Glioblastoma treatment GLIX1 enters human trials
Phase 1/2a study evaluating safety, efficacy in patients with recurrent disease
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GLIX1, a treatment for recurrent and progressive glioblastoma and other high-grade gliomas, has begun human testing in a Phase 1/2a clinical trial, said BioLineRx, which is developing the treatment in a joint venture along with Hemispherian.
The trial (NCT07464925) aims to assess GLIX1’s safety, pharmacological properties, and preliminary efficacy in up to 30 patients with recurrent glioblastoma. The U.S. Food and Drug Administration (FDA) gave clearance to the trial last summer.
The study will be conducted at three sites in the U.S. New York University Langone Health will be the first to start enrolling patients, followed by Northwestern University and Moffitt Cancer Center. Initial data from the trial are anticipated in the first half of 2027.
“The initiation of this study represents the culmination of many months of tireless work on behalf of our team and the team of our collaboration partner, Hemispherian AS, as well as a pivotal moment in our mission to identify and advance innovative drugs to treat diseases with significant unmet needs,” Philip Serlin, BioLineRx’s CEO, said in a company press release. “GLIX1 has a very compelling profile supporting this Phase 1/2a study.”
Gliomas are a group of brain tumors that originate in glial cells, which support and protect nerve cells in the central nervous system (CNS, the brain and spinal cord). Glioblastomas are aggressive and fast-growing forms of glioma, for which there is a high unmet medical need for more effective treatments.
Crossing the blood-brain barrier
GLIX1 is a first-in-class, oral small molecule designed to increase the activity of TET2, an enzyme that makes 5-hydroxymethylcytosine, a modified DNA building block that’s often decreased in cancer cells. By activating TET2, GLIX1 is expected to increase 5-hydroxymethylcytosine levels and trigger a DNA damage/repair response, selectively killing cancer cells while sparing healthy tissue.
In preclinical studies, including studies with animal models of glioblastoma, the treatment was shown to cross the blood-brain barrier, a protective barrier that prevents many drugs from reaching the brain. It also demonstrated potent anticancer activity and a favorable safety profile.
The Phase 1/2 trial will enroll adults with recurrent or progressive glioblastoma and other gliomas who have received up to two prior treatment lines. They will receive escalating doses of GLIX1, administered once daily in 28-day cycles.
The main goal of the trial is to determine GLIX1’s safety, as well as the maximum tolerated dose and/or a recommended dose for further studies. The maximum tolerated dose is the highest amount of a drug or treatment that can be given without causing unacceptable side effects or toxicity. The study will also look at preliminary efficacy.
Later in the study, other groups of patients, such as those with newly diagnosed glioblastoma and patients with other types of cancer, will be included.
“GLIX1 is a promising innovative molecule with impressive pre-clinical data, and I could not be more excited to participate in this study,” said Roger Stupp, an oncologist at Northwestern University in Chicago and the study’s lead investigator. “The protocol will rigorously assess the safety of an agent with an entirely novel mechanism of action, with the potential to ultimately integrate well and synergize with the current treatments.”
The FDA has granted GLIX1 orphan drug status, a designation that aims to incentivize companies developing treatments for rare diseases.
