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Improving Life Transforming Value. May 2015

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(1)

Improving Life… Transforming Value

(2)

Forward Looking Statement

This presentation contains forward-looking statements made pursuant to the safe harbor provisions of the U.S. Securities Litigation Reform Act of 1995. Forward-looking statements involve known and unknown risks and uncertainties that could cause the Company's actual results to differ materially from those in the forward-looking statements. Such risks and uncertainties include, among others, the availability of funds and resources to pursue R&D projects and clinical trials, the successful and timely completion of clinical studies, the risk that safety and efficacy data from any of our Phase 3 trials may not coincide with the data analyses from previously reported Phase 1 and/or Phase 2 clinical trials, the ability of the Company to efficiently commercialize one or more of its products or product candidates, the ability of the Company to take advantage of business opportunities in the pharmaceutical industry, uncertainties related to the regulatory process, the ability to protect our intellectual property, the potential of liability arising from shareholder lawsuits and general changes in economic conditions. Investors should consult the Company's quarterly and annual filings with the Canadian and U.S. securities commissions for additional information on risks and uncertainties relating to forward-looking statements. Investors are cautioned not to place undue reliance on these forward-looking statements. The Company does not undertake to update these forward-looking statements. We disclaim any obligation to update any such factors or to publicly announce the result of any revisions to any of the forward-looking statements contained herein to reflect future results, events or developments, unless required to do so by a governmental authority or by applicable law.

(3)

Highlights

Commercial-stage promotion

EstroGel

®

: Leading non-patch transdermal estrogen therapy product commercialized by

Ascend Therapeutics in the US

Saizen

®

: Growth hormone replacement therapy in children and adults with growth

hormone deficiency (GHD) commercialized by EMD Serono in the United States

Clinical-stage development

Zoptarelin doxorubicin: potential therapy for endometrial cancer

• ZoptEC Phase 3 trial (under SPA with FDA); wholly-owned, product candidate

• Potential additional indications (ex: ovarian, prostate, breast cancer therapies)

Macrilen™: potential drug for evaluating Adult GHD

• Confirmatory Phase 3 efficacy trial to be initiated before year-end

Growth

Actively pursuing other in-licensing, acquisition & promotion/co-promotion opportunities

Innovative technology platform supporting long-term growth (“cytotoxic peptide

conjugates” and “cell signaling”)

Leadership

Proven leadership with expertise in delivering revenue and earnings growth

(4)

At a Glance

Transitioning to a specialty biopharmaceutical company

Heavily focused on establishing revenues, cash and

profitability

Resource optimization / burn reduction is a priority

NASDAQ: AEZS, TSX: AEZ

Cash on hand as of March 31, 2015: $53.2 million with no

debt

(5)

Focused On Value Transformation

Aeterna Labs Founded Zentaris GmbH Acquired Clinical Development Strategic Shift to Commercial Development Expansion of Commercial Operations

1993

2002

2003

2012

2013

2014

2015

ZoptEC Trial Launched Focus On Business Development Organization Restructuring; focus on reducing burn-rate ZoptEC Interim Analysis Portfolio Expansion & Development Leaner, More Focused Organization 5 Initiated selling Ascend’s EstroGelin U.S.

(6)

Leadership with Significant Industry Success and Expertise

Executive

Title

Prior Affiliation(s)

Years of

Experience

David Dodd

Chairman, CEO

Abbott, BMS, Wyeth, Solvay,

Serologicals and others

30+

Jude Dinges

Senior VP and CCO

Merck, Novartis, Amgen

25+

Richard Sachse

Senior VP, CMO and CSO

Boehringer Ingelheim, Bayer,

Schwarz Pharma and UCB

20+

Phil Theodore

Senior VP, CAO, General

Counsel, Corporate Secretary

King & Spalding, Serologicals,

BioReliance, John H. Harland

and Zep

28+

Dennis Turpin

Senior VP and CFO

Coopers & Lybrand (now PwC)

25+

(7)

Strategic Growth Plan

Achieve successful commercial presence and growth

Successful licensing/acquisition/promotional opportunities

Field selling & development of EstroGel

®

, Saizen

®

& additional

products

Leverage sales force

Utilize collaborations to exploit portfolio value & growth

Pursue successful development and commercialization of our

internal product portfolio

Zoptarelin doxorubicin in oncology

Macrilen™ in endocrinology

Become a growth oriented specialty

biopharmaceutical company

(8)

Pipeline – Supporting Long-Term Growth

Product Candidate Discovery Preclinical Phase 1 Phase 2 Phase 3

LHRH - Disorazol Z Oncology

Erk inhibitors Oncology

AEZS-120 Prostate Cancer(4)

Zoptarelin doxorubicin Endometrial Cancer

Compound Library – MUSC(5)

Zoptarelin doxorubicin Ovarian(1)and Prostate(2) Cancer

(1) Phase 2 in ovarian cancer completed. (2) Investigator-driven and sponsored.

(3)Confirmatory Phase 3 efficacy trial to be initiated before year-end. (4)Potential oral prostate cancer vaccine available for out-licensing.

(5)Compound library transferred to MUSC, Aeterna Zentaris to access future candidates.

Macrilen(macimorelin) AGHD(3)

(9)

Aeterna Zentaris

(10)

Women’s Health – EstroGel

Non-patch transdermal estrogen

therapy product commercialized by

Ascend Therapeutics in the US

Co-promotion agreement with

Ascend in the US

Promotion by Aeterna Zentaris

sales force in the US on a sales

commissions basis

10 Please see full Prescribing Information and boxed warning, or visit

(11)

35 years of worldwide patient use

Approved in over 70 countries

#1 prescribed estrogen product in Europe

#1 prescribed transdermal estrogen product

in Canada

EstroGel

®

is Established Worldwide

(12)

$3.6 billion annual US sales (2013)

Comprised of oral, transdermal, vaginal, IM products

Non-patch transdermal products

$100 million annual sales & fastest growing segment

Promotionally responsive market

Estrogen Replacement Market

12

(13)

Aeterna Zentaris EstroGel

®

Goals & Strategy

Be #1 in every territory we occupy

Sales and promotional campaigns to sustain Ascend’s

share of voice in the marketplace

Expand, defend and further define/differentiate

EstroGel

®

in the US marketplace

(14)

Aeterna Zentaris

(15)

Æterna Zentaris

Growth Hormone Market Overview

Growth hormones (GH) are used to treat growth hormone deficiency

(GHD) in both adult and pediatric

patients

Annually, approximately 40,000 pediatric patients or 0.2% of the child

population are treated with Growth Hormone

$1.6 billion market that is growing 10% annually

Six companies are actively promoting a GH brand

No true generic competition in the market

All products utilize the same molecule,

somatropin

Products are differentiated by mode of delivery, reimbursement, and

patient support programs

(16)

Æterna Zentaris

Saizen

®

Overview

EMD Serono owns the worldwide rights to the Saizen

®

brand

Saizen

®

is indicated for the treatment of GHD in children and

adults

Patients and providers can choose from 1 of 4 delivery options

Saizen

®

is currently the only product available that offers a

needle-free delivery system

Patients receive 24/7 nursing support for device training, and a

dedicated case manager to help resolve access &

reimbursement issues

Saizen

®

is currently supported by an internal commercial team

and promoted by only 8 Key Account Managers

(17)

Æterna Zentaris

AEZS and Ascend Promotional Partnership

Under a five year co-promotion agreement AEZS and Ascend

Therapeutics will allocate time from 53 sales representatives to

promote Saizen

®

Collectively AEZS and Ascend will significantly increase the

field selling of Saizen

®

with EMD Serono maintaining their

existing selling

AEZS/Ascend will be compensated for New Patient Starts,

above an agreed-upon baseline

AEZS/Ascend selling is scheduled to launch Q2 2015

(18)

Aeterna Zentaris

(19)

Zoptarelin Doxorubicin –

Targeted Cytotoxic Therapy

Ref.: Westphalen et al. Int J Oncol. 2000

Binding

Internalization

Nucleus

Migration

Zoptarelin Doxorubicin

Apoptosis induction

Bypassing the

MDR-1 system

19

(20)

Zoptarelin Doxorubicin

– A Potential Breakthrough in Cancer Therapy

Product

New Chemical Entity (NCE) composed of a targeted synthetic peptide

carrier linked to doxorubicin

Value proposition:

Improved, targeted delivery of doxorubicin with improved

benefit-risk profile

Status

ZoptEC Phase 3 trial in endometrial cancer currently enrolling in

North America, Europe and Israel

FDA Special Protocol Assessment (SPA)

CRO Ergomed to assume 30% up to $10 million of clinical and reg. costs

DSMB recommendation to continue ZoptEC trial following interim

analysis, April 2015

(21)

Zoptarelin Doxorubicin

Multiple Potential Applications

21

Source: Cancer Facts & Figures 2015, ACS; literature estimates

Tumor Site Estimated New US Cases 2015

Clinical Evaluation

Endometrium 54,870

Locally advanced, recurrent or metastatic, failure after platinum-taxane

9,000 Phase 3; ZoptEC trial; NCT01767155 (n=500), zoptarelin dox vs doxorubicin

Ovary 21,290

Platinum refractory or resistant 14,180 Phase 2; NCT00569257 (n=42)

Prostate 220,800

Castration and taxane resistant 33,000 Phase 1/2; NCT01240629 (n=55)

Breast 234,190

Chemotherapy refractory triple negative breast cancer

39,800 Phase 2; NCT01698281 Terminated (n=8)

Bladder 74,000

Locally advanced unresectable or metastatic

(22)

Zoptarelin Doxorubicin

Compelling Phase 2 Results

22

AGO-GYN5:

Stratum B : Advanced or recurrent endometrial cancer expressing LHRH receptors Stratum A : Platinum resistant ovarian cancer expressing LHRH receptors

Tumor Site/Study

Zoptarelin Doxorubicin

Doxorubicin

Overall Survival

Cardiotoxicity

Overall Survival

(Literature data)

Cardiotoxicity

(Literature data)

Endometrium

(AGO-GYN5)

15.0 months

0/44 reports

Endometrium

Thigpen et al. (2004), J Clin Oncol 22:3902-3908

9.2 months

7/150 Grade 1 15/150 Grade 2 4/150 Grade 3 2/150 Grade 4

Endometrium

Aapro et al. (2003), Annals of Oncology 14:441-448

7.0 months

1/87 Grade 1 1/87 Grade 3

Ovary

(23)

Potentially the first FDA approved medical therapy for

treating advanced, recurrent endometrial cancer

Providing a medical option for significant unmet

medical needs

Resulting in rapid adoption as a core therapy for patient

treatment & management

Addressing Significant Unmet Medical Needs

(24)

ZoptEC Phase 3 Study Under SPA

Randomize

Median OS

Zopt dox 267 mg/m2 Day 1 of each cycle

Median OS

Doxorubicin 60 mg/m2 Day 1 of each cycle

Evaluation of response (every 3 cycles)

~ 384 events* (Death)

Primary Endpoint Overall Survival (OS)

~ 250 patients

~ 250 patients

Each cycle = 21 days

* Pre-planned interim analysis at ~ 128 events & ~ 192 events

(25)

Target Medical Oncologists

Universe includes 12,500 total

2,000 gynecological oncologists

Vertical Oncology Specialty Sales Force

Segmented strategy focused on high value targets and key

commercial insurers

Zoptarelin doxorubicin alone, 30-50 sales representatives

Zoptarelin doxorubicin + in-license brand, 70-100 sales

representatives

AEZS Commercial Plans in the US

(26)

Significant Market Opportunity Potential

Endometrial Cancer

$300 million – $400 million

Other Cancers

(Ovarian; Prostate; etc.)

> $500 million

26

Estimated US Annual Market Potential

(27)

Clinical development

Full recruitment of patients (n

500) in H1-2015

Second interim analysis in H2-2015 (~192 events)

Commercial plan

Prepare commercial launch in the US and other territories

Establish partnerships in strategic territories (ROW)

Out-licensing agreement with Sinopharm A-Think

for China, including Hong Kong and Macau

Other potential out-licensing agreements to come

Zoptarelin Doxorubicin… Next Steps

(28)

Strengthen patent protection

Process patent filed

Reduction of manufacturing costs >50%

Following successful development in endometrial

cancer therapy…

Ovarian cancer therapy (phase 2 successfully completed)

Prostate cancer therapy (phase 2 ongoing)

Additional indications with clinical data (bladder, breast

cancer)

LHRH-Disorazol Z (new patent protected optimized

follow-up molecule)

Zoptarelin Doxorubicin

… Life Cycle Management

(29)

Aeterna Zentaris

(30)

Aeterna Zentaris

Macrilen

TM

(Macimorelin)

Product

Novel orally-active ghrelin agonist that induces a fasting

patient’s GH secretion

Status

FDA issued a Complete Response Letter related to the

submitted NDA (November 6, 2014)

Following successful discussions with the FDA, Aeterna

Zentaris announced commitment to rapid clinical development

(April 13, 2015)

(31)

Aeterna Zentaris

Macrilen

TM

… Strong Value Proposition in AGHD

Novel

: the

only FDA approved

product for AGHD

(once approved)

Accurate

: comparable to current standard procedures

Safe

: well tolerated

Convenient

vs ITT

− Oral vs IV/IM injections

− Simple: single blood draw possible

− Shorter follow-up

− Less medical supervision

(32)

Aeterna Zentaris

Macrilen

TM

… Next Steps

Submit final protocol to FDA and initiate

confirmatory Phase 3 efficacy and QT trials

Commercial plan for product launch in the US

Core sales force of

20 reps

30 centers for GH evaluation in adults

2,500 specialists to target

(33)

Aeterna Zentaris

(34)

Milestones for 2015

STATUS

Commercial Development

EstroGel

®

revenue growth at 2x segment growth rate

Launch Saizen

®

selling

o

o

Expanding Commercial Portfolio

In-licensing, acquisition and/or promotion of additional registered

products for commercialization by Aeterna Zentaris

o

Resource & Organizational Improvement

Complete implementation of global optimization program to streamline

R&D activities, increase commercial activities and overall workforce

flexibility

Focused on Delivering on our Milestones

34

(35)

Milestones for 2015

STATUS

Zoptarelin doxorubicin

Achieve first interim analysis for ZoptEC trial

Complete patient recruitment for ZoptEC trial

Achieve second interim analysis for ZoptEC trial

o

o

o

Macrilen™

Resolve Go/No Go decision related to clinical development program

through FDA interactions

Initiate confirmatory Phase 3 efficacy trial

o

o

Erk Inhibitor Development Program

Selection of an optimized molecule for development

o

Focused on Delivering on our Milestones

35

(36)

Vision

Transform Aeterna Zentaris from R&D stage to a

commercially operating specialty biopharmaceutical

company through:

Working towards successful development and

commercialization of our pipeline

Pursuing successful in-/out-licensing opportunities

(37)

Improving Life… Transforming Value

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