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
SYS-CON Events announced today that DatacenterDynamics has been named “Media Sponsor” of SYS-CON's 18th International Cloud Expo, which will take place on June 7–9, 2016, at the Javits Center in New York City, NY. DatacenterDynamics is a brand of DCD Group, a global B2B media and publishing company that develops products to help senior professionals in the world's most ICT dependent organizations make risk-based infrastructure and capacity decisions.
Between the mockups and specs produced by analysts, and resulting applications built by developers, there exists a gulf where projects fail, costs spiral, and applications disappoint. Methodologies like Agile attempt to address this with intensified communication, with partial success but many limitations. In his session at 18th Cloud Expo, Charles Kendrick, CTO & Chief Architect at Isomorphic Software, will present a revolutionary model enabled by new technologies. Learn how business and devel...
You think you know what’s in your data. But do you? Most organizations are now aware of the business intelligence represented by their data. Data science stands to take this to a level you never thought of – literally. The techniques of data science, when used with the capabilities of Big Data technologies, can make connections you had not yet imagined, helping you discover new insights and ask new questions of your data. In his session at @ThingsExpo, Sarbjit Sarkaria, data science team lead ...
The IoT has the potential to create a renaissance of manufacturing in the US and elsewhere. In his session at 18th Cloud Expo, Florent Solt, CTO and chief architect of Netvibes, will discuss how the expected exponential increase in the amount of data that will be processed, transported, stored, and accessed means there will be a huge demand for smart technologies to deliver it. Florent Solt is the CTO and chief architect of Netvibes. Prior to joining Netvibes in 2007, he co-founded Rift Technol...
If there is anything we have learned by now, is that every business paves their own unique path for releasing software- every pipeline, implementation and practices are a bit different, and DevOps comes in all shapes and sizes. Software delivery practices are often comprised of set of several complementing (or even competing) methodologies – such as leveraging Agile, DevOps and even a mix of ITIL, to create the combination that’s most suitable for your organization and that maximize your busines...
Struggling to keep up with increasing application demand? Learn how Platform as a Service (PaaS) can streamline application development processes and make resource management easy.
New Relic, Inc. has announced a set of new features across the New Relic Software Analytics Cloud that offer IT operations teams increased visibility, and the ability to diagnose and resolve performance problems quickly. The new features further IT operations teams’ ability to leverage data and analytics, as well as drive collaboration and a common, shared understanding between teams. Software teams are under pressure to resolve performance issues quickly and improve availability, as the comple...
The proper isolation of resources is essential for multi-tenant environments. The traditional approach to isolate resources is, however, rather heavyweight. In his session at 18th Cloud Expo, Igor Drobiazko, co-founder of elastic.io, will draw upon their own experience with operating a Docker container-based infrastructure on a large scale and present a lightweight solution for resource isolation using microservices. He will also discuss the implementation of microservices in data and applicat...
Join IBM June 8 at 18th Cloud Expo at the Javits Center in New York City, NY, and learn how to innovate like a startup and scale for the enterprise. You need to deliver quality applications faster and cheaper, attract and retain customers with an engaging experience across devices, and seamlessly integrate your enterprise systems. And you can't take 12 months to do it.
See storage differently! Storage performance problems have only gotten worse and harder to solve as applications have become largely virtualized and moved to a cloud-based infrastructure. Storage performance in a virtualized environment is not just about IOPS, it is about how well that potential performance is guaranteed to individual VMs for these apps as the number of VMs keep going up real time. In his session at 18th Cloud Expo, Dhiraj Sehgal, in product and marketing at Tintri, will discu...
Machine Learning helps make complex systems more efficient. By applying advanced Machine Learning techniques such as Cognitive Fingerprinting, wind project operators can utilize these tools to learn from collected data, detect regular patterns, and optimize their own operations. In his session at 18th Cloud Expo, Stuart Gillen, Director of Business Development at SparkCognition, will discuss how research has demonstrated the value of Machine Learning in delivering next generation analytics to im...
This is not a small hotel event. It is also not a big vendor party where politicians and entertainers are more important than real content. This is Cloud Expo, the world's longest-running conference and exhibition focused on Cloud Computing and all that it entails. If you want serious presentations and valuable insight about Cloud Computing for three straight days, then register now for Cloud Expo.
As you respond to increasing requests for new analytics, you need fast and flexible technology in your arsenal so that you can deploy the right workload to the right platform for the need at hand. Do you need self-service and fast time to value? Do you have data and application control and privacy needs, along with strict SLAs to meet? IBM dashDB™ is data warehouse technology powered by in-memory computing and in-database analytics that are designed for fast results, scalability and more.
SYS-CON Events announced today that SoftLayer, an IBM Company, has been named “Gold Sponsor” of SYS-CON's 18th Cloud Expo, which will take place on June 7-9, 2016, at the Javits Center in New York, New York. SoftLayer, an IBM Company, provides cloud infrastructure as a service from a growing number of data centers and network points of presence around the world. SoftLayer’s customers range from Web startups to global enterprises.
So, you bought into the current machine learning craze and went on to collect millions/billions of records from this promising new data source. Now, what do you do with them? Too often, the abundance of data quickly turns into an abundance of problems. How do you extract that "magic essence" from your data without falling into the common pitfalls? In her session at @ThingsExpo, Natalia Ponomareva, Software Engineer at Google, will provide tips on how to be successful in large scale machine lear...