 XenoPort, Inc. Investor Presentation October 2014 © Copyright 2014 XenoPort, Inc. All rights reserved. NASDAQ:XNPT Exhibit 99.1 |
 These slides and the accompanying oral presentation by XenoPort, Inc. contain forward-looking statements that involve risks and uncertainties, including statements relating to the commercial opportunity and value proposition for HORIZANT; potential future sales and commercialization activity for HORIZANT and REGNITE; the planned development of HORIZANT with the NIAAA for AUD; the planned development of AP by Reckitt; the XP23829 clinical development program, including the initiation or conduct of current or potential future clinical trials and regulatory submissions and the timing thereof; expected patent coverage; and the therapeutic and commercial potential of XP23829. XenoPort can give no assurance with respect to these statements, and we assume no obligation to update them. For detailed information about the risks and uncertainties that could cause actual results to differ materially from those implied by, or anticipated in, these forward-looking statements, please refer to the Risk Factors section of our Quarterly Report on Form 10-Q for the quarter ended June 30, 2014 and filed with the SEC. October 2014 Investor Slide Presentation 2 Safe Harbor Language |
 A commercial-stage biopharmaceutical company with an internally discovered pipeline focused on CNS and dermatology indications Growing revenues from HORIZANT sales from 2 FDA-approved indications. New indication (alcohol use disorder) will enter potential registration trial in 1H 2015 in collaboration with NIAAA Differentiated MMF prodrug (XP23829) currently in Phase 2 trial for psoriasis with results expected in 3Q 2015. Plans to enter Phase 3 in psoriasis and/or relapsing forms of MS in 2016 Preparing to enter Phase 3 development in advanced Parkinson’s disease (XP21279) and Phase 2 in alcohol use disorder (arbaclofen placarbil) October 2014 Investor Slide Presentation 3 XenoPort Overview |
 October 2014 Investor Slide Presentation 4 GABAPENTIN ENACARBIL Restless Legs Syndrome (RLS) – U.S. Restless Legs Syndrome – Japan Postherpetic Neuralgia (PHN) – U.S. Alcohol Use Disorder (AUD)– U.S. XP23829 Psoriasis Relapsing Forms of MS ARBACLOFEN PLACARBIL (AP) AUD XP21279 Advanced Parkinson’s Disease( (Pending resources), ) PHASE 1 PHASE 2 PHASE 3 NDA FILED MARKETED PRECLINICAL PARTNER XenoPort Pipeline |
 HORIZANT net sales increased 66% in 2Q vs. 1Q 2014 to $4.9 million $124.9 million of cash, cash equivalents and short- term investments at 6/30/14 $5.0 million in non-dilutive cash in 3Q 2014 associated with AP licensing agreement No debt 5 Investor Slide Presentation October 2014 Financials |
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 Approved for moderate-to-severe primary RLS in adults in April 2011 Approved for the management of PHN in adults in June 2012 XenoPort promotional efforts began in June 2013 6 Orange Book listed patents with expiry dates from 2022 - 2029 • Patent term extension requested for composition-of-matter patent from 2022 to 2025 October 2014 Investor Slide Presentation 7 Please review the full prescribing and safety information for HORIZANT. The most common adverse reactions of HORIZANT in RLS patients: somnolence/sedation and dizziness, and in PHN patients: somnolence, dizziness and headache. HORIZANT: XenoPort’s First Commercial Product |
 Drug class: alpha-2-delta ligand (gabapentin, pregabalin) Actively transported prodrug of gabapentin • Addresses pharmacokinetic deficiencies of gabapentin Only approved extended-release alpha-2-delta product Not interchangeable with other gabapentin products October 2014 Investor Slide Presentation 8 Please review the full prescribing and safety information for HORIZANT. The most common adverse reactions of HORIZANT in RLS patients: somnolence/sedation and dizziness, and in PHN patients: somnolence, dizziness and headache. HORIZANT Different by Design |
 Over 5 million U.S. adults suffer from moderate-to-severe primary RLS Widespread use of dopamine agonists Growing awareness of issues related to dopamine agonist use in treatment of RLS • New treatment guidelines October 2014 Investor Slide Presentation 9 Sources: RLS Prevelance-NINDs, NIH, Sleep Medicine, Volume 14, No. 7 , 2013, Mayo Clinic Proceedings, Volume 88, No. 9, 2013, Sleep, Vol. 35, No. 8, 2012 Moderate-to-Severe Primary RLS Market Opportunity in U.S. |
 First and only non-dopamine agonist approved for the treatment of moderate-to- severe primary RLS in adults Proven effective in relieving RLS symptoms (clinical trial data) Convenient once-a-day dosing No titration required No evidence of augmentation, rebound or impulse control disorders* Recommended as a first-line treatment in recently published treatment guidelines October 2014 *In two 12-week clinical trials, patients taking HORIZANT showed no evidence of symptom augmentation. The duration of these trials may not have been sufficient to adequately assess symptom augmentation. Investor Slide Presentation 10 Please review the full prescribing and safety information for HORIZANT. The most common adverse reactions of HORIZANT in RLS patients: somnolence/sedation and dizziness, and in PHN patients: somnolence, dizziness and headache. HORIZANT Attributes for RLS |
  Results from damage that occurs to the peripheral nerve fibers during a shingles outbreak Pain associated with PHN can be very intense About 200,000 patients suffer from PHN in the U.S. Clear unmet medical need • ~30% of patients receive >50% reduction in PHN pain with gabapentin, the most widely used agent to treat PHN October 2014 Sources: Decision Resources, Inc. 2010, Neurontin Product Label Investor Slide Presentation 11 Postherpetic Neuralgia (PHN) |
 Simple 4-day titration Efficacy as early as one week Pharmacokinetic differentiation • High bioavailability (75%) • Sustained 24-hour gabapentin blood levels Pivotal trial showed 42% of PHN patients experienced >50% reduction in pain intensity score from baseline Pain relief over 24 hours October 2014 Please review the full prescribing and safety information for HORIZANT. The most common adverse reactions of HORIZANT in RLS patients: somnolence/sedation and dizziness, and in PHN patients: somnolence, dizziness and headache. Investor Slide Presentation 12 1. Adapted from Lal R, et al. J Clin Pharmacol. 2013;53(1):29-40 HORIZANT Attributes for PHN |
 Initial Plan Upon Return of Product Rights • Establish responsiveness quickly and efficiently • Focus on specialists and high prescribing PCPs of RLS and PHN drugs • Personal promotion and marketing efforts focused in ~ 40 territories XenoPort Initial Plan: Sales specialists called on <10% of the potential market October 2014 Investor Slide Presentation 13 Initial Plan Current Plan • Expanded to additional ~25 new territories • Trained and in field HORIZANT Commercialization |
 October 2014 Investor Slide Presentation 14 Source: SHA, PHAST 2014 Previous Partner’s Sales Team of ~300-500 XenoPort Sales Team of ~40 Approved Dose: RLS = 1 tablet per day; PHN = 2 tablets per day Currently >85% of prescribed tablets from XenoPort territories XenoPort Launch HORIZANT Dispensed Tablets per Month |
 Source: SHA, PHAST 2014 XenoPort Launch & First Promotion of PHN Indication Investor Slide Presentation October 2014 15 XenoPort Launch & First Promotion of PHN Indication Prescriptions by Specialty % Prescriptions 1200 mg/day* *Source: Healthcare Analytics, a Symphony Health Solutions Company The information attributed to Source Healthcare Analytics herein is provided as is, and Source Healthcare Analytics, LLC makes no representation and/or warranty of any kind, including but not limited to the accuracy and/or completeness of such information. Source Healthcare Analytics is credited as a source of certain data only. The attribution of Source Healthcare Analytics as the source of such data shall not be construed as an endorsement by Source Healthcare Analytics of the views, opinions or findings expressed, shared or otherwise published herein. HORIZANT Prescribers and Daily Dose |
 Randomized, double-blind, placebo-controlled trial of the safety and efficacy of HORIZANT in patients who have AUD XenoPort to supply clinical trial material NIAAA will conduct and pay all other expenses associated with proposed clinical trial • Six-month treatment duration; Enrolling approximately 350 patients; expect initiation in 1H 2015 XenoPort and NIAAA to meet with FDA to discuss possibility of utilizing the results of this trial as the basis for a potential supplemental new drug application (sNDA) submission for HORIZANT 16 October 2014 Investor Slide Presentation Clinical Trial Agreement “Scientists at XenoPort designed gabapentin enacarbil extended-release tablets to address certain limitations of drug levels in the body, which may make it a more attractive treatment option for people with AUD.” - NIAAA press release 9/9/14 XenoPort and NIAAA Clinical Trial Agreement |
    XP23829 for Potential Treatment of Psoriasis and/or Relapsing Forms of MS |
 FUMADERM mixture of dimethylfumarate (DMF) and monoethyl fumarate salts • Approved in 1990s and widely used for the treatment of psoriasis in Germany TECFIDERA (dimethylfumarate) • Approved in March 2013 in the U.S. for the treatment of relapsing forms of MS • Approved in February 2014 in the EU for relapsing-remitting MS • 2Q 2014 TECFIDERA revenues were $700 million ($585 million in U.S.; $115 million in sales outside the U.S.) XP23829 has a novel chemical structure that produces monomethylfumarate (MMF), the same active metabolite as dimethylfumarate October 2014 Investor Slide Presentation 18 Background: Fumaric Acid Ester Products |
 October 2014 Investor Slide Presentation 19 XP23829 DMF MMF MMF Promoiety Methanol + + Esterases XP23829 and DMF produce the same active metabolite (MMF) in the body DMF and XP23829 are Prodrugs of MMF |
 Lower incidence and/or less severe GI side effects and flushing • Improved compliance; fewer treatment failures Onset and/or magnitude of efficacy • Earlier onset of immunomodulation Dosing frequency • QD rather than BID (TECFIDERA) or TID (FUMADERM)* Indication • TECFIDERA and FUMADERM not approved for psoriasis in the U.S. October 2014 Investor Slide Presentation 20 *QD: once daily; BID: twice daily; TID: three times daily. Potential XP23829 Advantages and Areas of Differentiation |
 Completed preclinical PK and safety studies including 13-week toxicology studies in 3 animal species. Studies included DMF comparison arms • Demonstrated less skin and GI irritation compared to DMF • No adverse findings that were not observed with DMF Demonstrated efficacy in animal models of MS and psoriasis Completed three Phase 1 trials establishing human PK, metabolites and disposition and comparison of PK to TECFIDERA • XP23829 produced total MMF exposure in blood similar to TECFIDERA Demonstrated known pharmacodynamic effects on immune blood cells with once-a-day dosing in humans Selected novel delayed and extended-release formulation for Phase 2/3 October 2014 Investor Slide Presentation 21 XenoPort: A Leader in Development of 2 nd Generation MMF Prodrugs |
 October 2014 Investor Slide Presentation 22 • pH-independent delayed-release mechanism to reduce gastric irritation and food effects on pharmacokinetics • Releases XP23829 over 8-10 hours avoiding high local concentrations in upper small intestine, lower Cmax and extended blood levels of MMF Differentiation from TECFIDERA/FUMADERM In Vitro Dissolution XP23829 in a Novel Formulation to Potentially Provide Differentiation |
 XP23829 Intellectual Property October 2014 Investor Slide Presentation 23 • Three issued U.S. patents (US8148414, US8778991 and US8785443) including claim covering composition-of-matter of XP23829 (expiration date 2029) • 17 filed patent application families with pending claims including: • Specific MMF prodrug compositions and their uses • Crystalline forms of XP23829 • Oral dosage forms of MMF prodrugs* • Methods of treatment with MMF prodrugs* • Methods of selecting and/or administering MMF prodrugs* to reduce side effects • Applications filed broadly in major pharmaceutical markets * “MMF prodrug” may include XP23829, DMF and certain other MMF prodrug molecules |
 Conducting Phase 2 psoriasis study to assess the effect of dose and dosing regimen on efficacy, tolerability, safety and immune cell modulation of XP23829 • Trial initiated June 2014 • Top-line results expected in 3Q 2015 • Optimal dose(s) expected to translate to relapsing forms of MS, based on TECFIDERA precedent Preclinical studies and manufacturing scale-up are underway to allow Phase 3 study in 2016 Clear precedent for psoriasis Phase 3 program • Two Phase 3 trials, at least one demonstrating maintenance of efficacy at one year Discussions ongoing with FDA on potential abbreviated Phase 3 program for relapsing form of MS 24 October 2014 Investor Slide Presentation XP23829 Current Development Plan |
 Study Design Randomized, double-blind, multicenter, parallel group, placebo-controlled, dose-finding efficacy and safety study in subjects with moderate-to-severe chronic plaque-type psoriasis Number of Sites ~35 sites in United States Number of Subjects ~200 randomized 1:1:1:1 Primary Endpoint The percent change in Psoriasis Area and Severity Index (PASI) score from Baseline (12 weeks) October 2014 Investor Slide Presentation 25 400 mg BID Placebo 800 mg QD 400 mg QD Screening/ Washout Week -4 Week 0 Week 3 Week 12 Week 16 Post-Treatment Follow-up Titration Maintenance Phase XP23829 Phase 2 Clinical Trial in Psoriasis Patients |
 October 2014 Investor Slide Presentation 26 Phase 2 Psoriasis Study Biogen Press Release 2004 Langner, J Am Acad Dermatol 2005 Week 12 Phase 2 Relapsing Forms of MS Study Kappos, Lancet 2008 Similar Dose Response for TECFIDERA in Psoriasis & Relapsing Forms of MS |
 October 2014 Investor Slide Presentation 27 • Worldwide psoriasis prevalence rates vary from 0.6% to 4.8% Epidemiology • In the U.S., it is estimated that direct costs on health care approach ~$7B • Psoriasis affects the daily lives of patients; studies estimate 60% of psoriasis patients missed an average of 26 days of work per year due to their illness Economic Burden Sources: UpToDate, BioMedTracker, National Psoriasis Foundation, Horn EJ, et al 2007 J Am Acad Dermatol. 57:963-71; Fowler et al- 2008 J of Am Acad Derm 3 Clin Exp Rheumatol 2002; 20 (Suppl. 28): S27-S33 Psoriasis is Prevalent, Reduces Quality of Life and is an Economic Burden to Health Systems + In the U.S., estimates of psoriasis prevalence range from 2.5-3.5% + Epidemiological studies of prevalence in the EU range from 1-3% + Moderate and severe psoriasis represent 35% and 12% of all patients, respectively |
 October 2014 Investor Slide Presentation 28 * Assumes no arthritic involvement Sources: Back Bay Physician Interviews, DataMonitor 2013 Treatment Flow and Physician Quantitative Survey U.S. • Orals have 40-50% market share as 1 st and 2 nd line Germany • Orals have 80-90% market share as 1 st line • FUMADERM has highest market share Oral Therapy Market Share for Moderate and Severe Psoriasis Differs in U.S. and Germany |
 October 2014 Investor Slide Presentation 29 Safety/Tolerability Unfavorable Favorable Qualitative Positioning Efficacy similar to FUMADERM (PASI 75 = 40-50%) with favorable tolerability and long-term safety Desired XP23829 Profile Desired Product Positioning for XP23829 in Moderate-to-Severe Chronic Plaque-Type Psoriasis |
 October 2014 Investor Slide Presentation 30 2Q 2014 Sales U.S. Ex-U.S. $585M $115M $91M $27M $284M $322M Source: Symphony Health Solutions, PHAST Prescription Monthly, April 2013 – August 2014 1% market share is ~$100M in annual U.S. product sales at current prices Oral Treatments for Relapsing Forms of MS |
    FUTURE OPPORTUNITIES XP21279 for Parkinson’s Disease AP for Alcohol Use Disorder |
       October 2014 Investor Slide Presentation 32 C avg C avg Threshold for “off-time” SINEMET SINEMET Dyskinesia Threshold XP21279 XP21279 A dose of XP21279 resulting in a higher Cavg concentration could reduce “off” time without increasing dyskinesias High peak-trough ratios of levodopa concentrations result from SINEMET’s inherent PK properties Therapeutic Window XP21279: Designed to Keep Levodopa Exposure in the Target Therapeutic Window |
 Achieved levodopa levels with XP21279/Carbidopa tablet within the target therapeutic window Minimal Fluctuation in Levodopa Blood Levels October 2014 Investor Slide Presentation 33 XP21279 Phase 2 Study in Advanced Parkinson’s Patients |
 A single successful pivotal 12-week Phase 3 study comparing optimized doses of XP21279 to SINEMET could support approval for treatment of patients with advanced Parkinson’s disease with motor fluctuations • Data comparing to SINEMET would be in product label Gained agreement on long-term safety database size and duration of exposures A single successful 3-month placebo-controlled study could support approval of XP21279 in patients with early Parkinson’s disease • Based on 505(b)(2) cross-reference to SINEMET Program ready to enter Phase 3 development with further funding October 2014 Investor Slide Presentation 34 FDA Agreements For Potential XP21279 Phase 3 Program |
 Exclusive world-wide rights granted to Reckitt Benckiser Pharmaceuticals • Proven leader in commercialization of addiction treatments, e.g., SUBOXONE Planned initial development focus: AUD $20 million up-front plus $5 million for material transfer Up to $70 million in development and regulatory milestones Up to $50 million in commercial milestones Tiered double-digit royalty payments up to mid-teens on a percentage basis on potential future net sales in the U.S. High single-digit royalty payments on potential future net sales outside the U.S. October 2014 Investor Slide Presentation 35 AP Agreement with Reckitt Benckiser Pharmaceuticals |
 HORIZANT • Growing net sales in U.S. and royalties in Japan • Additional ex-U.S. partnership(s) • FDA agreement of AUD development plan • Initiation of AUD pivotal study in 1H 2015 XP23829 • Top-line results of Phase 2 psoriasis study expected in 3Q 2015 • Completion of non-clinical studies to support Phase 3 in 2016 • FDA agreement on Phase 3 plan for psoriasis and/or relapsing forms of MS XP21279 • Partner and/or initiate Phase 3 development activities as resources permit AP • Expected initiation of Phase 2 study in AUD by partner Reckitt Benckiser Pharmaceuticals October 2014 Investor Slide Presentation 36 XenoPort Anticipated Milestones |
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