Phase II Trial of Sanofi JAK2 Inhibitor in Myelofibrosis Met
Primary Endpoint
PARIS, Dec. 9, 2012 /PRNewswire/ -- Sanofi
(EURONEXT: SAN and NYSE: SNY) announced today new Phase II data
showing that treatment with a novel, investigational, selective
JAK2 inhibitor (SAR302503) reduced
spleen size and improved constitutional symptoms in patients with
intermediate-2 or high-risk primary or secondary myelofibrosis
(MF), a hematologic malignancy with unmet medical needs. The data
were presented today during the 2012 Annual Meeting of the American
Society of Hematology in Atlanta,
Ga., December 8-11, 2012.
"The results observed in our trial with SAR302503 are encouraging," said
Moshe Talpaz, M.D., Professor,
Department of Internal Medicine, University of
Michigan and lead investigator of the study. "New
treatment options are needed to fulfill existing treatment gaps for
patients with these debilitating blood disorders, and specifically
targeting the JAK2 enzyme appears to offer a promising
approach."
Results from this Phase II trial support the two doses (400 mg
and 500 mg) selected for the SAR302503 Phase III JAKARTA trial that is currently under way.
JAKARTA enrolled 289 patients over
nine months and initial results are expected in the second quarter
of 2013.
"I am very pleased with how much progress has been made in
the development of our JAK2 inhibitor. This study confirms the
once-daily oral administration of SAR302503 identified for use in the Phase III
trial in this difficult-to-treat patient population," said
Debasish Roychowdhury, M.D., Senior
Vice President and Head, Sanofi Oncology. "We believe
SAR302503 could provide a benefit to
these patients with primary and secondary myelofibrosis and
we look forward to our Phase III results next
year."
The Phase II, open label, randomized dose-ranging study
evaluates the efficacy of once-daily oral doses of 300 mg, 400 mg,
and 500 mg of SAR302503 for the
reduction of spleen volume. The primary endpoint is change in
spleen volume at the end of cycle three assessed by MRI with
independent central review. Secondary endpoints include
spleen response (reduction in spleen volume greater than or equal
to 35 percent vs. baseline), safety and symptom response using the
MPN-SAF scale.
According to the study results, treatment was associated with
reductions in spleen size and other disease symptoms in 31
randomized patients.
- Mean percentage reductions in spleen volume vs. baseline were
30% (n=10), 33% (n=10) and 42% (n=11), in each group,
respectively
- The proportion of patients who achieved a greater than or equal
to 35% reduction in spleen volume by MRI was 30%, 50% and 63.6% in
each group, respectively.
- The proportion of patients who achieved greater than or equal
to 50% reduction in the Myeloproliferative Neoplasm Symptom
Assessment Form (MPN-SAF) score, a sum of six key constitutional
symptoms (night sweats, itching, abdominal discomfort, abdominal
pain, bone pain, early satiety), was similar in all dose groups
(44%, 50% and 44%).
Consistent with data reported in previous trials, the most
common serious (grade 3-4) hematologic adverse event was anemia
with rates across the 300, 400, and 500 mg doses of 33%, 30% and
55%, respectively. Rates of grade 3-4 thrombocytopenia were
20%, 0% and 9%, respectively. The most common grade 3-4
non-hematological events were diarrhea (10%, 20%, 0%), nausea (10%,
10%, 0%) and vomiting (10%, 10%, 0%). Two patients in the 300
mg group discontinued treatment due to an adverse event (grade 3
anemia, grade 4 transaminase elevation).
About Myelofibrosis
Myelofibrosis (MF) is a rare,
debilitating and life-threatening progressive malignant hematologic
disease characterized by abnormal blood cell production and
fibrosis (scarring) within the bone marrow. Scarring in the bone
marrow interferes with blood cell production; the spleen and liver
try to produce and store extra blood cells, which can cause these
organs to become enlarged.
Most patients with MF have greatly enlarged spleens
(splenomegaly) that can result in a range of vague symptoms with
dramatic impact on quality of life. These include fatigue,
abdominal pain, night sweats, feeling full without eating, cough or
shortness of breath and decreased physical activity. Other signs
and symptoms of MF include anemia, thrombocytopenia, weight loss
and severe itching.
The exact prevalence of MF is not known. The latest
research estimates that the prevalence of MF ranges from 4.2 to 5.6
per 100,000 people in the U.S., or approximately 15,000
patients. Prevalence estimates in Europe are less clear. People over age
sixty are most likely to develop this disease, with men and women
equally at risk.
About JAK2 and SAR302503
JAK2 is a key enzyme for blood
cell development. Mutations in JAK2 can lead to dysregulated JAK2
signaling and are thought to be a cause of MF. Patients with
wild type JAK2 have also been shown to have persistent,
dysregulated activation of the JAK2 signaling pathway.
SAR302503 is a novel,
investigational, selective inhibitor of the JAK/STAT signaling
pathway that preferentially inhibits JAK2. Sanofi Oncology is
developing the compound for the treatment of the three main types
of myeloproliferative neoplasms: primary myelofibrosis,
polycythemia vera and essential thrombocythemia. Sanofi is also
studying the effect of the compound on reducing/reversing scarring
in the bone marrow.
About Sanofi Oncology
Based in Cambridge, Massachusetts, USA and Vitry,
France, Sanofi Oncology is
dedicated to translating science into effective therapeutics that
address unmet medical needs for cancer and organ transplant
patients. Starting with a deep understanding of the disease and the
patient, Sanofi Oncology employs innovative approaches to drug
discovery and clinical development, with the ultimate goal of
bringing the right medicines to the right patients to help them
live healthier and longer lives. We believe in the value of
partnerships that combine our internal scientific expertise with
that of industry and academic experts. Our portfolio includes 11
marketed products and more than 15 investigational compounds in
clinical development, including small molecules and biological
agents.
About Sanofi
Sanofi, a global and diversified
healthcare leader, discovers, develops and distributes therapeutic
solutions focused on patients' needs. Sanofi has core strengths in
the field of healthcare with seven growth platforms: diabetes
solutions, human vaccines, innovative drugs, consumer healthcare,
emerging markets, animal health and the new Genzyme. Sanofi is
listed in Paris (EURONEXT: SAN)
and in New York (NYSE: SNY).
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