Hemispherian gets FDA green-light for glioblastoma trial of GLIX1
Therapy was granted orphan drug status by FDA, EMA
The U.S. Food and Drug Administration (FDA) will allow Hemispherian to test its first-in-class experimental therapy for glioblastoma in a Phase 1 clinical trial.
The first-in-human study, to be conducted at sites in the U.S., starting at Northwestern University in Chicago, will evaluate the safety, tolerability, pharmacological properties, and preliminary efficacy of GLIX1 in people with recurrent glioblastoma, a type of malignant glioma. No other details have been released.
“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,” Adam Robertson, PhD, Hemispherian’s chief scientific officer, said in a company press release.
The FDA and the European Medicines Agency have granted GLIX1 orphan drug status, a designation given to investigational therapies that have the potential to treat rare diseases. The award provides Hemispherian with support and financial incentives for GLIX1’s development, along with a period of market exclusivity, if approved.
Gliomas are a group of tumors that affect glia, cells that support nerve cells in the brain and spinal cord. Glioblastoma is a fast-growing, aggressive cancer that can invade surrounding tissues. Treatment options are limited and outcomes are generally poor.
How does GLIX1 work?
GLIX1 is designed to increase the activity of TET2, an enzyme that makes 5-hydroxymethylcytosine, a type of modified DNA building block that’s often depleted in cancer cells. By boosting TET2, GLIX1 should cause 5-hydroxymethylcytosine levels to rise, triggering a DNA damage/repair response that selectively kills cancer cells while sparing healthy tissue, according to Hemispherian.
In preclinical studies, GLIX1 has shown anti-tumor activity in multiple glioblastoma models, limited side effects, and a favorable safety profile in preclinical toxicology studies, said the company. The therapy can also penetrate the blood-brain barrier, a protective membrane that can prevent drugs from reaching brain tissue.
“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,” Robertson said.
While no details have been released about the number of participants and the treatment regimen, Ditte Primdahl, MD, the principal investigator at Northwestern, said: “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.”
The company also will investigate if GLIX1 can be used to prevent tumors from recurring after successful initial treatment.
“Targeting DNA repair is the next frontier in improving outcome for patients with glioblastoma and other deadly brain tumors,” said Roger Stupp, MD, co-director of the Malnati Brain Tumor Institute of the Lurie Comprehensive Cancer Center at Northwestern. “It will delay tumor recurrences in patients who have initially been successfully treated.”
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