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23andMe Looks to Advance Drug Discovery Pipeline as GSK Partnership Winds Down

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A previous version of this story had an incorrect characterization of the 23andMe contract with GSK.

CHICAGO – A five-year exclusive drug discovery partnership between 23andMe and GSK will expire in July. At that time, 23andMe will be allowed to engage other partners to augment its discovery pipeline, though the firm can and has entertained discussions with potential new collaborators.

Jennifer Low, head of therapeutics development for the South San Francisco, California-based consumer genetic testing firm, said that the GSK collaboration has resulted in more than 50 programs "that reached the point where we started creating chemical matter" for potential future therapeutics.

The contract expiration does not signal the end of the 23andMe-GSK relationship.

In 2018, the companies partnered to use 23andMe's extensive genotype-phenotype database and base of customers willing to donate personal data to identify targets for personalized therapeutics. Four years later, the drug company opted to extend this exclusive collaboration for a fifth and final year, until July 2023.

GSK made a final payment of $50 million to 23andMe in October, but the drug company continues to hold a significant stake in 23andMe, as it made a $300 million investment at the start of the partnership five years ago.

Everything that GSK and 23andMe started together will continue beyond July. "Those teams will remain joint teams," Low said. There are milestones within the development process that require joint decisions and have opt-out opportunities.

So far, one compound from the collaboration has reached a Phase I clinical study: an immuno-oncology antibody targeting CD96. 23andMe said early last year that it elected to take a royalty option in the CD96 program, so GSK is fully responsible for the drug's development in later-stage clinical trials and will handle all development costs going forward.

Low said that 23andMe has opted out of several other programs it launched with GSK, also choosing the royalty route. She did not elaborate, citing the pipeline secrecy that is common for Big Pharma firms like GSK.

23andMe has publicly commented on only one other therapeutic program, 23ME-00610, an investigational monoclonal antibody targeting CD200R1 in patients with advanced solid tumors, to include indication-specific cohorts. 

The firm discovered CD200R1 as a new checkpoint prior to the GSK partnership. Low said that the drug company had the opportunity to opt into that program but chose not to, so the compound has been wholly owned by 23andMe from the start.

The investigational 23ME-00610 is a checkpoint inhibitor. "This isn't a PD1 add-on. It is hopefully a different checkpoint inhibitor that will have the opportunity to be broadly efficacious," Low said. She promised an update on that development at an upcoming scientific meeting, but did not offer details.

In November, 23andMe said that it is expanding its study of 23ME-00610 to evaluate monotherapy activity of the compound on new indications, including clear cell renal cell carcinoma; epithelial ovarian, fallopian tube, or primary peritoneal carcinoma; several neuroendocrine cancers; and microsatellite instability-high or tumor mutational burden-high (TMB-H) cancers that have not responded well to existing treatments. Shortly thereafter, company scientists presented a trials-in-progress poster for 23ME-00610 at the annual Society for Immunotherapy of Cancer meeting.

Based on preclinical testing, 23andMe said that the CD200R1-targeting compound "has the potential to restore the ability for both T cells and myeloid cells to kill cancer cells."

The therapeutics group has been around since 2015, and Low expressed optimism that the firm will be fine in the therapeutics arena after the end of the GSK exclusivity window, citing the potential of the massive 23andMe research database.

23andMe said in its 2023 second quarter financial release in November that it had genotyped 13.4 million people. The firm did not give an update in its Q3 report this month, but had been adding about 300,000 to 400,000 customers per quarter in fiscal 2022 and the early part of the current fiscal year.

While customers can opt out of joining the firm's research database, roughly 80 percent have chosen to participate in research, according to a company spokesperson. 23andMe combines its proprietary database with publicly available resources for data analysis and research.

Low said that the 23andMe database has helped the company build an immuno-oncology pipeline in a way that other commercial entities cannot.

For example, internal researchers queried the database for patients who had immune-related phenotypes, including autoimmune diseases as well such things as allergic reactions to insect bites. They then looked at those who had developed cancer.

"We saw that there was commonality of genes in these two big buckets and that the gene activity was going in the opposite direction," Low said. "It may be overactive for autoimmune and underactive for cancer."

The researchers then conducted "more traditional pharma biology," according to Low, looking at gene function to predict whether those with certain variants might respond to drugging.

"This is important because … there's hundreds of immuno-oncology targets and basically everything that's on an immune cell is being targeted," Low explained. But this additional information helps locate more specific genetic pathways to test compounds on.

Low said that CD200R1 was particularly interesting because the ligand, the receptor, and a downstream signaling protein for the receptor apparently had opposite effects on autoimmune diseases than they did for cancer. "It's one of the reasons why we think this is a really interesting target," she said.

According to Low, an earlier study from another company was unable to saturate the ligand using a slightly different mechanism. "We're much more optimistic we can saturate [the ligand and] the receptor and be able to potentially have clinical activity" because 23andMe is working from a much larger and richer genetic and phenotypic database.

23andMe is in the process of building a new business line called its genomic health service, facilitated by the company's $400 million acquisition of telehealth platform developer and telepharmacy services firm Lemonaid Health in 2021. The company is trying to gain a foothold in primary care with this acquisition by offering the ability to integrate genetic health risk information into care.

The company did not fulfill a request to interview with genomic health service leaders. 23andMe executives said in a conference call about the Q3 results that they would not provide updates in this area until midyear.

Low gave some generalities about how 23andMe is modernizing its technology to combine information from its genotyping database, public datasets, and the Lemonaid platform to, among other things, find genetic overlap and commonality in various diseases.

23andMe has publicly stated that it is happy genotyping with microarrays and has no plans to move into sequencing anytime soon. "It's just not really cost-effective to sequence everybody," Low said.

The company does give customers the option of banking their saliva samples for future testing beyond the original microarray genotyping.

23andMe has a series of US Food and Drug Administration (FDA) clearances for its pharmacogenetics reports that now fall under the firm's genomics health service. Low said that these reports do not have a major bearing on the therapeutics business, though there is some crossover.

The pharmacogenetics reports are specific to how certain enzymes process specific small-molecule drugs. "On the therapeutic side, we're taking a larger, more holistic view that we hope … we'll be able to apply more broadly to other genetic methods for predicting efficacy," Low said.

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