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| 8-K - FORM 8-K - Paratek Pharmaceuticals, Inc. | d8k.htm |
 January 2011
January 2011
A specialty pharmaceutical company focused on the development
and commercialization of proprietary products to address important
therapeutic needs in the field of neuroscience
Exhibit 99.1 |
 Forward
looking statements Forward looking statements
This
presentation
contains
forward-looking
statements
that
involve
substantial
risks
and
uncertainties.
All
statements,
other
than
statements
of
historical
facts,
included
in
this
presentation
are
forward-
looking
statements.
Examples
of
such
statements
include
our
expectation
regarding
the
sufficiency
of
the
Intermezzo®
NDA
resubmission
to
satisfy
FDA
issues
and
gain
FDA
approval;
expectations
that
Intermezzo®
will
become
the
first
prescription
sleep
aid
indicated
for
use
in
the
middle
of
the
night;
plans
to
commercialize
Intermezzo®
under
favorable
market
conditions
through
the
Purdue
collaboration
and
the
receipt
by
Transcept
of
payments
from
Purdue
thereunder;
expectations
that
intellectual
property
protection
will
be
obtained
and
maintained
for
Transcept
product
candidates;
and
plans
to
begin
a
Phase
2,
double-blind,
placebo-controlled
study
of
Setrodon™
in
2011.
Transcept
may
not
actually
meet
these
expectations
and
carryout
these
plans.
Various
important
factors
that
could
cause
actual
events
to
differ
materially
from
such
forward-looking
statements
include
FDA
deemed
insufficiencies
in
our
Intermezzo®
NDA
resubmission,
competitive
commercial
pressures;
adverse
patent
decisions
at
the
USPTO
or
in
court;
the
willingness
of
Purdue
to
commercialize
Intermezzo®
and
its
ability
to
do
so
successfully;
dependence
on
third
parties
to
manufacture,
and
carry-out
the
planned
study
of
Setrodon™
,
and
variability
in
the
business
of
Transcept
generally.
These
and
other
risks
are
described
in
greater
detail
in
the
"Risk
Factors"
section
of
Transcept
periodic
reports
filed
with
the
Securities
and
Exchange
Commission.
Forward-looking
statements
do
not
reflect
the
potential
impact
of
any
future
in-licensing,
collaborations,
acquisitions,
mergers,
dispositions,
joint
ventures,
or
investments
Transcept
may
enter
into
or
make.
Transcept
does
not
assume
any
obligation
to
update
any
forward-looking
statements,
except
as
may
be
required
by
law.
2 |
 Therapeutic
focus Large markets,
unmet needs
Commercial
platform
U.S. primary care partnership: Purdue Pharma
Co-promote
option
1
yr
post
Intermezzo
®
launch
9/30/10: $74M cash, equivalents and investments
no debt
CNS / Psychiatry
Intermezzo
®
:
middle
of
the
night
awakenings
Setrodon
™
: treatment resistant OCD
Transcept: preparing for Intermezzo
®
commercialization
Transcept: preparing for Intermezzo
®
commercialization
3
Strong balance
sheet |
 Intermezzo
®
(zolpidem
tartrate
sublingual tablet)
Intermezzo
®
(zolpidem
tartrate
sublingual tablet)
Proposed
indication
statement:
Intermezzo
®
is
indicated
for use as needed for the treatment of insomnia when a
middle of the night awakening is followed by
difficulty returning to sleep
*
*
*
*
* |
 Middle of the
night (MOTN) awakening: A major unmet medical need in the insomnia category
Middle of the night (MOTN) awakening:
A major unmet medical need in the insomnia category
Large U.S. insomnia market
–
$2.1
billion
(ex-factory)
(1)
–
78
million
new
and
refill
prescriptions
(2)
Insomnia is an under-treated condition
–
11
million
patients
receive
Rx
(3)
–
4x
to
6x
more
are
not
diagnosed
or
treated
by
a
physician
(3)(4)
MOTN
awakening:
the
most
prevalent
insomnia
symptom
(5)
–
35% of Americans suffer from MOTN awakenings at least 3x / week
–
At least 75% of the MOTN group reported no difficulty going to sleep
–
10.9% of MOTN sufferers consult a physician
5
Source:
(1)
IMS
NSP
2009;
(2)
Wolters
Kluwer,
PHAST
2010;
(3)
BluePrint
Research
Group;
(4)
Institutes
of
Medicine -
Sleep disorders and sleep deprivation Apr. 2006; (5) Ohayon: Nocturnal Awakenings and
comorbid
disorders in the American general population, J of Psych Research (2008).
|
 No product
currently indicated for prn treatment at the
time of a middle of the night awakening
No product currently indicated for prn
treatment at the
time of a middle of the night awakening
MOTN awakenings typically do not occur every night
7-8 hr sleep aids (Ambien
®
, Ambien CR
®
, Lunesta
®
) require
bedtime prophylactic dosing to prevent awakenings
An ideal therapeutic would:
–
Be used only at the time patients need help returning to sleep,
not every night in advance of a problem that may not occur
–
Return patients to sleep rapidly
–
Use a low dose to avoid next day residual effects
6 |
 Novel
formulation Novel formulation
Low dose
Low dose
Fast acting
Fast acting
Sleep lab, objective data: 14 minute sleep
onset with 3.5 mg dose
No residual next-morning effects vs. placebo in
sleep lab and outpatient studies
Driving study primary statistical analysis: no
significant next morning effect vs. placebo 4 hrs
after dosing, consistent with proposed label
Sublingual tablet dissolves in ~ 2 minutes
pH
drives
zolpidem
base,
rapidly
absorbed
72%
lower
dose
than
Ambien
CR
®
Intermezzo
®
: anticipated to be the first sleep aid for
use in the middle of the night at the time of awakening
Intermezzo
®
: anticipated to be the first sleep aid for
use in the middle of the night at the time of awakening
7
Favorable residual
effects profile
Favorable residual
effects profile |
 Intermezzo
®
3.5 mg delivered greater early zolpidem
bioavailability than a ~3x higher Ambien
®
dose
Intermezzo
®
3.5 mg delivered greater early zolpidem
bioavailability than a ~3x higher Ambien
®
dose
8 |
 Intermezzo
®
commercial opportunity
Intermezzo
®
commercial opportunity
*
*
*
* |
 Leading
pain therapeutic franchise 2009 rev: >$2.5 billion
Branded products include:
OxyContin
®
, MSContin
®
, Dilaudid
®
, Butrans
®
Sales force of 500 field reps calling on primary care
physicians and high prescribing specialists
High value pain prescribers tend to be significant insomnia
prescribers
Multi-year sales and marketing agreement with psychiatry
co-promote option, royalties and milestone payments
Commercialization partnership with Purdue Pharma
Commercialization partnership with Purdue Pharma
10 |
 Commercialization agreement: key Transcept benefits
Commercialization agreement: key Transcept benefits
Co-promote option: foundation for a commercial future
–
Transcept
option: co-promote to psychiatrists as early as the first
anniversary after Purdue launch
Milestone payments
–
Upfront license fee: $25M received August 2009
–
FDA approval milestone of $30M, reduced by $2M each 30-day period
after June 30, 2010 (estimated $0 -
$6M upon approval)
–
Up to $90M additional upon the achievement of certain patent
milestones and net sales targets, including $10M for first formulation
patent listed in Orange Book
Royalty structure
–
Base royalty: double digit up to the mid 20% level on net sales
–
Co-promote
royalty:
additional
double
digit
royalty
on
psychiatrist
Rx
net sales
11 |
 Intermezzo
®
partnership structure enables
significant Transcept marketing efficiency
Intermezzo
®
partnership structure enables
significant Transcept marketing efficiency
Purdue responsibilities
–
U.S. product launch
–
Primary care sales and marketing activities
–
Managed care and formulary placement
–
Manufacturing and distribution
–
Post marketing studies, if required
–
Book revenues
Transcept responsibilities
–
U.S. product approval
–
Psychiatry co promote option 1 yr after commercial launch
12 |
 Favorable
market
conditions
for
Intermezzo
®
launch
Favorable
market
conditions
for
Intermezzo
®
launch
Intermezzo
®
: anticipated to be the first strongly
differentiated insomnia treatment strategy since
launch
of
Ambien
®
in
1993
Continuing decline in sales presentations to
physicians in insomnia segment
Significantly reduced DTC spending
13 |
 Intermezzo
®
NDA resubmission
Intermezzo
®
NDA resubmission
*
*
*
* |
 FDA Complete
Response Letter (October 2009) and subsequent discussions
FDA Complete Response Letter (October 2009) and
subsequent discussions
FDA indicated Transcept
has submitted substantial evidence
of effectiveness for Intermezzo
®
in its intended indication
FDA recognized that the Intermezzo
®
Phase 3 data did not
indicate significant next morning residual effects at 4 hrs
However, given the unique MOTN indication, FDA requested:
–
additional data on next morning driving effects
–
demonstration that inadvertent dosing errors can be minimized,
or that the consequences of such errors are acceptable
•
inadvertent re-dosing in a single night
•
inadvertent dosing with < 4 hrs of bedtime remaining
–
arguments against the conduct of an in-use study
15 |
 Resubmit
Intermezzo ®
NDA Q1 2011, PDUFA Q3 2011
Resubmit Intermezzo
®
NDA Q1 2011, PDUFA Q3 2011
Driving study primary statistical analysis: no significant
next morning effect when dosed per proposed label
Comparative analysis vs. previous driving study data:
even when dosed off label (< 4hrs prior to driving),
Intermezzo
®
next day effects are relatively small
New epidemiology study offers first demonstration of
current and widespread middle of the night (off-label)
use of 7-8 hour sleep aids
Packaging changes, patient tools and instructions to
address FDA concerns re: dosing errors
16 |
 Redesigned
unit dose packaging for NDA resubmission Redesigned unit dose packaging for NDA
resubmission 17
Prototype
package:
Intermezzo
®
is
not
approved
by
the
U.S.
FDA |
 Intermezzo
®
highway driving study overview
Intermezzo
®
highway driving study overview
Approximately 40 healthy adult volunteers
Highway driving over a one-hour period
Single-center (Maastricht),
double-blind,
randomized,
placebo-controlled
crossover design
Key comparisons:
–
Intermezzo
®
3.5mg vs. placebo, dosed 4 hours prior to driving
–
Intermezzo
®
3.5mg vs. placebo, dosed 3 hours prior to driving
–
Zopiclone
7.5mg vs. placebo (positive control)
Key measure of driving performance: standard
deviation of lateral position (variability in lane position)
as compared to placebo
18 |
 Intermezzo
®
highway driving study results
Intermezzo
®
highway driving study results
4
hrs
after
MOTN
Intermezzo
®
dosing
–
Test condition consistent with proposed label
–
Primary analysis (symmetry): no statistically significant effect
–
Secondary analysis (mean): statistically significant but small
effect of 0.8 cm
SDLP
3
hrs
after
MOTN
Intermezzo
®
dosing
–
Test condition included to characterize risk profile of
Intermezzo
®
when not used according to label
–
Primary analysis (symmetry): statistically significant effect
–
Secondary analysis (mean): statistically significant but small
effect of 1.5 cm
SDLP
–
One drive discontinued due to excessive drowsiness
FDA may consider other statistical analyses
19 |
 0
1
2
3
4
5
6
7
8
4h
post-dose
3h
post-dose
9h
post-dose
10-11h
post-dose
10-11h
post-dose
10-11h
post-dose
10-11h
post-dose
10-11h
post-dose
Effect on driving produced by various hypnotics and
blood alcohol concentrations: cross study comparison
Effect on driving produced by various hypnotics and
blood alcohol concentrations: cross study comparison
Intermezzo
®
highway driving study
Zopiclone
and US-marketed hypnotics
Brookhuis
K. Hum. Psychopharmacol. Clin. Exp.
1998;13:S64–S69.
0.05%
EU
Note:
The
cross
study
comparisons
reflected
above
should
be
viewed
with
caution.
Cross
study
meta-analyses
are
subject
to
interpretational
and
other
risks
due
to
a
number
of
factors,
including
unaccounted
for
variables
that
may
render
comparisons
difficult
or
invalid,
and
potential
inconsistencies
in
methods
of
normalizing
study
results.
Additional
studies
from
the
Brookhuis
publication
reflecting
data
from
drugs
not
approved
in
the
United
States
are
not
reflected
above.
The
above
collection
of
studies
is
not
presented
as
a
complete
presentation
of
highway
driving
studies
analyzing
hypnotic
drugs
approved
in
the
United
States.
0.08%
USA
DUI blood
alcohol
concentration |
 Intermezzo
®
:
intellectual property
Intermezzo
®
:
intellectual property
*
*
*
* |
 Intermezzo
®
: two formulation patents issued; method
of use patents pending
Intermezzo
®
: two formulation patents issued; method
of use patents pending
Formulation for transmucosal absorption
–
Two issued U.S. patents -
7,682,628 and 7,658,945
–
Patents expire no sooner than February 2025
–
Low dose zolpidem, ~1mg to ~5mg
–
Formulation with buffer system for transmucosal absorption
Method of treating MOTN awakenings
–
Priority date: May 2006
–
Low dose zolpidem, ~1mg to ~5mg
–
Treatment of middle of the night awakenings
Administration as needed after the subject awakens
–
Proposed claims cover multiple dosage forms
22 |
 Setrodon™
(ultra low dose ondansetron)
Setrodon™
(ultra low dose ondansetron)
23
Adjunctive therapy in patients with obsessive compulsive
disorder not adequately responsive to SSRI treatment
*
*
*
* |
 Significant
unmet medical need: OCD patients not responding adequately to SSRI treatment
Significant unmet medical need: OCD patients not
responding adequately to SSRI treatment
Obsessive Compulsive Disorder
–
Intrusive thoughts and repetitive actions to reduce distress
–
Affects 1% to 2% of U.S. adult population, 40% to 50% seek treatment
–
Significantly impacts everyday life activities of patients and their families
–
40% to 60% of OCD patients do not respond adequately to first-line
SSRI
treatment
(e.g.,
Prozac
®
,
Luvox
®
,
Paxil
®
,
Zoloft
®
)
No FDA approved treatment for SSRI resistant OCD
–
Atypical antipsychotics are often used off-label to augment SSRIs
•
~68% of SSRI resistant OCD patients do not respond
•
Frequently reported adverse events: weight gain, metabolic disorder
24 |
 Pilot
studies: ultra low dose adjunctive ondansetron therapy in SSRI resistant OCD
Pilot studies: ultra low dose adjunctive ondansetron
therapy in SSRI resistant OCD
Ondansetron
(5-HT
3
antagonist approved as Zofran
®
)
–
Affects serotonin and dopamine pathways
–
Typical daily Zofran
®
doses of 16 mg to 24 mg for chemotherapy-
induced nausea and vomiting
Two 12 week open-label adjunctive therapy studies
with ondansetron
titrated to 0.5 mg BID dose
–
Pilot
Study
A
(1)
:
Adjunctive
ondansetron
therapy
in
patients
who
responded
poorly
to
at
least
12
weeks
of
SSRIs
combined
with
an
atypical antipsychotic, n=14
–
Pilot
Study
B
(2)
:
Adjunctive
ondansetron
therapy
in
patients
who
responded poorly to at least 12 weeks of SSRI treatment, n=21
25
(1)
S.
Pallanti,
S.
Bernardi,
S.
Antonini,
N.
Singh,
E.
Hollander:
Ondansetron
augmentation
in
Treatment-Resistant
Obsessive-Compulsive
Disorder,
CNS
Drugs
(2009).
(2)
S.
Pallanti,
S.
Bernardi,
E.
Hollander:
Ondansetron
augmentation
in
Treatment-Resistant
OCD
(TR-OCD):
Relapse
in
Y-BOCS
symptoms
following
discontinuation
of
ondansetron,
Poster
presentation,
American
College
of
Neuropsychopharmacology
49th
Annual
Conference,
December
2010. |
 Improvement:
measured as a % decrease over baseline on the Yale Brown Obsessive Compulsive Scale
(YBOCS) Improvement: measured as a % decrease over baseline on the
Yale Brown Obsessive Compulsive Scale (YBOCS)
26
All Patients: n=21
26.3% improvement at 12 weeks
Responders: n=12 of 21 (57%)
44.3% improvement at 12 weeks
Pilot Study B, n=21
Week
2
Week
4
Week
6
Week
8
Week
10
Week
12
50%
40%
30%
20%
10%
0%
All patients
Week
2
Week
4
Week
6
Week
8
Week
10
Week
12
50%
40%
30%
20%
10%
0%
Responders
Non-responders |
 Week
0
Week
2
Week
4
Week
6
Week
8
Week
10
Week
12
Week
14
Week
16
10
20
30
40
Relapse following discontinuation in responders
Relapse following discontinuation in responders
27
38.3%
YBOCS
worsening
from Week 12
Phase 1
Ondansetron augmentation
Phase 2
Discontinuation
Pilot Study B, n=12 of 21 (responders only) |
 Setrodon™:
development
overview
Setrodon™:
development
overview
505b2 NDA pathway
Phase
2
study,
n
150
–
To start Q1 2011, top-line results 2012
–
Approx. $10M investment through end of Phase 2 study
Intellectual property
–
Method of use patent application filed, priority date May 19, 2009
–
Ondansetron, up to ~1.5 mg/day
–
Pending claims for treating SSRI resistant OCD with ondansetron
augmentation
Managed care survey (~108M lives): unmet medical need
acknowledged, Tier 3 formulary placement expected
Strategic fit: psychiatry
–
~87% of patient visits for OCD were to psychiatrists in 2009
*
–
Complementary to Intermezzo
®
psychiatry co-promote option with Purdue
28
* SDI Physician Drug and Diagnosis Audit |
 Financial
overview Financial overview
*
*
*
* |
 Financial
position: September 30, 2010 Financial position: September 30, 2010
Cash & investments:
$74.2 M
Q3 2010 cash burn rate:
$ 1.6 M / month
Shares outstanding:
13.4 M
Options / warrants / other:
2.5
Total:
15.9 M
Employees:
31
30 |
 Key
Transcept activities and goals
Key Transcept
activities and goals
Completed Intermezzo
®
US marketing partnership, July 09
Patent coverage: two Intermezzo
®
formulation patents issued
Highway driving study data, Oct 2010
Resubmit Intermezzo
®
NDA early Q1 2011, 6 month review
expected
Intermezzo
®
pre-launch planning
Begin Setrodon
™
Phase 2 study Q1 2011
31 |
 Intermezzo
®
is a registered trademark of Transcept
Pharmaceuticals, Inc.
Ambien
®
and
Ambien
CR
®
are
registered
trademarks
of
sanofi-aventis
Lunesta
is a registered trademark of Sunovion
Pharmaceuticals Inc.
Zofran
®
and
Paxil
®
are
registered
trademarks
of
The
GlaxoSmithKline
Group
of
Companies
Prozac
®
is a registered trademark of Eli Lilly & Co.
Luvox
®
is a registered trademark of Solvay Pharmaceuticals, Inc.
Zoloft
®
is a registered trademark of Pfizer Inc. |