Screen Washout Omalizumab 150 mg Omalizumab 300 mg G ro u p A G ro u p B Follow- up UAS7 ≤6 UAS7 <16 Stop therapy Omalizumab 300 mg Omalizumab 150 mg R a n d o m iz a ti o n 4 :3 −5 to −1Week 0 4 8 12 16 2420 0 4 8 12 0 4 UAS7 >6 UAS7 ≤6 UAS7 <16 Stop therapy Omalizumab 300 mg Omalizumab 300 mg UAS7 ≥16 UAS7 ≥16 UAS7 >6 Screen 1st Dosing Study treatment withdrawal 2nd Dosing Follow- up 0 4 8 REFERENCES 1. ClinicalTrials.gov – NCT02161562. 2. Sussman G, et al. Design and rationale of the OPTIMA study: retreatment or step-up therapy with omalizumab in patients with chronic idiopathic/spontaneous urticaria (CIU/CSU). FCDC 2017. Poster. ACKNOWLEDGMENTS The complete OPTIMA study team comprised: 35 active sites in the following countries: Argentina, Brazil, Canada, Chile, Dominican Republic, Guatemala, Mexico, and Panama; Syreon Clinical Research for project management, data management, and medical writing; and Novartis Pharmaceuticals Canada and Novartis in participating countries. All authors participated in the development of the poster and approved the final poster for presentation. Editorial assistance in the development of this poster was provided by Jessica Donaldson of Fishawack Communications Ltd, Oxford, UK. This poster was previously presented at the European Academy of Allergy and Clinical Immunology Congress, June 17–21, 2017, Helsinki, Finland. FUNDING This study was funded by Novartis Pharmaceuticals Canada Inc. DISCLOSURES Authors declare the following, real or perceived conflicts of interest: GS, JH, WG, CL, and WHY received honoraria as investigators and consultants. GS received honoraria as speaker of this corresponding study. OC, AV, FdT, and LR are employees of Novartis Pharmaceuticals. CONTACT INFORMATION Lenka Rihakova – Lenka.Rihakova@novartis.com Antonio Vieira – Antonio.Vieira@novartis.com Novartis Pharmaceuticals Canada: +1(514) 631 6775 INTRODUCTION • The OPTIMA (efficacy of optimized retreatment and step-up therapy with omalizumab in patients with chronic idiopathic/spontaneous urticaria [CIU/CSU]; NCT02161562) study was designed to address some of the key gaps in the knowledge of optimal CIU/CSU treatment with omalizumab • Owing to the intermittent nature of CIU/CSU, physicians may want to consider stopping omalizumab treatment in patients who are symptom free for a period of time • Symptoms may re-emerge after a period of treatment withdrawal; the primary objective of the study was therefore to determine the efficacy and safety of retreatment in patients who respond to an initial course of omalizumab OBJECTIVES • Four objectives were to be answered in OPTIMA: − If a patient is well controlled and therefore treatment is stopped, will the patient relapse? How long will it take to relapse? − If treatment is restarted, will the patient respond to retreatment? − If the patient does not respond to omalizumab 150 mg, will step-up therapy help? − If the patient does not respond to omalizumab 300 mg, will treatment extension help? • This poster will cover the first two questions METHODS Study design • OPTIMA is a Phase 3b, international, multicenter, randomized, open-label, noncomparator study.1 For details about the study design, please see the companion poster being presented at this congress (Sussman et al. FCDC 2017)1,2 • Patients with CIU/CSU who were symptomatic despite H 1 -antagonists were randomized 4:3 to omalizumab 150 mg or 300 mg for 24 weeks (1st dosing period) • Based on weekly urticaria activity scores (UAS7), patients entered one of the following phases: treatment withdrawal (if UAS7 ≤6), step-up to 300 mg (if 150 mg initially and UAS7 >6 at Weeks ≥8 to 24), or continued treatment for 12 more weeks (if 300 mg initially and UAS7 >6 at Week 24) CONCLUSIONS • After being well controlled (UAS7 ≤6), upon withdrawal 44.4% of patients on omalizumab 150 mg and 50.0% of patients on omalizumab 300 mg relapsed (UAS7 ≥16) • The overall time to relapse for both dosages was 4.7 weeks • Retreatment with both dosages is effective. Overall, 87.8% of patients regained symptom control upon retreatment, after initially being well controlled and subsequent relapse 314 randomized patients Omalizumab 300 mg 136 patients Omalizumab 150 mg 178 patients Discontinued 10 patients (5.6%) Well controlled 27 patients (15.2%) Not controlled – step-up 141 patients (79.2%) Discontinued 5 patients (3.7%) Well controlled 88 patients (64.7%) Extended treatment 43 patients (31.6%) Relapsers 12 patients (44.4%) Nonrelapsers 15 patients (55.6%) Nonrelapsers 44 patients (50.0%) Relapsers 44 patients (50.0%) Figure 3. Disposition after withdrawal period – patients to receive retreatment 314 randomized patients Omalizumab 150 mg, 178 patients Omalizumab 300 mg, 136 patients Figure 2. Patient randomization ratio • If patients relapsed (UAS7 ≥16) upon withdrawal, they were retreated with their starting dose for 12 weeks Inclusion criteria • Men or women at least 18 years of age • Diagnosis of CIU/CSU and the presence of symptoms for ≥6 months prior to the screening visit • Patients must have been on an approved dose of nonsedating H 1 -antihistamine for CIU/CSU, and no other concomitant CIU/CSU treatment, for at least the 7 consecutive days immediately prior to the randomization visit and must have documented current use on the day of the randomization visit • UAS7 ≥16 (scale 0−42) and itch component of UAS7 ≥8 (scale 0−21) during 7 days prior to randomization Exclusion criteria • Patients with a clearly defined underlying etiology for chronic urticaria other than CIU/CSU • Patients with urticarial vasculitis, urticaria pigmentosa, erythema multiforme, mastocytosis, hereditary or acquired angioedema, lymphoma or leukemia, active atopic dermatitis, bullous pemphigoid, dermatitis herpetiformis, senile pruritus, or other skin disease associated with itch that could interfere with study outcomes • Patients with a history of malignancy of any organ system • Patients should stay on same approved dose of nonsedating H 1 -antihistamine during all trial duration. No rescue medication allowed RESULTS Baseline characteristics OMALIZUMAB RETREATMENT OF PATIENTS WITH CHRONIC IDIOPATHIC URTICARIA/SPONTANEOUS URTICARIA (CIU/CSU) FOLLOWING RETURN OF SYMPTOMS: PRIMARY RESULTS OF THE OPTIMA STUDY Gordon Sussman,1 Jacques Hébert,2 Wayne Gulliver,3 Charles Lynde,4 William H. Yang,5 Olivier Chambenoit,6 Antonio Vieira,7 Frederica DeTakacsy,7 Lenka Rihakova7 1Department of Medicine, University of Toronto, Toronto, ON, Canada; 2Department of Medicine, Centre Hospitalier de l’Université Laval, Québec, QC, Canada; 3Faculty of Medicine, Memorial University of Newfoundland, St. John’s, NL, Canada; 4Lynde Institute for Dermatology, Markham, ON, Canada; 5Ottawa Allergy Research Corporation, University of Ottawa Medical School, ON, Canada; 6Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA; 7Novartis Pharmaceuticals Canada Inc., Dorval, QC, Canada Table 1. Demographics and baseline characteristics Characteristic Omalizumab 150 mg (n=178) Omalizumab 300 mg (n=136) Overall (N=314) Age, mean (range), years 46.7 (18–79) 45.8 (20–78) 46.3 (18–79) Gender, % Male Female 27.0 73.0 27.2 72.8 27.1 72.9 Race, % White Asian Black Am. Indian/Alaska Native Other 76.4 8.4 5.6 1.1 8.4 83.1 7.4 4.4 2.2 2.9 79.3 8.0 5.1 1.6 6.1 Time to CIU/CSU symptoms, n (%) ≤1 year >1–≤2 years >2–10 years >10 years 28 (15.7) 25 (14.0) 84 (47.2) 41 (23.0) 22 (16.2) 25 (18.4) 54 (39.7) 35 (25.7) 50 (15.9) 50 (15.9) 138 (43.9) 76 (24.2) Baseline UAS7, mean (range) 29.7 (16.0–42.0) 30.0 (16.0–42.0) 29.8 (16.0–42.0) # Prior medications used for CIU/CSU, mean (range) 1.8 (0.0–12.0) 2.1 (0.0–8.0) 1.9 (0.0–12.0) CIU/CSU, chronic idiopathic/spontaneous urticaria; UAS7, weekly urticaria activity score. Table 2. Mean time to replase Omalizumab 150 mg Omalizumab 300 mg Overall 4.8 weeks 4.7 weeks 4.7 weeks Scan this QR code Visit the web at: http://novartis.medicalcongressposters.com/Default.aspx?doc=44671 Mobile Friendly e-Prints 3 ways to instantly download an electronic copy of this poster to your mobile device or e-mail a copy to your computer or tablet Text Message (SMS) Text: Q44671 to: 8NOVA (86682) US Only +18324604729 North, Central and South Americas; Caribbean; China +447860024038 UK, Europe & Russia +46737494608 Sweden, Europe Standard data or message rates may apply. Presented at the Fall Clinical Dermatology Conference, October 12–15, 2017, Las Vegas, NV, USA Figure 1. Study design for retreated patients UAS7, weekly urticaria activity score. 40 35 30 25 20 M e a n ( ± 9 5 % C I) U A S 7 15 10 5 0 Day 0 Month 1 Month 2 Month 3 1st Dosing period Month 4 Month 5 Month 6 Month 7 Month 8 Month 9 Month 10 Month 11 Omalizumab 150 mg (n=12) 29.8 1.3 3.8 29.4 Omalizumab 300 mg (n=37*) 30.1 1.2 28.3 2.3 Withdrawal period 2nd Dosing period Figure 4. Mean UAS7 of retreated patients throughout the study *7 out of 44 patients on omalizumab 300 mg did not complete the 2nd dosing period or did not submit the participant diary as per protocol. CI, confidence interval; UAS7, weekly urticaria activity score. P a ti e n ts ( % ) 83.3% Omalizumab 150 mg (n=12) 89.2% Omalizumab 300 mg (n=37*) 87.8% Overall (N=49) 100 75 50 25 0 Figure 5. Proportion of patients regaining symptom control upon retreatment *7 out of 44 patients on omalizumab 300 mg did not complete the 2nd dosing period or did not submit the participant diary as per protocol. Error bars represent 95% confidence intervals.