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
Recursion (NASDAQ: RXRX) announced positive top-line results from its SYCAMORE trial, a Phase 2 study of REC-994 for symptomatic Cerebral Cavernous Malformation (CCM). The trial met its primary endpoint of safety and tolerability, with similar profiles across placebo and both 200mg and 400mg dosage arms. Encouraging trends in MRI-based secondary efficacy endpoints were observed, particularly at the highest dose (400mg), showing reduced lesion volume and hemosiderin ring size compared to placebo.
The company plans to advance REC-994's development for potential CCM treatment in subsequent studies. A meeting with the FDA is anticipated to discuss plans for an additional clinical study. Recursion aims to present the data at a future medical conference and submit it for peer-reviewed publication.
Recursion (NASDAQ: RXRX) ha annunciato risultati positivi dai dati primari del suo studio SYCAMORE, uno studio di Fase 2 su REC-994 per la Malformazione Cavernosa Cerebrale (CCM) sintomatica. Lo studio ha raggiunto il suo obiettivo principale di sicurezza e tollerabilità, mostrando profili simili tra il placebo e le dosi di 200mg e 400mg. Sono state osservate tendenze promettenti negli endpoint secondari di efficacia basati su risonanza magnetica, in particolare alla dose più alta (400mg), evidenziando una riduzione del volume delle lesioni e della dimensione dell'anello di emosiderina rispetto al placebo.
L'azienda prevede di portare avanti lo sviluppo di REC-994 per il potenziale trattamento della CCM in studi successivi. È prevista una riunione con la FDA per discutere i piani per uno studio clinico aggiuntivo. Recursion intende presentare i dati in un futuro congresso medico e inoltrarli per pubblicazione su riviste sottoposte a peer review.
Recursion (NASDAQ: RXRX) anunció resultados positivos de los datos primarios de su ensayo SYCAMORE, un estudio de Fase 2 de REC-994 para la Malformación Cavernosa Cerebral (CCM) sintomática. El ensayo cumplió su objetivo principal de seguridad y tolerabilidad, con perfiles similares entre el placebo y las dosis de 200mg y 400mg. Se observaron tendencias alentadoras en los puntos finales secundarios de eficacia basados en resonancia magnética, especialmente en la dosis más alta (400mg), mostrando una reducción en el volumen de la lesión y en el tamaño del anillo de hemosiderina en comparación con el placebo.
La compañía planea avanzar en el desarrollo de REC-994 para un posible tratamiento de la CCM en estudios posteriores. Se espera una reunión con la FDA para discutir los planes de un estudio clínico adicional. Recursion tiene como objetivo presentar los datos en una futura conferencia médica y someterlos a publicación en una revista revisada por pares.
Recursion (NASDAQ: RXRX)는 증상 있는 뇌 해면종(CCM)을 위한 REC-994의 2상 연구인 SYCAMORE 시험에서 긍정적인 주요 결과를 발표했습니다. 이 시험은 안전성 및 내약성의 주요 목표를 달성하였으며, 플라시보 및 200mg 및 400mg 용량군 간에 유사한 프로파일을 보였습니다. 특히 최고 용량(400mg)에서 MRI 기반의 고무적인 추세가 관찰되어, 플라시보에 비해 병변 부피와 헤모시데린 링 크기가 감소했습니다.
회사는 향후 연구에서 CCM 치료를 위한 REC-994 개발을 진행할 계획입니다. 추가 임상 연구 계획에 대해 논의하기 위해 FDA와의 미팅이 예정되어 있습니다. Recursion은 향후 의료 회의에서 데이터를 발표하고 동료 심사를 거친 출판을 위해 제출할 계획입니다.
Recursion (NASDAQ: RXRX) a annoncé des résultats positifs des données préliminaires de son essai SYCAMORE, une étude de Phase 2 sur REC-994 pour la Malformation Cavernouse Cérébrale (CCM) symptomatique. L'essai a atteint son objectif principal de safety and tolerabilité, avec des profils similaires entre le placebo et les dosages de 200mg et 400mg. Des tendances encourageantes dans les critères d'évaluation secondaires basés sur l'IRM ont été observées, en particulier à la dose la plus élevée (400mg), montrant une réduction du volume des lésions et de la taille de l'anneau d'hémosidérine par rapport au placebo.
L'entreprise prévoit de faire avancer le développement de REC-994 pour un traitement potentiel de la CCM dans les études suivantes. Une réunion avec la FDA est anticipée pour discuter des plans pour une étude clinique supplémentaire. Recursion vise à présenter les données lors d'une future conférence médicale et à les soumettre pour publication dans une revue arbitrée.
Recursion (NASDAQ: RXRX) hat positive Ergebnisse aus seiner SYCAMORE-Studie bekannt gegeben, einer Phase-2-Studie zu REC-994 bei symptomatischer zerebraler Kavernomatosis (CCM). Die Studie erfüllte ihren primären Endpunkt der Sicherheit und Verträglichkeit, mit ähnlichen Profilen zwischen Placebo und den Dosierungen von 200mg und 400mg. Ermutigende Trends in den sekundären Wirksamkeitsendpunkten, die auf MRI basieren, wurden insbesondere bei der höchsten Dosis (400mg) beobachtet, wobei ein reduziertes Läsionsvolumen und eine kleinere Hämosiderinringgröße im Vergleich zum Placebo festgestellt wurden.
Das Unternehmen plant, die Entwicklung von REC-994 für eine potenzielle CCM-Behandlung in nachfolgenden Studien voranzutreiben. Ein Treffen mit der FDA ist geplant, um die Pläne für eine zusätzliche klinische Studie zu erörtern. Recursion hat das Ziel, die Daten auf einer zukünftigen medizinischen Konferenz zu präsentieren und zur Veröffentlichung in einer peer-reviewed Zeitschrift einzureichen.
- REC-994 met its primary endpoint of safety and tolerability in the Phase 2 SYCAMORE trial
- Encouraging trends in MRI-based secondary efficacy endpoints observed at the highest dose (400mg)
- Plans to advance REC-994's development for potential CCM treatment in subsequent studies
- First investigational therapy to demonstrate safety alongside promising trends in exploratory efficacy endpoints for CCM
- No improvements in patient or physician-reported outcomes at the 12-month time point
- Additional clinical study with longer duration and larger patient cohort needed
Insights
The Phase 2 SYCAMORE trial results for REC-994 in symptomatic Cerebral Cavernous Malformation (CCM) patients are promising. The drug met its primary endpoint of safety and tolerability, showing a similar profile across placebo and both dosage arms. Importantly, there's a trend towards reduced lesion volume and hemosiderin ring size in the
However, it's important to note that improvements in patient or physician-reported outcomes were not observed at the 12-month mark. This suggests the need for longer-term studies to fully assess REC-994's efficacy. The planned FDA meeting and future larger study will be critical in determining the drug's potential as a first-in-disease treatment for CCM, a condition affecting approximately 360,000 symptomatic individuals in the US and EU5.
Recursion's announcement is a positive development for the company and potentially for CCM patients. As the first investigational therapy to demonstrate safety with promising efficacy trends in CCM, REC-994 could capture a significant market share if approved. With no current pharmacological treatments available for CCM, Recursion is tapping into an unmet medical need.
However, investors should temper expectations. The lack of improvement in patient or physician-reported outcomes at 12 months suggests that further studies and longer timeframes may be necessary before potential commercialization. This could mean additional R&D expenses and a longer timeline to market. The company's plan to advance to further studies indicates confidence in the drug's potential, but it's important to monitor future trial results and regulatory interactions for a clearer picture of REC-994's commercial prospects.
The SYCAMORE trial results are intriguing from a neurological perspective. The trend towards reduced lesion volume and hemosiderin ring size in the high-dose group is particularly noteworthy. Hemosiderin, a byproduct of blood breakdown, often indicates past microbleeds. Its reduction could potentially signify decreased bleeding risk, a important factor in CCM management.
However, the lack of improvement in clinical outcomes at 12 months is a concern. CCM's unpredictable nature means that longer observation periods may be necessary to detect meaningful clinical benefits. The planned larger study should focus on longer-term follow-up and potentially explore even higher doses. If successful, REC-994 could offer a much-needed alternative to current invasive treatments like microsurgical resection and stereotactic radiosurgery, especially for patients with lesions in high-risk locations.
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
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.
Media Contact
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Forward-Looking Statements
This document contains information that includes or is based upon "forward-looking statements" within the meaning of the Securities Litigation Reform Act of 1995, including, without limitation, those regarding Recursion’s anticipated meeting with the FDA; Recursion’s plans to present SYCAMORE trial data at a medical conference and submit the data for publication; the clinical relevance of the SYCAMORE trial data and obtaining additional confirmatory data; promising trends in REC-994 efficacy endpoints; advancing potential transformational therapies for CCM and beyond; subsequent REC-994 studies and their results and advancing Recursion’s REC-994 program further; the size of the potential CCM patient population; Recursion OS and other technologies potential and advancement of the future of medicine; business and financial plans and performance; and all other statements that are not historical facts. Forward-looking statements may or may not include identifying words such as “plan,” “will,” “expect,” “anticipate,” “intend,” “believe,” “potential,” “continue,” and similar terms. These statements are subject to known or unknown risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements, including but not limited to: challenges inherent in pharmaceutical research and development, including the timing and results of preclinical and clinical programs, where the risk of failure is high and failure can occur at any stage prior to or after regulatory approval due to lack of sufficient efficacy, safety considerations, or other factors; our ability to leverage and enhance our drug discovery platform; our ability to obtain financing for development activities and other corporate purposes; the success of our collaboration activities; our ability to obtain regulatory approval of, and ultimately commercialize, drug candidates; our ability to obtain, maintain, and enforce intellectual property protections; cyberattacks or other disruptions to our technology systems; our ability to attract, motivate, and retain key employees and manage our growth; inflation and other macroeconomic issues; and other risks and uncertainties such as those described under the heading “Risk Factors” in our filings with the U.S. Securities and Exchange Commission, including our most recent Annual Report on Form 10-K, our subsequent Quarterly Reports on Form 10-Q, and our Current Reports on Form 8-K. All forward-looking statements are based on management’s current estimates, projections, and assumptions, and Recursion undertakes no obligation to correct or update any such statements, whether as a result of new information, future developments, or otherwise, except to the extent required by applicable law.
FAQ
What were the results of Recursion's Phase 2 SYCAMORE trial for REC-994 in CCM patients?
What is the next step for Recursion (RXRX) in developing REC-994 for CCM treatment?
Did REC-994 show any improvements in patient or physician-reported outcomes in the Phase 2 trial?
How many symptomatic individuals are affected by Cerebral Cavernous Malformation (CCM)?
