On July 1, 2021 Kintara Therapeutics, Inc. (Nasdaq: KTRA) ("Kintara" or the "Company"), a biopharmaceutical company developing novel cancer therapies for patients who are failing or are resistant to current treatment regimens, reported topline data results from the recurrent arm of its open-label, Phase 2 clinical study of its lead compound VAL-083 being conducted at the MD Anderson Cancer Center (MD Anderson) in Houston, Texas (Press release, Kintara Therapeutics, JUL 1, 2021, View Source [SID1234584550]).

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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 recurrent arm of the study addressed patients who have been pre-treated with temozolomide prior to disease recurrence.

The recurrent arm of the trial enrolled 89 patients, with 35 patients (35 efficacy evaluable) initially receiving a dose of VAL-083 at 40 mg/m2/day, and 54 patients (48 efficacy evaluable) initially receiving the treatment dose of 30 mg/m2/day on days 1, 2 and 3 of a 21-day cycle. This 30 mg dose corresponds to the dose being studied in the recently initiated and currently enrolling VAL-083 study arm of the GBM AGILE study.

Summary of results:

– Median overall survival (mOS) for the 48 efficacy evaluable patients initially receiving the treatment dose of 30 mg/m2/day is 8.0 months (95% confidence interval: CI 5.9-9.9 months). While this is not a head-to-head trial, historically, lomustine, which is the most commonly used chemotherapy for these patients, has demonstrated mOS of 7.2 months*

– Consistent with prior studies, myelosuppression was the most common adverse event. In the 30 mg/m2/day starting dose cohort, five patients experienced a serious adverse event (SAE) possibly related to VAL-083

– For the 83 efficacy evaluable patients who have completed at least one cycle of treatment mOS was 7.5 months (CI 6.1-9.0 months)

"I’m extremely pleased with the outcome of the recurrent arm of the study as it provided important safety and efficacy data to support further evaluation of VAL-083 for the treatment of GBM," said Saiid Zarrabian, Kintara’s Chief Executive Officer. "The study of VAL-083 continues in GBM AGILE, an adaptive registration study where it is currently the only therapeutic agent being evaluated for all three GBM patient subtypes: newly-diagnosed methylated MGMT, newly-diagnosed unmethylated MGMT, and recurrent."

Dr. Barbara O’Brien, the Principal Investigator for the Phase 2 study at MD Anderson added, "These data continue to support VAL-083’s compelling potential as a potent DNA targeting cytotoxic agent for the treatment of GBM, which remains a deadly disease with an urgent need for improved treatment options."

VAL-083 is independent of the MGMT resistance mechanism and has been assessed in over 40 Phase 1 and Phase 2 clinical trials in multiple indications sponsored by the U.S. National Cancer Institute (NCI). Published pre-clinical and clinical data indicate that VAL-083 has activity against a range of tumor types, including lung, brain, cervical, and ovarian tumors and hematologic (blood) cancers. VAL-083 has been granted Orphan Drug Designation for GBM by the FDA and EMA and has also been granted Orphan Drug Designations for medulloblastoma and ovarian cancer by the FDA. In addition, 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. VAL-083 has not been approved for any indications outside of China.

* Wick et al N.Eng.J.Med . 377:1954 1963 (2017)