New Brain Cancer Drug to Be Studied in Two Clinical Trials

Patients can now enroll in a phase 1 clinical trial evaluating an oral formulation of the novel drug OKN-007 for the treatment of patients with recurrent high-grade glioma, according to HLB Therapeutics, the manufacturer of the drug.

"We have decided to develop an oral formulation in consideration of convenience for the patient, which is an important aspect for future commercialization, and plan to have a strategy to increase the value of the new drug in the market with great unmet needs through diversification of treatment options,” Ki Hong Ahn, CEO of HLB Therapeutics said in a press release.

The main goal of the trial is to determine the proper dose of OKN-007, as well as how the drugs move through and are absorbed within the body — an endpoint known as pharmacokinetics. Based on the findings from the phase 1 trial, researchers will determine the best dosage of OKN-007 for future studies. They will also look at safety and preliminary cancer-fighting qualities of the regimen.

“Through discussions with the FDA, we have already incorporated their suggestions on the clinical study protocol. Along with investigating the efficacy in existing clinical studies with the OKN injectable formulation, our plan for this phase 1 clinical trial is to determine the safety of an oral dosage form of OKN-007 and define a dose for further clinical trials, thus allowing multiple options for future development,” Ahn said.

According to the National Cancer Institute, OKN-007 has the potential to stop tumor cell growth by blocking enzymes that glioma cells need to grow.

HLB Therapeutics also announced that they are also conducting a phase 2 clinical trial studying injectable OKN-007 plus Temodar (temozolomide), a type of chemotherapy, for patients with recurrent glioblastoma multiforme (GBM), which is the disease classification for grade 4 glioma, and accounts for nearly half (49.1%) of all primary malignant brain tumors, according to the National Brain Tumor Society.

Temodar and other chemotherapy agents work differently than OKN-007 to fight cancer; they stop the growth of tumor cells either by killing them directly, inhibiting their ability to divide or stop them from spreading. According to the National Cancer Institute, “Giving OKN-007 and (Temodar) chemo-radiotherapy may work better in treating patients with glioblastoma multiforme after surgery.”

This trial includes 57 patients with recurrent GBM that has returned or spread after being treated with standard-of-care treatments such as surgical removal, radiation and chemotherapy.

The main goals of the phase 2 trial are incidence of side effects within 24 months, number of patients who experience decreased neurological function or decreased performance (meaning the ability to perform daily activities independently) and overall survival, defined as the time from treatment until patient death of any cause.

Final data for this trial is expected to be collected in March of 2024.

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