2. 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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3. 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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4. 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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5. 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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6. 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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7. 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.
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Near-term value milestones identified.
8. The Aestus Pipeline
Pre-clinical Patent filing, Clinical Proof-
Project Data analysis Compound ID validation in-licensing of-efficacy
Neuropathic ATx08-001, ATx09-002, ATx03-005
pain
ALS
(Lou Gehrig’s 4 compounds
disease)
Schizophrenia 30 compounds
Epilepsy
Other disease
areas
~ 1 year ~ 1 year ~ 1.5 – 2 years
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9. 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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10. 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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11. 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
11 of three Aestus products.
= milestone payment for entry into Phase 3 trials = milestone payment for NDA filing
12. 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
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13. ATx In-license and IP Status
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
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15. The Aestus Engine
1. Aestus-proprietary disease-relevant genomics databases 2. Aestus-proprietary data QC,
• Also leverage public genomics databases (GEO, EBI) statistical meta-analysis across
multiple datasets
c
a
b
4. Mapping of gene clusters to biological pathways: 3. Aestus-proprietary
a) Known pathways – validate analysis biologically-informed cluster
b) Novel pathways – may be of value for discovery analysis
c) Aestus-proprietary novel association of disease to
15 well-studied pathways
16. The Aestus Engine
4. Mapping of gene clusters to biological pathways:
c) Aestus-proprietary novel association of
disease to well-studied pathways
5. Identify Phase 1+ compounds 6. Validate novel pathways using Phase 1+ compounds in
acting at these pathways, pre-clinical disease models (e.g., pain models above)
developed for other indications - Aestus-proprietary use patents (e.g., Aestus
patent application US2010/0076037)
- In-license for Phase 2 clinical development
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17. 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 for
other 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
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development
19. 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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20. 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,
20 (names withheld))
21. 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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22. 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 consultants
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25. 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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26. 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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27. 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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