Biocryst Pharmaceuticals, Inc. · NASDAQ:BCRX
Biocryst Pharmaceuticals — Corporate Update (September 2020)
13 slides from Biocryst Pharmaceuticals's corporate update, Sep 30, 2020
Slide 1: BCX9930 Data Update
BCX9930 Data Update September 30, 2020
Slide 2: Forward-Looking Statements
Forward-Looking Statements BioCryst’s presentation may contain forward-looking statements, including statements regarding future results, unaudited and forward-looking financial information and company performance or achievements. These statements are subject to known and unknown risks and uncertain
Slide 3: Six Major Program Updates Expected in Next 100 Days
Six Major Program Updates Expected in Next 100 Days Q32020 Q42020 PDUFA 2021 Dec. 3, 2020 Data update from Data from part 1 ORLADEYO Approval decision Data update from Phase 1 data from BCX9930 200/400 mg galidesivir COVID-19 trial approval in U.S. on ORLADEYO BCX9930 200/400 mg BCX9250 trial in FOP
Slide 4: Factor D: An Outstanding Drug Target for Complement-mediated Diseases
Factor D: An Outstanding Drug Target for Complement-mediated Diseases •Factor D is essential to initiate the Alternative Pathway •Blocking Factor D blocks the Alternative Pathway and all downstream products Spectrum of Alternative Pathway Dysregulation Diseases Factor D BCX9930 Hematology Rheumatolo
Slide 5: Oral Monotherapy with BCX9930 Offers Advantage of Controlling Both IVH and EVH
Oral Monotherapy with BCX9930 Offers Advantage of Controlling Both Intravascular (IVH) and Extravascular (EVH) Hemolysis Goal of Total Patient Outcomes from Hemolytic Control Controlled Hemolysis over Time Relieve Anemia IVH Control Hemoglobin LDH Hemolytic Control RBC/Granulocyte Reduce PNH Clinica
Slide 6: PNH Proof of Concept Study Design: BCX9930 as Monotherapy
PNH Proof of Concept Study Design: BCX9930 as Monotherapy BCX9930 Study Design: Patients with PNH who are Naïve to C5-INH Treatments Days 1 - 14 Days 15 - 28 Extension after 28 days Cohort 1 (N = 4 enrolled) 50 mg BID 100 mg BID Patients benefitting on treatment may continue on BCX9930 and dose- Coh
Slide 7: Treatment-naïve PNH Patients Had Severe Disease Prior to Treatment
Treatment-naïve PNH Patients Had Severe Disease Prior to Treatment Pre-treatment Characteristics Cohort 1 Cohort 2 Sequential Patient # in Cohort 1 2 3 4 1 2 3 Patient Code A B C D E F G PNH duration, years 8 4 4 5 2 5 1 Compromised bone marrow function no no yes no yes yes yes History of thrombosis
Slide 8: Duration of BCX9930 Treatment in PNH Patients
Duration of BCX9930 Treatment in PNH Patients 50 mg BID 100 mg BID 200 mg BID 400 mg BID Patient A Cohort 1 Patient B Patient C Data update from 4 patients on 400 mg BID an Patient D average of 53 days to date Patient E Cohort 2 Patient F Patient G 0 28 56 84 112 140 168 196 Days on therapy with BCX
Slide 9: Meaningful Changes in Key Biomarkers Indicating Control of Hemolysis
Meaningful Changes in Key Biomarkers Indicating Control of Hemolysis Hemoglobin Patient Duration at Hemoglobin RBC Clone Size % g/dL 400 mg BID g/dL of Granulocyte # of Clone Size Transfusions @ 200/400 14 Most Most mg Pre-Rx Pre-Rx Recent Recent 12 B 56 days 7.0 11.4 42% 100% 0 C 57 days 6.0 9.5 53
Slide 10: BCX9930 Dose-response in Hemoglobin and LDH in PNH Patients
BCX9930 Dose-response in Hemoglobin and LDH in PNH Patients Patient B Patient C Patient D Patient E Dose, mg BID Dose, mg BID Dose, mg BID Dose, mg BID 14 500 14 500 14 500 14 500 12 12 12 12 400 400 400 400 10 10 10 10 300 300 300 300 8 8 8 8 6 6 6 6 200 200 200 200 4 4 4 4 100 100 100 100 2 2 2 2
Slide 11: Hemolysis Biomarkers and Clinical Assessment Support Clinical Benefit of BCX9930 as Monotherapy in PNH
Hemolysis Biomarkers and Clinical Assessment Support Clinical Benefit of BCX9930 as Monotherapy in PNH Clinical Data at 400 mg BID Dose-dependent and clinically meaningful changes in key disease biomarkers were observed • Mean hemoglobin increase from baseline was 3.8 g/dL • Hb maintained at 400 mg
Slide 12: BCX9930 has been Safe and Well Tolerated in PNH Patients
BCX9930 has been Safe and Well Tolerated in PNH Patients Overall Safety • No discontinuations due to related AEs • No BCX9930-related serious AEs or safety signals • No safety signals in routine monitoring of vital signs, ECGs, or laboratory evaluations of hematology, coagulation, urinalysis, or cli
Slide 13: Next Steps for BCX9930 Development
Next Steps for BCX9930 Development Q32020 Q42020 2021 Data update update from from Complete dose-ranging Meet with regulators Data update from Begin multiple BCX9930 200/400 200/400mgmg in C5 inhibitor naïve to discuss advanced BCX9930 200/400 mg advanced development treatment-naïve PNH patients wit
BioCryst Pharmaceuticals — Corporate Update (September 2020) on 6ix