|[March 07, 2014]
Incyte Announces Positive Top-Line Results from Phase III Study of Ruxolitinib in Patients with Polycythemia Vera
WILMINGTON, Del. --(Business Wire)--
Incyte Corporation (Nasdaq: INCY) today announced that a pivotal Phase
III trial of ruxolitinib compared to best available therapy in patients
with polycythemia vera who are resistant to or intolerant of hydroxyurea
has met its primary endpoint of achieving phlebotomy independence and
reducing spleen size by 35 percent or more. The safety profile of
ruxolitinib was generally consistent with previous studies based on
initial review of the data. Ruxolitinib, marketed as Jakafi®
(ruxolitinib) in the United States, is approved to treat people with
intermediate or high-risk myelofibrosis (MF), including primary MF,
post-polycythemia vera MF and post-essential thrombocythemia MF.
"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.
These Phase III data give us confidence that ruxolitinib may offer a
welcome new treatment option," stated Herve Hoppenot, President and
Chief Executive Officer, Incyte.
The global, randomized, open-label Phase III trial, called RESPONSE, was
conducted at 109 sites. The trial included 222 patients with
polycythemia vera resistant to or intolerant of hydroxyurea. Patients
were randomized 1:1 to receive either ruxolitinib (10 mg twice-daily) or
best available therapy. The dose was adjusted as needed throughout the
The primary endpoint of the trial is the proportion of patients whose
hematocrit level is controlled in absence of phlebotomy and whose spleen
volume is reduced by 35 percent or more from baseline as assessed by
imaging at 32 weeks. In addition to safety, key secondary endpoints
include durable response and complete hematological remission.
Data from RESPONSE are expected to be presented at an upcoming
scientific meeting and submitted to the U.S. Food and Drug
Administration this year.
About Polycythemia Vera
Polycythemia vera (PV) is a myeloproliferative neoplasm (MPN)
characterized by an overproduction of normal red blood cells, white
blood cells and platelets that leads to an increased risk of thrombosis.1-4
Erythrocytosis (elevated red blood cell mass) is the most prominent
clinical manifestation of PV, distinguishing it from other MPNs.5
PV may occur at any age but often presents later in life, with a median
age at diagnosis of 60 years.6,7 Approximately 100,000
patients in the United States are living with PV.8 and
approximately 25 percent of patients with PV develop resistance to or
intolerance of hydroxyurea9,10 and are considered
Although patients may be asymptomatic for many years, PV is associated
with significant symptom burden, and the most common signs and symptoms
of PV are fatigue, pruritus, night sweats, bone pain, fever and weight
loss.11 Splenomegaly is present in 30 percent to 4 percent
of patients with PV.11 In patients who experience severe and
burdensome symptoms, data show that the disease causes a significant and
clinically meaningful erosion of quality of life.12,13
Jakafi is a prescription medicine approved by the U.S. Food and Drug
Administration to treat people with intermediate or high-risk
myelofibrosis (MF), including primary MF, post-polycythemia vera MF and
post-essential thrombocythemia MF. Jakafi is marketed by Incyte in the
United States and by Novartis as Jakavi® (ruxolitinib)
outside the United States.
Important Safety Information
Jakafi can cause serious side effects including:
Low blood counts: Jakafi may cause your platelet, red blood cell,
or white blood cell counts to be lowered. If you develop bleeding, stop
taking Jakafi and call your healthcare provider. Your healthcare
provider will perform blood tests to check your blood counts before you
start Jakafi and regularly during your treatment. Your healthcare
provider may change your dose of Jakafi or stop your treatment based on
the results of your blood tests. Tell your healthcare provider right
away if you experience unusual bleeding, bruising, fatigue, shortness of
breath, or a fever.
Infection: You may be at risk for developing a serious infection
while taking Jakafi. Tell your healthcare provider if you develop
symptoms such as chills, nausea, vomiting, aches, weakness, fever, or
painful skin rash or blisters.
The most common side effects of Jakafi include dizziness and headache.
These are not all the possible side effects of Jakafi. Ask your
healthcare provider or pharmacist for more information. Tell your
healthcare provider about any side effect that bothers you or that does
not go away.
Before taking Jakafi, tell your healthcare provider about all the
medications, vitamins, and herbal supplements you are taking and all
your medical conditions, including if you have an infection, have or had
liver or kidney problems, are on dialysis, or have any other medical
condition. Do not drink grapefruit juice while taking Jakafi.
Women should not take Jakafi while pregnant or planning to become
pregnant, or if breast-feeding.
Please see the Full Prescribing Information available at www.jakafi.com,
which includes a more complete discussion of the risks associated with
Incyte Corporation is a Wilmington, Delaware-based biopharmaceutical
company focused on the discovery, development and commercialization of
proprietary small molecule drugs for oncology and inflammation. For
additional information on Incyte, please visit the Company's website at www.incyte.com.
Except for the historical information set forth herein, the matters set
forth in this press release, including without limitation, statements as
to Incyte's confidence that ruxolitinib may offer a welcome new
treatment option for patients with uncontrolled PV and the expectation
that data from the RESPONSE trial will be presented at an upcoming
scientific meeting and submitted to the U.S. Food and Drug
Administration this year, contain predictions, estimates and other
These forward-looking statements are based on Incyte's current
expectations and subject to risks and uncertainties that may cause
actual results to differ materially, including unanticipated
developments in and risks related to the efficacy or safety of
ruxolitinib, risks related to market competition, the results of further
research and development, risks that results of clinical trials may be
unsuccessful or insufficient to meet applicable regulatory standards,
other market or economic factors and technological advances,
unanticipated delays, the ability of Incyte to compete against parties
with greater financial or other resources, and other risks detailed from
time to time in Incyte's reports filed with the Securities and Exchange
Commission, including its Form 10-K for the year ended December 31, 2013.
Incyte disclaims any intent or obligation to update these
1. Vannucchi AM, Guglielmelli P, Tefferi A. CA (News - Alert) Cancer J Clin. 2009;59:171-191.
2. Marchioli R, Finazzi G, Specchia G et al. N Engl J Med. 2013;368:22-33.
3. Tefferi A. Am J Hematol. 2013;88:507-516.
4. Spivak JL. Blood. 2002;100:4272-4290.
5. Spivak JL. Ann Intern Med. 2010;152:300-306.
6. Tefferi A, Rumi E, Finazzi G et al Leukemia. 2013;27:1874-1881.
7. Gruppo Italiano Studio Policitemia. Ann Intern Med. 1995;123:656-664.
8. Data on file. Incyte Corporation
9. Barosi G, Birgegard G, Finazzi G et al. Br J Haematol.
10. Alvarez-Larrán A, Pereira A, Cervantes F et al. Blood.
11. Passamonti F. Blood. 2012;120(2):275-284.
12. Mesa RA, Niblack J, Wadleigh M, et al. Cancer.
13. Johansson P, Mesa R, Scherber R, et al. Leuk Lymphoma. 2012;53(3):441-444.
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