SOLIRIS is a Complement Inhibitor Indicated for the Treatment of Patients With PNH to Reduce Hemolysis
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1 SOLIRIS (eculizumab) SOLIRIS is a Complement Inhibitor Indicated for the Treatment of Patients With PNH to Reduce Hemolysis SOLIRIS is the First and Only Approved Therapy for PNH SOLIRIS (eculizumab) [package insert]. Alexion Pharmaceuticals; 2009.
2 SOLIRIS (eculizumab) Humanized First in Class Anti - C5 Antibody Human Framework Regions No mutations Germline Hinge Complementarity Determining Regions (murine origin) Human IgG 2 Heavy Chain Constant Region 1 and Hinge (Eliminates Fc receptor binding) CH2 CH3 Human IgG 4 Heavy Chain Constant Regions 2 and 3 (Eliminates complement activation) Rother R et al. Nat Biotech 2007;25:1256 2
3 Terminal Proximal Co mp le me nt Ca sc ad e SOLIRIS Blocks Terminal Complement SOLIRIS C3 C3a SOLIRIS binds with high affinity to C5 C3b Terminal complement - C5a and C5b-9 activity blocked C5 C5a Proximal functions of complement remain intact C5b C5b-9 Cause of Hemolysis in PNH Weak anaphylatoxin Immune complex clearance Microbial opsonization Figueroa JE, Densen P. Clin Microbiol Rev. 1991;4(3): Walport MJ. N Engl J Med. 2001;344(14): SOLIRIS (eculizumab) [package insert]. Alexion Pharmaceuticals; Rother RP et al. Nature Biotech. 2007;25(11):
4 SOLIRIS PNH Clinical Studies Pilot Study NEJM N = 11 Primary endpoint: reduction of hemolysis TRIUMPH NEJM Pivotal Phase III, Double-Blind, Placebo-Controlled Trial, N = 87 SHEPHERD Blood Broader patient population, including those receiving minimal transfusions or with thrombocytopenia, N = 97 Long-Term Extension Trial Hillmen Blood Evaluated long-term safety, efficacy and effect on thrombosis; Placebo patients switched to SOLIRIS N = 187 4
5 Dosing Schedule Pretreatment Induction Phase Maintenance Phase 2 weeks before induction Week and every 2 weeks thereafter Neisseria meningitidis vaccination SOLIRIS dose, mg X 900 X 900 In clinical trials all patients received a meningococcal vaccination SOLIRIS should be administered via IV infusion over 35 minutes every 7 days during induction and every 14 days during maintenance SOLIRIS dose adjustment to every 12 days may be necessary for some patients to maintain LDH reduction Concomitant medications allowed: Steroids, immunosuppressant drugs, anti-clotting agents and hematinics 1 SOLIRIS (eculizumab) [package insert]. Alexion Pharmaceuticals; Hillmen P et al. N Engl J Med. 2004;350(6):
6 Lactate Dehydrogenase (U/L) 86% Reduction in LDH: TRIUMPH and SHEPHERD % response after the first dose TRIUMPH Placebo/Extension TRIUMPH SOLIRIS /Extension SHEPHERD SOLIRIS Time, Weeks TRIUMPH placebo patients switched to SOLIRIS after week 26. All TRIUMPH patients entered the long-term extension study. P<0.001 at all measured time points. Hillmen P et al. Blood. 2007;110(12):
7 Median Units Transfused 73% Reduction in Mean Units Transfused Across all Subgroups: TRIUMPH Patients not on SOLIRIS (n=44) SOLIRIS (n=43) * * * * (n=87) (n=30) (n=35) (n=22) Overall >25 Pre-treatment Transfusion Strata 51% of SOLIRIS patients achieved transfusion independence vs 0% of patients not on SOLIRIS 1 Patients with concomitant bone marrow dysfunction may continue to require minimal transfusions *P< Transfusion data obtained during 12 months before treatment; values were normalized for a 6-month period 1. Hillmen P et al. N Engl J Med. 2006;355; Schubert J. Br. J Haematol. 2008;142(2):
8 Standard Effect Size (SES) Patients Report Rapid and Sustained Improvement Across Broad Range of Measures Cognitive* Pain* Insomnia* Constipation Nausea Diarrhea Large Impact Moderate Impact Small Impact 0 FACIT-Fatigue EORTC Fatigue EORTC Functioning *P<0.05. P< Brodsky R et al. Blood. 2006;108(11): Abstract Data on file. Alexion Pharmaceuticals. Global Health Physical Role Dyspnea EORTC Symptoms 8
9 Thrombotic Events (#) 92% Reduction in Thrombotic Events Pre-SOLIRIS Treatment N=195 P= SOLIRIS Treatment 63% of patients received concomitant anticoagulants 1 The effect of anticoagulant withdrawal was not studied 2 Events observed in both venous and arterial sites 3 PI: There were fewer thrombotic events with SOLIRIS treatment than during the same period of time prior to treatment. 1.Brodsky R et al. Blood. 2008;111(4): SOLIRIS (eculizumab) [package insert]. Alexion Pharmaceuticals; Hillmen P, et al. Blood. 2007;110:
10 Is the Primary Cause of Fatigue in PNH Anemia or Hemolysis? 10
11 Hemoglobin, g/dl Change from Baseline FACIT-Fatigue Score TRIUMPH Demonstrated that Improvement in Fatigue Occurred Independent of Hemoglobin Response or more points denotes a clinically significant improvement P<0.001 FACIT-Fatigue Score Hgb Level FACIT-Fatigue Score Time, Weeks SOLIRIS (n=43) Patients not on SOLIRIS (n=44) SOLIRIS Hgb In SHEPHERD, 78% patients reported a significant improvement in fatigue 1 FACIT = Functional Assessment of Chronic Illness Therapy Adapted from: Hillmen P et al. NEJM. 2006;355: Brodsky R et al. Blood Rev. 2008; 22: Hill A et al. Haematologica. 2008; 93 (Suppl 1): 359. Abstract Brodsky R et al. Blood. 2008;111:
12 What Is The Long-Term Experience with SOLIRIS? 12
13 SOLIRIS PNH Clinical Studies Pilot Study NEJM N = 11 Primary endpoint: reduction of hemolysis TRIUMPH NEJM Pivotal Phase III, Double-Blind, Placebo-Controlled Trial, N = 87 SHEPHERD Blood Broader patient population, including those receiving minimal transfusions or with thrombocytopenia, N = 97 Long-Term Extension Trial Hillmen Blood Evaluated long-term safety, efficacy and effect on thrombosis; Placebo patients switched to SOLIRIS N =
14 Patien t (n) 187/ Study Year Lactate Dehydrogenase, U/L * * * * 86% Reduction in LDH Sustained Out Past 4 ½ Years: Long-Term Extension Results *P<0.001 P= patients who participated in the pilot study demonstrated sustained reduction in LDH out past 5 years Patients followed for up to 54 months Socié G et al. Blood. 2007;110(11): Abstract SOLIRIS (eculizumab) [package insert]. Alexion Pharmaceuticals; 2009.
15 Summary of Clinical Efficacy In clinical trials, SOLIRIS significantly reduced hemolysis 1 the underlying cause of morbidity and mortality in PNH 86% sustained reduction in hemolysis as measured by LDH 2 Fewer thrombotic events were observed with SOLIRIS in clinical trials 1,3 The majority of patients (63%) received concomitant anticoagulant therapy 1 The effect of anticoagulant withdrawal during SOLIRIS treatment has not been studied 1 78% clinically meaningful improvement in fatigue Fatigue in PNH impacted by hemolysis Significant improvement noted in pain and dyspnea along with a broad range of QoL measures 4 73% reduction in need for transfusions across all patient populations 2 1. SOLIRIS (eculizumab) [package insert]. Alexion Pharmaceuticals; Hillmen P et al. N Engl J Med. 2006;355: Hillmen P et al. Blood. 2007;110(12): Socie G et al. Blood. 2007;110(11)::Abstract
16 Important Safety Information About SOLIRIS All Patients Should Receive a Medication Guide Before Starting SOLIRIS Treatment Please See Full Prescribing Information for SOLIRIS
17 Warning WARNING: SERIOUS MENINGOCOCCAL INFECTION SOLIRIS increases the risk of meningococcal infections. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early. Vaccinate patients with a meningococcal vaccine at least 2 weeks prior to receiving the first dose of SOLIRIS Revaccinate according to current medical guidelines for vaccine use Monitor patients for early signs of meningococcal infections, evaluate immediately if infection is suspected, and treat with antibiotics if necessary SOLIRIS (eculizumab) [package insert]. Alexion Pharmaceuticals;
18 Safety: Contraindications SOLIRIS is contraindicated for patients with unresolved serious Neisseria meningitidis infection or who are not currently vaccinated against Neisseria meningitidis SOLIRIS (eculizumab) [package insert]. Alexion Pharmaceuticals;
19 Safety: Warnings and Precautions SOLIRIS therapy increases the risk of meningococcal infections. Meningococcal infection may become rapidly lifethreatening or fatal if not recognized and treated early All patients must be vaccinated against Neisseria meningitidis 2 weeks prior to receiving SOLIRIS Use caution when administering SOLIRIS to patients with any systemic infection Other infections: SOLIRIS blocks terminal complement; therefore patients may have increased susceptibility to infections, especially with encapsulated bacteria Use caution when administering SOLIRIS to patients with any systemic infection SOLIRIS (eculizumab) [package insert]. Alexion Pharmaceuticals;
20 Safety: Warnings and Precautions (cont) The effect of withdrawal of anticoagulant therapy during SOLIRIS treatment has not been established. Therefore, treatment with SOLIRIS should not alter anticoagulant management Patients who discontinue SOLIRIS must be monitored closely for signs of serious hemolysis If serious hemolysis occurs after SOLIRIS discontinuation, consider the following procedures/treatments: blood transfusion (packed RBCs), or exchange transfusion if the PNH RBCs are >50% of the total RBCs by flow cytometry; anticoagulation; corticosteroids; or reinstitution of SOLIRIS In clinical trials, 16 of 196 PNH patients discontinued SOLIRIS treatment; no serious hemolysis was observed SOLIRIS (eculizumab) [package insert]. Alexion Pharmaceuticals;
21 Safety: Warnings and Precautions (cont) LDH levels may be used to monitor hemolysis SOLIRIS dose adjustment to every 12 days may be necessary for some patients to maintain LDH reduction Infusion reactions may occur In clinical trials, no patients experienced infusion reactions that required discontinuation SOLIRIS treatment should be interrupted in all patients experiencing severe infusion reactions and appropriate medical therapy administered SOLIRIS (eculizumab) [package insert]. Alexion Pharmaceuticals;
22 Serious Adverse Events: Clinical Trial Experience Meningococcal infections are the most important adverse events that may be experienced by patients receiving SOLIRIS In clinical studies, 2 out of 196 patients developed serious meningococcal infections while receiving treatment with SOLIRIS Both patients had been vaccinated In clinical studies among non-pnh patients, meningococcal meningitis occurred in one patient, who was unvaccinated In post-marketing experience, cases of serious or fatal meningococcal infections have been reported SOLIRIS (eculizumab) [package insert]. Alexion Pharmaceuticals;
23 Adverse Reactions Reported in 5% of SOLIRIS Treated Patients in TRIUMPH Patients, n (%) Reaction SOLIRIS (n = 43) Placebo (n = 44) Headache 19 (44) 12 (27) Nasopharyngitis 10 (23) 8 (18) Back pain 8 (19) 4 (9) Nausea 7 (16) 5 (11) Fatigue 5 (12) 1 (2) Cough 5 (12) 4 (9) Herpes simplex virus infections 3 (7) 0 Sinusitis 3 (7) 0 Respiratory tract infection 3 (7) 1 (2) Constipation 3 (7) 2 (5) Myalgia 3 (7) 1 (2) Pain in extremity 3 (7) 1 (2) Influenza-like illness 2 (5) 1 (2) SOLIRIS (eculizumab) [package insert]. Alexion Pharmaceuticals;
24 Patient Counseling Prior to treatment, patients should be informed and fully understand: The risks and benefits of SOLIRIS, in particular the risk of meningococcal infection Meningococcal vaccine does not prevent all meningococcal infections They are required to receive a meningococcal vaccination at least 2 weeks prior to receiving the first dose of SOLIRIS, if they have not previously been vaccinated There is a potential for serious hemolysis when SOLIRIS is discontinued and that they will be monitored by their healthcare professional for at least 8 weeks following SOLIRIS discontinuation SOLIRIS (eculizumab) [package insert]. Alexion Pharmaceuticals;
25 SOLIRIS OneSource Program OneSource provides education, assistance with access and treatment support for people living with paroxysmal nocturnal hemoglobinuria (PNH) and their caregivers. It is staffed by Alexion Nurse Case Managers, who are registered nurses with healthcare and insurance experience. 25
26 Patients should be informed that they will be provided with a Patient Safety Card Patients should carry the card with them at all times The card describes symptoms, which if experienced, should prompt the patient to seek immediate medical attention Instruct patients to show the card to all health care providers involved in their care Patient Safety Card SOLIRIS (eculizumab) [package insert]. Alexion Pharmaceuticals;
27 Global, observational, non-interventional study to collect real world safety, effectiveness and QoL data Open to all physicians treating patients with PNH regardless of therapy Objectives: Database for publications to enhance understanding of disease and improve outcomes Promote evidence-based medicine Current enrollment: Over 500 patients enrolled Participation in 14 countries, including the United States, Argentina, Denmark, Netherlands, Belgium, Australia, France, New Zealand, Germany, and Taiwan Enrollment information: (800) or 27
28 Thank You Jack Goldberg M.D. FACP Clinical Professor of Medicine University of Pennsylvania
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