Cosciens Biopharma Inc. · TSX:CSCI
Cosciens Biopharma — Aeterna Zentaris — Investor Presentation (November 2013)
41 slides from Cosciens Biopharma's investor presentation, Nov 19, 2013
Slide 1: Exhibit 99.1
Exhibit 99.1 A final This relating Providing in November each Oncology document base of to Therapies the the 2013 shelf &provinces securities does Endocrinology prospectus, not for Unmet of offered, provide Canada. asMedical supplemented full before Adisclosure copy making Needs of the by of anfinal
Slide 2: Forward Looking Statement
Forward This resources the risks and except 2Aeterna regulatory uncertainties, disclaims presentation and ifZentaris Looking we uncertainties to pursue are any process required contains Statement which obligation R&Dand relating could to projects, forward-looking general dotoso cause to update the b
Slide 3: Investment Highlights
Investment A Company commercializing High –Pipeline Multiple Innovative Growth Proven Leadership consistent 3Aeterna AEZS-120: Macimorelin Zoptarelin specialty value, leadership Zentaris product revenue technology Highlights biopharmaceutical wholly doxorubicin: Prostate acetate: treatments candidat
Slide 4: Agenda
Agendaplans Advancing Future Aeterna Zentaris our product 4today pipeline
Slide 5: Aeterna Zentaris at a Glance
spin-out Late and Resource NASDAQ: –Cash Aeterna Addition North Germany: Market Expected endocrinology stage, onAmerica: Zentaris hand ofcap optimization of Asta specialty AEZS, cash 70~$45M Chief asstaff Medica position of at15 5formed TSX: September aScientific Commercial (down biopharmaceutical s
Slide 6: Seasoned Management Team
Seasoned Years Executive Abbott, Experience David Serologicals Jude Cephalon, Paul ICI Senior Nicholas and Dennis 6Aeterna Pharmaceuticals Quality Blake Dinges of A. VP, Turpin BMS, Zentaris J. Dodd Management Title SmithKline Regulatory Senior Pelliccione Affairs Senior and Wyeth, Senior Prior Pres
Slide 7: Strategic Growth Plan
Strategic Work of –Pursue Become operating 7Aeterna AEZS-120 Addition Macimorelin Zoptarelin Integrated Leverage our towards pipeline successful Zentaris aGrowth company specialty of sales commercial doxorubicin Chief successful acetate force Plan in-/out-licensing biopharmaceutical Scientific Comme
Slide 8: Agenda
Agendaplans Advancing Future Aeterna Zentaris our product 8today pipeline
Slide 9: Product Pipeline
Pipeline Product Macimorelin Zoptarelin AEZS-120 Ozarelix Perifosine AEZS-112 AEZS-129-136 AEZS-137 AEZS-138 Cooperation *Compound Aeterna Fully sponsored Candidate Zentaris –Prostate Supporting Multiple doxorubicin Multiple Recombinant Disorazol LHRH library with acetate Erk 9Cancer* NCI through –1
Slide 10: Advancing Our Lead Products
Advancing Lead Product NDA AGHD Macimorelin Designation acetate Estimated Zoptarelin Phase doxorubicin ~ Clinical AEZS-120 Prostate approved cancer Aeterna 1095,000 Potential submitted 3 evaluation (under Indication Trial Zentaris ~annual Endometrial Application Our 505,000 cancer ~Estimated 100,000
Slide 11: Macimorelin: Adult Growth Hormone Deficiency Background
Macimorelin Growth Background body Adult results Symptoms Adverse (accelerated Altered Reduced Increased particularly Current Patient Provoke Conduct Source: Aeterna 11 fat, Growth from in cardiac Diagnosis Navigant Hormone hormone Zentaris lipid GH standard lean bone exercise quality muscle fasting
Slide 12: The Current Test: Insulin Tolerance Test and Unmet Need
Induction The Current Insulin Seizure Elderly –Ischemic Requires Test Potential Need Aeterna Significant Contraindicated Lengthy Labor High Unmet site for administrative Tolerance disorders method intensive Zentaris with alternative medical need heart of procedure Need Growth potential emergency for
Slide 13: Macimorelin Profile and Status
MNDA Novel Product GH Status AGHD -(FDA Phase cancer-induced Aeterna a secretion c iorphan-drug 3orally-active 2 submitted m completed under ongoing Profile Zentaris o r eSPA land cachexia ias 13 n designation) as aghrelin Status cpotential oral e t aghrelin agonist t e treatment agonist that induce
Slide 14: Macimorelin Unique Mode of Action
M Unique hormone sugar GH growth the Macimorelin mimetic (AEZS-130) + Hypothalamus SRIF GHRH Pituitary Ghrelin -IG Liver Stomach Aeterna GF-1 H -apituitary + ac potent mimetic and im hormone Mode potently Zentaris Gland (GH) oprotein rgland regulator directly is acetate eoflrelease aiAction induces
Slide 15: Macimorelin Positive Phase 3 Results
Macimorelin Positive Patients 48 Efficacy results Dosing Sensitivity Adverse events(greater Phase Source: (3%) Aeterna matched 3Operating 0.5 JM Summary trial) Zentaris 50 Phase Most Primary AGHD mg/kg Garcia controls acetate were common than 3 Results 15 efficacy: Characteristic given bitter and pa
Slide 16: Annual AGHD Market Opportunity
Annual A US P T 1Total 2S Aeterna 16ediatrics ource: raumatic dultGHD 36,000 Market Agha Navigant Centers Fernandez-Rodrigues Popovic Zentaris 41,000 Brain tests: etOpportunity for al., Injury et 233,000 2009 al. Disease British AGHD, (adult) (pediatrics) Journal Control foret Frontiers 122,000 GHD
Slide 17: Significant Expansion Opportunity for Traumatic Brain Injury
Significant 215,000 Traumatic An hospitalized GHD evaluation, U.S. *Would ** ***Source: Aeterna Source: Source: estimated alone*** patients fall Zentaris adult Centers Brain into Agha Fernandez-Rodrigues Expansion for Popovic TBI 40,850 patients should aour potential Injury et victims 17 for initial
Slide 18: Current Provocative Tests and Costs
Current Provocative (materials Insulin Glucagon GHRH/Arginine *No Source: Aeterna longer Tolerance Tests Billing Zack Zentaris Stimulation &available Tests physician Consulting Billing (Geref*) 18 TestCosts and Test (ITT) services) Services withdrew (GST) ~$2,000 –from 2013thetomarket $3,200since 20
Slide 19: Macimorelin in Cancer-Induced Cachexia
MPrimary Additional Cachexia performance. Macimorelin weight Proof-of-concept under –Houston Significant Aeterna Phase Double-blind, a c CRADA i in m2A Zentaris Associated leads preclinical oobjectives: Indication unmet trial r acetate eNo lat torandomized, in i19 approved study nthe significant med
Slide 20: Macimorelin Next Steps
MCancer-induced Next NDA Commercial –Continue Aeterna Core 30 2,500 Inducement a ccenters Steps submitted i sales mspecialists Zentaris development o rfor force plan eofl GH to iGH 20 for ncachexia of evaluate toaevaluation release: ~product ctarget e20t areps AGHD t epediatrics launch in adults in
Slide 21: Advancing Our Lead Products
Advancing Lead Product NDA AGHD Macimorelin Designation acetate Estimated Zoptarelin Phase doxorubicin ~ Clinical AEZS-120 Prostate approved cancer Aeterna 95,000 Potential submitted 3 evaluation (under Indication Trial Zentaris ~annual Endometrial Application Our 505,000 cancer ~Estimated 100,000 S
Slide 22: Endometrial Cancer: Unmet Need
Endometrial Develops Disease (the Bleeding Symptoms uncontrollably Difficult Pain The –Prognosis Often Current Stage paclitaxel) Source: Aeterna Stage endometrium) five during III spreads I: Treatments NCI Zentaris year or or when 70-95%, and/or painful IV: sexual discharge Cancer and survival tosur
Slide 23: Large Endometrial Cancer Market Opportunity
Large Endometrial 95k in (U.S., No US Source: ~20% Disease: Recurrent Aeterna 2013 approved and new Market CAN, most invasive Datamonitor Zentaris cases cancer: EU-G5) of product Opportunity Europe gynecologic 23 most 2010 in the cancer
Slide 24: LHRH Receptor Expression in Human Tumor Tissue
LHRH are on system Aeterna many over-expressed Receptor receptors tumors Zentaris reproductive Expression 24 in Human Tumor Tissue
Slide 25: Zoptarelin Doxorubicin Profile and Status
Z CRO Patented Product chemo Completed Status ZoptEC results –America, Exploring Aeterna FDA EMA o p t aagent Special Ergomed Scientific Profile Zentaris Phase rpeptide-drug Europe eclinical Phase l doxorubicin i n3Protocol and dtrial 25 to and Advice 2oefficacy studies assume Status xin Israel ocon
Slide 26: Zoptarelin: Unique Mode of Action
Zoptarelin Unique Allows –•LHRH *Source: Aeterna Breast Endometrial Ovarian Prostate Expressed Only LHRH Companion receptor detectable specific Mode ~ Zentaris agonist Limonta ~ and 60% d80% in odiagnostic bladder of ~xhuman –targeting 80% oAction can 26 ideal in retureproductive al., be bcancer tar
Slide 27: Zoptarelin: Powerful Targeting of Cancer Cells
Zoptarelin Powerful LHRH DOX targeting Binding Migration MDR-1 Cell Aeterna death LHRH targeted Zentaris on agent Trojan to doxorubicin the theLHRH DOX nucleus Horse 27 conjugate receptor for Targeting Cancer Cells
Slide 28: Zoptarelin: Encouraging Phase 2 Results
Zoptarelin Encouraging Phase Patients Efficacy Partial results Stabilization Time 2D IV Adverse Hematological events Overall Source: Aeterna osing infusion to 2response: 267 response survival: survival Emons Summary progression: Zentaris nNo Complete =devery mg/m 43 cardiotoxicity oof Phase xtoxicit
Slide 29: Phase 2 Comparison with Historical First-Line Chemotherapy
Phasepatients Randomized Metastatic Response Study Thigpen Doxorubicin Doxorubicin/cyclophosphamide Fleming Doxorubicin/cisplatin Doxorubicin/cisplatin/paclitaxel Zoptarelin *No Ref.: endometrial Aeterna Temkin 2and Endometrial Zentaris etRate Regimen dEndometrial doxorubicin: al.* cancer. oTrials 2
Slide 30: Phase 3 ZoptEC Study Design
Phase advanced, received first Patients Randomized 2Zoptarelin Dosing IV (doxorubicin) Primary Overall 365 Interim Aeterna 2 infusion events line 3 267 analysis survival Study “ZoptEC” Zentaris nendpoint treatment) one =recurrent devery doxorubicin mg/m 500 oone chemotherapeutic xDesign oat (minimum
Slide 31: Zoptarelin Next Steps
Zoptarelin Next Initiate Phase Interim Continue prostate Pursue doxorubicin –Commercial Aeterna Prepare Establish Steps 3development all results ZoptEC Zentaris cancers the commercial clinical dpartnerships and oplan Phase xinotrial successor 31 2015 rsites 2uof in programs blaunch (~128 aiand endom
Slide 32: Advancing Our Lead Products
Advancing Lead Product NDA AGHD Macimorelin Designation acetate Estimated Zoptarelin Phase doxorubicin ~ Clinical AEZS-120 Prostate approved cancer Aeterna 95,000 Potential submitted 3 evaluation (under Indication Trial Zentaris ~annual Endometrial Application Our 505,000 cancer ~Estimated 100,000 S
Slide 33: Prostate Cancer: Fast Growing Market Opportunity
Prostate Fast 2010 U.S. Europe Japan $3.6B $10.1B Vaccines 9% Aeterna ofGrowing Actual total Zentaris Cancer and$10.1B 2020 immunotherapies Market –Source: Large Forecast market Market Decision in 2020 expected Opportunity Resources to represent 33
Slide 34: AEZS-120: Innovative Product Profile and Status
AEZS-120 Innovative Product –Induces Preclinical, Status prostate Aeterna May bypass Off-the-shelf cellular Profile Zentaris cancer patented planning immunotolerance product (Herlev and and 34 Status live innate initiation Hospital, recombinant Today immune ofthrough Denmark) Phase response oral 1T-
Slide 35: AEZS-120 Mode of Action
Mode AEZS-120 Carrier thyphoid Induction –Adjuvant 1- (Salmonella Secretion antigenic protein All death growth 2- The Recombinant non-toxic DNA toxin Aeterna Salmonella PSA-CtxB Genetically PSA-CtxB sequences fusion (CtxB) or ofstrain: (PSA) inZentaris retardation Action vaccine fusion activity of B
Slide 36: Advantages of AEZS-120
Advantages AEZS-120 Oral Provoke No Aeterna specific administration Zentaris a specific an efficient preparation of AEZS-120 36tumor andneeded long-lasting immuneforresponse each immune patient against response PSALess Potential sensitive to overcome to tolerance keyor hurdles suppressive for cancer
Slide 37: AEZS-120 Next Steps
Next Steps AEZS-120 Initiate Aeterna Phase Zentaris 1 / 37 Proof-of-Concept IND in the U.S. under consideration Establish a proof-of-concept in humans
Slide 38: Agenda
Agendaplans Advancing Future Aeterna Zentaris our product today pipeline 38
Slide 39: Delivering on Milestones
Delivering Milestones C STATUS ® Macimorelin NDA Complete Zoptarelin Agreement First Continue Initiate AEZS-120 Aeterna etrotide Transfer patient submission >Zentaris Phase 50% Phase commercial doxorubicin on of for signed recruited acetate 1Cetrotide our of 2013 /239 Proof-of-Concept clinical to tr
Slide 40: Near-term Value Drivers
Near-term Value Macimorelin Launch Initiate Zoptarelin -ZoptEC Results Proof-of-concept AEZS-120 Aeterna Complete First Drivers interim clinical from in Zentaris trial: the Milestones doxorubicin sites patient Phase acetate U.S. analysis program enrolment 40 inrecruitment as 2humans trial programs a
Slide 41: Vision: Transform Aeterna Zentaris Commercially
Transform Vision commercially Working commercialization Pursuing Aeterna Zentaris towards successful Aeterna operating 41 successful of Zentaris in-/out-licensing ourcompany pipeline from development R&D through: opportunities stage andto a
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