NPS Pharma Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide
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1 NPS Pharma Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide Jefferies 2014 Global Healthcare Conference June 2, 2014 Francois Nader, MD President and Chief Executive Officer
2 Safe harbor statement Statements made in this presentation, which are not historical in nature, constitute forward-looking statements for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of These statements are based on the company's current expectations and beliefs and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forwardlooking statements. Forward looking statements include, but are not limited to, statements concerning the company s future financial performance and plans for the commercialization of its products, beliefs or expectations regarding our products in development, statements concerning the company s plans for international expansion, beliefs or expectations regarding potential revenue and earnings from product sales, including beliefs regarding our ability to grow sales, expectations regarding the market size for our products, including those in development, and beliefs or expectations regarding our operating expenses. Risks associated to the company's business include, but are not limited to, the risks associated with any failure by the company to successfully commercialize Gattex/Revestive (teduglutide [rdna origin]) for injection, including the risk that physicians and patients may not see the advantages of Gattex/Revestive and may therefore be reluctant to utilize the product, the risk that private and public payers may be reluctant to cover or provide reimbursement for Gattex, risks related to regulatory and reimbursement approvals for Natpara/Natpar (recombinant human parathyroid hormone 1-84 (rhpth 1-84)), the risks associated with the company's strategy, global macroeconomic conditions, the impact of changes in management or staff levels, the effect of legislation effecting healthcare reform in the United States, as well as other risk factors described in the company's periodic filings with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K and Form 10-Qs. All information in this presentation is as of the date of this presentation and NPS undertakes no duty to update this information, whether as a result of new information, future events or otherwise. June
3 NPS is advancing four strategic priorities to achieve value creation In Short Bowel Syndrome Build on success of U.S. launch In Hypoparathyroidism Secure U.S. BLA approval PDUFA date: Oct. 24, 2014 International Expansion Launch Revestive for Short Bowel Syndrome in EU in 2H14 File MAA for Natpar in hypoparathyroidism in 2014 Advancing Pipeline Progress global study of pediatric Short Bowel Syndrome Begin P2 study of NPSP795 in Autosomal Dominant Hypocalcemia Actively seek assets for in-licensing
4 Gattex (U.S.) in Adult Short Bowel Syndrome (SBS) In the US, GATTEX (teduglutide [rdna origin]) for injection is indicated for the treatment of adult patients with Short Bowel Syndrome (SBS) who are dependent on parenteral support.. 4
5 Gattex is the first major treatment advance in adult SBS in 40 years SBS is a potentially fatal disorder in which the body is unable to absorb enough nutrients and fluids through the gastrointestinal tract Gattex/Revestive is a proprietary analog of GLP-2, a naturally occurring peptide involved in gastrointestinal rehabilitation ~3,000 to 5,000 addressable Gattex-eligible patients in the U.S. Launched in U.S. February 2013 Penetrated 6 to 10% of addressable market and delivered sales of $32M in 2013 U.S. exclusivity expected through October
6 On track to deliver 200% year-over-year growth in net sales First quarter 2014 results: Net sales of $18M; sequential growth of 17% Physician interest remains high March and April prescriptions higher than 4Q13 averages Positive patient anecdotal feedback on response including significant reductions in or independence from parenteral support 2014 outlook: FY14 sales guidance of $100 to $110M Maximize market potential with new commercial initiatives Strengthened U.S. commercial leadership Increasing field-based sales organization Expand prescribers to surgeons (GI & others) Increase patient education and awareness In the US, GATTEX (teduglutide [rdna origin]) for injection is indicated for the treatment of adult patients with Short Bowel Syndrome (SBS) who are dependent on parenteral support. 6
7 Natpara in hypoparathyroidism 7
8 Hypoparathyroidism is a rare, multidimensional disorder Parathyroid hormone precisely regulates calcium and phosphate homeostasis Calcium is essential in muscle, skeletal, neurological functions, and in cardiac contraction Hypoparathyroidism characterized by deficient or absent PTH Untreated hypoparathyroidism typically results in a multitude of physical, cognitive, and emotional comorbidities. ~50,000 patients in U.S.; ~20,000 uncontrolled with current management will be initial commercial focus 8
9 Robust epidemiological research supports unmet medical need and high burden of illness Current approach is limited to large doses of calcium and active vitamin D However, 99% of patients remain symptomatic 80% 70% 37% 20% Waking hours of symptoms Comorbidities Mental health medications Employment changes 5-17x 4x >50% >50% 33% Renal complications 1,4 Increased seizures 1 B. ganglia calcification 2,4 Morphometric fractures 3 Increased ER visits 4 Source: PARADOX and 1. Unberbjerg L. et al. JBMR Goswani et al. Clin End Mendonca et al. BMC Mitchel et al. JCEM
10 % of patients % of patients Natpara BLA supported by the largest clinical program ever conducted in hypoparathyroidism Natpara is the only bioengineered replica of the full-length 1-84 endogenous parathyroid hormone Landmark 24-week Phase 3 REPLACE study published in The Lancet Diabetes and Endocrinology PTH replacement therapy observed Clinically meaningful reductions in oral calcium and active vitamin D Serum calcium maintained without increasing mean urinary calcium excretion Serum phosphate improved 60% 40% 20% 0% Triple Primary Efficacy Endpoint 53% 2% NATPARA Placebo N=90 N=44 p< Primary endpoint: decrease of at least 50% in calcium and vitamin D while maintaining normocalcemia 60% 40% 20% 0% Secondary Efficacy Endpoint 43% p< % NATPARA Placebo N=90 N=44 Secondary endpoint: achieve independence from Vitamin D therapy and require only 500 mg/day or less of oral calcium *Therapy of Hypoparathyroidism with PTH(1 84): A Prospective Four-Year Investigation of Efficacy and Safety Cusano et al. J Clin Endocrinol Metab, January 2013, 98(1):
11 Active Natpara US BLA review and pre-launch activities U.S. BLA filing: BLA supported by the largest clinical program ever conducted in hypoparathyroidism Advisory Committee tentative date: July 24, 2014 PDUFA date: October 24, 2014 Pre-launch activities are laying the groundwork for successful U.S. introduction Educating patients Educating physicians Profiling the condition through PARADIGHM U.S. exclusivity expected through
12 NPS Pharma International expansion 12
13 International expansion of orphan disease franchise underway Ex-U.S. rights to Gattex and Natpara regained March 2013 International leadership team now in place First EU launches of Revestive expected in 2H14 Elected to shift filing in Germany to 2H14 to enhance value dossier with new long-term data, including parenteral support weaning data EU exclusivity expected through August 2024 Building SBS awareness with KOLs in Japan and Latin America Filing for orphan drug status of Revestive in Japan in 2014 EU regulatory filing for Natpar planned for 2014 Exclusivity expected through (EU/ROW) 13
14 Peak international opportunity for Revestive could exceed that of Gattex in the US United States European Union Rest of World 3,000-5,000 patients 3,000-6,000 patients 2,500-6,000 patients In the US, GATTEX (teduglutide [rdna origin]) for injection is indicated for the treatment of adult patients with Short Bowel Syndrome (SBS) who are dependent on parenteral support. In the EU, REVESTIVE (teduglutide) is indicated for the treatment of adult patients with SBS; patients should be stable following a period of intestinal adaptation after surgery. 14
15 Hypoparathyroidism affects ~130K patients in key ex-us markets* Initial commercial focus will be uncontrolled patients Patients who are controlled with current management ~80K ~50K Uncontrolled patients Patients who are uncontrolled with current management *EU5, Japan, and Latin America. 15
16 NPS Pharma product pipeline 16
17 NPS strategy is focused on long-term growth by building a global pipeline of innovative rare disease therapeutics Gattex/Revestive in pediatric SBS Complex and highly morbid condition Global study underway NPSP795 in Autosomal Dominant Hypocalcemia Life-long genetic disease caused by a mutation of the calcium-sensing receptor gene P2 proof-of-concept study to begin mid-2014 Active Business Development First-in or best-in class products to treat rare disorders of high unmet medical need 17
18 NPS Pharma Financials and Milestones 18
19 NPS strong financial position and outlook will enable investment in long-term growth Strong cash and investments: $176M at 3/31/14 Gattex/Revestive sales continue to ramp 1Q14 sales $18M; FY13 sales $32M Significant and consistent royalties from Amgen s global sales of Sensipar 1Q14 royalties $23M; FY13 royalties $113M 2014 financial guidance: $100 to $110M in net Gattex/Revestive sales $180 to $200M in operating expenses, excluding COGS and share-based compensation $1 $5 Revenues Net Gattex/Revestive sales $s millions $11 $15 $18 1Q13 2Q13 3Q13 4Q13 1Q14 19
20 NPS is well-equipped to deliver key milestones in 2014 as it advances its vision to become the world s premier orphan drug business Milestone Status Grow the Gattex / Revestive Franchise $100 to $110M in 2014 net sales 4Q14 Secure U.S. approval of Natpara Successful U.S. FDA Advisory Committee Approval of U.S. BLA Expand international business Named-patient programs for REVESTIVE First EU country launch for REVESTIVE EU MAA filing for Natpar 3Q14 4Q14 2H14 2H14 Advance product pipeline Progress global study of Gattex/Revestive in pediatric SBS 2014 File IND for NPSP795 in ADH FPI Phase 2a study of NPSP795 in ADH Mid-2014
21 NPS Pharma Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide Francois Nader, MD President and Chief Executive Officer
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