AVEO receives EMA orphan medicinal product designation for tivozanib

AVEO Pharmaceuticals, Inc. (NASDAQ: AVEO), a biopharmaceutical company focused on discovering, developing and commercializing cancer therapeutics, today announced it has received orphan medicinal product designation for tivozanib (N-{2-chloro-4-[(6,7-dimethoxy-4-quinolyl)oxy]phenyl}-N'-(5-methyl-3-isoxazolyl) urea hydrochloride monohydrate), its oral, triple VEGF receptor inhibitor, for the treatment of renal cell carcinoma by the European Medicines Agency (EMA). According to the EMA, tivozanib was awarded orphan medicinal product designation based on the prevalence of renal cell carcinoma among people in the European Union (EU); the life-threatening nature of the disease particularly for those with advanced or metastatic renal cell carcinoma; and the assumption that tivozanib may provide significant benefit for patients with renal cell carcinoma, and may be more potent and specific than existing treatments with similar mechanism of action as supported by preliminary clinical results.

“We are conducting a number of clinical trials of tivozanib both as monotherapy and in combination with other anti-cancer agents, and we believe tivozanib may represent an important and differentiated new therapeutic option to treat RCC.”

"Following the positive results from our Phase 2 clinical trial of tivozanib, we are very pleased to receive the EMA's orphan medicinal product designation for tivozanib, which represents an important milestone as we continue with the clinical development of tivozanib in renal cell cancer (RCC)," said Tuan Ha-Ngoc, president and chief executive officer of AVEO. "We are conducting a number of clinical trials of tivozanib both as monotherapy and in combination with other anti-cancer agents, and we believe tivozanib may represent an important and differentiated new therapeutic option to treat RCC."

Orphan medicinal product designation is awarded by the EMA for drugs that are shown to diagnose, prevent or treat life-threatening or rare, serious conditions that affect fewer than five in 10,000 people in the EU. Companies that receive orphan medicinal product designation by the EMA receive, among other benefits, market exclusivity in the EU for ten years following market authorization, protocol assistance, reduced fees and the opportunity to receive grants from the EU and member states supporting research and development. Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. Demonstration of quality, safety and efficacy is necessary before a designated orphan medicinal product can be granted a marketing authorization.

In February 2010, AVEO initiated patient enrollment in the TIVO-1 trial, a global Phase 3 clinical trial, for tivozanib in advanced RCC. TIVO-1 is a randomized, controlled trial evaluating tivozanib compared to sorafenib (Nexavar®). Sorafenib is approved by both the U.S. Food and Drug Administration (FDA) and the EMA for use in patients with advanced RCC. The primary endpoint of the trial is to compare the progression-free survival (PFS) of tivozanib vs. sorafenib. Secondary endpoints include overall survival, objective response rate, duration of response and quality of life. Independent radiology review will be conducted for all CT scans and radiographs. Patients with RCC of clear cell histology that have had a prior nephrectomy and that have not received prior VEGF-targeted therapy are eligible for this trial. Patients will be randomized 1:1 to tivozanib or sorafenib. Patients who demonstrate disease progression during treatment with sorafenib will have the opportunity to be treated with tivozanib by participating in a separate long-term treatment protocol. Additional information on the trial design can be found online at www.clinicaltrials.gov.

Prior to launching TIVO-1, AVEO successfully completed a 272-patient Phase 2 clinical trial of tivozanib in patients with advanced RCC. In March, AVEO presented an updated analysis of the results from its Phase 2 clinical trial of tivozanib at the 2010 Genitourinary Cancers Symposium, that reported the median PFS achieved by patients with advanced clear cell RCC who had undergone a prior nephrectomy was 14.8 months - comparing favorably to historical data from trials testing other currently approved VEGF receptor inhibitors in RCC.

Source AVEO Pharmaceuticals