Biocryst Pharmaceuticals, Inc. · NASDAQ:BCRX
Biocryst Pharmaceuticals — Q2 2020 Earnings Presentation
21 slides from Biocryst Pharmaceuticals's earnings presentation, Aug 6, 2020
Slide 1: Second Quarter 2020 Results Call
Second Quarter 2020 Results Call Corporate Update & Financial Results August 6, 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: Agenda
Agenda ◆ Corporate Update: Jon Stonehouse – President, Chief Executive Officer ◆ Global Berotralstat Launch Update: Charlie Gayer – Chief Commercial Officer Megan Sniecinski – Chief Business Officer ◆ BCX9930 and Galidesivir Clinical Update Dr. Bill Sheridan – Chief Medical Officer ◆ Financial Updat
Slide 4: Global Berotralstat (BCX7353) Launch Update
Global Berotralstat (BCX7353) Launch Update: Charlie Gayer – Chief Commercial Officer Megan Sniecinski – Chief Business Officer 4
Slide 5: Coming Soon: Orladeyo
Coming Soon: Orladeyo™ diggeryst COMING SOON O rl a d eyo orladeyo (berotralstat) 150 mg capsule (berotralstat) 150 mg capsule
Slide 6: Clinical Update
Clinical Update: Dr. Bill Sheridan – Chief Medical Officer 6
Slide 7: Factor D: Outstanding Target for Complement-mediated Diseases
Factor D: Outstanding Target for Complement-mediated Diseases Factor D is an ideal target: Application to BCX9930 Development: Doses of BCX9930 that block Factor D will inhibit the Required for the alternative pathway (AP) to work AP independent of the disease setting Target is the same in PNH, neph
Slide 8: Targeting Overactive Alternative Pathway Could Treat Many Complement-mediated Diseases
Targeting Overactive Alternative Pathway Could Treat Many Complement-mediated Diseases 8
Slide 9: BCX9930 28-day PNH Proof of Concept Study Design
BCX9930 28-day PNH Proof of Concept Study Design Key Outcome Measures • LDH, hemoglobin • Safety • PK • PD Total of 28 days of BCX9930 dosing Period 1 days 1-14 Period 2 days 15-28 Subjects with PNH who are naïve to C5-INH treatments: BCX9930 monotherapy Cohort 1: n = up to 4* 50 mg BID days 1-14 10
Slide 10: Treatment-naïve PNH Patients had Severe Disease
Treatment-naïve PNH Patients had Severe Disease Pre-treatment Characteristics Subject 1 Subject 2 Subject 3 PNH duration, years 8 4 3 History of aplastic anemia no no yes History of thrombosis yes no no LDH, IU/L 2205 2497 835 LDH × ULN 9.8 11.0 3.7 Hemoglobin, g/dL 8.2 7.0 6.0 Reticulocytes, 103cel
Slide 11: Dose-dependent Improvement Across Key Indicators in Treatment-naïve PNH Subjects
Dose-dependent Improvement Across Key Indicators in Treatment-naïve PNH Subjects Receiving BCX9930 Monotherapy Lactate Dehydrogenase (LDH) Reticulocytes Total Bilirubin Hemoglobin PNH Clone Size 14 300 3 12 100 12 10 80 10 200 2 8 Reticulocytes, 10 /L Total Bilirubin x ULN Hemoglobin, g/dL * 60 3 8
Slide 12: BCX9930 Data Provides Strong Support for Oral Monotherapy in PNH
BCX9930 Data Provides Strong Support for Oral Monotherapy in PNH Safety & Tolerability in PNH, n=3 Activity in PNH at low doses, n=3 • Prompt and sustained reductions in LDH (3/3) • BCX9930 has been safe and generally well- and reticulocytes (2/3) tolerated in cohort 1 at low doses of 50 mg bid days
Slide 13: Single Dose PK Profile of Oral BCX9930 in Healthy Subjects
Single Dose PK Profile of Oral BCX9930 in Healthy Subjects PK Profile of Single Dose BCX9930 Dose-Proportional Exposure of Single Dose BCX9930 100000 ng/mL, mean (SEM) Cmax ng/mL AUC0-24h ng.h/mL 10000 mean (SD) Cmax mean (SD) AUC0-24h 100 mg 10000 regression (95% CI band) regression (95% CI band) 3
Slide 14: Suppression of AP Activity After Single Oral Doses of BCX9930
Suppression of AP Activity After Single Oral Doses of BCX9930 Alternative pathway complement activity in healthy subjects : oral BCX9930 single dose Assay: AP Hemolysis Assay: AP Wieslab 100 100 100 mg 100 mg AP Hemolysis, % of predose, mean (SEM) AP Wieslab, % of predose, mean (SEM) 300 mg 300 mg 8
Slide 15: Clear Dose-response in AP Inhibition
Clear Dose-response in AP Inhibition – Consistent, Sustained Suppression at 200/400 mg Q12h AP Hemolysis Individual Subject Plots at Steady-State AP Weislab Individual Subject Plots at Steady-State 100 100 100 100 50 mg Q12h 100 mg Q12h 50 mg Q12h 100 mg Q12h 80 80 80 80 AP Hemolysis, % of predose,
Slide 16: Greater Exposure at 200/400 mg with >98% Sustained Alternative Pathway Suppression
Greater Exposure at 200/400 mg with >98% Sustained Alternative Pathway Suppression in Both Assays BCX9930 PK & PD Profile at Steady State BCX9930 PD Profile: Day 1 and Steady State PK Troughs AP Hemolysis AP Wieslab 50 mg Q12hr - PK 100 100 100 AP Hemolysis, % of predose, mean (SEM) 100 mg Q12hr - P
Slide 17: Successful BCX9930 SAD/MAD Supports Monotherapy for Diseases of the Alternative Pathway
Successful BCX9930 SAD/MAD Supports Monotherapy for Diseases of the Alternative Pathway Safety & Tolerability: Healthy Subjects PK/PD in Healthy Subjects • Study drug was safe and generally well- • Linear, dose-proportional exposure tolerated at all doses • Dose-related suppression of AP of compleme
Slide 18: Galidesivir Clinical Trial in COVID-19 Enrolling at Sites in Brazil
Galidesivir Clinical Trial in COVID-19 Enrolling at 4 Sites in Brazil Part 1 (n=24) Cohort 1 GVR n=6 , PBO n=2 10 mg/kg then 2 mg/kg q12h×13 Key Outcome Measures Cohort 2 GVR n=6 , PBO n=2 10 mg/kg then 5 mg/kg q12h×13 • Safety Cohort 3 • PK GVR n=6 , PBO n=2 20 mg/kg then 5 mg/kg q12h×13 • Viral Lo
Slide 19: Financial Update
Financial Update: Anthony Doyle– Chief Financial Officer 19
Slide 20: Cash position & 2020 guidance
Cash position & 2020 guidance (in millions) Cash & investments at December 31, 2019 $138 Cash & investments at March 31, 2020 $115 Cash & investments at June 30, 2020 $192 Senior credit facility $50 REVISED FY 2020 GUIDANCE Net operating cash utilization $150-165 Operating expenses A $180-195 A - Ex
Slide 21: Thank You… Questions and Answers
Thank You… Questions and Answers
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