BioCryst Pharmaceuticals, Inc. · NASDAQ:BCRX
BioCryst Pharmaceuticals — Investor Presentation (November 2022)
17 slides from BioCryst Pharmaceuticals's investor presentation, November 2022
Slide 1: BioCryst Corporate Presentation
BioCryst Corporate Presentation November 2022
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: What We Do: Oral Medicines for Rare Disease
What We Do: Oral Medicines for Rare Disease Our Focus Our Approach ◆ Develop novel oral medicines for rare diseases ◆ Design potent, specific, bio-available molecules with high unmet need atom-by-atom ◆ Pursue indications with validated target biology ◆ Combine our biology, medicinal chemistry and c
Slide 4: Our Molecules
Our Molecules Potent Specific Orally Bioavailable Pursue best-in-class efficacy Minimize off-target effects Improve quality of life for patients by eliminating burden of therapy 4
Slide 5: Our Programs
Our Programs EARLY APPROVED / PROGRAM LEAD OPTIMIZATION PRECLINICAL PIVOTAL DEVELOPMENT COMMERCIAL HEREDITARY ANGIOEDEMA (HAE) ORLADEYO® (berotralstat) Oral Plasma Kallikrein Inhibitor COMPLEMENT-MEDIATED DISEASES BCX9930 – Oral Factor D Inhibitor (PNH) BCX9930 – Oral Factor D Inhibitor (C3G/IgAN/PM
Slide 6: Orladeyo: The 1st and Only Oral for HAE Prophylaxis
Orladeyo: The 1st and Only Oral for HAE Prophylaxis ORLADEYO Net Revenue ($m) Key Highlights ◆ Solid launch continues into 2nd year: on track to 300 more than double sales in 2022 250 ◆ Patient desire for oral option remains strong +109% 200 y-o-y ◆ Steady patient growth driven by both prophy switch
Slide 7: Orladeyo Market Research: Steady Growth Expected
Orladeyo Market Research: Steady Growth Expected Previous HAE Medication Current and Future Prescribing of Prophy Medication: Mean % of Patients 100% 90% 18% 17% Takhzyro 21% 80% 4% 2% 70% On-demand/None 60% No Known Prophy 55% Androgens 50% 58% 49% Haegarda 14% 40% Other IV/SC Long- Term Prophy 30%
Slide 8: Going Global: Bringing Orladeyo to Patients Everywhere
Going Global: Bringing Orladeyo to Patients Everywhere US, Canada, and EU BioCryst sales territories Japan Out-licensed to Torii Pharmaceutical Israel Distribution partnership with Neopharm UAE and Saudi Arabia LATAM Distribution partnership with Distribution partnership with NewBridge PINT Pharma 8
Slide 9: ACAAI: Orladeyo Real-World Data Show Sustained Reduction in Attacks
ACAAI: Orladeyo Real-World Data Show Sustained Reduction in Attacks, Regardless of Prior Therapy1 1. Marc A. Riedl et al. Presented at: American College of Allergy Asthma and Immunology Annual Scientific Meeting; November 10-14, 2022. 9
Slide 10: Approved Label: ORLADEYO (berotralstat) Safety
Approved Label: ORLADEYO® (berotralstat) Safety In APeX-2 (part 1), the most commona treatment-emergent adverse reactions were abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease (GERD) a≥10% and higher than placebo. bIncludes abdominal pain, abdominal discomfort, abdo
Slide 11: Launch Trajectory + Opportunity Supports Confidence in $1B Peak ORLADEYO Sales
Launch Trajectory + Opportunity Supports Confidence in $1B Peak ORLADEYO Sales 2,000 OUT OF ~7,500 HAE patients diagnosed Paid patients on Orladeyo and treated in US1 $800m $200m From US at peak Ex-US 25-30% market share in the US gets us to our $1B goal 1. BioCryst HAE claims analysis, as of Nov’20
Slide 12: A Comprehensive Approach to Complement
A Comprehensive Approach to Complement BCX9930 BCX10013 Other Targets ◆ Potential First-in-Class oral ◆ Potential Best-in-Class oral ◆ New discovery targets for oral Factor D inhibitor Factor D inhibitor medicines across the classical, lectin and terminal pathways of ◆ In pivotal trials for PNH ◆ Pr
Slide 13: Key Efficacy Parameters for 400 mg bid and 500 mg bid in Proof of Concept Study
Key Efficacy Parameters for 400 mg bid and 500 mg bid in Proof of Concept Long-Term Extension Study Support Similar Efficacy at Both Dose Levels Parameter 400 mg BID period 500 mg BID period N subjects (treatment naïve) 7 7 Duration of Treatment, days 134 (109) 445 (130) RBC Transfusions, (n) 0 0 La
Slide 14: REDEEM 1: Pivotal Trial of BCX9930 Oral Monotherapy for PNH
REDEEM 1: Pivotal Trial of BCX9930 Oral Monotherapy for Treatment of PNH in Patients with Inadequate Response to C5-Inhibitor Therapy SCREENING PART 1 PART 2 4-week run-in Randomized open-label treatment | Weeks 1-24 Open-label treatment extension | Weeks 24-52 BCX9930 BCX9930 BCX9930 Randomization:
Slide 15: REDEEM 2: Pivotal Trial of BCX9930 Oral Monotherapy for PNH
REDEEM 2: Pivotal Trial of BCX9930 Oral Monotherapy for Treatment of PNH in Patients Not Currently Receiving C5-Inhibitor Therapy SCREENING PART 1 PART 2 4-week run-in Randomized blinded treatment | Weeks 1-12 Open-label treatment | Weeks 12-52 BCX9930 BCX9930 BCX9930 200 mg BID 400 mg BID 400 mg BI
Slide 16: RENEW: Open-label Proof-of-concept Study of BCX9930
RENEW: Open-label, Safety, Tolerability, and Proof-of-concept Study of BCX9930 Oral Monotherapy in Patients with CG3, IgAN, or PMN 8-week run-in Weeks 1-2 Weeks 2-52* Weeks 52-56 BCX9930 BCX9930 Safety Screening 200 mg BID 400 mg BID follow-up 2 weeks 50 weeks Clinical benefit assessment at Week 24
Slide 17: Finance Summary
Finance Summary (figures in millions) 3Q22 Cash Position Cash, cash equivalents, restricted cash & investments at September 30, 2021 $204 Cash, cash equivalents, restricted cash & investments at September 30, 2022 $463 Senior Credit Facility1 $232 2022 FY Guidance ORLADEYO Revenue $255 Operating Exp
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