A biopharmaceutical company developing innovative therapeutics in the fields of oncology and infectious diseases (TSX:AMB) Corporate Presentation Last update March 14, 2007
A biopharmaceutical company developing innovative therapeutics in the fields of oncology and infectious diseases
(TSX:AMB)
0
Corporate Presentation Last update March 14, 2007
This presentation contains forward-looking statements that reflect the Company’s current expectation regarding future events. The forward-looking statements involve risks and uncertainties. Actual events could differ materially from those projected herein and depend on a number of factors including, but not limited to, changing market conditions, successful and timely completion of clinical studies, uncertainties related to the regulatory approval process, establishment of corporate alliances and other risks detailed from time to time in the Company’s filings. Such statements are also based on various assumptions, including the successful and timely completion of clinical studies on Ambrilia’s products demonstrating efficacy and safety for human use, their successful commercialization within the forecasted timelines and the attainment of the forecasted milestone payments and other revenues. While Ambrilia anticipates that subsequent events and developments may cause Ambrilia’s views to change, Ambrilia specifically disclaims any obligation to update these forward‑looking statements.
Ambrilia’s Forward-Looking Statements
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Investment Highlights
Lead HIV/AIDS proprietary product licensed to top Pharma, Merck & Co. for $17 million upfront, potential $212 million (U.S.) in milestones, and royalties
Near-term product revenue opportunities from two new formulations of existing drugs, Octreotide and Goserelin
2 leading Pharma companies as licensees, TEVA and Mallinckrodt, of improved formulation of Octreotide
Product pipeline addressing infectious disease and cancer
4(1) Licensed to Mallinckrodt (U.S.), TEVA (France, Germany, Benelux, Spain, Scandinavia), Archimedes
Pharma (U.K.), Gerolymatos (Greece, Turkey, other export countries), Kwidza Pharma (Austria), Pharmis (Portugal, Brazil)
(2) Licensed to Merck & Co. worldwide
Pipeline Overview
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Ambrilia & Merck: Licensing Agreement
Terms
• Merck receives exclusive worldwide rights to Ambrilia’s
HIV/AIDS PI Program, incl. lead compound PPL-100
• Upfront licensing fee: $US 17M, additional milestones:
$US 212M
• Royalties on sales
• Merck assumes all future development costs
• Potential additional payments and royalties on
development and commercialization of each related
compound
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PPL-100: Market potential
• HIV/AIDS market(1) estimated to reach US$ 9 billion
at the projected time of launch of PPL-100 (2012)
• PIs: a healthy 1/3 of the HIV/AIDS treatment options - Today’s Market leader Abbott’s Kaletra® : US$ 1.0 B(2)
• PPL-100 market share and revenues expected to be significant
based on its competitive advantages:
Excellent safety and tolerability
Favorable cross-resistance profile
High genetic barrier
Potential once-a-day dosage, ritonavir free
(1) Decision Resource – 2004 HIV Infectious Diseases Study #68, Frost & Sullivan 2004 Strategic Analysis World HIV Market, 2006 DataMonitor - Pipeline Insight HIV
(2) Life Science Analytics – Medtrack Database
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PPL-100: Phase I• Phase I single dose escalation study in healthy volunteers
With neat drug filled capsules, PPL-100 demonstrated a favorable PK profile (elimination half life from 30-37 hours) and the potential as a once daily PI without ritonavir boosting PPL-100 was generally safe and well tolerated up to 2400 mg
--- Estimated protein binding adjusted EC90 against wild-type virus (~ 40-60ng/mL)Ctrough for both 600mg and 1200mg doses above EC90
0 4 8 12 16 20 2410
100
1000
600 mg600 mg + light meal
Time (h)
Pla
sma
PL
-100
(n
g/m
L)
0 4 8 12 16 20 2410
100
1000
1200 mg1200 mg + light meal
Time (h)
Pla
sma
PL
-100
(n
g/m
L)
C24 ~ 67 ng/mL C24 ~ 75 ng/mL
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PPL-100: Next Steps
• Phase 1 repeat dosing study in healthy volunteers
completed by Ambrilia
Good safety profile: only mild (grade 1) adverse events and
no moderate or severe cardiovascular, lipidic or hepatic adverse
events observed PK data support the potential for once or twice daily treatment,
without ritonavir boosting, for PI-naïve and PI-experienced
HIV/AIDS patients
• Merck has initiated formulation development
• Further development in patients will follow
Significant near-term product revenues from new formulations of existing drugs
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Oncology: Improved formulation of Octreotide
• Therapeutic alternative to Novartis’ Sandostatin®LAR
(somatostatin analogue)• Indicated for acromegaly and some digestive tumors
• 2006 Sandostatin® sales(1) : ~ $US 1B (+ 4 %), over 70% for the LAR
• Three dosages of LAR: 10, 20 and 30 mg per unit
• 30mg (over 50% of market) sells for $US 2,830 per unit
• 13 injections needed per year ( $US 25,000 to > $US 36,000)
• Improved prolonged release formulation developed with patented technology (C2L)
Much simpler and easier to use
With pharmacokinetic advantages
Manufactured in-house, cGMP compliant
(1) Novartis Annual Report 2006
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Mean plasma concentrations in healthy volunteers (12/12)
0
500
1000
1500
2000
0 7 14 21 28 35 42 49 56 63
Days
Oc
tre
oti
de
C2L-oct
S LAR
Superior bioavailability
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Calculation supports use of C2L every 6 weeks
Third injection
C2L (42D) vs SLAR (28D)
1
1000
0 20 40 60 80 100 120 140
Time (days)
Oct
reo
tid
e (p
g/m
l)
TEST
SLAR
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Octreotide: Clinical Plan
• Limited Phase 3 clinical studies
• in Europe and in the U.S.
• 3 months activity + 3 months safety follow-up
• Endpoints: Surrogate marker (Growth Hormone & IGF-1 levels)
• Upcoming milestones
• Start of clinical studies in acromegaly patients
• Filing in Europe and in the U.S. H1 2008
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Octreotide: Marketing Strategy
• Ambrilia manufactures and will sell the finished product to its licensees, at contractually fixed price
• Licensing agreements concluded with leading companies worldwide
• Agreements have been concluded for commercialization and have already generated
milestone payments
• Mallinckrodt (U.S.), TEVA (France, Germany, Benelux, Spain, Scandinavia),
Archimedes Pharma (U.K.), Gerolymatos (Greece, Turkey, other export countries),
Kwidza Pharma (Austria), Pharmis (Portugal, Brazil)
• C2L to be promoted as improved prolonged release
formulation, with three advantages : less expensive, easier to
use because more stable product, less frequent injections (8 or 9 instead of 13 per year)
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Oncology: New formulation of Goserelin
• Potential first-to-market therapeutic alternative to
Astra Zeneca’s Zoladex LP® (LHRH analogue)
• Indicated for hormone sensitive prostate cancer
• 3-month prolonged release formulation developed with
patented technology
• Zoladex® 2005 sales(1) : US$ 1.0 B (+9%), primarily in Europe
(70% for the 3 month formulation)
(1) Life Science Analytics – Medtrack Database
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Goserelin: Developments
• Upcoming Milestones
• In house formulation optimization Ongoing
• Human PK study, single dose 2007
• Clinical study in prostate cancer patients H2 2007
• Licensing-out H1 2008
• Filing in Europe H2 2008
Proprietary products in cancer and infectious diseases
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Oncology: PCK3145
• Therapeutic anti-cancer peptide with signal transduction
mediated effects on tumor metastasis
• Indicated for hormone-resistant prostate cancer, a condition
with very poor prognosis.
• Studied in close to 50 patients
Safe, well-tolerated with some disease stabilization observed
• Completed initial U.S. pilot study Improved dosing Study fully enrolled
- Results expected in H1 2007
- Safety confirmed, and potential efficacy signals
• Development partnership discussions ongoing
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PSP94 Test: Novel Biomarker Assay
• A test to measure the plasma levels of PSP94, a powerful new prostate cancer prognosticator
Confirmed diagnostic value in published study in patients with prostate cancer
Confirmed prognostic value in patients with risk of recurrence
• The PSP94 kit can help
Avoid biopsies in patients diagnosed with probable prostate cancer Help follow-up patients in observation before deciding to treat
• Partnership negotiations ongoing
• Potential launch in Europe in 2007
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Oncology: TVT (Tumor Vasculature Targeting) Platform
• NGR peptides-based targeted drug delivery technology that delivers anti-cancer drugs to the vasculature of solid tumors and surrounding tumor cells
Technology consists of a carrier vesicle coated with a tumor homing peptide (NGR)
which binds specifically to a receptor (CD13) exclusively expressed in tumor cells and
newly formed tumor blood vessels
The carrier is then internalized and releases the anti-cancer drug which, in turn,
specifically damages the tumor vasculature and kills the tumor
• Targeted NGR peptide can be attached to the surface of liposomes or other nanoparticles loaded with a single or combination of anti-cancer agents
• Proof-of-concept with Doxorubicine (Dox)• NGR targeted liposomal Dox with convincing results in animal solid tumors models
• Next Steps• Term Sheet negotiation for TVT technology by year-end
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HIV Integrase Inhibitor Program
• Integrase Inhibitor is a high-profile platform for the
treatment of HIV/AIDS
• Merck and Gilead have Integrase Inhibitor products in
late-stage development for the treatment of HIV
• Ambrilia to utilize Integrase Inhibitor Program to
develop drugs to treat HIV/AIDS
• Status: Lead optimization
• Novel structures and new mechanism of action
• 12 months to a drug candidate
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Financial Snapshot
Ticker Symbol (TSX) AMB
Recent share price CDN$ 3.00
Market capitalization ~ CDN$ 87 M
Shares outstanding 29 M
Cash available (Q3/06) Incl. US$ 17 M Merck upfront
CDN$ 27.7 M
Months of cash (Q3/06) >18
Analyst Coverage
BUY Recommendation As of Dec. 01, 2006
David Martin, Dundee Securities Nigel deGruyther, LOM Douglas Loe, Versant Partners
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AMB-T vs. U.S. & C.A. Biotech Indices
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01-mars-06
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AMB NBI BTK TTHC
Since 3/01/06: AMB-T +73.9% Nasdaq Biotech Index (NBI): -4.0%Amex Biotech Index (BTK): +5.0%S&P/TSX Capped Healthcare Index (TTHC): -8.2%
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Management Team
Hans J. MäderPresident & CEOHeld numerous senior executive positions at Sandoz / Novartis.
Monique Létourneau, CFA, M.Sc., Exec. VP, Finance & CFOFormerly held financial executive positions with Power Corporation of Canada and M&A Group at Deloitte and Touche.
Bonabes de Rougé, M.D. Senior Exec. VP & CSOFormerly President and Chief Executive Officer of Cellpep S.A.
Chandra J. Panchal, Ph.D. Exec. VP, Bus. Develop., Licensing & IPCo-founder of Procyon, scientist and former group leader at John Labatt Ltd.
Richard La Rue, LL.L, D.D.N. VP, Legal Affairs, HR & Corporate SecretaryLawyer with > 30 yrs of national and international experience as a private practitioner and corporate counsel.
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Summary – Upcoming Milestones
2007 • PPL-100 – Out-licensing • Octreotide - Start of clinical trials • PCK 3145 - Clinical results (4 months exposure) H1• PSP94 Assay – Licensing • TVT technology - Term-sheet negotiation H2
2008• Octreotide – Regulatory filing worldwide H1• Goserelin – Out-licensing H1• Goserelin – Regulatory filing Europe H2• TVT – 2nd pay-load out-licensing H2
2009• Octreotide – Worldwide launch • Goserelin – European launch • TVT – 3rd pay-load out-licensing
A biopharmaceutical company developing innovative therapeutics in the fields of oncology and infectious diseases
(TSX:AMB)
www.ambrilia.com