looks good to me
GenStar Reports Clinical Results of the First Patient to Receive MAX-AD--TM-- FVIII Treatment for Hemophilia A
Findings Presented at XVIII Congress of the International Society on Thrombosis and Haemostasis
PARIS--(BW HealthWire)--July 11, 2001-- GenStar Therapeutics (AMEX: GNT - news) today reported early Phase I clinical trial data from the study of its hemophilia A treatment, MAXIMUM AD Factor VIII (MAX-AD(TM) FVIII) at the XVIII Congress of the International Society on Thrombosis and Haemostasis. The findings, based on the dosing of the first patient, demonstrated an increase in Factor VIII clotting factor levels from less than 1% up to 3%. Further evaluation is required to determine if this effect will persist. 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.
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 initial patient was treated with MAX-AD FVIII on June 13, 2001. 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. 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 doing well presently.
``We are pleased to observe Factor VIII expression in the first patient treated with MAX-AD FVIII. However, these results are preliminary and further clinical evaluation of the system is needed to demonstrate if the increase in FVIII levels will be sustained and well tolerated,'' said Robert E. Sobol, M.D., chief executive officer of GenStar, who presented the data.
Consistent with the study design, the trial will not proceed until GenStar, the clinical trial sites and regulatory agencies have reviewed the findings and agreed upon potential protocol modifications. The Company 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.
GenStar Therapeutics is a biopharmaceutical company developing innovative gene therapy products for the treatment of serious medical disorders. The Company's research and development efforts, utilizing advanced gene delivery technologies, are focused on hemophilia, cancer, and vaccines. The Company's MAX-AD FVIII program is being developed in collaboration with Baxter Healthcare and its prostate cancer product development program is supported, in part, by a grant from the National Cancer Institute |
