Pain Therapeutics, Inc.

Pain Therapeutics, Inc.
Pioneers in Preventing Prescription Drug Abuse
Remi Barbier
President & CEO
June 2014
1
Forward-Looking Statements
This presentation contains forward-looking statements within the meaning of the Private Securities
Litigation Reform Act of 1995, including statement about our prospects for growth, intellectual property
protection, market size or growth, competitive position, regulatory agency action, and the anticipated
development, timing, data readouts and therapeutic scope and value of our development stage
products. You should not place undue reliance on these statements.
These statements involve significant risks and uncertainties. Our results may differ materially from those
contained in such statements, including, among others: our inability to protect our intellectual property
rights and to have sufficient rights to commercialize our products; product competition; clinical trials of
our products may fail or not initiated or conducted in a timely manner; our products may show
insufficient therapeutic effects or unacceptable safety profiles; adverse decisions by regulatory
authorities; existing preclinical and clinical data with respect to our products may not be indicative of
future clinical or commercial results; unfavorable market launch of our product by Pfizer; reimbursement
for our products; and the inability to manufacture successfully our products.
Additional factors that could cause actual results to differ significantly from those projected in our
forward-looking statement are discussed in our Annual Report on Form 10-K, Quarterly Reports on Form
10-Q and recent Current Reports on Form 8-K. Our forward-looking statements are based on our current
beliefs and expectations and speak only as of the date of this presentation. We do not undertake any
obligation to update any forward-looking statements.
The term “Abuse-resistance” or “Tamper-resistance” as used in this presentation are not intended to
designate an indication or a medical claim but rather a general description of agents designed to
address the misuse, abuse and diversion of opioids. Oxycodone (CII) is a controlled-substance with
high potential for abuse. The active ingredient in all opioids (including REMOXY) are highly addictive.
2
Pain Therapeutics Highlights
 Lead compound represents
next-generation abuse-resistance
 Phase III efficacy study met all
primary endpoints under an SPA
 Large target market ~$2.5 billion,
with favorable demographics
REMOXY
 Attractive deal economics, 15-20%
royalty on net sales in the U.S.
3
Balanced Pipeline
Registration
Phase I/II
Pre-clinical
Three Undisclosed Assets in
Pain and Other Indications.
One Undisclosed Asset;
Abuse-resistant Hydromorphone;
Abuse-resistant Hydrocodone.
REMOXY®
(oxycodone CII)
4
REMOXY - Product Overview
 A unique, extended-release formulation of oxycodone (CII)
for patients with moderate-to-severe chronic pain.1
‒ Phase III achieved all clinical endpoints under an SPA
 Next-generation abuse-resistance technology designed to
discourage common methods of tampering.
‒ Intellectual property protection beyond 2030
 Large, established target market (~$2.5 billion) with
demographics in favor of volume growth.
(1) This is not intended to designate an indication or a medical claim but rather a general description of strong
opioids designed to address certain types of clinical pain. Oxycodone (CII) is a controlled-substance with high
potential for abuse. The active ingredient in all opioids (including REMOXY) are highly addictive.
5
An Attractive Partnership
Pfizer has worldwide rights to REMOXY1
 $185 million cash received to
date from partnership.
 $15 million milestone payment
on FDA approval of REMOXY.
 100% of R&D, commercial costs
paid by Pfizer.
(1)
Except Australia & New Zealand.
6
An Attractive Partnership
Pfizer has worldwide rights to REMOXY1
 $185 million cash received to
date from partnership.
 $15 million milestone payment
on FDA approval of REMOXY.
 100% of R&D, commercial costs
paid by Pfizer.
20% ROYALTY on net sales in the U.S., except as to the
first cumulative billion in sales, where the royalty is 15%.2
(1)
Except Australia & New Zealand.
(2)
We will also receive a supplemental royalty equal to 6 to 11.5% of net sales, directly offsetting our financial
obligation to one of our suppliers of excipients.
7
Pain Management Today
Annual market for pain medications exceeds $7 billion;
Over 27 million people have moderate-to-severe pain in U.S.
MILD
MODERATE
SEVERE
ORTHOPEDIC
SOFT
TISSUE
INJURIES
 Acetaminophen
 COX-2 Inhibitor
BREAK-THROUGH
POST-OP
BACK PAIN
CANCER
 Hydrocodone
 Hydromorphone
 Oxycodone
 Morphine
8
Opioids For Pain Management
Global Opioid Consumption
6 principal opioids used to treat moderate to severe pain
1980 - 2010
70
60
50
Oxycodone
40
30
20
10
0
1980
1985
1990
1995
2000
2005
2010
Source: Pain & Policy Studies Group (2013)
9
Opioid Misuse is Epidemic
The World Health Organization estimates
that approximately 2 million people in the
U.S. alone are addicted to prescription opiates.
An estimated 52 million
people (20% of those age
12 and older) have used
prescription drugs for
nonmedical reasons at
least once in their lifetime.
Opiate dependency is
estimated to affect nearly
10% of modern day
medical practitioners.
The problem is not limited
to the medical community,
first use of an opiate seems
to be getting younger.
Heroin is the
most widely
used illegal
opiate
1 in 12 high school seniors
reported past-year
nonmedical use of the
prescription pain reliever,
Vicodin.
Prescription opiate
painkillers are just as
prevalent, and just as
dangerous
50%
40%
20%
More than half of teens
agree that prescription
drugs are easier to get
than illegal drugs.
Almost 40% of teens
don’t perceive any
great risk in trying
heroin once or twice.
3 of 10 teens
believe that
prescription pain
relievers are not
addictive.
300
270
240
210
180
150
120 78
90
60
30
0
1991
210
2010
The total number of opiate
prescriptions dispensed by
retail pharmacies in the US
rose from 76 million in 1991
to 210 million in 2010.
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Regulators Are Committed
“Let me say, in no uncertain terms: the prevention of
prescription opioid abuse is of the highest priority for
the FDA.”
- April 2014 Margaret A. Hamburg, M.D., Commissioner of the U.S.
Food and Drug Administration
“Prescription painkiller overdoses are a public health epidemic.
Prescription painkiller overdoses killed nearly 15,000 people in the US in 2008.
This is more than 3 times the 4,000 people killed by these drugs in 1999.”
‒ Nov 2011
“[Our]action plan primarily focuses on the growing and often deadly problem
of prescription opioid abuse…From 1997 to 2007, the milligram per person
use of prescription opioids in the U.S. increased from 74 milligrams to 369
milligrams, an increase of 402 percent.”
‒ Nov 2013
11
Common Methods of Abuse
VAPORIZE
DISSOLVE
FREEZE
CHEW
MICROWAVE
SNORT
CRUSH
INJECT
GRIND
12
1st Gen Abuse Resistance
Optimized to resist some methods of abuse
VAPORIZE
DISSOLVE
FREEZE
CHEW
MICROWAVE
SNORT
CRUSH
INJECT
GRIND
Examples: Crush-resistant polymer matrices (“hard shell”); sequestered
antagonists (“beads”); gelling agents that prevent injection.
13
Next Gen Abuse Resistance
REMOXY:
Designed to resist all common methods of formulation abuse.
VAPORIZE
DISSOLVE
FREEZE
CHEW
MICROWAVE
SNORT
CRUSH
INJECT
GRIND
This is not intended to designate an indication or a medical claim but rather a general description of strong opioids
designed to address certain types of clinical pain. Oxycodone (CII) is a controlled-substance with high potential for abuse.
The active ingredient in all opioids (including REMOXY) are highly addictive.
14
A Gold Standard
REMOXY may challenge market dynamics
with a gold standard of abuse resistance…
VAPORIZE
DISSOLVE
FREEZE
CHEW
MICROWAVE
SNORT
CRUSH
INJECT
GRIND
…and help ensure patients in pain have appropriate
access to opioid analgesics.
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Phase III Design Under SPA
Primary Endpoint - Pain Scores vs. Placebo
 412 osteoarthritic patients, moderate-severe pain
 12-Week treatment period
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Phase III Result Summary
Mean Reduction of
Pain Intensity Over 12 Weeks
0
Mean Pain Intensity AUC
-10
 Quality of Analgesia
and Global Assessment
of medication were
superior for REMOXY
-20
-30
-40
(p=0.004 and p=0.007)
-50
-60
 Achieved Primary
Endpoint of pain
reduction vs. placebo
Placebo
(N=205*)
Remoxy
(N=201*)
-70
(p=0.007)
 Most AEs were typical
opioid-related AEs, mild
and transient in nature
17
REMOXY – Regulatory History
 REMOXY NDA submitted by PTI: June 2008
 1st Complete Response: Dec 2008 - MANUFACTURING ISSUES
 2nd Complete Response: June 2011 - MANUFACTURING ISSUES
‒ Pfizer has handled CMC remedial work, at its own expense.
‒ Pfizer obtained FDA endorsement of the REMOXY program in March 2013:
“In summary, it appears there is a regulatory path forward for REMOXY”.
18
Pfizer’s Press Release (excerpt)
Pfizer Inc. Continues Development Program For Remoxy®
(oxycodone) Extended-Release Capsules CII
“October 22, 2013. Pfizer Inc. (NYSE: PFE) announced today that,
having achieved technical milestones related to manufacturing, it
will continue the development program for Remoxy® (oxycodone)
Extended-Release Capsules CII. Following guidance received from
the U.S. Food and Drug Administration (FDA) earlier this year, Pfizer
will proceed with the additional clinical studies and other actions
required to address the Complete Response Letter received in June
2011.”
19
Pfizer: NDA Re-filing mid-2015
Clinical Milestones
Study
# Subjects
Est. Timelines
60
On-going
Abuse Potential
Pharmacokinetics
Bioavailability
60
Dose Proportionality
18
Effects of Food
18
Sources: ClinicalTrials.gov, May 2014 and Pfizer, Inc.
Mid-2014
20
REMOXY – Path To Market
 Market launch by Pfizer in the U.S., following FDA approval and
risk-mitigation negotiations
 We believe REMOXY’s unique chemical properties have potential
to set a high bar for a gold standard of abuse-resistance in a $2.5
billion target market
 REMOXY is in Pfizer’s Global Innovative Products unit
‒ Unit sales ~$12 billion in FY2014, most profitable segment
‒ Established relationships with health care systems, primary care
providers & consumers
‒ Cornerstone pain drug brands include Celebrex® & Lyrica®
21
Efficient Capital Management
 ~ $47 million of cash & equivalents at Q1 2014
 ~ $12 million net cash use expected in 2014
 ~ 45 million shares outstanding
 ~ 300,000 shares trade daily
 ~ $33 million NOL
Management has ~ 30% ownership
22
Pain Therapeutics Highlights
 Lead compound represents
next-generation abuse-resistance
 Phase III efficacy study met all
primary endpoints under an SPA
 Large target market ~$2.5 billion,
with favorable demographics
REMOXY
 Attractive deal economics, 15-20%
royalty on net sales in the U.S.
23