Seeking Alpha
We cover over 5K calls/quarter
Profile| Send Message|
( followers)  

AMAG Pharmaceuticals, Inc. (NASDAQ:AMAG)

Positive Preliminary Results from IDA-303 Study Conference Call

March 15, 2013 8:00 am ET

Executives

Amy Sullivan – Vice President-Corporate Communications and Investor Relations

William K. Heiden – Chief Executive Officer

Lee F. Allen – Chief Medical Officer

Analysts

Anupam Rama – JPMorgan Chase & Co.

Joseph Schwartz – Leerink Swann & Company

Operator

Good morning. My name is Kenya, and I will be your conference operator today. At this time, I would like to welcome everyone to the AMAG Pharmaceuticals conference call. All lines have been placed on mute to prevent any background noise. After the speakers' remarks there will be a question-and-answer session. (Operator Instructions) Thank you. Ms. Sullivan, you may begin your conference.

Amy Sullivan

Thank you, Kenya. Good morning and welcome to those of you that have joined us for this morning’s conference call. We issued a press release earlier today that contains the preliminary top line data from the IDA-303 Extension Study that is evaluated ferumoxytol for the treatment of iron deficiency anemia in a broad range of patients.

We have focused the data release on key points to allow us to preserve our options to present the full data set at appropriate medical conferences and journals in the future. If you haven’t received a copy of the release, you can get it from the investor section of our website at www.amagpharma.com. Additionally, we filed a Form 8-K earlier today, which is also available on our website.

Before proceeding with this call, please be reminded that any statements we make during the course of this conference call that are other than historical facts are forward-looking statements made pursuant to the Safe Harbor provisions of the Private Securities Litigation Reform Act of 1995.

We want to emphasize that these forward-looking statements involve risks and uncertainties that could cause actual results to differ materially from those discussed in such forward-looking statements. Please refer to our recent filings with the SEC including our Form 10-K for 2012 for a full review of the risks and uncertainties associated with our business.

The agenda for our call today will be brief. Bill Heiden, our CEO will provide a review of 8-K from this morning and the Phase III registrational program for Feraheme, including our expected regulatory review timeline in both the U.S. and Europe. Lee Allen, our Chief Medical Officer, will review the preliminary data from the study report today and then Bill will close the prepared remarks and we will open the call for Q&A. Frank Thomas, our Chief Operating Officer is also with us today.

With that, I will now turn the call over to Bill.

William K. Heiden

Thank you, Amy and thanks to all of you for joining us this morning. Before we jump into the data, I wanted to update you on a few developments that were covered in the Form 8-K that was filed this morning. Three topics were covered in that filing. First, since we released the Q4 2012 financial results earlier this month without a conference call, we thought it would be helpful to furnish the slides that we usually issue in conjunction with our quarterly earnings to provide continuity of data for our investors.

We choose not to hold the call because the results were in line with those previously reported in early January and we presented these results at a number of recent investor conferences.

The quarterly financial slides were filed with the 8-K and are now available on our website. We are not going to be covering those this morning, as we are going to be focused on the clinical data. But if you have any questions as you review the slides, feel free to call. We look forward to reporting the Q1 2013 financial results in about six weeks and we will be holding our usual conference call to update investors at that time.

Second, in the filing today, we announced that Dr. Lee Allen, our Chief Medical Officer is leaving the company to pursue other interests. Lee has been with us since 2007 and has been instrumental in both the original NDA filing for Feraheme, and the FDA approval for the CKD indication, and more recently in the design and execution of our broad IDA clinical program. His contribution to the supplemental new drug application for the broad IDA indication, which was submitted to the FDA in December 2012, helped create what we all believe is the straightforward case for approval in this broader patient population. Lead trials in an environment with lots of development activity and with AMAG’s current focus on commercialization and in-licensing of marketed products, there was a mutual agreement to part ways now with the conclusion of the ferumoxytol Phase III IDA clinical program and the sNDA having been filed and accepted by the FDA.

I want to take this opportunity to thank Lee for his service and also let you know that Lee has agreed to serve as a consultant on an as-needed basis, during the sNDA review and approval process. Lee is with us today to provide an overview of the IDA-303 data and answer any questions that you may have about the data. I’m confident that with the development and regulatory team at AMAG and these continuing help if and when needed. we have all the resources we need to support the regulatory review and approval of our ferumoxytol label expansion.

The third topic covered in the 8-K is the press release related to the data from IDA-303. as Amy mentioned, the release is available on our website.

Before I turn the call over to Lee, I want to remind every one of the IDA program timelines. We completed the two large Phase III studies, IDA-301 and 302 in 2012 and submitted our sNDA to the FDA in December of 2012. the filing was accepted earlier this month and a PDUFA date has been set for late October 2013. While we did include an interim analysis of the IDA-303 clinical data we discussed today in our sNDA filing, the trial was done primarily for European registration.

Our partner, Takeda is preparing the dossier for European submission as we speak. We expect the IDA filing in Europe to be submitted this year with a subsequent approval sometime in 2014. This European filing is important to us and our partner, Takeda, as we look to expand the label and provide Feraheme to a group of patients who have failed an oral iron therapy.

Financially, the approval in Europe for the broad IDA label would entitle us to significant milestone payment from our partner as well as continuing double-digit tier royalties on net product sales. Across all of the now completed Phase III IDA ferumoxytol trials, I have to say that we are very pleased with the consistency of both the safety and efficacy data observed across the IDA program in studies, IDA-301, IDA-302 and now IDA-303.

With that brief background, I’ll now turn the call over to Lee to review the data.

Lee F. Allen

Thank you, Bill. And thank you to those of you have joined us this morning for the call. 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 recurrent iron deficiency anemia regardless of the underlying cause. As with each study in the IDA program, these patients had a history of iron deficiency anemia and history of unsatisfactory oral iron therapy over IDA patients in oral iron could not be used.

Patients were eligible to enroll in IDA-303, the extension study after they had completed IDA-301, which is you’ll recall was a double-blind placebo-controlled study that randomized subjects to either one gram intravenous course of ferumoxytol or to placebo. The results of that study were previously reported by AMAG and presented at the American Society of Hematology Annual Meeting in December of last year.

634 patients from the IDA-301 study were enrolled in the IDA-303 study and qualified for ongoing observation and has appropriate treatment with ferumoxytol. Of the patients enrolled in IDA-303, 471 had previously received one treatment course with ferumoxytol in the IDA-301 study, which was a standard one gram course of therapy consisting of two doses of ferumoxytol 500 milligrams given two to eight days apart. The remainder of the patients in IDA-303 had received placebo in IDA-301.

For patients enrolled in the extension study, hemoglobin and transferrin saturations were followed monthly over the six month course of the study. Patients were eligible to receive ferumoxytol treatment in IDA-303 if their hemoglobin levels fell below 11 g/dL and their transferrin saturation levels were less than 20%.

During the course of the IDA-303, 337 patients were treated with ferumoxytol. A 151 of these patients had previously received placebo in IDA-301, and therefore they received their first course of ferumoxytol in the IDA-303 study. 58 of these patients then also went to receive the second course of ferumoxytol in IDA-303.

A total of 244 patients received a second course of ferumoxytol, of whom 186 had received their first treatment course in IDA-301. The remainder of patients enrolled in IDA-303 did not receive treatment, suggesting the durability of the response to the first course of ferumoxytol that they had received 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 and clinically important increase in the mean hemoglobin from baseline to week five of 2.6 g/dL. This change was consistent with the 2.7 g/dL increase in hemoglobin we previously reported for ferumoxytol-treated patients in both the IDA-301 and IDA-302 studies.

A secondary endpoint for this study, assess the efficacy of repeat dosing and with 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 course of ferumoxytol, as I mentioned earlier totaled 244 and they were a total of 69 patients who received the third course treatment with ferumoxytol in IDA-303. With repeat dosing, ferumoxytol also demonstrated a statistically significant and clinically meaningful increase in the mean hemoglobin from baseline to week five, with a mean hemoglobin increase of 1.5 g/dL following treatment course two and 1.1 g/dL following treatment course three.

Importantly, the mean hemoglobin levels achieved by patients at week five following each of the three treatment courses with ferumoxytol were all comparable. Following the treatment course one, the mean week five hemoglobin level was a 11.7 g/dL, and it was a 11.9 g/dL, after course two and a 11.4 g/dL after course three, suggesting a consistent clinical effect of ferumoxytol treatment. The magnitude of the increases in hemoglobin following a repeat dosing in course two and course three was not as large as in treatment course one.

We believe that this is likely due to the higher baseline hemoglobin in course two and course three compared to the first course of therapy. The baseline hemoglobin for courses two and three were over 10 g/dL compared to the 8.9 g/dL baseline per patients receiving treatment course one. Only a small number of subjects, 18 received four or more courses of ferumoxytol over the six month duration of this study, limiting the analyzes of these data.

No new safety signals were observed with repeat dosing ferumoxytol in IDA-303. The types of reported adverse events were consistent with those that we’re seeing both in the previous IDA and CKD Phase III studies, and also those contained in the approved U.S. package insert for Feraheme. Importantly there were no hypersensitivity reactions or hypotension reported in patients in the IDA-303 study. The rate of adverse events reported with the first course of ferumoxytol treatment in IDA-303 was approximately 48%, which is comparable to the 49% adverse event rates reported in IDA-301.

A similar rate of adverse events approximately 38% was reported in ferumoxytol treated patients receiving treatment course two and treatment course three. Serious adverse events were reported in approximately 5% of ferumoxytol treated patients in each of the three courses in this study. And importantly none of the series adverse events were considered to be related to treatment by the study investigators.

The IDA studies defined a category of adverse events called protocol-defined adverse events of special interest or AESI, which included mild to severe hypotension or hypersensitivity reactions or their associated symptoms. One adverse event of special interest was reported in the IDA-303 study, which was dyspnea or shortness of breath in one patient who receive the second course of ferumoxytol, and it was assessed as not related to treatment by the study investigators.

Cardiovascular adverse events were reported in approximately 1% of ferumoxytol treated patietns, and each of this retreatment courses, similar to the rates in IDA-301 and IDA-302. All of these events were considered not related to ferumoxytol. No deaths were reported in the IDA-303 study. These data from the IDA-303 study support data previously reported in the IDA Phase III program, and demonstrates the consistent safety and efficacy profile of ferumoxytol in the broader IDA patient population. In particular these data reinforce the positive effect of the first course of ferumoxytol treatment, and further demonstrate the safety and efficacy of repeat dosing, in the treatment of iron deficiency anemia in patients who cannot tolerate or otherwise take oral iron.

That concludes the data portion of the call. But before I turn it back to Bill, I would like to comment on the difficult decision that I have made to leave my current role at AMAG. I’ve been with the Company for over five years now, and as I look back, it’s been amazing and very productive experience, helping to successfully develop ferumoxytol, first for adult iron deficiency anemia patients with chronic kidney disease and more recently completing the IDA Label Expansion Program and submitting the sNDA was a true highlight in my career. I remain committed to the project and confident that it will be successful and I plan to support the team through the approval process as a consultant for AMAG.

Iron deficiency anemia is an extremely prevalent condition and having lived the life of practicing oncologist, I know first hand the important adverse effects of iron deficiency anemia on patient’s quality of life. Ferumoxytol is a great product with an established safety and efficacy profile, and I believe that when it gets approved for the broader indication, it will be an important treatment options for patients with iron deficiency anemia who cannot tolerate or take oral iron.

I also want to take this opportunity to thank the full medical development team at AMAG. It’s been a great pleasure to work with such a talented and committed group of individuals.

Now, I’d like to turn the call back to Bill for some quick closing remarks and thank you for your attention.

William K. Heiden

Thank you very much, Lee. Regarding the study results, you can imagine we are really pleased with the outcome reported today. We believe that Feraheme could provide an important treatment option for patients with iron deficiency anemia who cannot take or tolerate oral iron. Currently, in the U.S., the only IV irons with a broad IDA label are the older iron dextran products.

The market in the U.S. for IV iron outside the dialysis setting is approximately 800,000 grams of which we estimate approximately half is currently used for the treatment of CKD patients, so that current label. And the other half is for the treatment of IDA for other causes, the focus of this clinical program. We believe that nearly all of this non-CKD IV iron is being administered to patients in the same clinical care settings, where CKD patients get their iron today, primarily hospitals and hematology and oncology infusions centers.

So should we get FDA approval for the broader IDA label in the U.S., we believe the market opportunity will double and we’ll be able to promote and to contract with the same customers that we target today with our current sales force. Therefore, no significant expansion of our commercial footprint will be required to call on these physicians who currently treat patients with IDA.

I mentioned the significant existing IV iron market opportunity for the broad IDA label and given Feraheme’s profile, we believe that through physician education and increased patient awareness, we could expand that market even further. There are approximately 4 million Americans who have been diagnosed with IDA today, and for many of them oral iron doesn’t work and the approved IV iron options require more doses and have boxed safety warnings.

We believe if approved that an IV iron with the efficacy, dosing and safety profile of Feraheme in which a complete one gram dose of iron can be delivered in two visits, three to eight days apart and has the potential to improve the fatigue, vitality and energy that more physicians and patients would consider IV iron and in particular Feraheme.

Small share gains in treating IDA patients who have not been successful with oral iron would represent a significant future opportunity for Feraheme. As I mentioned earlier outside the U.S., our partner Takeda Pharmaceuticals will be responsible for filing the regulatory application with the EU filing expected this year and approval for the broad IDA indication next year, which would trigger a significant milestone payment from Takeda.

And with that, I’ll close our prepared remarks and we’ll open the call for Q&A. Operator?

Question-and-Answer Session

Operator

(Operator Instructions) We have a question from the line of Geoff Meacham from JPMorgan.

Anupam Rama – JPMorgan Chase & Co.

Hey, guys, this is Anupam Rama in for Geoff Meacham. I just wanted to ask a quick question. In the press release you noted that, there was only a small number of patients receiving four or more courses of ferumoxytol, so it’s kind of – you couldn't do a formal analysis. But I was wondering if you could, may be talk about the general trends you were seeing in that small population? Thanks.

William K. Heiden

Yeah, again, it’s hard to make any real definitive conclusions from the data, but there certainly are increases in hemoglobin, and no real safety signals that are coming out of that patient population, just difficult to make more conclusions than that from the data sets are small.

Anupam Rama – JPMorgan Chase & Co.

Okay. And you talked about the regulatory strategy in Europe. Just wondering if you're going to submit these data to the FDA as well?

William K. Heiden

So, again, we did actually submit a subset of this data and interim analysis was performed and was included in the sNDA filing. It’s likely that we will update the FDA with these data as part of the safety supplement that the FDA requires at day 120.

Anupam Rama – JPMorgan Chase & Co.

Got it. Thanks so much.

Operator

We have a question from the line of Joseph Schwartz with Leerink.

Joseph Schwartz – Leerink Swann & Company

Great. Thank you very much and congratulations on the data. I was wondering if you can tell from this experience if you can provide us with any estimate of how much iron you expect patients with different underlying causes of IDA to receive in the marketplace? How should we think about maintenance, since I don't think there is a maintenance protocol, as much as a repletion protocol currently in the market. Do you have any thoughts on how this might change any of that?

Lee F. Allen

Yeah, we haven’t broken down the usage in terms of treatment courses by the different subgroups at this point in time. But just in general, I mean, obviously it’s a very variable disease state. So, women with abnormal uterine bleeding, obviously, when they are actively bleeding there require additional repletion courses because they are loosing actively blood and iron with that blood. And the treatment conditions, if they have a deficiency in the loss, the mechanism for their loss of the iron has resolved, then once they get repleted, they wouldn’t require long-term chronic therapy. But I think again, the answer to your question is, it’s very variable really depends upon the underlying condition and the activity of the disease state.

William K. Heiden

Joe, this is Bill. So that’s the medical explanation. If we just look at the market data across IV iron, it looks like an average patient receives about 1.6 grams of iron on an annual basis. And so, I think that’s probably a pretty safe assumption going forward because that market data includes IDA patients and so our assumption is that it would be the same for Feraheme for this broader label.

Joseph Schwartz – Leerink Swann & Company

That’s very helpful. Thanks. And if I could just ask one follow-up. Did you notice any trends among the patients who had an AE in terms of things like ferritin levels, pre and post therapy, underlying cause of their IDA or any comorbidities?

William K. Heiden

We’re still doing those analysis but nothing has jumped out from the data set at this point in time.

Joseph Schwartz – Leerink Swann & Company

Okay, great. Thanks again.

Operator

(Operator Instructions) At this time, I’d like to turn the call back over to Bill Heiden.

William K. Heiden

Thank you very much Kenya. So the results announced today, really conclude the formal Phase III IDA development program for the US and EU. We’ve already filed the sNDA in the United States and these results presented today will permit our partner in the EU markets, Takeda Pharmaceuticals to finalize their regulatory submission for the IDA label in Europe. We are pleased to have delivered data from these three trials which demonstrated treatment with ferumoxytol resulted in a consistent rises in hemoglobin, significant improvement in patient reported outcomes which correlated well, with rises in hemoglobin to reflect as a well established safety profile as reported in all of our Phase III trials and now more than 600,000 patient exposures in market.

I want to thank you all for joining us on this call today. And we look forward to speaking with you again in just a few weeks when we report our first quarter 2013 financial results. Have a great Friday and a nice weekend.

Operator

Thank you. This concludes today’s conference. You may now disconnect.

Copyright policy: All transcripts on this site are the copyright of Seeking Alpha. However, we view them as an important resource for bloggers and journalists, and are excited to contribute to the democratization of financial information on the Internet. (Until now investors have had to pay thousands of dollars in subscription fees for transcripts.) So our reproduction policy is as follows: You may quote up to 400 words of any transcript on the condition that you attribute the transcript to Seeking Alpha and either link to the original transcript or to www.SeekingAlpha.com. All other use is prohibited.

THE INFORMATION CONTAINED HERE IS A TEXTUAL REPRESENTATION OF THE APPLICABLE COMPANY'S CONFERENCE CALL, CONFERENCE PRESENTATION OR OTHER AUDIO PRESENTATION, AND WHILE EFFORTS ARE MADE TO PROVIDE AN ACCURATE TRANSCRIPTION, THERE MAY BE MATERIAL ERRORS, OMISSIONS, OR INACCURACIES IN THE REPORTING OF THE SUBSTANCE OF THE AUDIO PRESENTATIONS. IN NO WAY DOES SEEKING ALPHA ASSUME ANY RESPONSIBILITY FOR ANY INVESTMENT OR OTHER DECISIONS MADE BASED UPON THE INFORMATION PROVIDED ON THIS WEB SITE OR IN ANY TRANSCRIPT. USERS ARE ADVISED TO REVIEW THE APPLICABLE COMPANY'S AUDIO PRESENTATION ITSELF AND THE APPLICABLE COMPANY'S SEC FILINGS BEFORE MAKING ANY INVESTMENT OR OTHER DECISIONS.

If you have any additional questions about our online transcripts, please contact us at: transcripts@seekingalpha.com. Thank you!

Source: AMAG Pharmaceuticals' CEO Presents at Positive Preliminary Results from IDA-303 Study Conference (Transcript)
This Transcript
All Transcripts