Biocryst Pharmaceuticals, Inc. · NASDAQ:BCRX
Biocryst Pharmaceuticals — Q1 2025 Earnings Presentation
24 slides from Biocryst Pharmaceuticals's earnings presentation, May 5, 2025
Slide 1: First Quarter 2025 Results Call
First Quarter 2025 Results Call Corporate Update & Financial Results May 5, 2025 CONFIDENTIAL & PROPRIETARY
Slide 2: Forward-looking statements
Forward-looking statements BioCryst’s presentation contains forward-looking statements, including, but not limited to, statements regarding future results and forward-looking financial information, company performance, achievements, future market share or size, and expectations regarding pipeline de
Slide 3: Agenda
AGENDA Jon Stonehouse Corporate update President and Chief Executive Officer Charlie Gayer ORLADEYO® update Chief Commercial Officer Dr. Helen Thackray Pipeline update Chief Research and Development Officer Jon Stonehouse Financial update President and Chief Executive Officer Q&A 3
Slide 4: BioCryst: durable, profitable growth through the decade with pipeline optionality
BioCryst: durable, profitable growth through the decade with pipeline optionality Marketed Product growing to $1B peak annual sales Financially Clinical Pipeline Independent with near-term milestones with near-term path to profitability 4
Slide 5: Excellent Q1 ORLADEYO result sets up FY 2025 for record year
Excellent Q1 ORLADEYO result sets up FY 2025 for record year ORLADEYO REVENUE QUARTERLY & QUARTERLY AVERAGE BY YEAR ($M) 160 140 • Better-than-expected commercial execution in Q1 120 • ~84% paid rate across all patients 100 • More revenue captured during reauthorizations • Revenue will flow through
Slide 6: ORLADEYO growth + focused capital allocation to drive sustained profitability
ORLADEYO growth + focused capital allocation to drive sustained profitability $1B peak 1,000 800 By 2027: >$300M Operating Profit1 600 >$550M Cash2 400 200 0 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030 ORLADEYO revenue Non-GAAP operating expense¹ 1. Not including stock-based compensa
Slide 7: Market research: patient preference for oral route of administration for HAE LTP
MARKET RESEARCH: Patient preference for an oral route of administration for HAE LTP has increased steadily year over year since 2023 Prophy Medication Route of Admin Preferences (n=421) 100% 80% 51% 60% 70% 60% 40% 30% 22% 13% 20% 18% 18% 17% 0% 2023 Survey 2024 Survey 2025 Survey (n=181) (n=180) (n
Slide 8: Comprehensive annual research + market simulation
Comprehensive annual research + market simulation OUR MODEL STARTS WITH PREFERENCE AND SIMULATES 6,000 MARKET INTERACTIONS BETWEEN HCPS, PATIENTS, & PAYERS Research Sample* Market Model Simulation (Monte Carlo) 1.1 A patient, physician, and payer are randomly selected from survey respondents. 2 The
Slide 9: Monte Carlo simulation outcome: U.S. prophy market share
Monte Carlo simulation outcome: U.S. prophy market share ORLADEYO REACHES A STEADY STATE OF OVER 2,000 PATIENTS IN U.S. DURING 2028, EVEN AS NEW PRODUCTS GAIN SHARE 100% 80% Prophylaxis Patient Share 60% 40% 20% 21% 22% 24% 25% 24% 24% 24% 23% 23% 20% 0% 2024 2025 2026 2027 2028 2029 2030 2031 2032
Slide 10: Our pipeline
Our pipeline ASSET PROGRAM LEAD PRE- PROOF OF PIVOTAL‡ APPROVED / OPTIMIZATION CLINICAL CONCEPT† COMMERCIAL ORLADEYO® (berotralstat) Hereditary Oral Plasma Kallikrein Angioedema Inhibitor (HAE) ORLADEYO® (berotralstat) Hereditary Oral Plasma Kallikrein Angioedema Inhibitor (age ≥2 years) (HAE) BCX17
Slide 11: This is BIG: bringing ORLADEYO to children
This is BIG: bringing ORLADEYO to children • Despite significant innovation in HAE prophylaxis for adults, there is still high unmet need in children • Injectable therapies are the only FDA-approved options for children ages 2 to 11 • Positions ORLADEYO to be the market leading prophylaxis for child
Slide 12: Treating Netherton syndrome with a targeted KLK5 inhibitor: BCX17725
Treating Netherton syndrome (NS) with a targeted KLK5 inhibitor: BCX17725 Severe, rare, genetic and lifelong disease High Premature separation of skin layers, unmet severe inflammation and infection risk need No approved targeted therapies Well-understood biological cause: Validated mutation in SPIN
Slide 13: BCX17725 targets KLK5, the key player in Netherton syndrome
BCX17725 targets KLK5, the key player in Netherton syndrome BCX17725 KLK5 • KLK5 initiates the pathologic Degrades protease cascade (KLK7, KLK14 KLK7 epithelial KLK14) and inflammation junctions (via PAR2) in the skin • BCX17725 designed to stop KLK5 overactivity at the top PAR2 of the pathway Downs
Slide 14: BCX17725 Phase 1: healthy volunteers and patients with Netherton syndrome
BCX17725 Phase 1: healthy volunteers and patients with Netherton syndrome Parts 1 & 2: Single Ascending Dose Healthy volunteers Multiple Ascending Dose Potential early signals from measures of: Part 3: Multiple Dose • IGA NS patients • KLK5 activity in skin 4 weeks of treatment, open-label, safety a
Slide 15: Addressing DME with a potent plasma kallikrein inhibitor: avoralstat
Addressing DME with a potent plasma kallikrein inhibitor: avoralstat suprachoroidal injection1 Alternative Right drug Significant MOA + delivery market opportunity Compelling evidence for plasma High potency and low kallikrein activity in DME solubility/depot effect are attractive pathobiology ~1.5M
Slide 16: Preclinical evidence: plasma kallikrein pathway may reduce vascular leakage
Preclinical evidence demonstrates plasma kallikrein pathway may reduce vascular leakage Pre-Dosing Avoralstat (7 Days post dose) Pre-Dosing Avoralstat (6 Days post dose) Vascular leakage Vascular leakage Vascular Stability Vascular stability Source: BioCryst Pharmaceuticals nonclinical data on file
Slide 17: Suprachoroidal injection leads to 3+ months of sustained avoralstat levels in the retina
Suprachoroidal injection leads to 3+ months of sustained avoralstat levels in the retina1 1. BioCryst Pharmaceuticals nonclinical data on file 2025 This slide shows preclinical results, and the outcome of preclinical testing and early clinical trials may not be predictive of the success of later cli
Slide 18: Avoralstat Phase 1: patients with DME
Avoralstat Phase 1: patients with DME • Single dose Low dose cohort • 24-week follow-up • Data collected every 4 weeks • Safety Mid dose cohort • Central subfield thickness • BCVA • Enrollment: • Newly diagnosed patients High dose cohort • Patients previously treated with anti-VEGF BCVA: best correc
Slide 19: Path to profitability significantly accelerated
Path to profitability significantly accelerated 2025 2024 2026+ 2026 Full year Meaningful & operating profit1 Achieve positive net income & cash flows 2 sustainable cash generation 1. Not including stock-based compensation 2. This positive cash flow is the improvement in cash, cash equivalents, rest
Slide 20: Key milestones in 2025
Key milestones in 2025 ORLADEYO revenue advances to royalty-free tier (>$550M) Pediatric NDA for ORLADEYO Initial BCX17725 and avoralstat clinical data Accelerated full-year profitability 20
Slide 21: Finance summary
Finance summary (Figures in millions) Q1 2025 CASH POSITION Cash, cash equivalents, restricted cash & investments at December 31, 2024 $343 Cash, cash equivalents, restricted cash & investments at March 31, 2025 $317 Senior credit facility1 $324 PRO FORMA Q1 2025 CASH POSITION (ADJUSTED FOR DEBT PAY
Slide 22: Traditional debt and royalty breakdown (Q1'25)
Traditional debt and royalty breakdown (Q1'25) Does not include subsequent paydown of $75 million March 31, 2025 December 31, 2024 Royalty financing obligations - current 34,305 32,676 Royalty financing obligations - long-term 466,613 481,053 Total royalty financing obligations 500,918 513,729 Secur
Slide 23: Royalty obligations: terms
Royalty obligations: terms Rate Tiers Rate Tiers Cumulative Upfront Product (Key Territories2) (Other Markets2) Payback Cap $0-350M: 8.75% $0-150M: 20% RP $125M ORLADEYO $350M-550M: 2.75% $150M-230M: 10% None 2020 Over $550M: None Over $230M: None $0-350M: 0.75% $0-150M: 3% RP $150M1 ORLADEYO $350M-
Slide 24: First Quarter 2025 Results Call
First Quarter 2025 Results Call Corporate Update & Financial Results May 5, 2025 CONFIDENTIAL & PROPRIETARY
BioCryst Pharmaceuticals — Q1 2025 Earnings Presentation on 6ix