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Investment Opportunity
Non-Confidential Status Update
June, 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

2
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.
3
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.



4
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.

5
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.




6
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.

7
     Near-term value milestones identified.
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

8
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

9
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)‫‏‬



10
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
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



12
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
13
Aestus Core Technology
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
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

16
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
17
      development
Aestus Platform vs. Compound Reprofiling




18
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


19
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))‫‏‬
Intellectual Property
      Three provisional and three non-provisional PCT
      applications filed, including:

       – WO2008/057930A2

       – WO2008/057933A2

       – WO2008/063842A2

       – US 2010/0076037

21
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
22
Supplemental information
Selected data from validation of ATx08-001 and
ATx09-002 in animal models of neuropathic pain
Clinically-validated Neuropathic
     Pain Models




24
     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.

25
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.

26
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).

27

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Aestus Non Confidential Intro 062410

  • 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 2
  • 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. 3
  • 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. 4
  • 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. 5
  • 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. 6
  • 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. 7 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 8
  • 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 9
  • 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)‫‏‬ 10
  • 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 12
  • 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 13
  • 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 16
  • 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 17 development
  • 18. Aestus Platform vs. Compound Reprofiling 18
  • 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 19
  • 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 21
  • 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 22
  • 23. Supplemental information Selected data from validation of ATx08-001 and ATx09-002 in animal models of neuropathic pain
  • 24. Clinically-validated Neuropathic Pain Models 24 Confidential
  • 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. 25
  • 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. 26
  • 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). 27