Incyte Drug Jakafi® (ruxolitinib) Improved Overall Survival in Phase III
Trial of Patients with Myelofibrosis
*New three-year COMFORT-II study data presented at the European Hematology
Association (EHA) meeting show that patients treated with Jakafi
experienced sustained reductions in spleen size and a 52 percent reduction
in the risk of death relative to patients treated with best available
*COMFORT-II data also show that Jakafi continues to be well-tolerated after
three years of treatment
*Results from a Phase I/II study also presented at EHA offer evidence that
long-term treatment with Jakafi may stabilize or reverse bone marrow
fibrosis, a key marker of worsening disease in patients with myelofibrosis
EHA Congress 2013
STOCKHOLM -- June 16, 2013
Incyte Corporation (Nasdaq: INCY) today announced results from two ongoing
clinical trials of Jakafi^® (ruxolitinib), an oral JAK1 and JAK2 inhibitor
that is FDA-approved for the treatment of patients with intermediate or
high-risk myelofibrosis (MF), that were presented at the 18^th Congress of the
European Hematology Association (EHA) in Stockholm, Sweden. In a three-year
follow-up analysis of the Phase III COMFORT-II study, treatment with Jakafi,
which is marketed as Jakavi^® by Novartis outside the United States, was
associated with improved overall survival and sustained reductions in spleen
size compared to best available therapy. In a separate exploratory analysis of
bone marrow fibrosis data from an ongoing Phase I/II single-arm, open-label
clinical trial, by 48 months of treatment, Jakafi stabilized or reversed
fibrosis of the bone marrow in 56 percent and 22 percent, respectively, of
patients with MF, a magnitude of an effect not seen historically with best
“Results of these studies represent the continuing evolution in our
understanding of the clinical benefits of Jakafi for patients with
intermediate or high-risk myelofibrosis and further support my confidence that
long-term treatment with Jakafi may modify this progressive and
life-threatening blood cancer,” stated Srdan Verstovsek, M.D., Ph.D.,
Professor, Department of Leukemia, Division of Cancer Medicine, The University
of Texas MD Anderson Cancer Center in Houston.
“Data from the Phase I/II study provide the first evidence that long-term
treatment with Jakafi may stabilize or improve bone marrow fibrosis, a key
marker of worsening disease in patients with myelofibrosis. These findings, in
addition to what was presented at ASCO, provide a result not seen before with
best available therapy, including hydroxyurea. Future studies should improve
our understanding of the significance of these findings,” stated presenting
author Hans Michael Kvasnicka, M.D., of the University of Frankfurt in
Long-Term Outcomes from a Phase III Study Comparing Ruxolitinib with Best
Available Therapy for the Treatment of Myelofibrosis: A 3-Year Update of
In a three-year follow-up analysis of the COMFORT-II study, an overall
survival advantage was observed in patients treated with Jakafi compared to
patients receiving best available therapy. A 52 percent reduction in risk of
death was observed in the Jakafi arm compared with best available therapy
(HR=0.48; 95% CI, 0.28-0.85; p=0.009)^1, and the estimated probability of
overall survival was significantly greater with Jakafi compared to best
available therapy (81 percent compared to 61 percent, respectively) at 144
weeks. Additionally, 51.4 percent of patients treated with Jakafi achieved a
≥35 percent reduction from baseline in spleen size over the course of the
study. Spleen response was maintained, with the median duration of this
response not yet reached in the study.
Anemia and thrombocytopenia were the most common adverse events over the
three-year follow-up; however, the rates of these events decreased over time.
Among patients randomized to Jakafi and included in the extension phase, the
general frequency of the most common non-hematologic adverse events
(peripheral edema, diarrhea and asthenia) did not change over time.
The results are consistent with previous COMFORT-II and COMFORT-I study
analyses, which demonstrate that Jakafi provides significant clinical benefits
over best available therapy and placebo for patients suffering from
intermediate or high-risk myelofibrosis.
The slides used during the presentation can be accessed at: EHA 2013 - 3-Year
Long-Term Intervention Effects on Bone Marrow Morphology in Myelofibrosis:
Patients Treated With Ruxolitinib and Best Available Therapy
Data were presented from an exploratory analysis that evaluated long-term data
of patients with MF who were treated with Jakafi (n=68) in a Phase I/II trial.
Biopsies were obtained at baseline and at 24 (n=68) and 48 (n=18) months, and
bone marrow fibrosis grade was determined by three expert hematopathologists
using the World Health Organization scoring system and blinded to patient data
and outcome. Stabilization or improvement of bone marrow fibrosis was observed
at both time points, and by month 48, bone marrow fibrosis was stabilized in
56 percent of ruxolitinib-treated patients and improved in 22 percent. The
percentage of ruxolitinib-treated patients with bone marrow fibrosis grade
worsening at 24 and 48 months was 37 percent and 25 percent, respectively.
Separate samples were also collected from a multicenter observational database
from three European Union countries (160 biopsies in a cohort of 139 patients)
in patients treated with best available therapy at 24 months (n=97) and 48
months (n=63)^9. Additional research is needed to understand the clinical
impact of these findings.
The slides used during the presentation can be accessed at: EHA 2013 - Bone
Marrow Fibrosis Presentation
Myelofibrosis (MF) is a life-threatening blood cancer that belongs to a group
of diseases referred to as myeloproliferative neoplasms (or MPNs). MF has a
poor prognosis and limited treatment options.^2,3 While the exact prevalence
of MF is uncertain, and estimates vary widely, based on extensive market
research, Incyte estimates MF affects about 16,000 to 18,500 people in the
U.S.^4 The enlarged spleen and debilitating symptoms of MF are linked to
dysregulated signaling in the Janus kinase (JAK) pathway. This dysregulation
may be caused by various mechanisms and mutations, such as the JAK2 V617F
mutation.^5,6 Although allogeneic stem cell transplantation may cure
myelofibrosis, the procedure is associated with significant morbidity and
transplant-related mortality and is available to less the 5 percent of
patients who are young and fit enough to undergo the procedure.^7
Jakafi is a prescription medicine used 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
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.incyte.com, which
includes a more complete discussion of the risks associated with Jakafi.
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 with respect to
the potential efficacy and therapeutic and commercial value of Jakafi
(ruxolitinib), including that treatment with Jakafi may stabilize or reverse
bone marrow fibrosis in patients with myelofibrosis, that Jakafi may provide a
survival advantage, and that long-term treatment with Jakafi may modify this
progressive and life-threatening blood cancer, and statements with respect to
future studies improving our understanding of the significance of these
findings, 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 Jakafi, the results of further research and development,
other market or economic factors 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, 2013. Incyte disclaims any intent or obligation to
update these forward-looking statements.
Links to third party websites or pages are provided for convenience only. Each
consult these policy statements. Incyte has no control over third party sites
and does not endorse or recommend these sites, and expressly disclaims any
responsibility for the accuracy of content or opinions set forth in any third
party website or your use of that information.
Vannucchi, A, et al. Long-Term Outcomes From A Phase 3 Study Comparing
1. Ruxolitinib With Best Available Therapy (BAT) For The Treatment of
Myelofibrosis (MF): A 3 Year Update of Comfort II. Abstract #S1111.18th
Congress of European Hematology Association (EHA). Stockholm, Sweden.
Kvasnicka, HM, et al. Long Term Intervention Effects on Bone Marrow
2. Morphology in Myelofibrosis: Patients Treated With Ruxolitinib and Best
Available Therapy. Abstract #S591.18th Congress of European Hematology
Association (EHA). Stockholm, Sweden.
Cervantes F, et al. New prognostic scoring system for primary
3. myelofibrosis based on a study of the International Working Group for
Myelofibrosis Research and Treatment. Blood. 2009;113:2895-2901.
Gangat N, et al. DIPSS Plus: a refined dynamic international prognostic
4. scoring system for primary myelofibrosis that incorporates prognostic
information from karyotype, platelet count, and transfusion status. J
Clin Oncol. 2011;29(4):392-397.
5. Data on File. Incyte Corporation.
Levine RL, Pardanani A, Tefferi A, Gilliland DG. Role of JAK2 in the
6. pathogenesis and therapy of myeloproliferative disorders. Nat Rev Cancer.
Vannucchi AM, Guglielmelli P, Tefferi A. Advances in understanding and
7. management of myeloproliferative neoplasms. CA Cancer J Clin.
Patriarca F, Bacigalupo A, Sperotto A, et al. Allogeneic hematopoietic
8. stem cell transplantation in myelofibrosis: the 20-year experience of the
Gruppo Italiano Trapianto di Midollo Osseo (GITMO). Haematologica.
Pamela M. Murphy, 302-498-6944
Vice President, Investor Relations & Corporate Communications
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