SEATTLE, April 27, 2015 /PRNewswire/ -- VentiRx Pharmaceuticals, Inc., a clinical stage biopharmaceutical company focused on the development of novel Toll-like receptor 8 (TLR8) immunotherapies in the treatment of cancer, announced today that the European Commission (EC), acting on the positive recommendation from the European Medicines Agency (EMA) Committee for Orphan Medical Products (COMP), has granted orphan designation to motolimod (formally VTX-2337) as an orphan medicinal product for the treatment of ovarian cancer. VentiRx previously announced in April 2014 that the U.S. Food and Drug Administration (FDA) granted orphan drug designation to motolimod for the treatment of ovarian cancer.
"Motolimod has shown encouraging initial activity as a novel immunotherapy used in combination with pegylated liposomal doxorubicin for the treatment of women with recurrent or persistent ovarian cancer who have failed a prior platinum-containing regimen," commented Dr. Robert Hershberg, President and CEO of VentiRx. "We believe that targeted stimulation of the innate immune system by motolimod has the potential to make a real difference for women with ovarian cancer and look forward to reporting results from the fully enrolled randomized, placebo-controlled Phase 2 trial. Data from the primary endpoint of overall survival are expected in early 2016."
Orphan drug designation by the European Commission provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than 5 in 10,000 persons in the European Union (EU) and where no satisfactory treatment is available. In addition to the 10-year period of market exclusivity in the EU after product approval, orphan drug designation provides incentives for companies seeking protocol assistance from the EMA during the product development phase and direct access to the centralized authorization procedure.
About Motolimod (formally VTX-2337)
Motolimod is a novel Toll-like Receptor 8 (TLR8) agonist that directly activates multiple components of the innate immune system. This includes activation of human myeloid dendritic cells (mDCs), monocytes and natural killer (NK) cells resulting in the production of high levels of mediators known to orchestrate the integration of innate and adaptive antitumor responses.
Motolimod is in Phase 2 development in both ovarian and head and neck cancer. Over 290 women were enrolled in the GOG-3003 randomized, placebo-controlled Phase 2 trial of motolimod in combination with Pegylated Liposomal Doxorubicin (PLD) in patients with recurrent or persistent epithelial ovarian, fallopian tube or primary peritoneal cancer who have failed prior platinum-based chemotherapy. The study is being performed in collaboration with the Gynecologic Oncology Group (GOG) Partners Program. Enrollment in this trial is complete and VentiRx is currently awaiting results. The primary endpoint of the study is overall survival.
A second, company-sponsored, randomized, Phase 2 placebo-controlled trial, called Active8, (NCT01836029) is enrolling patients with locally advanced and metastatic squamous cell carcinoma of the head and neck at more than 50 sites in the United States. The study is evaluating motolimod in combination with cetuximab and chemotherapy. The primary endpoint of the trial is progression-free survival.
About Ovarian Cancer
There is an estimated 21,000 new cases of ovarian cancer in the US and an estimated 44,000 new cases in the European Union. Ovarian cancer is one of the most lethal gynecologic cancers. Women with recurrent or persistent ovarian cancer usually recur within 6-12 months of completion with a platinum-containing regimen and there remains a high unmet need for improved treatment options.
About VentiRx Pharmaceuticals
VentiRx Pharmaceuticals Inc. is a clinical stage biopharmaceutical company committed to the development and commercialization of novel Toll-like receptor 8 (TLR8) immunotherapies for the treatment of cancer, respiratory and inflammatory diseases. The company's lead product candidate, motolimod, is a small molecule TLR8 agonist for the treatment of cancer. VentiRx is a privately held organization based in Seattle, Washington. VentiRx has an exclusive, world-wide collaboration with Celgene Corporation for motolimod.