nps pharma jun13pres

NPS Pharmaceuticals:

Pioneering and delivering therapies that transform the lives of patients with rare

diseases worldwide

June 2013

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Safe harbor statement

Statements made in this presentation, which are not historical in nature, such as the timing of the commercialization of the company’s approved product, the expected uptake and use of the approved product, expected revenues from the approved product, the timing of the potential regulatory submission, approval process and commercialization of the company’s late-stage registration program, the company’s expected future cash flows from the company’s royalty-based portfolio of products and product candidates, the company’s future operations and the company’s 2013 cash burn guidance, 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, such as market acceptance of its approved product, the risk that third-party payers don’t provide reimbursement for the cost of the company’s approved product, the risk that the company does not receive regulatory approval to market its late-stage registration program in a timely manner, or at all, the risk that the company fails to maintain its existing collaborative relationships related to the company’s royalty-based portfolio of products, the risk that the company’s contract manufacturers will be unable to supply products and clinical supplies and the risk that the company’s cash flows are lower than expected due to increased expenses or lower cash in-flows from applicable collaborations and operations 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.

June 2013

NPS: a global biopharmaceutical company focused on rare disorders

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Prescriptions

Teduglutide (Gattex® (U.S.); Revestive® (EU)) approved for adult Short Bowel Syndrome; expected exclusivity 2020(U.S.)/2022(EU)

Sensipar royalties generating $60+M in annual cash flow; expected to increase to $100+M in 2015 through 2018 patent expiry

Commercially differentiated assets

Attractive pipeline of orphan products

Reacquired global rights for teduglutide and PTH 1-84 from Takeda in March 2013

Initiating reimbursement process for Revestive in Europe Defining named patient programs in select ex-EU countries

Gated international expansion underway

Consistently delivered on stated milestones $90M in cash and investments at 03/31/13; received cash proceeds

of $93M from secondary offering of 6.9M shares in May 2013 Proven expertise in developing/launching orphan products

Proven successful track record

Marketing submissions for PTH 1-84 in hypoparathyroidism (U.S. 2H13 and EU 1H14); expected exclusivity 2026(U.S.)/2025(EU)

Initiation of new development programs in 2013: pediatric Short Bowel Syndrome and autosomal dominant hypocalcemia (ADH)

2013 strategic priorities focus on positioning NPS as a leading global orphan specialty drug company

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Flawless execution of the Gattex® (teduglutide) launch Launched February 2013 in the U.S. for the treatment of adult short bowel

syndrome (SBS)

Submit Biologic License Application (BLA) for Natpara® (recombinant human parathyroid hormone (PTH) 1-84) in 2H13 Could be first approved replacement therapy for hypoparathyroidism

Advance additional pipeline opportunities to build long-term sustainable growth

Implement targeted international expansion of NPS In March, NPS reacquired worldwide rights to teduglutide (approved in EU as

Revestive®) and PTH 1-84

Short Bowel Syndrome (SBS) is a debilitating orphan condition with high unmet medical need

Results from surgical resection, congenital defect or disease-associated loss of absorption of the intestine

Can result in dependence on long-term parenteral support1 to receive nutrients and fluids intravenously to sustain life

Challenges of parenteral support:

Typically infused 5 to 7 days per week for up to 10-12 hours per day

Does not improve intestinal absorption

Life-threatening complications include: infections, blood clots or liver damage

Patients make up to 25 trips to bathroom/day

Annual costs of $185K-$568K3

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Factors affecting

parenteral support

dependence2

Length and function of remaining

bowel

Comorbidconditions

Patient’s age

Presence/absence of colon

& ileocecalvalve

1: Thompson JS , et al. Curr Probl Surg. 2012;49:52-115. 2: Buchman AL, et al. Gastroenterology. 2003;124(4):1111-1134.3: NIH publication (Nurs Econ. 2010 August; 28 (4) : 255-264)

Gattex launched in the U.S. in February 2013

Gattex is a proprietary analog of GLP-2, a naturally occurring peptide involved in gastrointestinal rehabilitation

Enhances intestinal absorption

Significantly reduces dependence on parenteral support

15% (13 of 88) patients achieved independence from parenteral support on Gattex in STEPS 2 study

Approved December 2012 in the U.S. for adult patients with SBS who are dependent on parenteral support

First long-term treatment for adult SBS

Self-injected subcutaneous once-a-day

Orphan status; U.S. patent exclusivity until April 2020

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Heading

U.S. launch off to a strong start with key revenue drivers performing at or ahead of expectations (as of May 3)

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160 prescriptions receivedPrescriptions

42 patients on GattexPatients

Field activities and launch data support 3,000-5,000 Gattex-eligible patients in the U.S.Addressable patients

Presented to 34 healthcare plans (145M+ lives); all payers have indicated coverage

Payer receptivity

Note: information as of first quarter update on May 9, 2013

Co-pays in-line with expectations; ranging from $0 to $200 per month for commercial plans

Co-pay

High-caliber field force presenting Gattex to target prescribers

27-person strong sales team deployed with detailed launch ‘play book’

~15 years industry experience from premier orphan companies like Alexion, Genzyme, Shire, and ViroPharma

Successful track records with orphan biologics and/or gastrointestinal products

Targeting gastroenterologists practicing in ~1,000 institutions

Reinforced by significant medical affairs resources

8 Medical Science Liaisons

7 key publications supporting launch, including pivotal Phase 3 Gattex studies

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Reimbursement tracking in line/slightly ahead of expectations

Payers recognizing Gattex value proposition

Prescriptions approved by many key national and regional health plans, including marquee names:

− United Health, Wellpoint, Aetna, Humana, Cigna, Express Scripts/Medco, and CVS/Caremark have approved prescriptions

− Several government-sponsored health plans have also approved prescriptions

Time from prescription-to-dispense shorter than expected for first patients

Patients’ out-of-pocket exposure – as expected

Prior authorization to the label – as expected

Payer interactions reaffirm value Gattex brings to patients, caregivers, and healthcare system

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NPS Advantage™ provides personalized patient assistance

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NPS Care Coordinator assigned to each patient

Proving to be a critical resource for patients and healthcare providers

Conduct benefits evaluations

Help with patient assistance programs for co-pay/co-insurance

Coordinate home nursing services

Communicate benefits information, answers to questions, and resources to patients, healthcare providers, and treatment team

Assist with compliance and adherence

Gattex in adult SBS: U.S. commercialization metrics

Patients 200-300 patients expected to be on therapy by end of 2013

− Most toward the latter part of the year

Expected time from prescription-to-dispense 90-120 days

Expected patient adherence and compliance rates of 70-80%

Gattex pricing Annual list price (equivalent of WAC) of $295,000

Gross-to-net expected to be between 10-14%

− Includes industry-standard prompt-pay discount, contracted clinical service fees, patient assistance, returns, rebates, etc.

Meaningful revenues expected to begin during 3Q13

U.S. peak annual revenues expected to be well in excess of $350M Does not include ex-U.S. opportunity

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syndrome (SBS)





Hypoparathyroidism is a rare endocrine disorder with high unmet medical need and significant burden of illness

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Typically arises after removal or damage to the parathyroid glands during neck surgery

Inadequate production of parathyroid hormone results in hypocalcemia and hyperphosphatemia

Calcium plays a crucial role in functioning of skeletal, neurological, and muscular systems

Symptoms: paresthesias, muscle spasms, tetany, back pain, myalgia, muscle twitching, throat tightness, musculoskeletal pain, anxiety, depression

Current approaches aim to reduce severity of symptoms with calcium and active vitamin D supplementation

Supplements can cause irreversible complications including calcifications in the kidneys, heart or brain

Currently quantifying U.S. addressable patients

Approved replacement

therapyEndocrine deficiency

√ Hypothyroidism

√ Diabetes

√ Hypoaldosteronism

√ Hypopituitarism

Hypoparathyroidism

NATPARA is the first and only bioengineered replica of the native full-length endogenous 84-amino acid parathyroid hormone

Natpara’s mechanism of action offers a physiological solution

Maintaining normocalcemia while potentially reducing large serum calcium fluctuations

Potential to reduce long-term complications associated with supplements

NATPARA will be available in multiple dosages to allow for personalized treatment

U.S. BLA submission expected 2H13

Final user study of injection pen completed

Advancing multiple tracks to resolve fill-finish manufacturing issue

BLA market exclusivity expected through 2026

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Data from PARADOX study in hypoparathyroidism underscore significant burden of illness

Significant misconceptions about burden of illness of hypoparathyroidism

Especially related to the magnitude of negative impact on patients, caregivers, and healthcare system

Current palliative options aim to manage symptoms and do not treat the underlying issue of parathyroid deficit

NPS commissioned PARADOX -- largest epidemiological landmark study in hypoparathyroidism

374-patient study

Conducted in partnership with the Mayo Clinic and the Hypoparathyroidism Association

Characterized the daily physical, emotional, and financial challenges of patients suffering from hypoparathyroidism despite calcium and vitamin D intake

Results to be reported at ENDO 2013 (June, San Francisco)

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syndrome (SBS)



Implement targeted international expansion of NPS In March, NPS reacquired worldwide rights to teduglutide (approved EU as


NPS is implementing a targeted international expansion to build long-term commercial success and value creation

Regained ex-U.S. rights to teduglutide (Revestive®) and PTH 1-84 (Preotact®) in March 2013 from Takeda GmbH

Takeda received 6.1M shares of NPS common stock valued at ~$55M on date of transaction

NPS received:

− $37M in API inventory

− Next-generation manufacturing process for teduglutide

− New pen device for PTH 1-84

Advancing a multi-prong strategy:

Prioritizing EU countries for Revestive (teduglutide) launch-sequencing

− Revestive approved in EU in August 2012

− Developing core value dossier for Revestive to start pricing and reimbursement

Defining key ex-U.S. countries to begin paid named-patient programs

Maximizing PTH 1-84 potential (hypoparathyroidism, osteoporosis)

Assessing market potential/regulatory requirements for both products in Japan

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syndrome (SBS)





Maximizing the potential of Gattex through proactive life-cycle management; first program will be pediatric SBS

Traumatizing/socially debilitating for children

Significant average costs over a 5-year period1

− ~$1.6M cost of hospital/home care; 146 days in hospital

Estimated prevalence ~10-20% of adult population

Pediatric and adult SBS treated by same KOLs/centers of excellence

Recently met with FDA to discuss development strategy

− Agency recognized high unmet medical need and potential benefits of Gattex in pediatric SBS

Next step: meet with EMA to discuss global development program

191 Pediatric short-bowel syndrome: the cost of comprehensive care. Spencer et al. Am J Clin Nutr 2008;88:1552-9

Calcilytics could be a novel treatment for the rare disorder, autosomal dominant hypocalcemia (ADH)

Earlier-stage pipeline includes calcilytics (NPSP795/NPSP790)

Calcilytics are small molecule antagonists of the calcium receptor

ADH is an ultra-rare disorder caused by a gain-of-function mutation in the calcium-sensing receptor gene (CaSR)

CaSR is the body’s primary regulator of systemic calcium homeostasis

Characterized by: hypocalcemia, low levels of PTH, hyperphosphatemia

Symptoms include: tingling, cramping, kidney stones, nephrocalcinosis, bone pain, and seizures

No approved therapy; current approaches aim to manage symptoms

Calcilytics’ mechanism of action indicates therapeutic potential in ADH

Meeting requested to discuss global development program with FDA and EMA

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2013 Financials and Milestones

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Net Gattex sales $0.7

Pro forma cash and investments1 $184

Adjusted current liabilities2 $17

5.75% convertible debt due 2014 ($5.44 conversion) $17

Non-recourse debt (Sensipar/Mimpara-secured $74M) $153

Pro forma shares outstanding1 100

2013 operating expense guidance3 $135-145

First quarter 2013 financial results

1 Adjusted for May 2013 secondary offering of 6.9M shares at $14.53 per share (estimated net proceeds of $93.4 million after deducting underwriting discounts and commissions and estimated offering expenses)

2 Adjusted current liabilities of $17M is a non-GAAP financial measure that excludes $6M related to the current portion of the company’s non-recourse Sensipar/Mimpara-secured non-recourse debt. Total current liabilities at 03.31.13 according to U.S. GAAP were $23M.

3 Includes share-based compensation and excludes cost of goods sold

03/31/13(in millions)

Milestone Status

GATTEX in short bowel syndrome:Commercial launch of GATTEX Present STEPS 2 results 4Q13

Gattex launch updates (# prescriptions / patients on therapy)

2Q-4Q

End 2013 with 200-300 U.S. patients on Gattex 4Q

NATPARA in hypoparathyroidism:U.S. BLA submission 2H13

Report results from PARADOX study June 2013

Ex-U.S. commercial strategy 2H13

Gattex global pediatric development strategy 2013

Calcilytics global development strategy 2013

2013 is becoming a breakout year for NPS as it transforms into a premier global orphan drug business

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NPS Pharmaceuticals:

Pioneering and delivering therapies that transform the lives of patients with rare

diseases worldwide

June 2013

nps pharma jun13pres

Health & Medicine

companys approved product

companys cash flows

companys future operations

gattex teduglutide

revestive eu

risk factors

companys periodic filings

future cash flows