Recursion מודיעה על נתוני שלב 2 של REC-994, טיפול ניסיוני ראשון במחלה למום מוחי סימפטומטי (CCM), שעמד בנקודת הקצה העיקרית של בטיחות וסבילות

REC-994 מדגים גם מגמות מעודדות במדדי יעילות גישוש אובייקטיביים מבוססי MRI במינון הגבוה ביותר, והחברה מתכננת לקדם את הפיתוח של REC-994 לטיפול פוטנציאלי ב-CCM סימפטומטי במחקרים הבאים.

סולט לייק סיטי, 3 בספטמבר 2024 (GLOBE NEWSWIRE) –

Recursion (נאסד"ק: RXRX), חברת טכנוביולוגיה מובילה בשלבים הקליניים המפענחת ביולוגיה כדי לשפר באופן קיצוני את החיים, הודיעה היום על תוצאות השורה הראשונה של ניסוי SYCAMORE, מחקר שלב 2 אקראי כפול סמיות, מבוקר פלצבו, בטיחות, סבילות ויעילות גישוש שלב 2 עבור REC-994 בחולי CCM סימפטומטיים.

Recursion Announces Phase 2 Data of REC-994, a First-in-Disease Investigational Treatment for Symptomatic Cerebral Cavernous Malformation (CCM), has Met its Primary Endpoint of Safety and Tolerability

REC-994 also demonstrates encouraging trends in objective MRI-based exploratory efficacy measures at the highest dose and the company plans to advance development of REC-994 for the potential treatment of symptomatic CCM in subsequent studies.

SALT LAKE CITY, Sept. 03, 2024 (GLOBE NEWSWIRE) –

Recursion (NASDAQ: RXRX), a leading clinical stage TechBio company decoding biology to radically improve lives, today announced top-line results of the SYCAMORE trial, a 12-month Phase 2 randomized double-blind, placebo-controlled, safety, tolerability and exploratory efficacy study for REC-994 in symptomatic CCM patients.

REC-994 met its primary endpoint of safety and tolerability, demonstrating a similar profile across placebo and both 200mg and 400mg 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. Improvements in either patient or physician-reported outcomes were not yet seen at the 12 month time point. A meeting with the 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.

"These studies are making significant strides in the development of therapeutics for CCM. 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,” said Dr. Jan-Karl Burkhardt, MD, Division Head, Cerebrovascular Surgery, University of Pennsylvania and Principal Investigator of the study. Connie Lee, Psy.D., founder and CEO of the Alliance to Cure Cavernous Malformation added: “I speak for the patients who have participated in the trial and those who have been cheering from the sidelines while waiting for news. This promising start is a critical step forward and will bring hope to thousands of families who currently have no options but brain or spinal cord surgery. The Alliance to Cure Cavernous Malformation looks forward to partnering with Recursion as they move to the next stage of the REC-994 program.”

“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 Najat Khan, Ph.D., Chief R&D Officer and Chief Commercial Officer of Recursion. "This is the first of several key clinical readouts for the company and represents an early proof-of-platform milestone for our constantly evolving Recursion OS, as we build upon our success in drug discovery with expertise and execution in mid-phase development. We are deeply grateful to the patients and investigators, and we are committed to advancing potential transformational therapies for CCM and beyond.”

Background on Cerebral Cavernous Malformation (CCM)

CCM is a neurovascular condition that impacts approximately 360,000 symptomatic individuals in the US and EU5. The disease is often underdiagnosed and potentially affects over 1 million patients worldwide. CCM manifests as vascular malformations of the spinal cord and brain characterized by abnormally enlarged capillary cavities without intervening brain parenchyma. Patients with CCM lesions are at substantial risk for seizures, headaches, progressive neurological deficits, and potentially fatal hemorrhagic stroke. Currently, only non-pharmacologic treatments including microsurgical resection and stereotactic radiosurgery are available options for this high unmet need patient population. However, surgical resection or stereotactic radiosurgery is not always feasible because of location and may not be curative.

About REC-994

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. Recursion has sought and received Orphan Drug Designation for REC-994 in symptomatic CCM in the US and Europe.

About the Trial

Our Phase 2 SYCAMORE clinical trial is a randomized, double-blind, placebo-controlled study of two doses of REC-994 in participants with CCM. The primary endpoint of the study is safety and tolerability. Secondary efficacy endpoints include MRI-based endpoints, clinician and patient reported outcomes, as well as selected biomarkers. This trial was fully enrolled in June 2023 with 62 participants, and 80% of participants who completed 12 months of treatment have entered the long-term extension study. This signal-finding study was not powered to demonstrate statistical significance.

About Recursion

Recursion (NASDAQ: RXRX) is a clinical stage TechBio company leading the space by decoding biology to radically improve lives. Enabling its mission is the Recursion OS, a platform built across diverse technologies that continuously generate one of the world’s largest proprietary biological and chemical datasets. Recursion leverages sophisticated machine-learning algorithms to distill from its dataset a collection of trillions of searchable relationships across biology and chemistry unconstrained by human bias. By commanding massive experimental scale — up to millions of wet lab experiments weekly — and massive computational scale — owning and operating one of the most powerful supercomputers in the world, Recursion is uniting technology, biology and chemistry to advance the future of medicine.

Recursion is headquartered in Salt Lake City, where it is a founding member of BioHive, the Utah life sciences industry collective. Recursion also has offices in Toronto, Montréal, London, and the San Francisco Bay Area. Learn more at www.Recursion.com, or connect on X (formerly Twitter) and LinkedIn.

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