Incyte生物医药公司CEO Hervé Hoppenot与诺华公司合作研制的治疗罕见血液疾病新药Ruxolitinib三期研究获得成功，这也将有助于公司向FDA申请突破性药物地位。Ruxolitinib此前已经被批准用于治疗骨髓纤维化，此次在Incyte公司关于治疗真性多血症的三期研究中，Ruxolitinib成功完成率其预期目标，血细胞体积明显减小、脾脏尺寸降低了35%。

不出意外的话，一旦该药物上市，其市场销售峰值可能达到10亿美元，按照约定，Incyte公司 将获得其美国市场权利，而诺华公司将获得除美国以外的全球市场销售权。

在美国有10万人患有真性多血症，虽然已经有标准疗法，但现有疗法很难在一些临床病例中很难达到理想效果。Ruxolitinib的上市很可能改变这一困境，因而令人期待。

Incyte ($INCY) CEO Hervé Hoppenot has been telling anyone who'll listen that his biotech is undervalued and underestimated, and now, with promising late-stage results for its lead drug in a rare blood cancer, the company has a shot at blockbuster sales alongside partner Novartis ($NVS).

Ruxolitinib, already approved for myelofibrosis, met its primary endpoint in a 222-patient trial on polycythemia vera, which affects about 100,000 patients in the U.S., Incyte said. Tested on patients for whom hydroxyurea doesn't work, ruxolitinib hit its goals of normalizing red blood cell volume and reducing spleen size by 35% or more, according to the company, which is keeping full results under wraps until a later scientific meeting.

Analysts figure that success should be enough to convince regulators to expand indications for the drug, marketed as Jakafi in the U.S. J. Safra Sarasin analyst David Kaegi told Reuters he expects the treatment to win approvals for polycythemia vera by next year, figuring that'll be enough to get its peak sales over $1 billion. Incyte holds domestic rights to the JAK1/JAK2 inhibitor, while Novartis reaps worldwide profits.

"One out of four patients with polycythemia vera remain uncontrolled despite existing standard therapies, and these patients face a profound symptom burden and are at greater risk of cardiovascular complications," Hoppenot said in a statement. "These Phase III data give us confidence that ruxolitinib may offer a welcome new treatment option."

Meanwhile, Incyte's ruxolitinib fortunes will only further improve if the biotech can prove its drug's effectiveness in pancreatic cancer. Back in August, Incyte unveiled top-line Phase II results in which the drug significantly boosted overall survival in a subset of patients, sending the company's shares up nearly 30%.

And, as Hoppenot is happy to point out, Incyte's pipeline goes well beyond hoped-for new indications for ruxolitinib. The company's second lead candidate is baricitinib, a Phase III JAK1/JAK2 inhibitor for rheumatoid arthritis and autoimmune diseases codeveloped with Eli Lilly ($LLY), and it's developing INCB39110 for myelofibrosis and the PI3K-targeting INCB40093 as a combo cancer therapy. The company also struck a deal with Merck ($MRK) to study that outfit's much-lauded MK-3475 in tandem with its INCB24360 in lung cancer.

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