Clinical Trials

Date: 2017-07-06

Type of information: Submission of a clinical trial application

phase: 2

Announcement: submission of a clinical trial application

Company: Shire (UK - USA)

Product: SHP654 (BAX 888)

Action mechanism:

• gene therapy. SHP654 (BAX 888) is an investigational factor VIII (FVIII) gene therapy intended to treat hemophilia A using a recombinant adeno-associated virus serotype 8 (rAAV8) vector to deliver a codon-optimized, B-domain deleted FVIII (BDD-FVIII). This gene therapy selectively targets the liver. It involves the delivery of a functional copy of FVIII to the body’s liver to enable its own production of FVIII, rather than relying on a factor-based treatment. The FVIII expression is further controlled in patients by incorporating the liver-specific transthyretin (TTR) promoter/enhancer.
• This gene therapy includes technology acquired from Chatham Therapeutics,  a spin-out of Asklepios Biopharmaceutical.

Disease: hemophilia A

Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases

Country: USA

Trial details:

Latest news:

• • On July 6, 2017, Shire announced the submission of an investigational new drug (IND) application to the FDA for SHP654, also designated as BAX 888, an investigational factor VIII (FVIII) gene therapy for the treatment of hemophilia A. SHP654 aims to protect hemophilia A patients against bleeds through the delivery of a long-term, constant level of factor expression.
• The IND filing for SHP654 was based on the results of pre-clinical and phase 1 studies demonstrating the potential utility of this candidate, including data to be presented at the International Society on Thrombosis and Haemostasis (ISTH) 26th Biennial Congress in Berlin, Germany, from July 8 – 13, 2017 (“Development of SHP654 a highly efficient AAV8-based BDD-FVIII gene therapy vector for treatment of hemophilia A.
• and “Dose response and long-term expression of a human FVIII gene therapy construct in hemophilia A mice.”
• http://onlinelibrary.wiley.com/doi/10.1111/rth2.2017.1.issue-S1/issuetoc)

 

Is general: Yes