jay Lichter, managing director of Avalon Ventures and president and CEO of COI Pharmaceuticals

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