On January 9, 2025 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to R289 for the treatment of myelodysplastic syndromes (MDS) (Press release, Rigel, JAN 9, 2025, View Source [SID1234649549]). R2891, Rigel’s potent and selective dual inhibitor of IRAK1 and IRAK4, is being studied in an ongoing Phase 1b study evaluating the safety, tolerability, pharmacokinetics and preliminary activity in patients with LR-MDS who are relapsed or refractory to prior therapies.
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"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 U.S. Under the Orphan Drug Act, orphan drug designation qualifies a company for incentives including tax credits, exemptions from certain FDA fees for clinical trials and the potential for seven years of market exclusivity following drug approval.
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.