Improving Life Transforming Value May 2015
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. 2
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 Growth Confirmatory Phase 3 efficacy trial to be initiated before year-end 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 3
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 4
Focused On Value Transformation Expansion of Commercial Operations Initiated selling Ascend s EstroGel in U.S. ZoptEC Interim Analysis Strategic Shift to Commercial Development Focus On Business Development Portfolio Expansion & Development Aeterna Labs Founded Zentaris GmbH Acquired Clinical Development ZoptEC Trial Launched Organization Restructuring; focus on reducing burnrate Leaner, More Focused Organization 1993 2002 2003 2012 2013 2014 2015 5
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+ 6
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 7
Pipeline Supporting Long-Term Growth Product Candidate Discovery Preclinical Phase 1 Phase 2 Phase 3 Zoptarelin doxorubicin Zoptarelin doxorubicin Ovarian (1) and Prostate (2) Cancer Endometrial Cancer Macrilen (macimorelin) AGHD (3) AEZS-120 Prostate Cancer (4) Erk inhibitors LHRH - Disorazol Z Oncology Oncology Compound Library MUSC (5) (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. 8
Aeterna Zentaris EstroGel
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 Please see full Prescribing Information and boxed warning, or visit www.estrogel.com for more information about this product. 10
EstroGel is Established Worldwide 35 years of worldwide patient use Approved in over 70 countries #1 prescribed estrogen product in Europe #1 prescribed transdermal estrogen product in Canada 11
Estrogen Replacement Market $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 Source: Wolters Kluwer 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 13
Aeterna Zentaris Saizen
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 15
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 16
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 Æterna Zentaris 17
Aeterna Zentaris Zoptarelin Doxorubicin
Zoptarelin Doxorubicin Targeted Cytotoxic Therapy Zoptarelin Doxorubicin Binding Bypassing the MDR-1 system Internalization Nucleus Migration Apoptosis induction Ref.: Westphalen et al. Int J Oncol. 2000 19
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 20
Zoptarelin Doxorubicin Multiple Potential Applications Tumor Site Endometrium 54,870 Locally advanced, recurrent or metastatic, failure after platinum-taxane Ovary 21,290 Estimated New US Cases 2015 Clinical Evaluation 9,000 Phase 3; ZoptEC trial; NCT01767155 (n=500), zoptarelin dox vs doxorubicin 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 16,000 Phase 1; NCT01234519 (n=13) Source: Cancer Facts & Figures 2015, ACS; literature estimates 21
Zoptarelin Doxorubicin Compelling Phase 2 Results Tumor Site/Study Endometrium (AGO-GYN5) Zoptarelin Doxorubicin Overall Survival Cardiotoxicity 15.0 months 0/44 reports Overall Survival (Literature data) Doxorubicin Cardiotoxicity (Literature data) 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 Ovary (AGO-GYN5) 15.6 months 0/42 reports 7.0 months 1/87 Grade 1 1/87 Grade 3 AGO-GYN5: Stratum B : Advanced or recurrent endometrial cancer expressing LHRH receptors Stratum A : Platinum resistant ovarian cancer expressing LHRH receptors 22
Addressing Significant Unmet Medical Needs 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 23
ZoptEC Phase 3 Study Under SPA Randomize ~ 250 patients ~ 250 patients Zopt dox 267 mg/m 2 Day 1 of each cycle Evaluation of response (every 3 cycles) Doxorubicin 60 mg/m 2 Day 1 of each cycle ~ 384 events* (Death) Median OS Median OS Primary Endpoint Overall Survival (OS) Each cycle = 21 days * Pre-planned interim analysis at ~ 128 events & ~ 192 events 24
AEZS Commercial Plans in the US 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 25
Significant Market Opportunity Potential Estimated US Annual Market Potential Treating Advanced, Recurrent Cancers Endometrial Cancer Other Cancers (Ovarian; Prostate; etc.) $300 million $400 million > $500 million 26
Zoptarelin Doxorubicin Next Steps 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 27
Zoptarelin Doxorubicin Life Cycle Management 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) 28
Aeterna Zentaris Macrilen TM (Macimorelin)
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 30
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 31
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 32
Aeterna Zentaris Committed to Delivering our Goals
Focused on Delivering on our Milestones Commercial Development Milestones for 2015 EstroGel revenue growth at 2x segment growth rate Launch Saizen selling STATUS o o Expanding Commercial Portfolio In-licensing, acquisition and/or promotion of additional registered products for commercialization by Aeterna Zentaris Resource & Organizational Improvement Complete implementation of global optimization program to streamline R&D activities, increase commercial activities and overall workforce flexibility o o 34
Focused on Delivering on our Milestones Zoptarelin doxorubicin Milestones for 2015 Achieve first interim analysis for ZoptEC trial Complete patient recruitment for ZoptEC trial Achieve second interim analysis for ZoptEC trial Macrilen Resolve Go/No Go decision related to clinical development program through FDA interactions Initiate confirmatory Phase 3 efficacy trial Erk Inhibitor Development Program Selection of an optimized molecule for development STATUS o o o o o o 35
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 36
Improving Life Transforming Value May 1 st, 2015