Biocryst Pharmaceuticals, Inc. · NASDAQ:BCRX
Biocryst Pharmaceuticals — Q1 2020 Earnings Presentation
31 slides from Biocryst Pharmaceuticals's earnings presentation, May 6, 2020
Slide 1: First Quarter 2020 Results Call
First Quarter 2020 Results Call Corporate Update & Financial Results May 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 ◆ Clinical Update Dr. Bill Sheridan – Chief Medical Officer ◆ Financial Update Anthony Doyle – Chief
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: Strong HAE Patient Demand for BCX7353
Strong HAE Patient Demand for BCX7353: 59% of Patients Expressed High Willingness to use BCX7353 Rises to 71% with Physician Recommendation Patient Willingness to Patient Willingness to Use BCX7353 Use BCX7353 (N=100) with MD Recommendation (N=100) 71% 59% 21% 22% 14% 5% 3% 5% Unsure Not Willing Som
Slide 6: Switch Opportunity—Even Those Very Satisfied with Current Injectable Treatment
Switch Opportunity—Even Those Very Satisfied with Their Current Injectable Prophylactic Treatment Prophylaxis Patients VERY WILLING to Use BCX7353 % of those Very Satisfied on current 63% 60% therapy % of Total 50% 50% 48% 47% 15 of 24 15 of 25 5 of 10 8 of 16 14 of 30 13 of 27 Cinryze (N=24) Haegar
Slide 7: Patients Report Breakthrough Attacks with Injectable/Infused Treatments
Patients Report Breakthrough Attacks with Injectable/Infused Treatments Mean Patient-Reported Attacks in Past 3 Months by Prophylaxis Medication 1.8 1.6 0.9 Cinryze (n=24) Haegarda (n=25) Takhzyro (n=30) Currently Taking Medication Prophylactically Source: BioCryst Proprietary Market Research Study,
Slide 8: Physicians Expect to Prescribe BCX7353 for Over 40% of HAE Patients
Physicians Expect to Prescribe BCX7353 for Over 40% of HAE Patients 80% of HAE Patients Expected to be on Some Form of Prophylaxis Patient Allocation by Medication Today and in Future with BCX7353 Available – Mean % of Patients 100% 4% 5% 4% 8% 6% 80% 25% Not sure No medication 60% 10% 39% Acute onl
Slide 9: Rapid and Sustained HAE Attack Rate Reduction
Rapid and Sustained HAE Attack Rate Reduction: Analyses for APeX-2: 150 mg and Placebo 48-week Completers APeX-2 Attack Rate by Month, Completers Analysis APeX-2 Attack Rate by Month, Completers Analysis BCX7353 150 mg QD Placebo and Placebo-to-Active Attack rate per 4-week month, mean (SEM) Attack
Slide 10: Consistent Mean Attack Rates in APeX-2 and APeX-S
Consistent Mean Attack Rates in APeX-2 and APeX-S APeX-2 and APeX-S Completers Analyses, 150 mg QD BCX7353 4 Mean (SEM) attack rate per 4-week month 3 APeX-2 150 mg QD, n=30 APeX-S 150 mg QD, n=73 2 1 0 BL 1 2 3 4 5 6 7 8 9 10 11 12 Month of study drug 10
Slide 11: Median Attack Rates in 48-week Completers
Median Attack Rates in 48-week Completers: Zero Attacks per Month in 6 of 12 Months in APeX-S APeX-2 Completers Analysis, 150 mg QD BCX7353 APeX-S Completers Analysis, 150 mg QD BCX7353 4 4 Median attack rate per 4-week month Median attack rate per 4-week month 150 mg QD N = 30 150 mg QD N = 73 3 3
Slide 12: Safety and Tolerability Confirmed in Integrated 48-week Analysis
Safety and Tolerability Confirmed in Integrated 48-week Analysis Integrated Safety Summary – APeX-2 and APeX-S BCX7353 110 mg BCX7353 150 mg Placebo Subjects enrolled and dosed [Safety Population] N = 158 N = 184 N = 39 Subject Incidence of SAEs or Discontinuations due to AEs Drug-Related Serious AE
Slide 13: Clinical Trial Experience Consistent with Market Research
Clinical Trial Experience Consistent with Market Research— Patients on Injectable Prophylaxis Switch to Oral Berotralstat Themes In patients’ own words ~50% of future use of berotralstat will come from patients switching from other Physicians’ expectations in prophylaxis treatments market research 4
Slide 14: Insights from Long-term Patients in APeX-2
Insights from Long-term Patients in APeX-2: Why they StayInon Themes Oral, Once-Daily Berotralstat patients’ own words Efficacy “In the past 3 months I may have had to fall back on rescue maybe 3 times, which is fantastic. I’ll take that all day long. Three times in 3 months compared to twice a week
Slide 15: Preparing for a Successful Commercial Launch
Preparing for a Successful Commercial Launch Building out critical launch elements based on our detailed market understanding • Marketing strategy, messages and tactics • Sales force structure and targeting • Market access strategies • Developing a best-in-class patient services and hub program Medi
Slide 16: Berotralstat for HAE Prophylaxis: Global Peak Market Opportunity >$500M
Berotralstat for HAE Prophylaxis: Data Supports Global Peak Market Opportunity >$500M Clinical Data Prevalence Treatment Paradigm Consistent, clinically meaningful benefit ~10,000 (US) demonstrated through HAE Patients Physicians expect 48 weeks shift to ~80% ~7,500 diagnosed prophylaxis Safe and an
Slide 17: Clinical Update
Clinical Update: Dr. Bill Sheridan – Chief Medical Officer 17
Slide 18: 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 19: Targeting Overactive Alternative Pathway Could Treat Many Complement-mediated Diseases
Targeting Overactive Alternative Pathway Could Treat Many Complement-mediated Diseases 19
Slide 20: 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 21: 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 22: 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 23: 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 24: 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 25: 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 26: 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 27: Greater Exposure at 200/400 mg with >98% Sustained AP 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 at 12 hr and Steady State PK Troughs 50 mg Q12hr - PK 100 100 100 AP Hemolysis AP Wieslab AP Hemolysis, % of predose, mean (SEM) 100 mg Q12hr -
Slide 28: 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 29: Galidesivir Clinical Trial in COVID-19 Enrolling Patients
Galidesivir Clinical Trial in COVID-19 Enrolling Patients 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 Load Reduction
Slide 30: Cash position & 2020 guidance
Cash position & 2020 guidance (in millions) Cash & investments at December 31, 2019 $137 Cash & investments at March 31, 2020 $115 Senior Credit Facility $50 FY 2020 GUIDANCE Net operating cash utilization $125 – 150 Operating expenses A $135 – 160 A - Excludes equity-based compensation. 30
Slide 31: Thank You… Questions and Answers
Thank You… Questions and Answers
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