Biotech Values

[DewDiligence]

REGN’s Arcalyst Hits All Endpoints in Second Phase-3 Gout Study

[The only thing missing in this study was a difference between the high and low doses, which had been seen in the first phase-3 gout study that reported in Jun 2010 (#msg-51061561). Arcalyst is a proprietary “trap” protein (incorporating the same technology as VEGF-Trap-Eye) that fuses portions of the IL-1 receptor into an antibody-like compound. Arcalyst is similar to NVS’ Ilaris (a conventional IL-1 mAb); both drugs are approved in the US and EU for the orphan indication known as CAPS. REGN’s own PR is at http://finance.yahoo.com/news/ARCALYST-rilonacept-Meets-prnews-2436673596.html?x=0&.v=1 .]

http://blogs.forbes.com/matthewherper/2011/02/28/regeneron-announces-strong-results-in-gout-study

›Feb. 28 2011 - 7:36 am
By MATTHEW HERPER

Regeneron Pharmaceuticals of Tarrytown, N.Y., announced positive results from a second late-stage clinical trial showing that its drug Arcalyst, currently approved for a rare disease, can treat gout, a much more lucrative opportunity.

Gout, a condition in which the buildup of uric acid in the joints causes pain and swelling, is treated with alopurinol, a drug that prevents the uric acid buildup and has been available in the United States since 1964. But in some patients, starting alopurinol increases the risk of painful flare ups that actually make the joint problems worse. Arcalyst, an expensive, injectible protein drug, is being tested as a way to prevent those flare-ups. Mark Monane, the biotechnology analyst at Needham & Co., thinks Arcalyst could reach 10% to 15% of the available patient pool and generate $300 million to $500 million in annual sales.

The new results, issued via press release, show that patients who injected themselves with either 80 milligrams or 160 mg of Arcalyst once a week had a 72% decrease in the average number of gout flares compared to a placebo group. The differences compared to placebo were highly statistically significant. There were 248 patients in the study, divided evenly between each group.

An earlier study of the same size and design had shown a 73% reduction on the 80 mg dose and an 83% reduction on the 160 mg dose. It’s not immediately clear why the newer result does not show a difference between the two doses. I asked Regeneron about this, and got the following response:

The trial was not designed to test differences between doses. Nevertheless, while the primary endpoint in the PRE-SURGE 2 trial does not appear to have any dose response, as you noted, the endpoints in PRE-SURGE 1 and the secondary endpoints in PRE-SURGE 2 appear to trend slightly better with the 160 mg dose. We will work with the FDA to determine the best dose for approval.

Patients also showed benefits from taking Arcalyst on other measures. Regeneron has said it expects file with the U.S. Food and Drug Administration to market Arcalyst for gout in the middle of this year.

Although the gout result could be important for Regeneron, most of the controversy [is] around its eye drug, VEGF-Trap, which will compete with Roche’s eye drug Lucentis. A key controversy is whether an upcoming trial from the National Institutes of Health will show that Roche’s cancer drug Avastin works as well as Lucentis. Some investors think that could make it harder for Regeneron’s drug to grab a toe hold. Although many of Regeneron’s drugs are being developed in a partnership with Sanofi-Aventis, the company owns the rights the Arcalyst outright.‹