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February 23, 2015
Baxter International reported results of a
phase III study of BAX 855 for hemophilia A.
The prospective, global, multicenter, openlabel,
two-arm phase III study evaluated BAX
855 among 137 previously treated hemophilia
A patients (PTP) who were 12 years or older.
Patients were assigned either to twice weekly
prophylaxis (40-50IU/kg, n=120) or on-demand
treatment (10-50IU/kg, n=17). In addition to
a reduced annualized bleed rate (ABR), BAX
855 also was effective in treating bleeding
episodes, 96% of which were controlled with
one or two infusions at a median dose of 29IU/
kg per infusion. Treatment was rated excellent
or good for nearly all episodes (96.2%). In the
prophylactic group (n=101), 40% of patients
experienced no bleeds. No patients developed
inhibitors to BAX 855 and no treatment-related
serious adverse events, including hypersensitivity,
were reported. Seven adverse reactions in six
patients, including headache, diarrhea, nausea
and flushing were reported. The study findings
supported Baxter’s December 2014 submission
for approval of BAX 855 to the FDA. Baxter’s
continuation study for patients who completed
the pivotal trial and the phase III study among
previously treated patients under the age of
12 with severe hemophilia A remain ongoing.
Upon completion of the pediatric study, Baxter
expects to file for marketing authorization with
the EMA in 2016.
September 1, 2014
Baxter International reported results
of a phase III clinical trial of BAX 855 for
treatment of treatment for hemophilia A.
The global, multi-center, open-label study
evaluated BAX 855 among 138 adolescent
and adult patients with previously-treated
hemophilia A. Patients received treatment
twice weekly (45IU/kg) or on-demand, and
were followed for six months. The study
demonstrated that BAX 855 met its primary
endpoint in the control and prevention of
bleeding, routine prophylaxis and perioperative
management for patients 12 years or
older. Patients in a twice-weekly prophylaxis
arm experienced a 95% reduction in median
ABR as compared to those in the on-demand
arm (1.9 v. 41.5, respectively). BAX 855 also
was effective in treating bleeding episodes,
96% of which were controlled with one or
two infusions. The half-life of BAX 855 was
1.4 to 1.5 times that of ADVATE, consistent
with the findings from the phase I study. No
patients developed inhibitors to BAX 855 and
no treatment-related serious adverse events,
including hypersensitivity, were reported.
The most common (three patients) productrelated
adverse event was headache. Baxter
expects to submit a BLA for BAX 855 to the
FDA before the end of 2014.
April 21, 2014
Biogen Idec and Swedish Orphan
Biovitrumreported results of a phase III trial
of ELOCTATE for the treatment of children
under 12 with severe hemophilia A. The global,
open-label, multi-center study involved
71 boys with severe hemophilia A (factor VIII
activity less than 1IU/dL, or 1%) with at least
50 prior exposure days to factor VIII therapies.
The study was conducted at 23 hemophilia
treatment centers in eight countries. Overall,
67 participants (94%) completed the study
(33 under six years old and 34 six-to-11 years
old). The average time participants spent in
the study was 25 weeks, and 61 participants
received ELOCTATE infusions on at least
50 separate days (exposure days) to assess
inhibitor development. All study participants
were to be initially treated with twice-weekly
prophylactic infusions of ELOCTATE [25IU/kg
day one, 50IU/kg day four]. Study investigators
could adjust the dose or interval based
on individual response. Approximately 90%
of study participants were on twice-weekly
dosing at the end of the study. The relative
increase in half-life in children with severe
hemophilia A was consistent with the 1.5-fold
increase in half-life seen in the A-LONG study
of adults and adolescents. Children treated
prophylactically with ELOCTATE had an overall
median ABR of two and a median ABR for
spontaneous bleeds of zero. Forty-six percent
of participants in the study experienced zero
bleeding episodes. Overall, 93% of bleeding
episodes were controlled by one to two infusions
of ELOCTATE. ELOCTATE was generally
well-tolerated. This is considered a milestone
that will enable regulatory submission in
August 5, 2013
rEVO Biologics issued results of a multi-center study of LR769, a novel recombinant form of human Factor VIIa (rhFVIIa), for the treatment of hemophilia A and B patients with inhibitors to Factor VIII or IX. Conducted in the U.S. and Europe, LR769 was administered in three dose levels— 25μg/kg, 75μg/kg and 225μg/kg, with each patient receiving two doses. LR769 is pharmacodynamically active after a single dose, and dose-related effects on core coagulation markers (aPTT, PT, and F1+2) were found. LR769 is well-tolerated in hemophilia A or B patients when administered at doses up to 225μg/kg and no immune reactions to LR769 were found in any patients after repeated dosing. There are plans for registrational trials later this year.
October 1, 2012
Biogen Idec and Swedish Orphan Biovitrum released results from a phase III trial of rFIXFc for the treatment of hemophilia B. This open-label, multi-center, multi-arm study, B-LONG, enrolled 123 male patients aged 12 years and older. Subjects were divided into four arms and received rFIXFc 50IU/kg weekly as prophylaxis, rFIXFc 100IU/kg every 10 days as an individualized interval prophylaxis, episodic treatment or perioperative management. Data showed that the overall median annualized bleeding rates (including spontaneous and traumatic bleeds) were 2.95 in the weekly prophylaxis arm; 1.38 in the individualized interval prophylaxis arm; and 17.69 in the episodic treatment arm. In the individualized interval prophylaxis arm, the median dosing interval during the last six months on study was 14 days. Overall, 90.4% of bleeding episodes were controlled by a single injection of rFIXFc. The drug was well tolerated. The most frequent adverse events were nasopharyngitis, influenza, arthralgia, upper respiratory infection, hypertension and headache. Based on these findings, Biogen Idec plans to submit a Biologics License Application to the FDA in the first half of 2013. Additional analyses of the B-LONG study are ongoing.
July 16, 2012
Inspiration Biopharmaceuticals reported interim results from a phase II/III trial of OBI-1 for the treatment of acquired hemophilia A. The open-label study enrolled seven patients experiencing serious bleeds. Subjects received 200U/kg of OBI-1 on first injection and were subsequently dosed based on target FVIII levels. All seven subjects experienced successful control of bleeds at 24 hours and subsequent resolution of their bleeds. Therapeutic FVIII activity levels were achieved and maintained with intermittent OBI-1 administration based on FVIII levels. Of the adverse events reported during the study thus far, five were serious, but these were not considered by investigators to be treatment-related. Inspiration Biopharmaceuticals plans to continue the phase II/III trial.
February 27, 2012
Inspiration Biopharmaceuticals reported results from a clinical trial evaluating IB1001 for hemophilia B. The trial enrolled 14 subjects ages 12 and older with hemophilia B who were scheduled for major surgical procedures. The subjects received either bolus or continuous infusion of IB1001 during surgery and for a minimum of three days post procedure. Hemostasis was evaluated at 12 hours and 24 hours post procedure. IB1001 resulted in effective control of bleeding. Estimated intra-operative blood loss was rated as expected or less than expected in all subjects. IB1001 was well-tolerated with few adverse events.
July 19, 2010
Biogen Idec and Swedish Orphan Biovitrum issued positive results from a phase I/II trial of their fully-recombinant factor IX Fc fusion protein, rFIXFc, for the treatment of hemophilia B. This open-label, multi-center, dose-escalation study, SYN-FIXFc-07-001, enrolled 14 previously-treated subjects with severe hemophilia B. The subjects received a single dose of rFIXFc given as an intravenous injection at doses ranging from 1 to 100 IU/kg. rFIXFc was well tolerated and demonstrated an approximately three-fold increase in half-life compared to historical data for existing therapies.
August 29, 2005
Baxter Healthcare reported positive results from a multi trials investigating Advate, a recombinant antihemophilic factor for the treatment of hemophilia A. The first results were from a preliminary assessment of a prospective international, multi-center study enrolling 53 pediatric subjects less than six years of age who were previously treated with factor VIII. Results demonstrated that no patients discontinued therapy as a result of an adverse event and no incidence of factor VIII antibodies were reported in this study. The second were of preliminary results of a multi- center, prospective, open-label, uncontrolled study designed to test the efficacy, safety and immunogenicity on 57 hemophilia A subjects undergoing surgery or other invasive procedures. An interim assessment of the first 44 procedures performed demonstrated an efficacy rating of excellent/good in 97.7% of surgeries. The company presented the data at the 20th Congress of the International Society on Thrombosis and Haemostasis held in Sydney, Australia.