Welcome!

News Feed Item

Phase II Trial of Sanofi JAK2 Inhibitor in Myelofibrosis Met Primary Endpoint

- New Data Presented at American Society of Hematology Annual Meeting Show Activity Against Disease and Symptom Alleviation -

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).

Forward Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words "expects", "anticipates", "believes", "intends", "estimates", "plans" and similar expressions. Although Sanofi's management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the absence of guarantee that the product candidates if approved will be commercially successful, the future approval and commercial success of therapeutic alternatives, the Group's ability to benefit from external growth opportunities, trends in exchange rates and prevailing interest rates, the impact of cost containment policies and subsequent changes thereto, the average number of shares outstanding as well as those discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under "Risk Factors" and "Cautionary Statement Regarding Forward-Looking Statements" in Sanofi's annual report on Form 20-F for the year ended December 31, 2011. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements.


Contacts: 




Sanofi 


Media Relations  

Investor Relations

Marisol Peron         

Sebastien Martel

Tel. : + (33) 1 53 77 45 02 

Tel. : + (33) 1 53 77 45 45

E-mail: [email protected]    

E-mail: [email protected]



Lauren Musto 


Oncology Division Communications


Tel: 1 (617) 768-1993; Mobile 1 (781) 572-1147


E-mail: [email protected]




Carrie Brown


U.S. Oncology Division Communications 


Tel: 1 (908) 981-6486; Mobile 1 (908) 247-6006


E-mail: [email protected]


SOURCE Sanofi

More Stories By PR Newswire

Copyright © 2007 PR Newswire. All rights reserved. Republication or redistribution of PRNewswire content is expressly prohibited without the prior written consent of PRNewswire. PRNewswire shall not be liable for any errors or delays in the content, or for any actions taken in reliance thereon.

Latest Stories
DXWorldEXPO LLC, the producer of the world's most influential technology conferences and trade shows has announced the 22nd International CloudEXPO | DXWorldEXPO "Early Bird Registration" is now open. Register for Full Conference "Gold Pass" ▸ Here (Expo Hall ▸ Here)
Join IBM November 1 at 21st Cloud Expo at the Santa Clara Convention Center in Santa Clara, CA, and learn how IBM Watson can bring cognitive services and AI to intelligent, unmanned systems. Cognitive analysis impacts today’s systems with unparalleled ability that were previously available only to manned, back-end operations. Thanks to cloud processing, IBM Watson can bring cognitive services and AI to intelligent, unmanned systems. Imagine a robot vacuum that becomes your personal assistant tha...
Your homes and cars can be automated and self-serviced. Why can't your storage? From simply asking questions to analyze and troubleshoot your infrastructure, to provisioning storage with snapshots, recovery and replication, your wildest sci-fi dream has come true. In his session at @DevOpsSummit at 20th Cloud Expo, Dan Florea, Director of Product Management at Tintri, provided a ChatOps demo where you can talk to your storage and manage it from anywhere, through Slack and similar services with...
Containers are rapidly finding their way into enterprise data centers, but change is difficult. How do enterprises transform their architecture with technologies like containers without losing the reliable components of their current solutions? In his session at @DevOpsSummit at 21st Cloud Expo, Tony Campbell, Director, Educational Services at CoreOS, will explore the challenges organizations are facing today as they move to containers and go over how Kubernetes applications can deploy with lega...
In their session at @DevOpsSummit at 21st Cloud Expo, Michael Berman, VP Engineering at TidalScale, and Ivo Jimenez, Engineer at TidalScale, will describe how automating tests in TidalScale is easy thanks to WaveRunner. They will show how they use WaveRunner, Jenkins, and Docker to have agile delivery of TidalScale. Michael Berman is VP Engineering at TidalScale. TidalScale is developing a scale up compute and resource architecture for customers to perform big data exploration and real time anal...
I think DevOps is now a rambunctious teenager - it's starting to get a mind of its own, wanting to get its own things but it still needs some adult supervision," explained Thomas Hooker, VP of marketing at CollabNet, in this SYS-CON.tv interview at DevOps Summit at 20th Cloud Expo, held June 6-8, 2017, at the Javits Center in New York City, NY.
"MobiDev is a software development company and we do complex, custom software development for everybody from entrepreneurs to large enterprises," explained Alan Winters, U.S. Head of Business Development at MobiDev, in this SYS-CON.tv interview at 21st Cloud Expo, held Oct 31 – Nov 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA.
"With Digital Experience Monitoring what used to be a simple visit to a web page has exploded into app on phones, data from social media feeds, competitive benchmarking - these are all components that are only available because of some type of digital asset," explained Leo Vasiliou, Director of Web Performance Engineering at Catchpoint Systems, in this SYS-CON.tv interview at DevOps Summit at 20th Cloud Expo, held June 6-8, 2017, at the Javits Center in New York City, NY.
The “Digital Era” is forcing us to engage with new methods to build, operate and maintain applications. This transformation also implies an evolution to more and more intelligent applications to better engage with the customers, while creating significant market differentiators. In both cases, the cloud has become a key enabler to embrace this digital revolution. So, moving to the cloud is no longer the question; the new questions are HOW and WHEN. To make this equation even more complex, most ...
Recently, WebRTC has a lot of eyes from market. The use cases of WebRTC are expanding - video chat, online education, online health care etc. Not only for human-to-human communication, but also IoT use cases such as machine to human use cases can be seen recently. One of the typical use-case is remote camera monitoring. With WebRTC, people can have interoperability and flexibility for deploying monitoring service. However, the benefit of WebRTC for IoT is not only its convenience and interopera...
Learn how to solve the problem of keeping files in sync between multiple Docker containers. In his session at 16th Cloud Expo, Aaron Brongersma, Senior Infrastructure Engineer at Modulus, discussed using rsync, GlusterFS, EBS and Bit Torrent Sync. He broke down the tools that are needed to help create a seamless user experience. In the end, can we have an environment where we can easily move Docker containers, servers, and volumes without impacting our applications? He shared his results so yo...
Creating replica copies to tolerate a certain number of failures is easy, but very expensive at cloud-scale. Conventional RAID has lower overhead, but it is limited in the number of failures it can tolerate. And the management is like herding cats (overseeing capacity, rebuilds, migrations, and degraded performance). In his general session at 18th Cloud Expo, Scott Cleland, Senior Director of Product Marketing for the HGST Cloud Infrastructure Business Unit, discussed how a new approach is neces...
"This week we're really focusing on scalability, asset preservation and how do you back up to the cloud and in the cloud with object storage, which is really a new way of attacking dealing with your file, your blocked data, where you put it and how you access it," stated Jeff Greenwald, Senior Director of Market Development at HGST, in this SYS-CON.tv interview at 18th Cloud Expo, held June 7-9, 2016, at the Javits Center in New York City, NY.
"ZeroStack is a startup in Silicon Valley. We're solving a very interesting problem around bringing public cloud convenience with private cloud control for enterprises and mid-size companies," explained Kamesh Pemmaraju, VP of Product Management at ZeroStack, in this SYS-CON.tv interview at 21st Cloud Expo, held Oct 31 – Nov 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA.
Cloud-enabled transformation has evolved from cost saving measure to business innovation strategy -- one that combines the cloud with cognitive capabilities to drive market disruption. Learn how you can achieve the insight and agility you need to gain a competitive advantage. Industry-acclaimed CTO and cloud expert, Shankar Kalyana presents. Only the most exceptional IBMers are appointed with the rare distinction of IBM Fellow, the highest technical honor in the company. Shankar has also receive...