Mezigdomide

Bristol Myers Squibb said a regimen that includes its experimental drug iberdomide led to no detectable cancer cells in a greater proportion of multiple myeloma patients than those who received standard treatments in a pivotal trial. The New Jersey pharma company appears poised to test the waters of a potential accelerated approval based on the outcome, though it shied away from explicitly saying so in Tuesday’s press release . The pivotal portion of the Phase 3 EXCALIBER-RRMM trial had 664 patients with multiple myeloma that returned or stopped responding to treatment. The participants were given the experimental regimen, which consisted of iberdomide, Johnson & Johnson’s Darzalex and dexamethasone, or the standard regimen of bortezomib alongside the latter two drugs. Bristol Myers said the iberdomide arm had a statistically significant improvement in minimal residual disease (MRD) negativity rates in a planned interim analysis compared to the control group. MRD negativity means there are no detectable cancer cells, though it does not necessarily convey that all cancer cells are gone. An FDA advisory committee last year backed the use of ​​minimal residual disease as an endpoint in accelerated approvals for multiple myeloma drugs. Bristol Myers then changed the design of its study in January to make MRD negativity a dual primary endpoint, according to clinicaltrials.gov . In other words, the study only needs to hit that endpoint or its other primary endpoint of progression-free survival to succeed (as opposed to hitting both endpoints). Other large drugmakers like Gilead have likewise added MRD negativity as a primary endpoint for their multiple myeloma trials in the hopes of hustling their experimental drugs to approval. The FDA has not yet approved any multiple myeloma drugs using MRD negativity as a primary endpoint. Bristol Myers could be the first to ask the FDA to do so. “The company plans to discuss these results with health authorities,” Bristol Myers said. It also noted in the release that “MRD is increasingly being used in clinical trials as a surrogate endpoint for progression-free survival (PFS) and is gaining recognition from regulatory authorities for its role in accelerating therapeutic development.” The company did not immediately respond to a request for comment about its regulatory plans. Iberdomide is a next-generation targeted protein degrader, akin to Bristol Myers’ blockbuster multiple myeloma drug Revlimid. The way Revlimid works — by binding to a part of the body’s natural protein recycling system — was discovered after its approval, and drug developers have since set out to purposefully make new drugs that leverage that system. In multiple myeloma, tagging certain proteins for recycling leads to the death of the cancer cells. Bristol Myers is also developing another protein degrader for multiple myeloma called mezigdomide. The drugmaker said it’s continuing the Phase 3 trial of iberdomide for the other dual primary endpoint of progression-free survival as well as the key secondary endpoint of overall survival. Editor’s note: This story was corrected to note the FDA has not approved any multiple myeloma drugs using MRD as a primary endpoint.

Two posters highlight the differentiated approach of KTX-1001 as a potential first-in-class therapy for patients with relapsed or refractory multiple myeloma who have exhausted standard options CAMBRIDGE, Mass., Sept. 18, 2025 /PRNewswire/ -- K36 Therapeutics, Inc. ("K36"), a privately held clinical-stage biotechnology company developing novel, targeted therapies for cancers with unmet medical need, today announced the presentation of two posters at the 22nd Annual International Myeloma Society (IMS) Meeting in Toronto, taking place September 17-20. The presentations will feature preclinical and translational data from K36's lead program, KTX-1001, a first-in-class targeted therapy currently being evaluated in an ongoing Phase 1 clinical trial for patients with relapsed or refractory multiple myeloma (MM). KTX-1001 is a selective inhibitor of NSD2, also known as MMSET, a histone methyltransferase frequently dysregulated in patients with the t(4;14) chromosomal translocation, a high-risk subtype of multiple myeloma. K36 recently began dosing patients in multiple cohorts of its Phase 1b dose-expansion study assessing KTX-1001 in combination with standard of care and mezigdomide for t(4;14) MM. The new data presented at IMS highlight the mechanism of action and potential clinical utility of KTX-1001. The data include bone marrow analyses from patients enrolled in the Phase 1 trial that support its clinical relevance. Additional findings from large multi-omics datasets identify a subgroup of newly diagnosed patients with high NSD2 expression in the absence of the t(4;14) translocation, highlighting the opportunity to expand investigation of NSD2 inhibition in patients that lack the translocation. Together, these findings provide important validation of KTX-1001's differentiated mechanism and support its continued development as a first-in-class targeted therapy for multiple myeloma. "These new findings strengthen the scientific rationale supporting KTX-1001 as a treatment for multiple myeloma and demonstrate how its differentiated mechanism of action can be translated into the clinic," said Erin Flynt, PhD, Executive Director of Translational Medicine, K36 Therapeutics. "Importantly, the identification of a distinct subgroup of patients with high NSD2 expression beyond the t(4;14) population highlights the potential to broaden the clinical development of KTX-1001 and expand its impact for patients with multiple myeloma." Poster Details: Anti-adhesion Properties of KTX-1001, a Selective NSD2/MMSET Inhibitor, Enhance Carfilzomib Sensitivity in Multiple Myeloma Poster Number: PA-293 Location: Exhibit Hall, Hall E Presentation Time: Friday, September 19, 12:15 - 1:15 p.m. ET High Expression of NSD2 in Non-t(4;14) Newly-diagnosed Multiple Myeloma Patients May Mimic t(4;14) Biology Poster Number: PA-259 Location: Exhibit Hall, Hall E Presentation time: Thursday, September 18, 12:00 – 1 p.m. ET Full abstracts can be found at the IMS Annual Meeting website. About KTX-1001 KTX-1001 is a novel, first-in-class, potent, and selective methyltransferase inhibitor of the catalytic activity of MMSET/NSD2. It is an orally administered small molecule developed initially for the treatment of relapsed and refractory multiple myeloma, with a focus on patients with the t(4;14) translocation. This inhibitor offers a promising avenue for addressing this challenging high risk patient population. About Multiple Myeloma Multiple myeloma (MM) is the second most common hematologic malignancy, driven by the uncontrolled proliferation of plasma cells in the bone marrow. According to the American Cancer Society, approximately 36,000 new cases are diagnosed each year. While recent therapeutic advances have extended survival, MM remains incurable, and most patients eventually relapse. High-risk MM, defined by genetic abnormalities such as t(4;14) and other adverse prognostic markers, is associated with aggressive disease biology, shorter survival, and limited benefit from standard-of-care regimens. Addressing this high-risk population represents one of the greatest unmet needs in MM research and treatment. About K36 Therapeutics, Inc. Founded in February 2021, K36 Therapeutics is a privately held biotechnology company backed by Atlas Venture, F-Prime , Eight Roads Ventures, Nextech and Bristol Myers Squibb. Our mission is to translate epigenetic modulation of oncogenic pathways into first-in-class small molecule therapeutics for the benefit of cancer patients worldwide. For more information, please visit and follow us on LinkedIn. CONTACTS K36: Soo Bang [email protected] Media: Sarah Sutton (518) 932-3680 [email protected] SOURCE K36 Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED