Biocryst Pharmaceuticals, Inc. · NASDAQ:BCRX
Biocryst Pharmaceuticals — Q2 2025 Earnings Presentation
23 slides from Biocryst Pharmaceuticals's earnings presentation, Aug 4, 2025
Slide 1: Second Quarter 2025 Results Call
Second Quarter 2025 Results Call Corporate Update & Financial Results August 4, 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 Jon Stonehouse President and Chief Executive Officer Financial update Babar Ghias Chief Financial 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 $1B peak annual sales Financially Clinical Pipeline Independent with near-term milestones with near-term path to profitability 4
Slide 5: Strong Q2 led by jump in ORLADEYO demand
Strong Q2 led by jump in ORLADEYO demand ORLADEYO REVENUE QUARTERLY & QUARTERLY AVERAGE BY YEAR ($M) • Best quarter ever for new patient prescriptions in 160 the US 140 120 • Revenue growth exceeded expectations due to: • Robust underlying demand (US and 100 international) 80 • Efficiency in getting
Slide 6: Market research: patient preference for an 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 7: 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 8: 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 9: 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 10: Bringing ORLADEYO granules to children
Bringing ORLADEYO granules 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 children
Slide 11: 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 12: 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 13: 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 14: 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 15: Preclinical evidence demonstrates 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 16: 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 17: 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 18: 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 19: 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 and sustainable cash flow 19
Slide 20: Finance summary
Finance summary (Figures in millions) PRO FORMA3 CASH POSITION JUN 30, 2025 JUN 30, 2025 Cash, cash equivalents, restricted cash & investments1 $287 $235 Senior credit facility $249 $199 6ME ORLADEYO ROYALTIES Q2 2025 JUN 30, 2025 Royalty revenues paid and payable $31 $57 Non-cash interest expense $
Slide 21: ORLADEYO royalties: terms
ORLADEYO royalties: terms Cumulative Upfront Rate Tiers Rate Tiers Payback (Direct2) (Indirect) Cap $0-350M: 8.75% $0-150M: 20% RP $125M $350M-550M: 2.75% $150M-230M: 10% None 2020 Over $550M: None Over $230M: None $0-350M: 0.75% $0-150M: 3% RP $150M1 $350M-550M: 1.75% $150M-230M: 2% None 2021 Over
Slide 22: ORLADEYO royalty rate now declining
ORLADEYO royalty rate now declining (Figures in millions) RPI rate OMERS rate¹ Expected rate Expected rate at peak 20% 17% 14% • We expect total royalties to be steady and blended rate to decline through peak, now that revenue has reached 4% royalty free tier (>$550M) • Rate expected to settle ~4% a
Slide 23: Second Quarter 2025 Results Call
Second Quarter 2025 Results Call Corporate Update & Financial Results August 4, 2025 CONFIDENTIAL & PROPRIETARY
BioCryst Pharmaceuticals — Q2 2025 Earnings Presentation on 6ix