Kintara Therapeutics (NASDAQ:KTRA) Inc has announced topline data results from the newly-diagnosed adjuvant arm of its open-label, Phase 2 clinical study being conducted at the MD Anderson Cancer Center in Houston.
The Phase 2 trial is a two-arm, biomarker-driven study testing VAL-083 in glioblastoma multiforme (GBM) patients who have an unmethylated promoter of the methylguanine DNA-methyltransferase (MGMT) gene.
The company previously announced topline data results from the recurrent GBM arm of the study which provided important safety and efficacy data to support the continued evaluation of VAL-083 as a treatment option for GBM.
READ: Kintara Therapeutics reports topline results from Phase 2 clinical study of VAL-083 for recurrent GBM
Kintara said the newly-diagnosed adjuvant arm of the study addressed GBM patients requiring adjuvant therapy after chemoradiation with temozolomide. The trial arm enrolled 39 patients (36 efficacy evaluable) initially receiving a dose of 30 milligrams/m2/day on days 1, 2 and 3 of a 21-day cycle.
"On behalf of the entire Kintara team, I wish to extend gratitude to MD Anderson, and all of the patients who participated in both arms of the trial," CEO Saiid Zarrabian said in the statement.
"The topline results from the newly-diagnosed adjuvant arm are a particularly important milestone for the company as it further affirms the efficacy and safety data reported this past July from the recurrent arm, thus providing additional support and momentum to continue the evaluation of VAL-083 for the treatment of GBM."
Summary of results:
- Progression Free Survival (PFS) for the 36 efficacy evaluable patients is 10.0 months (95% Confidence Interval (CI) 8.2-10.8 months). While this is not a head-to-head trial, historical data for this patient population has demonstrated PFS of 5.3-6.9 months.
- Median overall survival (mOS) for the 36 efficacy evaluable patients is 16.5 months (CI 13.3-19.3 months). While this is not a head-to-head trial historical data for this patient subpopulation has demonstrated mOS of 12.7-16.0 months.
- Consistent with prior studies, myelosuppression was the most common adverse event. One patient experienced a serious adverse event (SAE) possibly related to VAL-083.
- The dosing regimen (30 mg/m2/day) of the MD Anderson study mirrors the trial design of the newly-diagnosed adjuvant study arm of the GBM AGILE study.
GBM AGILE, which is sponsored by the Global Coalition for Adaptive Research (GCAR), is a revolutionary, patient-centered, registrational, seamless Phase 2/3 adaptive platform trial evaluating multiple therapies for patients with newly-diagnosed and recurrent GBM.
VAL-083 currently represents the only therapeutic agent being evaluated in all three GBM patient subtypes: methylated MGMT, newly-diagnosed unmethylated MGMT, and recurrent.
"I continue to be impressed by the clinical data generated by both arms of the study and remain excited by VAL-083's potential to be a game-changing therapeutic agent to help patients suffering from this deadly disease,” principal investigator for the Phase 2 study at MD Anderson Dr Barbara O’Brien said.
VAL-083 has been granted Orphan Drug Designation for GBM by the US Food & Drug Administration (FDA) and the European Medicines Agency (EMA) and has also been granted Orphan Drug Designations for medulloblastoma and ovarian cancer from the FDA. As well, the FDA has granted Fast Track Designation for VAL-083 in recurrent GBM.
VAL-083 is approved as a cancer chemotherapeutic in China for the treatment of chronic myelogenous leukemia and lung cancer. But the drug has not been approved for any indications outside of China.
Contact the author at stephen.gunnion@proactiveinvestors.com