K36 Therapeutics to Present New Preclinical Data Supporting Ongoing Phase 1 Clinical Trial of KTX-1001 at the 22nd Annual International Myeloma Society Meeting

On September 18, 2025 K36 Therapeutics, Inc. ("K36"), a privately held clinical-stage biotechnology company developing novel, targeted therapies for cancers with unmet medical need, reported the presentation of two posters at the 22nd Annual International Myeloma Society (IMS) Meeting in Toronto, taking place September 17-20 (Press release, K36 Therapeutics, SEP 18, 2025, View Source [SID1234656079]). 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).

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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.