Clinical OMICS

SEP-OCT 2018

Healthcare magazine for research scientists, labs, pathologists, hospitals, cancer centers, physicians and biopharma companies providing news articles, expert interviews and videos about molecular diagnostics in precision medicine

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8 Clinical OMICs September/October 2018 www.clinicalomics.com News 2 3andMe has received a $300 million equity investment from GlaxoSmithKline (GSK) under a collaboration designed to use genetic data to identify new drug targets, and thus accelerate development of new therapies from both companies. The collaboration is intended to combine 23andMe's expertise in gathering and analyzing data from the 5 million customers in its research platform—which the company says includes the world's largest consented, re-contactable database—with GSK's know-how in R&D and commercialization. GSK has disclosed one potential new therapy it plans to study with 23andMe—a small-molecule Leucine-Rich Repeat Kinase 2 (LRRK2) inhibitor now in preclinical devel- opment for Parkinson's disease, having been implicated in the autosomal dominant form of the disorder. The inhibitor of the LRRK2 mutated protein appears in only 1% to 2% of people with Parkinson's, the second-most common neurodegenerative disease affecting roughly 10 million people worldwide—1 million of them in the U.S. according to the Parkinson's Foundation. Two hundred-fifty customers of 23andMe reportedly have the disease. "The idea that there's 250 patients in the 23andMe data- base, most of whom have probably consented to research, and for whom there might be an available therapy, but would have taken maybe many, many years to conduct a trial, could be done significantly faster," Hal Barron, M.D., GSK's CSO and president, R&D, told reporters during a conference call. "We can really speed up the development time as well, making us very excited about the opportunity of pursuing this." Added Anne Wojcicki, CEO and co-founder of 23andMe: "With over 80% of customers consenting to participate in research, it's really clear that a lot of our customers want to play an active role in discovering and developing treat- ments and cures for diseases." Last year, GSK terminated a Phase I observational study (NCT01424475) designed to assess the phenotypic neuro- cognitive abnormalities of Parkinson's patients with the LRRK2 mutation—with the aim of identifying potential PD endpoints related to the LRRK2 mutation for future Phase I or II clinical trials of LRRK2 inhibitors. But finding patients proved to be a challenge. Only five were recruited, GSK disclosed on ClinicalTrials.gov in May 2017: "Following extensive efforts to increase recruitment, it will not be possible to complete the study protocol within a reasonable time." Strength in Numbers The prospect of much larger patient populations for future trials of the LRRK2 inhibitor and other candidates is attrac- tive to both companies, said Henry T. Greely, director of the Center for Law and the Biosciences at Stanford University, where he is also director of the Stanford Program in Neuro- science and Society. "GSK is not just paying $300 million to be able to pur- sue this Parkinson's trial, but other similar trials. That's a nice example of a potential payoff in terms of being able to Patient Investment GSK, 23andMe Forge $300 Million Alliance to Develop Drugs via Genetics By Alex Philippidis GSK's $300 million investment and collaboration with 23andMe comes as the company has refocused its drug development efforts and has increased its genomics partnerships. Jeff J Mitchell / Staff / Getty Images Don Murray / Stringer / Getty Images

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