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Caution should be exercised when HETLIOZ is administered to a nursing woman. HETLIOZ has not been studied in patients with severe hepatic impairment and is not recommended in these patients. Safety and effectiveness of HETLIOZ in pediatric patients have not been established. Full HETLIOZ Prescribing Information can be found at: www.hetlioz.com . 26 Vanda Pharmaceuticals Inc. 27
HETLIOZ should be avoided in combination with rifampin or other CYP3A4 inducers, because of a potentially large decrease in exposure of HETLIOZ , with reduced efficacy. There are no adequate and well controlled studies of HETLIOZ in pregnant women. Based on animal data, HETLIOZ may cause fetal harm. HETLIOZ should be used during pregnancy only if the potential benefit justifies the potential risks.
The risk of adverse reactions may be greater in elderly ( 65 years) patients than younger patients because exposure to HETLIOZ is increased by approximately 2 fold compared with younger patients. Use of HETLIOZ should be avoided in combination with fluvoxamine or other strong CYP1A2 inhibitors, because of a potentially large increase in exposure of HETLIOZ , and a greater risk of adverse reactions.
Important Safety Information HETLIOZ may cause somnolence: After taking HETLIOZ , patients should limit their activity to preparing for going to bed, because HETLIOZ can impair the performance of activities requiring complete mental alertness. The most common adverse reactions (incidence 5% and at least twice as high on HETLIOZ than on placebo) were headache, increased alanine aminotransferase, nightmares or unusual dreams, upper respiratory or urinary tract infection.
This includes intangible asset amortization expense of $2.5 million and $6 to $8 million of non cash stock based compensation Full year 2014 expenses are expected to reflect lower research and development spending as compared to 2013 and an increase in commercial spending to support the commercial launch of HETLIOZ in the US 1H 2014 includes $30 million for the Non 24 Awareness Campaign 2014 HETLIOZ revenue recognition is expected to follow the sell through method and reflect direct sales from Specialty Pharmacies to patients 25 (1) Cash, cash equivalents and marketable securities HETLIOZ Important Safety Information Indication and Important Safety Information About HETLIOZ Indication HETLIOZ is indicated for the treatment of Non 24 Hour Sleep Wake Disorder (Non 24).
Combined R D and G A includes $1.4M in noncash stock based compensation 5.Cash, cash equivalents and marketable securities 24 Financials Full Year 2014 Guidance FY 14 Operating Expenses $110M $120M 1H 14 Operating Expenses $67M $72M 2H 14 Operating Expenses $43M $48M Year End Cash (1) $20M $30M Before HETLIOZ income FY 2014 Total 2014 operating expenses are expected to be between $110 and $120 million.
Composition of Matter Patent 2. Assumes full 5 year Hatch Waxman extension in US 3. Eligible for up to 10 years commercial exclusivity in the EU 4. Composition of Matter plus 5 year Hatch Waxman extension Approval + 10 Years (3) 23 Financial Results First Quarter 2014 Licensing Agreement (1) $7.5M Royalty Revenue $1.7M Revenue $9.1M Research Development (2)(4) $7.3M General Administrative (3)(4) $27.9M Intangible Asset Amortization $0.6M Operating Expense $35.7M Net Loss ($26.5M) Cash (5) $100.4M Q1 2014 1.Licensing agreement of $7.5M reflects the period amortization of the $200M upfront payment from Novartis for Fanapt US/Canada rights 2.Includes a $2 million HETLIOZ approval milestone 3.Include $15 million for the Non 24 Awareness Campaign 4.
Composition of Matter Patent 2. Assumes full 5 year Hatch Waxman extension in US 3. Published but not issued, Expiry times listed as expected based on filing date 1 3 3 3 2 VLY 686 NK 1R Antagonist 19 VLY 686 Treatment Resistant Pruritus VLY 686 for Treatment Resistant Pruritus in Atopic Dermatitis Phase II Study initiated in Q4 2013 20 Sites / Enrollment Two sites in Germany Subjects = 68 (enrollment ongoing) Design Atopic dermatitis patients with chronic pruritus refractory to conventional treatments Randomized, double masked, placebo controlled 4 week treatment duration followed by a 2 week washout period Endpoints Primary Pruritus intensity (VAS): change from baseline Evaluation of decrease on VRS (Item Itch of the PGA Likert scale) from baseline Fanapt (iloperidone) An atypical antipsychotic agent for the treatment of schizophrenia in adults 21 Fanapt Oral Formulation Status US Canada Rest of World Approvals / Partners Mexico Probiomed Israel Megapharm EMA application to be re filed after maintenance study is concluded Vanda has full commercial rights for Fanapt outside of the US and Canada Partnered with Novartis 2010 US launch for schizophrenia in adults $7M royalty on 2013 US sales $1.7M royalty on Q1 14 US sales 22 Other Programs Intellectual Property United States Europe VLY 686 (1) 2024 (2029) (2) Approval + 10 Years (3) Fanapt Oral 2016 (4) 1.
Wholesale acquisition cost Mix of Commercial and Government Payors Financial Assistance Programs in place 2014 WAC (1) is $84,231 per year HETLIOZ U.S. Launch Initiatives 11 Non 24 Awareness Case Management Significant efforts in the first half of 2014 to increase Non 24 awareness Focus in the second half of 2014 is to help patients obtain proper diagnosis and treatment Advocacy Radio Web TV Education materials to people who opt in Case management through health educators Patient Directed Physicians Program Non 24 Awareness Campaign 9,500 individuals from across the US have responded and Opted In to learn more about Non 24 12 5,900 ~60% 3,600 ~40% Likely Patients Friends Family/Other HETLIOZ US Launch: June 4, 2014 Update 13 Majority of new prescriptions come from patients who have previously responded to the Non 24 Awareness Campaign Most payors require prior authorization Formulary decisions expected over the next 6 months Encouraging uptake to date Prescriptions Written: Over 220 Prescriptions sent to Specialty Pharmacy: Over 110 68 are clinical study patients over 152 are new patients More than half have been filled dispensed to date Benefits investigation completed HETLIOZ Launch Campaign 14 Its time to treat Non 24 Its time for HETLIOZ HETLIOZ EMA Filing HETLIOZ Marketing Authorization Application (MAA) for the treatment of Non 24 was accepted for review under Centralised Procedure in June 2014 HETLIOZ has been granted orphan drug designation for the treatment of Non 24 in the totally blind by the European Commission 15 HETLIOZ Pediatric Non 24 16 Clinical protocols under development for further EMA and FDA discussions Pediatric suspension formulation development is underway HETLIOZ Smith Magenis Syndrome (SMS) 17 Genetic disorder due to chromosome 17p abnormality Developmental defects, sleep and behavior problems Inversion of circadian rhythm reported SMS observational study underway to better characterize the disorder HETLIOZ Intellectual Property 18 Tasimelteon 2017 (2022) United States Expiry Date Tasimelteon Synthesis 2026 Tasimelteon Dose Range 2027 (if issued) Method to Treat in Non 24 2033 (if issued) Method to Diagnose in Non 24 2033 (if issued) Patent ID US7,754,902 Family PCT/US2007/069411 PCT/US2013/023312 PCT/US2013/023315 US5,856,529 1.
The risk of adverse reactions may be greater in elderly ( 65 years) patients than younger patients because exposure to HETLIOZ is increased by approximately 2 fold compared with younger patients See full prescribing information at www.HETLIOZ.com 9 HETLIOZ Solutions A hub to address patient and physician needs Patient Support Non 24 disease awareness HETLIOZ information Insurance benefits verification Financial assistance programs Prescription follow up support HETLIOZ prescribing information Prescription intake processing Triage to specialty pharmacy Physician Support HETLIOZ Access Reimbursement Principle: Ensure patient access to HETLIOZ 10 Programs for co pay assistance Foundation support Patient assistance programs Key Facts: 1.
Vanda estimate 2. EU prevalence reflects EU 28 member states 6 Non 24 Overview 2014 National Non 24 Awareness Campaign initiated (January 2014) FDA approval (1/31/14) HETLIOZ commercial launch (4/21/14) 9,500 individuals have responded to the Non 24 Awareness Campaign in the US (6/3/14) EMA accepts HETLIOZ Non 24 MAA filing for review (6/3/14) Significant 2014 HETLIOZ Milestones 7 HETLIOZ First FDA Approved Treatment for Non 24 FDA Approval: January 31, 2014 US Commercial Launch: April 21, 2014 8 Indication: HETLIOZ (tasimelteon) 20mg is indicated for the treatment of Non 24 Hour Sleep Wake Disorder (Non 24) Common Adverse Events: The most common adverse reactions (incidence 5% and at least twice as high on HETLIOZ than on placebo) were headache, increased alanine aminotransferase, nightmares or unusual dreams, upper respiratory or urinary tract infection.
Treatment Resistant Pruritus in Atopic Dermatitis 2. Marketed in the US by Novartis EU Non 24 Vanda Product Pipeline 5 Non 24 is a circadian rhythm disorder that primarily affects the totally blind Results in misalignment of the sleep wake cycle to the 24 hour day Causes serve and chronic impairment in social and occupational functioning An estimated 80K Americans suffer from Non 24 (1) ; EU prevalence is estimated at 130K (1)(2) 1.
There can be no assurance that the actual results or developments anticipated by Vanda will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on Vanda. Therefore, no assurance can be given that the outcomes stated in such forward looking statements and estimates will be achieved. CNS Specialty Company Focused on unmet medical needs Proven Track Record of Development and Drug Approvals Vanda Overview HETLIOZ FDA approved treatment for Non 24 Hour Sleep Wake Disorder (Non 24) April 2014 commercial launch in US full rest of world rights Additional novel commercial and clinical stage programs 3 Phase I Phase II Phase III NDA / MAA US Schizophrenia (2) US Non 24 Fanapt HETLIOZ Market EU Schizophrenia Pruritus (1) VLY 686 4 1.
Forward looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. Important factors that could cause actual results to differ materially from those reflected in the company's forward looking statements include, among others: Vanda's ability to successfully commercialize HETLIOZ (tasimelteon) for the treatment of Non 24 Hour Sleep Wake Disorder ( Non 24 ) in the U.S.; uncertainty as to the market awareness of Non 24 and the market acceptance of HETLIOZ ; Vanda s dependence on third party manufacturers to manufacture HETLIOZ in sufficient quantities and quality; Vanda s limited sales and marketing infrastructure; the regulatory status of tasimelteon in Europe; Vanda s ability to obtain the capital necessary to fund its research and development or commercial activities; Vanda s loss of rights to develop and commercialize its products under its license and sublicense agreements; the failure to obtain, or any delay in obtaining, regulatory approval for Vanda s products, particularly HETLIOZ outside the U.S., or to comply with ongoing regulatory requirements; the extent and effectiveness of the development, sales and marketing and distribution support Fanapt receives; Vanda s inability to successfully commercialize HETLIOZ globally or Fanapt outside of the U.S. and Canada; a failure of Vanda s products to be demonstrably safe and effective; Vanda s expectations regarding trends with respect to its revenues, costs, expenses and liabilities; Vanda s failure to identify or obtain rights to new products; a loss of any of Vanda s key scientists or management personnel; limitations on Vanda s ability to utilize some or all of its prior net operating losses and orphan drug and research and development credits; the costs and effects of potential litigation; losses incurred from product liability claims made against Vanda and other factors that are described in the Risk Factors and Management's Discussion and Analysis of Financial Condition and Results of Operations sections of Vanda's annual report for the fiscal year ended December 31, 2013 and quarterly report for the quarter ended March 31, 2014, which are on file with the SEC and available on the SEC's website at www.sec.gov .
The information in this presentation is provided only as of the date of this presentation, and Vanda undertakes no obligation, and specifically declines any obligation, to update or revise publicly any forward looking statements, whether as a result of new information, future events or otherwise. Various statements in this presentation, including, but not limited to, Vanda s financial guidance for 2014, are forward looking statements under the securities laws.