Recursion has enrolled the first subject in the Phase II SYCAMORE clinical trial of its small molecule, REC-994, to treat cerebral cavernous malformation (CCM).
An orally bioavailable small molecule superoxide scavenger, REC-994 is the first treatment candidate to progress to an industry-sponsored trial for CCM.
The multicentre, randomised, double-blind, placebo-controlled trial will analyse the safety, efficacy and pharmacokinetics of REC-994 for the treatment of patients with CCM.
It is anticipated to enrol nearly 60 participants.
Recursion co-founder and CEO Chris Gibson said: “This is a big day for Recursion and the patients we hope to serve, as we announce the enrolment of the first patient in the first human efficacy trial in the company’s history.
“I have had the opportunity to get to know and work with many patients with CCM.
“It is a disease with incredible unmet need, affecting hundreds of thousands of people around the world and sometimes multiple generations within the same family.”
In Phase I single-ascending dose (SAD) and multiple-ascending dose (MAD) trials that enrolled healthy subjects, REC-994 was found to be tolerable and suitable for chronic dosing.
Previously, the US Food and Drug Administration and the European Commission had granted Orphan Drug status to REC-994 for CCM.
A neurovascular disease, CCM is caused by hereditary or somatic mutations in one of three genes associated with endothelial function: CCM1, CCM2, or CCM3.
CCM develops as vascular malformations of the spinal cord and brain, putting impacted patients at high risk for headaches, seizures, progressive neurological impairments and disabling and possibly deadly haemorrhagic strokes.