 Pioneering Genetically-Targeted Cardiovascular Therapies April 23, 2013 NASDAQ: ABIO Exhibit 99.1 |
 Safe Harbor Statement 2 This presentation contains "forward-looking statements" for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements regarding the Company’s anticipated timing for initiation or completion of its clinical trials for any of its product candidates; the potential for Gencaro to be an effective potential treatment for atrial fibrillation and, the Company’s ability to fund future operations. Such statements are based on management's current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, the risks and uncertainties associated with: the Company's financial resources and whether they will be sufficient to meet the Company's business objectives and operational requirements; the Company’s ability to complete a strategic transaction to support the continued development Gencaro, and/or obtain additional financing; the Company’s anticipated timing for initiation or completion of its clinical trials for any of its product candidates; the Company's ability to identify, develop and achieve commercial success for products and technologies; drug discovery and the regulatory approval process; estimated timelines for regulatory filings and the implications of interim or final results of the Company’s clinical trials; the extent to which the Company’s issued and pending patents may protect its products and technology; the potential of the Company’s clinical development program to lead to the approval of the Company’s New Drug Application for Gencaro; and, the impact of competitive products and technological changes. These and other factors are identified and described in more detail in ARCA’s filings with the SEC, including without limitation the Company’s annual report on Form 10-K for the year ended December 31, 2012, the Company’s Registration Statement on Form S-1 (Registration No. 333-187508), and subsequent filings. The Company disclaims any intent or obligation to update these forward-looking statements. |
 Offering Summary • Ticker Symbol ABIO • Offering $20,000,000 Common stock + warrants • Use of proceeds To fund Phase 2b development of Company’s lead product candidate, Gencaro, working capital and general corporate purposes • Placement agent Dawson James Securities, Inc. 3 |
 Corporate and Lead Product Highlights • Late stage cardiovascular company • Pharmacogenomic drug development/personalized medicine approach – Potentially first genetically-targeted cardiovascular drug • Experienced, successful management team • Lead product Gencaro TM (bucindolol hydrochloride) – 4 th generation beta-blocker with extensive patient data – 1 st potential indication - atrial fibrillation (AF) • Clearly defined regulatory pathway – Similar endpoint basis for two most recent AF approvals – Beta blocker profile well characterized and understood • Significant market opportunities in a major unmet medical need 1 – 2.7 million AF patients in U.S.; 250,000-500,000 new onset AF/year (2010) – Current therapies associated with adverse events • Robust IP portfolio – US and EU market exclusivity, with potential patent extension, into 2029/2030 4 1 Bristow MR, Aleong RG. JACC-Heart Fail 1:29-30, 2013 |
 ARCA biopharma Leadership Michael R. Bristow, MD, PhD: President /CEO Patrick Wheeler, Chief Financial Officer Chris Ozeroff: General Counsel Thomas Keuer: EVP, Pharmaceutical Operations Monique Plamondon: VP, Regulatory/Quality Board of Directors Michael R. Bristow, MD, PhD Jean-Francois Formela, MD Linda Grais, MD, JD (Lead Independent) Burton E. Sobel, MD John Zabriskie, PhD 5 |
 Pharmacogenomics: The science of personalized medicine 6 Personalized medicine refers to the tailoring of medical treatment to the individual genetic characteristics of each patient in order to classify individuals into subpopulations that differ in their susceptibility to a particular disease or their response to a specific treatment. Preventative or therapeutic interventions can then be concentrated on those who will benefit, sparing expense and side effect for those who will not. 1 Potential Benefits Better diagnoses and earlier intervention More efficient drug development More effective therapies Improved patient outcomes Reduced adverse drug reactions Reduced healthcare costs Extension of market exclusivity 1 – President’s Council of Advisors on Science and Technology, Priorities for Personalized Medicine, 2008 |
 Patient Population Genotypes Results from Phase 3 BEST trial DNA sub-study, HF endpoints 7 Source: ARCA biopharma ‘Very Favorable’ genotype {ß1 389 Arg/Arg + any 2C } ~50% ‘Favorable’ genotype {ß1 389 Gly carrier + 2C Wt/Wt} ~40% ‘Unfavorable’ Pt. genotype {ß1 389 Gly carrier + 2C Del carrier} ~10% 1,040 Patients provided DNA for sub-study Target population for Phase 2b/3 AF trial Gencaro Unique MOA: 1) NE lowering (Arg is NE AR) 1 2) Inverse agonism 2 1 Liggett et al, PNAS 2006 2 O'Connor et al, PLOS ONE 2012 BEST trial participants – 2,708 Patients – |
 LabCorp – Strategic Partnership • ARCA has exclusive rights to Gencaro companion genetic test, has partnered with LabCorp • Easy-to-administer, one-time genetic test that identifies genetic markers which predict clinical response • Quick turnaround time for results 8 Strategic Partner S&P 500 Company $5.7B in revenues in 2012 34,000 employees worldwide 220,000 clients Conducts over 1M tests daily |
 1 389 Arg/Arg (n = 441; 36 events) Hazard Ratio = 0.26 (0.12 – 0.57) P-value = 0.0003 Risk reduction 74% BEST adrenergic receptor polymorphism substudy Interaction p = 0.008 Prevention of new onset AF in BEST by 1 389 Arg/Gly genotype Aleong et al, Circulation 124: A10438, 2011 1 - Abi Nasr I et al, EHJ 28: 457–462, 2007 0.70 0.75 0.80 0.85 0.90 0.95 1.00 0 6 12 18 24 30 36 42 48 Months After Randomization Placebo Bucindolol In a meta-analysis of Phase 3 HF trials covering ~12,000 randomized patients, there was a 27% average reduction in incidence of new onset AF in heart failure where new onset AF was reported.¹ 9 |
 LVEF <0.50, Class II-III HF w/in 90 days No contra-indications to -blockers 1 389 Arg/Arg genotype Bucindolol Metoprolol CR/XL ECV @ 4 wks if still in AF Phase 2b: 1° Endpoint = recurrent AF or ACM, 24 weeks; Co 1° EP = AF burden n = 100 (2b) (Ph 3, 310) n = 100 (2b) (Ph 3, 310) Phase 2b Ph 3 Adaptive Design Superiority Trial Bucindolol vs. Metoprolol CR/XL, Prevention of Recurrent Atrial Fibrillation in Persistent AF HFREF Patients with the 1 389 Arg/Arg Genotype post Electrical Cardioversion (ECV), + Adaptive Design to Phase 3 Projected timeline: Trial Initiation – Q4 2013 Ph 2b Trial Data –Q4 2015 Ph 3 Trial data- Q4 2017 Time 0 (conversion to AF counted as if ECV) Ph 3 1° EP: Recurrent AF or ACM , 24 wks 10 Recent onset Sx AF, 1 wk – 90 d Class I-III HF |
 Strategic Partnership - GENETIC-AF Clinical Trial Collaboration- • Medtronic, Inc – World’s largest medical technology company – Leader in medical technologies to improve the treatment of chronic diseases, including cardiac rhythm disorders – 45,000 employees – $16 Billion – 2012 Revenues – $1.5 Billion – 2012 Research & Development Investment • Collaboration - Substudy of P2b portion of GENETIC-AF - Measure AF burden data by means of Medtronic continuous monitoring devices - Medtronic to provide support for AF burden substudy and for collection and analysis of substudy data 11 Medtronic and ARCA have executed agreement to collaborate on GENETIC-AF trial |
 Atrial Fibrillation - Regulatory Strategy - Clearly defined Regulatory Pathway – Similar endpoints used for most recent AF FDA approvals – Safety profile – well characterized based on BEST & prior development – Company has understanding of pathway, safety profile and trial design 12 Obtain an atrial fibrillation approval in a genotype specific heart failure population via adaptive design Phase 2b/3 trial of 200/620 patients Possibility of approval, based on GENETIC-AF (Phase3) if p 0.01, when submitted with BEST trial data Second trial will likely be required if GENETIC-AF p >0.01 • • • |
 Hatch-Waxman [5 years from approval] Hatch-Waxman Patent Extension [7.5 years from approval] EU Data Exclusivity [10 – 11 years from EU approval] Bucindolol Patents [use of drug w/ genetic markers] Bucindolol Market Exclusivity / Intellectual Property Protection 13 Potential US Approval Patent Expires 2029* Worldwide Rights Bucindolol Patents Issued US – 2010,11,12 Europe –2010 * 2029 US/2030 EU w/term extension from current terms of 2026/2025 |
 Capital Structure 14 (as of December 31, 2012) • Shares outstanding: 2.66 million • Dilutive securities outstanding: 1.68 million – Stock issued post 12/31/12: 521K – Warrants outstanding: 930K (at $9.10 on weighted average) – Options outstanding: 144K (at $18.29 on weighted average) – Shares under Equity Plan: 80K • Current stock price (4/10/13): $2.20 /share • Current market capitalization: $7.01 million • Net cash: $2.92 million |
 Summary • AF is an epidemic CV disease afflicting ~2.7 million patients & growing; • Significant unmet need for treatment/prevention of AF, particularly in HFREF patients, and for rate control in HFREF (no other approved drugs for HFREF patients) • Prior clinical data have indicated Gencaro to be safe and has shown potential treatment benefits in AF, both in the entire patient cohort, and especially in the "very favorable" genotype (~50% of patient population) • Management team with extensive experience in CV disease, drug development and corporate execution • Potential to be only beta-blocker indicated for AF prevention • Potential to be the first genetically-targeted AF treatment 15 |