U.S. FDA Grants Orphan Drug Designation to ABM-1310 for the Treatment of Patients with Glioblastoma Harboring BRAF V600 Mutation

SAN DIEGOandSHANGHAI,Aug. 3,2023 -- ABM Therapeutics (ABM) announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to ABM-1310,a novel small molecule BRAF inhibitor developed by the company,for the treatment of patients with glioblastoma (GBM) bearing BRAF V600 mutation.

Orphan Drug Designation is a significant recognition bestowed by the FDA to the drugs used in the treatment of rare diseases. This designation intends to encourage and support the development of drugs that are vital to patients with rare medical conditions. ODD could provide the drug developer various regulatory support and financial incentives. Obtaining Orphan Drug Designation is a prestigious recognition from the FDA,which encourages ABM to accelerate the development of ABM-1310 for treating patients with GBM.

ABM-1310 is an orally administered medicine with high BRAF-mutation selectivity,high water solubility,and high blood-brain barrier permeability. It is one of innovative drugs independently developed by ABM. ABM-1310 is in Phase I studies at multiple clinical sites in the U.S. and China for BRAF V600-mutant advanced solid tumors. The interim result from its U.S. Phase 1 study was presented in June 2023 at the American Society of Clinical Oncology (ASCO) Annual Meeting,demonstrating ABM-1310's promising anticancer activity and good safety profile in patients with advanced BRAF V600 mutant solid tumors including primary brain tumors such asGBM and other gliomas. A new Phase I clinical trial specifically targeting GBM has recently been initiated in China.

GBM is a hard-to-treat,highly aggressive brain tumor. Due to the complexity in tumorigenesis and resistance to the therapy,the treatment of GBM remains significant challenges. Conventional treatments,including surgical resection,radiation therapy,and chemotherapy,are still suboptimal. As of the fact that very few treatment options are available after disease progression,development of more effective therapies is with a high demand.

ABM is dedicated to the development of novel medicines to provide patients with more effective treatment options. The FDA-granted Orphan Drug Designation marks another important milestone for the company. ABM is highly encouraged and will continue its endeavors in developing more innovative drugs,and bringing new hope and more choices to patients with cancers.

About ABM Therapeutics

ABM Therapeutics (ABM),a clinical-stage biopharmaceutical company with a mission to focus on small molecule research and development of novel medicines for the treatment of cancer,especially for brain cancer and brain metastases from solid tumors. ABM has been building a broad and robust proprietary pipeline to construct a brain medicine R&D platform (BPKddTM). ABM's pipeline includes several programs at various stages of discovery and development,most of which have improved brain permeability to address the unmet needs of treating cancers and metastases in the brain.