NVIDIA-BACKED Recursion Pharmaceuticals said on Tuesday (Sep 3) its experimental drug to treat a rare brain-related condition was safe and tolerable in a mid-stage study, but showed mixed results for efficacy.
Shares of the company, which uses artificial intelligence to discover new drug candidates, dropped about 14 per cent.
The drug, REC-994, was being tested to treat cerebral cavernous malformation, or abnormal collection of small blood vessels that form lesions in the brain, which could cause bleeding in the brain, seizures and paralysis.
According to MRI-based data, the highest dose of the drug displayed signs of reduced number of lesions in the study, but improvements as reported by patients or doctors were not yet seen at the end of the 12-month treatment period, Recursion said.
“The path forward for this drug is still unclear,” Jefferies analyst Dennis Ding said. “There are virtually no other drugs that are being developed for this indication, and it’s also an indication that the FDA is very unfamiliar with.”
Recursion is expected to meet with the US Food and Drug Administration to discuss plans for an additional clinical study.
More data and feedback from the FDA would be required to “figure out a proper endpoint and the bar for clinical meaningfulness, so that any future trials would have a clearer bar in terms of what is needed” for a potential approval, Ding said.
Cerebral cavernous malformation affects more than one million people worldwide and has only surgical treatment options available, according to the company.
Last year, chip designer Nvidia had said it would invest US$50 million in Recursion to speed up training of its AI models for drug discovery. REUTERS
