Capstone Therapeutics (Trade Name of OrthoLogic Corp.)

Transcription

1 AZX100 AZX100 Capstone Therapeutics (Trade Name of OrthoLogic Corp.) Corporate Overview October

2 Safe Harbor Statement Statements in this presentation or otherwise attributable to Capstone Therapeutics regarding our business that are not historical facts are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of These forward-looking statements, which include the timing and acceptability of FDA filings and the efficacy and marketability of potential products, involve risks and uncertainties that could cause actual results to differ materially from predicted results. These risks include: delays in obtaining or inability to obtain FDA, institutional review board or other regulatory approvals of pre-clinical or clinical testing; unfavorable outcomes in our pre-clinical or clinical testing; the development by others of competing technologies and therapeutics that may have greater efficacy or lower cost; delays in obtaining or inability to obtain FDA or other necessary regulatory approval of our products; our inability to successfully and cost effectively develop or outsource manufacturing and marketing of any products we are able to bring to market; changes in FDA or other regulations that affect our ability to obtain regulatory approval of our products, increase our manufacturing costs or limit our ability to market our product; effects on our stock price and liquidity if we are unable to meet the requirements for continued listing on the NASDAQ Global Market; our need for additional capital in the future to fund the continued development of our product candidates; and other factors discussed in our Form 10-K for the fiscal year ended December 31, 2008, and other documents we file with the Securities and Exchange Commission. 2

3 Capstone Overview Capstone is a Clinical-Stage Biopharmaceutical Company AZX100 Multiple Phase 2 Studies in Dermal Scarring (1Q2009) Synthetic peptide with demonstrated effects Anti-fibrotic Smooth muscle relaxation Chrysalin (TP508) Pre-Clinical Proof-of-Concept in Acute Myocardial Infarction Synthetic peptide with demonstrated effects in vascular endothelial dysfunction Excellent safety profile in 565 human subjects in other indications Strong IP portfolio Internal Product Development Capabilities 22 FTEs (7 PhD and/or MD) Experienced management, public company infrastructure Expertise in science, pre-clinical, regulatory, clinical, administration 3

4 Capstone Summary Capstone Report Card 2008 / YTD2009 Achieved All Corporate Objectives AZX100 Successful Phase 1a safety study (30 subjects) Met safety endpoint Successful Phase 1b safety study (40 subjects) Met safety endpoint Significant pharmacologic effect Trend of efficacy in scar reduction Pre-clinical progress in additional high-value indications Published fibroblast mechanism data Initiated Phase 2 clinical program in two key areas of dermal scarring Reached key enrollment goals Chrysalin (TP508) Completed & published landmark pre-clinical proof-of-concept AMI studies Published key studies on cellular effects / mechanism of action Development collaboration process ongoing Restructured staff and programs to optimize efficient use of cash 4

5 AZX100 AZX100 5

6 AZX100 Development Overview AZX100: Scarring, Pulmonary & Vascular Indications Mechanism of Action: HSP20 + PTD Smooth Muscle Relaxation Anti-Fibrotic Clinical Targets: Dermal Scarring Atrophic Hypertrophic Keloid Pulmonary Disease Idiopathic Pulmonary Fibrosis 6

7 AZX100 Dermal Scarring Compelling Rationale Cellular (in vitro) AZX100 decreased expression of CTGF & Type 1 collagen AZX100 decreased stress fiber formation & altered morphology of human dermal keloid fibroblasts Pre-Clinical AZX100 significantly improved collagen organization in Siberian hamster scarring model Clinical Phase 1a & 1b Safety Profile Phase 1b Strong pharmacologic signal & early signal of efficacy Commercial Potential 21 million surgical procedures performed annually in the U.S. that can result in some form of dermal scarring Conclusion: Initiated Phase 2 Program 7

8 Dermal Scarring Market Categories Cosmetic / Aesthetics Post-surgical scars Severe scars (keloids) 20.9 MM U.S. procedures include Burns / Grafts / Donor Cosmetic Surgery Mole / Tumor Removal Reconstructive Surgery Elective, Emergency ER Procedures - Trauma / Injury Other Elective Surgery - Hospital, Office CABG C-Section Scar Revision $BB Market Potential 8

9 Dermal Scarring: Range of Indications Atrophic (Normal) Scars Various types: Flat, pale Sunken Red, dark, raised Result from surgery, burns acne Hypertrophic Scars Red, itching, raised fibrous lesions Typically do not expand beyond boundaries of initial injury May undergo partial spontaneous resolution Common after thermal and other injuries that involve deep dermis Keloid Scars Overgrowth of dense fibrous tissue Extend beyond borders of original wound Do not regress spontaneously Tend to recur after excision 9

10 Keloid Scars The tough problem Progressive growth that extends past original scar Invades and destroys normal skin and tissue Cause of keloid formation still not understood; several theories Alteration in Dermal Environment Collagen Turnover Genetic Immune Dysfunction Little new in treatment of keloids over past 20 years; standard treatment involves: Steroid injection Radiation Pressure 5-fluorouracil 10

11 AZX100 Dermal Scarring Clinical Program Arthroscopic Shoulder Surgery Study OL-ASCAR-03 Objective: Scar reduction in trocar sites; up to n=153 (per protocol) Placebo controlled; two active doses per patient 3.0 mg, 10.0 mg; 9 ± 2 days, 21 ± 2 days following surgery Internal interim analysis 2Q2010 Safety, Quality of Dataset, Definition of Measurement Techniques Keloid Surgery Study OL-ASCAR-04 & -05 Objective: Reduction or prevention of re-formation following keloid scar excision; up to n=120 combined (per protocol) Placebo controlled; four active dose groups 0.3, 1.0, 3.0, 10.0 mg per linear cm; 21 ± 2 days, 42 ± 2 days following excision Both studies fully enrolled Internal interim analysis 1Q2010 & 2Q2010 Safety, Quality of Dataset, Definition of Measurement Techniques FDA Guidelines: 12-month follow-up (4Q2010) 11

12 AZX100 Dermal Scarring Clinical Program Deliverables Primary Endpoint 12 months Secondary Endpoints 2D & 3D Digital Photography - VAS, Height, Length, Volume, Width OL-ASCAR-03 (Shoulder) Interim Analysis: 2Q2010 (Internal Use Only) OL-ASCAR-04 (Keloid Higher Doses) Observational: 1Q2010 (Internal Use Only) OL-ASCAR-05 (Keloid Lower Doses) Observational: 2Q2010 (Internal Use Only) 12

13 AZX100 in Dermal Scarring Pathway to Commercialization Market Characteristics Atrophic Scars Aesthetic; Private Pay Botox, Restylane examples General Surgical Scars Potential for third party reimbursement as adopted under standard of care Keloid Scars Unmet medical need; potential for third party reimbursement Target Audiences General surgeons Cosmetic, reconstructive plastic surgeons Aesthetic dermatologists 13

14 AZX100 in Dermal Scarring Pathway to Commercialization Current Phase 2 program yields results in 2010 CAPS will partner, joint develop, license to conduct future trials Latest Comp in Dermal Scarring: $125MM up-front (cash, equity investment) $700MM milestones 14

15 Chrysalin 15

16 Chrysalin (rusalatide acetate, TP508) in Cardiovascular Disease Elegant, compelling pre-clinical proof-of-concept data in AMI; multiple models Normal cholesterol Hypercholesterolemic Diabetic Solid intellectual property suite Effects replicated with TP508 progeny Partnering outreach & discussions ongoing 16

17 Capstone Financial Summary Capstone Key Financial Facts $41.1MM cash (30-Jun-2009) Within guidance No long-term debt 52-wk Range: $ $1.04 Mkt Cap as of 23-Oct-2009 : ~$32.6MM 17

18 Balance Sheet (in thousands, except per share data) June 30, December 31, (Unaudited) ASSETS Current assets Cash and cash equivalents $ 14,731 $ 23,088 Short-term investments 26,347 22,675 Prepaids and other current assets 835 1,094 Total current assets 41,913 46,857 Furniture and equipment, net Long-term investments - 2,221 Total assets $ 42,289 $ 49,514 LIABILITIES AND STOCKHOLDERS' EQUITY Current liabilities Accounts payable $ 995 $ 1,063 Accrued compensation Other accrued liabilities Total current liabilities 1,853 1,992 Stockholders' Equity Common Stock $.0005 par value; 100,000,000 shares authorized; 40,775,411 in 2009 and 40,775,411 in 2008 shares issued and outstanding Additional paid-in capital 188, ,314 Accumulated deficit (148,076) (140,812) Total stockholders' equity 40,436 47,522 Total liabilities and stockholders' equity $ 42,289 $ 49,514 18

19 Statement of Operations (in thousands, except per share data) Three months ended June 30, Six months ended June 30, As a Development Stage Company August 5, June 30, 2009 OPERATING EXPENSES General and administrative $ 761 $ 742 $ 1,568 $ 1,563 $ 21,643 Research and development 2,579 2,586 6,187 5,028 79,706 Purchased in-process research and development ,311 Other (375) Total operating expenses 3,340 3,328 7,755 6, ,285 Interest and other income, net (224) (565) (491) (1,171) (13,125) Loss from continuing operations 3,116 2,763 7,264 5, ,160 Income tax benefit (7) Loss from continuing operations 3,116 2,763 7,264 5, ,153 Discontinued operations - net gain on sale of the bone device business, net of taxes of $ (2,202) NET LOSS $ 3,116 $ 2,763 $ 7,264 $ 5,420 $ 119,951 Per Share Information: Net loss, basic and diluted $ 0.08 $ 0.07 $ 0.18 $ 0.13 Basic and diluted shares outstanding 40,775 41,186 40,775 41,415 19

20 Capstone Cash Burn Capstone Manages Cash with Discipline 2006 Guidance Orig: $35.0MM Rev: $15.0-$17.5MM Actual $13.4MM 2007 $ $19.0MM $9.6MM 2008 $ $15.0MM $12.6MM (includes $1.041MM stock repurchase) 2009 $ $16.0MM Guidance Reaffirmed 20

21 Capstone Therapeutics Summary 2009 & 2010 Focus: Execution of AZX100 Clinical Trials in Dermal Scarring Clinical Value Creation AZX100 Phase 2 Dermal Scarring Study AZX100 Phase 2 Keloid Studies Investment Thesis AZX100 success (efficacy and ongoing safety) in dermal scarring offers significant upside Downside event protected by cash asset 21

22 NASDAQ: CAPS Capstone Therapeutics 1275 West Washington Street Suite 101 Tempe, AZ (602)