UK-based biotech Charm Therapeutics has closed an oversubscribed Series B funding round worth $80 million, positioning the company to advance its AI-designed menin inhibitor for acute myeloid leukemia (AML) into clinical trials. The Phase I trial is expected to begin in Q1 2026, marking a significant milestone for the company's approach to overcoming resistance mutations that limit current therapies.
Breakthrough AI-Designed Therapy
Charm's menin inhibitor represents a departure from first-generation therapies in this class. Using its proprietary protein-ligand co-folding platform DragonFold, the company has developed a candidate designed to retain potency against all publicly described clinical resistance mutations associated with AML. According to the company, this molecule demonstrates robust tumor regression in preclinical models while being predicted to be efficacious at low human doses.
"Current menin inhibitors show promise in AML treatment but are fundamentally limited by the rapid emergence of resistance mutations that cause treatment failure," said Gary D. Glick, Ph.D., Executive Chair of Charm Therapeutics. "Our next-generation inhibitors, discovered using our proprietary DragonFold AI platform, are specifically designed to overcome these resistance mutations and deliver the durable responses that patients need."
Addressing Safety Concerns
The molecule chosen for clinical progression is designed to avoid common safety issues associated with first-generation menin inhibitors. Specifically, it aims to reduce the likelihood of cardiac arrhythmia and drug-drug interactions by not inhibiting enzymes responsible for drug interactions and avoiding QTc prolongation.
Strong Investor Confidence
The funding round was co-led by New Enterprise Associates (NEA) and SR One, with participation from existing investors OrbiMed, F-Prime, Khosla Ventures, and tech giant NVIDIA. The oversubscribed nature of the round reflects investor confidence in Charm's approach to addressing menin inhibitor resistance.
"The strength of the preclinical data and the clarity of the clinical development plan make CHARM well positioned to drive meaningful impact for patients facing treatment-resistant cancers," said Matthew McAviney, M.D., Partner at New Enterprise Associates.
Competitive Landscape
If approved, Charm's candidate would enter a lucrative but competitive AML market valued at $2.3 billion across eight major markets in 2022. The company would face direct competition from Syndax Pharmaceuticals' Revuforj (revumenib), which became the first FDA-approved menin inhibitor for AML in 2024. Analysts forecast Revuforj will earn $849 million for Syndax by 2031, representing 749% growth from its estimated 2025 revenue of $100 million.
Additional competition is emerging, with Kura Oncology and Kyowa Kirin having filed a new drug application for their candidate ziftomenib, which has received priority review with a PDUFA target action date of November 30, 2025.
Strategic Leadership Additions
To support its clinical transition, Charm has appointed Briggs Morrison, M.D., former CEO of Syndax, and Kim Blackwell, M.D., as non-executive directors. Morrison brings specific expertise in menin inhibitor development and deep understanding of the AML therapeutic landscape from his role in developing the first FDA-approved menin inhibitor.
Market Context
Acute myeloid leukemia is a rapidly progressing and aggressive blood cancer with poor prognosis for many patients. Menin inhibitors work by disrupting the binding of the KMT2A protein to menin, restoring normal gene regulation and triggering differentiation and apoptosis of malignant cells. However, first-generation therapies are limited by rapid emergence of resistance mutations in the menin protein, which reduce efficacy and lead to relapse and disease progression.
With over $150 million raised to date from leading international investors, Charm is positioned to advance its lead menin inhibitor candidate toward clinical development in early 2026, potentially offering AML patients a more durable treatment option.
