Baxalta Commences Phase 1 Clinical Trial of BAX 826, the Company’s Second Extended Half-Life Factor VIII Treatment for Hemophilia A

03/04/2016
  • First patient dosed in Phase 1 clinical trial to evaluate safety and pharmacokinetics of investigational treatment BAX 826 for hemophilia A
  • Using proprietary polysialic acid (PSA) technology, BAX 826 is under investigation as Baxalta’s second extended half-life treatment based on ADVATE [Antihemophilic Factor (Recombinant)], the world's most prescribed FVIII treatment
  • Baxalta continues to advance its pipeline of innovative treatments for hemophilia A, including BAX 826 and gene therapy

BANNOCKBURN, Ill.--(BUSINESS WIRE)-- Baxalta Incorporated (NYSE:BXLT), a global biopharmaceutical leader dedicated to delivering transformative therapies to patients with orphan diseases and underserved conditions, announced today that it has dosed the first patient in its Phase 1, first-in-human clinical trial of BAX 826, a recombinant Factor VIII (rFVIII) treatment for hemophilia A that uses proprietary polysialic acid (PSA) technology to extend its circulating half-life. BAX 826 is under investigation as Baxalta’s second extended half-life treatment based on ADVATE [Antihemophilic Factor (Recombinant)], the world's most prescribed FVIII treatment.

"This is an important step in evolving extended half-life treatments as we aim to improve bleed protection for hemophilia A patients while potentially offering once-weekly dosing,” said John Orloff, M.D., head of Research & Development and chief scientific officer, Baxalta. “Baxalta is dedicated to advancing innovative research on the principle of direct factor replacement, a proven treatment model, to support as many patients as possible.”

The open-label, dose-finding study evaluating the safety and pharmacokinetics of BAX 826 looks to enroll 30 patients in three dosing cohorts. Baxalta expects to complete enrollment by the end of 2016.

BAX 826 is a next-generation rFVIII treatment based on the full length ADVATE. 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 FVIII. Xenetic’s PolyXen™ technology utilizes the biopolymer PSA in order to extend the circulating half-life and potentially improve the pharmacokinetic profile of therapeutic proteins, peptides, and small molecules. Preclinical studies indicated BAX 826 offered an extended circulating half-life compared to standard rFVIII.

Baxalta leads the industry with the broadest portfolio of hemophilia treatments, and meets individual therapy choices with a range of options. With BAX 826, Baxalta strives to augment its growing portfolio of direct factor replacement treatments for hemophilia, including ADVATE and ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated], which was approved in the United States for on demand and prophylaxis treatment in adolescent and adult patients (12 years and older) with hemophilia A and is under regulatory review in Japan, Canada and Switzerland. The company also continues to drive hemophilia innovation with the development of new advances like gene therapy.

About ADVATE

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 67 countries worldwide, including the United States, Canada, 28 countries in the European Union, Algeria, Argentina, Australia, Brazil, Brunei, Chile, China, Colombia, Ecuador, Hong Kong, Iceland, India, Iraq, Israel, Japan, Kuwait, Macau, Malaysia, Mexico, Morocco, New Zealand, Norway, Panama, Puerto Rico, Qatar, Russia, Saudi Arabia, Serbia, Singapore, South Korea, Suriname, Switzerland, Taiwan, Tunisia, Turkey, Ukraine, Uruguay, and Venezuela.

Indications:

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.

Detailed Important Risk Information

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.

For full prescribing information for ADVATE, visit: http://www.baxalta.com/assets/documents/ADVATE_PI.pdf.

About ADYNOVATE

ADYNOVATE is built on the full-length ADVATE molecule, a leading treatment for hemophilia A that been used by patients worldwide for more than 12 years. Through a collaboration with Nektar Therapeutics (NASDAQ: NKTR), ADYNOVATE leverages proprietary PEGylation technology designed to extend the amount of FVIII available for use in the body. The technology was selected because it maintains the integrity of the parent molecule (ADVATE) and reduces the time at which the body clears ADYNOVATE, resulting in an extended circulating half-life. This proprietary technology has been used for more than 15 years in a number of approved medicines that treat chronic or serious conditions.

Indications:

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

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 Prescribing Information, visit http://baxalta.com/assets/documents/ADYNOVATE_PI.pdf.

About Baxalta

Baxalta Incorporated (NYSE: BXLT) is a global biopharmaceutical leader developing, manufacturing and commercializing therapies for orphan diseases and underserved conditions in hematology, immunology and oncology. 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. 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, with its Global Innovation Center in Cambridge, Mass., Baxalta employs 17,000 employees worldwide.

Forward-Looking Statements

This release includes forward-looking statements concerning BAX 826, including expectations with regard to clinical trials, including enrollment plans, as well as its potential impact on patients. 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: 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 filings with the Securities and Exchange Commission, 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.

Source: Baxalta Incorporated

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