Rigel Announces R289 Granted Orphan Drug Designation by the FDA for MDS
Rigel Pharmaceuticals (NASDAQ: RIGL) announced that its drug candidate R289 has received Orphan Drug designation from the FDA for treating myelodysplastic syndromes (MDS). R289, a dual inhibitor of IRAK1 and IRAK4, is currently in a Phase 1b study evaluating its safety, tolerability, and preliminary activity in patients with lower-risk MDS who haven't responded to previous treatments.
The Orphan Drug designation provides several benefits, including tax credits, FDA fee exemptions, and potential seven-year market exclusivity upon approval. R289 had previously received Fast Track designation for treating patients with previously-treated transfusion-dependent lower-risk MDS.
R289 is a prodrug of R835 that works by blocking inflammatory cytokine production in response to toll-like receptor and interleukin-1 receptor family signaling, which are thought to cause pro-inflammatory conditions in the bone marrow of lower-risk MDS patients.
Rigel Pharmaceuticals (NASDAQ: RIGL) ha annunciato che il suo candidato farmaco R289 ha ricevuto la designazione di Farmaco Orfano dalla FDA per il trattamento delle sindromi mielodisplastiche (MDS). R289, un inibitore duale di IRAK1 e IRAK4, è attualmente in uno studio di Fase 1b che valuta la sua sicurezza, tollerabilità e attività preliminare in pazienti con MDS a basso rischio che non hanno risposto ai trattamenti precedenti.
La designazione di Farmaco Orfano offre diversi benefici, tra cui crediti d'imposta, esenzioni dalle tasse FDA e una potenziale esclusività di mercato di sette anni dopo l'approvazione. R289 aveva precedentemente ricevuto la designazione Fast Track per il trattamento di pazienti con MDS a basso rischio e dipendenti da trasfusioni già trattati.
R289 è un pro-farmaco di R835 che agisce bloccando la produzione di citochine infiammatorie in risposta alla segnalazione dei recettori toll-like e della famiglia dei recettori interleuchina-1, che si pensa causino condizioni pro-infiammatorie nel midollo osseo dei pazienti con MDS a basso rischio.
Rigel Pharmaceuticals (NASDAQ: RIGL) anunció que su candidato a medicamento R289 ha recibido la designación de Medicamento Huérfano por parte de la FDA para el tratamiento de síndromes mielodisplásicos (MDS). R289, un inhibidor dual de IRAK1 e IRAK4, se encuentra actualmente en un estudio de Fase 1b que evalúa su seguridad, tolerabilidad y actividad preliminar en pacientes con MDS de bajo riesgo que no han respondido a tratamientos previos.
La designación de Medicamento Huérfano proporciona varios beneficios, incluidos créditos fiscales, exenciones de tarifas de la FDA y una posible exclusividad en el mercado de siete años tras la aprobación. R289 había recibido anteriormente la designación Fast Track para el tratamiento de pacientes con MDS de bajo riesgo dependientes de transfusiones que ya habían sido tratados.
R289 es un profármaco de R835 que actúa bloqueando la producción de citoquinas inflamatorias en respuesta a la señalización del receptor tipo toll y la familia del receptor interleucina-1, que se cree causan condiciones proinflamatorias en la médula ósea de pacientes con MDS de bajo riesgo.
리겔 제약 (NASDAQ: RIGL)은 약물 후보 R289가 MDS(골수이형성증후군) 치료를 위한 FDA의 희귀의약품 지정을 받았다고 발표했습니다. R289는 IRAK1과 IRAK4의 이중 억제제로, 현재 이전 치료에 반응하지 않은 저위험 MDS 환자에서 안전성, 내약성 및 초기 활동성을 평가하는 1b상 연구가 진행 중입니다.
희귀의약품 지정은 세금 크레딧, FDA 수수료 면제 및 승인 시 7년 간의 시장 독점 가능성을 포함한 여러 가지 혜택을 제공합니다. R289는 이전에 치료받은 저위험 MDS 환자 치료를 위한 신속 심사(Fast Track) 지정을 받았습니다.
R289는 염증성 사이토카인 생성을 차단하여 작용하는 R835의 프로드럭으로, 톨 유사 수용체 및 인터루킨-1 수용체 패밀리 신호 전달에 반응하여 발생하는 저위험 MDS 환자의 골수 내에서 발생하는 염증성 조건을 유발하는 것으로 추정됩니다.
Rigel Pharmaceuticals (NASDAQ: RIGL) a annoncé que son candidat médicament R289 a reçu la désignation de Médicament Orphelin par la FDA pour le traitement des syndromes myélodysplasiques (MDS). R289, un inhibiteur dual de IRAK1 et IRAK4, est actuellement en phase d'étude 1b pour évaluer sa sécurité, sa tolérabilité et son activité préliminaire chez des patients atteints de MDS à faible risque n'ayant pas répondu aux traitements antérieurs.
La désignation de Médicament Orphelin offre plusieurs avantages, notamment des crédits d'impôt, des exonérations de frais de la FDA et une exclusivité sur le marché pouvant aller jusqu'à sept ans après approbation. R289 avait précédemment reçu la désignation Fast Track pour le traitement des patients ayant déjà été traités et dépendants des transfusions pour un MDS à faible risque.
R289 est un pro-médicament de R835 qui 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, qui sont considérés comme responsables des conditions pro-inflammatoires dans la moelle osseuse des patients atteints de MDS à faible risque.
Rigel Pharmaceuticals (NASDAQ: RIGL) gab bekannt, dass sein Arzneimittelkandidat R289 von der FDA die Orphan Drug-Bezeichnung zur Behandlung von myelodysplastischen Syndromen (MDS) erhalten hat. R289, ein Dualinhibitor von IRAK1 und IRAK4, befindet sich derzeit in einer Phase-1b-Studie, die seine Sicherheit, Verträglichkeit und vorläufige Aktivität bei Patienten mit niedrigem Risiko für MDS, die auf frühere Behandlungen nicht angesprochen haben, bewertet.
Die Orphan Drug-Bezeichnung bietet mehrere Vorteile, darunter Steuervergünstigungen, Befreiungen von FDA-Gebühren und eine potenzielle Marktexklusivität von sieben Jahren nach der Genehmigung. R289 hatte zuvor die Fast Track-Bezeichnung für die Behandlung von bereits behandelten, transfusionsabhängigen Patienten mit niedrigem Risiko für MDS erhalten.
R289 ist ein Prodrug von R835, das die Produktion entzündlicher Zytokine blockiert, die als Reaktion auf die Signalübertragung durch tollähnliche Rezeptoren und die Interleukin-1-Rezeptorfamilie entstehen, die als ursächlich für entzündliche Bedingungen im Knochenmark von Patienten mit niedrigem Risiko für MDS angesehen werden.
- Received FDA Orphan Drug designation for R289
- Previously obtained Fast Track designation for R289
- Potential 7-year market exclusivity upon approval
- Eligible for tax credits and FDA fee exemptions
- Phase 1b study ongoing with encouraging initial data
- R289 still in early Phase 1b testing phase
- No approved product yet - investigational compound
- to rare disease affecting <200,000 people in US
Insights
The FDA's Orphan Drug designation for R289 represents a significant regulatory milestone for Rigel's MDS program. The designation, coupled with the previously granted Fast Track status, provides important benefits: tax credits for clinical trials, FDA fee waivers and potentially 7 years of market exclusivity upon approval. This substantially reduces development costs and provides a clear competitive advantage.
The mechanism of action targeting IRAK1/4 is particularly compelling in MDS treatment. By inhibiting these key inflammatory signaling pathways, R289 addresses the underlying bone marrow inflammation that characterizes lower-risk MDS - a novel approach compared to existing treatments. The prodrug formulation (R289 converting to R835) suggests improved bioavailability and potentially better safety profile.
For retail investors: Think of this like getting both a fast-pass and exclusive rights at a theme park - it speeds up the process and keeps competitors at bay for a significant period.
From a market perspective, this development strengthens Rigel's position in the $2.5+ billion MDS treatment market. The orphan designation is particularly valuable given that MDS affects fewer than 200,000 people in the U.S., creating a protected niche market with high unmet needs. The combination of Fast Track and Orphan designations typically correlates with higher probability of eventual approval and commercial success.
The current MDS market lacks effective treatments for lower-risk patients who become refractory to initial therapies. R289's novel mechanism targeting inflammation through IRAK1/4 inhibition could capture significant market share if proven effective. The regulatory incentives reduce development costs by approximately 25-30%, improving the program's potential return on investment.
The ongoing Phase 1b study's focus on lower-risk MDS patients who have failed prior therapies addresses a critical treatment gap. The dual inhibition of IRAK1 and IRAK4 represents an innovative approach targeting the inflammatory bone marrow microenvironment - a key driver of disease progression in MDS.
The "encouraging initial data" mentioned by management, while not detailed, suggests positive early safety signals and potential efficacy. For context, successful treatment in this patient population would typically mean reduced transfusion dependence and improved blood counts. The Fast Track designation indicates the FDA recognizes both the serious nature of the condition and R289's potential to address this unmet need.
"Receiving Orphan Drug designation for R289 supports the development of this therapeutic candidate for the treatment of MDS and highlights the significant unmet medical need that exists for these patients," said Raul Rodriguez, Rigel's president and CEO. "Orphan Drug and Fast Track designations, along with encouraging initial data from our ongoing Phase 1b study in patients with lower-risk MDS, represent significant milestones in the advancement of R289 as a potential new treatment option."
The FDA Office of Orphan Products Development grants orphan drug designation to support the development of medicines for rare disorders that affect fewer than 200,000 people in the
R289 was previously granted Fast Track designation by the FDA for the treatment of patients with previously-treated transfusion dependent lower-risk MDS.
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.2016.00151
Forward Looking Statements
This press release contains forward-looking statements relating to, among other things, the possible advantages of Orphan Drug and Fast Track designations, the potential benefits of R289 as a therapeutic for MDS and LR-MDS, the existence of patients with an unmet medical need for such therapy, certain potential benefits associated with orphan drug designation, and Rigel's ability to further develop its clinical stage product candidates, including the encouragement of initial data from, and progress of, current clinical trials of R289, and the potential for future trials. 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 and Orphan Drug designations 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:
Investors:
Rigel Pharmaceuticals, Inc.
650.624.1232
ir@rigel.com
Media:
David Rosen
Argot Partners
646.461.6387
david.rosen@argotpartners.com
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SOURCE Rigel Pharmaceuticals, Inc.
FAQ
What is the significance of R289's Orphan Drug designation for RIGL stock?
What phase of clinical trials is RIGL's R289 currently in for MDS treatment?
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