Capstone Holding Corp. · NASDAQ:CAPS
Capstone — Capstone Therapeutics — Corporate Update (May 2014)
20 slides from Capstone's corporate update, May 21, 2014
Slide 1: Capstone Therapeutics Operating Update
Capstone Therapeutics (OTCQB: CAPS) Operating Update May 21, 2014 SS CAPSTONE
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 May 20, 2014: = $11MM = $5.5MM cash at March 31, 2014 (includes $1.7MM held in and reserved for use by LipimetiX Develop
Slide 4: Cash Report
Cash Report CAPS. S$ (442,000) Lipimetix § (326,000) Consolidated S$ (768,000) Cash Balance S$ 5,490,000 Note: Cash Balance at March 31, 2014 includes $1.7 million held in and reserved for use by Lipimetix Development, LLC
Slide 5: Qui Tam Lawsuit Update
Qui Tam Lawsuit Update @ Discovery hearing held in December 2013 — duration of discovery process uncertain but underway @ Status conference with new judge held February 2014 to set rules of discovery @ Proposed DOJ/Orthofix settlement accepted by court - had slowed lawsuit progress @ DOJ/US Governme
Slide 6: AEM-28 is Derived from Native Apo E
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’ ‘APSTONE AAR ‘Angi72 ° rats Me & ina = @ lll kor 79) xe | oD| [Crs 5sSF = NHteNnal <domat e ) AEM-28 Adapted from Hatters et al., Trends Biochem Sci. 2006; 3
Slide 7: AEM-28 Market Indications
AEM-28 Market Indications Familial Hypercholesterolemia (HOFH) Orphan/Fast Track Severe Refractory Hypercholesterolemia Orphan Target Acute Coronary Syndrome (ACS) Non-Orphan Peripheral Artery Disease (PAD) Non-Orphan Diabetes Mellitus Type 2 Non-Orphan $200MM $800MM $10B $3B $35B (All Drugs) AEM-28
Slide 8: CardioVascular Lipid Lowering Drug Classes
CardioVascular Lipid Lowering Drug Classes STATINS. Lipitor (Pfizer) Zocor (Merck Crestor (Astra Zeneca) PCSK9 evolucumab (Amgen) INHIBITORS alirocumab (Sanofi/Regeron) bococizumab (Pfizer) APO Es AEM-28 Inhibits HMG-CoA reductase to limit liver cholesterol production. Inhibits PCSK9, enzyme that li
Slide 9: The Case for Apo E's
The Case for Apo E’s > Native Apolipoprotein E = Proven triglyceride/cholesterol regulating protein for 30 years = 299 Amino Acid — too large for therapeutic delivery > Apo E Mimetics (AEM-28 and Analogs) = University of Alabama at Birmingham — national research leader = Enhanced LDL and VLDL bindin
Slide 10: The Case for Apo E's (cont'd)
The Case for Apo E’s (cont’d) = Does not inhibit key regulatory enzymes = Low cost synthesis = AEM-28 composition of matter IP to 2020; 7 years Orphan exclusivity under Waxman-Hatch = Analogs of AEM-28 in development/potential new IP « Differentiated mechanistically from other cholesterol-lowering d
Slide 11: AEM-28 Clear and Rapid Path to Lead Indication for HoFH
AEM-28 Clear and Rapid Path to Lead Indication for HoFH > Regulatory approval path for orphan indication in US and EU established by Juxtapid (Aegerion) and Kynamro (Sanofi/Isis) >» Cholesterol reduction approvable endpoint in this population / no clinical outcomes endpoint > Juxtapid approved after
Slide 12: AEM-28: HoFH Preclinical Evidence
AEM-28: HoFH Preclinical Evidence Watanabe Heritable Hyperlipidemic (WHHL) rabbits have same genetic defect as human FH patients WHHL rabbits are refractory to statins, bile acid sequestrants, PCSK9 inhibitors, etc. Single dose of AEM-28 reduces non- HDL cholesterol 45% at 18 hours 0.08 7 s 3 0.06 |
Slide 13: AEM-28 Human Protocol Summary
AEM-28 Human Protocol Summary > Phase la = Objective: © Safety and Tolerability o Evaluate Pharmacokinetics o Evaluate Effects on non-HDL, LDL and Total Cholesterol = Format: © Single Ascending Doses (6 doses delivered IV) in Normal Healthy Volunteers with Elevated Cholesterol © Double-Blinded, Plac
Slide 14: AEM-28 Role in Acute Coronary Syndrome Therapy
AEM-28 Role in Acute Coronary Syndrome Therapy ACS Event | Critical Care 200 Stabilization/Maintenance 180 160 Non-HDL-C 140 120 Annual AEM-28 Follow Up 100 o 1 aa MO Tg 9 wo [=e AEM28 —=PCSK9 —i-Statin | (Not Based on Clinical Data) "High ACS Recurrence in Three Months Post Event = PCSK9 and Statin
Slide 15: AEM-28: ACS Preclinical Evidence
AEM-28: ACS Preclinical Evidence CAPSTONE Reduction in Atherosclerosis Far Exceeds Cholesterol Drop Control Animal Plasma Cholesterol = 1600 mg/dl 51% of aorta covered by fatty streaks AEM-28 Animal Plasma Cholesterol = 1100 mg/dl 8% of aorta covered by fatty streaks Direct Effects on Artery Wall Re
Slide 16: AEM-28: Athero Preclinical Beneficial Duration of Effect
AEM-28: Athero Preclinical Beneficial Duration of Effect Representative en face Lesions En face Lesion Area (n=13 in each group) 3 i i H : Figure 4. AEM-28 Sustained Effect on Atherosclerotic Lesion Regression in Fat-fed APO E Null Mice 4 Weeks After Stopping Drug Treatment Za SS CAPSTONE
Slide 17: LipimetiX Operating Update — May 2014
LipimetiX Operating Update — May 2014 > 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 p
Slide 18: LipimetiX Operating Update — May 2014
LipimetiX Operating Update — May 2014 > Budget: «Total Original Program Cost was = S6MM; Now Estimating = $6.5MM « Australia program possible 43.5% R&D cash rebate will assist funding Available Joint Venture funds anticipated to be adequate to complete development plan (refining final CRO Phase 1a/1
Slide 19: Capstone Summary
Capstone Summary @ Two Programs: > Apo E Mimetic Peptide AEM-28 in LDL and non-HDL Reduction > AZX100 in Pulmonary Fibrosis and Spinal Fibrosis @ Continue CAPS Virtual Operation (1.5 Employees/ Consultants) to Preserve Cash @ Cash Resources Believed to be Adequate to Reach Value Milestones
Slide 20: Capstone Therapeutics
A~ CAPSTONE THERAPEUTICS OTCQB: CAPS www.capstonethx.com Capstone Therapeutics 1275 West Washington Street - Suite 104 Tempe, AZ 85281 (602) 286-5520 20