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NX-5948 is an investigational medicine being developed by Nurix Therapeutics for certain B-cell blood cancers. It has no marketing authorisation and is given as capsules for oral use.[1] The substance has received orphan designation, meaning it targets a rare disease.[1]
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NX-5948 is a chemically synthesised substance classified in the therapeutic area of neoplasms (cancers).[1] It is an investigational medicine, meaning it has no marketing authorisation in the product records and is still being studied in clinical trials.[1] The substance is supplied by Nurix Therapeutics and carries an orphan designation for a rare disease.[1]
NX-5948 is designed to target a protein called Bruton's tyrosine kinase (BTK), which helps cancer cells grow in certain blood cancers such as CLL and SLL. Other medicines called BTK inhibitors can slow down or stop BTK, but cancer cells sometimes stop responding to them. NX-5948 works differently: it destroys the BTK protein rather than merely blocking it.[3]
In a separate early-phase study listed in the UK, NX-5948 is also being evaluated in other advanced B-cell malignancies, including diffuse large B-cell lymphoma, follicular lymphoma, mantle cell lymphoma, marginal zone lymphoma, Waldenström macroglobulinemia, and primary or secondary central nervous system lymphoma.[2]
NX-5948 is given as capsules for oral use, taken once daily in continuous 28-day cycles.[3] In the EU Phase 2 trial, the dose is 600 mg.[1]
One Phase 2 trial of NX-5948 is registered in the EU Clinical Trials Information System. It is currently authorised and plans to enroll 110 adults with relapsed or refractory CLL or SLL who have previously been exposed to a covalent BTK inhibitor, a non-covalent BTK inhibitor, and a BCL-2 inhibitor.[1] The study is single-arm, open-label, and multicenter, meaning all participants receive NX-5948 and both researchers and participants know which medicine is being given.[3] A separate first-in-human Phase 1a/1b study is also listed in UK sources for patients with advanced B-cell malignancies.[2]
The EU Phase 2 trial is open to adults aged 18 and over with relapsed or refractory CLL or SLL who have previously been treated with a Bruton's tyrosine kinase inhibitor and a B-cell lymphoma-2 inhibitor.[1] The study follows the 2018 International Workshop on CLL guidelines for assessing response.[1] The UK-listed early-phase study is open to adults with a wider range of advanced B-cell malignancies.[2]
The primary outcome of the EU Phase 2 trial is the overall response rate (ORR) without partial response with lymphocytosis (PR-L), assessed by an independent review committee.[1] The study also evaluates safety and tolerability, pharmacodynamics (the effect of the drug on the body), and pharmacokinetics (how the body processes the drug).[3] Participants are followed for safety after treatment stops and then at 12-week intervals for long-term follow-up of overall health.[3]
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