ACS is a major public health challenge In the US: Over 1.5 million people experience ACS annually1 In the EU: ACS is the most common cause of death, accounting for more than 741,000 deaths each year2 The 6-month post-discharge mortality rate is:3 3.6% in patients with UA 4.8% in patients with STEMI 6.2% in patients with NSTEMI 1. American Heart Association, 2008; 2. British Heart Foundation Health Promotion Research Group, 2008; 3. Goldberg et al, 2004 Event rate of CV death, MI or stroke at 12 months post event remains ~10% 25 CV death, MI or stroke Major bleeding Event rate (%) 20 –25% 15 –20% –16% –19% 10 5 0 20.0 0.8 None 1.3 15.0 ASA1,2 12.1 1.8* 9.9 2.4* 11.7 2.2* 9.8 2.8* ASA + ASA + ASA + ASA + 3 3 4 clopidogrel prasugrel clopidogrel ticagrelor4 *Major bleeding: non-CABG-related TIMI major bleeding 1. Antiplatelet Trialists' Collaboration, 1994; 2. Antithrombotic Trialists' Collaboration, 2002; 3. Wiviott et al, 2007; 4. Wallentin et al, 2009 Anticoagulants and antiplatelets have different yet complementary mechanisms of action Anticoagulants Rivaroxaban Apixaban Edoxaban Coagulation cascade Platelets Collagen + other mediators Factor Xa Thromboxane Thrombin ASA Clopidogrel Prasugrel Ticagrelor ADP Inflammation Cellular proliferation Antiplatelets Thrombin Activated platelet Fibrinogen Platelet aggregation Fibrin Clot Mackman, 2008 GPIIb/IIIa inhibitors GPIIb/IIIa Rivaroxaban has the potential to further improve secondary prevention of ACS Oral administration O O N N O O Predictable pharmacology Cl S H N O Rivaroxaban Rapid onset of action Fixed dose Balanced dual mode of elimination Low potential for drug–drug or food–drug interactions* No routine coagulation monitoring Rivaroxaban binds directly to the active site of Factor Xa (Ki 0.4 nM) *For full details, see the rivaroxaban Summary of Product Characteristics, available at http://www.xarelto.com Roehrig et al, 2005; Perzborn et al, 2005; Kubitza et al, 2005; 2006a,b,c; 2007a,b 2.5 and 5 mg bid rivaroxaban doses were chosen for phase III based on data from ATLAS ACS TIMI 46 Rivaroxaban 2.5 and 5 mg TIMI major bleeding Rivaroxaban 2.5 and 5 mg death, MI, stroke Placebo TIMI major bleeding Placebo death, MI, stroke 15 5 ASA 11.9% Incidence (%) 12 9 6 ASA plus thienopyridine 4 3.8% HR=0.54 (0.27–1.08) 3 HR=0.55 (0.27–1.11) 6.6% 2.0% 2 p=0.17 1.2% 1 3 p=0.03 0 0 90 1.2% 0.0% 180 0.2% 0 0 Time after start of treatment (days) Gibson, 2008; Data on file at Johnson & Johnson; Mega et al, 2009 90 180 ATLAS ACS 2 TIMI 51 N=15,526* Physician's decision to add thienopyridine or not Stratum 1: ASA alone (7%) Placebo n=355 Rivaroxaban 2.5 mg bid n=349 ASA dose = 75–100 mg/day Rivaroxaban 5 mg bid n=349 Stratum 2: ASA + thienopyridine (93%) Placebo n=4821 Rivaroxaban 2.5 mg bid n=4825 Rivaroxaban 5 mg bid n=4827 Event-driven study – 1002 events *184 patients were excluded from the efficacy analyses prior to unblinding because of trial misconduct at three sites Mega et al, 2011 Main inclusion and exclusion criteria Inclusion criteria Diagnosis of STEMI, NSTEMI, or UA with at least one of the following: ≥0.1 mV ST-segment deviation TIMI risk score ≥4 Patients aged ≥18 years; <55 years only with either: Diabetes mellitus or Prior MI Patients received ASA 75–100 mg/day alone or ASA plus a thienopyridine Based on national/local dosing guidelines Gibson et al, 2011 Exclusion criteria Increased bleeding risk, e.g. Low platelet count History of intracranial haemorrhage Active internal bleeding Prior stroke or TIA in stratum 2 patients Atrial fibrillation: except single episodes >2 years previously in patients aged <60 years with no evidence of cardiopulmonary disease Study endpoints/analyses Primary efficacy endpoint: composite of cardiovascular death, MI and stroke (ischaemic, haemorrhagic or uncertain) Main safety endpoint: incidence of major bleeding not associated with CABG surgery (according to TIMI bleeding definition) Primary analysis: log-rank test stratified by thienopyridine use in mITT population with confirmation in an ITT analysis mITT: all randomized patients and events from randomization up to earliest date of completion of treatment period (i.e. global treatment end date), 30 days after early discontinuation of study drug, or 30 days after randomization (patients randomized but not treated) ITT: all randomized patients and events observed from randomization up to global treatment end date Gibson et al, 2011 Patient characteristics Rivaroxaban 2.5 mg bid (n=5174) Rivaroxaban 5 mg bid (n=5176) Placebo (n=5176) 62 (9) 62 (9) 62 (9) Male sex, % 75 74 75 Median weight, kg 78 78 78 Median CrCl, ml/min 85 85 86 Prior MI 26 27 27 Hypertension 67 68 68 Diabetes mellitus 32 32 32 STEMI 50 50 51 NSTEMI 26 26 26 UA 24 24 24 PCI or CABG for index 61 60 60 Mean age, years (SD) Medical history, % Index diagnosis, % Mega et al, 2011 Primary efficacy endpoint (CV death/MI/stroke) Both rivaroxaban doses, both strata Estimated cumulative rate (%) 12 2-year Kaplan–Meier estimate 10.7% 10 Placebo 8.9% 8 Rivaroxaban 6 HR=0.84 (0.74–0.96) ARR=1.7% mITT p=0.008 4 ITT p=0.002 NNT=56 2 0 0 Number at risk Placebo 5113 Rivaroxaban 10,229 Mega et al, 2011 4 4307 8502 16 8 12 Months after randomization 3470 6753 2664 5137 1831 3554 20 24 1079 2084 421 831 Primary efficacy analysis: patient subgroups Both rivaroxaban doses, both strata HR (95% CI) Pinteraction Overall 0.84 (0.74 0.96) ASA ASA + thienopyridine 0.69 (0.45 -1.05) 0.86 (0.75 -0.98) 0.34 <65 years ≥65 years 0.83 (0.70 - 0.99) 0.94 STEMI NSTEMI UA 0.85 (0.70 - 1.03) 0.85 (0.68 - 1.06) 0.82 (0.62 - 1.07) 0.96 Male Female 0.87 (0.75 - 1.01) 0.40 Weight <60 kg Weight 60 to <90 kg Weight ≥90 kg 0.83 (0.56 - 1.25) Prior MI No prior MI 0.83 (0.68 - 1.01) Diabetes mellitus No diabetes mellitus 0.96 (0.77 - 1.20) CrCl <50 ml/min CrCl ≥50 ml/min 0.88 (0.62 - 1.26) North America South America Western Europe Eastern Europe Asia Other 0.57 (0.33 - 0.97) Mega et al, 2011 0.84 (0.70 - 1.01) 0.77 (0.60 - 0.99) 0.98 0.85 (0.72 - 0.99) 0.83 (0.64 - 1.08) 0.80 0.85 (0.72 - 1.01) 0.14 0.78 (0.67 - 0.92) 0.82 0.84 (0.73 - 0.96) 0.89 (0.59 - 1.34) 0.90 (0.59 - 1.37) 0.83 (0.69 - 1.00) 0.86 (0.63 - 1.17) 0.92 (0.60 - 1.39) 0.5 0.8 Favours rivaroxaban 1.0 1.25 2.0 Favours placebo 0.80 Primary efficacy endpoint Separate rivaroxaban doses, both strata Rivaroxaban 2.5 mg bid (n=5114) Rivaroxaban 5 mg bid (n=5115) Placebo (n=5113) 9.1% 8.8% 10.7% 0.84 (0.72–0.97) 0.85 (0.73–0.98) 0.02 0.03 2.7% 4.0% 0.66 (0.51–0.86) 0.94 (0.75–1.20) 0.002 0.63 6.1% 4.9% 0.90 (0.75–1.09) 0.79 (0.65–0.97) 0.27 0.02 1.4% 1.7% 1.13 (0.74–1.73) 1.34 (0.90–2.02) 0.56 0.15 Composite primary endpoint K–M estimate at 2 years HR versus placebo (95% CI) p value versus placebo CV death K–M estimate at 2 years HR versus placebo (95% CI) p value versus placebo 4.1% MI K–M estimate at 2 years HR versus placebo (95% CI) p value versus placebo 6.6% Stroke (haemorrhagic and ischaemic) K–M estimate at 2 years HR versus placebo (95% CI) p value versus placebo Mega et al, 2011 1.2% Components of primary endpoint Rivaroxaban 2.5 mg bid, both strata CV death/MI/stroke 13 Cardiovascular death 5 HR=0.84 Cumulative incidence (%) mITT p=0.02 ITT p=0.007 Placebo All-cause death 5 HR=0.66 mITT p=0.002 ITT p=0.005 10.7% HR=0.68 Placebo mITT p=0.002 ITT p=0.004 4.1% Placebo 4.5% 9.1% 2.9% 2.7% Rivaroxaban 2.5 mg bid Rivaroxaban Rivaroxaban 2.5 mg bid 2.5 mg bid NNT=63 0 NNT=71 0 0 6 12 18 Months 24 Mega et al, 2011; Gibson et al, 2011 NNT=63 0 0 6 12 18 Months 24 0 6 12 18 Months 24 Stent thrombosis* Both rivaroxaban, both strata 2-year Kaplan–Meier estimate 3 2.9% Estimated cumulative incidence (%) Placebo 2.3% 2 Rivaroxaban HR=0.69 (0.51–0.93) RRR=31% mITT p=0.02 ITT p=0.008 1 2-year K–M estimate HR versus placebo (95% CI) p value vs placebo (mITT) Rivaroxaban 2.5 mg bid Rivaroxaban 5 mg bid 2.2% 2.3% 0.65 (0.45–0.94) 0.73 (0.51–1.04) 0.02 0.08 0 0 4 8 12 16 Months after randomization 20 24 *Stent thrombosis events: definite, probable or possible (Academic Research Consortium definitions) Mega et al, 2011 Principal safety endpoint Separate rivaroxaban doses, both strata Non-CABG TIMI major bleed K–M estimate at 2 years p value versus placebo ICH K–M estimate at 2 years p value versus placebo Fatal bleeding K–M estimate at 2 years p value versus placebo Fatal ICH K–M estimate at 2 years p value versus placebo Mega et al, 2011 Rivaroxaban 2.5 mg bid (n=5115) Rivaroxaban 5 mg bid (n=5110) 1.8% <0.001 2.4% <0.001 0.6% 0.4% 0.04 0.7% 0.005 0.2% 0.1% 0.45 0.4% 0.20 0.2% 0.1% – 0.2% – 0.1% Placebo (n=5125) Treatment-emergent fatal bleeding events and ICH 2-year Kaplan–Meier estimate (%) Separate rivaroxaban doses, both strata Placebo 2.5 mg rivaroxaban 5.0 mg rivaroxaban 1.0 Rivaroxaban vs placebo Rivaroxaban vs placebo Rivaroxaban vs placebo p=NS p=0.009 p=NS 0.8 0.7 0.6 0.4 0.4 0.2 0.2 0.4 0.2 0.2 0.1 0.1 0.1 0.0 Fatal bleeding events Mega et al, 2011 ICH Fatal ICH Other safety endpoints Separate rivaroxaban doses, both strata Rivaroxaban 2.5 mg bid (n=5115) Rivaroxaban 5 mg bid (n=5110) Placebo (n=5125) Post-treatment ischaemic events* Raw percentage 1.4% 2.2% p value versus placebo p=NS p=NS 1.8% Liver function test (ALT >3× ULN)# Raw percentage 1.3% 1.4% p value versus placebo p=NS p=NS 1.6% Other adverse events (raw percentages) Dyspnoea 1.1% 1.3% 1.5% Cough 1.2% 1.1% 1.4% *CV death/MI/stroke (ischaemic, haemorrhagic, uncertain) events occurring 1–10 days after last rivaroxaban dose; #Abnormal values from first dose to 2 days post last dose in patients with normal baseline values Mega et al, 2011 ATLAS ACS 2 TIMI 51: summary Compared with placebo, rivaroxaban (2.5 or 5 mg bid) on top of ASA or ASA plus clopidogrel showed: Significant reductions in the rates of death, MI , and stroke Benefits in all types of ACS patients (UA, NSTEMI and STEMI ) More than a 30% reduction in risk of both CV and all-cause mortality (2.5 mg bid) No increase in fatal bleeding and fatal ICH A non-bleeding safety profile similar to placebo The addition of anticoagulation with rivaroxaban may represent a new treatment strategy in patients after recent ACS Mega et al, 2011