CHARM Therapeutics advances CHM-029, a next-generation menin inhibitor designed to overcome resistance mutations, as its first clinical candidate for acute myeloid leukaemia (AML)
PRESS RELEASE
- AI-discovered candidate CHM-029 demonstrates robust, dose-dependent tumor regression in pre-clinical AML models and retains potency against all known resistance mutations
- First-in-human trials expected to begin in Q2 2026
LONDON – 13 November 2025 -- CHARM Therapeutics (“CHARM”, “The Company”), today announced the advancement of CHM-029, a next-generation pan-menin inhibitor designed to overcome resistance mutations, as a development candidate for the treatment of acute myeloid leukemia (AML). It is expected that CHM-029 will enter first-in-human trials in Q2 2026.
Menin inhibitors have emerged as a clinically validated therapeutic class for the treatment of acute AML. However, first-generation inhibitors are limited by the rapid emergence of resistance mutations in menin that reduce efficacy and lead to disease progression.
CHM-029 was discovered using CHARM’s proprietary DragonFold platform, which applies deep learning to model protein–ligand interactions and enable structure-based drug design. It is designed to retain potency against all known menin clinical resistance mutations. The candidate has shown strong pre-clinical efficacy and a favorable safety profile, demonstrating dose-dependent tumor regression in both wild-type and mutant menin pre-clinical models. Further, it has not been possible to generate resistance to CHM-029 in in vitro forced mutation experiments, where resistance to multiple first generation menin inhibitors is rapidly seen. The pre-clinical data package is supportive of the potential for CHM-029 to deliver superior safety, efficacy, and more durable treatment responses.
Gary D. Glick, Ph.D., Executive Chair of CHARM Therapeutics, said: “The nomination of CHM-029 is a defining step as CHARM advances toward the clinic in Q2 2026 with a therapy built to address resistance to first-generation menin inhibitors. The pre-clinical data we’ve generated so far give us real confidence in the potential of CHM-029 to achieve deeper, more durable responses for patients with acute myeloid leukemia, where resistance continues to limit long-term treatment success.”
Dr. Erkut Bahceci, Chief Medical Officer at CHARM Therapeutics, added: “AML remains an aggressive disease with limited treatment options and a high rate of relapse driven by resistance mutations. CHM-029 offers a promising approach designed to overcome these challenges and deliver more effective and durable treatment responses for patients with AML. As we advance towards clinical development, we look forward to realizing the full potential of menin inhibition to improve the clinical outcomes in this difficult-to-treat cancer.”
This news follows CHARM’s recent $80 million Series B financing, supported by a strong syndicate of both existing and new global investors, reflecting confidence in the Company’s breakthrough approach to overcoming menin inhibitor resistance.
ENDS
For further information, please contact:
CHARM Therapeutics
Dr Beverley Carr, Chief Business Officer
ICR Healthcare
Amber Fennell
About CHARM Therapeutics
Founded by Laksh Aithani and David Baker, CHARM Therapeutics is a biotechnology company pioneering the next generation of precision oncology treatments through its proprietary AI-driven drug discovery platform.
CHARM's lead program is a next generation menin inhibitor for the treatment of acute myeloid leukemia (AML). Unlike first-generation menin inhibitors that rapidly lose potency due to menin resistance mutations, CHARM's candidates are specifically designed to maintain potency against all known clinical resistance mutations, potentially delivering the durable responses that patients desperately need.
Based in Cambridge and London, CHARM has raised over $150 million from leading international investors including New Enterprise Associates (NEA), SR One, OrbiMed, F-Prime and Khosla Ventures. The Company is advancing its lead menin inhibitor candidate, CHM-029, toward clinical development in Q2 2026.
For more information, please visit: www.charmtx.com
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