Definitive pharmacokinetic and comparative pain studies initiated for Makena subcutaneous auto-injector program
Estimated sNDA filing for Feraheme Phase 3 label expansion accelerated to mid-2017 based on rapid pace of trial enrollment
WALTHAM, Mass., Oct. 12, 2016 (GLOBE NEWSWIRE) -- AMAG Pharmaceuticals, Inc. (NASDAQ:AMAG) today announced that it has achieved two key milestones in its next generation development programs. The definitive pharmacokinetic study and the comparative pain study for the Makena® (hydroxyprogesterone caproate injection) subcutaneous auto-injector program (Makena SQ) have been initiated with the first patient dosed. The enrollment of patients in the Feraheme Phase 3 label expansion trial for the treatment of iron deficiency anemia (IDA) has surpassed approximately two-thirds of the total target enrollment of 2,000 patients, resulting in the acceleration of the estimated supplemental new drug application (sNDA) filing date to mid-2017.
“We are pleased to announce significant progress on these two next generation development programs,” said William Heiden, chief executive officer of AMAG. “The Makena subcutaneous auto-injector is designed for easier administration by healthcare providers and potentially less painful injections for patients, and we are focused on completing the development of this product and bringing it to market as quickly as possible. With an earlier sNDA filing for Feraheme, we hope to receive FDA approval for a broader label sooner than previously forecasted, which if approved, could significantly expand the number of patients who could benefit from Feraheme therapy in the future.”
Makena subcutaneous auto-injector development program
The definitive pharmacokinetic study for the Makena SQ program is a randomized open label parallel study designed to demonstrate comparable bioavailability between subcutaneous and intramuscular injections of Makena. The study is expected to enroll 120 healthy post-menopausal women with a 1:1 randomization.
The comparative pain study is also a randomized open label parallel study designed to compare the average injection pain of four weekly injections between the subcutaneous and intramuscular injections of Makena using a validated pain scale. The study is expected to enroll 60 healthy, post-menopausal women with a 1:1 randomization. The company intends to use the results from the pain study to establish clinical superiority of the SQ auto-injector over the intramuscular injection to support a submission to the FDA for orphan drug exclusivity for the Makena SQ auto-injector.
AMAG plans to file the sNDA for the Makena SQ auto-injector in the second quarter of 2017 and anticipates a decision on approval in the first quarter of 2018.
Phase 3 clinical trial evaluating the safety of Feraheme compared to Injectafer
This is the final trial that the company plans to conduct in preparation for filing the sNDA to broaden the use of Feraheme beyond the current chronic kidney disease (CKD) indication to include all adult IDA patients who have failed or cannot tolerate oral iron treatment or in whom oral iron was contraindicated. Patient enrollment in the clinical trial has surpassed two-thirds of the total target enrollment mark ahead of schedule. Based on the rapid pace of enrollment, AMAG now expects to file the sNDA in mid-2017, with a decision on approval expected in the first half of 2018. Approval of the broader IDA label would double the addressable patient population eligible for Feraheme.
The non-inferiority, randomized, double-blind, multicenter clinical trial is evaluating the incidence of moderate-to-severe hypersensitivity reactions (including anaphylaxis), and moderate-to-severe hypotension with Feraheme compared to Injectafer in adults with IDA. A total of approximately 2,000 patients will be randomized in a 1:1 ratio into one of two treatment groups—Feraheme IV infusion or Injectafer IV infusion. While the trial’s primary endpoint is safety, the trial will also assess efficacy.
“This clinical trial is progressing well and if the current pace of enrollment continues, we believe that the trial could be completed in early 2017,” said Dr. Julie Krop, chief medical officer and senior vice president of clinical development and regulatory affairs at AMAG. “We are grateful to the investigators participating in the trial whose enthusiasm and commitment have allowed us to enroll the trial much faster than anticipated, potentially allowing a broader population of patients suffering from IDA to benefit from Feraheme earlier than previously expected. We are also pleased that the Makena next generation subcutaneous auto-injector program is progressing well and we remain on track for our sNDA filing next year.”
About Makena® (hydroxyprogesterone caproate injection)
Makena® is a progestin indicated to reduce the risk of preterm birth in women pregnant with a single baby who have a history of singleton spontaneous preterm birth. The effectiveness of Makena is based on improvement in the proportion of women who delivered <37 weeks of gestation. There are no controlled trials demonstrating a direct clinical benefit, such as improvement in neonatal mortality and morbidity.
Limitation of use: While there are many risk factors for preterm birth, safety and efficacy of Makena has been demonstrated only in women with a prior spontaneous singleton preterm birth. It is not intended for use in women with multiple gestations or other risk factors for preterm birth.
Makena should not be used in women with any of the following conditions: blood clots or other blood clotting problems, breast cancer or other hormone-sensitive cancers, or history of these conditions; unusual vaginal bleeding not related to the current pregnancy, yellowing of the skin due to liver problems during pregnancy, liver problems, including liver tumors, or uncontrolled high blood pressure.
Before patients receive Makena, they should tell their healthcare provider if they have an allergy to hydroxyprogesterone caproate, castor oil, or any of the other ingredients in Makena; diabetes or prediabetes, epilepsy, migraine headaches, asthma, heart problems, kidney problems, depression, or high blood pressure.
In one clinical study, certain complications or events associated with pregnancy occurred more often in women who received Makena. These included miscarriage (pregnancy loss before 20 weeks of pregnancy), stillbirth (fetal death occurring during or after the 20th week of pregnancy), hospital admission for preterm labor, preeclampsia (high blood pressure and too much protein in the urine), gestational hypertension (high blood pressure caused by pregnancy), gestational diabetes, and oligohydramnios (low amniotic fluid levels).
Makena may cause serious side effects including blood clots, allergic reactions, depression, and yellowing of the skin and the whites of the eyes. The most common side effects of Makena include injection site reactions (pain, swelling, itching, bruising, or a hard bump), hives, itching, nausea, and diarrhea.
For additional product information, including the full prescribing information, please visit www.makena.com.
About Feraheme® (ferumoxytol) Injection
Feraheme received marketing approval from the FDA on June 30, 2009 for the treatment of IDA in adult CKD patients and was commercially launched by AMAG in the U.S. shortly thereafter. Ferumoxytol is protected in the U.S. by six issued patents covering the composition and dosage form of the product. Each issued patent is listed in the FDA's Orange Book, the last of which expires in June 2023.
Fatal and serious hypersensitivity reactions including anaphylaxis have occurred in patients receiving Feraheme. Initial symptoms may include hypotension, syncope, unresponsiveness, and cardiac/cardiorespiratory arrest. Feraheme is contraindicated in patients with a known hypersensitivity to Feraheme or any of its components, or a history of allergic reaction to any intravenous iron product.
For additional product information, please see the full prescribing information, including Boxed Warning, available at www.feraheme.com.
About Iron Deficiency Anemia
Approximately 4.5 million Americans suffer from IDA and many IDA patients fail treatment with oral iron due to intolerability or side effects.
In 2015, an estimated 776,000 patients were treated with IV iron outside the dialysis setting, approximately half of whom were estimated to have CKD, with the other half suffering from IDA caused by conditions other than CKD, including abnormal uterine bleeding, gastrointestinal disorders, inflammatory diseases and chemotherapy-induced anemia.
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, among others, expectations for the Makena subcutaneous auto-injector program, including design and size of the definitive pharmacokinetic study and the comparative pain study, AMAG’s intention to establish clinical superiority over the intramuscular injection by demonstrating a reduction in pain, the expected timing of an sNDA submission and related decision from the FDA; expectations for AMAG’s Phase 3 clinical trial for the broader indication for Feraheme, including its design and size, the expected timing of an sNDA submission and related decision from the FDA, the estimated timing of completion of the trial and the impact of approval of the broader IDA label on the addressable patient population eligible for Feraheme; and beliefs that newborn stem cells have the potential to play a valuable role in the development of regenerative medicine 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, among others, those risks identified in AMAG’s filings with the U.S. Securities and Exchange Commission (SEC), including its Annual Report on Form 10-K for the year ended December 31, 2015, its Quarterly Reports on Form 10-Q for the quarters ended March 31, 2016 and June 30, 2016 and subsequent filings with the SEC. Any such risks and uncertainties could materially and adversely affect AMAG’s results of operations, its profitability and its cash flows, which would, in turn, have a significant and adverse impact on AMAG’s stock price. AMAG cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made.
AMAG disclaims 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.
AMAG Pharmaceuticals® and Feraheme® are registered trademark of AMAG Pharmaceuticals, Inc. Makena® is a registered trademark of AMAG Pharmaceuticals IP, Ltd. Cord Blood Registry® and CBR® are registered trademarks of CBR Systems, Inc.
AMAG is a biopharmaceutical company focused on developing and delivering important therapeutics, conducting clinical research in areas of unmet need and creating education and support programs for the patients and families we serve. Our products support the health of patients in the areas of maternal health, anemia management and cancer supportive care. Through CBR®, we also help families to preserve newborn stem cells, which are used today in transplant medicine for certain cancers and blood, immune and metabolic disorders, and have the potential to play a valuable role in the ongoing development of regenerative medicine. For additional company information, please visit www.amagpharma.com.
Last updated on: 13/10/2016
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