BioCryst Pharmaceuticals, Inc. · NASDAQ:BCRX
BioCryst Pharmaceuticals — Investor Presentation (September 2020)
41 slides from BioCryst Pharmaceuticals's investor presentation, September 2020
Slide 1: September 2020 Corporate Presentation
September 2020 Corporate Presentation
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 200/400mg galidesivir COVID-19 trial approval in U.S. on ORLADEYO BCX9930 200/400mg BCX9250 trial in FOP t
Slide 4: Coming Soon: Orladeyo
Coming Soon: Orladeyo™ Diggeryst COMING SOON Orladeyo: | ‘vnc. (berotralstat) 150 mg capsule (berotralstat) 150 mg capsule
Slide 5: Rapid and Sustained HAE Attack Rate Reduction: APeX-2 Analyses
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 6: 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 6
Slide 7: 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 8: 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 9: Robust Market Research Since APeX-2
Robust Market Research Since APeX-2 Market Sizing US HAE Patients US Physicians US Payors • US prevalence • 100 quantitative, • 175 quantitative, • 16 interviews study using 25-minute online 20-minute online with medical and administrative surveys surveys pharmacy claims data directors from • 26 ind
Slide 10: Administrative Claims Analysis Estimates US HAE Population
Administrative Claims Analysis Estimates US HAE Population at ~10,000 Patients with ~7,500 Diagnosed & Treated 1.Diagnosed and treated with HAE-specific medication 2.Diagnosed but not treated with HAE-specific medication HAE Patient 3.Treated with HAE-specific medication but not diagnosed cohorts Da
Slide 11: Large, Quantitative Market Research Studies with US Patients and Physicians
Large, Quantitative Market Research Studies with US Patients and HAE-treating Physicians in July 2019 with 24-week APeX-2 Profile 100 HAE Patients 175 HAE-Treating Physicians • 25-minute online survey • 20-minute online survey • Age 18+, diagnosed with • Allergist/Immunologist Type I or II HAE (n=10
Slide 12: Respondents Viewed a Blinded Profile of BCX7353
Respondents Viewed a Blinded Profile of BCX7353 Based on 24-week Results from APeX-2 Indication Prophylactic treatment of HAE for patients 12 years and above Dosage Take 1 capsule by mouth once per day Clinical trial Patients who were experiencing an average of 3 HAE attacks per month took Treatment
Slide 13: 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 14: Prophylaxis Patients Very Willing to Use BCX7353
Prophylaxis Patients are Very Willing to Use BCX7353—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
Slide 15: 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 16: 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 17: 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 18: 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 19: US Payors Anticipate Providing Coverage for Berotralstat
US Payors Anticipate Providing Coverage for Berotralstat 80M Positive reaction to therapeutic value of profile Medical & Pharmacy covered Directors lives Accept that treatment approach is individualized choice made by patients and HCPs Willing to cover new therapies in HAE 110M PBMs covered Broad wi
Slide 20: Berotralstat Data Supports 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 21: Ready for Successful Commercial Launch
Ready for Successful Commercial Launch Sales force hired + trained • Average 20 years of experience, including 8 in rare disease • Top performers Medical Affairs team deployed and engaging with the KOL community Patient access/support systems in place Robust dual-source supply chain to support comme
Slide 22: Multiple Potential Global Approvals in 2020-2021
Multiple Potential Global Approvals in 2020-2021 Orphan Drug 2017 Sakigake 2015 Orphan Drug 2018 NDA accepted JNDA accepted MAA accepted Mar 2020 PDUFA Date: 12/3/20 Approval Dec 2020 CHMP Opinion:~12 mo 2020 2020 2021 22
Slide 23: APeX-J – Primary Efficacy Endpoint Met for Berotralstat 150 mg
APeX-J – Primary Efficacy Endpoint was Met for Berotralstat 150 mg Total Enrollment: 19 (7 at 150 mg, 6 at 110mg, 6 placebo) Primary endpoint: expert-confirmed angioedema attacks, rate/month* Screening / Run-in Attack rate ratio Percent reduction from Part 1 24 WeekTreatment Arm N Rate P value 8 Wee
Slide 24: Consistent, Sustained Reduction in Attacks Over 24 Weeks
Berotralstat 150 mg Shows Consistent, Sustained Reduction in Attacks Over 24 Weeks APeX-J Attack Rate by Month, mean (SEM) APeX-J Change from Baseline in Attack Rate by Month, mean (SEM) berotralstat 150 mg and placebo berotralstat 150 mg and placebo 4 1 berotralstat 150 mg berotralstat 150 mg CFB i
Slide 25: Overall Safety Summary: Berotralstat Safe and Generally Well Tolerated
Overall Safety Summary: Berotralstat was Safe and Generally Well Tolerated Treatment-emergent (TE) Adverse Events (AEs) or Berotralstat 110 mg Berotralstat 150 mg Placebo Discontinuations (DCs) due to TEAEs N=6 N=7 N=6 Any Drug-Related TEAEs 2 (33.3%) 2 (28.6%) 2 (33.3%) Drug-Related Serious TEAEs 0
Slide 26: Unique Market Opportunity in Japan
Unique Market Opportunity in Japan Japanese Market Growth Potential 3000 • Berotralstat would be 1st approved prophylactic HAE 2500 therapy in Japan 2000 • Active KOL base of treating physicians with strong interest 1500 Japan Current Patients in new therapies for patients 1000 Japan Prevalence 500
Slide 27: Japanese Partnership with Torii
Berotralstat for HAE Prophylaxis: Japanese Partnership with Torii Non-dilutive Capital + Access to Unique Market with Large Unmet Need • $42 million in upfront and milestones • $22 million upfront • Up to $20 million with approval + threshold pricing • Royalties from mid-teens up to potentially 40%
Slide 28: Goal for BCX9930: Oral Factor D Inhibitor as Monotherapy
Goal for BCX9930: Oral Factor D Inhibitor as Monotherapy for Multiple 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 se
Slide 29: Significant Unmet Need in PNH Due to Uncontrolled Hemolysis
Significant Unmet Need in PNH Due to Uncontrolled Hemolysis Anemia and PNH Symptoms Persist in Many Patients Despite C5 Inhibitor Treatment C5 Inhibitor Hb not Stabilized Fatigue Hemoglobinuria Dyspnea Ravulizumab 32% 29% 10% 14% Eculizumab 35% 30% 9% 14% J. W. Lee et al., Blood 133, 530-539 (2019)
Slide 30: Factor D Inhibition Controls Both Intravascular and Extravascular Hemolysis
Factor D Inhibition in PNH Offers the Advantage of Controlling Both Intravascular (IVH) and Extravascular (EVH) Hemolysis Goal of Total Patient Outcomes from Controlled Hemolytic Control Hemolysis over Time Relieve Anemia IVH Control Hemoglobin LDH Reduce PNH Clinical Symptoms Appearance of symptoms
Slide 31: Goal of Ongoing Dose-ranging Study
Goal of Ongoing Dose-ranging Study: Choose Optimized Dose for Advanced Development as Monotherapy Key Outcome Measures • LDH, hemoglobin • Safety • PK Total of 28 days of BCX9930 dosing • PD Period 1 days 1-14 Period 2 days 15-28 Subjects with PNH who are naïve to C5-INH treatments: BCX9930 monother
Slide 32: 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 33: Dose-dependent Improvement Across Key Indicators in 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 34: 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 35: 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 36: 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 37: 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 38: 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: 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 39: COVID-19 Antiviral Status Update: Galidesivir (BCX4430)
COVID-19 Antiviral Status Update: Galidesivir (BCX4430) $ 129 M of program support to date: New $47M of NIAID Contract Awards to Support: • Completion of Parts 1 & 2 of ongoing clinical trial of galidesivir in Brazil • Conducting a phase 2 trial of galidesivir in non-hospitalized COVID-19 patients a
Slide 40: 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 41: September 2020 Corporate Presentation
September 2020 Corporate Presentation
BioCryst Pharmaceuticals — Investor Presentation (September 2020) on 6ix