Blood Publishes Phase III Data on Baxalta's Investigational Treatment for Von Willebrand Disease, the Most Common Type of Inherited Bleeding Disorder

  • Von Willebrand patients may experience slower blood clot formation and prolonged bleeding due to insufficient or defective factor1
  • In the study, 100 percent of patients treated with Baxalta's investigational recombinant von Willebrand factor achieved success for control of bleeding2
  • BAX 111 is another example of Baxalta's commitment to deliver transformative and personalized therapies to improve the lives of patients with blood disorders

DEERFIELD, Ill., August 3, 2015 – Baxalta Incorporated (NYSE: BXLT), a global biopharmaceutical leader dedicated to delivering transformative therapies to patients with orphan diseases and underserved conditions, announced today the publication of pivotal phase III data for BAX 111 in Blood, the journal of the American Society of Hematology. The data showed that 100 percent of the patients treated with BAX 111, a highly purified recombinant von Willebrand factor (rVWF) candidate, achieved success in the management of bleeding episodes.2 BAX 111 is currently under review by the U.S. Food and Drug Administration (FDA). If approved, BAX 111 would become the first recombinant replacement treatment for managing bleeding episodes for von Willebrand patients.3

"Von Willebrand disease is the most common hereditary bleeding disorder, yet few treatment options exist," said John Orloff, M.D., head of Research & Development and chief scientific officer, Baxalta. "BAX 111 has the potential to transform the standard of care, especially for patients with severe von Willebrand disease, by offering an effective individualized treatment option."

There is no cure for von Willebrand disease.4 The primary treatments for severe von Willebrand disease are transfusion therapy and plasma-derived VWF concentrates.2,5 Unlike other marketed VWF concentrates, BAX 111 contains a more consistent concentration of ultra-large molecular weight multimers, which have been shown to demonstrate high functional activity. 2,6

The phase III multicenter, open-label clinical trial assessed the safety, efficacy and pharmacokinetics of BAX 111 in the on-demand treatment of 37 patients with severe von Willebrand disease at trial sites in the United States, Europe, Australia, Japan, Russia and India. The primary endpoint was the number of patients experiencing successful treatment for bleeding episodes. Secondary endpoints included additional efficacy measures, pharmacokinetics, the number of infusions and the number of units administered per bleeding episode.

All study participants (100 percent) reported a mean efficacy rating of <2.5 on a four-point scale where 1=excellent control and 4=no bleeding control. Data also indicated that one infusion was sufficient to control 81.8 percent of bleeds, with a median of two infusions required to treat major bleeds. No thrombotic events or severe allergic reactions were observed, and none of the participants developed anti-VWF binding or neutralizing antibodies to VWF.2

The most common adverse events in the study were headache, vomiting/nausea and anemia (iron deficiency anemia), which were not considered to be related to treatment. One patient experienced two simultaneous serious adverse events related to treatment, characterized by chest discomfort and increased heart rate during infusion, which were resolved within three hours without further symptoms observed.2 BAX 111 was developed using a plasma- and albumin-free manufacturing method.

Both the FDA and the European Medicines Agency granted orphan drug designation in November 2010, which is reserved for products that meet medical needs for a disease that is classified as rare (affecting fewer than 200,000 people in the U.S.). Orphan exclusivity is not granted until the time of regulatory approval.

About Baxalta
Baxalta Incorporated (NYSE: BXLT) is a $6 billion global biopharmaceutical leader developing, manufacturing and commercializing therapies for orphan diseases and underserved conditions in hematology, oncology and immunology. Driven by passion to make a meaningful impact on patients' lives, Baxalta's broad and diverse pipeline includes biologics with novel mechanisms and advanced technology platforms such as gene therapy. The Baxalta Global Innovation and R&D Center is located in Cambridge, Massachusetts. Launched in 2015 following separation from Baxter International, Baxalta's heritage in biopharmaceuticals spans decades. Baxalta's therapies are available in more than 100 countries and it has advanced biological manufacturing operations across 12 facilities, including state-of-the-art recombinant production and plasma fractionation. Headquartered in Northern Illinois, Baxalta employs 16,000 employees worldwide.

Forward-Looking Statements
This release includes forward-looking statements concerning BAX 111, including expectations with regard to its potential impact on patients and related regulatory actions. Such statements are made of the date that they were first issued and are based on current expectations, beliefs and assumptions of management. Forward-looking statements are subject to a number of risks and uncertainties, many of which involve factors or circumstances that are beyond Baxalta's control and which could cause actual results to differ materially from those in the forward-looking statements, including the following: clinical trial results; satisfaction of regulatory and other requirements; actions of regulatory bodies and other governmental authorities; changes in laws and regulations; product quality, manufacturing or supply issues; patient safety issues; and other risks identified in Baxalta's Registration Statement on Form 10 and other Securities and Exchange Commission filings, all of which are available on Baxalta's website. Baxalta expressly disclaims any intent or obligation to update these forward-looking statements except as required by law. 

1.    Center for Disease Control and Prevention. Von Willebrand Disease (VWD). Accessed on July 24, 2015. Available at:
2.    Gill JC, et al. Hemostatic efficacy, safety and pharmacokinetics of a recombinant von Willebrand factor in severe von Willebrand disease. Blood. 2015
3.    Turecek PL, Mitterer A, Matthiessen HP, et al. Development of a plasma- and albumin-free recombinant von Willebrand factor. Hamostaseologie. 2009;29 (Suppl 1):32-38.
4.    National Heart, Lung and Blood Institute. In Brief: Your Guide to von Willebrand Disease.  Accessed on July 24, 2015. Available at:
5.    Tuohy E, Litt E, and Alikhan R. Treatment of patients with von Willebrand disease. J Blood Med. 2011; 2: 49–57
6.    Reininger AJ. The function of ultra-large von Willebrand factor multimers in high shear flow controlled by ADAMTS13. Hamostaseologie. 2015 May 18;35(3)

Baxalta Media Relations

Albert Liao, +1-617-834-9285, [email protected]

Baxalta Investor Relations
Mary Kay Ladone, +1-224-948-3371, [email protected] 
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