On August 5, 2025 Hemispherian AS, a pioneering biotech company developing next-generation therapeutics for aggressive cancers, reported that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for GLIX1, a first-in-class small molecule targeting DNA repair vulnerabilities in glioblastoma and other solid tumors (Press release, Hemispherian, AUG 5, 2025, View Source [SID1234654815]).
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The IND clearance allows Hemispherian to proceed with a first-in-human Phase 1 clinical trial to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of GLIX1 in patients with recurrent glioblastoma.
Important Milestone – targets DNA repair pathways in tumor cells while sparing healthy tissue
"This milestone marks a major inflection point for Hemispherian and brings us one step closer to delivering a much-needed therapeutic option to patients facing glioblastoma, a cancer with devastating outcomes and few effective treatments," said Adam Robertson, Chief Scientific Officer of Hemispherian. "GLIX1’s unique mechanism of action selectively targets DNA repair pathways in tumor cells while sparing healthy tissue, and we are encouraged by its strong preclinical profile and regulatory recognition in both the U.S. and Europe."
GLIX1 is the lead candidate from Hemispherian’s proprietary GLIX platform and has demonstrated:
Potent anti-tumor activity in multiple glioblastoma models
Excellent blood-brain barrier penetration
Favorable safety profile in preclinical toxicology studies
Study to be conducted by world leading investigators in the field of Glioblastoma
The Phase 1 study will be conducted at leading neuro-oncology centers across the United States, starting at Northwestern University in Chicago.
Dr. Ditte Primdahl, Principal Investigator at Northwestern University in Chicago, commented: "I look forward to evaluating this novel therapeutic approach in patients with recurrent glioblastoma. The trial will generate valuable clinical data on safety, tolerability, and early signs of biological activity."
Dr. Roger Stupp, Co-Director of the Malnati Brain Tumor Institute of the Lurie Comprehensive Cancer Center at Northwestern University, Chicago, and Chairman of Hemispherian’s Scientific Advisory Committee, added: "Targeting DNA repair is the next frontier in improving outcome for patients with glioblastoma and other deadly brain tumors. It will delay tumor recurrences in patients who have initially been successfully treated. GLIX1 has a novel and unique mechanism of action, and this first-in-human trial will provide a novel therapeutic option for our patients, and insights for subsequently preventing tumor recurrences."
GLIX1 has also been granted Orphan Drug Designation by both the FDA and the European Medicines Agency (EMA), highlighting the urgent need for new treatments for malignant glioma and recognizing the potential of GLIX1 to provide meaningful benefit for patients facing this devastating disease.