Pivotal Phase III Data in Polycythemia Vera Show that Jakafi® (ruxolitinib)
Achieved Superior Disease Control Compared to Best Available Therapies
*The trial met the primary composite endpoint of hematocrit control and at
least a 35 percent reduction in spleen volume
*77 percent of patients treated with ruxolitinib versus 20 percent on best
available therapy achieved one or both of the components of the primary
*Approximately half of patients in the ruxolitinib group had at least a 50
percent improvement in symptom score, compared with 5 percent on best
*Global regulatory filings are underway based on these data; if approved,
ruxolitinib would be the first JAK1/JAK2 inhibitor available for PV
Incyte to host a webcast for investors featuring key results from this trial
on Tuesday, June 3, at 1:15 p.m. CDT
2014 ASCO Annual Meeting
CHICAGO -- June 3, 2014
Incyte Corporation (Nasdaq: INCY) today announced results from the RESPONSE
trial, the first pivotal Phase III study evaluating a JAK1/JAK2 inhibitor for
the treatment of polycythemia vera (PV). Ruxolitinib, compared to best
available therapy (BAT), significantly improved hematocrit control (red blood
cell volume) without the need for phlebotomy (a procedure to remove blood from
the body to reduce the concentration of red blood cells) and reduced spleen
size in patients with uncontrolled PV -- those who are resistant to or
intolerant of hydroxyurea (HU). Findings from the RESPONSE study are being
presented in an oral presentation at the 50th Annual Meeting of the American
Society of Clinical Oncology in Chicago.
“Patients with advanced PV whose disease is not well-managed with existing
therapies are at increased risk for thrombosis and suffer from multiple
debilitating symptoms,” stated Srdan Verstovsek, M.D., Ph.D., Professor,
Department of Leukemia, Division of Cancer Medicine, The University of Texas
MD Anderson Cancer Center. “Data from the RESPONSE trial demonstrated that
treatment with ruxolitinib can consistently control hematocrit, reduce spleen
size, and improve symptoms such as fatigue and itching. Importantly, there
appears to be a lower rate of thrombosis in the ruxolitinib arm compared to
best available therapy.”
Seventy-seven percent of ruxolitinib-treated patients versus 20 percent on BAT
achieved at least one component of the primary endpoint: hematocrit control
from week 8 to 32 and/or at least a 35 percent reduction in spleen volume. A
greater proportion of patients treated with ruxolitinib achieved the composite
primary endpoint compared to BAT (21 percent vs 1 percent, respectively; P<
.0001); 91 percent of patients in the ruxolitinib group achieving this
endpoint maintained their response at week 48.
A greater proportion of patients in the ruxolitinib treatment arm had complete
hematologic remission, a key secondary endpoint, when compared to the BAT arm
(24 percent compared to 9 percent, P=.003). Patients treated with ruxolitinib
also experienced meaningful improvements in PV-related symptoms: 49 percent,
compared to 5 percent treated with BAT, had a 50 percent or greater
improvement in symptom score at week 32 as measured by the 14-item MPN-SAF
(Myeloproliferative Neoplasm Symptom Assessment Form). At week 32, one patient
in the ruxolitinib group and six in the BAT group had a thromboembolic event.
At a median follow-up of 81 weeks, 85 percent of patients in the ruxolitinib
arm were still receiving treatment. Because most patients in the BAT group
crossed over to receive ruxolitinib therapy at week 32, adverse events were
evaluated at this time when exposure between groups was similar. The most
common non-hematologic adverse events of any grade in the ruxolitinib group
compared to the BAT group were headache (16.4 percent vs 18.9 percent),
diarrhea (14.5 percent vs 7.2 percent), fatigue (14.5 percent vs 15.3
percent), and pruritus (13.6 percent vs 22.5 percent). Based on laboratory
assessments, the rates of new or worsening grade 3 or 4 anemia and
thrombocytopenia in the ruxolitinib group versus the BAT group were 1.8
percent vs 0 percent and 5.5 percent vs 3.6 percent, respectively.
“One out of four patients with polycythemia vera remain uncontrolled, face a
profound symptom burden and are at greater risk of cardiovascular
complications such as stroke and heart attack,” stated Hervé Hoppenot,
President and Chief Executive Officer, Incyte. “These Phase III data give us
confidence that ruxolitinib has the potential to become an important new
treatment option for patients with uncontrolled PV who are no longer
responding to or are intolerant of hydroxyurea.”
These data will support global regulatory filings anticipated this year,
including a submission to the U.S. Food and Drug Administration expected this
The slides used during the presentation can be accessed at 2014 ASCO -
About the RESPONSE Trial
RESPONSE is an open-label randomized trial of 222 patients conducted in North
America, Europe, Asia, and Australia. Patients with splenomegaly who were
resistant to or intolerant of hydroxyurea (HU) and required phlebotomy were
randomized 1:1 to receive ruxolitinib 10 mg twice daily or BAT, which was
defined as investigator selected monotherapy or observation only. From week
32, patients in the BAT group could cross over to receive ruxolitinib therapy.
The primary endpoint of the study is the proportion of patients who achieved
both hematocrit control without the need for phlebotomy from week 8 through 32
and a spleen volume reduction of at least 35 percent from baseline at 32
weeks. Key secondary endpoints include durable primary response and complete
hematologic remission. Complete hematologic remission was defined as
maintaining hematocrit control without the need for phlebotomy, a platelet
count ≤400 x 10^9/L and white blood cell count ≤10 x 10^9/L. Other secondary
endpoints include safety, symptom improvement, and quality of life.
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 hydroxyurea^9,10 and are considered uncontrolled.
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 40 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
About Jakafi® (ruxolitinib)
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
Please see the Full Prescribing Information available at www.jakafi.com, which
includes a more complete discussion of the risks associated with Jakafi.
About the Webcast
Incyte will host an investor meeting, which will be webcast live at 1:15 p.m.
CDT on June 3, 2014, and can be accessed at www.incyte.com under Investor
Relations, Events and Webcasts. A replay of the event will be available for 60
Incyte Corporation is a Wilmington, Delaware-based biopharmaceutical company
focused on the discovery, development and commercialization of proprietary
small molecule drugs, primarily in oncology. 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 with respect to
the potential efficacy, safety and therapeutic value of, and Incyte’s plans
for, ruxolitinib in polycythemia vera, including the potential for ruxolitinib
to become an important new treatment option for patients with uncontrolled
polycythemia vera and the expectation to file a regulatory submission with the
FDA this month, contain predictions and estimates and are forward-looking
statements within the meaning of the "safe harbor" provisions of the Private
Securities Litigation Reform Act of 1995. 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,
the results of further research and development, the high degree of risk and
uncertainty associated with drug development, clinical trials and regulatory
approval processes, other market or economic factors, competitive and
technological advances, and other risks detailed from time to time in Incyte's
filings with the Securities and Exchange Commission, including its Quarterly
Report on Form 10-Q for the quarter ended March 31, 2014. Incyte disclaims any
intent or obligation to update these forward-looking statements.
1. Vannucchi AM, Guglielmelli P, Tefferi A. CA Cancer J Clin. 2009;59:171-91.
2. Marchioli R, Finazzi G, Specchia G, et al. N Engl J Med. 2013;368:22-33.
3. Tefferi A. Am J Hematol. 2013;88:507-16.
4. Spivak JL. Blood. 2002;100:4272-90.
5. Spivak JL. Ann Intern Med. 2010;152:300-6.
6. Tefferi A, Rumi E, Finazzi G, et al. Leukemia. 2013;27:1874-81.
7. Gruppo Italiano Studio Policitemia. Ann Intern Med. 1995;123:656-64.
8. Data on file. Incyte Corporation
9. Barosi G, Birgegard G, Finazzi G, et al. Br J Haematol. 2010;149:961-3.
10. Alvarez-Larrán A, Pereira A, Cervantes F, et al. Blood. 2012;119:1363-9
11. Passamonti F. Blood. 2012;120(2):275-84.
12. Mesa RA, Niblack J, Wadleigh M, et al. Cancer. 2007;109(1):68-76.
13. Johansson P, Mesa R, Scherber R, et al. Leuk Lymphoma. 2012;53(3):441-4.
Pamela M. Murphy
Vice President, Investor Relations & Corporate Communications
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