jefferies 2015 global healthcare conference ......*source: healthcare analytics, a symphony health...

© COPYRIGHT 2015 XENOPORT, INC. ALL RIGHTS RESERVED.

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

| JEFFERIES GLOBAL HEALTHCARE CONFERENCE | JUNE 2015

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.


Growing, commercial business

Unique prodrug NCEs with long patent lives

Pipeline of product candidates

XENOPORT BUSINESS DRIVERS


PRODUCT/INDICATION DEVELOPMENT STAGE PARTNER

GABAPENTIN ENACARBIL

Alcohol Use Disorder (AUD) – U.S. NIAAA Planning Pivotal Trial – 2Q’15

XP23829

Psoriasis Phase 2 Ongoing

Relapsing Forms of MS 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

POTENTIAL FOR SIGNIFICANT NEW OPPORTUNITIES FROM DEVELOPMENT PIPELINE

http://www.niaaa.nih.gov/

http://www.google.com/url?sa=i&rct=j&q=&esrc=s&frm=1&source=images&cd=&cad=rja&uact=8&ved=0CAcQjRw&url=http://indivior.com/&ei=vXqwVP_2HIOvogSW-4KYBw&bvm=bv.83339334,d.cGU&psig=AFQjCNEOyLTrq3K7u9N3MYx07rWCsoFBIA&ust=1420938291472267


XP23829 FOR POTENTIAL TREATMENT OF PSORIASIS AND RELAPSING FORMS OF MS


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


XP23829 A FUMARIC ACID ESTER COMPOUND

Releases the same active metabolite (MMF) in the body as TECFIDERA and FUMADERM

XP23829

DMF

MMF

MMF

Promoiety

Methanol

+

+

Esterases


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


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.


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


PSORIASIS RESULTS RELEVANT TO DEVELOPMENT FOR RELAPSING FORMS OF MS

11

TECFIDERA Phase 2 Psoriasis Study

6%

31%

52%

71%

0%

10%

20%

30%

40%

50%

60%

70%

80%

Placebo 120 mg/Day 360 mg/Day 720 mg/Day

MEDIAN PERCENT REDUCTIONS FROM BASELINE PASI

(n=36) (n=36) (n=36) (n=36)

Biogen Press Release 2004 Langner, J Am Acad Dermatol 2005

WEEK 12

TECFIDERA Phase 2 Relapsing Forms of MS Study

4.5

3.3 3.1

1.4

0

0.5

1

1.5

2

2.5

3

3.5

4

4.5

5

Placebo(N=65)

120 mg/day(N=64)

360 mg/day(N=64)

720 mg/day(N=64)

NUMBER OF NEW GDE LESIONS (WEEK 12-24)

Kappos, Lancet 2008


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)

XP23829 PHASE 2 CLINICAL TRIAL IN PSORIASIS PATIENTS

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

Top-line results expected by end of 3Q’15


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


Safety/Tolerability

Effic

acy

UN

FAVO

RAB

LE

FAVO

RAB

LE

UNFAVORABLE FAVORABLE

Qualitative Positioning

XP23829

DESIRED PRODUCT POSITIONING FOR XP23829 AS ORAL TREATMENT FOR PSORIASIS

http://www.google.com/url?sa=i&rct=j&q=&esrc=s&frm=1&source=images&cd=&cad=rja&uact=8&docid=bdMlmNKuKS-yyM&tbnid=8ms5GoY7LIRgzM:&ved=0CAcQjRw&url=http://contionor.cz/poznejte-svoje-cilove-publikum-a-definujte-svoje-cile/&ei=-GI1VL_oKYrwoASu04CwDg&bvm=bv.76943099,d.cGU&psig=AFQjCNGdL4-FYa3_a4Yu1HMeh8NuXY55eg&ust=1412871299629727


0

10,000

20,000

30,000

40,000

50,000M

ar-0

9Ap

r-09

May

-09

Jun-

09Ju

l-09

Aug-

09Se

p-09

Oct

-09

Nov

-09

Dec

-09

Jan-

10Fe

b-10

Mar

-10

Apr-1

0M

ay-1

0Ju

n-10

Jul-1

0Au

g-10

Sep-

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ct-1

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AUBAGIO GILENYA TECFIDERA

ORAL TREATMENTS FOR RELAPSING FORMS OF MS

U.S

. TR

x

2014 SALES U.S. Ex-U.S.

$2.4 B $0.5 B

$0.3 B $0.5 B

$1.2 B $1.3 B

Source: Symphony Health Solutions, PHAST Prescription Monthly, April 2013 – April 2015

1% market share is ~$100M in annual U.S. product sales at current prices


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


HORIZANT OUR FIRST APPROVED PRODUCT


Approved in U.S. for moderate-to-severe 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 2013

6 Orange Book listed patents with expiry dates from 2022 – 2029

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® (GABAPENTIN ENACARBIL) EXTENDED- RELEASE TABLETS


*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.

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


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.

Source: Pivotal trial data

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

TIME (HR)

0

5

10

0.00 12.10 24.00

Predicted Plasma Concentration of Gabapentin After HORIZANT 600 mg BID

for PHN1

1. Adapted from Lal R, et al. J Clin Pharmacol. 2013;53(1):29-40

STEA

DY-

STR

EAM

G

ABAP

ENTI

N C

ON

CEN

TRAT

IO

(µg/

ml)

Peak to Trough Ratio = 1.5


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’13

2015

Planned expansion to up to 120 territories by mid-2015 Maintaining focus on specialists and high prescribing PCPs of RLS and PHN drugs


$1.6 $2.0

$2.7 $3.0

$4.9

$5.6

$6.6 $6.6

$0.00

$1.00

$2.00

$3.00

$4.00

$5.00

$6.00

$7.00

2Q'13 3Q'13 4Q'13 1Q'14 2Q'14 3Q'14 4Q'14 1Q'15

GROWING HORIZANT SALES

Regions with NO XenoPort Promotion

Prescribed Tablets HORIZANT Quarterly Net Sales

*Reflects XenoPort net sales of HORIZANT following its reacquisition on May 1, 2013.

*

*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.

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50

100

150

200

250

300

350

400

450

500

2011

-06

2011

-08

2011

-10

2011

-12

2012

-02

2012

-04

2012

-06

2012

-08

2012

-10

2012

-12

2013

-02

2013

-04

2013

-06

2013

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2013

-10

2013

-12

2014

-02

2014

-04

2014

-06

2014

-08

2014

-10

2014

-12

2015

-02

2015

-04

In T

hous

ands

Pre- XP Launch

Post- XP Launch



1,311

2,290

979

0

400

800

1200

1600

2000

2400

R4W

Rxs

Horizant R4 NRx, RRx, TRx

Horizant Rolling 4 NRx Horizant Rolling 4 TRx Horizant Rolling 4 RRxs



-

20,000

40,000

60,000

80,000

100,000

120,000 Horizant Rolling Average 4 Week Tabs

116,526


EXPANDING HORIZANT OPPORTUNITY

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

CLINICAL TRIAL AGREEMENT

http://www.niaaa.nih.gov/


PROOF OF CONCEPT: GABAPENTIN / ALCOHOL DEPENDENCE


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


FINANCIALS

$191.6 million of cash, cash equivalents and short- and 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


BACK UP SLIDES


XP23829 IS DIFFERENT BY DESIGN

XP23829 has reduced local GI irritation compared to DMF based on XenoPort preclinical studies A pH-independent delayed- and extended-release tablet

• Avoids high local concentrations particularly in stomach/duodenum

• Potential to increase drug exposure to immune cells found throughout the intestine

• Reduces Cmax and extends blood levels of MMF

IN VITRO DISSOLUTION*

0 HOURS 2 HOURS 8 HOURS 4 HOURS 12 HOURS

*XenoPort data on file

pH=1.2 pH=6.8


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


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50

100

150

200

250

300

350

400

450

500

2011

-06

2011

-07

2011

-08

2011

-09

2011

-10

2011

-11

2011

-12

2012

-01

2012

-02

2012

-03

2012

-04

2012

-05

2012

-06

2012

-07

2012

-08

2012

-09

2012

-10

2012

-11

2012

-12

2013

-01

2013

-02

2013

-03

2013

-04

2013

-05

2013

-06

2013

-07

2013

-08

2013

-09

2013

-10

2013

-11

2013

-12

2014

-01

2014

-02

2014

-03

2014

-04

2014

-05

2014

-06

2014

-07

2014

-08

2014

-09

2014

-10

2014

-11

2014

-12

2015

-01

2015

-02

2015

-03

2015

-04

GROWING HORIZANT TABLET GROWTH POST-LAUNCH OF HORIZANT BY XENOPORT

Prescribed Tablets

*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.

In T

hous

ands

Pre- XenoPort Launch

Post- XenoPort Launch



1,311

2,290

979

0

400

800

1200

1600

2000

2400

24-J

an24

-Feb

24-M

ar24

-Apr

24-M

ay24

-Jun

24-J

ul24

-Aug

24-S

ep24

-Oct

24-N

ov24

-Dec

24-J

an24

-Feb

24-M

ar24

-Apr

24-M

ay24

-Jun

24-J

ul24

-Aug

24-S

ep24

-Oct

24-N

ov24

-Dec

24-J

an24

-Feb

24-M

ar24

-Apr

R4W

Rxs

Horizant R4 NRx, RRx, TRx

Horizant Rolling 4 NRxHorizant Rolling 4 TRxHorizant Rolling 4 RRxs

-

20,000

40,000

60,000

80,000

100,000

120,000

3-M

ay3-

Jun

3-Ju

l3-

Aug

3-Se

p3-

Oct

3-N

ov3-

Dec

3-Ja

n3-

Feb

3-M

ar3-

Apr

3-M

ay3-

Jun

3-Ju

l3-

Aug

3-Se

p3-

Oct

3-N

ov3-

Dec

3-Ja

n3-

Feb

3-M

ar3-

Apr

3-M

ay

Horizant Rolling Average 4 Week Tabs

116,526

jefferies 2015 global healthcare conference ......*source: healthcare analytics, a symphony health...

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