Aeterna Zentaris. 11 th Annual Needham Healthcare Conference April 3, Committed to cure

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1 11 th Annual Needham Healthcare Conference April 3, 2012

2 Forward-Looking Statements This presentation contains forward-looking statements made pursuant to the safe harbor provisions of the U.S. Securities Litigation Reform Act of Forward-looking statements involve known and unknown risks and uncertainties, which could cause Æterna Zentaris 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, the successful and timely completion of clinical studies, the ability of the Company to take advantage of business opportunities in the pharmaceutical industry, uncertainties related to the regulatory process 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 the forward-looking statements. Investors are cautioned not to rely on these forward-looking statements. The Company does not undertake to update these forward-looking statements and disclaims 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, except if we are required to do so by a governmental authority or under applicable law. 2

3 Company Overview Late-stage for various cancers drug development company primarily focused on potential treatments Perifosine: In Phase 3 Negative top-line Phase 3 results in metastatic colorectal cancer: Perifosine failed to demonstrate a benefit in overall survival Multiple myeloma Phase 3 study in collaboration with our partners: We will evaluate and decide with our partners the next steps AEZS-108: Phase 3 endometrial cancer expected to initiate in 2012 Other strategic indications to develop in Phase 1/2 AEZS-130: Phase 3 completed as oral diagnostic for Adult Growth Hormone Deficiency (AGHD) NDA filing expected in 2012 Earlier stage oncology compounds Oral vaccine (AEZS-120) AEZS-112: Improved formulation, new Phase 1 to start ERK/PI3K kinase inhibitors 3

4 Pipeline Compound Indication Preclinical Phase 1 Phase 2 Phase 3 Commercial Perifosine AEZS-108 AEZS-112 AEZS-120 Tumor Vaccine AEZS-129/131-2/136 PI3K/Erk Inhibitor AEZS-137 (disorazol Z) Cetrotide AEZS-130 AEZS-130 Ghrelin Agonist Multiple Myeloma CLL, Renal Cancer & Others Endometrial Cancer Ovarian Cancer Refractory Prostate Cancer Refractory Bladder Cancer Triple Negative Breast Cancer Solid Cancers Prostate Cancer Oncology Oncology In Vitro Fertilization Diagnostic Adult Growth Hormone Deficiency Cancer Cachexia and Other Therapeutic Use Handok (Korea) / Hikma (MENA) / Keryx (N. A.) / Yakult (Japan) Handok (Korea) / Hikma (MENA) / Keryx (N. A.) / Yakult (Japan) Merck Serono, Nippon Kayaku / Shionogi (Japan) 4

5 AEZS-108 5

6 AEZS-108 Rationale LHRH receptor Ideal target for personalized medicine approach Expressed in human cancer tissue (e.g. breast, endometrial, ovarian, prostate and bladder cancer) Only detectable in reproductive tissue and pituitary LHRH agonist can be used for targeting LHRH receptors Companion diagnostic test can be developed 6

7 AEZS-108 Trojan Horse for Cancer Cells AEZS-108 (LHRH targeted DOX conjugate) DOX LHRH targeting agent Binding Internalization Nucleus Migration Ref.: Westphalen et al. Int J Oncol

8 AEZS-108 Phase 2 in Endometrial and Ovarian Cancer Open-label study in patients with advanced or recurrent endometrial and platinum-resistant ovarian cancer Study performed by German Oncology Group (GOG) Two-stage design (Simon Design) with up to 82 patients, 41 patients for each indication Status Response criteria for opening stage 2 were met for both indications The primary endpoint of 5 or more responders was met for both indications Positive ovarian cancer results presented at ASCO 2010 Positive endometrial cancer final results presented at ESGO

9 AEZS-108 Phase 2 Recurrent Endometrial Cancer Patients CR PR SD CBR TTP OS (n) n (%) n (%) n (%) n (%) (months) (months) 42 2 (4.8%) 11 (26.2%) 18 (42.8%) 31 (73.8%) 7 15 Treatment regimen: 267mg/m 2 IV infusion every 3 weeks Toxicity No cardiotoxicity was observed Hematological toxicity was rapidly reversible OS compares favorably to modern triple combination chemotherapy Ref.: P. Wimberger et al. ESGO 2011 G. Emons et al. German Cancer Congress

10 AEZS-108 Phase 2 Endometrial Cancer Historical Comparison Randomized Trials of Combination Chemotherapy in Metastatic Endometrial Cancer Study and Regimen Patients Response Rate (RR) (%) Median OS (months) Thigpen et al.* 356 Doxorubicin Doxorubicin/cyclophosphamide Fleming et al.* 273 Doxorubicin/cisplatin Doxorubicin/cisplatin/paclitaxel AEZS-108**: RR = 31%, TTP = 7 months and OS = 15 months *No patients had received prior therapy with cytotoxic drugs **Ref.: P. Wimberger et al. ESGO 2011 Ref.: Temkin S, Fleming G: Current treatment of metastatic endometrial cancer. Cancer Control 2009;16:3 10

11 AEZS-108 Phase 2 in Recurrent Endometrial Cancer Maximum change from baseline (%) 140% 120% 100% 80% 60% 40% 20% 0% -20% -40% -60% -80% -100% -120% < -30 %: PR Reviewer: not evaluable Reviewer: confirmed > +20 %: PD G. Emons et al. German Cancer Congress

12 AEZS-108 in Advanced Endometrial Cancer Single Phase 3 study planned in recurrent disease following platinum and taxane therapy Comparison against doxorubicin as Caelyx/Doxil currently not available and Myocet not approved in U.S. Primary endpoint: Overall Survival Secondary endpoints: Overall Response Rate Progression Free Survival Safety Companion diagnostic being developed with Ventana 12

13 AEZS-108 Phase 1/2 in Castration- and Taxane-Resistant Prostate Cancer PI Dr. J. Pinski, USC* 13 patients treated with AEZS-108 for up to 8 cycles 3 at 160 mg/m 2 ; 3 at 210 mg/m 2 ; and 7 patients at 267 mg/m 2 Well tolerated at all dose levels and no dose limiting toxicity seen Grade 3 and 4 toxicities were hematologic Early evidence of anti-tumor activity even at low dose level PSA regression in 6 patients (46%); Tumor response per RECIST criteria Drug internalization demonstrated in captured circulating tumor cells After completion of 3 additional patients at 210 mg/m 2, the study to be extended in the Phase 2 portion *S. Liu et al., Poster D3, Abstract #60. ASCO GCS, February 2, 2012, San Francisco 13

14 AEZS-108 Phase 1/2 in Castration- and Taxane-Resistant Prostate Cancer Maximal PSA Response PSA Changes from Baseline 150 Maximal PSA Changes Any Time During Treatment (n=13) Dose Level 1 (n=3) Dose Level 2 (n=3) Dose Level 3 (n=7) Waterfall plot of maximal PSA response J. Pinski et al.; Poster ASCO GU

15 AEZS-108 Internalization Initial 1-3 hours 24 hours AEZS-108 PSA DAPI Merged Co-localization of AEZS-108 with PSA in CTCs 15

16 AEZS-108 Phase 1/2 in Urothelial (Bladder) Cancer Who Failed Platinum-Chemotherapy Phase 1 Phase 2 Maximum tolerated dose (MTD) determination in 4 sequential cohorts of patients (3-6 patients/cohort) AEZS-108 at MTD to determine efficacy in 40 patients AEZS , 160, 210 or 267 mg/m 2, 2-hour intravenous (IV) infusion, Day 1 of 21-day cycles, until toxicity or progression, up to 6 cycles AEZS-108 at MTD 2-hour IV infusion, Day 1 of 21-day cycles, until toxicity or progression, up to 6 cycles 16

17 AEZS

18 AEZS-130 Orally-Administered Ghrelin Agonist Phase 3 as diagnostic test for Adult Growth Hormone Deficiency (AGHD) First oral diagnostic test in Growth Hormone Deficiency Orphan-Drug Designation in U.S. Study completed per SPA agreement Primary efficacy: Achieved > 90% AUC of the Receiver Operating Characteristic (ROC) Curve which determines Specificity and Sensitivity 8 of 10 new AGHD patients correctly classified by prespecified peak GH threshold level 18

19 AEZS-130 Orally-Administered Ghrelin Agonist Next steps Subject to successful ongoing discussions with FDA on NDA structure and format: NDA filing during 2012 Potential for therapeutic indications being studied in cachexia associated with cancer: proof-of-concept study initiated under CRADA with Michael E. DeBakey, VA Medical Center, Houston 19

20 Financials 20

21 Consolidated Results IFRS (in millions of US dollars) (unaudited) Years ended December 31, Revenues Sales and royalties License fees and other Operating Expenses Cost of sales R&D costs, net SG&A expenses Loss from operations (32.2) (24.9) Net finance income (costs) 6.2 (3.6) Income tax expense (1.1) - Net loss (27.1) (28.5) 21

22 Consolidated Cash Flows IFRS (in millions of US dollars) (unaudited) Years ended December 31, Cash and cash equivalents Beginning of year Net cash used in operating activities (26.2) (31.7) Net cash provided by financing activities Net cash provided by investing activities Effect of exchange rate changes - (0.4) Net change in cash and cash equivalents 14.9 (6.1) Cash and cash equivalents End of year

23 2012 Milestones 23

24 2012 Milestones AEZS-108 Define regulatory strategy for endometrial cancer with FDA and EMA through Parallel Scientific Advice procedure Initiate first pivotal program in endometrial cancer (Phase 3) Initiate triple-negative breast cancer Phase 2 study Update on ongoing Phase 1/2 studies in refractory prostate cancer and refractory bladder cancer Update on ongoing companion diagnostic program 24

25 2012 Milestones AEZS-130 File an NDA as diagnostic test for AGHD in the U.S. Update on Phase 2 study in cancer-induced cachexia 25

26 2012 Milestones Others AEZS-120 (oral vaccine) Obtain IND or CTA Initiate Phase 1 in prostate cancer AEZS-112 Initiate Phase 1/2 study with new improved formulation Kinase Inhibitors Selection of clinical candidates 26