AI specialist Recursion says lead drug is safe, but efficacy less clear

 

• Recursion Pharmaceuticals on Tuesday said results from a mid-stage trial showed its lead experimental drug to be safe, the first readout in a series of important data disclosures for the AI drug discovery specialist.

• But the summary findings Recursion announced provide little indication of whether the drug, a treatment for a potentially dangerous kind of vascular malformation in the brain, might be effective. MRI scans of the brains of study participants given the highest dose showed a “trend” toward smaller lesions, Recursion said.

• Shares in Recursion fell by double digits Tuesday morning. The Salt Lake City-based company intends to submit its data for publication in a scientific journal, and to discuss plans for a follow-up clinical trial with the Food and Drug Administration “as soon as practical.”

This year has been big for Recursion, which claims its vast datasets, and the technology it uses to mine them, will allow the company to “turn drug discovery from sequential trial-and-error into a search problem.”

Recursion made a splash at the J.P. Morgan Healthcare Conference in January, pitching its approach to biopharmaceutical industry executives at an event it co-hosted with chip giant Nvidia. Then, in August, Recursion announced a deal to combine with Exscientia, an AI drug discovery rival that had ranked among the field’s most well resourced. The companies touted the potential of their combined drug pipeline, which they expect to deliver around 10 clinical trial readouts over 18 months.

Thursday’s announcement involves a drug compound Recursion selected with earlier iterations of its technology and then licensed. Dubbed REC-994, the drug is designed to treat a neurovascular condition known as cerebral cavernous malformation. Typically sporadic in nature, the condition develops as irregular bunches of small blood vessels. Leaking blood from these formations can cause seizures, headaches and potentially fatal hemorrhagic stroke.

While Recursion’s trial was a Phase 2 test, its main goals centered on safety. The company said its drug met those goals. The frequency and severity of adverse events after one year of treatment were similar across the placebo, 200 milligram drug dose and 400 milligram drug dose groups.

Efficacy evaluations were less clear. Along with the “trend” toward reduced lesion size, Recursion also reported that treatment with REC-994 led to a similar trend on MRI scans in the size of hemosiderin rings — iron-containing halos caused by blood leaking from malformed vessels.

Notably, an evaluation of patient- and physician-reported outcomes showed no improvement at the one-year mark.

In a statement provided by Recursion, Jan-Karl Burkhardt, the division head of cerebrovascular surgery at the University of Pennsylvania and the study’s lead investigator, described the results as an “impressive start” that “strongly supports the need for a future study.”

Recursion CEO Chris Gibson has made in recent remarks a point of emphasizing the company’s overall pipeline, rather than talking up the importance of specific readouts.

“There are going to be successes. There are going to be failures,” he told reporters at the JPM meeting, speaking generally of AI drug discovery. “People are going to make some predictions about the industry based on those early data points.”

Still, Tuesday’s readout and those that follow this year and early next will likely do much to shape investors’ views of whether Recursion’s technology is more effective than more traditional approaches to drug discovery.

Data are expected this year from a candidate it’s developing for neurofibromatosis type 2, and in the first half of 2025 for a treatment for familial adenomatous polyposis.

https://finance.yahoo.com/m/0c753ec8-8167-30c0-b970-eab2a00ee948/ai-specialist-recursion-says.html