BioLineRx Ltd. (NASDAQ:BLRX) (TASE: BLRX), a clinical development stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, today announced the initiation of a first-in-human, Phase 1/2a study of GLIX1 for the treatment of recurrent and progressive GBM and other high-grade glioma (NCT07464925).

 

GLIX1 is an oral small molecule with a novel mechanism of action applicable to a broad range of cancers. By restoring TET2 activity, GLIX1 selectively targets DNA damage repair in cancer cells only. GBM was selected as the first indication for GLIX1 due to the low level of TET2 activity in this aggressive brain cancer, for which there remains a high unmet medical need for novel and more effective treatments. In extensive preclinical studies, including in-vivo GBM models, GLIX1 demonstrated potent anti-tumor activity and excellent blood-brain-barrier penetration, combined with a favorable safety profile in toxicology studies. 

Three renowned academic centers will ultimately participate in this clinical trial. The first center ready to start enrolling patients is NYU Langone Health, led by Dr. Alexandra Miller. This will be followed by Northwestern University, led by Dr. Roger Stupp and Dr. Ditte Primdahl, and by Moffit Cancer Center, led by Dr. Patrick Grogan.

"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," stated Philip Serlin, Chief Executive Officer of BioLineRx. "GLIX1 has a very compelling profile supporting this Phase 1/2a study and, to that end, we look forward to initial data in the first half of 2027." 

Dr. Alexandra Miller, Chief of Neuro-Oncology and Co-Director of the Brain and Spine Tumor Center at the Perlmutter Cancer Center, NYU Langone Health, stated, "I am pleased to be the first investigator able to enroll patients into this critical study, which brings new hope to patients who are in desperate need of innovative and novel treatment options."

Dr. Roger Stupp, Medical Director of the Malnati Brain Tumor Institute at Northwestern University in Chicago, and lead investigator of the study, added, "GLIX1 is a promising innovative molecule with impressive pre-clinical data, and I could not be more excited to participate in this study. 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. We urgently need breakthrough innovations for our patients suffering from glioblastoma, one of the most aggressive and difficult malignancies to treat."

Phase 1/2a clinical trial design (NCT07464925)

The Phase 1 part of the trial is expected to recruit up to 30 patients with recurrent and progressive GBM and other high-grade glioma. The objective is to establish a maximum tolerated dose (MTD) and/or a recommended dose based on safety, PK/PD and preliminary efficacy. Data from the Phase 1 part of the trial are anticipated in H1 2027.

The Phase 2a expansion part of the trial is planned to include various population cohorts, including GBM (newly diagnosed and/or recurrent), as well as additional cancers with/without standard of care (e.g., PARP inhibitors). These cohorts are expected to identify preliminary efficacy, PD assessments and dose optimization data, serving as the basis for a rapid and effective advanced clinical development plan.