K36 Therapeutics' Breakthrough Data on KTX-1001 Sets Stage for Targeted Multiple Myeloma Treatment at Upcoming ASH Meeting

K36 Therapeutics is poised to present critical clinical findings that advance the scientific rationale for MMSET/NSD2 inhibition as a therapeutic strategy in t(4;14) multiple myeloma. The company will showcase multiple presentations at the 66th American Society of Hematology Annual Meeting and Exposition, scheduled for December 6-10, 2024, in San Diego, California.

Clinical Progress Demonstrates Target Engagement

The centerpiece of K36 Therapeutics’ presentation agenda features initial Phase 1 dose escalation results for KTX-1001, an oral, first-in-class inhibitor of MMSET. Early trial data reveals a dose-dependent increase in drug exposure coupled with corresponding reductions in the H3K36me2 biomarker—a key indicator of target engagement. This pharmacodynamic activity aligns with preclinical predictions and supports continued development.

According to Benjamin Winograd, M.D., Ph.D., Chief Medical Officer of K36 Therapeutics, “The safety profile observed at therapeutically relevant doses provides a strong foundation to advance into the dose expansion phase. We anticipate initiating combination studies in 2025, pairing our investigational oral agent with standard-of-care approaches including proteasome inhibitors and immunomodulatory drugs to optimize treatment for t(4;14) patients.”

The trial, characterized as a single-arm, open-label study in relapsed and refractory multiple myeloma (RRMM) patients, has demonstrated favorable tolerability alongside encouraging clinical activity. Pierre Bories, MD, PhD, Hematologist at Toulouse University Cancer Institute, noted that the dose-escalation phase clearly validates MMSET as a viable therapeutic target for this high-risk patient population.

Three Poster Presentations Highlight Mechanism and Preclinical Validation

Mechanistic Insights from Biochemical Characterization

K36 Therapeutics will present detailed biochemical analysis of KTX-1001’s mechanism using surface plasmon resonance (SPR) technology. The data illustrate how the compound achieves H3K36me2 reduction by displacing S-adenosyl methionine (SAM), the essential cofactor responsible for methylating H3K36. This novel analytical approach provides unprecedented clarity on KTX-1001’s molecular action.

Preclinical Efficacy of KTX-1029 Program

A companion presentation will showcase in vitro and in vivo efficacy data for KTX-1029, another potent MMSET selective inhibitor developed by K36 Therapeutics. KTX-1029 demonstrated robust single-agent activity in t(4;14) multiple myeloma models and showed synergistic potential when combined with established proteasome inhibitors (bortezomib and carfilzomib) across both sensitive and resistant disease settings. These findings reinforce the therapeutic hypothesis underlying MMSET targeting.

Meeting the Unmet Medical Need

Terry Connolly, Ph.D., President and Chief Executive Officer of K36 Therapeutics, emphasized the clinical significance: “Enrollment momentum has accelerated substantially since our initial ASH presentation last year. This reflects genuine investigator and patient interest in oral therapeutic options for high-risk multiple myeloma. For patients with t(4;14) who have exhausted conventional treatments, KTX-1001 represents a genuinely novel therapeutic approach designed specifically for their disease biology.”

The collective data presented by K36 Therapeutics underscore both the scientific rationale and practical potential of MMSET inhibition in transforming outcomes for a particularly challenging patient subset. Combined clinical, biochemical, and preclinical evidence from KTX-1001 and KTX-1029 construct a compelling narrative for this emerging therapeutic class.

About K36 Therapeutics and Future Development Plans

Established in February 2021, K36 Therapeutics is a privately held biotechnology company supported by prominent venture investors including Atlas Venture, F-Prime Capital, Eight Roads Ventures, Nextech, and Bristol Myers Squibb (NYSE:BMS). The company’s mission centers on translating epigenetic pathway modulation into first-in-class small molecule therapeutics for oncology patients.

For additional information about the Phase 1 trial (NCT05651932) and participating clinical centers, interested parties can visit the trial registry. More details about K36 Therapeutics are available at www.k36tx.com, with regular updates shared via LinkedIn.