K36 Therapeutics Announces Multiple Poster Presentations Highlighting the First Clinical Data for KTX-1001 and Other Developmental Compounds at the 66th American Society of Hematology (ASH) Annual Meeting

Results collectively support the mechanism of action and rationale for targeting MMSET in patients with multiple myeloma

CAMBRIDGE, Mass., Dec. 3, 2024 /PRNewswire/ -- K36 Therapeutics, Inc. ("K36"), a clinical-stage biotech company focused on developing its first-in-class MMSET / NSD2 inhibitor KTX-1001 for t(4;14) multiple myeloma, today announced upcoming poster presentations outlining data from its KTX-1001 and KTX-1029 programs at the 66^th American Society of Hematology (ASH) Annual Meeting and Exposition held December 6-10, 2024 in San Diego, California.

The KTX-1001 presentations will feature the first results from the dose escalation part of the Phase 1 study and characterization of the asset's biochemical activity. A third poster will characterize the in vitro and in vivo efficacy of KTX-1029, a novel, potent, selective inhibitor of MMSET. Taken together, the results collectively support the mechanism of action and rationale for targeting MMSET in patients with multiple myeloma.

"I am excited to report on the clinical progress of KTX-1001, a potent, oral MMSET inhibitor being developed for multiple myeloma patients with translocation t(4;14). The dose-escalation phase of the clinical trial demonstrates an increase in KTX-1001 exposure by dose and a corresponding decrease in H3K36me2 biomarker, reflecting clear target engagement that is consistent with preclinical models" said Pierre Bories, MD, PhD, Hematologist, Early Phase Clinical Research Unit Hematology and Clinical Research, Onco-Occitanie Network, Toulouse University Cancer Institute Oncopole.

"We've seen significant momentum in enrollment since our first presentation at ASH last year, highlighting the opportunity for KTX-1001 to meet the unmet need for oral therapies in high-risk patients," said Terry Connolly, Ph.D., President and Chief Executive Officer of K36. "I want to thank the patients, investigators, and their teams for their commitment to generating crucial data from our trial. KTX-1001 has shown a favorable tolerability profile, and promising clinical activity reinforcing its potential as a first in class targeted therapy for t(4;14) multiple myeloma patients who have exhausted standard treatments."

Additional presentation details are outlined below:

Title: First Results from the Dose Escalation Part of the Phase 1 Study of KTX1001, an Oral, First-in-Class, Potent Inhibitor of MMSET/NSD2 for Relapsed/Refractory Multiple Myeloma (RRMM)
Poster Number: 3370
Session Name: 654. Multiple Myeloma: Pharmacologic Therapies: Poster II
Date & Time: Sunday, December 8, 2024, 6:00 PM-8:00 PM PST

"This trial highlights the potential of personalized oral therapies like KTX-1001 to treat multiple myeloma with a targeted medicine designed for high-risk patients," said Benjamin Winograd, M.D. Ph.D., K36's Chief Medical Officer. "The safety profile of the oral therapy at relevant doses allows us to move forward with the dose expansion phase of the trial. In 2025, we will combine our oral investigational drug with standard of care agents, starting with a proteasome inhibitor and IMiDS to continue the development of KTX-1001 for the treatment of t(4;14) patients"

Title: Characterization of the Biochemical Activity of KTX-1001, a Selective Small Molecule NSD2 Inhibitor, in Surface Plasmon Resonance (SPR)
Poster Number: 2205
Session Name: 802. Chemical Biology and Experimental Therapeutics
Date & Time: Saturday, December 7, 2024, 5:30 PM-7:30 PM PST

• Describes a novel method for utilizing surface plasmon resonance (SPR) to interrogate the binding of KTX-1001 to the SET domain of MMSET.
• These data demonstrate that one of the ways that KTX-1001 reduces H3K36me2 is by displacing S-adenosyl methionine (SAM), the cofactor responsible for donating the methyl groups to H3K36.

Title: KTX-1029, a Potent, Selective MMSET/NSD2 Inhibitor Is Effective in t(4;14) Multiple Myeloma Preclinical Models
Poster Number: 1878
Session Name: 651. Multiple Myeloma and Plasma Cell Dyscrasias
Date & Time: Saturday, December 7, 2024, 5:30 PM-7:30 PM PST

• Describes the in vitro and in vivo efficacy of KTX-1029, a novel, potent, selective inhibitor of MMSET. KTX-1029 demonstrated efficacy in MM preclinical models as a single agent and in combination with the proteasome inhibitors bortezomib and carfilzomib in both PI-sensitive and -resistant settings.
• The data generated with KTX-1029 compliments the data generated with the company's orally-available clinical candidate, KTX-1001 and adds to the body of evidence for targeting MMSET in multiple myeloma patients with t(4;14) and for further exploration of combination regimens with multiple myeloma standards of care.

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

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 the KTX-1001 Phase 1 Clinical Trial
The Phase 1 clinical trial is a single-arm, open-label study in subjects with relapsed and refractory multiple myeloma. It is a multi-part clinical trial 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. For more information and participating centers visit NCT05651932. 

About K36 Therapeutics, Inc.
Founded in February 2021, K36 is a privately held biotech company backed by Atlas Venture, F-Prime Capital, Eight Roads Ventures, Nextech and Bristol Myers Squibb (NYSE:BMS). 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.

K36 COMPANY CONTACT
Soo Bang | [email protected] 

SOURCE K36 Therapeutics