Hemispherian Initiates Phase 1/2a Clinical Trial of GLIX1 in Glioblastoma

Key Terms

phase 1/2a medical

Phase 1/2a is an early stage in testing new medicines or treatments, combining two steps into one process. It helps researchers quickly assess whether a treatment is safe and shows signs of working, while also gathering initial information on the best dosage. For investors, this stage indicates how close a potential new therapy is to becoming available and its initial safety profile.

glioblastoma medical

Glioblastoma is a fast-growing and aggressive type of brain tumor that can affect a person's thinking, movement, or senses. Its seriousness and difficulty to treat can lead to significant health impacts, making it a concern for medical research and drug development. For investors, advances or setbacks in glioblastoma treatments can influence biotech companies and healthcare markets focused on cancer therapies.

high-grade gliomas medical

High-grade gliomas are aggressive, fast-growing tumors that start in the brain’s support cells and tend to invade nearby tissue, often causing serious symptoms and poor prognosis. For investors, they matter because their severity drives demand for new treatments, influences clinical trial urgency and regulatory scrutiny, and can create significant market opportunities or risks for companies developing drugs, devices, or diagnostics—think of them as a rapidly spreading problem that creates urgent need and potential value.

tet2 medical

TET2 is a gene that makes a protein helping control which genes are turned on or off by removing chemical “tags” on DNA, like a dimmer switch for gene activity. Mutations in TET2 can allow abnormal blood cell growth and are linked to age-related clonal hematopoiesis and certain blood cancers, so it matters to investors as a biomarker that can affect diagnostic tests, patient selection, drug targets, and the commercial prospects of therapies or testing companies.

dna damage response medical

A DNA damage response is the set of cellular systems that detect and repair breaks or errors in a cell’s genetic material; think of it as a repair crew and alarm system that keeps DNA functioning properly. It matters to investors because drugs that enhance, inhibit, or exploit these repair pathways can change a therapy’s effectiveness, safety, market potential and regulatory risk, affecting the value of biotech and pharmaceutical companies.

blood-brain barrier medical

A protective barrier of tightly packed cells and supporting tissue that controls what substances in the blood can enter the brain, acting like a security checkpoint that keeps out most pathogens and many drugs while allowing essential nutrients through. For investors, the barrier matters because whether a therapy can cross or safely bypass it often determines clinical success, regulatory approval and commercial potential for treatments of brain disorders.

pharmacokinetics/pharmacodynamics medical

Pharmacokinetics/pharmacodynamics (PK/PD) describes how a drug moves through the body (absorption, distribution, breakdown and elimination) and how it affects the body (the relationship between dose and effect). Think of PK as the drug’s journey and PD as the drug’s impact at the destination; together they predict dosing, safety and effectiveness, which matter to investors because strong, predictable PK/PD data reduces development risk and informs commercial potential.

parp inhibitors medical

PARP inhibitors are a class of cancer drugs that block an enzyme cells use to repair damaged DNA, effectively preventing cancer cells from fixing themselves and causing them to die. Investors watch them because their clinical approvals, safety profiles, patent status, and tests that identify which patients will benefit determine market size and revenue potential—much like backing a tool that only works on certain problems but can be very valuable when it does.

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First-in-class TET2 activator targets DNA damage response in GBM and other cancers

Strong preclinical efficacy, brain penetration, and safety profile support clinical advancement

Study led by world-leading glioblastoma investigators, including Dr. Roger Stupp

OSLO, Norway--(BUSINESS WIRE)-- Hemispherian AS, a clinical-stage oncology company developing novel small molecule therapeutics, today announced the initiation of a first-in-human Phase 1/2a clinical trial of GLIX1 in patients with recurrent and progressive glioblastoma (GBM) and other high-grade gliomas (NCT07464925). The study is being conducted in collaboration with BioLineRx Ltd. (NASDAQ/TASE: BLRX).

GLIX1 is an orally available, first-in-class small molecule designed to activate TET2 and drive tumor-selective DNA damage. By restoring TET2 activity, GLIX1 induces DNA damage selectively in cancer cells, representing a differentiated approach to targeting the DNA damage response with potential applicability across a broad range of tumors.

Glioblastoma was selected as the initial indication due to its highly suppressed TET2 activity and significant unmet medical need. Despite existing therapies, GBM remains one of the most aggressive and treatment-resistant cancers.

In extensive preclinical studies, including orthotopic in vivo GBM models, GLIX1 demonstrated:

• Potent anti-tumor activity
• Robust blood-brain barrier penetration
• Favorable safety profile in toxicology studies

The trial will be conducted across three leading academic centers. The first site to initiate patient enrollment is NYU Langone Health, led by Dr. Alexandra Miller. Additional sites include Northwestern University, led by Dr. Roger Stupp and Dr. Ditte Primdahl, and Moffitt Cancer Center, led by Dr. Patrick Grogan.

“The initiation of this Phase 1/2a study marks a defining milestone for Hemispherian and represents the culmination of years of dedicated research and development work by our team and our collaboration partner, BioLineRx. GLIX1 has a compelling preclinical profile and a truly differentiated mechanism of action, and we look forward to bringing this innovative therapy to patients who urgently need new treatment options. We are proud to be advancing this program alongside world-leading glioblastoma investigators and anticipate initial data readout in the first half of 2027,” said Zeno Albisser, Chief Executive Officer of Hemispherian AS.

“GLIX1 is built on a fundamentally new understanding of how to exploit DNA repair vulnerabilities in cancer. The strength and consistency of the preclinical data give us confidence as we now transition this mechanism into the clinic,” remarks Adam Robertson, Chief Scientific Officer at Hemispherian.

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.”

Clinical Trial Design (NCT07464925)

The Phase 1 portion of the study will enroll up to 30 patients with recurrent and progressive GBM and other high-grade gliomas. The primary objective is to establish the maximum tolerated dose (MTD) and/or a recommended dose based on safety, pharmacokinetics/pharmacodynamics (PK/PD), and preliminary efficacy. Data from Phase 1 are anticipated in the first half of 2027.

The Phase 2a expansion is expected to include multiple patient cohorts, including newly diagnosed and recurrent GBM, as well as additional tumor types. Combination approaches, including with PARP inhibitors, will also be evaluated. These cohorts are designed to generate early efficacy signals, inform dose optimization, and support subsequent clinical development.

About Hemispherian

Hemispherian AS is a clinical-stage pharmaceutical company developing first-in-class small-molecule cancer therapies. Its lead program, GLIX1, is being advanced in partnership with BioLineRx for the treatment of glioblastoma and a broad range of solid tumors.

The company is headquartered in Oslo, Norway, and collaborates with leading academic and clinical institutions worldwide.

Learn more at www.hemispherian.com or on LinkedIn.

View source version on businesswire.com: https://www.businesswire.com/news/home/20260407249206/en/

Zeno Albisser, CEO – zeno@hemispherian.com

Source: Hemispherian AS