Slide 1: Long-Term Safety and Efficacy of Oral Deucrictibant: RAPIDe-2 Extension Study
Long-Term Safety and Efficacy of Oral Deucrictibant for Treatment of Hereditary Angioedema Attacks: Results of the RAPIDe-2 Extension Study G. Spadaro, J. Anderson, E. Aygören-Pürsün, L. Bouillet, H. Chapdelaine, H. Farkas, D. Gobert, R. Hakl, J.S. Jacobs, R. Lleonart, M.E. Manning, A. Reshef, M. St
Slide 2: Conflicts of interest disclosure
Conflicts of interest disclosure Research grant support, consultancy fees, speaker fees, advisory board, investigator, and/or clinical trial fees G.S.: Pharvaris, Takeda; J.A.: BioCryst, BioMarin, CSL Behring, Cycle Pharma, KalVista, Pharming, Pharvaris, Takeda; E.A-P.: Astria, BioCryst, CSL Behring
Slide 3: Hereditary angioedema (HAE) is a bradykinin-mediated condition with unmet medical needs
Hereditary angioedema (HAE) is a bradykinin-mediated condition with unmet medical needs Bradykinin is the main mediator of the clinical manifestations of HAE attacks.1 Current guidelines recommend HAE attacks are treated as early as possible.2-4 Parenteral administration often leads to on-dema
Slide 4: Deucrictibant is a selective, bradykinin B2 receptor antagonist
Deucrictibant is a selective, bradykinin B2 receptor antagonist Currently under development for both prophylactic and on-demand treatment of HAE attacks1-9: DEUCRICTIBANT DEUCRICTIBANT extended-release (XR) tablet immediate-release (IR) capsule sustained absorption9 rapid absorption2 Plasma concen
Slide 5: RAPIDe-2: a two-part, double-blind Phase 2/3 extension study
RAPIDe-2: a two-part, double-blind Phase 2/3 extension study of deucrictibant for on-demand treatment of repeat HAE attacks Part A: Part B: Double-blind phase Open-label phase Deucrictibant IR capsule 10 mg RAPIDe-1 Deucrictibant IR participants Deucrictibant IR capsule 20 mg capsule 20 mg Deucricti
Slide 6: RAPIDe-2 study endpoints and post-hoc analyses
RAPIDe-2 study endpoints and post-hoc analyses Primary endpoint: safety including TEAEs, clinical laboratory tests, vital signs, and ECG findings. Secondary endpoints: efficacy endpoints using PRO tools PGI-C and PGI-S. Post-hoc analyses: Safety and efficacy for on-demand treatment of upper ai
Slide 7: Deucrictibant IR capsule was well tolerated across all doses
Deucrictibant IR capsule was well tolerated across all doses Participants who received ≥1 dose of Deucrictibant IR capsule deucrictibant IR capsule in the study (Combined dose group) at data cutoff (10 June 2024). Adverse events (n=19) 337 attacks from 19 participants. Attacks with any TEAE, n
Slide 8: Median time to achieving key efficacy endpoints
Median time to achieving key efficacy endpoints At data cutoff (01 March 2024): 265 attacks from 17 participants. Onset of Reduction in Substantial Complete symptom relief attack severity symptom relief attack resolution Median time by PGI-C by PGI-S by PGI-C by PGI-S to (95% CIs) 1.1 hours 2.6 ho
Slide 9: Majority of attacks achieved key efficacy endpoints within timeframe
Majority of attacks achieved key efficacy endpoints within timeframe of attacks achieved of attacks achieved 98.5% onset of symptom relief by 12 hours 97.7% reduction in attack severity by 12 hours (261/265) (255/261a) PGI-C PGI-S of attacks achieved of attacks achieved 96.2% substantial symptom rel
Slide 10: Similar times to symptom relief for upper airway and other attacks
Similar times to symptom relief for upper airway and other attacks Difficulty in swallowing and/or voice change were reported as attack manifestations of 3 upper airway attacks before treatment. Non-upper airway Upper airway attacks in participants Total non-upper attacks with upper airway airway
Slide 11: Majority of upper airway attacks were treated with a single dose of deucrictibant
Majority of upper airway attacks were treated with a single dose of deucrictibant 85.7% 97.4% 85.4% of upper airway of non-upper of all attacksa airway attacks in non-upper (6/7) participants with airway attacks upper airway attacks (280/328) (113/116) 1 dose of deucrictibant IR capsule 2nd dose of
Slide 12: Majority of upper airway and other attacks did not use rescue medication by 48 hours
Majority of upper airway and other attacks did not use rescue medication by 48 hours post-treatment 85.7% 100% 98.5% of upper of non-upper of all non-upper airway attacksa,b airway attacks in airway attacks (6/7) participants with (323/328) upper airway attacks (116/116) Not treated with rescue medi
Slide 13: Conclusions
Conclusions These data provide additional evidence on the long-term safety and efficacy of deucrictibant IR capsule for on-demand treatment of repeat HAE attacks. • Safety: Deucrictibant was well tolerated with no safety signals. • Efficacy – All attacks: ─ Median time to onset of symptom relief was