GLIX1 - A new drug to treat glioblastoma multiforme (GBM)

Glioblastoma multiforme is the highest global unmet need in oncology. Glioblastoma multiforme (GBM) is the 17th leading cause of cancer and the second leading cause of cancer in children. Owing to its aggressive nature, a diagnosis with GBM results in severe reduction in quality of life and the disease rapidly causes death. GBM is a leading cause of death in children, second only to road injuries in Western Europe and the third leading cause of child death in the USA. Through a decade of scientific research into DNA base modifications at Oslo University Hospital, our chief Scientist and team identified a previously unknown cell cycle regulatory mechanism that pointed towards a new DNA replication regulatory pathway. The team exploited this pathway to identify highly specific anti-tumour drugs and identified a few molecules, among which, one stood out as specifically killing GBM tumours - GLIX1. The research group headed the fundamental work that led to the filing of patent protection for this family of compounds. Given the very positive and recent developments of GLIX1, Hemispherian now aims to advance drug development using a drug-diagnostic co-development model strategy, searching for new biomarkers that can guide clinical development phases, and conducting preclinical studies, to determine if GLIX1 is safe for use in humans diagnosed with GBM. Hemispherian?s long-term vision is to commercialize a patient-centric approach, by combining therapy decisions with molecular insights, a first-of-a-kind drug combined with the respective diagnostic tool. As such, this project is of strategic importance for Hemispherian. The R&D outcome of this project will be a pre-clinically validated new candidate treatment for GBM (drug and companion diagnostic assay), ready for human clinical trials (Phase I clinical studies).

Actual outcomes: 1) A class of biomarkers that allow us to distinguish between GLIX1 sensitive and resistant cancers. 2) A biomarker that can identify patient samples that are more likely to benefit from GLIX1 treatment. 3) A cost effective production process for synthesising GLIX1. 4) Progressing towards fulfilling all safety requirements for initiating clinical trials in the EU/US.

Today, patients diagnosed with glioblastoma (GBM) have limited treatment options. The current standard of care for GBM treatment is a combination of surgical resection, radiation, and one or more courses of generally ineffective chemotherapy. With the currently available treatment, the median overall survival of GBM patients is 13.9 months with 2-year survival of 22.5% and 5-year survival of 5.3%. Compounding the poor patient survival rates, currently available treatments often cause debilitating side-effects, resulting in poor quality of life. This poor patient survival rate and horrendous end-of-life quality highlights an urgent need to identify novel strategies to target GBM. Through a decade of scientific research Hemispherian AS (HEMI) identified a molecule (GLIX1) that targets DNA replication and has been shown to kill GBM tumours. Furthermore, this molecule showed no side-effects and indicate a striking reduction in tumour mass or even total remission. Building on very positive results and milestones reached until today, HEMI aims now to further advance the GLIX1 development by using a drug-diagnostic co-development model strategy, searching for new biomarkers that can guide clinical development phases, while also conducting preclinical studies, to determine if GLIX1 is safe for initial use in humans. To enable this work HEMI will lead a consortium of world-class partners (OUH, NTNU, UIO). HEMI’s long term vision is to commercialize a patient-centric approach, by combining therapy decisions with molecular insights, a first-of-a-kind drug combined with the respective diagnostic tool. Funding from IPN programme would allow the participating SME to strengthen its current market position, expanding its vision of bringing a new and effective drug to the oncology market.