CHARM Therapeutics: $80 Million Series B Raised For Next-Generation AML Treatment

By Amit Chowdhry • Sep 3, 2025

CHARM Therapeutics has closed an oversubscribed Series B funding round, raising a substantial $80 million. This funding will be instrumental in propelling their innovative menin inhibitor into clinical testing. The funding attracted a diverse group of global investors, including previous supporters and new partners, demonstrating their strong confidence in CHARM’s approach. The company’s strategy centers on overcoming the resistance issues that have limited the effectiveness of earlier menin inhibitors—a significant step forward in treating acute myeloid leukemia.

AML, or acute myeloid leukemia, is a fast-growing and aggressive form of blood cancer that often carries a poor outlook for many patients. In recent years, scientists have identified menin inhibitors as promising therapeutic agents. These drugs work by restoring normal gene regulation within cancer cells, prompting them to differentiate or undergo apoptosis. However, the first-generation drugs faced significant hurdles. Resistance mutations in the menin protein would rapidly develop, reducing the effectiveness of the drugs and leading to disease relapse.

Additionally, some early menin inhibitors brought safety concerns, such as the risk of prolonging the QTc interval—a measure of the heart’s electrical cycle—and issues related to drug interactions, which sometimes forced the use of high doses with increased side effects.

CHARM Therapeutics is now leading the charge in developing a new class of menin inhibitors designed to address these challenges. Leveraging their proprietary platform, DragonFold, CHARM has identified a promising candidate that demonstrates robust activity against various resistance mutations seen in clinical settings.

Preclinical studies have yielded encouraging results, with the candidate demonstrating significant tumor regression in multiple models. This molecule is predicted to work effectively at low doses in humans, without the heart-related safety risks associated with some previous treatments. It has been engineered to avoid inhibiting enzymes responsible for drug-drug interactions, potentially making it safer and more convenient for patients.

Through the power of artificial intelligence and innovative design, CHARM aims to fully unlock the potential of menin inhibition to improve outcomes for AML patients. Their goal is to develop more potent, long-lasting therapies that provide enduring responses, addressing a significant unmet need in this aggressive cancer.

New board members: As they prepare to move their lead candidate into clinical trials, CHARM has also taken strategic steps to strengthen its leadership. This includes the addition of two experienced directors to their board. Briggs Morrison, a seasoned expert with deep knowledge in menin inhibitor development and AML, joins as a non-executive director, bringing insights from his previous role as CEO of Syndax, the company behind the first FDA-approved menin inhibitor. Alongside him, Kim Blackwell joins as a non-executive director, bringing extensive clinical development expertise in oncology that will be invaluable as CHARM advances its innovative therapies.

Following the Series B round, Matthew McAviney, M.D., Partner at NEA, and Mahesh Kudari, M.D., Senior Associate at SR One, will also join the board as non-executive directors, supporting CHARM’s strategic execution as it advances toward clinical trials.

KEY QUOTES:

“Securing funding from such a high-quality investor syndicate is a strong validation of our approach to overcoming menin inhibitor resistance. Current menin inhibitors show promise in AML treatment but are fundamentally limited by the rapid emergence of resistance mutations that cause treatment failure. 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. This program represents a significant opportunity to transform outcomes for patients, and we look forward to initiating clinical development early next year.”

Gary D. Glick, Ph.D., Executive Chair CHARM Therapeutics

“We are thrilled to support CHARM as it transitions into the clinic with its highly promising next-generation menin inhibitor. 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. We’re proud to support CHARM during this next phase of growth.”

Matthew McAviney, M.D., Partner at New Enterprise Associates

“CHARM has leveraged its proprietary protein-ligand co-folding platform DragonFold to rapidly identify highly potent, next-generation menin inhibitors that are active against resistant mutations. We are excited to support progression of this program into clinical trials and look forward to working with the team.”

Mahesh Kudari, M.D., Senior Associate at SR One