FDA Grants Orphan Drug Designation to ABM-1310 for Glioblastoma


ABM-1310 is a novel small molecule BRAF inhibitor that has shown promising anti-cancer activity and a good safety profile across a variety of advanced BRAF V600-mutant solid tumors.

The FDA has granted an orphan drug designation (ODD) to ABM-1310 for the treatment of patients with glioblastoma (GBM) harboring a BRAF V600 mutation, according to ABM Therapeutics, Inc.1

ABM-1310 is a novel small molecule BRAF inhibitor that is orally administered. The product has high BRAF-mutation selectivity, high water solubility, and high blood-brain barrier permeability, and is currently being developed by ABM Therapeutics, Inc. Multiple phase 1 studies are ongoing, evaluating ABM-1310 across clinical sites in the United States and China in patients with BRAF V600-mutant advanced solid tumors.

Interim findings from a phase 1 study presented at the American Society of Clinical Oncology (ASCO) Annual Meeting 2023 showed ABM-1310 to have promising anticancer activity and a good safety profile when used for the treatment of patients with advanced BRAF V600-mutant solid tumors. This included in patients with primary brain tumors, such as GBM and other gliomas.2

In the multicenter, open-label, dose-escalation trial, patients with BRAF V600-mutated solid tumors were enrolled and assessed to determine the primary end point of maximum tolerated dose and recommended phase 2 dose of ABM-1310 with or without cobimetinib (Cotellic), along with secondary end points of safety, tolerability, pharmacokinetics, and preliminary efficacy.

Findings revealed that as of November 28, 2022, 20 patients with a median age of 57.5 years were enrolled and treated with ABM-1310 monotherapy across 6 dose levels in part A of the study. Sixteen patients in part A were efficacy evaluable and among them, 2 had a partial response (PR) and 8 had stable disease (SD) as their best response.

Among patients, 100% experienced adverse events (AEs), with the most frequent being skin rash (40%) and QT prolongation (20%). Drug-related grade ≥3 AEs occurred in 3 patients and consisted of nausea/vomiting, QT prolongation and rash, and 2 patients had drug-related serious AEs, including nausea/vomiting and creatinine increase.

In part B of the study, ABM-1310 in combination with cobimetinib was assessed at 2 dose levels, 100 mg twice a day (n = 3) and 200 mg twice a day (n = 3). Four patients had AEs and drug-related AEs, including rash and QT prolongation, and no SAEs were reported. Of the 3 evaluable patients, 1 patient with melanoma had a PR and 1 had SD as their best response.

No DLTs were confirmed in either dose-escalation part of the trial and no patients discontinued treatment prematurely for any safety or tolerability reason. Additionally, no drug-related deaths were reported.

Overall, ABM-1310 at doses up to 200 mg twice a day, either alone with cobimetinib, was generally well tolerated, with no new safety signals. This trial is ongoing.

Additionally, a new phase 1 clinical trial taking place in China has been initiated, specifically evaluating ABM-1310 to target GBM.

  1. U.S. FDA grants orphan drug designation to ABM-1310 for the treatment of patients with glioblastoma harboring BRAF V600 mutation. News release. ABM Therapeutics. August 2, 2023. Accessed August 3, 2023. https://tinyurl.com/4p3z5xyf
  2. Piha-Paul SA, Nagpal S, Weise AM, et al. A phase 1, multicenter, open-label study of a new BRAF inhibitor ABM-1310 in adult patients (pts) with BRAFv600-mutated solid tumors. J Clin Oncol. 2023;41(16) 3098-3098. doi:10.1200/JCO.2023.41.16_suppl.3098

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