Next-Generation Menin Inhibitor Designed to Overcome Clinically Acquired Resistance Mutations Observed with First-Generation Molecules

Shanghai, May 15, 2026 — Beyang Therapeutics Co., Ltd, a clinical-stage biopharmaceutical company pioneering next-generation targeted therapies for hematologic malignancies, today announced that the U.S. Food and Drug Administration (FDA) has granted Ophan Drug Designation (ODD) to BY002, an oral, potent, and highly selective next-generation menin inhibitor. The designation covers the treatment of Acute Myeloid Leukemia (AML) characterized by KMT2A (MLL) rearrangements or NPM1 mutations. BY002 was specifically engineered to retaintherapeutic activity against acquired menin mutations, a key mechanism of resistance that has constrained the durability and long-term clinical benefit of first-generation menin inhibitors currently in clinical development or early commercialization.

Addressing the Challenge of Acquired Resistance

While first-generation menin inhibitors have shown significant promise in inducing clinical responses in genetically defined AML subtypes, recent clinical data suggest that many patients eventually progress due to the emergence of somatic mutations at the menin-KMT2A interface. These mutations disrupt drug binding while preserving the oncogenic protein-protein interaction. “Receiving Orphan Drug Designation is a significant regulatory milestone that underscores the urgent need for more durable treatment options for patients with KMT2A-rearranged or NPM1-mutated AML,” said Jerry Hu, Chief Excutive Officer of Beyang Therapeutics. “By specifically designing BY002 to overcome the resistance mutations that frequently emerge during treatment with earlier agents, we aim to provide a more sustainable therapeutic to benefit and improve long-term outcomes for patients who currently face limited options following relapse.”

About Menin Inhibition in AML

The interaction between menin and KMT2A (formerly MLL1) is a critical driver in specific leukemias. In KMT2A-rearranged and NPM1-mutated AML, the menin protein acts as a scaffold that facilitates the expression of a leukemogenic gene program, including HOXA and MEIS1. Inhibiting this interaction induces differentiation of leukemic blasts and promotes clinical remission.

About Orphan Drug Designation

The FDA's Orphan Drug Designation program provides orphan status to drugs and biologics intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. ODD provides Beyang Therapeutics with certain development incentives, including tax credits for qualified clinical testing, a waiver of the Prescription Drug User Fee (PDUFA), and potential for seven years of market exclusivity upon regulatory approval.

About Beyang

Beyang Therapeutics is an AI-driven innovative drug R&D company, aiming to become the "DreamWorks" in the field of innovative Drug Discovery. The company focuses on the development of drugs with clear mechanisms of action (MOA) and high potential for clinical translation, in the areas with unmet medical needs. Based on its proprietary ExCEED platform (molecular design, druggability assessment, and biological research), Beyang boasts industry-leading preclinical pipeline conversion rates. The core team comprises experienced scientists from leading pharmaceutical companies such as Pfizer, Merck, and Hengrui. They have participated in the R&D of three globally-launched small molecule drugs and successfully advanced over 10 projects into clinical trials. With strict target selection standards and an "AI + experience-driven" model, the company is actively deploying in high-potential fields, accelerating the development of effective drugs to address unmet patient needs while maximizing commercial value.

Contact：

Minghui Tong, PhD

Tel：+86-13265738517

Email：tongmh@beyangtx.com

Website：Beyang Therapeutics Co., Ltd.