Small-Molecule Chemotherapeutic (VAL-083) Wins Orphan Drug Designation for Ovarian Cancer

Drug candidate shows clinical activity in phase II studies

The FDA’s Office of Orphan Products Development has granted orphan drug status to VAL-083 (DelMar Pharmaceuticals), a first-in-class small-molecule chemotherapeutic, for the treatment of women with ovarian cancer. VAL-083 previously received an orphan drug designation for glioma and medulloblastoma in the United States.

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

Ovarian cancers are commonly treated with platinum-based chemotherapy regimens. Initial tumor response rates are relatively high; however, up to 75% of ovarian cancer patients who respond to initial treatment will relapse within approximately 18 months after completing first-line therapy. Median survival in platinum-resistant recurrent ovarian cancer patients has ranged from six to nine months in published studies.

At the recent annual meeting of the American Association for Cancer Research, investigators at the University of Texas MD Anderson Cancer Center presented preclinical data demonstrating that the mechanism of action of VAL-083 appears to differ from that of the platinum-based chemotherapies currently used to treat ovarian cancer. In these studies, VAL-083 demonstrated an ability to circumvent cisplatin resistance in all ovarian cell lines tested.

The American Cancer Society has estimated that approximately 22,000 women receive a new diagnosis of ovarian cancer and approximately 14,000 die from the disease each year in the U.S. Ovarian cancer ranks fifth in cancer deaths among women, accounting for more deaths than any other cancer of the female reproductive system. The market for ovarian cancer therapies is expected to be approximately $570 million in 2016 and is projected grow to more than $3.5 billion by 2022.

Source: DelMar Pharmaceuticals; April 21, 2016.