JEFFERIES 2015 GLOBAL HEALTHCARE CONFERENCE JUNE 4, 2015 VINCENT J. ANGOTTI, CHIEF OPERATING OFFICER

Transcription

1 JEFFERIES 2015 GLOBAL HEALTHCARE CONFERENCE JUNE 4, 2015 VINCENT J. ANGOTTI, CHIEF OPERATING OFFICER COPYRIGHT 2015 XENOPORT, INC. ALL RIGHTS RESERVED.

2 SAFE HARBOR LANGUAGE 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, promotional efforts and value proposition for HORIZANT (gabapentin enacarbil) Extended-Release Tablets, including XenoPort s planned sales force expansion and the timing thereof; potential future revenue, prescription growth and commercialization and partnering activities for HORIZANT; XenoPort s 2015 revenue guidance and other 2015 goals; the planned clinical development of HORIZANT by the NIAAA, including the initiation, conduct and results of the NIAAA's proposed clinical trial and the timing thereof; XenoPort s beliefs regarding the design of NIAAA s proposed pivotal clinical trial and its potential to support a potential supplemental NDA for HORIZANT as a potential treatment for AUD; the planned development of AP by Indivior PLC (formerly Reckitt Benckiser Pharmaceuticals) and the timing thereof; the XP23829 clinical development program, including XenoPort s development strategy as well as XenoPort's expectations to obtain top-line Phase 2 XP23829 study results by the end of the third quarter of 2015 and to accelerate and advance XP23829 into Phase 3 development; the initiation or conduct of current or potential future XP23829 clinical trials and related regulatory interactions and the timing thereof; partnering activities for XP23829; expected patent coverage; and the therapeutic and commercial potential of HORIZANT and XenoPort s product candidates. 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 March 31, 2015 and filed with the SEC. PAGE 2 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

3 XENOPORT BUSINESS DRIVERS Growing, commercial business Pipeline of product candidates Unique prodrug NCEs with long patent lives PAGE 3 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

4 POTENTIAL FOR SIGNIFICANT NEW OPPORTUNITIES FROM DEVELOPMENT PIPELINE PRODUCT/INDICATION DEVELOPMENT STAGE PARTNER GABAPENTIN ENACARBIL Alcohol Use Disorder (AUD) U.S. NIAAA Planning Pivotal Trial 2Q 15 XP23829 Psoriasis Relapsing Forms of MS Phase 2 Ongoing May Enter Phase 3 - Pending Psoriasis Data / Toxicology Studies XP21279 Parkinson s Disease Potential Phase 3 Development (Pending Resources) ARBACLOFEN PLACARBIL (AP) AUD Indivior Planning Phase 2 Development PAGE 4 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

5 XP23829 FOR POTENTIAL TREATMENT OF PSORIASIS AND RELAPSING FORMS OF MS PAGE 5 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

6 APPROVED FUMARIC ACID ESTER PRODUCTS FUMADERM Mixture of fumaric acid esters Available in Germany since 1994 for treatment of psoriasis TECFIDERA (dimethyl fumarate) A formulation of dimethyl fumarate (DMF) 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 Shown effective in 2 clinical trials in psoriasis patients PAGE 6 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

7 XP23829 A FUMARIC ACID ESTER COMPOUND Releases the same active metabolite (MMF) in the body as TECFIDERA and FUMADERM + Esterases XP23829 MMF Promoiety + DMF MMF Methanol PAGE 7 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

8 STATUS OF PRECLINICAL AND PHASE 1 TRIALS Completed preclinical PK and safety studies Including 13-week toxicology studies in 3 animal species with DMF comparison arms Demonstrated less skin and GI irritation compared to DMF No adverse findings that were not observed with DMF Completed three Phase 1 trials Established human PK, metabolites and disposition Compared PK/PD to TECFIDERA (dimethyl fumarate) Confirmed total MMF exposure in blood similar to TECFIDERA Demonstrated known pharmacodynamic effects on immune blood cells with once-a-day dosing in humans Ongoing chronic and reproductive toxicology studies Studies Include DMF control arm Data expected to be available in 2H 2015 in time for EOP2 meeting with FDA PAGE 8 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

9 POTENTIAL XP23829 ADVANTAGES Lower incidence and/or less severe GI side effects and flushing Onset and/or magnitude of efficacy Dosing frequency QD rather than BID (TECFIDERA) or TID (FUMADERM) Indication TECFIDERA and FUMADERM not approved for psoriasis in the U.S. PAGE 9 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

10 XP23829 DEVELOPMENT STRATEGY Conduct a Phase 2 psoriasis study to gain valuable knowledge for XP23829 product profile Efficacy Safety and Tolerability (including effects on lymphocytes) Dose and dose regimen (QD vs. BID) Plan to advance into Phase 3 psoriasis development, pending Phase 2 results Psoriasis study results may be used to justify dose-selection and support development strategy in MS Recently gained FDA agreement on key aspects of Phase 3 MS development program Proceed upon obtaining supportive data from chronic and reproductive toxicology studies and Phase 2 psoriasis results A single Phase 3 trial could support potential NDA submission Continuing to seek development strategy input from potential partners PAGE 10 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

11 PSORIASIS RESULTS RELEVANT TO DEVELOPMENT FOR RELAPSING FORMS OF MS TECFIDERA Phase 2 Psoriasis Study MEDIAN PERCENT REDUCTIONS FROM BASELINE PASI TECFIDERA Phase 2 Relapsing Forms of MS Study NUMBER OF NEW GDE LESIONS (WEEK 12-24) 80% 70% 71% % 50% WEEK 12 52% % 30% 20% 31% % 6% 0.5 0% Placebo 120 mg/day 360 mg/day 720 mg/day (n=36) (n=36) (n=36) (n=36) 0 Placebo (N=65) 120 mg/day (N=64) 360 mg/day (N=64) 720 mg/day (N=64) Biogen Press Release 2004 Langner, J Am Acad Dermatol 2005 Kappos, Lancet 2008 PAGE 11 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE

12 XP23829 PHASE 2 CLINICAL TRIAL IN PSORIASIS PATIENTS 800 mg QD 400 mg BID 400 mg QD Screening/ Washout Placebo Post-Treatment Follow-up Week -4 Week 0 Week 3 Week 12 Week 16 Titration Maintenance Phase Study Design Number of Sites Randomized, double-blind, multicenter, parallel group, placebo-controlled, dose-finding efficacy and safety study in subjects with moderate-to-severe chronic plaque-type psoriasis ~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) Top-line results expected by end of 3Q 15 PAGE 12 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

13 PLAQUE PSORIASIS MARKET IS UNDERSERVED Chronic, systemic, inflammatory disease Most prevalent autoimmune disease Afflicts 7.5 million Americans * 1.5 million with moderate-to-severe psoriasis** Treatment options not optimal Topicals Useful in mild cases Oral options have limited efficacy and/or potentially concerning side effects Acitreten, cyclosporine, methotrexate and OTEZLA Injectable biologics are efficacious but come with safety warnings ENBREL, HUMIRA, REMICADE, STELARA 52% of patients are dissatisfied with current treatment * * National Psoriasis Foundation Fact Sheet, as posted 2/23/15 ** The Psoriasis and Psoriatic Arthritis Pocket Guide, National Psoriasis Foundation, 2009 PAGE 13 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

14 DESIRED PRODUCT POSITIONING FOR XP23829 AS ORAL TREATMENT FOR PSORIASIS Qualitative Positioning UNFAVORABLE Efficacy FAVORABLE XP23829 UNFAVORABLE Safety/Tolerability FAVORABLE PAGE 14 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

15 ORAL TREATMENTS FOR RELAPSING FORMS OF MS 50,000 U.S SALES Ex-U.S. 40,000 $2.4 B $0.5 B U.S. TRx 30,000 20,000 $1.2 B $1.3 B 10,000 $0.3 B $0.5 B 0 Mar-09 Apr-09 May-09 Jun-09 Jul-09 Aug-09 Sep-09 Oct-09 Nov-09 Dec-09 Jan-10 Feb-10 Mar-10 Apr-10 May-10 Jun-10 Jul-10 Aug-10 Sep-10 Oct-10 Nov-10 Dec-10 Jan-11 Feb-11 Mar-11 Apr-11 AUBAGIO GILENYA TECFIDERA 1% market share is ~$100M in annual U.S. product sales at current prices Source: Symphony Health Solutions, PHAST Prescription Monthly, April 2013 April 2015 PAGE 15 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

16 XENOPORT S BROAD INTELLECTUAL PROPERTY Three issued U.S. patents Claims covering composition-of-matter of XP23829 (expiration date in 2029) 48 patents issued worldwide, covering: XP23829 and other 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 *May include XP23829, DMF and certain other MMF prodrug molecules PAGE 16 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

17 HORIZANT OUR FIRST APPROVED PRODUCT PAGE 17 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

18 HORIZANT (GABAPENTIN ENACARBIL) EXTENDED- RELEASE TABLETS Approved in U.S. for moderate-tosevere primary RLS in adults in April 2011 Approved in U.S. for the management of PHN in adults in June 2012 XenoPort promotional efforts began in June Orange Book listed patents with expiry dates from 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. PAGE 18 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

19 HORIZANT ATTRIBUTES: MODERATE-TO-SEVERE PRIMARY RLS Proven effective Convenient once-a-day dosing No titration Only non-dopamine agonist approved by FDA No evidence of augmentation, rebound or impulse control disorders* Recommended as a first-line treatment in recently published treatment guidelines *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. PAGE 19 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

20 HORIZANT ATTRIBUTES: MANAGEMENT OF PHN Simple 4-day titration Efficacy as early as one week Pharmacokinetic differentiation High bioavailability (75%) Sustained 24-hour gabapentin blood levels 42% of patients experienced 50% pain score reduction Pain relief over 24 hours STEADY-STREAM GABAPENTIN CONCENTRATIO (µg/ml) 10 5 Predicted Plasma Concentration of Gabapentin After HORIZANT 600 mg BID for PHN 1 Peak to Trough Ratio = TIME (HR) 1. Adapted from Lal R, et al. J Clin Pharmacol. 2013;53(1):29-40 Source: Pivotal trial data 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. PAGE 20 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

21 HORIZANT COMMERCIALIZATION STRATEGY JUNE 2013 Objective: Demonstrate promotional responsiveness quickly and efficiently Personal promotion and marketing efforts focused in ~ 40 territories Focus on specialists and high prescribing PCPs of RLS and PHN drugs OCTOBER 2014 Expanded to ~65 territories Observed 74% increase in prescribed tablets in new territories in 4Q 14 versus 4Q Planned expansion to up to 120 territories by mid-2015 Maintaining focus on specialists and high prescribing PCPs of RLS and PHN drugs PAGE 21 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

22 GROWING HORIZANT SALES Prescribed Tablets HORIZANT Quarterly Net Sales Pre- XP Launch Post- XP Launch $7.00 $6.00 $5.6 $6.6 $ $5.00 $4.9 In Thousands $4.00 $3.00 $2.7 $ $2.00 $1.00 $1.6 $2.0 - Regions with NO XenoPort Promotion $0.00 * 2Q'13 3Q'13 4Q'13 1Q'14 2Q'14 3Q'14 4Q'14 1Q'15 *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. *Reflects XenoPort net sales of HORIZANT following its reacquisition on May 1, PAGE 22 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

23 GROWING HORIZANT SALES 2400 Horizant R4 NRx, RRx, TRx 2, R4W Rxs , Horizant Rolling 4 NRx Horizant Rolling 4 TRx Horizant Rolling 4 RRxs PAGE 23 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

24 GROWING HORIZANT SALES 120,000 Horizant Rolling Average 4 Week Tabs 116, ,000 80,000 60,000 40,000 20,000 - PAGE 24 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

25 EXPANDING HORIZANT OPPORTUNITY CLINICAL TRIAL AGREEMENT HORIZANT clinical trial in patients who have AUD Six-month treatment duration NIAAA plans to enroll ~350 patients Initiation planned for 2Q 15. Results may be available by end of 2016 XenoPort to supply clinical trial material NIAAA plans to conduct trial and pay all other expenses FDA meeting outcome Accepted trial design XenoPort believes trial results may support filing of potential snda for HORIZANT if results are robust and compelling Additional confirmatory evidence from literature or other sources should support the clinical trial findings PAGE 25 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

26 PROOF OF CONCEPT: GABAPENTIN / ALCOHOL DEPENDENCE PAGE 26 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

27 2015 GOALS HORIZANT net sales of $39 to $43 million Initiation of HORIZANT AUD clinical trial by NIAAA in 2Q 15 Top-line data for XP23829 Phase 2 psoriasis clinical trial by the end of 3Q 15 Complete chronic and reproductive toxicology studies for XP23829 and initiate carcinogenicity studies Scale-up manufacturing of XP23829 to support potential Phase 3 Potential End-of-Phase 2 meeting with FDA for XP23829 PAGE 27 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

28 FINANCIALS $191.6 million of cash, cash equivalents and shortand long-term investments at March 31, 2015 Includes $111.4 million net proceeds secured through recent convertible senior debt financing 62.8 million shares outstanding at April 15, 2015 PAGE 28 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

29 THANK YOU VINCENT J. ANGOTTI, CHIEF OPERATING OFFICER COPYRIGHT 2015 XENOPORT, INC. ALL RIGHTS RESERVED.

30 BACK UP SLIDES PAGE 30 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

31 XP23829 IS DIFFERENT BY DESIGN XP23829 has reduced local GI irritation compared to DMF based on XenoPort preclinical studies A ph-independent delayed- and extendedrelease tablet Avoids high local concentrations particularly in stomach/duodenum Potential to increase drug exposure to immune cells found throughout the intestine Reduces C max and extends blood levels of MMF ph=1.2 IN VITRO DISSOLUTION* ph=6.8 *XenoPort data on file 0 HOURS 2 HOURS 4 HOURS 8 HOURS 12 HOURS PAGE 31 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

32 XP23829 POTENTIAL MS DEVELOPMENT PLAN Gained FDA agreement on key aspects of Phase 3 development program Proceed upon obtaining supportive data from chronic and reproductive toxicology studies and Phase 2 psoriasis results A single Phase 3 trial could support potential NDA submission Phase 2 psoriasis study results (efficacy, safety, tolerability) may be used to justify dose-selection and support development strategy Continuing to seek development strategy input from potential partners PAGE 32 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

33 GROWING HORIZANT TABLET GROWTH POST-LAUNCH OF HORIZANT BY XENOPORT Prescribed Tablets Pre- XenoPort Launch Post- XenoPort Launch In Thousands *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. PAGE 33 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015

34 GROWING HORIZANT SALES 120,000 Horizant Rolling Average 4 Week Tabs 116, Horizant R4 NRx, RRx, TRx 2, , , ,000 40,000 R4W Rxs , , May 3-Jun 3-Jul 3-Aug 3-Sep 3-Oct 3-Nov 3-Dec 3-Jan 3-Feb 3-Mar 3-Apr 3-May 3-Jun 3-Jul 3-Aug 3-Sep 3-Oct 3-Nov 3-Dec 3-Jan 3-Feb 3-Mar 3-Apr 3-May 0 24-Jan 24-Feb 24-Mar 24-Apr 24-May 24-Jun 24-Jul 24-Aug 24-Sep 24-Oct 24-Nov 24-Dec 24-Jan 24-Feb 24-Mar 24-Apr 24-May 24-Jun 24-Jul 24-Aug 24-Sep 24-Oct 24-Nov 24-Dec 24-Jan 24-Feb 24-Mar 24-Apr Horizant Rolling 4 NRx Horizant Rolling 4 TRx Horizant Rolling 4 RRxs PAGE 34 JEFFERIES GLOBAL HEALTHCARE CONFERENCE JUNE 2015