nps pharma jun13pres
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NPS Pharmaceuticals:
Pioneering and delivering therapies that transform the lives of patients with rare
diseases worldwide
June 2013
2
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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2013 strategic priorities focus on positioning NPS as a leading global orphan specialty drug company
12
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
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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2013 strategic priorities focus on positioning NPS as a leading global orphan specialty drug company
16
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 EU as
Revestive®) and PTH 1-84
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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2013 strategic priorities focus on positioning NPS as a leading global orphan specialty drug company
18
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
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
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