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NPS Corp Pres

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These statements are based on management's current expectations ... Pfizer, Schering-Plough, Novartis. CMO. Alan Harris, M.D., Ph.D. Chugai, Amersham, Pharmacia ... – PowerPoint PPT presentation

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Title: NPS Corp Pres


1
January 2008
2
Safe Harbor Statement
  • This presentation contains forward-looking
    statements. These statements are based on
    managements current expectations and beliefs.
    Actual results could differ materially from those
    described. Please refer to Company documents
    filed with the SEC for a more detailed discussion
    of risks. The Company is under no obligation (and
    expressly disclaims any such obligation) to
    update or alter its forward-looking statements,
    whether as a result of new information, future
    events, or otherwise.
  • January 2008

3
The New NPS Delivering Value
  • High-potential product pipeline
  • Two Phase III compounds in multiple areas of
    unmet medical need
  • Three IND-stage programs
  • Experienced leadership team
  • Strong financial profile
  • Over 200 M in value from Cinacalcet royalties
  • Over 130 in cash/short-term investments

4
The New NPS 2007 Accomplishments
  • Completed corporate restructuring
  • Expect 08 burn of 35M - 45M
  • Raised gt275M
  • Retired 2008 convertible debt
  • Completed Phase 3 GATTEX SBS study
  • Executed GATTEX co-development agreement with
    Nycomed
  • Strengthened leadership team

5
The New NPS Experienced Leadership
6
NPS Multiple Opportunities to Deliver Value
Product
Pre-IND
Phase 1
Phase 2
Phase 3
Commercial
Partner
cinacalcet
(Sensipar)
Amgen
Secondary HPT
(US/Europe)
Secondary HPT
(REGPARA)
Kirin
(Asia)
GATTEXTM
Nycomed
(ex-N.Am.)
SBS
Crohn's disease
CIGIM
NEC
Nycomed
PREOS
(ex-US)
Osteoporosis
(PREOTACT)
Hypoparathyroidism
calcilytics
GSK
(Worldwide)
Osteoporosis
NPSP156
CNS
7
GATTEX Unique and Valuable Asset
  • Analog of naturally occurring glucagon-like
    peptide 2 (GLP-2)
  • Promotes GI mucosal regeneration
  • Multiple potential indications with significant
    unmet needs
  • Short bowel syndrome (SBS) Orphan Status
  • Chemotherapy-induced GI mucositis (CIGIM)
  • Necrotizing enterocolitis (NEC)
  • Crohns disease
  • Co-development with Nycomed

8
GATTEX in SBS Study Overview
  • 24-week dosing (0.05 or 0.10 mg/kg/d vs. placebo)
  • Study enrollment
  • 139 pts screened
  • 83 pts randomized
  • 71 pts completed the trial
  • Efficacy measure
  • Reduction in PN volume of 20
  • Protocol required statistical significance in
    high-dose group before evaluating low-dose group

9
GATTEX in SBSPromising Efficacy Results
  • PN discontinuation
  • 2 low-dose subjects at week 16
  • 1 high-dose subject at week 24

10
GATTEX in SBS Excellent Safety Profile
  • Comparable incidence of SAEs
  • Placebo 31.3
  • Low dose 37.1
  • High dose 34.4
  • Most common AEs include
  • Abdominal distention and pain
  • Nausea and vomiting
  • Injection site bruising and erythema
  • Headache
  • Only AE with significant difference versus
    placebo.

11
GATTEX in SBS Path Forward
  • Meeting with FDA Q1 2008
  • Complete Phase 3-extension study Q1 2008
  • If FDA accepts current data package, submit NDA
    in Q3 2008
  • If not, start confirmatory study ASAP
  • Efficacy endpoint 20 reduction in PN
  • Adequate sample size and power
  • Total Cost Estimate - 25M to 30M over 3 yrs.
  • Simplified study design to be discussed with FDA

12
GATTEX Nycomed Partnership Accelerates Global
Development
  • Nycomed granted rights to develop and market
    GATTEX ex-N.Am.
  • NPS receives
  • 35M upfront (includes 25M post data release)
  • Double-digit royalties
  • Up to 150M in additional payments for regulatory
    approval of new indications and sales milestones
  • Nycomed pays 50 of joint external development
    expenses

13
GATTEX Addl Indications
  • Necrotizing enterocolitis (NEC)
  • Affects 12,000 neonates annually
  • Mortality rate gt 50 in premature infants lt 1500
    grams
  • No current treatment
  • Pre-IND meeting with FDA in 2H 2008
  • Chemotherapy-induced GI mucositis (CIGIM)
  • Dose-limiting side effect of chemotherapy
  • Affects gt 500,000 patients worldwide annually
  • No current treatment
  • Meeting with FDA in 2H 2008 to discuss Phase 2
    program
  • Potential combined annual sales gt 500M
  • Company estimate

14
PREOS Osteoporosis Update
  • After careful review, NPS has decided to focus on
    hypoparathyroidism (HypoPTH)
  • Osteoporosis remains a viable opportunity
  • PREOTACT performing well in Europe
  • Good post-marketing safety record
  • Requires US partner to assume cost and risk

15
PREOS in HypoPTHA Low-Risk, High-Value
Opportunity
  • FDA approved Orphan Drug status in Aug. 2007
  • US prevalence 65,000 patients
  • Symptoms Paresthesias, muscle spasms, peri-oral
    numbness, altered mental status, tetany and
    psychoses
  • Treatment options limited
  • Lifelong Vitamin D analogues, high Ca diet, Vit
    D2 and Ca supplementation
  • Some patients require permanent catheter for IV
    Ca

16
PREOS in HypoPTHA Low-Risk, High-Value
Opportunity
  • PREOS could be first true PTH replacement therapy
  • Chronic administration of native PTH (1-84)
    intuitive
  • Low mechanism of action risk
  • FDA granted Orphan Drug status in August 2007
  • Worldwide annual sales could exceed 200M

17
PREOS in HypoPTH Path Forward
  • Dr. Bilezikian (Columbia U.) IIT ongoing
  • NPS initiating pivotal registration study to
    demonstrate safety and efficacy
  • Efficacy endpoints Eucalcemia, reduced
    supplemental calcium and vitamin D analogues
  • 75 -100 patients 2 or 3 doses vs. placebo
  • 8 to 10-week screening/stabilization
  • 24-week dosing
  • Study preparation started Expect data 2H 2010
  • Total cost estimate 20 to 25M over 3 years

18
Calcilytics (ronacaleret)GSK Partnership Update
  • Target indication Osteoporosis
  • Currently recruiting for Phase 2 dose-range study
    in post-menopausal women
  • Efficacy endpoint BMD
  • Two active comparators with placebo
  • 520 patients
  • 12-month dosing
  • Study initiated May 2007
  • 6 additional clinical studies ongoing

19
NPS Financial Strength
  • Cash/ST investments over 130M
  • Adequate resources to fund late-stage studies
  • Liquidity to fund over 2 years burn
  • Modest debt 50M convert due 2014 (5.44
    convert price)
  • Nycomed partnership expected to reduce GATTEX
    development costs significantly
  • Cinacalcet royalties (US/EU) represent over 200M
    additional value

20
Cinacalcet Significant Value
  • Sensipar
  • NPS receives royalties from Amgen on US/EU sales
  • Royalties monetized through non-recourse debt
    with estimated repayment in 2012
  • Royalties projected to accrue to NPS 2013
  • NPV of royalties estimated over 200M or approx.
    4/share
  • REGPARA
  • NPS receives royalties from Kirin on Far East
    sales
  • Approved in Japan Oct. 2007
  • Kirin seeking pricing approval for 2008 launch

21
Building Value in 2008 Key Milestones
  • GATTEX in SBS
  • Complete Phase 3-extension study by Q1 2008
  • If FDA accepts current package, submit NDA Q3
    2008
  • If not, start confirmatory study ASAP
  • GATTEX in additional indications
  • Complete preclinical studies in NEC and CIGIM
  • Initiate at least one FIM study 2H 2008
  • PREOS in HypoPTH
  • FPFV by 2H 2008
  • REGPARA launch in Japan (Kirin)

22
The New NPS Delivering Value
  • Healthy balance sheet
  • Multiple pipeline opportunities focused on unmet
    medical needs
  • Strong partnerships with industry leaders
  • Commercialized products
  • Experienced leadership

23
January 2008
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