Jefferies Global Healthcare Conference · goal” through partnering ... NIH, Sleep Medicine,...

Jefferies Global

Healthcare Conference Ronald W. Barrett, Ph.D.

Chief Executive Officer

June 4, 2014

© Copyright 2014 XenoPort, Inc. All rights reserved. NASDAQ:XNPT

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 HORZIANT; potential future sales and commercialization

activity for HORIZANT and REGNITE; the XP23829 clinical development

program, including the initiation or conduct of planned 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 March 31, 2014 and filed with the SEC.

June 2014 Jefferies Healthcare Conference 2

Background on XenoPort

Founded in 1999; IPO in 2005

92 full-time employees at December 31, 2013

Developed innovative biology/chemistry platform to improve drug efficacy, tolerability, compliance

Discovered and developed 4 patented mid/late stage or marketed compounds

XP21279

June 2014 3 Jefferies Healthcare Conference

$122.5 million of cash, cash

equivalents and short-term

investments at 3/31/14

Operations expected to be funded

through 2015

Additional $25 million in non-dilutive

cash expected in 2014 associated

with licensing agreement

announced 5/15/14*

No debt

All financial data as of March 31, 2014.

*Subject to antitrust clearance of transaction

4 June 2014 Jefferies Healthcare Conference

5

Build significant value for HORIZANT® (gabapentin

enacarbil) Extended-Release Tablets

• Potential revenue through partnership or through

achieving profitability of commercial effort

• Provide funding source to capture the most value for

XP23829

Advance development of XP23829 as potential

treatment for psoriasis and/or relapsing forms of multiple

sclerosis

Monetize other assets and create “additional shots on

goal” through partnering

Jefferies Health Care Conference June 2014

Exclusive world-wide rights granted to Reckitt Benckiser Pharmaceuticals (RBP) announced on May 15, 20141

Initial development focus: Alcohol Use Disorder

$20 million up-front plus $5 million on technology transfer completion

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 sales outside the U.S.

1 Subject to antitrust clearance of transaction


RBP is the world’s largest addiction treatment business

• $1.3 billion (USD) 2013 sales

Long-standing track record with patients and physicians

• 5 million patients treated with Suboxone therapy (treats opioid addiction)

• ~ 27,500 certified prescribing physicians

Well funded for future development of arbaclofen placarbil

• ~ $720M in 2013 Operating Profit

1 Subject to clearance of transaction through HSR

8 June 2014 Jefferies Healthcare Conference

http://www.google.com/url?sa=i&rct=j&q=&esrc=s&frm=1&source=images&cd=&cad=rja&uact=8&docid=ET_gLUieRZv9FM&tbnid=itUd5P7Z--qTAM:&ved=0CAUQjRw&url=http://www.wkrb13.com/markets/277757/bank-of-america-increases-reckitt-benckiser-group-plc-price-target-to-gbx-5600-rb/&ei=mbZ2U82DK46CogTr14G4Bg&bvm=bv.66917471,d.cGU&psig=AFQjCNGiL3f-x0z5ISZxeQtiGMiAFz29qQ&ust=1400375307331113

http://www.google.com/url?sa=i&rct=j&q=&esrc=s&frm=1&source=images&cd=&cad=rja&uact=8&docid=Td0YHHlvdIRZKM&tbnid=eEAhfrkrD33uaM:&ved=0CAUQjRw&url=http://www.glassdoor.com/Reviews/XenoPort-Reviews-E13720.htm&ei=9rZ2U6rsGcfYoASJrYCgDQ&bvm=bv.66917471,d.cGU&psig=AFQjCNEIeHiSdeSVix0sqTVA6LeWD-T4Gw&ust=1400375408782837

Divider Image Here

(Size image to 4” by 4”)

Discovered and developed by XenoPort for moderate-to-severe primary RLS in adults

U.S. composition-of-matter patent expires 2022

• Patent term extension requested into 2025

FDA approved for moderate-to-severe primary RLS in adults in April 2011

• Launched by GSK in July 2011

FDA approved for the management of PHN in adults in June 2012

• Never launched for the indication by GSK

Re-acquired by XenoPort in May 2013

• Stock-out by GSK in 2013 prior to product return

XenoPort began promotional efforts in June 2013

June 2014 Jefferies Healthcare Conference

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.

10

Gabapentin enacarbil is a member of the alpha-2-delta

ligand class of drugs (gabapentin, pregabalin)

Gabapentin enacarbil is an actively transported prodrug of

gabapentin that addresses the pharmacokinetic

deficiencies of gabapentin

HORIZANT provides dose-proportional exposure to

gabapentin

HORIZANT is the only extended-release alpha-2-delta

product

HORIZANT is not interchangeable with other gabapentin

products because of differing pharmacokinetic profiles




11

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


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

12

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)

• 73% of patients taking HORIZANT 600 mg were “much improved” or “very much improved” on CGI-I Scale at Week 12 vs. 45% of patients on placebo

• Patients taking HORIZANT 600 mg achieved a 41% greater reduction in IRLS score at Week 12 compared to patients on placebo

• Most common AEs were somnolence/sedation and dizziness

Convenient once-a-day dosing

No titration to approved dose

Shows no evidence of augmentation, rebound or impulse control disorders

Recognized in recently published treatment guidelines




13

IRLS study group (IRLSSG) task force recommends that

alpha-2-delta ligands should be considered for first-line

treatment for patients with RLS

WED Foundation revised consensus statement on the

management of RLS recommends alpha-2-delta ligands

should be considered for initial treatment for patients with

RLS

American Academy of Sleep Medicine identified gabapentin

enacarbil as the only alpha-2-delta ligand with high level of

evidence of efficacy for patients with RLS


Sources: Sleep Medicine, Volume 14, No. 7 , 2013,

Mayo Clinic Proceedings, Volume 88, No. 9 , 2013,

Sleep, Vol. 35, No. 8, 2012

14

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


Sources: Decision Resources, Inc. 2010, Neurontin Product Label

15

http://www.google.com/url?sa=i&rct=j&q=&esrc=s&frm=1&source=images&cd=&cad=rja&docid=0xKBRO-5cUHNrM&tbnid=Z1V0YC57Xl1iEM:&ved=0CAUQjRw&url=http://acsh.org/2013/09/shingles-vaccine-continues-to-be-profoundly-under-used/&ei=tDWBUpvKIsKhigKRtIDoDA&bvm=bv.56146854,d.cGE&psig=AFQjCNED7RTDDYd2RLjgOeoL6Ejxw75lyw&ust=1384285906565421

Simple dosing

• Three days at 600 mg QD

• 4th day at approved 600 mg BID dose

Effective at one week

Pharmacokinetic differentiation

• High bioavailability (75%)

• Sustained 24-hr gabapentin blood levels (Peak/Trough = 1.5)

• “Not interchangeable with other gabapentin products” (FDA label)

Pivotal trial showed 42% of PHN patients experienced ≥50% pain

intensity score from baseline

24-hour pain reduction




16

Strategy • Measure responsiveness quickly and efficiently

• Build value in HORIZANT to provide strategic optionality (monetize or grow business)

• Closely monitor results to make sure continued investment is warranted

Tactics • Leverage $40 million and 50 metric tons of active ingredient acquired as part of

settlement

• Implement state-of-the-art promotional tools

• Personal promotion and marketing efforts focused in 40 territories

• XenoPort sales

specialists call on ~10%

of the potential market

• ~40 sales reps vs.

former partner’s ~300


June 2014

63,599

13,506

0

10,000

20,000

30,000

40,000

50,000

60,000

70,000

Ap

r-12

Ma

y-1

2

Jun

-12

Jul-1

2

Au

g-1

2

Se

p-1

2

Oct-

12

Nov-1

2

Dec-1

2

Jan

-13

Fe

b-1

3

Ma

r-13

Ap

r-13

Ma

y-1

3

Jun

-13

Jul-1

3

Au

g-1

3

Se

p-1

3

Oct-

13

Nov-1

3

Dec-1

3

Jan

-14

Fe

b-1

4

Ma

r-14

Ap

r-14

Ma

y-1

4

Jun

-14

Table

ts p

er

Week

XenoPort

Promoted

Territories

Non-Promoted

Territories

Former

Partner

Stockout

XenoPort

Commercialization

Begins

Weekly HORIZANT Prescribed Tablets

Jefferies Healthcare Conference 18

Former Partner Never Launched for PHN Indication

0

500

1000

1500

2000

2500

Jan-13 Feb-13 Mar-13 Apr-13 May-13 Jun-13 Jul-13 Aug-13 Sep-13 Oct-13 Nov-13 Dec-13 Jan-14 Feb-14 Mar-14 Apr-14

Monthly HORIZANT TRx by Specialty

PCP

Neuro

Psych

PUD/Sleep

Pain/Anes

Other

XenoPort

Begins

Promotion

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. Patients with renal insufficiency require a modified dose."


4-Week Rolling Average - HORIZANT Prescribed Tablets

May 31, 2013 to May 9, 2014

69,645

-

20,000

40,000

60,000

80,000

31

-May

14

-Jun

28

-Jun

12

-Jul

26

-Jul

9-A

ug

23

-Aug

6-S

ep

20

-Sep

4-O

ct

18

-Oct

1-N

ov

15

-Nov

29

-Nov

13

-Dec

27

-Dec

10

-Jan

24

-Jan

7-F

eb

21

-Fe

b

7-M

ar

21

-Mar

4-A

pr

18

-Apr

2-M

ay

69,645 tablets at current WAC price = $489,125 / week

Achieved with 40 sales reps promoting to physicians that

represent ~10% of the potential market


Divider Image Here

(Size image to 4” by 4”)

FUMADERM (mixture of dimethylfumarate 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 United States and February 2014 in EU for the

treatment of relapsing forms of MS

• Q1 2014 TECFIDERA revenues were $506 million ($460 million in U.S.; $46

million in sales outside the U.S.)

XP23829 has novel chemical structure that produces the same

active metabolite as TECFIDERA (dimethylfumarate)



Lower incidence/less severe GI side effects and flushing

• Improved compliance; fewer treatment failures

Onset and/or magnitude of efficacy

• Earlier onset of immunomodulation

Dosing frequency

• Once-a-day rather than BID (TECFIDERA) or TID (FUMADERM)

Indication

• TECFIDERA and FUMADERM not approved for psoriasis in the U.S.


XP23829 development progress

• Favorable physical properties and stability of API and formulations

• Desired metabolism in vitro, in preclinical species and in humans

• Preclinical safety established, including 13-week toxicology studies in 3 animal

species

• Demonstrated efficacy in animal models of MS and psoriasis

• Completed 3 Phase 1 trials establishing human PK, metabolites and disposition

• Demonstrated lower contact sensitization and GI irritation than DMF in preclinical

studies

• Identified 2 formulations with MMF exposure similar to TECFIDERA, including 1

with an extended-release profile

• Demonstrated known pharmacodynamic effects with once-a-day dosing in

humans


Planned Phase 2 psoriasis study expected to be quickest and

most cost effective way to assess the efficacy, tolerability, safety

and dose-response of XP23829

• Sufficient clinical trial material currently in hand

• 13-week duration of current toxicology study sufficient

• Established treatment effects of FUMADERM/TECFIDERA (low placebo)

• Effective at 12-weeks

• 50 subjects per arm

• Can include multiple arms (once-a-day and lower doses)

• Can be enrolled in North America

Optimal dose(s) could translate to MS, based on TECFIDERA

precedent

XP23829 - Potential Development for

Psoriasis


Ability to Establish Dose Response and Onset:

TECFIDERA in Psoriasis

27

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


Similar Dose Response for TECFIDERA in

Psoriasis & Relapsing Forms of MS

28

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

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



829

• Initiation of 829 Phase 2 psoriasis study by mid-year

• Top-line data expected 12-14 months after initiation

HORIZANT

• Continued growth of prescriptions and net sales

• Potential partnership to expand promotional effort

Arbaclofen Placarbil

• Initiation by RBP of a Phase 2 study for alcohol use disorder

29

Thank You

© Copyright 2014 XenoPort, Inc. All rights reserved. NASDAQ:XNPT

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