Nurix Therapeutics Receives U.S. FDA Fast Track Designation for NX-5948 for the Treatment of Relapsed or Refractory Waldenstrom’s Macroglobulinemia
Nurix Therapeutics (NRIX) has received FDA Fast Track designation for NX-5948, its selective BTK degrader, for treating relapsed/refractory Waldenstrom's macroglobulinemia patients who have undergone at least two prior therapies, including a BTK inhibitor. This designation follows positive Phase 1 clinical trial data and adds to the drug's existing Fast Track designation for CLL/SLL treatment received in January 2024. NX-5948 also received EMA PRIME designation in November 2024 for treating CLL/SLL. The company continues patient enrollment in the Phase 1b expansion cohort, with additional clinical data expected in 2025.
Nurix Therapeutics (NRIX) ha ricevuto la designazione di Fast Track da parte della FDA per NX-5948, il suo degradatore selettivo di BTK, per il trattamento di pazienti con macroglobulinemia di Waldenström in recidiva/riferimento che hanno ricevuto almeno due terapie precedenti, compreso un inibitore di BTK. Questa designazione segue dati positivi della sperimentazione clinica di Fase 1 e si aggiunge alla designazione di Fast Track esistente per il trattamento di CLL/SLL ricevuta a gennaio 2024. NX-5948 ha anche ricevuto la designazione EMA PRIME nel novembre 2024 per il trattamento di CLL/SLL. L'azienda continua il reclutamento dei pazienti nella coorte di espansione della Fase 1b, con ulteriori dati clinici attesi nel 2025.
Nurix Therapeutics (NRIX) ha recibido la designación Fast Track de la FDA para NX-5948, su degradador selectivo de BTK, para tratar a pacientes con macroglobulinemia de Waldenström en recaída/refractaria que han recibido al menos dos terapias previas, incluyendo un inhibidor de BTK. Esta designación sigue a datos positivos de ensayos clínicos de Fase 1 y se suma a la designación de Fast Track existente para el tratamiento de CLL/SLL recibida en enero de 2024. NX-5948 también recibió la designación PRIME de la EMA en noviembre de 2024 para el tratamiento de CLL/SLL. La compañía continúa reclutando pacientes en la cohorte de expansión de Fase 1b, con datos clínicos adicionales esperados para 2025.
Nurix Therapeutics (NRIX)는 FDA로부터 선택적 BTK 분해제인 NX-5948에 대해 재발/불응성 월덴스트롬 대세포매크로글로불린혈증 환자 치료를 위한 패스트트랙 승인을 받았습니다. 이는 최소 두 가지 이전 치료, BTK 억제제를 포함한 치료를 받은 환자에게 해당됩니다. 이 승인 결정은 긍정적인 1상 임상시험 데이터에 따른 것이며, 2024년 1월에 CLL/SLL 치료를 위해 받은 기존의 패스트트랙 디자인ation에 추가됩니다. NX-5948는 2024년 11월에 CLL/SLL 치료를 위한 EMA PRIME 디자인ation도 받았습니다. 회사는 1b상 확장 코호트에 대한 환자 모집을 계속하고 있으며, 2025년에 추가적인 임상 데이터가 예상됩니다.
Nurix Therapeutics (NRIX) a reçu la désignation Fast Track de la FDA pour NX-5948, son dégradeur sélectif de BTK, destiné au traitement des patients atteints de macroglobulinémie de Waldenström en rechute/réfractaire, ayant bénéficié d'au moins deux thérapies antérieures, y compris un inhibiteur de BTK. Cette désignation fait suite à des données positives de l'essai clinique de phase 1 et vient s'ajouter à la désignation Fast Track existante pour le traitement de la CLL/SLL reçue en janvier 2024. NX-5948 a également reçu la désignation EMA PRIME en novembre 2024 pour le traitement de la CLL/SLL. L'entreprise poursuit le recrutement de patients dans la cohorte d'expansion de la phase 1b, avec des données cliniques supplémentaires attendues en 2025.
Nurix Therapeutics (NRIX) hat von der FDA den Fast Track-Status für NX-5948, seinen selektiven BTK-Degrader, erhalten, um Patienten mit rezidivierender/therapieresistenter Waldenström-Makroglobulinämie zu behandeln, die mindestens zwei vorherige Therapien, einschließlich eines BTK-Inhibitors, durchlaufen haben. Diese Auszeichnung folgt positiven Daten aus der klinischen Phase-1-Studie und ergänzt die bereits bestehenden Fast Track-Designierung des Medikaments zur Behandlung von CLL/SLL, die im Januar 2024 erhalten wurde. NX-5948 erhielt außerdem im November 2024 die EMA PRIME-Designierung für die Behandlung von CLL/SLL. Das Unternehmen setzt die Patientenrekrutierung in der Phase-1b-Erweiterungskohorte fort, mit weiteren klinischen Daten, die im Jahr 2025 erwartet werden.
- FDA Fast Track designation received for NX-5948 in Waldenstrom's macroglobulinemia
- Positive Phase 1 trial data showing safety and efficacy
- Multiple regulatory designations secured (FDA Fast Track for CLL/SLL and EMA PRIME designation)
- Ongoing Phase 1b expansion cohort enrollment
- None.
Insights
The FDA Fast Track designation for NX-5948 in Waldenstrom's macroglobulinemia (WM) represents a significant regulatory milestone that could accelerate the drug's path to market. The designation, coupled with previous Fast Track for CLL/SLL and EMA's PRIME designation, creates a triple regulatory advantage that substantially de-risks the development pathway.
This development is particularly noteworthy as NX-5948, a selective BTK degrader, targets a critical market opportunity in the second-line-plus WM setting. The growing population of BTK inhibitor-resistant patients represents an expanding commercial opportunity. BTK degraders offer a mechanistically distinct approach compared to inhibitors, potentially overcoming resistance mechanisms.
For investors, this translates to: 1) Accelerated development timeline, reducing cash burn concerns 2) Enhanced probability of regulatory success 3) Stronger market positioning in an increasingly valuable therapeutic space. The combination of multiple expedited designations across different indications strengthens Nurix's negotiating position for potential partnerships or licensing deals.
The clinical implications of NX-5948's development are compelling from a therapeutic perspective. WM, a rare B-cell lymphoma, has treatment options after BTK inhibitor failure. A BTK degrader approach could be transformative because it: 1) Addresses the fundamental mechanism of resistance 2) May achieve more complete BTK suppression than inhibitors 3) Could potentially deliver more durable responses.
The positive Phase 1 data mentioned suggests early signs of efficacy, though specific metrics weren't disclosed. The timing for additional data in 2025 aligns with typical development timelines for targeted therapies in hematologic malignancies. The dual development strategy in both WM and CLL/SLL is strategically sound, as these indications share biological pathways and often respond to similar therapeutic approaches.
Fast track designation follows positive Phase 1 data presented at the 12th International Workshop on Waldenstrom’s Macroglobulinemia
SAN FRANCISCO, Dec. 19, 2024 (GLOBE NEWSWIRE) -- Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical stage biopharmaceutical company developing targeted protein modulation drugs designed to treat patients with cancer and inflammatory diseases, today announced 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.
“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).
About Nurix
Nurix Therapeutics is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of innovative small molecules and antibody therapies based on the modulation of cellular protein levels as a novel treatment approach for cancer, inflammatory conditions, and other challenging diseases. Leveraging extensive expertise in E3 ligases together with proprietary DNA-encoded libraries, Nurix has built DELigase, an integrated discovery platform, to identify and advance novel drug candidates targeting E3 ligases, a broad class of enzymes that can modulate proteins within the cell. Nurix’s drug discovery approach is to either harness or inhibit the natural function of E3 ligases within the ubiquitin-proteasome system to selectively decrease or increase cellular protein levels. Nurix’s wholly owned, clinical stage pipeline includes targeted protein degraders of Bruton’s tyrosine kinase, a B-cell signaling protein, and inhibitors of Casitas B-lineage lymphoma proto-oncogene B, an E3 ligase that regulates activation of multiple immune cell types including T cells and NK cells. Nurix is headquartered in San Francisco, California. For additional information visit http://www.nurixtx.com.
Forward-Looking Statements
This press release contains statements that relate to future events and expectations and as such constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. When or if used in this press release, the words “anticipate,” “believe,” “could,” “estimate,” “expect,” “intend,” “may,” “outlook,” “plan,” “predict,” “should,” “will,” and similar expressions and their variants, as they relate to Nurix, may identify forward-looking statements. All statements that reflect Nurix’s expectations, assumptions or projections about the future, other than statements of historical fact, are forward-looking statements, including, without limitation, statements regarding Nurix’s plans and strategies with respect to NX-5948, the potential advantages and therapeutic benefits of NX-5948, including its potential role in the treatment of B-cell malignancies, including Waldenstrom’s macroglobulinemia, the planned timing for the provision of updates from the NX-5948 clinical trial, and the potential benefits of Fast Track designation. Forward-looking statements reflect Nurix’s current beliefs, expectations, and assumptions. Although Nurix believes the expectations and assumptions reflected in such forward-looking statements are reasonable, Nurix can give no assurance that they will prove to be correct. Forward-looking statements are not guarantees of future performance and are subject to risks, uncertainties and changes in circumstances that are difficult to predict, which could cause Nurix’s actual activities and results to differ materially from those expressed in any forward-looking statement. Such risks and uncertainties include, but are not limited to: (i) the risks inherent in the drug development process, including the unexpected emergence of adverse events or other undesirable side effects during clinical development; (ii) uncertainties related to the timing and results of clinical trials; (iii) whether Nurix will be able to fund its research and development activities and achieve its research and development goals; (iv) the impact of economic and market conditions and global and regional events on Nurix’s business, clinical trials, financial condition, liquidity and results of operations; (v) whether Nurix will be able to protect intellectual property and (vi) other risks and uncertainties described under the heading “Risk Factors” in Nurix’s Quarterly Report on Form 10-Q for the fiscal period ended August 31, 2024, and other SEC filings. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. The statements in this press release speak only as of the date of this press release, even if subsequently made available by Nurix on its website or otherwise. Nurix disclaims any intention or obligation to update publicly any forward-looking statements, whether in response to new information, future events, or otherwise, except as required by applicable law.
Contacts:
Investors
Jason Kantor, Ph.D.
Nurix Therapeutics
ir@nurixtx.com
Elizabeth Wolffe, Ph.D.
Wheelhouse Life Science Advisors
lwolffe@wheelhouselsa.com
Media
Aljanae Reynolds
Wheelhouse Life Science Advisors
areynolds@wheelhouselsa.com
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¹ Bibas M., Sarosiek S., Castillo J.J. Waldenström Macroglobulinemia - A State-of-the-Art Review: Part 1: Epidemiology, pathogenesis, clinicopathologic characteristics, differential diagnosis, risk stratification, and clinical problems. Mediterr J Hematol Infect Dis 2024, 16(1): e2024061.
² McMaster ML. The Epidemiology of Waldenström Macroglobulinemia. Semin Hematol. 2023 March; 60(2): 65–72.
³ Kyle Robert A, et al, 50 Year Incidence of Waldenström Macroglobulinemia in Olmsted County, Minnesota From 1961–2010: A Population-Based Study With Complete Case Capture and Hematopathologic Review. Mayo Clin Proc. 2018; 93(6): 739–746.
⁴ Gertz M.A., et.al., Waldenstrom Macroglobulinemia: 2023 update on diagnosis, risk stratification, and management. Am J Hematol. 2023;98(1):348-358.
FAQ
What is the significance of FDA Fast Track designation for NRIX's NX-5948?
When will NRIX release additional clinical data for NX-5948?
What indications has NX-5948 received Fast Track designation for?
What regulatory designations has NRIX's NX-5948 received in 2024?
