Drug development is expensive, can take years to complete and doesn't guarantee a novel therapy for treating disease.
But consumer genetic testing company 23andMe is forging ahead to leverage its massive database of genetic data for research and to make and sell its own therapies.
Anne Wojcicki, 23andMe's CEO and co-founder, has often said the company's mission, from the beginning, is to help people "access, understand and benefit from the human genome."
Using genetic data based on the company's 12 million genotype customers and billions of phenotypic data points gives 23andMe a distinct competitive advantage for its therapeutics business, Kenneth Hillan, 23andMe’s head of therapeutics, said Thursday during the virtual Fierce JPM Week event.
"We talk about it as our secret power. It really is that statistical power, the size and scale of our databases, and using genetics to see things in terms of drug targets within diseases that others just can't see," he said.
"What's really underappreciated about the 23andMe databases is that we get to look not just at the genetics and a single indication, but we get to look at genetics across 1,200 or so different indications," he said. "That kind of data provides an enormous advantage, both in discovery but also in translation and not just within the research setting, but also when we move things into the clinic in terms of really matching our drug targets with patients. And, ultimately, moving those forward to the market in a way that we hope will maximize benefit while minimizing risk for patients."
WATCH THE EXECUTIVE INTERVIEW ON-DEMAND
Earlier this month, 23andMe announced it started clinical trials of its first drug developed entirely in-house, a cancer immunotherapy aimed at treating solid tumors. It's the second cancer drug the company has advanced into human trials, following an immuno-oncology therapy developed through a partnership with pharmaceutical giant GlaxoSmithKline.
GSK also exercised its option to extend a partnership with 23andMe. The company will receive a one-time $50 million payment as part of that agreement.
That collaboration has resulted in more than 40 genetically validated drug discovery programs in the GSK portfolio.
The company is betting its approach to drug development harnessing the "power" of its database will pay off.
"The first premise is that you start with a target that's validated with human genetics, as all of our targets are, and there is published evidence from multiple sources indicating that will double or even potentially triple the overall probability of success," Hillan said. "In an industry where an average of only maybe 1 in 10 to 1 in 14 programs makes it, when you double the probability of success, the impact of that actually on a portfolio is huge."
The company went public in June via a merger with Richard Branson's blank check company. The company raised nearly $600 million in gross proceeds through the transaction. It had about $700 million in cash on hand as of Sept. 30.
"We're interested in bringing forward meaningful medicines to patients that will help to transform their life. It's an expensive endeavor, but with the company going public and with the GSK collaboration, it allows us to carry forward a portfolio and it will allow us to make very data-driven decisions about the programs we continue versus those that we stop. We're really leveraging human genetics to increase that probability of overall success," Hillan said.
RELATED: 23andMe jumps into telehealth, prescription drug delivery with $400M buyout of Lemonaid Health
Going public has given 23andMe the financial firepower to make strategic investments, such as buying Lemonaid Health, a virtual care and pharmacy provider, in October in a $400 million deal.
The company also has pushed forward on the consumer side of its business. It recently tallied up its third FDA clearance for a genetic health risk report, this one to identify whether a biomarker for prostate cancer is present in a user’s DNA. To date, 23andMe’s trio of direct-to-consumer cancer risk tests are the only ones cleared by the FDA for use without requiring a prescription.
In the realm of drug discovery, 23andMe has set its sights on immuno-oncology therapies. "It's one area where we think our data lends itself to identifying novel targets," Hillan said.
He added, "Some examples of diseases in immunology include systemic lupus erythematosus and systemic sclerosis." The company is currently recruiting people for a research study focused on better understanding the underlying genetic and environmental factors that contribute to systemic sclerosis.
RELATED: 23andMe snags 3rd FDA nod for cancer risk test, this time for prostate cancer
But the company's ambitions may extend beyond that.
Hillan said 23andMe also is focused on areas of "high unmet medical need" as well as cardio-metabolic diseases.
"We just have an enormous amount of data. We're really looking to take advantage of that data in some specific indications, for example, Type 2 diabetes and those programs are at an earlier stage," he said.
Hillan, who previously worked at biotech companies before joining 23andMe in 2019, sees the promise of the company's ability to marry the consumer, research and therapeutics sides of the business.
"Drug discovery and development is a very slow and inefficient process. I'm excited about being able to use this data to move with greater agility and more nimbly and more quickly so that ultimately we can get therapies moving forward and into the clinic to test if these have the potential to become medicines that can really help patients," he said.