CHARM Therapeutics Advances AI-Discovered Menin Inhibitor CHM-029 to Overcome AML Drug Resistance
Key Insights
CHARM Therapeutics (search) has advanced CHM-029 (search), a next-generation pan-menin (search) inhibitor designed to overcome resistance mutations, as its lead development candidate for acute myeloid leukemia treatment.
The AI-discovered drug candidate demonstrated dose-dependent tumor regression in preclinical models and retained potency against all known menin (search) clinical resistance mutations.
CHM-029 (search) is expected to enter first-in-human trials in Q2 2026, following CHARM's recent $80 million Series B financing round.
CHARM Therapeutics (search) announced the advancement of CHM-029 (search), a next-generation pan-menin (search) inhibitor designed to overcome resistance mutations, as a development candidate for the treatment of acute myeloid leukemia (AML). The biotechnology company expects CHM-029 to enter first-in-human trials in Q2 2026.
Addressing First-Generation Menin Inhibitor Limitations
Menin (search) 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 (search) was discovered using CHARM's proprietary DragonFold platform, which applies deep learning to model protein-ligand interactions and enable structure-based drug design. The candidate is designed to retain potency against all known menin (search) clinical resistance mutations.
Preclinical Efficacy and Safety Profile
The candidate has shown strong preclinical efficacy and a favorable safety profile, demonstrating dose-dependent tumor regression in both wild-type and mutant menin (search) preclinical models. Notably, it has not been possible to generate resistance to CHM-029 (search) in in vitro forced mutation experiments, where resistance to multiple first-generation menin inhibitors is rapidly seen.
The preclinical data package is supportive of the potential for CHM-029 (search) to deliver superior safety, efficacy, and more durable treatment responses compared to existing therapies.
Leadership Perspectives on Clinical Potential
Gary D. Glick, Ph.D., Executive Chair of CHARM Therapeutics (search), said: "The nomination of CHM-029 (search) is a defining step as CHARM advances toward the clinic in Q2 2026 with a therapy built to address resistance to first-generation menin (search) inhibitors. The preclinical 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 (search), added: "AML remains an aggressive disease with limited treatment options and a high rate of relapse driven by resistance mutations. CHM-029 (search) 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 (search) inhibition to improve the clinical outcomes in this difficult-to-treat cancer."
Company Funding and Platform
This development 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 (search) inhibitor resistance.
Founded by Laksh Aithani and David Baker, CHARM Therapeutics (search) is a biotechnology company pioneering the next generation of precision oncology treatments through its proprietary AI-driven drug discovery platform. 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.