Clinical Trials

Date: 2016-05-25

Type of information: Presentation of results at a congress

phase: 1

Announcement: presentation of results at the 62nd annual Scientific and Standardization Committee (SSC) meeting of the International Society on Thrombosis and Haemostatis (ISTH) in Montpellier

Company: Baxalta (USA - IL), now Shire (UK - USA)

Product: BAX 930/SHP655

Action mechanism: enzyme. BAX 930 is a recombinant ADAMTS13 (ADintegrin-like And Metalloprotease with ThromboSpondin type 13 motifs). This metalloprotease limits platelet aggregation and microthrombi formation in the microcirculation by cleaving Von Willebrand Factor [VWF] between Tyrosine 1605-Methionine 1606 [Tyr1605-Met1606] to generate a series of small molecular weight multimers. A deficiency of ADAMTS13 either because of an inherited mutation within the ADAMTS13 gene or the development of an autoantibody leads to the potentially lethal syndrome of thrombotic thrombocytopenic purpura.

Disease: hereditary thrombotic thrombocytopenic purpura

Therapeutic area: Autoimmune diseases - Rare diseases

Country: Austria, Germany, Japan, Poland, Switzerland, UK, USA

Trial details: The purpose of this Phase 1, prospective, uncontrolled, open-label, multicenter, dose-escalation study is to evaluate the safety, including immunogenicity, and pharmacokinetics of BAX930 (rADAMTS13) in a total of 14 evaluable subjects diagnosed with severe hereditary thrombotic thrombocytopenic purpura (TTP) (plasma ADAMTS13 activity <6%) who are assigned to one of three dose cohorts.(NCT02216084)

Latest news:

• • On May 25, 2016, Baxalta reported positive results from a Phase 1 open-label, dose escalation study assessing the safety and pharmacokinetic 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 - page 40).
• 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.

Is general: Yes