Aveo (Barely) Passes Study With Kidney Cancer Drug, Stock Falls

[Updated] Aveo Pharmaceuticals is starting the New Year with news that it has passed the most important clinical trial in company history, although it appears to have barely eked out the victory, which disappointed investors.

Cambridge, MA-based Aveo (NASDAQ: AVEO) said today that its lead experimental drug reached its goal in a pivotal clinical trial known as TIVO-1. The study showed Aveo’s drug was able to keep kidney tumors from spreading for a longer period of time than another drug in its class, sorafenib (Nexavar) from Bayer and Onyx Pharmaceuticals. The trial of 517 kidney cancer patients showed that Aveo’s tivozanib was able to keep tumors from spreading a median of 11.9 months, compared with 9.1 months for the existing drug. The study was designed to show the Aveo drug could keep tumors from spreading about an extra three months, so it barely passed.

Today’s statement didn’t say anything specific about the new therapy’s side effects, although CEO Tuan Ha-Ngoc said the study showed a “favorable safety profile” for the Aveo drug. More details on safety and effectiveness will be presented in June at the American Society of Clinical Oncology’s (ASCO) annual meeting, Aveo said in a statement. And importantly, Aveo and its partner, Japan-based Astellas Pharma, said they now plan to file applications for approval from regulators in the U.S. and Europe this year to start selling the new treatment.

Despite reaching the study’s main goal, Aveo shares fell today, possibly because investors were expecting to see the Aveo drug demonstrate a bigger advantage over the competition. Aveo stock fell $2.13, or about 12 percent, to $15.07 at 10:20 am Eastern.

[Update: 11:50 am Eastern] If there was one surprise in the results, it was that patients on the competing drug did a bit better than expected, Ha-Ngoc said by phone this morning. Patients on Onyx and Bayer’s sorafenib have typically been able to live for five to nine months without their tumors spreading, so today’s result of 9.1 months was on the high end of the expected range, creating a higher-than-expected hurdle for Aveo to clear. But Ha-Ngoc said the result definitively shows Aveo met its goal of demonstrating superiority over an active drug, unlike other experimental cancer treatments in this class, which have typically been tested against placebos. He added that Aveo’s drug was able to keep tumors from spreading for a median of 12.7 months for patients getting their first round of treatment, which is the largest benefit ever shown for patients in that group, he says.

“We are extremely happy today. It’s a fantastic way to start the year for us,” Ha-Ngoc says. Given the drug’s advantage in its safety profile, and its ability to delay tumor progression, tivozanib “should be in position to be the first-line treatment of choice,” Ha Ngoc says.

He added that the result is just the first time Aveo has passed a pivotal trial, and it is looking forward to additional studies of tivozanib as a treatment for other tumor types, and to help provide momentum for the rest of its pipeline. “We are here to build a real company,” he says.

The Aveo drug is an oral pill designed to cut off the blood supply that nourishes tumors, by specifically blocking three different forms of VEGF receptors that are on the surface of cancer cells. Aveo’s tivozanib is supposed to be a more selective blocker of VEGF than two currently marketed drugs that work in a similar way-Pfizer’s sunitinib (Sutent) and Bayer and Onyx’s sorafenib. Researchers hope that more selective VEGF receptor can more completely shut off blood flow to tumors, and also avoid blocking similar receptors on healthy cells-which can cause side effects.

The market for treating kidney cancer is significant. About 61,000 new cases of kidney cancer were diagnosed in the U.S. last year, and 13,000 people died from the disease, according to the American Cancer Society. Pfizer’s sunitinib, first approved in January 2006, generated $1.06 billion in sales in 2010. Bayer and Onyx’s sorafenib generated $934 million in sales the same year

Investors, and researchers, have had high hopes for the Aveo drug since it passed a mid-stage study of 272 patients, which was presented at the ASCO meeting in June 2009. The Aveo drug showed in that study it was able to keep tumors from spreading a median of 11.8 months, with mild side effects reported, including high blood pressure and hoarseness of voice.

So today’s TIVO-1 study essentially confirms what researchers had seen in the prior study.

Aveo didn’t provide detailed breakdowns of how the drug performed in various patient demographic groups, although it did suggest the drug’s advantage was more pronounced in patients getting their first round of therapy. The Aveo drug kept tumors in check for a median of 12.7 months compared with 9.1 months for those on the competing drug who were previously “treatment naive.” About 70 percent of the patients in the study fit this description, Aveo said.

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