Biocryst Pharmaceuticals, Inc. · NASDAQ:BCRX
Biocryst Pharmaceuticals — H.C. Wainwright Global Investor Conference 2020
15 slides from Biocryst Pharmaceuticals's conference presentation, Sep 14, 2020
Slide 1: H.C. Wainwright Global Investor Conference
H.C. Wainwright Global Investor Conference Jon Stonehouse Dr. Bill Sheridan Chief Executive Officer Chief Medical Officer September 14, 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 200/400mg 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: 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 6: 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 7: Goal for BCX9930: Oral Factor D Inhibitor as Monotherapy for Multiple Complement-mediated Diseases
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 8: 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 9: Factor D Inhibition in PNH Offers the Advantage of Controlling Both IVH and EVH
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 10: Goal of Ongoing Dose-ranging Study: Choose Optimized Dose for Advanced Development as Monotherapy
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 11: Dose-dependent Improvement Across Key Indicators in Treatment-naïve PNH Subjects Receiving BCX9930 Monotherapy
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: 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 14: 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 15: H.C. Wainwright Global Investor Conference
H.C. Wainwright Global Investor Conference Jon Stonehouse Dr. Bill Sheridan Chief Executive Officer Chief Medical Officer September 14, 2020
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