Nurix Therapeutics has Received FDA’s Fast Track Designation for NX-5948 in Waldenstrom’s Macroglobulinemia

On 19th December, Nurix Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to NX-5948.

NX-5948 is an investigational, is an orally administered chimeric targeting molecule (CTM) that specifically degrades the BTK protein through the cereblon E3 ligase (CRBN) complex, without affecting other cereblon neo-substrates. By degrading BTK, NX-5948 exerts strong anti-inflammatory effects, inhibiting B cell activation.

Nurix Therapeutics is a clinical-stage biopharmaceutical company focused on developing targeted protein modulation drugs for cancer and inflammatory diseases.

This highly selective degrader of Bruton’s tyrosine kinase (BTK) is being developed for adult patients with relapsed or refractory Waldenstrom’s macroglobulinemia (WM) after at least two prior therapies, including a BTK inhibitor.

“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.

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.” – said Arthur T. Sands, M.D., Ph.D., president and chief executive officer of Nurix.

In addition to this designation, NX-5948 also received Fast Track status in January 2024 for relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) in patients who have received at least two prior therapies, including a BTK inhibitor and a BCL2 inhibitor.

Furthermore, 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.

About Waldenstrom’s Macroglobulinemia (WM)

WM is a rare, slow-growing form of non-Hodgkin’s lymphoma characterized by the infiltration of malignant lymphocytes into the bone marrow, resulting in monoclonal IgM production. This can cause anemia, bleeding, and impaired immune function, with elevated IgM levels potentially leading to neurological symptoms. The incidence in the U.S. ranges from 0.36 to 0.57 per 100,000 people, with approximately 12,000 to 19,000 individuals living with WM in the country. There are currently no approved therapies for patients with WM who have failed BTK inhibitor treatment.

About FDA Fast Track Designation

The Fast Track designation is a program established by the U.S. Food and Drug Administration (FDA) to facilitate the development and expedite the review of drugs intended to treat serious conditions and fill unmet medical needs.

The FDA’s Fast Track designation is designed to speed up the development and review of drugs that treat serious conditions and fulfill an unmet medical need. Drugs with this designation are eligible for more frequent interactions with the FDA and, if specific criteria are met, may qualify for Accelerated Approval or Priority Review.

About EMA’s PRIME Designation

In November 2024, NX-5948 received the European Medicines Agency (EMA) PRIME designation for the treatment of adult patients with relapsed or refractory CLL/SLL.

Launched in 2016, the Priority Medicines (PRIME) desi enhances EMA support for developing medicines that address unmet medical needs, aiming to provide early patient access. The EMA’s PRIME initiative supports promising medicines with early and enhanced development plans, aiming to bring treatments to patients more quickly.

About NX-5948

NX-5948 is an investigational, orally bioavailable small molecule that selectively degrades BTK. Currently being tested in a Phase 1 clinical trial, NX-5948 has shown strong potency against tumor cell lines resistant to existing BTK inhibitors, which is crucial for treating heavily pretreated CLL/SLL patients.

By degrading BTK, NX-5948 exerts strong anti-inflammatory effects, inhibiting B cell activation. It shows significant tumor growth inhibition in TMD8 xenograft models with either wild-type BTK or BTK inhibitor-resistant mutations. NX-5948 also demonstrates effectiveness in a mouse model of collagen-induced arthritis (CIA) and is capable of crossing the blood-brain barrier (BBB).

About Nurix Therapeutics

Nurix Therapeutics is a clinical-stage biopharmaceutical company specializing in the discovery and development of innovative small molecules and antibody therapies that modulate cellular protein levels to treat cancer, inflammatory diseases, and other challenging conditions.

Using its DELigase platform, Nurix leverages the natural function of E3 ligases in the ubiquitin-proteasome system to create drug candidates that can selectively modulate protein levels inside cells. The company is based in San Francisco, California.

Further Reading:

FDA Grants Fast Track Designation to Cothera Bioscience’s PC-002