AMAG to Host a Conference Call at
preliminary data from the IDA-303 study. IDA-303 is a single arm,
open-label extension study that evaluated the safety and efficacy of
repeat dosing with ferumoxytol in patients with persistent or recurring
iron deficiency anemia (IDA) regardless of the underlying cause and a
history of unsatisfactory oral iron therapy or in whom oral iron could
not be used. Patients were eligible to enroll in the IDA-303 extension
study after they completed IDA-301, which was a double-blind,
placebo-controlled trial that randomized patients to receive either a
one gram intravenous course of ferumoxytol or placebo, the results of
which were previously reported by the company and presented at the
A total of 634 patients were enrolled and qualified for ongoing
observation/treatment in IDA-303. Of the 634 patients enrolled in
IDA-303, 471 had previously received one treatment course with
ferumoxytol in the IDA-301 study, and the remainder had received
placebo. Patients were eligible for ferumoxytol treatment in IDA-303 if
their hemoglobin levels fell below 11 g/dL and their transferrin
saturation (TSAT) levels were less than 20%. During the course of the
IDA-303 study, 337 patients were treated with ferumoxytol; 151 of these
patients had received placebo in IDA-301 and therefore received their
first course of ferumoxytol in the IDA-303 study. A total of 244
patients received a second course of ferumoxytol, of whom 186 patients
had received their first course in IDA-301. The remainder of patients
enrolled in IDA-303 did not receive treatment, suggesting the durability
of response to the first course of ferumoxytol administered in the
IDA-301 study.
The primary efficacy endpoint of the extension study was the mean change
in hemoglobin from baseline to week five following the first course of
ferumoxytol. The 151 patients who received their first course of therapy
in IDA-303 achieved a statistically significant mean increase in
hemoglobin from baseline to week five of 2.6 g/dL. This change was
consistent with the 2.7 g/dL increase in hemoglobin reported for
ferumoxytol-treated patients in both the IDA-301 and IDA-302 studies.
The first secondary endpoint was the mean change in hemoglobin from
baseline to week five for each subsequent course of ferumoxytol after
treatment course one. The group of patients receiving a second (n=244)
and third (n=69) course of treatment with ferumoxytol in IDA-303
included patients who had received ferumoxytol in IDA-301 and patients
who had received their first course of ferumoxytol in IDA-303. With
repeat dosing, ferumoxytol also demonstrated a statistically significant
increase in mean hemoglobin from baseline to week five, with a mean
hemoglobin increase of 1.5 g/dL following treatment course two and a 1.1
g/dL increase following treatment course three. The mean hemoglobin
level achieved by patients at week five following each of the first
three treatment courses with ferumoxytol were all comparable, with the
mean hemoglobin levels in course one of 11.7 g/dL, in course two of 11.9
g/dL, and in course three of 11.4 g/dL, suggesting the consistent effect
of ferumoxytol treatment with repeat dosing. Only a small number of
patients (n=18) received four or more courses of ferumoxytol over the 6
month duration of the study, limiting formal analyses of these data.
No new safety signals were observed with repeat dosing of ferumoxytol
and the types of reported adverse events (AEs) were consistent with
those seen in both the previously reported IDA and CKD phase III
studies, and those contained in the approved U.S. package insert for
Feraheme; no hypersensitivity reactions or hypotension were
reported in the IDA-303 study. Approximately 48 percent of patients who
received ferumoxytol treatment course one in this study experienced an
adverse event, consistent with the AE rate reported in the IDA-301
study; in each of treatment courses two and three, approximately 38
percent of patients experienced an AE. Approximately 5 percent of
ferumoxytol-treated patients in this study experienced a serious adverse
event in each of the three treatment courses, none of which were deemed
related to treatment by study investigators.
Protocol-defined adverse events of special interest (AESI) for the IDA
studies were defined to include mild to severe hypotension or
hypersensitivity reactions or associated symptoms. There was one AESI,
shortness of breath (or dyspnea), which was reported as not related to
treatment in one patient who received a second course of ferumoxytol.
Cardiovascular AEs, deemed not related to treatment, were reported in
approximately 1 percent of ferumoxytol-treated patients in each of the
three treatment courses. No deaths were reported in this study.
“We are pleased to see the consistency of both the safety and efficacy
data observed across the full IDA phase III program in studies IDA-301,
IDA-302 and now IDA-303,” said
executive officer of AMAG. “The final data from this study will be
included in the registration package for ferumoxytol in
pursuit of the broader IDA indication, which we expect will be filed
this year.”
In
(sNDA) to the
Feraheme® (ferumoxytol) Injection for Intravenous (IV) requesting
approval to expand the indication for ferumoxytol beyond the current
approved indication for the treatment of IDA in adult patients with
chronic kidney disease to all adult patients with IDA who have failed or
could not tolerate oral iron treatment. This sNDA is currently under
review with the
guidelines, the
completion of their review.
Conference Call Information
AMAG will host a conference call and webcast at
access the conference call via telephone, please dial (877) 412-6083
from
telephone replay will be available from approximately
the conference call, dial (855) 859-2056 from
537-3406 for international access. The pass code for the live call and
the replay is 23976614.
The call will be webcast live and accessible through the Investors
section of the company’s website at www.amagpharma.com.
A replay of the webcast will be available from approximately
EDT
About Iron Deficiency Anemia
More than 4 million Americans have IDA; 1.6 million of whom are
estimated to have CKD, while the other 2.4 million suffer from anemia
due to other causes.1 For these patients with anemia due to
other causes, the underlying diseases or issues causing IDA include
abnormal uterine bleeding, gastrointestinal disorders, inflammatory
diseases and chemotherapy-induced anemia. Many IDA patients fail
treatment with oral iron due to intolerability or side effects.2
About AMAG
manufactures and markets Feraheme® in
driving organic growth of its lead product, AMAG intends to expand its
portfolio with additional commercial-stage specialty pharmaceuticals.
The company is seeking complementary products that leverage the
company’s commercial footprint and focus on hematology and oncology
centers and hospital infusion centers. For additional company
information, please visit www.amagpharma.com.
About Feraheme® (ferumoxytol)/Rienso
In
(IV) use is indicated for the treatment of iron deficiency anemia in
adult chronic kidney disease (CKD) patients. Feraheme received marketing
approval from the
was commercially launched by AMAG in the U.S. shortly thereafter.
Ferumoxytol is protected in the U.S. by three issued patents covering
the composition and dosage form of the product. Each issued patent is
listed in the FDA’s Orange Book. These patents are set to expire in
2020; a request for patent term extension has been filed, which, if
granted, may extend the patent term to 2023 for one of the patents.
Ferumoxytol received marketing approval in
where it is marketed by Takeda as Feraheme, and in the
as Rienso®.
Important Safety Information for Feraheme (ferumoxytol) Injection
Indication and contraindication
Feraheme (ferumoxytol) Injection for Intravenous (IV) use is indicated
for the treatment of iron deficiency anemia in adult patients with
chronic kidney disease. Feraheme is contraindicated in patients with
known hypersensitivity to Feraheme or any of its components.
Warnings and precautions
Serious hypersensitivity reactions, including anaphylactic-type
reactions, some of which have been life-threatening and fatal, have been
reported in patients receiving Feraheme. Observe patients for signs and
symptoms of hypersensitivity during and after Feraheme administration
for at least 30 minutes and until clinically stable following completion
of each administration. Only administer the drug when personnel and
therapies are immediately available for the treatment of anaphylaxis and
other hypersensitivity reactions. Anaphylactic-type
reactions, presenting with cardiac/cardiorespiratory arrest, clinically
significant hypotension, syncope, and unresponsiveness have been
reported in the post-marketing experience. In clinical studies,
serious hypersensitivity reactions were reported in 0.2%
(3/1,726) of subjects receiving Feraheme. Other adverse reactions
potentially associated with hypersensitivity (e.g.,
pruritus, rash, urticaria or wheezing) were reported in 3.7%
(63/1,726) of subjects.
Severe adverse reactions of clinically significant hypotension have
been reported in the post-marketing experience. In clinical studies,
hypotension was reported in 1.9% (33/1,726) of subjects, including three
patients with serious hypotensive reactions. Monitor for signs and
symptoms of hypotension following each Feraheme injection.
Excessive therapy with parenteral iron can lead to excess storage of
iron with the possibility of iatrogenic hemosiderosis. Patients should
be regularly monitored for hematologic response during parenteral iron
therapy, noting that lab assays may overestimate serum iron and
transferrin bound iron values in the 24 hours following administration
of Feraheme.
As a superparamagnetic iron oxide, Feraheme may
transiently affect magnetic resonance diagnostic imaging studies for up
to 3 months following the last Feraheme dose. Feraheme will
not affect X-ray, CT, PET, SPECT, ultrasound, or nuclear imaging.
Adverse reactions
In clinical trials, the most commonly occurring adverse reactions
in Feraheme treated patients versus oral iron treated
patients reported in ≥ 2% of chronic kidney disease patients were
diarrhea (4.0% vs. 8.2%), nausea (3.1% vs. 7.5%), dizziness (2.6% vs.
1.8%), hypotension (2.5% vs. 0.4%), constipation (2.1% vs. 5.7%) and
peripheral edema (2.0% vs. 3.2%).
In clinical trials, adverse reactions leading to treatment
discontinuation and occurring in 2 or more Feraheme treated
patients included hypotension, infusion site swelling, increased serum
ferritin level, chest pain, diarrhea, dizziness, ecchymosis, pruritus,
chronic renal failure, and urticaria.
Post-marketing safety experience
The following adverse reactions have been identified during
post-approval use of Feraheme. Because these reactions are reported
voluntarily from a population of uncertain size, it is not always
possible to reliably estimate their frequency or establish a causal
relationship to drug exposure.
The following serious adverse reactions have been reported from the
post-marketing spontaneous reports with Feraheme: life-threatening
anaphylactic-type reactions, cardiac/cardiorespiratory arrest,
clinically significant hypotension, syncope, unresponsiveness, loss of
consciousness, tachycardia/rhythm abnormalities, angioedema, ischemic
myocardial events, congestive heart failure, pulse absent, and cyanosis.
These adverse reactions have occurred up to 30 minutes after the
administration of Feraheme injection. Reactions have occurred following
the first dose or subsequent doses of Feraheme.
For additional product information, including full prescribing
information, please visit www.feraheme.com.
Forward-looking Statements
This press release contains forward-looking statements within the
meaning of the Private Securities Litigation Reform Act of 1995 and
other federal securities laws. Any statements contained herein which do
not describe historical facts, including but not limited to statements
regarding: the final data for the IDA-303 study and expectations,
including timing, regarding the EU registration package for ferumoxytol,
are forward-looking statements which involve risks and uncertainties
that could cause actual results to differ materially from those
discussed in such forward-looking statements.
Such risks and uncertainties include: (1) uncertainties regarding our
and Takeda’s ability to successfully compete in the intravenous iron
replacement market both in the US and outside the US, including the EU,
(2) uncertainties regarding our ability to successfully and timely
complete our clinical development programs and obtain regulatory
approval for Feraheme/Rienso in the broader IDA indication both in the
US and outside of the US, including the EU, (3) the possibility that
significant safety or drug interaction problems could arise with respect
to Feraheme/Rienso, (4) uncertainties regarding the manufacture
of Feraheme/Rienso, (5) uncertainties relating to our patents and
proprietary rights, both in the US and outside of the US, (6) the risk
of an Abbreviated New Drug Application (ANDA) filing following the FDA’s
recently published draft bioequivalence recommendation for ferumoxytol,
and (7) other risks identified in our Securities and Exchange
Commission filings, including our Annual Report on Form 10-K for the
year ended December 31, 2012 and subsequent filings with the
caution you not to place undue reliance on any forward-looking
statements, which speak only as of the date they are made.
We disclaim any obligation to publicly update or revise any such
statements to reflect any change in expectations or in events,
conditions or circumstances on which any such statements may be based,
or that may affect the likelihood that actual results will differ from
those set forth in the forward-looking statements.
Pharmaceuticals, Inc.
Rienso is a registered trademark of
Limited
1 U.S. Census; U.S. Renal Data System, USRDS 2010 Annual
Data Report: Atlas of Chronic Kidney Disease and End-Stage Renal Disease
in
Institute of Diabetes and Digestive
2010: 41-42; Fishbane, S. et al. Iron Indices in CKD in the NHANES
1998-2004. Clin J Am Soc Nephrol. 2009 January; 4(1): 57–61.
2 Barton, James et al. Intravenous iron dextran therapy in
patients with iron deficiency and normal renal function who failed to
respond to or did not tolerate oral iron supplementation. Am J
Medicine. 2000; 109: 27-32.
Source:
AMAG Pharmaceuticals, Inc.
Amy Sullivan, 617-498-3303
