Sanofi builds focus on rare blood disorders and cancers

SanofiSome of the most serious unmet patient needs today are in the field of hematology. Rare blood disorders and blood-related cancers continue to be a major focus of research as scientists look for new treatments for serious and life-threatening conditions. To help meet this need, Sanofi has significantly increased its focus on hematology, making a number of important investments to advance new therapies and improve the care of people affected by multiple myeloma, hemophilia, acquired thrombotic thrombocytopenic purpura (aTTP), cold agglutinin disease and other rare blood disorders. The company’s progress will be evident at the 60th American Society of Hematology (ASH) Annual Meeting and Exposition being held December 1 - 4 in San Diego, CA.

"Hematology has become a major focus for Sanofi, and we are very excited about the progress we are making in advancing research on a relatively broad range of potential new treatments for people with rare blood disorders and blood cancers," said Bill Sibold, Executive Vice President and Head of Sanofi Genzyme, the specialty care global business unit of Sanofi. "We look forward to sharing that progress with the hematology community at this year’s ASH meeting."

Advancements in Cancer (Hematological Malignancies)

Data presented at ASH include the latest research regarding isatuximab, an anti-CD38 monoclonal antibody in late-stage development for patients with multiple myeloma, a rare plasma cell malignancy with high unmet medical need. Isatuximab is currently being investigated in four Phase 3 clinical trials, including three company sponsored pivotal trials.

Multiple Myeloma Oral Presentations at ASH:

  • Results from a Phase II Study of Isatuximab As a Single Agent and in Combination with Dexamethasone in Patients with Relapsed/Refractory Multiple Myeloma (abstract 155; oral presentation on Saturday, December 1, 2018: 12:00-1:30 PM (PT))
  • Preliminary Results from a Phase I Study of Isatuximab (ISA) in Combination with Bortezomib, Lenalidomide, Dexamethasone (VRd), and in Patients with Newly Diagnosed Multiple Myeloma (NDMM) Non-Eligible for Transplant (abstract 595; oral presentation on Monday, December 3, 2018: 7:00-8:30 AM (PT))

Advancements in Rare Blood Disorders

Sanofi’s increased commitment to the rare blood disorders community follows the acquisition of two companies, Bioverativ and Ablynx, earlier this year. Sanofi will create a Rare Blood Disorders franchise in early 2019 that will become part of Sanofi Genzyme. In addition to two marketed therapies for the treatment of hemophilia, Sanofi’s pipeline includes novel investigational therapies in hemophilia, sickle cell disease(1), cold agglutinin disease and aTTP, a rare blood-clotting disorder.

Key Hemophilia Presentations at ASH:

  • BIVV001: The First Investigational Factor VIII Therapy to Break Through the VWF Ceiling in Hemophilia A, with Potential for Extended Protection for One Week or Longer (abstract 636; oral presentation on Monday, December 3, 2018: 10:30 AM-12:00 PM (PT)) (2)
  • ASPIRE Final Results Confirm Established Safety and Sustained Efficacy for Up to 4 Years of Treatment With rFVIIIFc in Previously Treated Subjects With Severe Hemophilia A (abstract 1192; poster on Saturday, December 1, 2018: 6:15-8:15 PM (PT)) (3)
  • B-YOND Final Results Confirm Established Safety, Sustained Efficacy, and Extended Dosing Interval for Up to 4 Years of Treatment With rFIXFc in Previously Treated Subjects With Severe Hemophilia B (abstract 1214; poster on Saturday, December 1, 2018: 6:15-8:15 PM (PT)) (3)

aTTP Oral Presentation at ASH:

  • Integrated Efficacy Results from the Phase II and Phase III Randomized Studies with Caplacizumab in Patients with Acquired Thrombotic Thrombocytopenic Purpura (abstract number 373; oral presentation on Sunday, December 2, 2018: 12:00-1:30 PM (PT))

Advancements in Rare Genetic Conditions

Sanofi Genzyme has worked in rare genetic conditions, including Gaucher disease for more than 30 years. Gaucher disease is an inherited genetic condition that can affect the blood, organs and bones. Because Gaucher disease has similar signs and symptoms of some more common hematologic malignancies, it is often misdiagnosed. Data presented at ASH includes oral eliglustat for the treatment of Gaucher disease type 1.

Gaucher Disease Presentations at ASH:

  • Long-Term Effects of Oral Eliglustat on Skeletal Manifestations of Gaucher Disease Type 1: Results from Four Completed Clinical Trials (abstract 2396; poster on Sunday, December 2, 6:00-8:00 PM (PT))
  • The Long-Term Adverse Event Profile of Oral Eliglustat for the Treatment of Gaucher Disease Type 1: Pooled Analysis of Data from 393 Patients in 4 Completed Trials (abstract 3692; poster on Monday, December 3, 6:00-8:00 PM (PT))

Isatuximab and BIVV001 are investigational agents and have not been approved by the U.S. Food and Drug Administration (FDA) or any other regulatory agency worldwide for the use under investigation.

The U.S. FDA has accepted for priority review the Biologics License Application for caplacizumab for treatment of patients 18 years of age and older experiencing an episode of aTTP. The European Commission approved caplacizumab as the first therapeutic specifically indicated for the treatment aTTP earlier this year.

About Sanofi

Sanofi is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.

With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.

Sanofi, Empowering Life

1. Bioverativ, a Sanofi company, and Sangamo Therapeutics, Inc. have an exclusive worldwide collaboration to develop and commercialize ZFN-mediated gene-edited cell therapies for the treatment of beta thalassemia and sickle cell disease.
2. Bioverativ, a Sanofi company, is responsible for the development of BIVV001
3. Bioverativ, a Sanofi company, and Sobi collaborate on the development and commercialization of rFVIIIFc and rFIXFc

Most Popular Now

Positive Phase 1 data from mRNA-based individualiz…

BioNTech SE (Nasdaq: BNTX, "BioNTech") announced initial data from an ongoing investigator-initiated first-in-human Phase 1 study evaluating the safety and tolerability o...

FDA approves RIABNI™ (rituximab-arrx), a biosimila…

Amgen (NASDAQ:AMGN) today announced that the U.S. Food and Drug Administration (FDA) has approved RIABNI™ (rituximab-arrx), a biosimilar to Rituxan®, in combination with ...

Pfizer to invest $120 million to produce COVID-19 …

Pfizer Inc. (NYSE: PFE) announced today that it will further strengthen its commitment to United States manufacturing with a $120 million investment at its Kalamazoo, Mic...

Proteomic study of 2,002 tumors identifies 11 pan-…

A new study that analyzed protein levels in 2,002 primary tumors from 14 tissue-based cancer types identified 11 distinct molecular subtypes, providing systematic knowled...

A new technology offers treatment for HIV infectio…

A new study from Tel Aviv University offers a new and unique treatment for AIDS which may be developed into a vaccine or a one time treatment for patients with HIV. The s...

Sanoff offers perspective on a promising rectal ca…

UNC Lineberger Comprehensive Cancer Center's Hanna K. Sanoff, MD, MPH, is the author of a viewpoint in the New England Journal of Medicine that provides a perspective on ...

Boehringer Ingelheim signs option to acquire Truti…

Boehringer Ingelheim announced the signing of an option to acquire Trutino Biosciences Inc. (the "Transaction"), a San Diego-based biotech company. Trutino Biosciences...

Broadly neutralizing antibodies could provide immu…

Two broadly neutralizing antibodies show great promise to provide long-acting immunity against COVID-19 in immunocompromised populations according to a paper published Ju...

Novel drug combo activates natural killer cell imm…

Most skin cancer drugs that activate the immune system work by triggering immune cells, called T cells, to attack tumors, but when T cells are activated for too long, the...

Novartis announces Nature Medicine publication of …

Novartis announced that Nature Medicine published final results from both the two- and three-copy cohorts of the completed Phase 3 SPR1NT trial as separate companion manu...

COVID-19 rebound after taking Paxlovid likely due …

Paxlovid is the leading oral medication for preventing severe cases of COVID-19 in high-risk individuals. However, symptoms returned in some patients after treatment was ...

Biomarkers found that could be drug targets agains…

Biomarkers that could be targets for novel drugs to treat glioblastoma brain tumors have been identified by investigators at Georgetown Lombardi Comprehensive Cancer Cent...