UK-based biotech Charm Therapeutics has closed an oversubscribed Series B funding round worth $80m, which will support the progression of its acute myeloid leukaemia (AML) menin inhibitor into clinical trials.
Co-led by New Enterprise Associates (NEA) and SR One, the round will see the cancer candidate enter early clinical development, with a Phase I trial expected to begin in Q1 2026. Other investors include tech giant NVIDIA, OrbiMed, F-Prime and Khosla Ventures.
According to Charm, its menin inhibitor differs from first-generation iterations as it is designed to overcome the emergence of resistance mutations and safety risks associated with this drug class.
Through the utilisation of its AI-driven protein-ligand co-folding platform, DragonFold, the biotech has developed a candidate it claims can remain potent against all logged clinical resistance mutations associated with AML.
The molecule chosen for clinical progression is also designed to be efficacious at low doses, reducing the likelihood of cardiac arrhythmia and drug-drug interactions – two common side effects of first-generation menin inhibitors.
Investors seem excited about the prospect of this early-stage menin inhibitor, with New Enterprise Associates partner Matthew McAviney noting that “the strength of the preclinical data and the clarity of the clinical development plan” has put Charm in a good position to progress the molecule.
If Charm’s candidate were to gain approval, it would join the lucrative AML market, which was worth $2.3bn across the eight major markets (8MM: US, Germany, France, Italy, Spain, UK, Japan and China) in 2022, according to GlobalData, parent company of Pharmaceutical Technology.
However, it would be in direct competition with Syndax Pharmaceuticals’ Revuforj (revumenib), which became the first menin inhibitor to gain approval from the US Food and Drug Administration (FDA) for AML during 2024.
According to analysts at GlobalData, Revuforj is primed for significant growth during the next five years, with forecasts predicting that the drug will earn $849m for Syndax by 2031. This value is up 749% from the drug's estimated 2025 revenue of $100m.
However, Revuforj’s status as the only FDA-approved menin inhibitor lies in the balance, as Kura Oncology and Kyowa Kirin have filed a new drug application (NDA) for their candidate, ziftomenib.
This follows the positive results of a pivotal Phase II trial exploring the drug’s efficacy in relapsed or refractory AML patients with nucleophosmin 1 (NPM1) mutations. In a 1 June 2025 statement, Kura noted that the drug has been granted priority review, while its Prescription Drug User Fee Act (PDUFA) target action date is set to 30 November 2025.
Meanwhile, a combination of Inqovi (decitabine and cadazuridine) plus Venclexta (venetoclax) is also in the pre-registration phase for AML, which – if approved – would be designated for use in newly diagnosed patients who are ineligible for induction chemotherapy. Its PDUFA data has been set for 25 February 2026.
Further back in the clinical pipeline, there are 28 candidates in Phase III trials for AML, including B-cell lymphoma 2 (Bcl 2), FMS-like tyrosine kinase 3 (FLT 3) and Nicotinamide adenine dinucleotide phosphate (NADP) inhibitors, according to GlobalData’s Intelligence Center.
