- First and Only FDA-Approved Treatment For Potentially
Life-Threatening Blood Cancer
- Product Available Next Week; Incyte Establishes Comprehensive
Patient Assistance Program
- Conference Call Scheduled, Today at 12:30 PM ET
WILMINGTON, Del.--(BUSINESS WIRE)--Nov. 16, 2011--
Incyte Corporation (Nasdaq: INCY) today announced that the U.S. Food and
Drug Administration (FDA) has granted marketing approval for Jakafi™
(ruxolitinib) for the treatment of patients with intermediate or
high-risk myelofibrosis (MF), including primary MF, post-polycythemia
vera MF and post-essential thrombocythemia MF. Patients with
intermediate and high-risk MF represent 80 to 90 percent of MF patients.
Jakafi (JAK-ah-fye) is the first and only product to be approved by the
FDA for MF, and the first in a new class of drugs, known as JAK
inhibitors, to be approved for any indication. Jakafi is an oral JAK1
and JAK2 inhibitor.
Jakafi(TM) (ruxolitinib) is the first and only product to be approved by the FDA for myelofibrosis, and the first in a new class of drugs, known as JAK inhibitors, to be approved for any indication. (Photo: Business Wire)
“The availability of Jakafi is a significant medical advancement for
people living with myelofibrosis, a debilitating disease,” stated Paul
A. Friedman, M.D., President and Chief Executive Officer of Incyte.
“This milestone marks a tremendous achievement for Incyte because a
scientific discovery from our research laboratories has become the first
JAK inhibitor to reach the market and provide a clinical benefit to
MF is a progressive, potentially life-threatening blood cancer with
limited treatment options.1 Patients with MF suffer a high
disease burden characterized by bone marrow failure, enlarged spleen
(splenomegaly) and debilitating symptoms including fatigue, severe
itching (pruritus), night sweats, bone pain, and early satiety (a
feeling of fullness), leading to impaired quality of life.2
The enlarged spleen and debilitating symptoms of MF are linked to
dysregulated signaling in the Janus kinase (JAK) pathway.3,4
“Today’s FDA approval of Jakafi has the potential to transform the way
we treat myelofibrosis,” said Srdan Verstovsek, M.D., Ph.D., Associate
Professor, Department of Leukemia, Division of Cancer Medicine, The
University of Texas MD Anderson Cancer Center and the principal
investigator of the COMFORT-I pivotal trial. “In this Phase III clinical
trial, we observed significant reductions in spleen size and significant
improvements in symptoms. Importantly, these benefits were achieved
early on, most within a month, and tended to be durable during
treatment. In contrast, most of the patients who received placebo saw
their spleens increase and their symptoms worsen.”
“We are very excited about the first FDA approval of a treatment for MF.
Not only is this a new therapy, but it brings additional education,
awareness and attention to a profoundly debilitating disease,” stated
Robert Rosen, President of MPN Research Foundation.
Phase III Clinical Trial Data
The FDA approval was based on results from two randomized Phase III
trials (COMFORT-I and COMFORT-II), which demonstrated that patients
treated with Jakafi experienced significant reductions in splenomegaly
(enlarged spleen). COMFORT-I also demonstrated improvements in symptoms
as measured by the modified Myelofibrosis Symptom Assessment Form
(MFSAF) v.2.0 electronic diary and the MFSAF Total Symptom Score (TSS)
comprised of six specific symptoms (abdominal discomfort, pain under the
left ribs, an early feeling of fullness, night sweats, bone and muscle
pain and itching) all of which contributed to the overall benefit. Most
patients taking placebo experienced worsening of these same parameters.
The COMFORT-I trial, conducted by Incyte, compared Jakafi to placebo in
309 patients with primary MF, post-polycythemia vera MF and
post-essential thrombocythemia MF. The trial met the primary endpoint,
showing that 41.9% of patients treated with Jakafi experienced a 35% or
greater reduction in spleen volume at 24 weeks, compared with 0.7% of
patients taking placebo (p<0.0001). A 35% reduction in spleen volume
correlates to approximately a 50% reduction in spleen size on palpation.
At week 24, the percentage of patients with a greater than or equal to
50% improvement in the TSS was 45.9% and 5.3% in patients treated with
Jakafi and placebo, respectively (p<0.0001), with a median time to
response of less than four weeks.
The COMFORT-II trial, conducted by Novartis, Incyte’s collaboration
partner outside of the U.S., compared Jakafi to best available therapy
in 219 patients with primary MF, post-polycythemia vera MF and
post-essential thrombocythemia MF. This trial also met the primary
endpoint, showing that 28.5% of patients treated with Jakafi experienced
a 35% or greater reduction in spleen volume at 48 weeks, compared with
0% of patients in the best available therapy arm (p<0.0001).
The most common adverse reactions in both studies were thrombocytopenia
and anemia. These events were manageable and rarely led to
discontinuation of Jakafi treatment. The most common non-hematologic
adverse reactions were bruising, dizziness, and headache.
Please see Important Safety Information below, and the full Prescribing
Information for Jakafi at www.jakafi.com
Indication, Usage and Dosing
Jakafi is indicated for treatment of patients with intermediate or
high-risk myelofibrosis, including primary MF, post-polycythemia vera MF
and post-essential thrombocythemia MF. Intermediate and high-risk MF
patients include anyone over the age of 65 or who have or have had any
of the following: anemia, constitutional symptoms, elevated white blood
cell or blast counts or platelet counts less than 100 X 109/L.1,5
The recommended starting dose for most patients is either 15 mg or 20 mg
given orally twice daily based on the patient’s platelet count. Dosage
should be adjusted based on safety and efficacy. A blood cell count must
be performed before initiating therapy with Jakafi and complete blood
counts should be monitored every 2-4 weeks until doses are stabilized.
Important Safety Information
Treatment with Jakafi can cause hematologic adverse reactions, including
thrombocytopenia, anemia and neutropenia, which are each dose-related
effects, with the most frequent being thrombocytopenia and anemia. A
complete blood count must be performed before initiating therapy with
Jakafi. Complete blood counts should be monitored as clinically
indicated and dosing adjusted as required. The three most frequent
non-hematologic adverse reactions were bruising, dizziness and headache.
Patients with platelet counts less than 200 X 109/L at the
start of therapy are more likely to develop thrombocytopenia during
treatment. Thrombocytopenia was generally reversible and was usually
managed by reducing the dose or temporarily withholding Jakafi. If
clinically indicated, platelet transfusions may be administered.
Patients developing anemia may require blood transfusions. Dose
modifications of Jakafi for patients developing anemia may also be
considered. Neutropenia (ANC <0.5 X 109/L) was generally
reversible and was managed by temporarily withholding Jakafi. Patients
should be assessed for the risk of developing serious bacterial,
mycobacterial, fungal and viral infections. Active serious infections
should have resolved before starting Jakafi. Physicians should carefully
observe patients receiving Jakafi for signs and symptoms of infection
(including herpes zoster) and initiate appropriate treatment promptly. A
dose modification is recommended when administering Jakafi with strong
CYP3A4 inhibitors or in patients with renal or hepatic impairment [see
Dosage and Administration]. Patients should be closely monitored and
the dose titrated based on safety and efficacy. There are no adequate
and well-controlled studies of Jakafi in pregnant women. Use of Jakafi
during pregnancy is not recommended and should only be used if the
potential benefit justifies the potential risk to the fetus. Women
taking Jakafi should not breast-feed. Discontinue nursing or discontinue
the drug, taking into account the importance of the drug to the mother.
Patient Assistant Program: IncyteCARES
Incyte has established IncyteCARES (Connecting to Access, Reimbursement,
Education and Support), a comprehensive program that provides
reimbursement support and educational resources for patients. Incyte is
committed to providing financial assistance for patients in need who
qualify for the available support programs. A toll-free number has been
established to provide support regarding benefit verification, prior
authorization and assistance with appeals. IncyteCARES also offers
patient educational materials, resources and access to trained nurse
professionals to answer questions regarding the program.
Jakafi will be available in the United States next week through a number
of specialty pharmacies. Patients can access information about Jakafi
and the IncyteCARES program by calling 1-855-4-Jakafi (855-452-5234) or
Myelofibrosis (MF) is a potentially 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.1 While the exact prevalence of MF is uncertain, and
estimates vary widely, based on extensive market research, Incyte
believes MF affects about 16,000 to 18,500 people in the U.S.6
About the MPN Research Foundation
The primary mission of the MPN Research Foundation is to promote, fund
and support the most innovative and effective research into the causes,
treatments, and potentially the cure for essential thrombocythemia,
polycythemia vera and myelofibrosis. For more information, go to http://www.mpnresearchfoundation.org/.
About the Incyte-Novartis Collaboration
In 2009, Incyte entered into a worldwide collaboration and license
agreement with Novartis. Incyte retained exclusive rights for the
development and commercialization of ruxolitinib (INCB424) in the United
States. Novartis received exclusive rights to the development and
potential commercialization of ruxolitinib in all hematology-oncology
indications outside of the United States.
Conference Call Information
Incyte will hold a conference call today at 12:30 PM ET to discuss the
FDA approval of Jakafi, its price, and Incyte’s patient assistance
program. To access the conference call, please dial 877-407-8037 for
domestic callers or 201-689-8037 for international callers. When
prompted, provide the conference identification number, 383585.
If you are unable to participate, a replay of the conference call will
be available for thirty days. The replay dial-in number for the U.S. is
877-660-6853 and the dial-in number for international callers is
201-612-7415. To access the replay you will need the conference account
number 278 and the identification number 383585.
The conference call will also be webcast live and can be accessed at www.incyte.com
under Investor Relations, Events and Webcasts.
Incyte Corporation is a Wilmington, Delaware-based biopharmaceutical
company focused on developing and commercializing proprietary small
molecule drugs for oncology and inflammation. For additional information
on Incyte, please visit www.incyte.com.
Except for the historical information contained herein, the matters set
forth in this press release, including statements with respect to Jakafi
having the potential to transform the way physicians treat myelofibrosis
and Jakafi being available in the United States next week through a
number of specialty pharmacies, are all 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 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, the
acceptance of Jakafi in the marketplace, risks related to market
competition, risks and uncertainties associated with sales, marketing
and distribution requirements, risks associated with Incyte’s dependence
on its third party manufacturers 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
September 30, 2011. Incyte disclaims any intent or obligation to update
these forward-looking statements.
Links to third party websites or pages are provided for convenience
encourages you to 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
Cervantes F, Dupriez B, Pereira A, et al. New prognostic scoring
system for primary myelofibrosis based on a study of the
International Working Group for Myelofibrosis Research and
Treatment. Blood. 2009;113:2895-2901.
Mesa RA, Niblack J, Wadleigh M, et al. The burden of fatigue and
quality of life in myeloproliferative disorders (MPDs): an
international internet-based survey of 1179 MPD patients. Cancer.
Levine RL, Pardanani A, Tefferi A, Gilliland DG. Role of JAK2 in
the pathogenesis and therapy of myeloproliferative disorders. Nat
Rev Cancer. 2007;7:673-683.
Vannucchi AM, Guglielmelli P, Tefferi A. Advances in understanding
and management of myeloproliferative neoplasms. CA Cancer J
Gangat N, Caramazza D, Vaidya R, et al. DIPSS Plus: A Refined
Dynamic International Prognostic Scoring System for Primary
Myelofibrosis That Incorporates Prognostic Information From
Karyotype, Platelet Count, and Transfusion Status. J Clin Oncol.
Data on File. Incyte Corporation.
Photos/Multimedia Gallery Available: http://www.businesswire.com/cgi-bin/mmg.cgi?eid=50075108&lang=en
Source: Incyte Corporation
Pamela M. Murphy
Investor Relations/Corporate Communications
Sam Brown Inc.