ABM-1310 receives Orphan Drug Designation from the FDA for Glioblastoma

FDA Grants Orphan Status to ABM-1310 for Glioblastoma

Source – ABM Therapeutics

ABM Therapeutics has received orphan drug designation from the FDA for ABM-1310, a novel small molecule BRAF inhibitor, in the treatment of glioblastoma (GBM) patients with a BRAF V600 mutation. ABM-1310 is administered orally and has demonstrated high selectivity for BRAF mutations, good water solubility, and high permeability through the blood-brain barrier. The drug is currently undergoing multiple phase 1 studies in the United States and China, focusing on patients with BRAF V600-mutant advanced solid tumors.

Preliminary results from a Phase I study presented at the American Society of Clinical Oncology (ASCO) Annual Meeting 2023 revealed promising anticancer activity and a favorable safety profile of ABM-1310 in treating patients with advanced BRAF V600-mutant solid tumors, including primary brain tumors like GBM and other gliomas.

The Phase I trial was a multicenter, open-label, dose-escalation study. The primary endpoint was to determine the maximum tolerated dose and recommended Phase II dose of ABM-1310 with or without cobimetinib (Cotellic), along with assessing secondary endpoints such as safety, tolerability, pharmacokinetics, and preliminary efficacy.

As of November 28, 2022, 20 patients with a median age of 57.5 years were enrolled in part A of the study and treated with ABM-1310 monotherapy across six dose levels. Among the 16 patients evaluable for efficacy, two patients achieved a partial response (PR), and eight patients showed stable disease (SD) as their best response. All patients experienced adverse events (AEs), with the most common benign skin rash and QT prolongation. Grade ≥3 drug-related AEs were reported in three patients, and two patients experienced drug-related serious AEs.

Part B of the study assessed the combination of ABM-1310 with cobimetinib at two dose levels. Among the three evaluable patients, one patient with melanoma achieved a partial response, and one patient showed stable disease.

The trial showed that ABM-1310, alone or in combination with cobimetinib at doses up to 200 mg twice a day, was generally well-tolerated, with no dose-limiting toxicities, no treatment discontinuations due to safety reasons, and no drug-related deaths reported.

Moreover, a new Phase I clinical trial has been initiated in China, specifically evaluating ABM-1310 for the treatment of glioblastoma (GBM). The research is ongoing, and ABM-1310 shows promise as a potential therapeutic option for patients with BRAF V600-mutant tumors.

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