Celgene Corporation (NASDAQ: CELG) announced an updated analysis
of data from the Phase 2 JAKARTA2 clinical study demonstrating
clinically meaningful response rates with investigational
fedratinib in patients with myelofibrosis previously treated with
ruxolitinib. This updated analysis of fedratinib employed
intent-to-treat (ITT) principles and utilized a narrower definition
of ruxolitinib relapsed, refractory, or intolerant patients.
Results were shared in a poster presentation today at the 2019
American Society of Clinical Oncology (ASCO) Annual Meeting in
Chicago.
In the ITT population (n=97), the proportion of patients who
exhibited a 35% or greater reduction in spleen volume at end of
cycle 6 was 31% (95% CI 22, 41). Of these 97 patients, 79 (81%) met
the narrower criteria for ruxolitinib resistance or intolerance. In
this cohort, the proportion of patients who exhibited a 35% or
greater reduction in spleen volume at end of cycle 6 was 30% (95%
CI 21, 42), consistent with the response rate observed in the ITT
population. In addition, the proportion of patients who exhibited a
50% or greater symptom response rate was 27% in both the ITT
population (95% CI, 18, 37) and the patients in the analysis of the
narrower criteria (95% CI, 17, 39).
The most common grade 3–4 hematologic abnormalities were anemia
(46%) and thrombocytopenia (24%). Most common non-hematologic
treatment-emergent adverse events (TEAEs) in all treated patients
were diarrhea (62%), nausea (56%), vomiting (41%), and constipation
(21%)
“Myelofibrosis is a serious and rare bone marrow disorder for
which there is only one currently approved treatment option,” said
Claire N. Harrison, M.D., Deputy Clinical Director of Cancer and
Haematology at Guy’s and St. Thomas’ NHS Foundation Trust Hospital
in London. “Patients may become intolerant or resistant to the
therapy. These updated results show meaningful reductions in spleen
volume and symptoms and reinforce the potential of fedratinib in
these difficult-to-treat patients who no longer receive benefit
from ruxolitinib.”
“Fedratinib has the potential to be the first new treatment
option since 2011 for patients with myelofibrosis,” said Dr. Alise
Reicin, President, Global Clinical Development, for Celgene.
“Patients with myelofibrosis who are relapsed, refractory or
intolerant to ruxolitinib represent a population of particularly
high unmet medical need and we are committed to bringing this
important treatment option forward.”
About JAKARTA2
JAKARTA2 is a Phase 2, multicenter, open label, single-arm trial
that evaluated the efficacy of a once daily dose of fedratinib (400
mg starting dose) in patients previously treated with ruxolitinib
and with a diagnosis of intermediate-1 with symptoms,
intermediate-2 or high-risk primary myelofibrosis,
post-polycythemia vera myelofibrosis or post-essential
thrombocythemia myelofibrosis. The study included 97 subjects
across 40 sites in 10 countries.
The primary endpoint was spleen volume response rate, defined as
the proportion of patients who had a reduction in spleen volume of
at least 35% as measured by MRI or CT scan after six one-month
treatment cycles. Secondary endpoints included symptom response
rate, defined as the proportion of patients with a 50% or greater
reduction in Total Symptom Score after six one-month treatment
cycles as measured by the modified Myelofibrosis Symptoms
Assessment Form (MFSAF) v2.0 diary.
In August 2017, the FDA removed the clinical hold on the
fedratinib development program that it had placed in 2013,
following potential cases of Wernicke's encephalopathy (WE) being
reported in eight out of 877 patients receiving one or more doses
(less than 1% of treated patients). WE is a neurological condition
induced by vitamin B1 deficiency that manifests itself in the form
of paralysis of one or more extraocular muscles, lack of muscle
coordination and confusion. Rates of WE range from 0.8% to 2.8% of
the general population, as determined by autopsy studies, however
the incidence in MPN patients is reportedly three times
greater.1
About Fedratinib
Fedratinib is an oral kinase inhibitor with activity against
wild type and mutationally activated Janus Associated Kinase 2
(JAK2) and FMS-like tyrosine kinase 3 (FLT3). Fedratinib is a
JAK2-selective inhibitor with higher potency for JAK2 over family
members JAK1, JAK3 and TYK2. Abnormal activation of JAK2 is
associated with myeloproliferative neoplasms (MPNs), including
myelofibrosis and polycythemia vera. In cell models expressing
mutationally active JAK2, fedratinib reduced phosphorylation of
signal transducer and activator of transcription (STAT3/5)
proteins, inhibited cell proliferation, and induced apoptotic cell
death. In mouse models of JAK2V617F-driven myeloproliferative
disease, fedratinib blocked phosphorylation of STAT3/5, improved
survival and disease-associated signs (including white blood cell
counts, hematocrit, splenomegaly, and fibrosis).
Fedratinib is an investigational compound that is not approved
for any use in any country.
About Myelofibrosis
Myelofibrosis is a serious and rare bone marrow disorder that
disrupts the body’s normal production of blood cells. Bone marrow
is gradually replaced with fibrous scar tissue, which limits the
ability of the bone marrow to make red blood cells.2 The disorder
can lead to anemia, weakness, fatigue and swelling of the spleen
and liver, among other symptoms.2 Myelofibrosis is classified as a
myeloproliferative neoplasm, a group of rare blood cancers that
derive from blood-forming stem cells.3 In the U.S. myelofibrosis
occurs in 1.5 of every 100,000 people each year,4 and between
16,000 and 18,500 people are living with the disease.5 Both men and
women are affected and, while the disease can affect people of all
ages, the median age at diagnosis ranges from 60 to 67
years.6,7
About Celgene
Celgene Corporation, headquartered in Summit, New Jersey, is an
integrated global biopharmaceutical company engaged primarily in
the discovery, development and commercialization of innovative
therapies for the treatment of cancer and inflammatory diseases
through next-generation solutions in protein homeostasis,
immuno-oncology, epigenetics, immunology and neuro-inflammation.
For more information, please visit www.celgene.com. Follow Celgene
on Social Media: Twitter, Pinterest, LinkedIn, Facebook and
YouTube.
FORWARD-LOOKING STATEMENTS
This press release contains forward-looking statements, which
are generally statements that are not historical facts.
Forward-looking statements can be identified by the words
"expects," "anticipates," "believes," "intends," "estimates,"
"plans," "will," "outlook" and similar expressions. Forward-looking
statements are based on management's current plans, estimates,
assumptions and projections, and speak only as of the date they are
made. We undertake no obligation to update any forward-looking
statement in light of new information or future events, except as
otherwise required by law. Forward-looking statements involve
inherent risks and uncertainties, most of which are difficult to
predict and are generally beyond our control. Actual results or
outcomes may differ materially from those implied by the
forward-looking statements as a result of the impact of a number of
factors, many of which are discussed in more detail in our Annual
Report on Form 10-K and our other reports filed with the U.S.
Securities and Exchange Commission, including factors related to
the proposed transaction between Bristol-Myers Squibb and Celgene,
such as, but not limited to, the risks that: management’s time and
attention is diverted on transaction related issues; disruption
from the transaction make it more difficult to maintain business,
contractual and operational relationships; any legal proceedings
are instituted against Bristol-Myers Squibb, Celgene or the
combined company that could delay or prevent the proposed
transaction; and Bristol-Myers Squibb, Celgene or the combined
company is unable to retain key personnel.
Hyperlinks are provided as a convenience and for informational
purposes only. Celgene bears no responsibility for the security or
content of external websites.
All trademarks are the property of their respective owners.
1 Harrison C, Mesa R, Jamieson C, et al. Case Series of
Potential Wernicke's Encephalopathy in Patients Treated with
Fedratinib. Blood 2017. 130.Suppl 1 (2017): 4197. Web.
2 Mayo Clinic. Myelofibrosis. Available at
https://www.mayoclinic.org/diseases-conditions/myelofibrosis/symptoms-causes/syc-20355057.
Accessed April 2019.
3 The Leukemia & Lymphoma Society. Myelofibrosis. Available
at https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis.
Accessed April 2019.
4 Mesa RA, Silverstein MN, Jacobsen SJ, et al. Population-based
incidence and survival figures in essential thrombocythemia and
agnogenic myeloid metaplasia: an Olmsted County Study, 1976-1995.
Am J Hematol. 1999;61(1):10-15.
5 Voices of MPN. About Myelofibrosis. Available at
https://www.voicesofmpn.com/myelofibrosis-information.aspx.
Accessed April 2019.
6 Abdel-Wahab O and Levine R. Primary myelofibrosis: Updates on
Definition, Pathogenesis and Treatment. Annual Review of Medicine.
2009;60:233-245.
7 Mesa R, Niblack J, Wadleigh M, et al. The burden of fatigue
and quality of life in myeloproliferative disorders (MPDs). Cancer.
2007;109:68-76.
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