23andMe has received a $300 million equity investment disclosed today by GlaxoSmithKline (GSK), under a collaboration designed to use genetic data to identify new drug targets, and thus accelerate development of new therapies for 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—which has overhauled its R&D effort over the past year—said today its partnership with 23andMe reflects a new strategy focusing on science related to the immune system, the use of genetic data, and investments in advanced technologies.

The pharma giant disclosed one potential new therapy it plans to study with 23andMe—a small molecule Leucine-Rich Repeat Kinase 2 (LRRK2) inhibitor that is in preclinical development as a potential treatment for Parkinson’s disease, having been implicated in the autosomal dominant form of the disorder.

The companies said they expect to make progress on the LRRK2 inhibitor by applying 23andMe’s base of customers who know about their LRRK2 variant status after having their genetic data collected and analyzed by 23andMe.

The inhibitor of the LRRK2 mutated protein is among existing early-stage programs that both companies said they plan to study in order to begin work quickly. The mutation appears in only 1% to 2% of people with Parkinson’s, the second-most common neurodegenerative disease with about 10 million people affected worldwide, about 1 million of them in the U.S. according to the Parkinson’s Foundation—including 250 customers of 23andMe.

“The idea that there’s 250 patients in the 23andMe database, 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. . .We can really speed up the development time as well, making us very excited about the opportunity of pursuing this,” Hal Barron, M.D., GSK’s CSO and President, R&D, said this morning during a conference call with reporters.

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 treatments and cures for diseases.”

Last year, GSK terminated a Phase I observational study (NCT01424475)  designed to assess the phenotypic neurocognitive 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.

Finding patients for that study proved to be a challenge, GSK disclosed on ClinicalTrials.gov upon terminating the study in May 2017: “Following extensive efforts to increase recruitment, it will not be possible to complete the study protocol within a reasonable time.”

‘Beginning to Discuss the Details’

Answering a Clinical OMICs question during the conference call, Richard Scheller, Ph.D., 23andMe’s CSO and head of therapeutics, said his company and GSK have yet to nail down the early-stage programs.

“We’re just beginning to discuss the details around those potential collaborations,” Dr. Scheller said. “We have a number of drug discovery projects that we’ve started in the areas of autoimmune, cancer immunotherapy, cardiovascular disease, osteoarthritis, liver disease, and so on.”

Dr. Scheller added that the company has not publicly disclosed its therapeutic targets for those programs: “Just today, we are disclosing them to GSK to begin the discussions of which might enter into a collaboration.”

“I’m looking forward to seeing that data,” Dr. Barron added, chuckling with Dr. Scheller.

The two were colleagues at Genentech, where Dr. Barron was once SVP of development and chief medical officer. He later served as president of R&D at Calico (California Life Sciences LLC), a Google-backed company launched in 2013, until joining GSK effective January 1. Dr. Scheller served as Genentech’s evp of research and early development until 23andMe appointed him in 2015 to lead its therapeutics group.

Since then, Dr. Scheller said, the group has launched several drug discovery programs and built out 23andMe’s capabilities for finding drug targets.

“We have a lot of information about immune phenotypes and immune dysregulated diseases, including psoriasis, osteoarthritis, way-way too many to even comment on. We also gain insights into the immune system from asking questions like, ‘How do you respond to poison oak or a mosquito bite?’ and so on,” Dr. Scheller said.

“One of the most interesting sets of observations that we’ve made has to do with potential cancer immunotherapy targets, when we find a certain group of patients have an increase in autoimmunity and a decrease in certain types of cancer, suggesting that these variants are in genes that may be good targets for cancer immunotherapy,” he added. “We have a vast amount of information on human variants and the response of the human immune system to these variants, and we look forward to working with GSK to exploit this data.”

Revamping R&D Strategy

GSK has overhauled its R&D pipeline, last year terminating more than 30 clinical and preclinical programs deemed by the company as “unlikely to generate sufficient returns.

GSK finished the second quarter with total operating profit of £779 million ($1.023 billion) on revenue of £7.31 billion ($9.6 billion). In announcing Q2 results today, GSK said it currently has over 40 new molecular entities in its pharmaceutical pipeline, with several candidates expected to reach the market over the next two years, including two combination treatments for HIV—dolutegravir+lamivudine and cabotegravir+ripilvirine—as well as the oncology treatment GSK 2857916, a BCMA antibody-drug conjugate.

Dr. Barron noted that GSK has had four approvals of new treatments in the past 10 months, with a fifth anticipated soon. He said the company needs to focus its R&D on the immune system and other therapeutic areas where targets can be developed to treat multiple diseases.

GSK’s revamped R&D strategy, he said, would emphasize “science related to immune system, as well as the use of human genetics and functional genomics to identify promising targets, while exploring other technologies that we think will be very, very helpful for that approach, which includes machine learning and other advanced analytics.”

“We think the combination of science and technology will generate new insights and direct us to many exciting targets, as well as help us focus to ultimately have a more exciting pipeline and improve our probability of success for the medicines that enter clinical testing,” Dr. Barron added.