DelMar Pharmaceuticals, Inc. announced that the FDA Office of Orphan Products Development (OOPD) has granted orphan drug designation for its lead product candidate, VAL-083, in the treatment of medulloblastoma. The investigational drug candidate previously received an orphan designation for glioblastoma in the United States and in Europe.

VAL-083 is a "first-in-class," small-molecule chemotherapeutic. In more than 40 phase I and II clinical studies sponsored by the US National Cancer Institute, VAL-083 demonstrated clinical activity against a range of cancers including lung, brain, cervical, ovarian tumours and leukemia both as a single-agent and in combination with other treatments.

"We are pleased to achieve this important regulatory milestone and to continue a collaborative relationship with the FDA and the OOPD as we continue to expand the development of VAL-083," commented Jeffrey Bacha, chairman and CEO of DelMar. "Orphan designation is a major step toward expediting this promising therapy to an additional patient population with few treatment options."

DelMar has been conducting clinical trials with VAL-083 as a potential new treatment for glioblastoma multiforme (GBM), the most common and aggressive form of brain cancer. In September 2015, DelMar announced completion of enrollment in a phase II clinical trial in refractory GBM. The company anticipates top-line overall survival data from this trial in the first half of 2016.

Through its research, DelMar has also been exploring the unique cytotoxic mechanism of VAL-083 in order to identify additional indications where VAL-083 may address modern unmet medical needs in the treatment of cancer. In November 2015, DelMar presented new pre-clinical data in a poster entitled, "Dianhydrogalactitol (VAL-083) Offers Potential Therapeutic Alternatives in the Treatment of Pediatric Brain Tumors," at the American Association for Cancer Research (AACR) Advances in Pediatric Research: From Mechanisms and Models to Treatment and Survivorship Conference.

Medulloblastoma is the most common malignant paediatric brain tumour, accounting for 15-30 per cent of all childhood intracranial neoplasms. Although multidisciplinary treatment has improved the 5-year survival rates in children significantly, the prognosis for certain subtypes of medulloblastoma and for recurrent disease remains poor with a median overall survival of less than one (1) year.

In historical NCI-sponsored clinical studies, VAL-083 demonstrated clinical activity against medulloblastoma. In these studies VAL-083 was investigated both as a stand-alone therapy and in combination with other chemotherapeutic regimens. DelMar's recent pre-clinical research demonstrates that VAL-083 is active against medulloblastoma cells with difficult to treat sonic hedgehog (SHH) characteristics and p53 mutations; and VAL-083 in combination with temozolomide completely inhibits self-renewal of pediatric brain cancer stem cells (CSCs).

"Taken together, we believe these data will serve as a basis for our clinical development strategy with VAL-083 in paediatric brain tumours," continued Bacha. "We plan to continue our discussions with leading clinical investigators in order to undertake the necessary steps to advance VAL-083 into clinical studies as a potential treatment for children suffering from recurrent and difficult-to-treat medulloblastoma subtypes."