- Company confirms submission of clinical trial application (CTA) to initiate the clinical study in the United Kingdom
- Baxalta’s second investigational extended half-life candidate based on ADVATE [Antihemophilic Factor (Recombinant)] uses proprietary polysialic acid (PSA) technology that aims to extend dosing intervals
- Ongoing pipeline development for hemophilia treatments aims to introduce new therapeutic options based on the innovative science of factor replacement to help as many patients as possible achieve a goal of zero bleeds
BANNOCKBURN, IL, USA I November 20, 2015 I Baxalta Incorporated (NYSE: BXLT), a global biopharmaceutical leader dedicated to delivering transformative therapies to patients with orphan diseases and underserved conditions, today announced the submission of a Clinical Trial Application (CTA) to the UK Medicines and Healthcare Products Regulatory Agency (MHRA) to initiate a first-in-human clinical trial to evaluate the safety and efficacy of BAX 826, an investigational, extended half-life recombinant Factor VIII (rFVIII) treatment for hemophilia A.
“We are advancing a number of approaches, including BAX 826 as well as our gene therapy program, to evaluate potential new options for hemophilia patients that can offer efficacy while also easing the treatment burden with a goal of once-weekly or even less frequent infusions,” said John Orloff, M.D., head of Research & Development and chief scientific officer, Baxalta. “The CTA submission represents an important advancement in our efforts to expand our portfolio of extended half-life treatments and further reinforces our leadership in hemophilia innovation.”
The open-label, dose-finding study of BAX 826 aims to enroll 30 patients; Baxalta expects to begin treating participants in the study by early 2016. BAX 826 is a next-generation rFVIII treatment based on the full length ADVATE [Antihemophilic Factor (Recombinant)] molecule. The compound is modified using proprietary polysialic acid (PSA) technology licensed from Xenetic Biosciences, Inc. (OTCQB: XBIO) to extend its circulating half-life. Baxalta has partnered with Xenetic to develop novel forms of polysialylated blood coagulation factors, including factor VIII. Xenetic’s PolyXen™ technology utilizes the biopolymer PSA in order to extend the circulating half-life and improve the pharmacokinetic profile of therapeutic proteins, peptides, and small molecules. Preclinical studies indicated BAX 826 offered an extended half-life compared to standard rFVIII.
With the BAX 826 program, Baxalta aims to augment its growing portfolio of direct factor replacement treatments for hemophilia, including ADVATE as well as ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated], which was recently approved in the United States. In addition, the company continues to advance hemophilia innovation with the development of new advances like gene therapy, which has the potential to transform the treatment of hemophilia.
About ADYNOVATE in the United States
ADYNOVATE, [Antihemophilic Factor (Recombinant), PEGylated], is a human antihemophilic factor indicated in adolescent and adult patients (12 years and older) with hemophilia A (congenital factor VIII deficiency) for:
- On-demand treatment and control of bleeding episodes
- Routine prophylaxis to reduce the frequency of bleeding episodes
ADYNOVATE is not indicated for the treatment of von Willebrand disease.
Detailed Important Risk Information for ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated]
CONTRAINDICATIONS
ADYNOVATE is contraindicated in patients who have had prior anaphylactic reaction to ADYNOVATE, to the parent molecule (ADVATE), mouse or hamster protein, or excipients of ADYNOVATE (e.g. Tris, mannitol, trehalose, glutathione, and/or polysorbate 80).
WARNINGS & PRECAUTIONS
Hypersensitivity Reactions
Hypersensitivity reactions are possible with ADYNOVATE. Allergic-type hypersensitivity reactions, including anaphylaxis, have been reported with other recombinant antihemophilic factor VIII products, including the parent molecule, ADVATE. Early signs of hypersensitivity reactions that can progress to anaphylaxis may include angioedema, chest tightness, dyspnea, wheezing, urticaria, and pruritus. Immediately discontinue administration and initiate appropriate treatment if hypersensitivity reactions occur.
Neutralizing Antibodies
Formation of neutralizing antibodies (inhibitors) to factor VIII can occur following administration of ADYNOVATE. Monitor patients regularly for the development of factor VIII inhibitors by appropriate clinical observations and laboratory tests. Perform an assay that measures factor VIII inhibitor concentration if the plasma factor VIII level fails to increase as expected, or if bleeding is not controlled with expected dose.
ADVERSE REACTIONS
Common adverse reactions (≥1% of subjects) reported in the clinical studies were headache and nausea.
For Full U.S. Prescribing Information, please visit
http://baxalta.com/assets/documents/ADYNOVATE_PI.pdf.
About ADVATE
ADVATE [Antihemophilic Factor (Recombinant)] is a recombinant antihemophilic factor indicated for use in children and adults with hemophilia A (congenital factor VIII deficiency) for:
- Control and prevention of bleeding episodes
- Perioperative management
- Routine prophylaxis to prevent or reduce the frequency of bleeding episodes
ADVATE is not indicated for the treatment of von Willebrand disease.
ADVATE has a demonstrated efficacy and safety profile for the treatment of hemophilia A. ADVATE is a full-length (derived from the complete FVIII gene) recombinant FVIII product that is processed without any blood-based additives. Because no blood-derived components are added at any stage of the manufacturing process, the potential risk of transmitting pathogens that may be carried in blood-based additives is virtually eliminated. There have been no confirmed reports of transmission of HIV, HBV or HCV with rFVIII treatments.
ADVATE is the world’s most prescribed FVIII treatment. It is currently approved in 64 countries worldwide, including the United States, Canada, 28 countries in the European Union, Algeria, Argentina, Australia, Brazil, Chile, China, Colombia, Ecuador, Hong Kong, Iceland, Iraq, Israel, Japan, Kuwait, Macau, Malaysia, Mexico, Morocco, New Zealand, Norway, Panama, Puerto Rico, Russia, Saudi Arabia, Serbia, Singapore, South Korea, Suriname, Switzerland, Taiwan, Tunisia, Turkey, Ukraine, Uruguay, and Venezuela.
Detailed Important Risk Information for ADVATE [Antihemophilic Factor (Recombinant)]
CONTRAINDICATIONS
ADVATE is contraindicated in patients who have life-threatening hypersensitivity reactions, including anaphylaxis, to mouse or hamster protein or other constituents of the product.
WARNINGS & PRECAUTIONS
Hypersensitivity Reactions
Allergic-type hypersensitivity reactions, including anaphylaxis, have been reported with ADVATE. Symptoms include dizziness, paresthesia, rash, flushing, facial swelling, urticaria, dyspnea, pruritus, and vomiting.
Discontinue ADVATE if hypersensitivity symptoms occur and administer appropriate emergency treatment.
Neutralizing Antibodies
Neutralizing antibodies (inhibitors) have been reported following administration of ADVATE predominantly in previously untreated patients (PUPs) and previously minimally treated patients (MTPs). Monitor all patients for the development of factor VIII inhibitors by appropriate clinical observation and laboratory testing. If expected plasma factor VIII activity levels are not attained, or if bleeding is not controlled with an expected dose, perform an assay that measures factor VIII inhibitor concentration.
ADVERSE REACTIONS
Serious adverse reactions seen with ADVATE are hypersensitivity reactions, including anaphylaxis, and the development of high-titer inhibitors necessitating alternative treatments to factor VIII.
The most common adverse reactions observed in clinical trials (frequency ≥5% of subjects) were pyrexia, headache, cough, nasopharyngitis, arthralgia, vomiting, upper respiratory tract infection, limb injury, nasal congestion, and diarrhea.
Please see full prescribing information for ADVATE at:
http://baxalta.com/assets/documents/ADVATE_PI.pdf.
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.
SOURCE: Baxalta
