aestus non confidential intro 062410
DESCRIPTION
Updated introduction to Aestus Therapeutics including news on Phase 2 trials of a first-in-class novel pain medication, and investment opportunities.TRANSCRIPT
Investment Opportunity
Non-Confidential Status UpdateJune, 2010
Aestus Therapeutics Inc
Founded in 2005, Aestus is a translational medicine company
focused on the accelerated development of first-in-class
therapeutics for nervous system disorders
The Aestus key proprietary element is our unique systems
biology engine to discover novel targets for the diseases of
interest
Aestus validates these targets using compounds developed for
other indications
Aestus opportunistically in-licenses active compounds from this
target validation for Phase 2 clinical studies
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Management
Tage Honore, President & CEO:
Previously, Vice President at Novo Nordisk, Novartis, and Purdue Pharma.
Brought more than 35 new drug concepts to early clinical studies in multiple disease conditions, of which three were launched.
Managed budgets for a total of $1B, achieving record pipeline productivity in several companies.
Ph D. in Medicinal Chemistry and Doctor of Science in Neurobiology from the Royal Danish School of Pharmacy in Denmark. Business training from European Management Centre and Harvard Business School.
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Management, cont
F. Aaron Dubberley, Director of Intellectual Property:
Previously, McAulay Nissen Goldberg Kiel & Hand (now the New York IP group for Reed Smith), Hoffmann-La Roche, Aventis and senior U.S. patent attorney at Organon International.
Thirteen years of pharma IP experience, including all aspects of patent acquisition, freedom-to-operate, IP due diligence and licensing.
MS. in Biochemistry and Molecular Biology from University of California, Davis, and JD. from Rutgers.
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Management, cont
Dan Lavery, Director, Research and Genomics:
Previously, Director, Molecular Biology, Chromocell Corporation, research and management positions at Glaxo SmithKline and Purdue Pharma
Twenty years of experience in differential gene expression, target ID & validation
Managed $36 million multi-year research collaboration on molecular biology of taste
BA, Johns Hopkins University and PhD., Mt. Sinai Medical School/NYU. Post-doctoral researcher and Lecturer, University of Geneva, Switzerland.
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Status Update
Our most advanced product, ATx08-001, enters clinical phase 2
proof of efficacy studies this quarter for treatment of post-
herpetic neuralgia (PHN) as a first-in-class novel pain
therapeutic.
These studies are funded by a non-dilutive SBIR grant totaling
$2.6 million from NIH.
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Investment Proposition
Aestus is seeking capital to expand our portfolio of products for
neuropathic pain in addition to the current phase 2 pain trial of
ATx08-001.
Proceeds will be used to progress two additional products in
neuropathic pain, ATx09-002 and ATx03-005, which act at
different targets than ATx08-001, in proof-of-concept phase 2
trials on PHN.
Proceeds will also fund pre-clinical and clinical studies on
treatments for schizophrenia and ALS, over the next 2-5 years.
Revenue will be created by sub-license to or collaboration with
pharma in late stage clinical studies, NDA and product launch.
Near-term value milestones identified.7
The Aestus Pipeline
Project Data analysis Compound IDPre-clinical validation
Patent filing, in-licensing
Clinical Proof-of-efficacy
Neuropathic pain
ALS (Lou Gehrig’s
disease)
Schizophrenia
Epilepsy
Other disease areas
ATx08-001, ATx09-002, ATx03-005
4 compounds
30 compounds
~ 1 year ~ 1 year ~ 1.5 – 2 years
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Clinical trial compounds
All three compounds showed robust performance in universally accepted pre-clinical
models of neuropathic pain (Chung, Bennett); excellent safety profile and no serious
adverse events in phase 1 and 2 clinical studies for original indications.
ATx08-001:
• MW: 468.4
• PPAR gamma agonist
• Aestus: Initiating phase 2 trials in PHN this quarter
ATx09-002:
• MW: 481.36
• Glycogen phosphorylase inhibitor
• Aestus: in-license term sheet negotiated
ATx03-005:
• MW 500.54
• Fructose 1,6-bisphosphatase inhibitor
• Aestus: in-license term sheet negotiated
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Investment and Value Creation
$10M investment:
Clinical Phase 2 proof of efficacy of two products (ATx09-002, ATx03-005) for
post-herpetic neuralgia
• Third product, ATx08-001, now beginning Phase 2 clinical trials for PHN,
financed by non-dilutive SBIR grant
Value creation steps:
Out-license of product(s) after successful Phase 2a pain trial (2-3 years)
Partnering for full development of product after successful Phase 2 trials (3+
years)
Acquisition, trade sale or IPO of Aestus (5+ years)
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Five Year Budget
Aestus Therapeutics Inc Five–year Financial Plan (all values in $)
2010 2011 2012 2013 2014
Revenue
SBIR phase 2 grant 1,500,000 1,500,000 0 0 0
Milestone payments to Aestus for ATx08-001 0 0 30,000,000 0 25,000,000
Milestone payments to Aestus for ATx09-002 0 0 30,000,000 0 25,000,000
Milestone payments to Aestus for ATx-Schizo 0 0 0 30,000,000 0
Total Revenue 1,500,000 1,500,000 50,000,000 30,000,000 50,000,000
Product cost
In-license payment for ATx08-001 1,000,000 0 0 0 0
Milestone payment for ATx08-001 0 0 5,000,000 0 5,000,000
In-license payment for ATx09-002 1,000,000 0 0 0 0
Milestone payment for ATx09-002 0 0 15,000,000 12,500,000
In-license payment for ATx-Schizo 0 1,000,000 0 0
Milestone payment for ATx-Schizo 5,000,000 0
Total product cost 2,000,000 1,000,000 20,000,000 5,000,000 17,500,000
Operating expenses
Clinical development costs 3,000,000 4,500,000 1,500,000 0 0
Research to expand portfolio, salaries/ benefits, infrastructure
1,400,000 1,400,000 1,400,000 1,400,000 1,400,000
Total operating expenses 4,400,000 5,900,000 2,900,000 1,400,000 1,400,000
EBIT -4,900,000 -5,400,000 37,100,000 23,600,000 31,100,000
Projections based on terms of comparable pharma license agreements, and assuming successful clinical development and partnering
of three Aestus products.
= milestone payment for entry into Phase 3 trials = milestone payment for NDA filing
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Investment summary
Reduced risk in PHN Phase 2 clinical trials
Exclusive WW license from COM owner in place
Managed by a high-calibre, highly experienced team of industry
executives
Extensive use of out-sourcing to keep burn rate low
Potential sub-licensees already identified and awaiting data
An attractive, balanced portfolio
Products
ATx09-002 ATx08-001 ATx03-005
In-license status Agreed Signed Negotiating
License scope Exclusive Exclusive Exclusive
Field NP Human therapy NP-DN
Territory Worldwide Worldwide Worldwide
Patent life
Composition of matter patent (issued) 2024* 2016* 2019*
Use patent (application) 2027* 2030* 2027*
* = Plus data exclusivity and any available patent term extension
NP = Neuropathic pain
DN = Diabetic neuropathy
ATx In-license and IP Status
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Aestus Core Technology
1. Aestus-proprietary disease-relevant genomics databases
• Also leverage public genomics databases (GEO, EBI)
3. Aestus-proprietary
biologically-informed cluster
analysis
4. Mapping of gene clusters to biological pathways:
a) Known pathways – validate analysis
b) Novel pathways – may be of value for discovery
c) Aestus-proprietary novel association of disease to
well-studied pathways
2. Aestus-proprietary data QC,
statistical meta-analysis across
multiple datasets
a
b
c
The Aestus Engine
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5. Identify Phase 1+ compounds
acting at these pathways,
developed for other indications
4. Mapping of gene clusters to biological pathways:
c) Aestus-proprietary novel association of
disease to well-studied pathways
6. Validate novel pathways using Phase 1+ compounds in
pre-clinical disease models (e.g., pain models above)
- Aestus-proprietary use patents (e.g., Aestus
patent application US2010/0076037)
- In-license for Phase 2 clinical development
The Aestus Engine
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Aestus Discovery & Development Model
Identify novel disease targets through the proprietary Aestus engine for systems biology data-mining
Identify de-risked compounds (Phase 2-ready) developed forother indications, acting at our targets
Validate pathways with these compounds in pre-clinical animal models
Patent active compounds for novel utility (Aestus IP)
Obtain exclusive worldwide license from composition of matter owner
Show proof of efficacy in clinical phase 2 trial
Co-develop/out-license with pharma partner for later-stage development
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Aestus Platform vs. Compound Reprofiling
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Aestus Platform vs. Compound Reprofiling
Aestus has succeeded where compound reprofiling failed:
Using Aestus platform, several novel pain targets were identified and validated with compounds acting at these targets
Aestus approached the composition of matter owner of one compound to negotiate in-licensing agreement for clinical development as pain product
Owner had already engaged a major compound reprofiler to identify other appropriate indications
Six months later and after failure to identify novel indications by reprofiler, owner contacted Aestus to negotiate our proposal
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Third Party Validation
Pain Research collaboration
– (Public traded pharmaceutical company, (name withheld))
Small Business Innovation Research Grants
– Phase I and Phase II (National Institute of Neurological Disorders and Stroke (NINDS))
– $2.6 million non-dilutive funding for Phase 2 clinical trial of Aestus pain product ATx08-001, initiated this year
Edison Innovation Research and Development Grant
– (New Jersey Commission on Science and Technology (NJCST))
– $500K non-dilutive funding
License partners
– (Four publicly traded Pharmaceutical companies, (names withheld))20
Intellectual Property
Three provisional and three non-provisional PCT applications filed, including:
– WO2008/057930A2
– WO2008/057933A2
– WO2008/063842A2
– US 2010/0076037
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Infrastructure
Located in Commercialization Centre for Innovative Technologies, New Brunswick, NJ
• Cost effective, state supported, located on US Highway 1
5 full time employees on payroll:
• Tage Honore, President & CEO
• Aaron Dubberley, Director Intellectual Properties
• Daniel Lavery, Director Research & Genomics
• Meredith Prysak, Project management
• Kathy Kerrigan, Administrative Assistant
3 further employees to be employed in the future:
• VP Commercial
• Director Informatics
• VP Clinical
All others are advisors and consultants22
Supplemental information
Selected data from validation of ATx08-001 and ATx09-002 in animal models of neuropathic pain
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Clinically-validated Neuropathic
Pain Models
Confidential
ATx08-001 in chronic pain model
ATx08-001 reverses pain behavior (cold allodynia) in rats in the Bennett chronic constriction model of neuropathic pain. Reversal of pain behavior by ATx08-001 was equal to or greater than equivalent doses of the positive control pain medication, carbamazepine. Error bars, SEM; * = p < 0.05 compared to vehicle.
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ATx09-002 in chronic pain model (I)
ATx09-002 reverses pain behavior (cold allodynia) in rats in the Bennett chronic constriction model in a dose-dependent manner. Reversal of pain behavior by ATx09-002 at 100 mg/kg was equal to that of 100 mg/kg carbamazepine, the positive control. Error bars = SEM; * = p < 0.05 versus vehicle.
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ATx09-002 in chronic pain model (II)
ATx09-002 reverses pain behavior (mechanical allodynia) in rats in the Chung spinal nerve ligation model of neuropathic pain. Reversal of pain behavior by ATx09-002 (30 mg/kg) was greater than that by greater doses of the positive control pain medication, carbamazepine (100 mg/kg; * = p < 0.05, ATx09-002 vs. carbamazepine; error bars = SEM).
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