Baxalta Reports Positive Phase 1 Results for BAX 930, Investigational Recombinant ADAMTS13 to Treat Hereditary Thrombotic Thrombocytopenic Purpura (hTTP)

05/25/2016
  • Single recombinant ADAMTS13 infusions demonstrated a favorable safety profile in adult and adolescent patients with severe hTTP
  • hTTP is a rare blood clotting disease related to ADAMTS13 deficiency, and in the absence of immediate treatment, mortality rates can approach ninety percent
  • In comparison to plasma infusions, recombinant ADAMTS13 may offer faster reconstitution and shorter administration times, and may be suitable for treatment at home

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, today reported positive results from a Phase 1 open-label, dose escalation study assessing the safety and pharmacokinetic (PK) profile of BAX 930, an investigational recombinant ADAMTS13 for the treatment of patients with severe hereditary thrombotic thrombocytopenic purpura (hTTP). These findings on BAX 930 were presented during a poster session at the 62nd annual Scientific and Standardization Committee (SSC) meeting of the International Society on Thrombosis and Haemostatis (ISTH) in Montpellier, France (Poster #FIBO4 – abstract available here, page 40).

“As a recombinant ADAMTS13, BAX 930 may provide an alternative treatment option for patients with hTTP and these Phase 1 results showed potential advantages of BAX 930 compared to conventional plasma infusions,” said John Orloff, M.D., head of Research & Development and chief scientific officer, Baxalta. “The positive results from the Phase 1 study demonstrated a favorable PK and safety profile for BAX 930 and provide the basis for proceeding to Phase 3 as we advance this innovative therapy for patients.”

BAX 930 is designed to provide an alternative to the current replacement of ADAMTS13 using large volumes of fresh frozen plasma that contain variable amounts of ADAMTS13 and typically require two hours or more for the preparation and infusion. In contrast, BAX 930 can be quickly reconstituted, and may thus be suitable for treatment in the patient’s home.

The Phase 1 data included results from fifteen patients with severe hTTP who completed the multicenter study. Each patient received a single dose of BAX 930 in one of three dosing cohorts. PK data demonstrated a consistent half-life across the BAX 930 doses and a linear dose response. No serious adverse events were reported. In the highest dosing cohort, three subjects reported three possibly related adverse events, nausea, flatulence and decreased VWF activity; all of these reported adverse events resolved quickly without medication. Immunogenicity tests performed at screening, pre-dose and upon study completion, were negative in all subjects.

hTTP is a rare, life-threatening blood clotting disease in which individuals are congenitally deficient in ADAMTS13, an enzyme that cleaves the von Willebrand factor (VWF) protein involved in blood clotting. Baxalta has been granted Orphan Drug Designation for BAX 930 by the U.S. Food and Drug Administration and by the European Medicines Agency.

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 930, including expectations with regard to clinical trials 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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Geoffrey Mogilner, 1-224-940-2202

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Mary Kay Ladone, 1-224-940-3371

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