Date: 2016-03-04
Type of
information: Treatment of the first patient
phase: 1
Announcement: treatment of the first patient
Company: Baxalta (USA - IL), now Shire (UK - USA)
Product: BAX 826 (rFVIII - recombinant factor VIII - SHP656)
Action
mechanism: protein/coagulation factor. 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, 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.
Disease: hemophilia A
Therapeutic
area: Rare diseases - Genetic diseases - Hematological diseases
Country: UK
Trial
details:
Latest
news:
- • On March 4, 2016, Baxalta announced 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 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.
- • On November 20, 2015, Baxalta 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. 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. 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.
Is
general: Yes
