On December 19, 2024 Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical stage biopharmaceutical company developing targeted protein modulation drugs designed to treat patients with cancer and inflammatory diseases, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for NX-5948, a highly selective degrader of Bruton’s tyrosine kinase (BTK), for the treatment of adult patients with relapsed or refractory Waldenstrom’s macroglobulinemia (WM) after at least two lines of therapy, including a BTK inhibitor (Press release, Nurix Therapeutics, DEC 19, 2024, View Source [SID1234649216]).
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"Fast Track designation for NX-5948 is an important recognition of the unmet patient need in Waldenstrom’s macroglobulinemia, particularly in the growing number of patients whose cancer has progressed following BTK inhibitor therapy," said Arthur T. Sands, M.D., Ph.D., president and chief executive officer of Nurix. "This designation follows encouraging safety and efficacy data from our ongoing Phase 1 clinical trial, demonstrating early promise of clinical benefit with potential for durable outcomes. We continue to enroll Waldenstrom’s macroglobulinemia patients in the ongoing Phase 1b expansion cohort and anticipate sharing additional clinical data in 2025."
In addition to the Fast Track designation announced today for Waldenstrom’s macroglobulinemia, NX-5948 previously received Fast Track designation in January 2024 for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) after at least two lines of therapy, including a BTK inhibitor and a B-cell lymphoma 2 (BCL2) inhibitor. In November 2024, the European Medicines Agency (EMA) granted NX-5948 PRIME designation for the treatment of adult patients with relapsed or refractory CLL/SLL after at least a BTK inhibitor and a BCL2 inhibitor.
About Waldenstrom’s Macroglobulinemia
WM is a rare, slow growing type of non-Hodgkin’s lymphoma that is characterized by the replacement of normal bone marrow cells by malignant lymphocytic cells that produce monoclonal IgM. This replacement leads to anemia, bleeding, and impaired immune function, while the elevated IgM levels may cause neurologic symptoms. The incidence of Waldenstrom’s macroglobulinemia ranges from 0.361,2 to 0.573 per 100,000 people in the United States or approximately 1,200 to 1,900 annually. With a median disease duration approaching 10 years,4 approximately 12,000 to 19,000 patients are living with Waldenstrom’s macroglobulinemia in the United States. Recommended first-line treatments including chemoimmunotherapy and BTK inhibitor therapy. There are no therapies approved to treat WM patients after BTKi.
About Fast Track Designation
The FDA’s Fast Track designation is intended to facilitate and expedite the development and review of drug candidates to treat serious conditions and fulfill an unmet medical need. To qualify, available clinical and non-clinical data need to demonstrate a therapeutic candidate’s potential to address this unmet medical need. A therapeutic candidate that receives Fast Track designation may be eligible for more frequent interactions with the FDA to discuss the candidate’s development plan and, if relevant criteria are met, eligibility for Accelerated Approval and Priority Review.
About PRIME Designation
The PRIME initiative, launched by the EMA in 2016, offers early, proactive and enhanced support to developers of promising medicines to optimize development plans and accelerate evaluation so these medicines can reach patients faster. To be eligible for PRIME, medicines must target an unmet medical need and show potential benefit for patients based on early clinical data.
About NX-5948
NX-5948 is an investigational, orally bioavailable, brain penetrant, small molecule degrader of BTK. NX-5948 is currently being evaluated in a Phase 1 clinical trial in patients with relapsed or refractory B cell malignancies. Nurix has previously reported that NX-5948 is highly potent against a range of tumor cell lines that are resistant to current BTK inhibitor therapies, an important consideration in heavily pretreated CLL/SLL patient populations. Additional information on the ongoing clinical trial can be accessed at clinicaltrials.gov (NCT05131022).