GenStar Thera: Study Shows MAX-AD Raises
Clotting Factor
Dow Jones Newswires
NEW YORK -- GenStar Therapeutics Corp. (GNT) said the first patient
who took the company's hemophilia A treatment, Maximum Ad Factor VIII,
in a Phase I trial showed an increase in Factor VIII clotting factor levels to up
to 3% from less than 1%.
Further evaluation is required to determine if this effect will persist, the
company said in a press release Wednesday.
Hemophilia A is a hereditary bleeding disorder characterized by a deficiency
in the blood clotting protein Factor VIII. Factor VIII levels of 1% are
characteristic of a severe condition and levels greater than 1% are associated
with a more moderate condition, GenStar said.
The Phase I clinical trial is a multi-center U.S. single-dose, dose-escalating
study intended to assess the safety of this therapeutic approach. It will also
measure the expression of Factor VIII following administration of the therapy.
The first patient was treated with MAX-AD FVIII on June 13. Following the
treatment, transient liver chemistries and hematologic abnormalities were
observed from an analysis of the patient's blood. While some of these
laboratory abnormalities were severe, these laboratory values returned to
normal shortly thereafter, and were not considered serious, GenStar said.
The patient, who has a history of multiple spontaneous bleeds, suffered a
spontaneous joint bleed at the time of the laboratory abnormalities requiring
treatment with recombinant Factor VIII protein, but is now doing well, the
company said.
Consistent with the study design, the trial won't proceed until GenStar, the
clinical trial sites and regulatory agencies have reviewed the findings and
agreed on potential protocol modifications. The company said it will continue
to work closely with the appropriate regulatory agencies to develop and
adopt a modified trial protocol, at which time the trial may resume.
GenStar's MAX-AD Factor VIII gene therapy product for hemophilia A is a
gene delivery system derived from the adenovirus. In contrast to earlier gene
delivery approaches, GenStar's delivery system has been engineered to
remove all the viral genes providing a large DNA-delivery capacity for
therapeutic genes that are responsible for the production of Factor VIII.
-Ralph Tasgal; Dow Jones Newswires; 201-938-5400 |
