NPS PharmaPioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide

Jefferies 2014 Global Healthcare Conference June 2, 2014

Francois Nader, MDPresident and Chief Executive Officer

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 1995. 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 forward-looking 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 20142

NPS Pharma | Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide

NPS is advancing four strategic priorities to achieve value creation

NPS Pharma | Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide

In Hypoparathyroidism

• Secure U.S. BLA approval• PDUFA date: Oct. 24, 2014

• Launch Revestive® for Short Bowel Syndrome in EU in 2H14

• File MAA for Natpar® in hypoparathyroidism in 2014

International Expansion

• Build on success of U.S. launch

In Short Bowel Syndrome

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

Gattex® (U.S.) in Adult Short Bowel Syndrome (SBS)

NPS Pharma | Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide

4In 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..

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 2020

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NPS Pharma | Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide

On track to deliver 200% year-over-year growth in net sales

6

NPS Pharma | Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide

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.

Natpara® in hypoparathyroidism

NPS Pharma | Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide

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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.

• ~50,000 patients in U.S.; ~20,000 uncontrolled with current management will be initial commercial focus

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NPS Pharma | Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide

Robust epidemiological research supports unmet medical need and high burden of illness

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NPS Pharma | Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide

Comorbidities

70%

Employment changes

20%

Mental health medications

37%

Waking hours of symptoms

80%

Renal complications1,4

5-17xIncreased seizures1

4x

IncreasedER visits4

33%B. ganglia

calcification2,4

>50%

Morphometric fractures3

>50%

Current approach is limited to large doses of calcium and active vitamin D

However, 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.

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

10

53%

2%0%

20%

40%

60%

NATPARA Placebo

p<0.0001

% o

f p

atie

nts

Triple Primary Efficacy Endpoint

N=90 N=44

Primary endpoint: decrease of at least 50% in calcium and vitamin D while maintaining normocalcemia

*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):137–144.

NPS Pharma | Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide

43%

5%

0%

20%

40%

60%

NATPARA Placebo

Secondary endpoint: achieve independence from Vitamin D therapy and require only 500 mg/day or less of oral calcium

Secondary Efficacy Endpoint

N=90 N=44

% o

f p

atie

nts

p<0.0001

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 2026

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NPS Pharma | Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide

NPS Pharma International expansion

NPS Pharma | Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide

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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 2025+ (EU/ROW)

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NPS Pharma | Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide

Peak international opportunity for Revestive could exceed that of Gattex in the US

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United States European Union Rest of World

3,000-6,000patients

2,500-6,000patients

3,000-5,000patients

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.

NPS Pharma | Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide

Patients who are controlled

with current management

Patients who areuncontrolled

with current management

~80K

Uncontrolledpatients

~50K

Hypoparathyroidism affects ~130K patients in key ex-US markets*

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NPS Pharma | Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide

*EU5, Japan, and Latin America.

Initial commercial focus will be uncontrolled

patients

NPS Pharma product pipeline

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NPS Pharma | Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide

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

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NPS Pharma | Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide

NPS Pharma Financials and Milestones

NPS Pharma | Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide

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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

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Revenues

$1

$5

$11

$15$18

0

5

10

15

20

1Q13 2Q13 3Q13 4Q13 1Q14

Net Gattex/Revestive sales$s millions

NPS Pharma | Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide

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 3Q14

Approval of U.S. BLA 4Q14

Expand international business

Named-patient programs for REVESTIVE

First EU country launch for REVESTIVE 2H14

EU MAA filing for Natpar 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

NPS Pharma | Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide

NPS PharmaPioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide

Francois Nader, MDPresident and Chief Executive Officer