DelMar Pharmaceuticals Announce FDA Grants Second Orphan Designation For VAL-083

DelMar Pharmaceuticals (OTCQX:DMPI) 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.
As quoted in the press release:

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

DelMar Pharmaceuticals Chairman and CEO, Jeffrey Bacha, stated:

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. Orphan designation is a major step toward expediting this promising therapy to an additional patient population with few treatment options.
Taken together, we believe these data will serve as a basis for our clinical development strategy with VAL-083 in pediatric brain tumors. 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.

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