BioCryst Pharmaceuticals, Inc. · NASDAQ:BCRX
BioCryst Pharmaceuticals — EACCI 2019
13 slides from BioCryst Pharmaceuticals's conference presentation, May 30, 2019
Slide 1: Final Results from the ZENITH-1 Study: Oral BCX7353 for HAE Attacks
Final Results from the ZENITH-1 Study: Oral Administration of Plasma Kallikrein Inhibitor BCX7353 for the Treatment of Attacks in Patients with Hereditary Angioedema Dr. Hilary Longhurst Department of Immunology, Addenbrooke’s Hospital, Cambridge University Hospitals NHS Foundation Trust, Cambridge,
Slide 2: Disclosures
Disclosures In relation to this presentation, I declare the following, real or perceived conflicts of interest: Type Company Employment full time / part time None Research Grant (P.I., BioCryst, CSL, Takeda (Shire), Kalvista collaborator or consultant; pending and received grants) Other research sup
Slide 3: BCX7353: orally available, potent, specific kallikrein inhibitor with demonstrated efficacy in C1-INH-HAE
BCX7353: orally available, potent, specific kallikrein inhibitor with demonstrated efficacy in C1-INH-HAE LSM Attack rate, 62.5 125 250 350 per-protocol analysis mg mg mg mg PK Profile PD Profileǂ WEEKS 2-4 1000 100 % Difference, Active-PBO -9% -74% -47% -58% 64 80 p-Value 0.657 <0.001 0.005 <0.001
Slide 4: ZENITH-1: Designed to conform with current HAE treatment guidelines
ZENITH-1: Designed to conform with current HAE treatment guidelines Drug pdC1-INH pdC1-INH ecallantide icatibant rhC1-INH BCX7353 Study CHANGE IMPACT-1 EDEMA-3 FAST-3 C-131O Trial ZENITH-1 Years subjects enrolled 2005-2007 2005-2007 2005-2007 2009-2010 2011-2012 2017-2019 Route IV infusion IV infusi
Slide 5: Design of ZENITH-1 Phase 2: At-home self-administered oral treatment
Design of ZENITH-1 Phase 2: At-home self-administered oral treatment Study methods are aligned with the current guidelines for on-demand treatment1,2 Screening Treatment Follow-Up ZENITH-1 protocol instructions Sequence 1: A, P, A • Subjects were to call the site PI and treat End of attacks within 1
Slide 6: Demographics and HAE attack metrics
Demographics and HAE attack metrics Part 1 Part 2 Part 3 Demographics* 750 mg 500 mg 250 mg Subjects randomized, n 36 15 12 Age in years, mean (SD) 44 (13) 42 (11) 35 (11) Sex, % female 52% 79% 64% Subjects treating 0/1/2/3 attacks, n 3/1/2/30 1/3/0/11 1/0/1/10 HAE attack metrics Attacks treated, ac
Slide 7: BCX7353 750 mg effectively treated abdominal and peripheral attacks
BCX7353 750 mg effectively treated abdominal and peripheral attacks Abdominal pain Cutaneous swelling Abdominal pain median VAS score (mm) for attacks with pre-dose VAS > 0* Cutaneous swelling median VAS score (mm) for attacks with pre-dose VAS > 0* 50 50 BCX7353 750 mg, n=53 attacks BCX7353 750 mg,
Slide 8: Dose-response in efficacy measures
Dose-response in efficacy measures Efficacy endpoints (difference, active-placebo) for each dose level in ZENITH-1 Endpoint 750 mg 500 mg 250 mg Number of attacks, active/placebo 64/31 25/11 21/11 ∆ from baseline in VAS at 4 hr, mm‡ -6.98*** -2.1 0.57 Improved or stable VAS at 4 hr, %€ 21.0* 18.5 6.
Slide 9: Dose-response observed in VAS scores, rescue medication use, and symptoms
Dose-response was observed in VAS scores, use of rescue medication, and improvement in symptoms CFB in VAS§ Use of Rescue Rx ¥ Symptoms ¥ placebo better Improved or Stable BCX7353 better § Change from baseline in 3-Composite VAS at 4 hours post-dose (least-squares mean difference from placebo in mm,
Slide 10: Safety summary, attack-level analysis
Safety summary, attack-level analysis BCX7353 All Placebo 750 mg 500 mg 250 mg Attacks treated, n* 64 25 21 53 Attacks with a reported TEAE, n (%) 16 (25.0%) 10 (40.0%) 10 (47.6%) 17 (32.1%) Attacks with a serious TEAE, n¥ 0 1 (4.0%) 0 1 (1.9%) Attacks with a drug-related TEAE as assessed by 7 (10.9
Slide 11: Most common AEs, attack-level analysis
Most common AEs, attack-level analysis BCX7353 Adverse Event (AE) Summaryǂ All 750 mg 500 mg 250 mg Placebo Number of treated attacks 64 25 21 53 Most common AEs Diarrhea 3 (4.7%) 3 (12.0%) 0 2 (3.8%) Abdominal Pain 2 (3.1%) 3 (12.0%) 1 (4.8%) 1 (1.9%) Nausea 2 (3.1%) 2 (8.0%) 2 (9.5%) 0 Headache 3
Slide 12: ZENITH-1 Proof-of-Concept Phase 2 Conclusions
ZENITH-1 Proof-of Concept Phase 2 Conclusions ◆ ZENITH-1 was a novel, early intervention trial of oral BCX7353 in the treatment of HAE attacks • Subjects self-treated with study drug at home within an hour onset of symptoms • Subjects were responsible for evaluating and documenting evolving HAE atta
Slide 13: Authors and Acknowledgments
Authors and Acknowledgments Authors Marcin Stobiecki (PL) Andrea Zanichelli (IT) Aarnoud Huissoon (GB) Dumitru Moldovan (RO) Marcus Maurer (DE) Markus Magerl (DE) Clinical trial staff at each Mauro Cancian (IT) Ricardo Senter (IT) Ania Manson (GB) participating clinical trial Emel Aygören-Pürsün V