Fixed-Dose Subcutaneous C1-Inhibitor Liquid for Prophylactic Treatment of C1-INH- HAE: SAHARA Randomized Study William R. Lumry, MD, Inmaculada Martinez-Saguer,

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Presentation transcript:

Fixed-Dose Subcutaneous C1-Inhibitor Liquid for Prophylactic Treatment of C1-INH- HAE: SAHARA Randomized Study William R. Lumry, MD, Inmaculada Martinez-Saguer, MD, William H. Yang, MD, Jonathan A. Bernstein, MD, Joshua Jacobs, MD, Dumitru Moldovan, MD, Marc A. Riedl, MD, MS, Douglas T. Johnston, DO, H. Henry Li, MD, Yongqiang Tang, PhD, Jennifer Schranz, MD, Peng Lu, MD, Moshe Vardi, MD, Henriette Farkas, MD P. Keith, W. Yang, M. Maurer, I. Martinez-Saguer, H. Farkas, A. Reshef, S. Kivity, D. Moldovan, T. Caballero, M. Guilarte, M.D. Hernandez, M.T. González-Quevedo, A. Banerji, J. Bernstein, A. Bewtra, T. Craig, S. Fineman, R. Gower, J. Jacobs, D. Johnston, J. Kashkin, H.H. Li, W.R. Lumry, M. Manning, D. McNeil, I. Melamed, N. Mumneh, T. Nickel, J. Panuto, D. Soteres, R. Tachdjian, J. Offenberger, J. Wedner William R. Lumry, MD, Inmaculada Martinez-Saguer, MD, William H. Yang, MD, Jonathan A. Bernstein, MD, Joshua Jacobs, MD, Dumitru Moldovan, MD, Marc A. Riedl, MD, MS, Douglas T. Johnston, DO, H. Henry Li, MD, Yongqiang Tang, PhD, Jennifer Schranz, MD, Peng Lu, MD, Moshe Vardi, MD, Henriette Farkas, MD P. Keith, W. Yang, M. Maurer, I. Martinez-Saguer, H. Farkas, A. Reshef, S. Kivity, D. Moldovan, T. Caballero, M. Guilarte, M.D. Hernandez, M.T. González-Quevedo, A. Banerji, J. Bernstein, A. Bewtra, T. Craig, S. Fineman, R. Gower, J. Jacobs, D. Johnston, J. Kashkin, H.H. Li, W.R. Lumry, M. Manning, D. McNeil, I. Melamed, N. Mumneh, T. Nickel, J. Panuto, D. Soteres, R. Tachdjian, J. Offenberger, J. Wedner The Journal of Allergy and Clinical Immunology: In Practice Volume 7, Issue 5, Pages 1610-1618.e4 (May 2019) DOI: 10.1016/j.jaip.2019.01.021 Copyright © 2019 The Authors Terms and Conditions

Figure 1 Normalized number of HAE attacks during treatment periods. Based on the full analysis set from the crossover sequences, consisting of all patients in the safety set who received ≥1 postbaseline (eg, randomization) primary efficacy assessment. CI, Confidence interval; HAE, hereditary angioedema; LS, least squares; NNA, normalized number of attacks; pdC1-INH, plasma-derived C1-INH; SD, standard deviation. †Reflects the entire study period. ‡Reflects the steady state period. The Journal of Allergy and Clinical Immunology: In Practice 2019 7, 1610-1618.e4DOI: (10.1016/j.jaip.2019.01.021) Copyright © 2019 The Authors Terms and Conditions

Figure 2 Proportion of patients achieving ≥50%, ≥70%, ≥90, and 100% reduction in NNA during treatment periods relative to placebo periods, from day 1 and from day 15. Clinical response (≥50% reduction in NNA vs placebo) was achieved by 77.6% and 76.6% of patients from days 1 and 15, respectively. Based on the full analysis set from the crossover sequences with patients who had data in both treatment periods. The proportion of patients achieving a reduction in normalized number of attacks with C1-INH liquid versus placebo was statistically significant for each category of response (from day 1: ≥50%, P < .0001; ≥70%, P < .0001; ≥90%, P =. 0005; 100%, P = .0233; from day 15: ≥50%, P < .0001; ≥70%, P < .0001; ≥90%, P = .0002; 100%, P = .0058) relative to a predetermined efficacy threshold of 20%. Error bars represent 95% CI values. CI, Confidence interval; NNA, normalized number of attacks; pdC1-INH, plasma-derived C1-INH. The Journal of Allergy and Clinical Immunology: In Practice 2019 7, 1610-1618.e4DOI: (10.1016/j.jaip.2019.01.021) Copyright © 2019 The Authors Terms and Conditions

Figure 3 Maximum attack severity. Based on the full analysis set from the crossover sequences; not restricted to patients who improved or who had data from both treatment periods. Percentages may not total 100 because of rounding. pdC1-INH, Plasma-derived C1-INH; SC, subcutaneous. The Journal of Allergy and Clinical Immunology: In Practice 2019 7, 1610-1618.e4DOI: (10.1016/j.jaip.2019.01.021) Copyright © 2019 The Authors Terms and Conditions

Figure E1 Patient disposition. One patient receiving pdC1-INH liquid experienced 2 TEAEs that led to treatment withdrawal (nausea and headache). Both events were considered treatment related and occurred within 24 hours of administration. Two patients receiving placebo experienced 2 TEAEs leading to withdrawal (one had cardiac arrest and the other had an HAE attack). Neither event was considered treatment related. HAE, Hereditary angioedema; pdC1-INH, plasma-derived C1-INH; SC, subcutaneous. The Journal of Allergy and Clinical Immunology: In Practice 2019 7, 1610-1618.e4DOI: (10.1016/j.jaip.2019.01.021) Copyright © 2019 The Authors Terms and Conditions

Figure E2 Occurrence of HAE attacks, attack severity, and use of on-demand medications. The height of vertical bars indicates the maximum attack severity of each dosing day, and the width indicates attack duration. The red dots indicate acute treatment. The horizontal lines indicate treatment duration. HAE, Hereditary angioedema. The Journal of Allergy and Clinical Immunology: In Practice 2019 7, 1610-1618.e4DOI: (10.1016/j.jaip.2019.01.021) Copyright © 2019 The Authors Terms and Conditions