Recursion Announces FDA Clearance of Investigational New Drug Application for REC-1245, a Potential First-In-Class RBM39 Degrader for Biomarker-Enriched Solid Tumors and Lymphoma
Recursion (NASDAQ: RXRX) has received FDA clearance for an Investigational New Drug (IND) application for REC-1245, a potential first-in-class RBM39 degrader targeting biomarker-enriched solid tumors and lymphoma. This program, developed using Recursion's AI-enabled active learning modules, progressed from target identification to IND-enabling studies in under 18 months.
The company plans to initiate a Phase 1/2 clinical trial in Q4 2024, evaluating REC-1245 in a biomarker-enriched patient population. Recursion identified RBM39's novel regulatory role associated with CDK12 using its maps of biology. The company estimates the initially addressable population for this potential therapeutic to be over 100,000 patients in the US and EU5.
Preclinical data suggest that RBM39 degradation induces splicing defects, downregulating DNA Damage Response networks and cell cycle checkpoints. The upcoming clinical trial will assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and potential monotherapy efficacy of REC-1245.
Recursion (NASDAQ: RXRX) ha ricevuto l'approvazione della FDA per una richiesta di farmaco sperimentale (IND) per REC-1245, un potenziale degradatore di RBM39 di prima classe che mira a tumori solidi arricchiti da biomarker e linfomi. Questo programma, sviluppato utilizzando i moduli di apprendimento attivo abilitati dall'IA di Recursion, è avanzato dall'identificazione del target agli studi abilitanti per l'IND in meno di 18 mesi.
L'azienda prevede di avviare un studio clinico di Fase 1/2 nel quarto trimestre del 2024, valutando REC-1245 in una popolazione di pazienti arricchita da biomarker. Recursion ha identificato il nuovo ruolo regolatorio di RBM39 associato a CDK12 utilizzando le sue mappe biologiche. L'azienda stima che la popolazione inizialmente trattabile per questa potenziale terapia superi i 100.000 pazienti negli Stati Uniti e nei cinque principali paesi dell'UE.
I dati preclinici suggeriscono che la degradazione di RBM39 induce difetti di splicing, riducendo le reti della risposta al danno del DNA e i checkpoint del ciclo cellulare. Il prossimo studio clinico valuterà la sicurezza, la tollerabilità, la farmacocinetica, la farmacodinamica e la potenziale efficacia della monoterapia di REC-1245.
Recursion (NASDAQ: RXRX) ha recibido la aprobación de la FDA para una solicitud de nuevo medicamento en investigación (IND) para REC-1245, un posible degradador de RBM39 de primera clase que se dirige a tumores sólidos enriquecidos con biomarcadores y linfomas. Este programa, desarrollado utilizando los módulos de aprendizaje activo habilitados por IA de Recursion, avanzó desde la identificación del objetivo hasta estudios habilitantes de IND en menos de 18 meses.
La empresa planea iniciar un ensayo clínico de Fase 1/2 en el cuarto trimestre de 2024, evaluando REC-1245 en una población de pacientes enriquecida con biomarcadores. Recursion identificó el nuevo rol regulador de RBM39 asociado con CDK12 utilizando sus mapas biológicos. La empresa estima que la población inicial tratable para esta potencial terapia supera los 100,000 pacientes en EE. UU. y en los cinco principales países de la UE.
Los datos preclínicos sugieren que la degradación de RBM39 induce defectos de empalme, desacelerando las redes de respuesta al daño del ADN y los puntos de control del ciclo celular. El próximo ensayo clínico evaluará la seguridad, la tolerancia, la farmacocinética, la farmacodinamia y la posible eficacia de la monoterapia de REC-1245.
Recursion (NASDAQ: RXRX)는 REC-1245에 대한 조사 신약 (IND) 신청에 대해 FDA 승인을 받았습니다. REC-1245는 바이오마커가 풍부한 고형 종양 및 림프종을 표적으로 하는 잠재적인 1세대 RBM39 분해제입니다. 이 프로그램은 Recursion의 AI 활성 학습 모듈을 사용하여 18개월 이내에 목표 식별에서 IND 지원 연구로 진행되었습니다.
회사는 바이오마커가 풍부한 환자 집단에서 REC-1245를 평가하는 1/2상 임상 시험을 2024년 4분기에 시작할 계획입니다. Recursion은 생물학적 지도를 사용하여 CDK12와 관련된 RBM39의 새로운 조절 역할을 확인했습니다. 이 회사는 이 잠재적 치료에 대한 초기 치료 가능 인구가 미국과 EU5에서 100,000명 이상이 될 것으로 추정합니다.
전임상 데이터는 RBM39 분해가 스플라이싱 결함을 유도하고 DNA 손상 응답 네트워크와 세포 주기 검문소를 하향 조절함을 시사합니다. 다가오는 임상 시험에서는 REC-1245의 안전성, 내약성, 약물 동태학, 약물 작용학 및 가능성 있는 단독 요법 효능을 평가할 것입니다.
Recursion (NASDAQ: RXRX) a reçu l'approbation de la FDA pour une demande de médicament expérimental (IND) pour REC-1245, un potentiel dégradateur de RBM39 de première classe ciblant des tumeurs solides enrichies en biomarqueurs et des lymphomes. Ce programme, développé à l'aide des modules d'apprentissage actif habilités par l'IA de Recursion, a progressé de l'identification de la cible aux études permettant l'IND en moins de 18 mois.
L'entreprise prévoit de lancer un essai clinique de Phase 1/2 au quatrième trimestre de 2024, évaluant REC-1245 dans une population de patients enrichie en biomarqueurs. Recursion a identifié le nouveau rôle régulateur de RBM39 associé à CDK12 en utilisant ses cartes biologiques. L'entreprise estime que la population initialement traitable pour cette thérapeutique potentielle dépasse les 100 000 patients aux États-Unis et dans les cinq pays principaux de l'UE.
Les données précliniques suggèrent que la dégradation de RBM39 induit des défauts d'épissage, régulant à la baisse les réseaux de réponse aux dommages de l'ADN et les checkpoints du cycle cellulaire. L'essai clinique à venir évaluera la sécurité, la tolérance, la pharmacocinétique, la pharmacodynamie et l'efficacité potentielle en monothérapie de REC-1245.
Recursion (NASDAQ: RXRX) hat von der FDA die Genehmigung für einen Antrag auf ein Investigational New Drug (IND) für REC-1245 erhalten, einen potenziellen ersten RBM39-Degrader, der auf biomarker-angereicherte solide Tumoren und Lymphome abzielt. Dieses Programm, das mit den KI-gestützten aktiven Lernmodulen von Recursion entwickelt wurde, hat sich in weniger als 18 Monaten von der Zielidentifikation zu IND-unterstützenden Studien weiterentwickelt.
Das Unternehmen plant, im vierten Quartal 2024 eine Phase 1/2 klinische Studie zu starten, in der REC-1245 in einer biomarker-angereicherten Patientenpopulation bewertet wird. Recursion hat die neuartige regulatorische Rolle von RBM39 in Verbindung mit CDK12 mithilfe ihrer biologischen Karten identifiziert. Das Unternehmen schätzt, dass die anfänglich ansprechbare Population für diese potenzielle Therapie über 100.000 Patienten in den USA und den EU5 beträgt.
Präklinische Daten deuten darauf hin, dass die Degradation von RBM39 Spleißdefekte induziert und Netzwerke der DNA-Schadenantwort sowie Kontrollpunkte des Zellzyklus herabreguliert. Die bevorstehende klinische Studie wird die Sicherheit, Verträglichkeit, Pharmacokinetik, Pharmacodynamik und die potenzielle monotherapeutische Wirksamkeit von REC-1245 bewerten.
- FDA clearance of IND application for REC-1245, a potential first-in-class RBM39 degrader
- Rapid development from target identification to IND-enabling studies in under 18 months
- Estimated addressable patient population of over 100,000 in the US and EU5
- Promising preclinical data showing RBM39 degradation's effects on DNA Damage Response networks
- Planned initiation of Phase 1/2 clinical trial in Q4 2024
- None.
Insights
The FDA clearance of the IND for REC-1245 marks a significant milestone in targeted cancer therapy. This potential first-in-class RBM39 degrader represents a novel approach to treating biomarker-enriched solid tumors and lymphoma. The rapid progression from target identification to IND in under 18 months is remarkable, showcasing the power of AI-enabled drug discovery platforms.
Key points to consider:
- REC-1245 targets RBM39, which is functionally similar to CDK12 but potentially more druggable
- Preclinical data suggests RBM39 degradation affects DNA Damage Response networks and cell cycle checkpoints
- The addressable patient population is estimated at
>100,000 in the US and EU5 - The Phase 1/2 trial, set to begin in Q4 2024, will assess safety, tolerability and potential efficacy
This development could potentially offer new treatment options for patients with alternatives, particularly those resistant to conventional therapies. However, as with any early-stage drug, clinical trial results will be important in determining its true potential and safety profile.
Recursion's announcement demonstrates the company's innovative approach to drug discovery, leveraging AI and machine learning to accelerate the development process. The 18-month timeline from target identification to IND is particularly noteworthy, as it significantly outpaces industry averages.
From an investor perspective, this development has several implications:
- Validates Recursion's AI-enabled platform and its potential to revolutionize drug discovery
- Positions the company as a leader in the emerging field of TechBio
- Expands Recursion's pipeline with a potentially high-value asset
- Could attract partnerships or collaborations with larger pharmaceutical companies
However, it's important to note that the road from IND to market approval is long and uncertain. Investors should monitor the progress of the Phase 1/2 trial closely, as positive results could significantly impact Recursion's valuation. The company's ability to replicate this rapid development process with other candidates will be important for long-term success.
- First program to combine Recursion’s end-to-end suite of AI-enabled active learning modules, resulting in target identification to IND enabling studies in under 18 months
- Plan to initiate dosing of Phase 1/2 in Q4 2024 to evaluate REC-1245 in a biomarker enriched patient population, including patients with solid tumors and lymphoma
SALT LAKE CITY, Oct. 02, 2024 (GLOBE NEWSWIRE) -- Recursion (NASDAQ: RXRX), a leading clinical stage TechBio company decoding biology to industrialize drug discovery, today announced that the U.S. Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for a Phase 1/2 clinical trial of REC-1245, a new chemical entity for the treatment of biomarker-enriched solid tumors and lymphoma.
Chris Gibson, Ph.D., Co-founder and CEO of Recursion said, “REC-1245 is a prime example of using an expansive AI-enabled platform for drug discovery. After exploring many predicted biological and chemical relationships across our maps of biology, we identified RMB39 as a novel target that looks functionally similar to the well-known but hard to drug target CDK12. We also identified and optimized small molecules that target RBM39 without directly impacting CDK12 or CDK13 using these same AI-enabled maps. In under 18 months, leveraging some of our newer chemistry tools, Recursion rapidly progressed REC-1245 from novel target biology to preclinical drug candidate, more than twice the speed of industry average.”
Recursion identified the novel regulatory role of RBM39 associated with CDK12 using its maps of biology and first reported this relationship in early 2023 at Download Day, Recursion’s R&D and investor event. Recursion believes the modulation of RBM39 may be associated with a therapeutic effect in certain biomarker-enriched solid tumors and lymphoma. Additionally, Recursion estimates that the initially addressable population for this potential therapeutic to be >100,000 patients in the US and EU5. REC-1245 is a potent and selective RBM39 degrader with a potential first-in-class profile. Preclinical data support that RBM39 degradation induces splicing defects which downregulate DNA Damage Response (DDR) networks and cell cycle checkpoints.
“RBM39 degraders may offer a promising therapeutic approach for patients with solid tumors, particularly those with limited treatment options,” said Najat Khan, Ph.D., Chief R&D Officer and Chief Commercial Officer at Recursion. “Recursion’s platform was among the first to rapidly uncover the therapeutic potential of RBM39 degradation, a finding now validated by independent research. This mechanism provides new opportunities for targeting tumors, which are often resistant to conventional treatments. By advancing this research, we aim to deliver a critical option for patients facing significant unmet needs, ultimately improving their prognosis and quality of life.”
The Phase 1/2 clinical trial will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and potential monotherapy efficacy of REC-1245, and is expected to initiate in Q4 2024.
About Recursion
Recursion (NASDAQ: RXRX) is a clinical stage TechBio company decoding biology to industrialize drug discovery. Enabling its mission is the Recursion OS, a platform built across diverse technologies that continuously expands 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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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 the potential efficacy of REC-1245; timing of and plans to initiate dosing of Phase 1 clinical trial of REC-1245; early and late stage discovery, preclinical, and clinical programs; licenses and collaborations; prospective products and their potential future indications and market opportunities; Recursion OS and other technologies; 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 Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. 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 is REC-1245 and what is its potential use?
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