First-in-human data highlights safety, pharmacodynamics, and single-agent activity supporting MMSET/NSD2 inhibition in heavily pre-treated multiple myeloma patients

Shinta Cheng, M.D., Ph.D., joins K36 to drive clinical development of lead programs and oncology pipeline expansion

CAMBRIDGE, Mass., Dec. 5, 2025 /PRNewswire/ — K36 Therapeutics, Inc. (“K36”), a clinical-stage biotechnology company developing first-in-class MMSET/NSD2 inhibitors for genetically defined cancers, today announced that clinical data from its lead program, gintemetostat (KTX-1001) will be presented at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, taking place December 6 -9, 2025, in Orlando, Florida. The oral presentation will feature the results from the dose escalation part of the Phase 1 MMSET study NCT05651932.

“I’m looking forward to sharing the clinical progress of gintemetostat (KTX-1001), a potent oral NSD2/MMSET inhibitor being developed for patients with t(4;14) multiple myeloma,” said Saad Usmani, M.D., MBA, Chief of Myeloma Service at Memorial Sloan Kettering Cancer Center. “In the dose-escalation phase, gintemetostat monotherapy showed a favorable safety and tolerability profile and demonstrated disease control and efficacy. Pharmacodynamic data confirm target engagement, and we look forward to advancing into the dose-expansion phase to evaluate combinations with proteasome inhibitors, IMiDs^™, and next-generation CELMoDs^™ such as mezigdomide.”

In parallel with this important clinical milestone, K36 is continuing to strengthen its leadership team to support the advancement of its expanding clinical pipeline. The company also announced the appointment of Shinta Cheng, M.D., Ph.D., a seasoned clinical leader with more than 20 years oncology and hematology drug development experience, including in prostate cancer and multiple myeloma, as its new Chief Medical Officer.

“We are highly encouraged by the durable disease control achieved with oral, single-agent gintemetostat, and by the strength of our emerging clinical data, including patients who continue to experience long-standing benefit while remaining on monotherapy,” said Dr. Cheng, Chief Medical Officer of K36 Therapeutics. “Gintemetostat is a first-in-class MMSET inhibitor with a novel mechanism of action and is being evaluated in combination with both standard and next-generation therapies. Its favorable safety and tolerability profile further support its potential as a foundational therapy for patients with t(4;14) multiple myeloma. We remain deeply grateful to the investigators, patients, and partners whose commitment enables our rapid clinical progress.”

Dr. Cheng was most recently vice president, clinical development at SpringWorks Therapeutics, where he led clinical collaborations combining nirogacestat with therapies targeting BCMA in multiple myeloma as well as global registration programs for nirogacestat in desmoid tumors and mirdametinib in plexiform neurofibromas in NF1 patients. Previously, he led development of apalutamide and niraparib in prostate cancer at Johnson & Johnson Innovative Medicines. He was also Asia-Pacific immuno-oncology development lead for nivolumab and ipilimumab at Bristol-Myers Squibb, where he advanced early clinical studies of dasatinib and first-in-human androgen receptor antagonists in prostate cancer. Board-certified in internal medicine, hematology, and medical oncology, he trained at Beth Israel Deaconess Medical Center, Harvard Medical School. He holds an M.D. and Ph.D. in viral oncogenesis from the University of Rochester School of Medicine.

Oral presentation details are outlined below:

Title: Phase 1 study of KTX-1001, a first-in-class oral MMSET/NSD2 inhibitor, demonstrates clinical activity in relapsed/refractory multiple myeloma
Date & Time: December 6, 2025, 2:45 PM – 3:00 PM EST
Location: Orange County Convention Center – West Hall D1

“We are fortunate to welcome Shinta to K36 as we enter a period of significant clinical expansion,” said Terry Connolly, Ph.D., Chief Executive Officer of K36 Therapeutics. “As we advance two first-in-class NSD2 inhibitor programs across multiple myeloma and prostate cancer, Shinta’s deep expertise in both diseases and his proven track record guiding therapies from early development through registration, comes at exactly the right moment. With our prostate cancer program now initiated and our multiple myeloma program expanding, his leadership will be pivotal in shaping this next phase of growth and strengthening our position in the emerging field of cancer epigenetics.”

The full abstracts can be found at the ASH Annual Meeting website at www.Hematology.org.

About Gintemetostat (KTX-1001)
Gintemetostat (KTX-1001) is a novel, first-in-class, potent, and selective inhibitor of the methyltransferase activity of MMSET/NSD2. It is an orally administered small molecule being developed for the treatment of relapsed and refractory multiple myeloma, with an initial focus on patients harboring the t(4;14) translocation. By targeting the underlying epigenetic driver of this high-risk subset, gintemetostat offers a promising avenue for patients with limited treatment options.

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 myeloma research and treatment.

About the KTX-1001 Phase 1 MMSET Clinical Trial
The Phase 1 clinical trial is a single-arm, open-label study in participants with relapsed and refractory multiple myeloma. It is a multi-part study with dose escalation followed by an expansion cohort in patients with the genetic translocation t(4;14) to evaluate the safety, tolerability, and preliminary efficacy of different doses of KTX-1001 in combination with standard of care and mezigdomide. For more information and participating centers, visit NCT05651932 and EUCTR: 2022-500801-41-00.

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 www.k36tx.com and follow us on LinkedIn.

CONTACTS:

K36 Therapeutics
Soo Bang
[email protected]

MEDIA:
Sarah Sutton
(518) 932-3680
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