News|Articles|August 6, 2025
FDA Clears IND for GLIX1 in Glioblastoma, Phase 1 Study to Proceed
Author(s)Sabrina Serani
Fact checked by: Jason M. Broderick
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Key Takeaways
- GLIX1, a first-in-class small molecule, targets DNA repair vulnerabilities, offering potential glioblastoma therapy.
- Hemispherian will initiate a phase 1 trial to evaluate GLIX1's safety, tolerability, pharmacokinetics, and preliminary efficacy.
Hemispherian's GLIX1 gains FDA approval for glioblastoma trials, promising a novel therapy targeting DNA repair in tumor cells.
The FDA has cleared the investigational new drug (IND) application of GLIX1, a first-in-class small molecule targeting DNA repair vulnerabilities, offering a potential therapy for glioblastoma.1
With this clearance, Hemispherian, the sponsor, will initiate a first-in-human phase 1 clinical trial to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of GLIX1 in patients with recurrent glioblastoma.
"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, in a press release. "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 US and Europe."
Preclinically, GLIX1 has demonstrated potent antitumor activity in glioblastoma models, good blood-brain barrier penetration, and a favorable safety profile. GLIX1 was also previously granted orphan drug designation by the FDA and EMA for the treatment of malignant glioma.2
The phase 1 study will recruit patients across the US and begin at Northwestern University in Chicago, Illinois.1
"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,” said Ditte Primdahl, MD, principal investigator at Northwestern University, in the press release.
REFERENCES:
1. Hemispherian Receives FDA IND Clearance for First-in-Class Glioblastoma Therapeutic, GLIX1. News release. Hemispherian AS. August 5, 2025. Accessed August 5, 2025. https://tinyurl.com/bdzb3pwk
2. Hemispherian Receives U.S. FDA Orphan Drug Designation for GLIX1 for the Treatment of Malignant Glioma. News release. Hemispherian AS. July 1, 2025. Accessed August 5, 2025. https://tinyurl.com/37wsxfh8
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