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VISIONARY THERAPEUTICS M IKE M ORADI, CEO. Business Summary Founded in 2004 – 21 employees Core Business #1 – Drug Dev for Eye Disease –Lead drug candidate.

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Presentation on theme: "VISIONARY THERAPEUTICS M IKE M ORADI, CEO. Business Summary Founded in 2004 – 21 employees Core Business #1 – Drug Dev for Eye Disease –Lead drug candidate."— Presentation transcript:

1 VISIONARY THERAPEUTICS M IKE M ORADI, CEO

2 Business Summary Founded in 2004 – 21 employees Core Business #1 – Drug Dev for Eye Disease –Lead drug candidate with 2 backups Diabetic Macular Edema (DME) Age-related Macular Degeneration (AMD) Retinal vein occlusion (RVO) Business #2 – Contract R&D (EyeCRO) –Clients in US, EU, Canada, incl. Fortune 500 –China primate CRO launch in 2009

3 Overview CLT-003: IND in 2011, clinical PoC in 2012 –2 backup compounds, 7 assets in pipeline A strategy built for the times –Over $10.5MM in non-dilutive capital to-date –No external investors or debt –$0 burn rate –Profitable specialty CRO, $300K EBITDA in 2008 Seeking $8-12MM Series A or Corporate Partner –May unlock $3-6MM in non-dilutive capital & sales

4 Management Michael Moradi – Co-Founder & CEO Founder of 7 companies, Nanosource acquired by DuPont Jian-Xing Ma, MD, PhD – Founder & Chief Scientist First to use anti-angiogenic compound in eye, $25MM in grants Philippe Margaron, PhD – VP of R&D Former Director of Ocular Research, Preclinical Development, QLT Curt Almen, Chief Financial Officer Former KPMG, private practice, former CFO of 2 other companies Mostafa Analoui, PhD - SVP of Business Development Former SVP of Global Clinical Dev., Site Head for Pfizer Global R&D Peter Gluck, Luce Forward – Ext. IP Counsel Former Chief Patent Counsel for Allergan, General Counsel for AMO Won 2 of 4 largest infringement cases in USPTO history

5 CLT-003 Indications  Diabetic Macular Edema Route of Administration  Intravitreal injection (2-3x / year) Primary Mechanism of Action  HIF 1α Inhibitor – Master switch for angiogenesis and vascular leakage Market Size Potential (U.S.)  $2.0B – $3.0B Competitors  Laser photocoagulation;  Intravitreal steroids and anti-VEGF Competitive Advantages  Upstream to VEGF  Blocks multiple disease targets  Sustained release formulation  Steroid-sparing therapy I.P.  Product by Process & Utility claims/ US Patent pending + PCT filed in 2008

6 Normal Baseline *P<0.05 Left Eye Right Eye Retinal Vascular Permeability in Diabetic Rats 2 days post intravitreal administration. The left eye of each animal received the vehicle, and the right eye received CLT-003.

7 DME Competitive Landscape PIPIIPIII Market Laser Photocoagulation SOC Competitors in Development Kenalog (triamcinolone) Avastin Posurdex (dexamethasone) Macugen Rapamycin Aflibercept (VEGF-Trap) Medidur (fluocinolone) Off-label Lucentis Arxxant Cortiject iCo-007 (antisense) Cortico- steroids Anti- VEGF Other Targeted Therapies Vitrectomy PF (siRNA) Choline Fenofibrate Microplasmin

8 CLT-003 – Key Differentiators New Chemical Entity Upstream from current VEGF drugs in development Early stage treatment for diabetic retinopathy Sustained release formulation Small molecule – low costs of good Chemically stable – can expand into different routes of administration, including eye drops

9 Lead Candidate Timeline ProgramRoAIndication Q3Q4Q1Q2Q3Q4Q1Q2Q3Q4Q1Q2Q3Q4 CLT-003 Intravitreal, sustained release DME CLT-001 CLT-002 Eye drop AMD, Ocular Inflammation :preclinical candidate selection : IND filing: clinical PoC (Phase Ib/IIa): Phase I

10 Summary Clinical Phase Ib/IIa Proof of Concept within 36 months $10.5MM+ in funding, no debt, no external investors –Seeking $8-12MM in funding from VC or strategic partner Capital efficient operation by strong early mgmt. No Approved Therapeutics for DME Charlesson’s CRO provides downside risk mgmt. –12.5x multiple on sale of specialty CROs (m)

11 Backup CLT-003 CRO Exit AMDDMECLT-002 CLT-005 IPFinancials Markets Timeline

12 Indications  Wet AMD/DR Route of Administration  Eye drop Primary Mechanism of Action  Antagonizes expression of VEGF Anti-inflammatory Market Size Potential (U.S.)  $2.0B – $4.0B Competitors  Anti-VEGFs  Laser photocoagulation  Intravitreal steroids Competitive Advantages  Upstream to VEGF  Blocks multiple disease targets  Potentially safer, advantageous dosing  Potential steroid-sparing therapy I.P.  June 2024 (expiration date w/o extension)  Utility patent filed in 2005 CLT-002

13 Indications  Ocular inflammation and Neovascularization  Posterior Uveitis  Dry/Wet AMD Route of Administration  Intravitreal injection Mechanism of Action  Stat3 inhibitor – anti-inflammatory / anti- angiogenesis Market Size Potential (U.S.)  $3.0B – $6.0B Competitors  Intravitreal steroids  Anti-VEGFs  Anti-TNF  ; complement inhibitors Competitive Advantages  New Mechanism of Action that targets upstream events leading to inflammation / angiogenesis  Highly specific safety profile I.P.  Composition of matter & utility claims filed in 2008  US and PCT Pending, with clean novelty search CLT-005

14 CLT-003 A Novel Potent Anti-Leakage Agent CLT-003 reduces retinal vascular leakage in OIR, STZ- diabetes and AMD models CLT-003 blocks activation of HIF-1α CLT-003 down-regulates VEGF and ICAM-1 expression Single injection of CLT-003 in nanoparticles suppresses vascular leakage at 6 weeks. CLT-003 inhibits neovascularization in OIR retina and in CAM assay CLT-003 does not cause detectable toxicities in the eye

15 Normalized Permeability (µg/mg) Normal OIR Rats OIR + CLT-003 (µg/eye) * * Left Eye Right Eye *P<0.05 Dose-dependent Inhibition of Retinal Vascular Permeability in the Rat OIR Model Experiments were performed 2-days following intravitreal administration. The left eye of each animal received the vehicle, and the right eye received CLT-003.

16 Normal Baseline *P<0.05 Left Eye Right Eye Retinal Vascular Permeability in Diabetic Rats Experiments were performed 2 days following intravitreal administration. The left eye of each animal received the vehicle, and the right eye received CLT- 003.

17 Expression of ICAM-1 in ARPE-19 cells

18 Expression of VEGF in ARPE-19 cells

19 CLT-003 attenuates HIF-1α activation in ARPE-19 cells

20 CLT µg/eye Vehicle CLT-003 Inhibits Retinal NV in Rat Model of Retinal ischemia (OIR) Intravitreal injections were performed at P16 and angiography/histology were performed at P20. The left eye of each animal received the vehicle, and the right eye received CLT-003.

21 *P<0.05 Retinal Vascular Permeability in the Vldlr -/- mouse model of AMD Vldlr -/- mice at P98 received an intravitreal injection of CLT-003 (1 µg) in the right eye and an equal volume of vehicle in the left eye. Experiments were performed at 2-days post-injection.

22 * *p< Normal Rats OIR + Vehicle OIR + CLT-003 (1.0 µg/eye) VEGF Concentration (pg/ml) CLT-003 reduces retinal VEGF levels in the rat OIR Model Experiments were performed at 2-days post intravitreal administration and VEGF levels were assessed with a commercial Elisa kit.

23 Normal Rats OIR + Vehicle OIR + CLT-003 (1.0 µg/eye) ICAM-1 Concentration (ng/ml) * *p<0.05 CLT-003 Reduces Retinal ICAM-1 Levels in the Rat OIR Model Experiments were performed at 2-days post intravitreal administration and ICAM-1 levels were assessed with a commercial Elisa kit.

24 Control NPs Left Eye CLT-003 NPs Right Eye Control NPs Bilateral Eyes CLT-003 NPs Bilateral Eyes Retinal Vascular Permeability (ug Evans Blue/ mg protein) n = 6 n = 6 n = 4 n = 4 p = p = CLT-003 NPs: LMW, 50:50, 14.5 % loading, 64 µg/eye * STZ-diabetic Rats 6 Weeks post intravitreal injection CLT-003 NP suppresses retinal vascular leakage 6 weeks after injection *

25 Diabetic Macular Edema DME can occur at early stages of DR and is the #1 cause of vision loss in diabetic patients No satisfactory treatment –Standard of care = laser photocoagulation reduces vision loss in 50% patients –Off-label corticosteroids are less efficacious than laser over time and cause cataract and increased IOP –Anti-VEGF therapies look promising (Phase II) but require frequent injections Need for a new potent and safe, conveniently administered therapy

26 DME Competitive Analysis Expected CLT- 003 Profile Laser Photocoagulation VitrectomyCortico-steroids Anti- VEGF Long-term Retinal SafetyYesNo Unclear Sustainable Therapeutic Effect > 3 Months Yes Formulation Dependent No Targets Multiple Pathogenic Cellular Pathways YesN/R YesNo Retains Efficacy Following Long-term Repeated Treatments YesN/R NoYes Cost-effectivenessYes No

27 What is Diabetic Retinopathy? DR is a common complication of Diabetes Mellitus –Two major changes leading to vision loss in DR: DME and retina NV –Vascular leakage leads to retinal swelling, new vessels –Causes permanent loss of photoreceptor cells = vision loss 5-year market estimation: $6.3billion – Assumes 10% market year 5, 20% AAGR –5.3 million patients in US ~100% of Type I diabetic patients will develop retinopathy ~60% of Type II diabetic patients

28 Histopathology of Diabetic Retinopathy (DR) Degeneration of pericytes Impairment of vascular endothelium – -impaired blood-retinal barrier Increased permeability - vascular leakage -Diabetic Macular Edema Basement membrane thickening Overproliferation of endothelial cells – -neovascularization (NV) – -proliferative diabetic retinopathy (PDR) Hemorrhage, retinal detachment Retinal neuron damage

29 What is Macular Degeneration? AMD afflicts 1/3 of those over age 70, 30-50MM total 3 rd leading cause of blindness (cataracts & glaucoma) –Prevalence to increase by 50% by 2020 Permanent loss of central vision: can’t perform basic tasks Current drugs prevent AMD progression, after vision loss

30 AMD Competition Company (Compound) Route of Admin. Frequency Treats Dry AMD? Treats DME? Cost per Year Genentech (Lucentis) Injection in eye 1-2 monthsNo $22,000 Genentech (Avastin) * (off label) Injection in eye 1-2 monthsNo $1,000? Controversial OSI/EyeTech (Macugen) Injection in eye 1.5 monthsNo $11,000 QLT (Visudyne™) Injection + laser in eye 3 monthsNo $7,500 Charlesson (CLT-002) Topical / eyedrop DailyYes ? Charlesson CLT-003 (ChiroVis™) Sustained Release Injectable 3-6 MonthsYes ?

31 CRO Business Perform non-GLP rodent studies –Diabetic Models –Angiogenesis Models –Retinal Degeneration Models –Ocular tolerance and distribution studies in rabbits Multiple biochemical and biological assays available Unique ability to develop sophisticated animal models.

32 R&D Outsourcing Trends Expedited discovery and development process Cost-efficient alternative –Reduction of capital expenditures Access to critical and unique expertise The global CRO market is expected to increase at a compound annual rate of 12.6%

33 Summary Pro Forma 2008(Actual) Revenue Grant$1,657,045$3,300,359$4,016,922$2,638,125$1,818,125 CRO Rev$354,665$400,000$1,100,000$1,900,000$2,400,000 Total Rev$2,011,711$3,700,359$5,116,922$4,538,125$4,218,125 Operating Expenses R&D / Clinical$1,244,504$4,400,000$6,200,000$8,000,000$9,000,000 G&A$382,516$900,000$1,400,000$2,100,000$2,500,000 CRO Exp$23,314$305,000$335,000$375,000$410,000 Total Exp$1,650,333$5,605,000$7,935,000$10,475,000$11,910,000 Operating Income$361,378($1,904,641)($2,818,078)($5,936,875)($7,691,875)

34 Markets Disease TypePatientsU.S. Market Potential Diabetic Macular Edema2.1 Million$3 Billion Wet AMD1.75 Million$1.6 Billion Diabetic Retinopathy5.6 Million$2.5 Billion TOTAL9.45 Million$7.1 Billion

35 Anticipated Milestones + Timing DatesCost ($k) Complete CMC Q ,400 Complete IND-enabling studiesQ2-111,600 IND filingQ Clinical Proof of Concept (Phase I/II) Q2-122,000

36 Intellectual Property 3 Lead Compounds, 10 patent families –CLT-003 – PCT filed 6/08, Utility 6/07, CIP pending –CLT-005 – PCT & CoM/Utility filed 9/08 –CLT-001 – 2 U.S. pending - partially licensed from OUHSC –CLT-002 – U.S. pending, Utility & Use of Nanoparticles Other IP –CLT-011 partially licensed from UCLA –6 other compounds with provisional/utility filings –ChiroVis™ trademark

37 Recent exits in Diabetes / Ocular CompanyAcquirerValueSummary OculexAllergan$230 MAcquisition of Phase II Macular Edema drug GlenmarkMerck$292MLicense for Phase II diabetes drug, GRC 8200 AlantosAmgen$300MAcquisition of Alantos, Ph 2 Type 2 drug, RegeneronBayer$320 MCollaboration of Phase I/II AMD drug OSiEli Lilly$385M License of PSN010, a preclinical Type 2 diabetes drug CoGenesysTeva$400MAcquisition of CoGenesys for drug platform AngiosynPfizer$571MAcquisition T2TrpRS – AMD drug Eye TechOsi/Pfizer$650 MAcquisition of Macugen™ Phase III AMD drug MacroGenicsEli Lilly$1.1BLicense for teplizumab, a Phase II/III drug SirnaMerck$1.1BAMD siRNA drug, + gene therapy platform

38 Biopharma IPOs in past 24 months CompanyMarket Cap at IPOSummary MAP Pharmaceuticals $ 230 MTwo products starting phase III Sucampo Pharma $ 360 MFDA Approval on small market drug Jazz Pharmaceuticals $ 235 MFDA approved drug for EDS Amicus Therapeutics $ 252 M2 drugs Phase II 1 Drug Phase I Sirtris Pharmaceuticals $ 361 MDiabetes drug finished Phase I Biodel $ 214 MPhase III injectable insulin Elixir Pharmaceuticals $ 281 MPhase III of diabetes & obesity drug Pharmasset $ 368 MMultiple clinical viral products EnteroMedics $ 130 MPhase I/II obesity/gastro drug Average $ 270 M

39 Pfizer Eyetech (wAMD) Novartis Genentech (wAMD) Bayer Regeneron (wAMD) Merck Sirna (wAMD) Novartis QLT (wAMD) Alcon Amgen (Opht. Therapies) Pfizer Angiosyn (wAMD) Allergan Sirna (wAMD + other diseases) OSI Eyetech (wAMD) Hot Therapeutic Area


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