FDA Grants Fast Track Designation for VAL-083 for the Treatment of Newly-Diagnosed Glioblastoma

The United States Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to dianhydrogalactitol (VAL-083) for the treatment of patients with newly-diagnosed unmethylated glioblastoma multiforme (GBM).  The drug us being developed by San Diego-based Kintara Therapeutics.

Fast Track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Some of the significant benefits of FTD include:

• Enhanced access to the FDA, including opportunities for more frequent meetings and written consultation throughout the remaining development of VAL-083.
• Drugs with FTD are eligible to apply for Accelerated Approval and Priority Review at the time of a New Drug Application (NDA) submission, which may result in faster product approval.
• FTD also allows for ‘rolling review’, whereby Kintara may submit completed sections of the VAL-083 NDA as they become available, rather than at the end of development.

Demonstrating potency
VAL-083 has been evaluated in more than 40 Phase 1 and Phase 2 clinical trials by the U.S. National Cancer Institute (NCI) and was found to have anti-cancer activities against various cancers, including brain tumors and ovarian cancers. The investigational agent is a structurally unique, “first-in-class”, small-molecule DNA-targeting agent. Based on historical data researchers believe that VAL-083 has the potential to offer physicians and patients a new paradigm in the treatment of Glioblastoma Multiforme (GBM).

In one open-label, phase 2 clinical study, which was conducted at the MD Anderson Cancer Center (MD Anderson) in Houston, Texas (NCT02717962) in patients with GBM who had an unmethylated promoter of the MGMT-gene researchers demonstrated that, among 36 efficacy-evaluable patients, VAL-083 elicited a median progression-free survival (PFS) of 10.0 months (95% CI, 8.2-10.8). In this study, the median overall survival (OS) for these patients was 16.5 months (95% CI, 13.3-19.3). Although this study was not a head-to-head trial, the PFS and OS data for VAL-083 represented improvements over historical comparisons for patient diagnosed with GBM.

The dosing regimen (30 mg/m²/day) in this MD Anderson study mirrors the trial design of the newly-diagnosed adjuvant study arm of the GBM AGILE study which is sponsored by the Global Coalition for Adaptive Research (GCAR), a revolutionary, patient-centered, registrational, seamless Phase 2/3 adaptive platform trial evaluating multiple therapies for patients with newly-diagnosed and recurrent GBM.

Improving outcomes for patients with GBM
“We believe Fast Track Designation is indicative of VAL-083’s potential to improve outcomes for patients with GBM, the most aggressive form of brain cancer,” stated Robert E. Hoffman, President and CEO of Kintara.

“We look forward to announcing top-line data from the international registrational phase 2/3 GBM AGILE Study around the end of calendar year 2023. Fast Track Designation allows us to work closely with the FDA and may expedite our commercial launch of VAL-083, if approved,” Hoffman concluded.

VAL-083 currently represents the only therapeutic agent being evaluated in all three GBM patient subtypes: methylated MGMT, newly-diagnosed unmethylated MGMT, and recurrent.

Clinical trials
A Trial to Evaluate Multiple Regimens in Newly Diagnosed and Recurrent Glioblastoma (GBM AGILE) – NCT03970447
Study of VAL-083 in Patients With MGMT Unmethylated, Bevacizumab-naive Glioblastoma in the Adjuvant or Recurrent Setting – NCT02717962

Featured image: MRI of the brain. Photo courtesy: © 2016 – 2022 Fotolia/Adobe. Used with permission.