NPS PharmaPioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide
Jefferies 2014 Global Healthcare Conference November 20, 2014
Francois Nader, MDPresident and Chief Executive Officer
NPS Pharma
Safe harbor statement
Statements made in this presentation, which are not historical in nature, constitute forward-looking statementsfor purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. These statementsare based on the company's current expectations and beliefs and are subject to a number of factors anduncertainties that could cause actual results to differ materially from those described in the forward-lookingstatements. Forward looking statements include, but are not limited to, statements concerning the company'sfuture financial performance and plans for the commercialization of its products, including statements regardinglaunch timelines and plans for Natpara, beliefs or expectations regarding our products in development,statements concerning the company's plans for international expansion, beliefs or expectations regardingpotential 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 orexpectations regarding our operating expenses. Risks associated to the company's business include, but are notlimited 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 theadvantages of Gattex/Revestive and may therefore be reluctant to utilize the product, the risk that private andpublic payers may be reluctant to cover or provide reimbursement for Gattex, risks related to regulatory approvalsfor recombinant human parathyroid hormone 1-84 (rhPTH [1-84]), the risks associated with the company'sstrategy, global macroeconomic conditions, the impact of changes in management or staff levels, the effect oflegislation effecting healthcare reform in the United States, as well as other risk factors described in thecompany's periodic filings with the U.S. Securities and Exchange Commission, including its Annual Report on Form10-K and Form 10-Qs. All information in this presentation is as of the date of this presentation and NPS undertakesno duty to update this information, whether as a result of new information, future events or otherwise.
November 2014
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© 2014 NPS Pharmaceuticals, Inc.
NPS Pharma
The NPS business plan focuses on long-term value creation
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MISSIONPioneer and deliver innovative therapies that transform the lives
of patients with rare diseases worldwide
Create a world where every person living with a rare disease has a therapyVISION
Deliver ‘first-in’ or ‘best-in’ disease therapies that provide quantifiable value to patients and society
STRATEGY
Patient-Centric Goal: Pioneer & deliver innovative therapies for 10 discrete rare diseases by 2023
LONG-TERM GOAL
Integrity, respect, excellence, personal ownership, teamwork, entrepreneurial spirit and fun
VALUES
Grow globalgastroenterology
Portfolio
Build global endocrinology Portfolio
Address newrare diseases
through in-licensing& acquisition
THREE GLOBAL PILLARS
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NPS Pharma
Three key growth initiatives are propelling NPS Pharma forward
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Build global endocrinology portfolio
Grow globalgastroenterology portfolio
© 2014 NPS Pharmaceuticals, Inc.
NPS Pharma
Gattex is the first major treatment advance in adult SBS in 40 years
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2014 highlights:
• Significant quarter-over-quarter growth
• $68M in net sales as of 9/30/14; full-year net sales expected at low-end of $100-110M guidance
• Physician and patient interest remains high
• Expanding prescriber base
• Expanded US field force
• Enhanced physician targeting
© 2014 NPS Pharmaceuticals, Inc.
Gattex launch continues to deliver significant growth with year-over-year 2014
growth on track for 200+%
$0.7
$4.8
$11.0
$15.2$17.9
$22.0
$28.1
$0
$5
$10
$15
$20
$25
$30
1Q13 2Q13 3Q13 4Q13 1Q14 2Q14 3Q14
Net sales$s millions
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International rollout of Revestive progressing
Europe: pricing and reimbursement process
• Germany: Filed AMNOG dossier marking the launch and start of IQWIG assessment process; expect to begin pricing discussions 1Q15
• France: Submitted Health Technology Assessment (value dossier); expect to begin pricing discussions 1Q15
• UK: Secured public pricing; Revestive accessible via individual funding requests (IFRs)
• Nordic countries: Secured public pricing; Revestive accessible via IFRs
Latin America
• Argentina: Processing named-patient requests from physicians
Japan development strategy
• Received orphan designation; on track to begin small local study this year
© 2014 NPS Pharmaceuticals, Inc.
NPS Pharma
Three key growth initiatives are propelling NPS Pharma forward
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Build global endocrinology portfolio
Grow globalgastroenterology portfolio
© 2014 NPS Pharmaceuticals, Inc.
NPS Pharma
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 co-morbidities.
• ~180K patients in key global markets (~50K in US)
– Commercial focus expected to be patients who are not adequately controlled with current management
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© 2014 NPS Pharmaceuticals, Inc.
NPS Pharma
Robust epidemiological research supports unmet medical need and high burden of illness
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Comorbidities
70%
Mental health medications
37%
Waking hours of symptoms
80%
Renal complications1,4
5-17xIncreased seizures1
4xIncreasedER visits4
33%B. ganglia
calcification2,4
>50%Morphometric
fractures3
>50%
Current approach is limited to large doses of calcium and active vitamin D
99% of patients remain symptomatic
Source: PARADOX and 1. Unberbjerg L. et al. JBMR. 2013. 2. Goswani et al. Clin End. 2012. 3. Mendonca et al. BMC. 2013. 4. Mitchel et al. JCEM. 2012.
© 2014 NPS Pharmaceuticals, Inc.
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Focused on advancing the US and EU regulatory processes for Natpara/Natpar in hypoparathyroidism
© 2014 NPS Pharmaceuticals, Inc.
September 2014: FDA AdCommvoted in favor of Natpara approval
November 2014: Filed MAA with EMA
January 2015: FDA PDUFA action date for Natpara
2Q15: US Commercial launch of Natpara
NPS Pharma
Preparing for the successful US introduction of Natpara as the first parathyroid replacement therapy for patients with Hypoparathyroidism
• Significant interest in digital and social media programs (hypoparathyroidism.com, Facebook, YouTube)
• E-book and Emergency Resource Kit (~2,000 requests)• Advancing PARADIGHM long-term natural history registry
• Field-based team profiled ~3,500 endocrinologists• MSLs building awareness at COEs• Sales leadership in place• Recruiting dedicated Natpara sales force
Market Access
• Establishing network of specialty pharmacies• NPS Advantage expanding to include Natpara-focused Care
Coordinators• Plan to offer a suite of patient assistance programs
Physician Profiling & Field Force Deployment
Disease Awareness
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NPSP795 in development for Autosomal Dominant Hypocalcemia (ADH)
• Ultra-rare, life-long genetic disease caused by a mutations of the calcium-sensing receptor gene affecting children and adults
– Estimated to affect only a few thousand patients worldwide
• Increased sensitivity to serum calcium and continuous excretion of calcium because receptor always senses serum calcium is too high
• Life-threatening neonatal complications
– Cardiac arrhythmias, seizures or laryngeal spasms
• Significant increased risk for renal complications
– Nephrocalcinosis and impaired renal function
• Currently no approved treatment for ADH
• NPSP795 is a selective calcium receptor antagonist
– Binds to the CaSR and decreases its sensitivity to serum calcium to restore normal calcium homeostasis
• Phase 2 proof-of-concept study is underway
© 2014 NPS Pharmaceuticals, Inc.
NPS Pharma
Three key growth initiatives are propelling NPS Pharma forward
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Address new rare diseases through in-licensing and acquisition
Build global endocrinology portfolio
Grow globalgastroenterology portfolio
NPS Pharma
NPS Pharma is driving its long-term ‘10 in 10’ goal through active business development
• Pursuing acquisition / in-licensing opportunities for first-in or best-in rare disease therapeutics that provide quantifiable value to patients and society
• Integrated customer offering
– Companion diagnostics via partnering
– Delivery technologies
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© 2014 NPS Pharmaceuticals, Inc.
NPS Pharma
NPS’ strong financial position and outlook will enable investment in long-term growth
• Strong cash and investments
– $169M at 9/30/14
• Gattex/Revestive sales continue to grow
– 3Q14 sales $28M (28% QOQ growth)
• Royalties from Amgen’s global sales of Sensipar® provide financial flexibility
– 3Q14 royalties $33M; FY13 royalties $113M
• 2014 financial guidance:
– $100 to $110M in net Gattex/Revestive sales; expect to be at low-end of guidance
– $185 to $195M in operating expenses, excluding COGS and share-based compensation
$18$22
$28
0
10
20
30
1Q14 2Q14 3Q14
Net Gattex/Revestive sales$s millions
© 2014 NPS Pharmaceuticals, Inc.
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NPS Pharma
NPS Pharma continues to deliver key milestones as it pursues its vision to create a world where every person with a rare disease has a treatment
Milestone Status
Growing global gastrointestinal franchise
Achieve FY 2014 net sales guidance for Gattex/Revestive 2014
Top-line data from global study in pediatric SBS 2014
Named-patient programs for Revestive
First EU country launch for Revestive
Building a global endocrinology franchise
US FDA Advisory Committee for Natpara
Approval of US BLA for Natpara PDUFA: 01/24/15
File EU MAA for Natpar
File IND for NPSP795 in ADH
FPI Phase 2a study of NPSP795 in ADH
© 2014 NPS Pharmaceuticals, Inc.
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NPS PharmaPioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide