Novartis drug helps rare tumors, filing this year

Patients with advanced neuroendocrine tumors, a rare type of cancer, lived 5.1 months longer without their disease worsening when given Novartis's drug Afinitor, researchers said on Monday.

The new data add to the case for using the potential blockbuster in this niche area, following its approval as a treatment for kidney cancer last year.

Novartis previously said the latest Phase III trial had produced clinically meaningful results, even though it slightly missed pre-defined statistical significance. But details were only released at the European Society for Medical Oncology (ESMO) congress in Milan.

Researchers involved in assessing the drug played down the statistical "miss," which they said reflected imbalances in the treatment arms of the so-called RADIANT-2 trial.

An earlier study, known as RADIANT-3, did meet its endpoint in extending progression-free survival in patients with advanced pancreatic neuroendocrine tumors (NETs).

The Swiss drugmaker reaffirmed that it planned to seek worldwide regulatory approval for Afinitor as a treatment for NETs this year.

The new drug works by blocking a protein known as mTOR and is given in combination with Novartis's older drug Sandostatin, or octreotide LAR.

$1 BILLION SALES

Industry analysts believe sales of Afinitor, or everolimus, will reach $1 billion a year by 2014, driven initially by its use in kidney cancer, according to consensus forecasts compiled by Thomson Reuters.

Overall, patients given Afinitor and Sandostatin lived a median 16.4 months before their disease progressed, against 11.3 months for those on placebo plus Sandostatin, in the latest 429-patient trial.

"When we designed the study, we tested for an improvement of at least 4.5 months compared to octreotide LAR and were pleased with the resulting 5.1 month difference," said lead investigator Dr Marianne Pavel from Charite University in Berlin.

Neuroendocrine tumors affect less than five people in 100,000 and, until now, there have been few treatment options. The tumors can occur throughout the body and although most are slow-growing, some can be highly malignant.

The rarity of disease has made conducting clinical trials a challenge, so Novartis and its research associates had to work with multiple medical centers around the world.

Dr Roberto Labianca from Ospedali Riuniti di Bergamo, Italy, who was not involved in the research, said the latest findings were "practice-changing" but he urged future studies to refine the selection of patients to identify those most likely to respond.

Dr James Yao of MD Anderson Cancer Center in Houston, who worked with Pavel on the study, told Reuters that while other drugmakers were investigating new approaches to fighting NETs Afinitor was some two or three years ahead.

(Editing by Michael Shields)