Rigel Announces R289 Granted Fast Track Designation by the FDA for Lower-Risk MDS
Rigel Pharmaceuticals (Nasdaq: RIGL) announced that the FDA has granted Fast Track designation to R289 for treating patients with previously-treated transfusion dependent lower-risk myelodysplastic syndrome (LR-MDS). R289, a dual inhibitor of IRAK1 and IRAK4, is currently being evaluated in a Phase 1b study. The Fast Track designation may provide benefits including more frequent FDA interactions and eligibility for Accelerated Approval and Priority Review. The drug works by blocking inflammatory cytokine production in response to toll-like receptor and interleukin-1 receptor family signaling, targeting the pro-inflammatory environment in bone marrow responsible for persistent cytopenias in lower-risk MDS patients.
Rigel Pharmaceuticals (Nasdaq: RIGL) ha annunciato che la FDA ha concesso la designazione Fast Track a R289 per il trattamento di pazienti con sindrome mielodisplastica a basso rischio dipendente da trasfusioni già trattati in precedenza (LR-MDS). R289, un inibitore duale di IRAK1 e IRAK4, è attualmente in fase di valutazione in uno studio di Fase 1b. La designazione Fast Track può fornire vantaggi, tra cui interazioni più frequenti con la FDA e idoneità per l'Approvazione Accelerata e la Revisione Prioritaria. Il farmaco agisce bloccando la produzione di citochine infiammatorie in risposta alla segnalazione dei recettori toll-like e della famiglia dei recettori dell'interleuchina-1, mirando all'ambiente pro-infiammatorio nel midollo osseo responsabile delle citopenie persistenti nei pazienti con MDS a basso rischio.
Rigel Pharmaceuticals (Nasdaq: RIGL) anunció que la FDA ha otorgado la designación Fast Track a R289 para el tratamiento de pacientes con síndrome mielodisplástico de bajo riesgo dependiente de transfusiones que han sido tratados previamente (LR-MDS). R289, un inhibidor dual de IRAK1 e IRAK4, se está evaluando actualmente en un estudio de Fase 1b. La designación Fast Track puede proporcionar beneficios, incluidas interacciones más frecuentes con la FDA y elegibilidad para Aprobación Acelerada y Revisión Prioritaria. El medicamento actúa bloqueando la producción de citoquinas inflamatorias en respuesta a la señalización del receptor tipo Toll y la familia de receptores de interleucina-1, dirigéndose al ambiente proinflamatorio en la médula ósea responsable de las citopenias persistentes en pacientes con MDS de bajo riesgo.
리겔 제약 (Nasdaq: RIGL)은 FDA가 신속 심사 지정을 R289에 부여하여 이전에 치료를 받은 수혈 의존 저위험 골수이형성증후군 (LR-MDS) 환자를 치료한다고 발표했습니다. R289는 IRAK1과 IRAK4의 이중 억제제로 현재 1b상 연구에서 평가되고 있습니다. 신속 심사 지정은 보다 빈번한 FDA 상호작용 및 가속 승인 및 우선 심사에 대한 자격을 포함한 혜택을 제공할 수 있습니다. 이 약물은 톨 유사 수용체 및 인터루킨 1 수용체 패밀리 신호에 대한 반응으로 염증성 사이토카인 생성을 차단하여 저위험 MDS 환자에서 지속적인 세포감소증을 유발하는 골수 내 프로염증 환경을 표적으로 작용합니다.
Rigel Pharmaceuticals (Nasdaq: RIGL) a annoncé que la FDA a accordé la désignation Fast Track à R289 pour traiter des patients précédemment traités pour un syndrome myélodysplasique à bas risque dépendant des transfusions (LR-MDS). R289, un inhibiteur dual de IRAK1 et IRAK4, est actuellement évalué dans le cadre d'une étude de Phase 1b. La désignation Fast Track peut offrir des avantages, notamment des interactions plus fréquentes avec la FDA et l'éligibilité pour une approbation accélérée et un examen prioritaire. Ce médicament agit en bloquant la production de cytokines inflammatoires en réponse à la signalisation des récepteurs de type Toll et de la famille des récepteurs de l'interleukine-1, ciblant l'environnement pro-inflammatoire dans la moelle osseuse responsable des cytopénies persistantes chez les patients atteints de MDS à bas risque.
Rigel Pharmaceuticals (Nasdaq: RIGL) gab bekannt, dass die FDA die Fast-Track-Bezeichnung für R289 zur Behandlung von zuvor behandelten transfusionsabhängigen niedrigrisiko myelodysplastischen Syndrom (LR-MDS) Patienten vergeben hat. R289, ein dualer Inhibitor von IRAK1 und IRAK4, wird derzeit in einer Phase-1b-Studie evaluiert. Die Fast-Track-Bezeichnung kann Vorteile bieten, darunter häufigere Interaktionen mit der FDA und die Berechtigung für beschleunigte Zulassung und priorisierte Überprüfung. Das Medikament wirkt, indem es die Produktion entzündlicher Zytokine als Reaktion auf die Signalgebung durch Toll-ähnliche Rezeptoren und die Interleukin-1-Rezeptor-Familie blockiert und somit die pro-inflammatorische Umgebung im Knochenmark angreift, die für persistierende Zytopenien bei Patienten mit niedrigem Risiko MDS verantwortlich ist.
- Received FDA Fast Track designation, potentially accelerating the drug development and review process
- Eligible for Accelerated Approval and Priority Review if criteria are met
- Addresses an unmet medical need in transfusion-dependent LR-MDS patients
- Drug is still in early Phase 1b testing stage
- No efficacy data or preliminary results provided from the ongoing study
Insights
The FDA Fast Track designation for R289 represents a significant regulatory milestone for Rigel's LR-MDS program. The designation, based on early Phase 1b data, provides several key advantages including more frequent FDA interactions and potential eligibility for Accelerated Approval. This could substantially expedite R289's path to market.
The mechanism of action targeting IRAK1/4 is particularly noteworthy. By inhibiting inflammatory signaling pathways implicated in MDS progression, R289 addresses a novel therapeutic approach. Current treatment options for transfusion-dependent LR-MDS patients are , making this an attractive market opportunity with significant unmet medical need.
While still in early clinical development, the Fast Track status validates the therapeutic potential and could accelerate development timelines by 6-12 months. This positions Rigel favorably in the competitive landscape for MDS treatments, though investors should note that Phase 2/3 trials and eventual commercialization are still years away.
"We are pleased that R289 has been granted Fast Track designation, which underscores the significant unmet need for patients with transfusion dependent lower-risk MDS," said Raul Rodriguez, Rigel's president and CEO. "By targeting inflammatory signaling, we believe that R289 has the potential to meaningfully improve the lives of those living with this disease."
"Lower-risk MDS affects a primarily elderly patient population that faces progressive cytopenias, particularly anemia, and treatment options for transfusion-dependent patients are limited," said Lisa Rojkjaer, M.D., Rigel's chief medical officer. "This designation is based on initial data from the ongoing Phase 1b study and highlights the potential of R289 to be a new therapeutic option for these patients. We look forward to working closely with the FDA to advance the clinical development of R289."
Fast track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. A drug that receives Fast Track designation may benefit from more frequent interactions with the FDA over the course of drug development. In addition, the Fast Track program allows for eligibility for Accelerated Approval and Priority Review, if relevant criteria are met.
About R289
R289 is a prodrug of R835, an IRAK1/4 dual inhibitor, which has been shown in preclinical studies to block inflammatory cytokine production in response to toll-like receptor (TLR) and interleukin-1 receptor (IL-1R) family signaling. TLRs and IL-1Rs play a critical role in the innate immune response and dysregulation of these pathways can lead to various inflammatory conditions. Chronic stimulation of both these receptor systems is thought to cause the pro-inflammatory environment in the bone marrow responsible for persistent cytopenias in lower-risk MDS patients.2
About Rigel
Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) is a biotechnology company dedicated to discovering, developing and providing novel therapies that significantly improve the lives of patients with hematologic disorders and cancer. Founded in 1996, Rigel is based in
- R289 is an investigational compound not approved by the FDA.
- Sallman DA et al. Unraveling the Pathogenesis of MDS: The NLRP3 Inflammasome and Pyroptosis Drive the MDS Phenotype. Front Oncol. June 16, 2016. DOI: https://doi.org/10.3389/fonc.2016.0015
Forward-Looking Statements
This press release contains forward-looking statements relating to, among other things, the potential benefits of Fast Track designation for R289 for the treatment of patients with lower-risk myelodysplastic syndrome (LR-MDS), its potential as a therapeutic, the existence of patients with an unmet medical need for such therapy, the potential for such therapy to meaningfully improve the lives of such patients, and Rigel's ability to further develop its clinical stage product candidates, including the progress of current and potential future clinical trials of R289. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements and as such are intended to be covered by the safe harbor for "forward-looking statements" provided by the PSLRA. Forward-looking statements can be identified by words such as "plan", "potential", "may", "look to", "expects", "will" and similar expressions in reference to future periods. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Rigel's current beliefs, expectations, and assumptions and hence they inherently involve significant risks, uncertainties and changes in circumstances that are difficult to predict and many of which are outside of Rigel's control. Therefore, you should not rely on any of these forward-looking statements. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, Fast Track designation may not result in a more expedited development or regulatory review process, and such a designation does not increase the likelihood that R289 will receive marketing approval in
Contact for Investors & Media:
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650.624.1232
ir@rigel.com
Media:
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FAQ
What is the Fast Track designation that RIGL received for R289?
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