Incyte Corp. (INCY) may soon learn the fate of its myelofibrosis drug Ruxolitinib, which if approved , will be the first effective drug for the potentially life-threatening disorder characterized by scarring of tissue in the bone marrow.
Myelofibrosis, which affects about 3,500 people annually in the United States, is characterized by significant anemia, an enlarged spleen, intense itching, weight loss and bone pain. There are currently no FDA-approved therapies for myelofibrosis.
The existing treatment options like androgen therapy, chemotherapy, radiation therapy, splenectomy (surgical removal of spleen) , blood transfusions and medications like thalidomide plus steroids are capable of only managing the symptoms of myelofibrosis but not effective in curing the disease. Stem cell transplants are considered the only cure for myelofibrosis.
Incyte's Ruxolitinib is an inhibitor of Janus kinases enzymes - JAK1 and JAK2, that mediate signaling of several important drivers of myeloproliferative neoplasms like myelofibrosis, other hematological malignancies and inflammatory diseases. The FDA as well as the European Commission have granted Ruxolitinib orphan drug status for treating myelofibrosis.
As part of a collaboration and license agreement signed in November 2009, Novartis AG (NVS) licensed Ruxolitinib from Incyte for the development and potential commercialization outside the U.S. The development and commercialization rights to Ruxolitinib in the United States are retained by Incyte.
Incyte submitted a New Drug Application for Ruxolitinib to the FDA, seeking approval of the drug in the treatment of myelofibrosis, in June of this year, and also requested a Priority Review of the application.
The NDA of Ruxolitinib was based on the results of two phase III trials - COMFORT-I and COMFORT-II, conducted as part of the Incyte-Novartis worldwide collaboration and license agreement. (COMFORT - COntrolled MyeloFibrosis Study With ORal JAK Inhibitor Treatment).
According to the company, in clinical trials, patients treated with Ruxolitinib experienced significant reductions in splenomegaly and improvement in symptoms and overall quality of life measures while patients in the control arms, (placebo and best available therapy) , experienced progressive splenomegaly and worsening of symptoms. However, Ruxolitinib was associated with adverse effects like anemia and thrombocytopenia, but only at the beginning of the treatment.
The FDA, which has granted Priority Review for the Ruxolitinib NDA, is expected to announce its decision whether or not to approve the drug for myelofibrosis on December 3, 2011.
Ruxolitinib is also under review in Europe and its Marketing Authorization Application was submitted by Novartis in June.
In addition to myelofibrosis, Ruxolitinib is tested for other indications like polycythemia vera, hematologic malignancies and solid tumors. A global phase III registration study, dubbed RESPONSE, is studying Ruxolitinib in patients with advanced polycythemia vera. This study is being conducted by Incyte in the US and Novartis outside of the US.
Yet another Janus Kinase inhibitor in Incyte's pipeline is LY3009104, which is under phase IIb trial for rheumatoid arthritis. Under an agreement signed in December 2009, Eli Lilly & Co. (LLY) received exclusive worldwide development and commercialization rights to LY3009104. However in July 2010, Incyte elected to co-develop LY3009104 with Lilly in rheumatoid arthritis in exchange for tiered royalty rates ranging up to the high twenties.
A quick look at the balance sheet...
The company had incurred losses from inception in 1991 through 1996 and in 1999 through 2010. As of June 30, 2011, the accumulated deficit totaled $1.5 billion. Incyte derives revenue from collaborations and from licensing its intellectual property.
In the second quarter ended June 30, 2011 Incyte incurred a net loss of $51.9 million or $0.41 per share, reversing a net income of $3.0 million or $0.02 per share in the year-ago quarter. Total revenue, which includes contract revenue as well as license & royalty revenues, was $16.8 million in the second quarter of 2011, down from $49.8 million in the comparable quarter a year before.
Incyte shares have traded in a 52-week range of $12.58 to $21.15. The stock closed Monday's trading at $16.29, up 4.09%.
As time draws nearer to the FDA decision, it will be interesting to see how the Ruxolitinib story plays out. Will Incyte be credited for developing the first oral JAK inhibitor drug for advanced myelofibrosis. Stay tuned...
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