Slide 1: Capstone Therapeutics Operating Update
Capstone Therapeutics (OTCQB: CAPS) Operating Update August 13, 2015 Capstone / Lipimetix
Slide 2: Safe Harbor Statement
Safe Harbor Statement Statements in this presentation or otherwise attributable to Capstone regarding our business that are not historical facts are forward looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking s
Slide 3: Corporate Facts
Corporate Facts > Capstone: Clinical Stage Biopharma in Peptide Therapeutics OTCQB: CAPS Based in Tempe, AZ Virtual business model: 2 FTEs / 3 Consultants Market Cap as of August 12, 2015: = S9MM $1.0MM cash at June 30, 2015 = No Long Term Debt > Lipimetix Development LLC (JV) = Based in Natick, MA
Slide 4: Milestones Over Last Months
Milestones Over Last 12 Months i. Reported Phase 1a/1b/2a Human Clinical Studies (51 Subjects) > Met All Study Objectives > Powerful and Rapid Reduction in VLDL and Triglycerides > Achieved Substantially On Schedule / On Budget Discovered Next Gen AEM-28/ New Formulation (AEM 28-14) > More Efficacio
Slide 5: Cash Report
Cash Report CAPS $ (393,000) Lipimetix $ (110,000) Consolidated $ (503,000) Cash Balance $ 1,037,000 Capstone / Lipimetix
Slide 6: Capitalization Status
Capitalization Status > Registered Unit Offering *S-1 Filed / Public Document «Awaiting Investment Bank FINRA Clearance «Not Yet Taking Investor Meetings or Raising Capital =Currently in Waiting Period Fundraising Road Show Targeted For September 2015 Capstone / Lipimetix
Slide 7: Molecule
MOLECULE AEM-28 is Derived from Native Apo E AEM-28 (Ac-hE18A-NH,) is a 28-amino acid peptide constructed from 2-domains of native Apo E Hinge region WSIS 6 . angie — Major Ipd-binding am regon 88 Cen) \co,H-torminal ldomain Receptor SH ‘ong me os H55 region AEM-28 Adapted from Hatters et al., Trend
Slide 8: AEM-28 Human Protocols
AEM-28 Human Protocols Phase 1a Health Volunteer Study Protocol/ Inclusion/Exclusion Normal Health Volunteers With elevated LDL Cholesterol 2 160 No Smokers No History of Heart Disease Fasted Subjects (overnight) Standard Safety Premedication Single Ascending Dose IV Administration over 1 hour 24 Ho
Slide 9: AEM-28 Human Protocol
AEM-28 Human Protocol Phase 1b/2a Multiple Ascending Dose Protocol/ Inclusion/Exclusion Refractory Hypercholesterolemic Subjects Normal Health Volunteers With elevated LDL Cholesterol > 160 High Body Mass Index Subjects with elevated fasting TG No Smokers Fasted Subjects (overnight) Standard Safety
Slide 10: Phase 1a Trial Results
Phase 1a Trial Results Rapid, Sustained Reductions in VLDL (3.54mg/kg) Dose: 3.54 mg/kg i a 8 i 3 i g Time (hr) > Subjects fasted at infusion and 2 hours post > Single 3.54 mg/kg dose of AEM-28 causes rapid drop in VLDL and TG > Approx. 70% net decrease from baseline >» AEM-28 delays increases in VL
Slide 11: Phase 1a Trial Results
Phase 1a Trial Results Rapid, Sustained Reductions in TG (3.54mg/kg) Dose: 3.54 mg/kg 20 2 Az Average —-a— Pacebo Grand Average! Fractional Triglycerides (vs Baseline) 00 ° 10 20 30 0 50 Time (hr) > Subjects fasted at infusion and 2 hours post > Single 3.54 mg/kg dose of AEM-28 causes rapid drop in
Slide 12: Phase 1b/2a Trial Results
Phase 1b/2a Trial Results 19) os] oa! 2 ‘inion Fractional Concentros.on verais Pre Dose in First 12 Hours Triglycerides: Comparison of Minimum Fractional Concentrations ‘VLDL: Comparison of Minimum Fractional Concentrations versus Pre-Dose Observed in First 12 Hours Post-Dose verus Pre-Dose Observed
Slide 13: New Chimeric Peptides: AEM-28 Analogs: AEM-28-14
New Chimeric Peptides: AEM-28 Analogs: AEM-28-14 >» Program will advance newly-discovered AEM-28-14 which has been developed by LipimetiX /University of Alabama > AEM-28-14 4X more efficacious than AEM-28 in mouse models. > Preclinical safety and toleration show >5X increase NOAEL dose in mouse mode
Slide 14: AEM-28 Analogs: Preclinical Efficacy
AEM-28 Analogs: Preclinical Efficacy Efficacy of Novel AEM-28 Analogs vs AEM-28-02 in apoE Null Mice: Percent Plasma Cholesterol - 50 ug Data 100 80 60 40 AEM-28-14 4 AEM-28-16 ——e-— AEM-28-18 =~ AEM-28-08 ¥. ‘AEM-28-02 20 Percent Baseline Plasma Cholesterol! 0 5 10 15 20 25 Time (hn) Decreases Chol
Slide 15: AEM-28 Analogs: Preclinical Efficacy
AEM-28 Analogs: Preclinical Efficacy Percentage of Baseline Plasma Cholestero! Comparison of Efficacy of Varying Doses of AEM-28-14 for Reducing Cholesterol in Apo E Null Mice (+ SEM) Dose vs Maximum Percent Decrease in Plasma Cholesterol with AEM-28-14 in Apo E Null Mice (Hyperbolic Curve Fit) Time
Slide 16: AEM-28-14 Development Plan
AEM-28-14 Development Plan What Does Pharma Want To See? 1) Full Pre-IND Tox/PK Package 2) Full CMC Package 3) Phase 1a — Single Ascending Dose — Safety/Efficacy 4) Phase 1b — Multiple Ascending Dose — Safety/Efficacy ) Phase 2 = Multi-center, Multi-dose in Subjects with Refractory Hypertriglyceride
Slide 17: Company Estimated AEM-28-14 Development Schedule
Company Estimated AEM-28-14 Development Schedule Milestone Ee Q415 | Q1N6 fee Q316 | Q46 [Be Q2/17 emi ee CMC SS} Pre-Ciinical aS CTAIND = Phase 1a = Phase 1b S53 Phase 2 a Estimate of 25 Months from Pre-Clinical to Phase 2 Data: Lipimetix Delivered Prior AEM-28 Phase 2a Data on Time and On Budget C
Slide 18: AEM-28-14 Development Plan
AEM-28-14 Development Plan Plan Is To “Partner” Phase 2b/3 Trials Which Can Be Targeted To Multiple TG/Cholesterol Indications: > CV > Hypertriglyceridemic Acute Pancreatitis > Severe Refractory Hypertriglyceridemia > HoFH > Diabetic / Vascular Complications > Metabolic Syndrome > Acute Coronary Syn
Slide 19: Acute Pancreatitis: Symptoms, Types, Diagnosis, Therapy
Symptoms: Types: Diagnosis: Therapy: Severe Abdominal Pain/Nausea/Loss of Appetite/Fever/ Hemodynamic Instability/Respiratory Distress (Defined by Extent of Cell Injury) Mild -TGs < 500mg/dL / Inflammation Moderate — TGs > 500mg/dL / Inflammation/Edema Severe — TGs > 1,000mg/dL / Inflammation/Autoim
Slide 20: Target Indications
Target Indications Homozygous Familial Hypercholesterolemia (HoFH) Homozygous Familial Fiperene ete erie ere] Orphan/Fast Track $200MM LDL, non-HDL > Rare genetic disease (= 12,000 worldwide) > Homozygous (both parents) bear offspring with no LDL receptors on liver: No clearance of LDL Cholesterol >
Slide 21: Target Orphan Indications in Hyperlipidemia
Target Orphan Indications in Hyperlipidemia AEM-28-14 Formulation Type 1a Familial Decreased Chylomicron Hyperchylomicronemia LPL remnants in 1,000,000=300 Infusion Clinic or LPL Lipase Deficiency Type 1b Familial apoprotein Altered Chylomicron/ C Ildeficiency Apo C2 VLDL 1in 250,000=1,200 Infusion
Slide 22: AEM-28-14: Future Long Term Target Indications
AEM-28-14: Future Long Term Target Indications Acute Coronary Syndrome Clinical Ath if i 1,300,000 (acs) Outeanes erosclerosis 300, Peripheral Artery Disease Clinical Intimal Hyperplasia/ 600,000 (PAD) Outcomes Atherosclerosis g High Risk Metabolic Clinical eS & Epidemic VLDI i id Syndrome Outcomes
Slide 23: Proven Clinical Management
Proven Clinical Management > Dennis Goldberg, PhD — LipimetiX President and CSO ¢ Uof Penn, UCSD, GelTex, Pfizer, Talaria Therapeutics * Led discovery and development of RenaGel® and WelChol® combined over $1B in annual sales > Eric Morrel, PhD — LipimetiX Vice President, Clinical Research « MIT, As
Slide 24: Opportunity Summary
Opportunity Summary > Proof-Of-Concept in AEM-28 Human Trials > Fresh Composition Of Matter Patent Filed — Potential Coverage Through 2035 > I.V. (vs. Oral) Delivery Suited To Smaller Orphan Markets > Capital Efficient Virtual Model Managed By Experienced Team > AEM-28-14 Showing 4X Greater Efficacy
Slide 25: Capstone Therapeutics Corp.
Capstone Therapeutics Corp. 1275 W. Washington Street, Suite 104 Tempe, AZ 85281 OTCQB: CAPS Jock Holliman 602-286-5586 jholliman@capstonethx.com Libime DeVelopment, LipimetiX Development, Inc. 5 Commonwealth Road, Suite 2A Natick, MA 01760 Dennis Goldberg, PhD. 508-651-3715 dgoldberg@lipimetix.com