Merck and Co. has acquired Calporta Therapeutics, a developer of small-molecule agonists for treating Niemann-Pick C disease, a rare genetic disorder characterized by an inability of the body to transport cholesterol and other fatty substances inside of cells, and other lysosomal storage diseases, for up to $576 million.

The Community of Innovation (COI Pharmaceuticals), established by Avalon Ventures, a venture-capital firm, announced the acquisition earlier this month (November 2019).

Calporta is developing 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 body’s way of cleaning out damaged cells in order to regenerate newer, healthier cells. The lysosome is a cellular compartment involved in the breakdown 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 have been shown to be toxic to the cell. Activating TRPML1 signaling with small molecules could re-establish lysosomal processes and restore cellular function, according to information from Calporta. 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.

Merck acquired Calporta for a total potential consideration of up to $576 million, including an upfront payment and contingent milestone payments.

Calporta Therapeutics was part of COI Pharmaceuticals, a venture-pharma entity. This Community of Innovation (COI) was established in 2013 to support the portfolio companies of Avalon Ventures. COI provides the intellectual capital to launch companies, a fully equipped R&D infrastructure, and industry mentors to help entrepreneurial scientists. COI is located in La Jolla, California. 

Source: COI Pharmaceuticals