Capstone Holding Corp. · NASDAQ:CAPS
Capstone — Capstone Therapeutics — Corporate Update (November 2014)
21 slides from Capstone's corporate update, Nov 13, 2014
Slide 1: Capstone Therapeutics Operating Update
Capstone Therapeutics (OTCQB: CAPS) Operating Update November 13, 2014 eS CAPSTONE Confidential
Slide 2: Safe Harbor Statement
Safe Harbor Statement Statements in this presentation or otherwise attributable to ‘apstone regarding our business that are not historical facts are These forward-looking statements involve risks and uncertainties that could cause actual results to differ materially from predicted results. These ris
Slide 3: Corporate Facts
Corporate Facts ptide Therapeutics = OTCQB: CAPS Based in Tempe, AZ . | Morket Cap as of November 10, oe ae M 4 (includ * Tipimetix Development, LLC) = Based in Natick, MA nership ae HenGer i i pre-clinical data for 40% ownership (data Q3 and Q4 2014) 28 and AEM-28-02) 1 \ } Indi eS CAPSTONE Confide
Slide 4: Cash Report
Cash Report CAPS $ (345,000) Lipimetix $ (737,000) Consolidated $ (1,082,000) Cash Balance $ 3,111,000 Note: h Bal 2014 includes $0.1 million held i use by LipimetiX Development, LLC eS CAPSTONE Confidential
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 OrthoLogic/CAPS learned of lawsuit in 2009 DOJ/US Government has not joined complaint against OrthoLog
Slide 6: LipimetiX Operating Update — November 2014
LipimetiX Operating Update — November 2014 > Major Milestones: = Augu: = October 2012 —-N EM f fl CMC validated December 2012 if Jesi i HoFH indication = November 2013 1 i linical studi «= December Regulatory nee -in-man studies « April 2 an 351 NSE SSH Jal beeinch g' cal lsh Australia . 1a AEM. 14
Slide 7: LipimetiX Operating Update (cont'd)
LipimetiX Operating Update (cont’d) > Target Schedule: = December 2014 — Report topline safety data and any pharmacodynamic/biomarker signals for Phase 1b/2a = January 2015 — Initiate strategic licensing/partnering discussions, assuming favorable clinical data Hitting Milestones Substantially On Tim
Slide 8: AEM-28 Target Indication: HoFH
AEM-28 Target Indication: HoFH Homozygous Famili lial Hypercholesterolemia (HoFH) Chetan yee WEES > ied ide) f LDL Cholesterol in teens and 20’s > Histori = Juxtapid (Aegerion = ky rion) pers (Sa nofi/Genzyme): . may limit usage > Rlaved fl underserved CAPSTONE Confidential
Slide 9: Advantages of Apo E Mimetics in HoFH
Advantages of Apo E Mimetics in HoFH > Rapid (one hour) and dramatic lipid reduction > Does not inhibit nacurah regulatory pathways > Acceptable saf bility (new proprietary formulation) > ApoE protective effect on arterial walls lessens atheroma burden > New proprietary formulation for acceptable sa
Slide 10: AEM-28: Clear and Rapid Path to HoFH Approval
AEM-28: 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
Slide 11: AEM-28 Human Protocol: Phase 1a
AEM-28 Human Protocol: Phase 1a Protocol/ Cohort 1: 0.032 mg/kg 2 Placebo Inclusion/Exclusion yore Normal Health Volunteers With elevated LDL Cohort 2: 0.10 mg/kg Cholesterol 2 160 BPliesbo Outcomes 4 Active No Smokers > Generally acceptable safety profile . . Cohort 3: 0.32 mg/kg No History of Hear
Slide 12: Phase 1a Pharmacodynamic Results: Escalating Dose Response Curve
Phase 1a Pharmacodynamic Results: Escalating Dose Response Curve VLDL chotesterol Trighcorides . , : sci 28 ose nat) AW. Don na) | [ | | l Ath bse nat) sem 28 Dos sta eS CAPSTONE Confidential 2
Slide 13: Rapid, Sustained Reductions in VLDL (3.54mg/kg)
Rapid, Sustained Reductions in VLDL (3.54mg/kg) Fractional VLDL (versus Baseline) Dose: 3.54 mg/kg > > Single 3.54 mg/kg d f AEM in VLDL and TG > AEM 1 ire ; > AEM / pi J 48 hrs post d eS CAPSTONE Confidential
Slide 14: Rapid, Sustained Reductions in TG (3.54mg/kg)
Rapid, Sustained Reductions in TG (3.54mg/kg) Dose: 3.54 mg/kg —2— AEM Average ? § ;: —-8— Placebo Grand Average wi qQ eo g 4 g 3 3 = 3 & g g 3 & * in a var 10 2 Fry 50 Time (hr) > Single 3.54 mg/kg dose of AEM 1d VLDLc and TG > AEM i intak > AEM H t {48h a V p eS CAPSTONE Confidential
Slide 15: 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 mice models > Game Changer: Opens new “clinical outcomes” indications 21 Year Patent Life Opens Larger Indications in
Slide 16: Efficacy Comparison (AEM-28 TO AEM-28-02)
Efficacy Comparison (AEM-28 TO AEM-28-02) Round 1 (AEM-28 we LPM-APOE-001 - Round 1 ogres a ime (hot = oe aie fis mE 2) AEM-28 1:20 mE 8) AEM-28 1:20 Round 5 (AEM-28-02) Study LPM-APOE-002 - Round 5 8 8 Total Cholesterol (mg/dL) ‘AEN-28-02 saline (100 ug) - mm fevzec2t 20100 oo} - Total Cholesterol
Slide 17: New Formulation
New Formulation: > Invented by Lipimetix > Enhances safety/toleration of Apo E mimetic peptides > No loss of efficacy > Manufacturing program in place 21 Year Patent Life and Enhanced Toleration Allows ‘or Expanded Dosing Range eS CAPSTONE Confidential
Slide 18: New AEM-28-02: Target Orphan Indications in Hyperlipidemia
New AEM-28-02: Target Orphan Indications in Hyperlipidemia Type da Type 1b Type 3 Types 4 and 5 Familial Hyperchylomicronemia or LPL Lipase Deficiency Familial apoprotein Cll deficiency Familial Disbetalipoproteinemia Refractory Familial lypertriglyceridemia teri Pancreati 1 in 1,000,000 = 300 1 in
Slide 19: New AEM-28-02: Future Target Indications
New AEM-28-02: Future Target Indications Acute Coronary Syndrome Clinical Atherosclerosis 1,300,000 Hospital (ACS) Outcomes Peripheral Artery Disease _Clinical Intimal Beast 600,000 Hospital and (PAD) Outcomes Atherosclero: Infusion Clinic High Risk Metabolic Clinical VLDL/Triglycerides Epidemic Hos
Slide 20: Program Summary
Program Summary > Clinical « Lead peptide, AEM-2! cacy it LDL Phase 1a trial subjects with duration of effect placebo in VLD! ad Regulat i i itapi {mipomersen) for FDA approval in HoFH >» CMC: oN > IP: allows “clinical outcomes” endpoints * Novel formulation application filed 2014 > Commercial: indi
Slide 21: Capstone Therapeutics
> SS CAPSTONE T HE RAPEU T OTCQB: CAPS www.capstonethx.com Capstone Therapeutics 1275 West Washinaron Steet Suite 104 ‘se 602) "206-5820" oA CAPSTONE 2
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