Improving Life… Transforming Value
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.
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
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
Focused On Value Transformation
Aeterna Labs Founded Zentaris GmbH Acquired Clinical Development Strategic Shift to Commercial Development Expansion of Commercial Operations1993
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.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+
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
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)
Aeterna Zentaris
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
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
$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
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
Aeterna Zentaris
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
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
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
Aeterna Zentaris
Zoptarelin Doxorubicin –
Targeted Cytotoxic Therapy
Ref.: Westphalen et al. Int J Oncol. 2000
Binding
Internalization
Nucleus
Migration
Zoptarelin DoxorubicinApoptosis induction
Bypassing the
MDR-1 system
19Zoptarelin 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
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
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 22:3902-39089.2 months
7/150 Grade 1 15/150 Grade 2 4/150 Grade 3 2/150 Grade 4Endometrium
Aapro et al. (2003), Annals of Oncology 14:441-4487.0 months
1/87 Grade 1 1/87 Grade 3Ovary
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
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
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
Significant Market Opportunity Potential
Endometrial Cancer
$300 million – $400 million
Other Cancers
(Ovarian; Prostate; etc.)
> $500 million
26
Estimated US Annual Market Potential
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
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
Aeterna Zentaris
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)
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
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
Aeterna Zentaris
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
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