AMAG PHARMACEUTICALS. June 2013

Transcription

1 AMAG PHARMACEUTICALS June 2013

2 FORWARD LOOKING STATEMENTS This presentation 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: (i) our statement that AMAG is well positioned for growth in 2013 and beyond; (ii) the expanded label opportunity for Feraheme; (iii) expectations regarding IV iron referrals and patient and physician behaviors; (iv) the potential for approval and potential launch of the supplemental new drug application in the U.S. and the timing and potential regulatory submission in the EU for Feraheme/Rienso for the broad iron deficiency anemia indication; (v) statements regarding our GI launch planning; (vi) statements regarding the potential size and expansion of the U.S. IV iron market opportunity and patient population; (vii) our expected financial results for 2013, including revenues, operating expenses and cost of goods sold; (viii) our expected cash and investments balance for 2013; (ix) our plans to expand the reach of Feraheme to new indications and geographic territories; (x) our business development activities and plans to execute transactions; (xi) strategies driving Feraheme volume growth; (xii) the emergence of shifting practice patterns in the IV iron market; (xiii) our expectation to increase our market share of the IV iron market in 2013; and (xiv) statements regarding our expectation to deliver on financial guidance, are forwardlooking 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, and our dependence on third parties for, 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 the US, (6) the risk of an Abbreviated New Drug Application (ANDA) filing following the FDA s draft bioequivalence recommendation for ferumoxytol, and (7) other risks identified in our Securities and Exchange Commission (SEC) filings, including our Quarterly Report on Form 10-Q for the quarter ended March 31, 2013 and subsequent filings with the SEC. We 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 2

3 AMAG: LEVERAGING OUR ASSETS AND CORE COMPETENCIES TO BUILD A PROFITABLE, MULTI-PRODUCT SPECIALTY PHARMACEUTICAL COMPANY Product #3 Product #2 Feraheme US U.S. CKD Business Feraheme IDA label Expansion IV Iron Market Expansion Feraheme Geographic Expansion Strong Balance Sheet Experienced Management Team Commercial Infrastructure Focus on Hematology/Oncology, Hospital and Nephrology 3

4 U.S. IV IRON MARKET

5 CURRENT US NON-DIALYSIS IV IRON MARKET >$400 MILLION (FERAHEME DOLLARS)* IV Iron Grams 50% of market is for treatment of patients with CKD Primary sites of treatment are hematology / oncology clinics and hospitals ~70% of market reimbursed at ASP+ model Hosptital In-patient Hospital Out-patient Heme/Onc Nephrology * Sources: IMS Health IV iron market data; Market opportunity estimated by AMAG at >$500/gram (500,000 patients receiving 1.6 grams of iron per year). 5

6 FERAHEME PERFORMANCE IN THE U.S.

7 FERAHEME ONASOLID GROWTH TRAJECTORY Quarterly US Feraheme Net Sales +14% volume growth +5% price growth $16 vs. 1Q % volume growth -5% price decline vs $15 illions) (in mi $14 $13 $12 $11 Q Q Q Q Q *Excludes reduction of reserves for product returns and Medicaid in

8 IV IRON HISTORICAL DOSING PARADIGM Attribute Many Other IV Irons One-gram Dose Dosing 1 Schedule 5-10 x mg Delivery Slow push or minute infusion Regimen (1 g) 5-10 treatments within one month Safety Dextran products have boxed warnings 2 Observation Period 30 minutes post dosing 2 1. One gram of IV iron is the usual therapeutic course. 2. Labels of Venofer and Ferrlecit were harmonized by the FDA in the summer of 2011 to all include post-dose observation periods and similar language regarding hypersensitivity reactions in the warnings and precautions sections. Dexferum and Infed do not have observation periods, but have boxed warnings. 8

9 FERAHEME IS AS EASY AS GRAM, 2 DOSES, 3 DAYS APART Attribute Feraheme One-gram Dose Dosing 1 Schedule 2 x 510 mg doses Delivery Rapid IV Injection (under 1 minute) Regimen (1 g) 2 treatments 3 to 8 days apart Safety No-boxed warning Observation Period 30 minutes post dosing 1. One gram of IV iron is the usual therapeutic course and that which was studied in the Feraheme clinical trials. Feraheme Indication and contraindications Feraheme (ferumoxytol) Injection for Intravenous (IV) 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. Serious hypersensitivity reactions, including anaphylactic-type reactions, some of which have been life-threatening and fatal, have been reported in patients receiving Feraheme. Anaphylactic type reactions, presenting with cardiac/cardiorespiratory arrest, clinically significant hypotension, syncope, and unresponsiveness have been reported in the post-marketing experience. In clinical trials, the most commonly occurring adverse reactions for Feraheme-treated patients were diarrhea, nausea, dizziness, hypotension, constipation and peripheral edema. A full list of adverse events can be found in the full prescribing information for Feraheme. For full prescribing information, please visit 9

10 1Q13 RECORD FERAHEME NON-DIALYSIS PROVIDER DEMAND 30,000 1Q13 demand results 25,000 11% increase in Feraheme provider demand; 110 evolution index vs 1Q12 24% growth in hospital 20, Grams 15,000 14% share of non-dialysis IV iron market 26% of hem/onc segment; EI 104 9% of hospital segment; EI ,000 40% of nephrology segment 5, Q12 1Q13 Hem/Onc Hospital Nephrolgy Other Source: IMS Health Data. 10

11 STRATEGIES DRIVING DOUBLE-DIGIT VOLUME GROWTH Targeting accounts, with differentiated strategy, message and tactics Double-digit Volume Growth Solid Execution Specific Messages Smart Targeting Customer Segmentation Tracking progress against growth & volume goals across all segments 11

12 SIGNIFICANT OPPORTUNITY TO EXPAND IV IRON TREATED IDA PATIENTS IN THE U.S. IDA Patients in the U.S. IV Iron Treated (500,000) ~10% of IDA patients are treated t with IV iron today ~$400 million market opportunity in Feraheme dollars Oral Iron Treated 4,000,000 Many patients fail oral iron treatment Existing IV Iron Market CKD Expansion Opportunity IDA Expansion Opportunity Converting even a third of patients that t fail oral iron to IV iron would double the market opportunity Sources: U.S. Census; U.S. Renal Data System, USRDS 2010 Annual Data Report: Atlas of Chronic Kidney Disease and End-Stage Renal Disease in the United States, National Institutes of Health, National Institute of Diabetes and Digestive and Kidney Diseases, Bethesda, MD, 2010: 41-42; Fishbane, S. et al. Iron Indices in CKD in the NHANES Clin J Am Soc Nephrol January; 4(1): 57 61; AMAG estimates market opportunity at $500/gram. 12

13 BROAD IDA LABEL EXPANSION PLANS 2012 Global 1,400 patient phase III program completed snda submitted to FDA 2013 IDA submission i in EU Potential US U.S. snda approval 2014 Potential IDA approval in EU (milestone to AMAG) 13

14 IV IRON REFERRALS -TODAY Patients with Iron deficiency anemia IV Iron Administration Nephrology Non -dialysis CKD Hematologist /Oncologist OB/GYN Fail or intolerant IV iron Gastroenterology Abnormal Uterine Bleeding Oral Iron Hospital outpatient Infusion Center IBD, Celiac, bariatric surgery Hospital In-patient Cardiology CHF Rheumatology Crohn s, arthritis 14

15 IV IRON REFERRALS -TODAY TOMORROW Patients with Iron deficiency anemia IV Iron Administration Nephrology OB/GYN Non -dialysis CKD Fail or intolerant IV iron IV iron Hematologist /Oncologist Gastroenterology Abnormal Uterine Bleeding Oral Iron Hospital outpatient Infusion Center IBD, Celiac, bariatric surgery Hospital In-patient Cardiology Rheumatology CHF Crohn s, arthritis INCREASE THE RATE OF ORAL IRON FAILURE PATIENTSTHATARE REFERRED FOR IV IRON TREATMENT 15

16 GASTROENTEROLOGY A PRIME OPPORTUNITY UPON APPROVAL Chronic GI disorders lead to IDA Inflammatory bowel disease (>150,000 with IDA) Bariatric surgery patients (>150,000 with IDA) Patients cannot tolerate or absorb oral iron Relatively small specialty (13,000) easily targeted 150 hospital-based IBD centers of excellence 570 CMS-approved bariatric surgery centers Physicians are highly concerned about the impact of anemia on their patients Developing launch strategies and tactics Conducting market research and hosting advisory boards Meeting with key opinion leaders and patient advocacy groups Publishing & presenting clinical trials results of GI patients (DDW & ACG) Education: Physicians & patients 16

17 THE UNMET NEED THAT EXISTS WITH IDA Patient Symptoms Diagnosis Treatment/ progression The answer 42, breast cancer patient After undergoing treatment for breast cancer, experienced extreme fatigue. Iron deficiency anemia diagnosed as IDA progressed post-cancer treatment Prescribed oral iron three times a day; eatiron-rich rich foods. Had a very hard time tolerating the oral iron and often stopped taking. Anemia unresolved. Visit to a hematologist who put together an IV iron treatment plan tailored to correct her iron deficiency. I refused to continue to feel like the walking dead so I took matters into my own hands. Now that my iron deficiency has been corrected, I am a different person. I am more active with my children and am not dreading the next decade of my life I m embracing it. 17

18 BUILDING DISEASE STATE AWARENESS IRON MATTERS 18

19 INTERNATIONAL EXPANSION BRINGS NEAR-TERM GROWTH Takeda Takeda AMAG 3SBio Takeda Approved for CKD indication in Europe and Canada Launches began in 4Q12 and will continue through 2013 Deal terms $60 million upfront payment $33 million in milestone payments received in 2012 $187 million in milestones remaining Broad IDA approval in EU Sales milestones Royalties on sales: tiered, double-digit 19

20 FUTURE ANNUAL SALES OPPORTUNITY IDA Label GI Market Expansion International ti Expansion CKD 20

21 BUSINESS DEVELOPMENT TARGET CATEGORIES Bulls-Eye Hem/onc or hospital company or product $10-60 MM / yr. revenue potential IP runway Immediately accretive Opportunity for growth Strategic Opportunities aligned with Feraheme growth strategy (e.g. GI, Rheum) Financial Similarly sized (or smaller) specialty company Eliminate overlapping infrastructure and increase EBITDA 21

22 FINANCIAL OVERVIEW

23 2013 FINANCIAL OUTLOOK 2013 Guidance Reiterated Revenues (in millions) Total Revenues of $73 - $77 million $63 $67 million in U.S. Feraheme net sales Operating expenses of $78 $82 $90 $80 $70 $60 million (excluding COGS) $50 Feraheme Ex US COGS: 14%-18% of global Feraheme sales $40 $30 Milestone and Royalty Revenue End 2013 with $206 $211 million of cash and investments, not including the impact of business development transactions $20 $ Est. Expenses (in millions) $130 Feraheme US $110 $90 $70 SG&A $50 R&D $30 $ Est. 23

24 BUILDING OFF 1Q SUCCESS IN 2013 Achieve full year double-digit growth of Feraheme in CKD patient population Continue to realize increasing net revenue per gram of Feraheme Regulatory filings for broad IDA indication Formal acceptance of snda by FDA 1Q13 EU regulatory submission mid-2013 Expected approval & launch in US 4Q13 Pre-launch activities for potential broader IDA indication Execute one or more business development transaction(s) Deliver on financial guidance 24

25 AMAG PHARMACEUTICALS Well positioned for growth in 2013 and beyond

26 ABOUT FERAHEME (FERUMOXYTOL)/RIENSO In the United States, Feraheme (ferumoxytol) Injection for Intravenous (IV) use is indicated for the treatment of iron deficiency anemia in adult chronic kidney disease (CKD) patients. Feraheme received marketing approval from the U.S. Food and Drug Administration on June 30, 2009 and was commercially launched by AMAG in the U.S. shortly thereafter. Ferumoxytol received marketing approval in Canada in December 2011, where it is marketed by Takeda as Feraheme, and in the European Union in June 2012 and Switzerland in August 2012, where it is marketed by Takeda as Rienso. For additional i product information, i please visit i Feraheme (ferumoxytol) Injection for Intravenous (IV) 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. Serious hypersensitivity reactions, including anaphylactic-type reactions, some of which have been life-threatening and fatal, have been reported in patients receiving Feraheme. Serious adverse reactions of clinically significant hypotension have been reported. In the post-marketing setting, life-threatening anaphylactic type reactions, cardiac/cardiorespiratory arrest, clinically significant hypotension, syncope, unresponsiveness and other safety events have been reported in patients being treated with Feraheme. In clinical trials, the most commonly occurring adverse reactions for Feraheme-treated patients were nausea, dizziness, hypotension, peripheral edema, headache, h edema and vomiting. i A full list of adverse events can be found in the full prescribing information for Feraheme. 26