Capstone Holding Corp. · NASDAQ:CAPS
Capstone — Capstone Therapeutics — Corporate Update (March 2015)
22 slides from Capstone's corporate update, Mar 26, 2015
Slide 1: Capstone Therapeutics Operating Update
Capstone Therapeutics (OTCQB: CAPS) Operating Update March 26, 2015 AS CABSTONE
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 March 24, 2015: = $8MM « $2.2MM cash at December 31, 2014 > Lipi imetiX Development LLC (JV) = Based in Natick, MA « Vi
Slide 4: Cash Report
Cash Report CAPS $ (503,000) Lipimetix $ (444,000) Consolidated $ (947,000) Cash Balance $ 2,164,000 oN CAPSTONE
Slide 5: Qui Tam Lawsuit Update
Qui Tam Lawsuit Update > Basic legal complaint is “rent vs. buy” disclosure for bone stimulation device sold by OrthoLogic/CAPS in 1998 — 2003 > OrthoLogic/CAPS sold bone stimulation business in 2003 > Lawsuit filed in 2005 (under seal) > OrthoLogic/CAPS learned of lawsuit in 2009 > DOJ/US Governmen
Slide 6: LipimetiX Operating Update
LipimetiX Operating Update > Major Milestones: = August 2012 —JV formation and initial funding = October 2012 — New AEM-28 drug material commercially manufactured/ CMC validated = December 2012 — Orphan Drug designation for HoFH indication = November 2013 — Completion of comprehensive preclinical st
Slide 7: AEM-28 Human Protocol
AEM-28 Human Protocol Phase 1b/2a Multiple Ascending Dose Protocol/ Inclusion/Exclusion Refractory Hypercholesterolemic Subjects Cohort 7: 1.0 mg/kg Normal Health Volunteers 1 Placebo With elevated LDL 4 Active e Cholesterol > 160 Tian boay Mand nde Gupecs > Generally Acceptable Safety Profile with
Slide 8: Phase 1b/2a Trial Results
Phase 1b/2a Trial Results Triglycerides: Comparison of Minimum Fractional Concentrations VLDL: Comparison of Minimum Fractional Concentrations Versus Pre-Dose Observed In Fist 12 Hours Post Dose Verus Pre-Dose Observed in First 12 Hours Post-Dose (Mean # SEM) {Mean 2 SEM) 10 14 [ : 8g | —— Ege! : Ez
Slide 9: PD in Phase 1a/1b/2a Clinical Studies
PD in Phase 1a/1b/2a Clinical Studies > Part A (Phase 1a) = In Part A of the study, when compared to placebo, a decrease in VLDL-C and triglyceride levels was observed for approximately 8 hours following administration of a single dose of 2.0 mg/kg and 3.54 mg/kg AEM-28. = VLDL-C levels decreased by
Slide 10: PD in Phase 1a/1b/2a Clinical Study
PD in Phase 1a/1b/2a Clinical Study > Fad B (Phase 1b/2a) In Part B of the study, similarly to Part A, VLDL-C and triglyceride levels decreased for approximately 4 to 8 hours following administration of 2.0 mg/kg and 3.54 mg/kg AEM-28, when compared to placebo. =" Maximum decreases from baseline of
Slide 11: New Analog: AEM-28-02
New Analog: AEM-28-02 > Invented by Lipi/UAB > New composition of matter filed July: AEM-28-02 > Will develop with new formulation > More efficacious/better tolerated than AEM-28 in mouse models > Game Changer: Opens new “clinical outcomes” indications 21 Year Patent Life Extends Commercial Value AS
Slide 12: New Formulation
New Formulation: > Invented by Lipimetix > Enhances safety/toleration of Apo E mimetic peptides > Minimal trade-off in efficacy > Manufacturing program in place > Preclinical models show increase of 6x MTD 21 Year Patent Life and Enhanced Toleration Allows for Expanded Dosing Range AS CABSTONE Z
Slide 13: AEM-28-02 Target Orphan Indications
AEM-28-02 Target Orphan Indications > Homozygous Familial Hypercholesterolemia (HoFH) > Acute Pancreatitis with High Triglycerides > Other Hyperlipidemic Conditions AS CABSTONE
Slide 14: AEM-28-02 Target Indication: HoFH
AEM-28-02 Target Indication: HoOFH Homozygous Familial Tneensete cere ern) 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 > Blood often cream colored: life t
Slide 15: Clear and Rapid Path to HoFH Approval
Clear and Rapid Path to HoFH Approval > Regulatory approval path for orphan indication in US and EU established by Juxtapid (Aegerion) and Kynamro (Sanofi/Genzyme) > Cholesterol reduction (biomarker) approvable endpoint in this population / no “clinical outcomes” endpoint > Juxtapid approved after a
Slide 16: AEM-28-02 Target Indication: Acute Pancreatitis
AEM-28-02 Target Indication: Acute Pancreatitis with High Triglycerides Regulatory Prevalence in US Target Orphan/Fast Track 45,000 Acute Hospital VLDL/Chylomicron Cases/Year Remnants (TGs) Acute episodes triggered by gallstones, alcohol and genetic causes 74,000 hospital admissions in US, some 45,0
Slide 17: US acute market for AP with high TG is 45K doses/year
US acute market for AP with high TG is 45K doses/year; maintenance of refractory patients could be 5.7M doses/year Estimated Annual Market for AEM-28-02 # of AEM-28 Doses/year 45,000 KOLs were reluctant to speculate on 6,000,000) —~ acute care outcomes benefits for an doses i 5,000,000 experimental
Slide 18: Pharmacoeconomic Case Study (Company Prepared)
Pharmacoeconomic Case Study (Company Prepared): Acute Pancreatitis with High TGs * TG2 1,000 + ICU/Acute Hospitalization + Genetic Origin/Alcohol/Gallstones + Prescribed Therapy: “No Fat Diet / + 5% Mortality Fibrates / Opiates/ Prayer” + 45,000 Hospital Subjects + Avg 3 Day ICU Stay @ $10K/Day $30K
Slide 19: Target Orphan Indications in Hyperlipidemia
New AEM-28-02: Target Orphan Indications in Hyperlipidemia Type 1a Familial Decreased —Chylomicron Hyperchylomicronemia LPL remnants 1in 1,000,000=300 _Infusion Clinic or LPL Lipase Deficiency Type 1b Familial apoprotein Altered Chylomicron Cll deficiency Apo C2 /VLDL 1in 250,000 = 1,200 —_Infusion
Slide 20: Future Long Term Target Indications
New AEM-28-02: Future Long Term Target Indications Acute Coronary Syndrome Clinical Atherosclerosis 1,300,000 Hospital (ACS) Outcomes Peripheral Artery Disease _Clinical Intimal Hyperplasia/ 600,000 Hospital and (PAD) Outcomes Atherosclerosis Infusion Clinic High Risk Metabolic Clinical VLDL/Triglyc
Slide 21: Summary Goals
Summary Goals > Continue Ongoing, Early Stage Partnering Discussions > Optimize New Formulation > Develop New Molecule AEM-28-02 as Lead Commercial Candidate Demonstrated Drug Class Efficacy in Phase 1a/1b/2a Trials Serving Valuable Underserved Orphan Markets AS CABSTONE 24
Slide 22: Capstone Therapeutics
~~ CAPSTONE. T HERAPEU T OTCQB: CAPS www.capstonethx.com Capstone Therapeutics 4275 West Washington Street - Suite 104 Tempe, AZ 85281 (602) 286-5520 ao CAPSTONE 2
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