+ All Categories
Home > Documents > External Slide Template - Jefferies Group · NPS Pharma 12 NPSP795 in development for Autosomal...

External Slide Template - Jefferies Group · NPS Pharma 12 NPSP795 in development for Autosomal...

Date post: 19-Jan-2021
Category:
Upload: others
View: 1 times
Download: 0 times
Share this document with a friend
17
NPS Pharma Pioneering and delivering innovative therapies that transform the lives of patients with rare diseases worldwide Jefferies 2014 Global Healthcare Conference November 20, 2014 Francois Nader, MD President and Chief Executive Officer
Transcript
Page 1: External Slide Template - Jefferies Group · NPS Pharma 12 NPSP795 in development for Autosomal Dominant Hypocalcemia (ADH) •Ultra-rare, life-long genetic disease caused by a mutations

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

Page 2: External Slide Template - Jefferies Group · NPS Pharma 12 NPSP795 in development for Autosomal Dominant Hypocalcemia (ADH) •Ultra-rare, life-long genetic disease caused by a mutations

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

2

© 2014 NPS Pharmaceuticals, Inc.

Page 3: External Slide Template - Jefferies Group · NPS Pharma 12 NPSP795 in development for Autosomal Dominant Hypocalcemia (ADH) •Ultra-rare, life-long genetic disease caused by a mutations

NPS Pharma

The NPS business plan focuses on long-term value creation

3

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

1 2 3

Page 4: External Slide Template - Jefferies Group · NPS Pharma 12 NPSP795 in development for Autosomal Dominant Hypocalcemia (ADH) •Ultra-rare, life-long genetic disease caused by a mutations

NPS Pharma

Three key growth initiatives are propelling NPS Pharma forward

4

321

Build global endocrinology portfolio

Grow globalgastroenterology portfolio

© 2014 NPS Pharmaceuticals, Inc.

Page 5: External Slide Template - Jefferies Group · NPS Pharma 12 NPSP795 in development for Autosomal Dominant Hypocalcemia (ADH) •Ultra-rare, life-long genetic disease caused by a mutations

NPS Pharma

Gattex is the first major treatment advance in adult SBS in 40 years

5

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

Page 6: External Slide Template - Jefferies Group · NPS Pharma 12 NPSP795 in development for Autosomal Dominant Hypocalcemia (ADH) •Ultra-rare, life-long genetic disease caused by a mutations

NPS Pharma 6

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.

Page 7: External Slide Template - Jefferies Group · NPS Pharma 12 NPSP795 in development for Autosomal Dominant Hypocalcemia (ADH) •Ultra-rare, life-long genetic disease caused by a mutations

NPS Pharma

Three key growth initiatives are propelling NPS Pharma forward

7

321

Build global endocrinology portfolio

Grow globalgastroenterology portfolio

© 2014 NPS Pharmaceuticals, Inc.

Page 8: External Slide Template - Jefferies Group · NPS Pharma 12 NPSP795 in development for Autosomal Dominant Hypocalcemia (ADH) •Ultra-rare, life-long genetic disease caused by a mutations

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

8

© 2014 NPS Pharmaceuticals, Inc.

Page 9: External Slide Template - Jefferies Group · NPS Pharma 12 NPSP795 in development for Autosomal Dominant Hypocalcemia (ADH) •Ultra-rare, life-long genetic disease caused by a mutations

NPS Pharma

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

9

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.

Page 10: External Slide Template - Jefferies Group · NPS Pharma 12 NPSP795 in development for Autosomal Dominant Hypocalcemia (ADH) •Ultra-rare, life-long genetic disease caused by a mutations

NPS Pharma 10

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

Page 11: External Slide Template - Jefferies Group · NPS Pharma 12 NPSP795 in development for Autosomal Dominant Hypocalcemia (ADH) •Ultra-rare, life-long genetic disease caused by a mutations

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

11

© 2014 NPS Pharmaceuticals, Inc.

Page 12: External Slide Template - Jefferies Group · NPS Pharma 12 NPSP795 in development for Autosomal Dominant Hypocalcemia (ADH) •Ultra-rare, life-long genetic disease caused by a mutations

NPS Pharma 12

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.

Page 13: External Slide Template - Jefferies Group · NPS Pharma 12 NPSP795 in development for Autosomal Dominant Hypocalcemia (ADH) •Ultra-rare, life-long genetic disease caused by a mutations

NPS Pharma

Three key growth initiatives are propelling NPS Pharma forward

13

321

Address new rare diseases through in-licensing and acquisition

Build global endocrinology portfolio

Grow globalgastroenterology portfolio

Page 14: External Slide Template - Jefferies Group · NPS Pharma 12 NPSP795 in development for Autosomal Dominant Hypocalcemia (ADH) •Ultra-rare, life-long genetic disease caused by a mutations

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

14

© 2014 NPS Pharmaceuticals, Inc.

Page 15: External Slide Template - Jefferies Group · NPS Pharma 12 NPSP795 in development for Autosomal Dominant Hypocalcemia (ADH) •Ultra-rare, life-long genetic disease caused by a mutations

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.

15

Page 16: External Slide Template - Jefferies Group · NPS Pharma 12 NPSP795 in development for Autosomal Dominant Hypocalcemia (ADH) •Ultra-rare, life-long genetic disease caused by a mutations

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.

16

Page 17: External Slide Template - Jefferies Group · NPS Pharma 12 NPSP795 in development for Autosomal Dominant Hypocalcemia (ADH) •Ultra-rare, life-long genetic disease caused by a mutations

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


Recommended