Merck Snaps Up Preclinical Neurodegenerative Asset in $576 Million Buy


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Merck plunked down $576 million to acquire San Diego-based Calporta to gain access to preclinical TRPML1 agonists that are seen as potential treatments for neurodegenerative disorders such as Alzheimer’s or Parkinson’s.

Calporta was launched as a collaboration between GlaxoSmithKline and Avalon Ventures. The company is located at COI Pharmaceuticals the Community Of Innovation site established by Avalon Ventures. GSK had the option to acquire Calporta as part of that collaboration but opted against that earlier this year, which opened the door to Merck.

Calporta Therapeutics focuses on the development of selective small-molecule agonists to TRPML1 (transient receptor potential cation channel, mucolipin subfamily), which is believed to play a role in lysosomal function by promoting autophagy and lysosomal exocytosis. The lysosome is a cellular compartment involved in the breakdown and recycling of cellular waste products, such as fats, proteins and other macromolecules, the company said. Damaged TRPML1 function has been implicated in several pathological conditions, including Niemann-Pick C Disease, as well as muscular dystrophy and Alzheimer’s disease. 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. It is believed that activating TRPML1 signaling with small molecules could reestablish lysosomal processes and restore cellular function.

Jay Lichter, the managing director of Avalon Ventures and CEO of COI Pharmaceuticals said that TRPML1 was identified in 2014 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,” he said in a statement.

Fiona Marshall, vice president, neuroscience discovery Merck Research Laboratories, said in a brief statement that there is increasing evidence that shows the accumulation of toxic proteins are a common mechanism in neurodegenerative conditions such as Parkinson's disease, amyotrophic lateral sclerosis (ALS) and Alzheimer's. Marshall said Merck is excited about conducting its own research to evaluate the potential of TRPML1 agonists as a means to “activate a natural clearance mechanism the brain employs to clear toxic proteins.”

Under terms of the deal, Merck will pay up to $576 million for Calporta, including an undisclosed upfront payment and contingent milestone payments.

Calporta Chief Executive Officer Sanford J. Madigan called the deal with Merck an “important milestone” toward the development of novel therapeutics that could benefit millions of people with degenerative disorders that are caused by toxic accumulation of proteins, fats, or other cellular macromolecule. Madigan added that he was proud of the Calporta team for its diligence in advancing the preclinical research toward human trials.

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