Recursion gets FDA approval to begin phase 1 trials of AI-discovered cancer treatment

AI drug pioneer Recursion Pharmaceuticals said Wednesday that one of its experimental treatments hit a key milestone.

Recursion was able to use its artificial intelligence-enabled drug discovery platform to identify an area of biology to target for the treatment of solid tumors and lymphoma, match it with a drug candidate and move all the way to gaining regulatory approval to begin studies in less than 18 months.

“We think that’s a really exciting proof point, not only for us as a company, but I think for the techbio industry as well,” said CEO and co-founder Chris Gibson, in an interview with CNBC.

The Food and Drug Administration cleared the investigational new drug application for a phase 1/2 clinical trial of an experimental drug candidate known as REC-1245. The company said the potential market for this treatment could be more than 100,000 patients in the U.S. and European Union.

The trial will evaluate the safety and tolerability of REC-1245 and will begin in the fourth quarter of this year. The phase 1 data from the dose-escalation portion of the study could be completed by the end of next year, the company has said.

The drug will target RBM39, which Recursion said appears functionally similar to a well-known but hard to target marker known as CDK12 to treat advanced biomarker-enriched cancers such as ovarian, prostate, breast and pancreatic cancers.

“I think what’s really exciting about this particular program of Recursion is that this small molecule and novel target came out from essentially a Google-search equivalent, from this giant map of biology that we’ve already built,” Gibson said, referring to the massive datasets that Recursion has created over the past 11 years.

“This is the first program that’s come of that,” he continued. “It’s the first program that really is leveraging many of these new tools that we’ve built in one unit.”

There is much hope that artificial intelligence will be able to significantly speed up drug discovery and make it less costly by eliminating some of the time-consuming trial-and-error as drug candidates are screened and selected. But investors have been wanting to see that the reality can live up to the hype.

Recursion, which counts Nvidia among its investors, has seen its shares fall 38% in 2024. But the stock is still more than 60% below a 52-week high it hit in late February.

The company is planning to merge with fellow AI-drug discovery company Exscientia, which will allow it to harness even more data. The deal is expected to close early next year.

The majority of analysts rate Recursion shares a hold, but two analysts do have a buy rating on the stock, according to FactSet. The average analyst price target of $10.14, implies a 64% return.