Paratek Pharmaceuticals, Inc. - FORM 8-K - EX-99.1 - COMPANY SLIDE PRESENTATION DATED SEPTEMBER 2011

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8-K - FORM 8-K - Paratek Pharmaceuticals, Inc.	d233658d8k.htm

September 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

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 planned timing for filing

and

FDA

review

the

Intermezzo

NDA;

the

sufficiency

the

Intermezzo

NDA

gain

FDA

approval;

that

Intermezzo

will

become

the

first

prescription

sleep

aid

indicated

for

use

the

middle

the

night;

the

potential

market

size

for

middle

the

night

sleep

aid;

plans

commercialize

Intermezzo

through

the Purdue collaboration and the receipt by Transcept of payments from Purdue thereunder;

reimbursement coverage for Transcept product candidates; intellectual property protection being obtained

and maintained for Transcept product candidates; and enrollment plans for the Phase 2 study of TO-2061

and plans to report results therefrom in late 2012. Transcept may not actually meet these expectations

and carryout

these

plans.

Various

important

factors

that

could

cause

actual

events

differ materially

from such

forward-looking

statements

include

FDA

deemed

insufficiencies

our

Intermezzo

NDA

resubmission,

competitive

product

commercialization;

adverse

patent

decisions

the

USPTO

court;

the

willingness

Purdue

commercialize

Intermezzo

and

its

ability

successfully;

payer

opinion

Intermezzo

approved;

dependence

third

parties

manufacture,

and

carry-out

the

planned study of TO-2061, and variability in the business of Transcept generally. These and other risks

are

described

greater

detail

the

"Risk

Factors"

section

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.

U.S. primary care partnership: Purdue Pharma

Co-promote

option

post

Intermezzo

launch

6/30/11: ~$60M cash, equivalents & investments

No debt

Neuroscience / psychiatry

Intermezzo

: middle of the night awakenings

TO-2061

treatment

resistant

OCD

Transcept:

preparing

for

Intermezzo

commercialization

Transcept:

preparing

for

Intermezzo

commercialization

Intermezzo

NDA

resubmission:

est.

Sept

2011

TO-2061

Phase

results:

est.

late

2012

Therapeutic focus

Large markets,

unmet needs

Commercial

platform

Strong balance

sheet

Near term

catalysts

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)

–

million

new

and

refill

prescriptions

(2)

Insomnia is an under-treated condition

–

million

patients

receive

(3)

–

are

not

diagnosed

treated

physician

(3)(4)

MOTN

awakening:

the

most

prevalent

insomnia

symptom

(5)

–

35%

Americans

suffer

from

MOTN

awakenings

least

week

(5)

–

>90% report awakenings persist more than six months;

50%

report

awakenings

persist

than

five

years

(6)

(1)

IMS

NSP

2009;

(2)

Wolters

Kluwer,

PHAST

2010;

(3)

BluePrint

Research

Group;

(4)

Institutes

Medicine

Sleep

disorders

and

sleep deprivation

Apr. 2006; (5) Ohayon, Nocturnal awakenings and comorbid disorders in the American general population. J of Psych Research (2009); (6) Ohayon,

Difficulty in resuming or inability to resume sleep and the links to daytime impairment, J of Psych Research (2009).

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

Sleep lab: sleep onset, least squares mean

men 13 min, 3.5 mg; women 16 min, 1.75 mg

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% -

86% lower dose than comparable

Ambien CR

dose

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

Low dose

Novel formulation

Fast acting

Favorable 4 hr

residual effects

Intermezzo

3.5 mg delivered

more zolpidem earlier than a ~3x

higher

Ambien

dose

Intermezzo

3.5 mg delivered

more zolpidem earlier than a ~3x

higher

Ambien

dose

PK comparison study: Intermezzo

3.5mg vs. Ambien 10mg PO (n=33)

Time (min)

9.6x

3.0x

1.5x

Intermezzo

3.5 mg

Ambien

10 mg

Intermezzo

commercial opportunity

Intermezzo

commercial opportunity

Estimating the MOTN market size at branded prices

Ohayon, et al., Nocturnal Awakenings in the American

General Population: Prevalence and Consequences

235 M U.S. adults

(3)

x Ohayon

MOTN sufferers at least 3x week

35.5%

(1)

83 million

…with difficulty returning to sleep

43.0%

(1)

…without difficulty initiating sleep

62.6%

(1)

…who seek MD treatment

16.0%

(2)

x mean length of therapy, currently marketed Rx sleep aids

108 to 169 days

(4)

x per tablet branded pricing, currently marketed Rx sleep aids

$5.05 to $5.67

(5)

= 100% market estimate: treatment seeking MOTN patients

$1.9 B to $3.4 B

3.6

(1)

Ohayon,

al.,

Nocturnal

awakenings

the

American

general

population:

Prevalence

and

consequences.

World

Sleep

Conference

2007;

(2)

Estimated

based

percentage

applied

adults

with

difficulty

returning

sleep,

with

and

without

difficulty

initiating

sleep

Ohayon,

al.,

Using

difficulty

resuming

sleep

define

nocturnal

awakenings,

Sleep

Medicine

(2010);

(3)

U.S.

Census

data,

2010,

adults

and

over;

(4)

IMS:

Length

therapy in the

sleep

disorder

market,

March

2007.

Products

evaluated

are

indicated

for

bedtime

use:

Ambien

Lunesta

Restoril

Rozerem

, Sonata

Desyrel

;

(5)

Wolters

Kluwer

WAC

pricing

(branded)

Jan

2011

(products

evaluated:

Ambien

Lunesta

Silenor

;

Ambien

and

Ambien

are

currently

available

generic

form).

Leading pain therapeutic franchise

2010 net revenue: ~$2.4 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 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

–

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: mid-teens up to mid 20% level on net sales

–

Co-promote

royalty:

additional

double

digit

royalty

psychiatrist

net sales

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

Intermezzo

managed markets advisory board with

decision makers covering >170M lives

Intermezzo

managed markets advisory board with

decision makers covering >170M lives

Broad formulary access expected

Unmet medical need generally recognized by payers

–

Unique insomnia indication

–

Low dose used less often

–

Delivery technology drives rapid bioavailability

Tier 3 formulary placement anticipated by most plans

Utilization management criteria consistent with other brands

Intermezzo

brand parity pricing not viewed as a major

barrier with managed markets

Proven Purdue managed markets team to drive

formulary access

Intermezzo

NDA resubmission

Intermezzo

NDA resubmission

July 2011 FDA Complete Response Letter

January 2011 NDA resubmission: driving study including

comparative analysis, epidemiology study assessing current

MOTN use of bedtime hypnotics, and packaging changes

FDA newly expressed concern that patients with high outlier

zolpidem blood levels may be at risk of next day impairment

FDA further hypothesized that such patients may belong to

distinct and identifiable demographic groups

FDA recommendations

–

Investigate whether demographic factors contribute to elevated blood

levels

–

Consider modifications in dose, time of dose and patient selection

Sept 14, 2011 End of Review meeting with FDA

FDA generally agreed:

–

Change

Intermezzo

dose

for

women

from

3.5

1.75

mg;

3.5

mg dose for men unchanged

–

Intermezzo

should

only

taken

the

patient

has

hours

bedtime remaining. Patients should refrain from driving for at least

1 hour after arising and until not less than 5 hours post dose.

–

the

planned

resubmission

“adequately

concise”

summarizing

morning zolpidem levels and evidence that the levels are safe given

the proposed labeling, FDA may be able to consider this

resubmission as a two-month, Class 1 review.

No additional clinical studies are planned prior to

NDA resubmission

Sleep lab study: 1.75 mg efficacy in women similar

to 3.5 mg efficacy in men

Sleep lab study: 1.75 mg efficacy in women similar

to 3.5 mg efficacy in men

<10

<20

<30

100

Latency to persistent sleep (min)

<10

<20

<30

100

Latency to persistent sleep (min)

Women 1.75 mg (n=58)

Men 3.5 mg (n=24)

60%

63%

Intermezzo®

3.5 mg

Placebo

Baseline

Intermezzo®

1.75 mg

Placebo

Baseline

New Intermezzo

instructions are consistent with

current patient behavior

New Intermezzo

instructions are consistent with

current patient behavior

Originally

proposed

instructions:

“Intermezzo

should

only

taken if the patient has 4 hours of bedtime remaining before

being active again”

Newly

proposed

instructions:

“Intermezzo

should

only

taken

if the patient has 4 hours of bedtime remaining. Patients should

refrain

from

driving

for

least

hour

after

arising

and

until

not

less than 5 hours post dose.”

The typical American: time between arising and commuting to

work

–

1 hour 53 minutes (mean), National Sleep Foundation 2008

–

1 hour (median), Bureau of Labor Statistics 2003-2010

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

~5mg

–

Formulation with buffer system for transmucosal absorption

Method of treating MOTN awakenings

–

Priority date: May 2006

–

Low

dose

zolpidem,

~1mg

~4.75mg

–

Treatment of middle of the night awakenings

Administration as needed after the subject awakens

–

Proposed claims cover multiple dosage forms

TO-2061

(low dose ondansetron)

TO-2061

(low dose ondansetron)

Proposed indication: adjunctive therapy in adult patients

with obsessive compulsive disorder (OCD) not adequately

responsive to currently approved OCD medication

Significant unmet medical need: OCD patients not

responding adequately to approved medication

Significant unmet medical need: OCD patients not

responding adequately to approved medication

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 approved

medications,

which

include

the

SSRIs

Prozac

Luvox

Paxil

Zoloft

No FDA approved medication for treatment resistant

OCD

–

Atypical antipsychotics are often used off-label to augment approved

medications

•

~68% of treatment resistant OCD patients do not respond

•

Frequently reported adverse events: weight gain, metabolic disorder

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

antagonist

approved

Zofran

)

–

Affects serotonin and dopamine pathways

–

Typical

daily

Zofran

doses

for

nausea

and

vomiting induced by chemotherapy, radiation therapy and surgery

Two 12 week open-label adjunctive therapy studies

with ondansetron titrated to 0.5 mg BID dose

–

Pilot

Study

(1)

Adjunctive

ondansetron

therapy

patients

who

responded poorly to at least 12 weeks of SSRIs combined with an

atypical antipsychotic, n=14

–

Pilot

Study

(2)

Adjunctive

ondansetron

therapy

patients

who

responded poorly to at least 12 weeks of SSRI treatment, n=21

(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

49 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)

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

50%

40%

30%

20%

10%

All patients

Week

50%

40%

30%

20%

10%

Responders

Non-responders

505b2 NDA pathway

Phase

study,

150,

top

line

data

est.

late

2012

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

Strategic fit: psychiatry

–

~87% of patient visits for OCD were to psychiatrists in 2009

–

Complementary

Intermezzo

psychiatry

co-promote

option

with

Purdue

* SDI Physician Drug and Diagnosis Audit

TO-2061

development

overview

Managed care survey (~108M lives): unmet medical need

acknowledged, Tier 3 formulary placement expected

Financial overview

Financial position

Cash & investments (6/30/11):

$59.6 M

Q2 2011 cash burn rate:

$ 1.2 M / month

Shares outstanding (9/15/11):

13.5 M

Options / warrants / other:

3.5

Total:

17.0 M

Employees (9/15/11):

Intermezzo

is a registered trademark of Transcept Pharmaceuticals, Inc.

Ambien

and

Ambien

are

registered

trademarks

sanofi-aventis

Lunesta

is a registered trademark of Sunovion Pharmaceuticals Inc.

Zofran

and

Paxil

are

registered

trademarks

The

GlaxoSmithKline

Group

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.

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