Sanofi highlights positive PhIII study for myelofibrosis contender

Sanofi ($SNY) scored upbeat data from a Phase III study of its JAK 2 inhibitor for a rare hematological cancer known as myelofibrosis. The pivotal study for SAR302503 nailed the primary endpoint for the proportion of patients with a greater than 35% reduction in the volume of their spleens, which become enlarged as a result of abnormal blood cell production.

The French drug giant has highlighted the compound, which the company acquired in a 2010 buyout of San Diego-based TargeGen, as one of the 17 late-stage programs with a shot at approval in the coming years. The drug candidate also poses a potential competitive threat to the myelofibrosis drug Jakafi from Incyte ($INCY) and Novartis ($NVS), with Sanofi studying its contender in patients who fail treatment on Jakafi/ruxolitinib. And Sanofi has mounted an effort to rapidly advance the compound through trials for three types of myeloproliferative dieases and, if approved, onto the market.

"Since Sanofi's acquisition of the molecule," Dr. Debasish Roychowdhury, head of Sanofi Oncology, stated, "SAR302503 has moved from Phase I to the completion of pivotal Phase III studies in less than three years, and now we are planning regulatory filings with authorities to make this medicine available for patients."

Like many of its peers, Sanofi has banked on new oncology drugs for future growth. Lately, the company has had mixed results in its cancer-drug efforts. In January, the company revealed that late-stage development of ombrabulin for sarcoma was halted because of insufficient evidence of benefit, and midstage work on the compound was stopped in ovarian cancer as well.

Sanofi expects to report full results of the late-stage trial called "JAKARTA" at an upcoming medical meeting. In the top-line results, the company noted that as in previous studies, the most common side effects included anemia, diarrhea, nausea and vomiting.

Myelofibrosis affects about 15,000 patients in the U.S., and those with the disease have median survival of 6 years and a one in 5 risk of developing a more deadly blood cancer known as acute myelogenous leukemia, according to Sanofi.

- here's the press release
- see Reuters' article
- and the item from Dow Jones Newswires

Special Report: Sanofi - Biopharma's Top R&D Spenders - 2012

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