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KUALA LUMPUR, Recursion, a clinical stage TechBio company, announced top-line results of the SYCAMORE trial, a 12-month Phase 2 randomised double-blind, placebo-controlled, safety, tolerability and exploratory efficacy study for REC-994 in symptomatic Cerebral Cavernous Malformation (CCM) patients.
‘We are encouraged by the recent data from our signal-finding Phase 2 study in CCM, where the trial successfully met its primary safety endpoint and became the first investigational therapy to demonstrate safety alongside some promising trends in exploratory efficacy endpoints.
‘These results provide critical insights that will inform our next study design, including exploring study duration, higher doses, and a larger cohort of patients,’ said Recursion Chief RandD Officer and Chief Commercial Officer, Najat Khan.
Meanwhile, University of Pennsylvania Division Head, Cerebrovascular Surgery, and Principal Investigator of the study, Dr Jan-Karl Burkhardt said the data from this readout is an impressive start and
will provide a valuable contribution to the existing CCM literature and strongly supports the need for a future study, with a longer duration and a larger patient cohort.
REC-994 met its primary endpoint of safety and tolerability, demonstrating a similar profile across placebo and both 200 milligramme (mg) and 400 mg dosage-arms with regard to the frequency and severity of adverse events after 12 months of treatment.
Magnetic resonance imaging-based secondary efficacy endpoints showed a trend towards reduced lesion volume and hemosiderin ring size in patients at the highest dose (400mg) as compared to placebo.
Time-dependent improvement in these trends at the 400mg dose was also observed in this signal-finding study, with improvements in either patient or physician-reported outcomes were not yet seen at the 12 months time point.
A meeting with the Food and Drug Administration (FDA) is anticipated as soon as practical to discuss plans for an additional clinical study. Recursion plans to present data from
this trial at a forthcoming medical conference and intends to submit these data for publication in a peer reviewed scientific journal.
REC-994 is an orally bioavailable, superoxide scavenger small molecule under development for the treatment of symptomatic CCM. The potential of REC-994 in CCM was demonstrated using the earliest version of what would become the foundational technology underlying the Recursion OS.
Subsequently, REC-994 demonstrated preclinical activity in models for CCM and tolerability and suitability for chronic dosing in Phase 1 single ascending dose escalation (SAD) and multiple ascending dose escalation (MAD) trials in healthy volunteers directed and executed by Recursion.
Source: BERNAMA News Agency
