Tivozanib demonstrated superiority over sorafenib in patients with advanced renal cell cancer in Phase 3 TIVO-1 trial

AVEO Pharmaceuticals, Inc. (NASDAQ: AVEO) and Astellas Pharma Inc. (TSE: 4503) today announced that tivozanib demonstrated superiority over sorafenib in the primary endpoint of progression-free survival (PFS) in TIVO-1, a global, randomized Phase 3 clinical trial evaluating the efficacy and safety of investigational drug tivozanib compared to sorafenib in 517 patients with advanced renal cell carcinoma (RCC). TIVO-1 is the first registration study in first-line RCC that is comparing an investigational agent against an approved VEGF therapy.

All patients in TIVO-1 had clear cell RCC, had undergone a prior nephrectomy, and had not previously been treated with either a VEGF or mTOR therapy. Based on the top-line analysis of events in TIVO-1, determined by a blinded, independent review committee, key top-line findings include:

• tivozanib demonstrated a statistically significant improvement in PFS with a median PFS of 11.9 months compared to a median PFS of 9.1 months for sorafenib in the overall study population
• tivozanib demonstrated a statistically significant improvement in PFS with a median PFS of 12.7 months compared to a median PFS of 9.1 months for sorafenib in the pre-specified subpopulation of patients who were treatment naïve (no prior systemic anti-cancer therapy); this subpopulation was approximately 70% of the total study population
• tivozanib demonstrated a well-tolerated safety profile consistent with the Phase 2 experience; the most commonly reported side effect was hypertension, a well established on-target and manageable effect of VEGFR inhibitors

Based on these data, AVEO and Astellas currently plan to submit for marketing approval of tivozanib in the United States and Europe in 2012, subject to final collection and analyses of all available data from the trial.

“We are very pleased by these results, especially the PFS benefit demonstrated in the treatment naïve population, which represents the most significant market opportunity for tivozanib,” said Tuan Ha-Ngoc, president and chief executive officer, AVEO. “In addition, we were delighted with the favorable safety profile observed in TIVO-1. We would like to acknowledge the investigators and patients who participated in TIVO-1 for their important contributions and commitment to the treatment of patients with RCC.”

The study participants continue to be observed to gather additional data for further analyses. AVEO and Astellas plan to submit detailed findings from TIVO-1 for presentation at the 2012 Annual Meeting of the American Society of Clinical Oncology (ASCO) being held June 1-5, 2012 in Chicago.

“We are delighted with the outcome of TIVO-1 and to be collaborating with AVEO on tivozanib at this critical juncture,” said Steven Ryder, M.D., president, Astellas Pharma Global Development. “Tivozanib is an important asset to our strategy of becoming a global category leader in oncology. We look forward to working closely with our global partner AVEO on next steps for regulatory filings for RCC and advancing clinical development of tivozanib in colorectal cancer and other solid tumors.”

AVEO Conference Call Information

AVEO will hold a conference call to discuss the top-line results of the TIVO-1 trial today at 8:30 a.m. ET. The call can be accessed by dialing 1-866-825-3354 (domestic) or 1-617-213-8063 (international) five minutes prior to the start of the call and providing the passcode 17542026. A replay of the call will be available approximately two hours after the completion of the call and can be accessed by dialing 1-888-286-8010 (domestic) or 1-617-801-6888 (international), providing the passcode 21404848. The replay of the call will be available for two weeks from the date of the live call.

A live, listen-only webcast of the conference call can also be accessed by visiting the investors section of the AVEO website at investor.aveopharma.com. A replay of the webcast will be archived on the company’s website for two weeks following the call.

About Renal Cell Carcinoma

Advanced RCC, or kidney cancer, is the ninth most commonly diagnosed cancer in men and women in the U.S.i Worldwide it is estimated that more than 250,000 people are diagnosed and more than 100,000 people die from the disease each year.ii RCC accounts for 90 percent of all malignant kidney tumors.iii Despite advances in RCC therapies, significant unmet need persists.

About Tivozanib

Tivozanib is a potent, selective and continuous inhibitor of all three vascular endothelial growth factor (VEGF) receptors that is designed to optimize VEGF blockade while minimizing offtarget toxicities. Tivozanib is an oral, once-daily, investigational tyrosine kinase inhibitor for which positive top-line results from a Phase 3 clinical study in advanced renal cell carcinoma have been reported, and is being evaluated in other tumors.

About the AVEO/Astellas Collaboration

In February 2011, AVEO and Astellas entered into a worldwide agreement outside of Asia to develop and commercialize tivozanib for the treatment of a broad range of cancers. Tivozanib, AVEO’s lead investigational drug, is a potent, selective and continuous inhibitor of all three vascular endothelial growth factor (VEGF) receptors that is designed to optimize VEGF blockade while minimizing off-target toxicities. Subject to regulatory approval, AVEO will lead commercialization of tivozanib in North America and Astellas will lead commercialization of tivozanib in the European Union (EU). AVEO and Astellas are evaluating tivozanib in clinical trials in multiple solid tumors; updates on the progress of those trials are expected to be available in the coming months.

Reference

i U.S. Cancer Statistics Working Group. United States Cancer Statistics: 1999–2007 Incidence and Mortality Web-based Report. Atlanta: U.S. Department of Health and Human Services, Centers for Disease Control and Prevention and National Cancer Institute; 2010. Available at: www.cdc.gov/uscs.

ii Cancer Research UK http://info.cancerresearchuk.org/cancerstats/world/the-global-picture/#Common; http://publications.cancerresearchuk.org/downloads/Product/cs_pdf_worldwide_2011.pdf

iii Motzer RJ, et al. JCO 2009- Sutent.