AMAG Pharmaceuticals, Inc. (NASDAQ: AMAG) today reported preliminary
results from the second phase III study from its global registrational
program for Feraheme® (ferumoxytol) in patients with iron
deficiency anemia (IDA) regardless of the underlying cause. The study
being reported today, IDA-301, compared Feraheme treatment to
placebo and enrolled 808 patients at 136 sites in the US, Canada, India,
Latvia, Hungary, and Poland. The patients enrolled in the study had a
history of unsatisfactory response to, or could otherwise not tolerate,
oral iron therapy. Patients in this study had IDA associated with
various conditions including abnormal uterine bleeding, cancer,
gastrointestinal disorders or other causes. Feraheme demonstrated
superiority on all primary efficacy endpoints evaluated in this study.
The efficacy and safety of Feraheme in this study were comparable
to that reported earlier this year in the IDA-302 study, the phase III
IDA study comparing Feraheme to iron sucrose.
The IDA-301 study was a double-blind, placebo-controlled trial that
randomized patients 3:1 to receive a one gram IV course of Feraheme
or placebo, and it was powered to demonstrate superiority on efficacy.
In this study, 608 patients were treated with Feraheme and 200
received placebo, with the demographics and all baseline parameters well
balanced between the two treatment groups. The primary efficacy endpoint
of the study for US regulators is the proportion of subjects who
achieved a ≥ 2.0 g/dL increase in hemoglobin at any time from baseline
to week five; the primary efficacy endpoint of the study for EU
regulators is the mean change in hemoglobin from baseline to week five.
Patients enrolled in this study were eligible to enter an ongoing
extension study, IDA-303, to evaluate repeat dosing with Feraheme; the
extension study is fully enrolled with 634 patients.
In the IDA-301 study, Feraheme demonstrated robust efficacy,
achieving superiority on both primary efficacy endpoints. Patients
treated with Feraheme achieved a statistically significant mean
increase in hemoglobin at week five of 2.7 g/dL, compared to a mean
increase of only 0.1 g/dL in patients who received placebo; importantly,
these data are consistent with the 2.7 g/dL increase in hemoglobin
reported in the IDA-302 study. In addition, a ≥ 2.0 g/dL increase in
hemoglobin at any time from baseline to week five was achieved in a
statistically significantly greater proportion, 81.1%, of patients
treated with Feraheme in this study, compared with only 5.5% of
patients who received placebo; these data are also consistent with the
data from IDA-302, in which 84.0% of Feraheme-treated patients
achieved a ≥ 2.0 g/dL increase in hemoglobin. Further, a statistically
significant improvement in fatigue, as assessed by patient reported
outcome measures, was demonstrated at week five in Feraheme-treated
No new safety signals were observed with Feraheme and the types
of reported adverse events (AEs) were consistent with those seen in both
the previously reported IDA phase III study and the CKD phase III
studies, and those contained in the approved U.S. package insert for Feraheme.
Overall, AEs were reported in both study arms with AEs reported in 49.2%
of Feraheme-treated patients, compared to 43.0% of patients who