On May 6, 2019 DelMar Pharmaceuticals, Inc. (Nasdaq: DMPI) ("DelMar" or the "Company"), reported a biopharmaceutical company focused on the development of new cancer therapies, on May 3, 2019 presented data supporting Dianhydrogalactitol (VAL-083) as a potential therapy for pediatric brain tumors at the Society for Neuro-Oncology (SNO) Pediatric Neuro-Oncology Basic and Translational Research Conference held May 3-4, 2019 in San Francisco, CA (Press release, DelMar Pharmaceuticals, MAY 6, 2019, View Source [SID1234535749]). The Company, which has been working in collaboration with investigators from the University of California, San Francisco (UCSF), University of British Columbia, and the Vancouver Prostate Centre on evaluating VAL-083 as a treatment for diffuse intrinsic pontine glioma (DIPG), an extremely aggressive brain tumor, presented promising in vivo DIPG model data demonstrating VAL-083’s potential as a single agent, and in combination with Wee1 inhibitor AZD1775, in significantly prolonging survival.
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VAL-083 presentation highlights as a treatment for DIPG included:
As a single agent significantly increases median survival in DIPG in vivo compared to untreated controls
In combination with AZD-1775, a Wee1 inhibitor, further increases survival in DIPG in vivo
Is active against DIPG cell lines with varying genetic profiles including p53, H3.3/H3.1 and K27M mutations
Is synergistic with AZD1775 against DIPG and pediatric GBM cell lines
"We are encouraged by this data that demonstrates VAL-083’s ability as a single agent and in combination with Wee1 inhibitor AZD1775 to significantly increase survival in an in vivo DIPG model. An intact blood-brain barrier impeding drug penetration is a major obstacle to successful treatment of DIPG and VAL-083’s ability to concentrate in the cerebral spinal fluid (CSF) may offer an important advantage," commented Dr. Sabine Muller, Associate Professor of Clinical Neurology, UCSF.
DIPG is a difficult-to-treat, inoperable, rare pediatric brain tumor with very poor prognosis and a dismal survival outlook. Approximately 300 children in the U.S. are diagnosed with DIPG each year. While DIPGs are usually diagnosed when children are between the ages of 5 and 9, they can occur at any age in childhood. These tumors occur in boys and girls equally and do not generally appear in adults.
"While it’s certainly early in the evaluation process for the DIPG indication, I’m extremely pleased by these results demonstrating VAL-083’s potential in this difficult-to-treat pediatric brain cancer population. I am also encouraged to see the potential therapeutic benefit afforded by VAL-083 due to its ability to concentrate in the CSF. Recent data reported at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting in April 2019 from our clinical study for newly-diagnosed, adult, unmethylated MGMT GBM patients demonstrated that drug levels in the CSF were generally higher in comparison to plasma levels at two hours after administration. This compares favorably to standard of care temozolomide where CSF concentration levels are approximately 80% lower than in plasma and may offer an important advantage for VAL-083," commented Saiid Zarrabian, DelMar’s Chief Executive Officer.
About VAL-083
VAL-083 (dianhydrogalactitol) is a "first-in-class," bifunctional DNA-targeting agent that introduces interstrand DNA cross-links at the N7-position of guanine leading to DNA double-strand breaks and cancer cell death. VAL-083 has demonstrated clinical activity against a range of cancers including GBM and ovarian cancer in historical clinical trials sponsored by the U.S. National Cancer Institute (NCI). DelMar has demonstrated that VAL-083’s anti-tumor activity is unaffected by common mechanisms of chemoresistance, including MGMT, in cancer cell models and animal studies. Further details regarding these studies can be found at:
VAL-083 has been granted orphan drug designations by the U.S. FDA Office of Orphan Products for the treatment of glioma, medulloblastoma and ovarian cancer, and in Europe for the treatment of malignant gliomas. VAL-083 has been granted fast-track status for the treatment of recurrent GBM by the US FDA.