Slide 1: Capstone Therapeutics Operating Update
Capstone Therapeutics Operating Update March 27, 2014 AN 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: Cash Report
Cash Report Cash Use and Ending Balance 2013 CAPS $ (1,490,000) Lipimetix $ (2,457,000) Consolidated $ (3,947,000) Cash Balance S$ 6,258,000 Note: Cash Balance at December 31, 2013 includes $2.0 million held in and reserved for use by Lipimetix Development, LLC
Slide 4: 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 5: LipimetiX Development LLC (JV)
LipimetiX Development LLC (JV) @ CAPS contributed $6M for 60% (ownership) @ 2 tranche funding structure @ S5M of CAPS contribution has preferential distribution right @ Lipimetix LLC (Lipi) contributed UAB license for current/future IP, for 40% (ownership) @ JV contracted Dennis Goldberg, Ph.D. and
Slide 6: AEM-28 Rational Design at UAB
AEM-28 Rational Design at UAB — 299 aa down to 28 aa with full function "Hinge region’ a a a F % Apolipoprotein E Gq A aire 5 ape touting (Gn bes on a ¥5) es 55555 region
Slide 7: Target Indications
Target Indications @ Homozygous Familial Hypercholesterolemia (HOFH) > Absence of LDL receptor function > Cannot clear LDL or control cholesterol levels > Orphan indication, potential for rapid development/ approval > Potential $200 million market @ Severe Refractory Hypercholesterolemia > Not respo
Slide 8: Target Indications (cont'd)
Target Indications (cont’d) @ Acute Coronary Syndrome > 1,300,000 hospitalizations/year in US alone > 10-25% recurrence rate in first year after event > Reduce atherosclerosis burden and mortality > Potential $11 Billion annual market > Will require an outcome study and funding by big Pharma
Slide 9: Homozygous FH Animal Evidence
Homozygous FH Animal Evidence: AEM -28 Reduces LDL & VLDL > Watanabe Heritable Hyperlipidemic (WHHL) Rabbits have same genetic defect as human FH patients > WHHL rabbits are refractory to statins, bile acid sequestrants, etc. > Single dose of AEM-28 reduces non-HDL cholesterol 45% at 18 hours 0.08 7
Slide 10: ACS Animal Evidence
ACS Animal Evidence: Direct Artery Wall Effects Reduce Atherosclerosis 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
Slide 11: AEM-28 Sustained Effect on Atherosclerotic Lesions in Fat-fed APO E Null Mice
AEM-28 Sustained Effect on Atherosclerotic Lesions in Fat-fed APO E Null Mice 4 Weeks After Stopping Drug Treatment En face Lesion Area (n=13 in each group) Representative en face Lesions Control AEM-28 TTT 117 i a Saline AEM.28 Figure 4. Za CAPSTONE
Slide 12: Summary — Apo E Mimetics
Summary — Apo E Mimetics @ Novel mimetics of key lipid/cholesterol metabolism protein > Utilizes alternate receptors on liver to clear atherogenic lipoproteins > Induces direct anti-inflammatory and lesion regression effects on artery walls > Believed to be only product in development with both effe
Slide 13: LipimetiX Operating Update — March 2014
LipimetiX Operating Update — March 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
Slide 14: LipimetiX Operating Update — March 2014
LipimetiX Operating Update — March 2014 Budget: > Total Original Program Cost was = S$6M; Now Estimating = $6.5M > Australia program possible 45% R&D cash rebate may assist funding > Remaining escrow funds to be released > Available Joint Venture funds anticipated to be adequate to complete developm
Slide 15: AEM-28 Human Protocol Summary
AEM-28 Human Protocol Summary @ Phase Ja > Objective: Safety and Tolerability Evaluate Pharmacokinetics © Evaluate Effects on non-HDL, LDL and Total Cholesterol > Format: © Single Ascending Doses (6 doses delivered IV) in Normal Healthy Volunteers with Elevated Cholesterol en=36 @ Phase 1b/2a > Obje
Slide 16: Capstone Summary
Capstone Summary @ Two Programs: > Apo E Mimetic Peptide AEM-28 in LDL and non-HDL Reduction > AZX100 in Pulmonary Fibrosis — LRRI Results @ Continue CAPS Virtual Operation (1.5 Employees/ Consultants) to Preserve Cash @ Cash Resources Believed to be Adequate to Reach Value Milestones AS CAPSTONE 6
Slide 17: Capstone Therapeutics
~~ CAPSTONE. T H ER APEU T OTCQB: CAPS www.capstonethx.com Capstone Therapeutics 1275 West Washington Street - Suite 101 Tempe, AZ 85281 (602) 286-5520