The final patient with glioblastoma multiforme has been enrolled in the ongoing phase 2 clinical trial of VAL-083, which is evaluating the efficacy, safety, and pharmacokinetics of the agent in patients who have been pre-treated with temozolomide prior to disease recurrence.
The final patient with glioblastoma multiforme (GBM) has been enrolled in the ongoing phase 2 clinical trial of VAL-083, which is evaluating the efficacy, safety, and pharmacokinetics of the agent in patients who have been pre-treated with temozolomide (Temodar) prior to disease recurrence, according to a press release from Kintara Therapeutics, Inc.
VAL-083 is a first-in-class small molecule bifunctional alkylating agent that has the ability to cross the blood-brain barrier. VAL-083 was previously granted Orphan Drug Designation for GBM by the FDA as well as a Fast Track designation for the treatment of patients with recurrent GBM.
The study, which is being conducted at the MD Anderson Cancer Center in Houston, Texas, enrolled 83 patients into the recurrent arm to determine whether the drug will improve overall GBM survival. Of the 83 patients, 35 patients will receive 40 mg/m2/day of VAL-083 and 48 patients will receive 30mg/m2/day. Efficacy is determined by overall survival (OS) as well as progression-free survival (PFS).
The second arm of the 2-arm, open label, biomarker-driven study enrolled 36 newly diagnosed patients who have undergone surgery and chemoradiation with temozolomide, are receiving VAL-083 as opposed to the standard of care of temozolomide for adjuvant therapy.
In a follow-up presented in November 2020, the median OS for the 77 evaluable patients who completed at least 1 treatment cycle was 7.6 months. The second arm of the study had a median PFS of 10.0 months. In the 30 mg/m2/day, group, 3 patients experienced a serious adverse event related to the treatment.
"Given the urgent need for improved treatment options for this deadly disease, we are pleased to have reached the very important milestone of full enrollment in the recurrent arm of this Phase 2 clinical study," said Saiid Zarrabian, chief executive officer, Kintara Therapeutics, in a press release.