Download Zoptarelin doxorubicin

Providing Therapies for Unmet Medical Needs
in Oncology & Endocrinology
April, 2014
Forward Looking Statement
This presentation contains forward-looking statements made pursuant to the safe harbour
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, 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 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 rely 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.
Aeterna Zentaris
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Investment Highlights
Company

A specialty biopharmaceutical company focused on developing and
commercializing treatments in oncology and endocrinology
Pipeline

High-value, wholly-owned, late-stage product candidates
–
–

MACRILENTM (macimorelin): Orphan Drug designation to evaluate GH deficiency in
adults (AGHD) / NDA under review
Zoptarelin doxorubicin: targeted therapy for endometrial cancer / Phase 3 (under SPA
with FDA)
Multiple additional product candidates & indications in development
Growth

Innovative technology platform supporting long-term growth
Leadership

Proven leadership with expertise in delivering rapid and
consistent revenue and earnings growth
Aeterna Zentaris
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Aeterna Zentaris at a Glance
 Late-stage specialty biopharmaceutical company
 Heavily focused on establishing revenues, cash and
profitability
– In-licensing of products, product acquisition and co-promotions
 Resource optimization / burn reduction is a priority
 NASDAQ: AEZS, TSX: AEZ
– Market cap ~$70M
 Cash on hand as of Dec. 31, 2013: $43.2M with no debt
 Financing completed in Jan. 2014: $12.2M
in net proceeds
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Leadership with Significant Industry Success and Expertise
Executive
Title
Prior Affiliation(s)
Years of
Experience
David Dodd
President and CEO
Abbott, BMS, Wyeth, Solvay,
Serologicals and others
30+
Jude Dinges
Senior VP and CCO
Merck, Novartis, Amgen
25+
Richard Sachse
Senior VP and CSO
Boehringer Ingelheim, Bayer,
Schwarz Pharma and UCB
20+
Dennis Turpin
Senior VP and CFO
Coopers & Lybrand
25+
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Strategic Growth Plan
 Pursue successful development and commercialization of our
pipeline
– MACRILENTM
– Zoptarelin doxorubicin
 Achieve successful in-/out-licensing opportunities
– Integrated commercial activities
– Leverage sales force
 Become a growth oriented specialty biopharmaceutical
company
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Pipeline – Supporting Long-Term Growth
Product Candidate
Discovery
Preclinical
Phase 1
Phase 2
MACRILENTM (macimorelin)
NDA
Evaluate AGHD
Cancer Induced Cachexia (1)
Macimorelin
Zoptarelin doxorubicin
Endometrial Cancer
Ovarian(2) and Prostate(1) Cancer
Zoptarelin doxorubicin
AEZS-120
Phase 3
Prostate Cancer
Prostate Cancer(3)
Ozarelix
Multiple Cancers(3)
Perifosine
AEZS-129-136 - Erk/PI3K Inhibitors
Oncology
AEZS-137 - Disorazol Z
Oncology
AEZS-138 - LHRH - Disorazol Z
Oncology
Compound library
(1) Investigator-driven and sponsored
Aeterna Zentaris
(2) Phase 2 in ovarian cancer completed
(3) Sponsored entirely by license partners
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Macimorelin
Product Profile and Status
Product
 Novel orally-active ghrelin agonist that induces a fasting patient’s
GH secretion
 Phase 3 completed in AGHD under SPA
Status
 NDA under review by the FDA as an oral ghrelin agonist for the
evaluation of AGHD, under the trade name MACRILENTM
– Canadian registration to be pursued ASAP
– Pediatric indication to be pursued ASAP
 Investigator-driven Phase 2 trial as a potential
treatment for cancer-induced cachexia
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Annual Market Opportunity for AGHD Evaluation
US
Existing Evaluations in Adults1
36,000
Potential Evaluations Resulting from
Traumatic Brain Injury in Adults2
180,000
 216,000
1
Source: Navigant, 2009
Source: Centers for Disease Control and Prevention (CDC), 2010
2 Source: Fernandez-Rodrigues et al., Frontiers in Endocrinology, 2011
2 Source: Agha et al., British Journal of Neurosurgery, 2007
2 Source: Popovic et al., AGHD, Frontiers of Hormone Research, Basel, Karger, 2005
2
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Significant Expansion Opportunity for AGHD Evaluation
Traumatic Brain Injury (TBI) – U.S. Annually
 950,000 emergency department visits or hospitalizations
resulting from TBI event1
  1/3 will have moderate to severe TBI with  60,000
developing GHD2-3
 GHD patients should be evaluated at 3, 6 and 12 months
following TBI, for a potential total of 180,000 annual tests in
the U.S. alone4
Falls into our anticipated approved indication
1
Source: Centers for Disease Control and Prevention (CDC), 2010
Source: Fernandez-Rodrigues et al., Frontiers in Endocrinology, 2011
3 Source: Agha et al., British Journal of Neurosurgery, 2007
4 Source: Popovic et al., AGHD, Frontiers of Hormone Research, Basel, Karger, 2005
2
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Current Tests Billing Costs
Provocative Tests
Total Current Billing Costs
(materials & physician services)
Insulin Tolerance Test (ITT)
~$2,000 to $3,200
Glucagon Stimulation Test (GST)
Source: Zack Consulting Services – 2013
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MACRILENTM
Next Steps
 NDA under review to evaluate AGHD
– PDUFA date: November 5, 2014
 Commercial plan for product launch in the U.S.
–
–
–
–
Core sales force of  20 reps
30 centers for GH evaluation in adults
2,500 specialists to target
Achieve highest share of promotional activity within the category
(50% - 75%)
 Pursue registration to evaluate AGHD in Canada
 Continue development in pediatric use and
for other territories
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LHRH Receptor Expression in Human Tumor Tissue
LHRH receptors
Aeterna Zentaris
LHRH receptors
are over-expressed
on many reproductive
system tumors
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Zoptarelin Doxorubicin
Product Profile and Status
Product
 Patented peptide-drug conjugate (PDC) targets receptors delivering potent
chemo agent doxorubicin to tumor cells
 Focused on delivering targeted chemotherapy with reduced adverse events
Status
 Completed Phase 2 studies in endometrial and ovarian cancer with positive
results
 ZoptEC Phase 3 trial in endometrial cancer currently enrolling in North
America, Europe and Israel
– FDA Special Protocol Assessment (SPA)
– EMA Scientific Advice and Voluntary Harmonization Process
– CRO Ergomed to assume 30% up to $10M of clinical and regulatory costs
 Exploring clinical efficacy in other LHRH-receptor positive cancers
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Zoptarelin Doxorubicin
Phase 3 Study Design in Recurrent Endometrial Cancer Under SPA
Phase 3 “ZoptEC” (Zoptarelin doxorubicin in Endometrial Cancer) trial in
women with advanced, recurrent or metastatic endometrial who have progressed
and who have received one chemotherapeutic regimen with platinum and taxane
(either as adjuvant or first line treatment)
Patients
 n = 500
 Randomized one to one
 Zoptarelin doxorubicin against doxorubicin
Dosing
 267 mg/m2 (zoptarelin doxorubicin) against 60 mg/m2
(doxorubicin)
 IV infusion every 3 weeks
Primary endpoint
 Overall survival (minimum 3 month improvement)
 384 events
Interim analysis at ~128 events and ~192 events
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Zoptarelin Doxorubicin
Next Steps
 Activate all clinical sites and associated recruitment for the
Phase 3 ZoptEC trial in endometrial cancer
 Interim results in 2015 (~128 events)
 Continue the sponsored investigator-driven Phase 2 program in
prostate cancer
 Commercial plan
– Prepare commercial launch in North America and Europe
– Establish partnerships in ROW
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Delivering on our Milestones
Milestones for 2013
STATUS
Cetrotide®
 Transfer of Cetrotide® manufacturing rights to Merck KGaA
o

MACRILENTM
 NDA submission to evaluate AGHD
 Complete commercial plans for initial product launch
o

o

Macimorelin
 Continue Phase 2 trial in cancer-induced cachexia
o

Zoptarelin doxorubicin
 Agreement signed with Ergomed as CRO
 First patient recruited for the Phase 3 ZoptEC trial in endometrial cancer
 Initiate > 50% of clinical sites
 Continue Phase 2 programs in prostate and triple-negative breast cancers

o

o

o

o
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Delivering on our Milestones
Milestones for 2014
STATUS
MACRILENTM
 NDA review completed in AGHD
 Complete pre-launch activities in AGHD
 Development plan to expand opportunity in other regions & indications
o
o
o
Macimorelin
 Results from investigator-driven Phase 2 in cancer-induced cachexia
o
Zoptarelin doxorubicin
 Complete clinical sites initiation (over 100 sites) for ZoptEC
Phase 3 trial
 Reach expected patient enrolment to secure a first interim analysis for
ZoptEC Phase 3 trial in H1-2015
 Results from the investigator-driven Phase 2 study in prostate cancer
Aeterna Zentaris
o
o
o
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Value Drivers
MACRILENTM
 U.S. launch as an oral ghrelin agonist inducing GH release to evaluate AGHD
 Canadian registration for evaluating AGHD
 Expand development and application to evaluation of GHD in children
Zoptarelin doxorubicin
 ZoptEC Phase 3 trial:
− First interim analysis (128 events)
− Second interim analysis (180 events)
− Results (365 events)
 Phase 2 results in prostate cancer
Addition of commercial products
 In-licensing products, product acquisition or co-promotions
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Vision
Transform Aeterna Zentaris from R&D stage to a
commercially operating company through:
 Working towards successful development and
commercialization of our pipeline
 Pursuing successful in-/out-licensing opportunities
Aeterna Zentaris
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