Biocryst Pharmaceuticals, Inc. · NASDAQ:BCRX
Biocryst Pharmaceuticals — J.P. Morgan Healthcare Conference 2026
23 slides from Biocryst Pharmaceuticals's conference presentation, Jan 12, 2026
Slide 1: BioCryst Pharmaceuticals, Inc. J.P. Morgan Healthcare Conference
BioCryst Pharmaceuticals, Inc. J.P. Morgan Healthcare Conference January 12, 2026 CONFIDENTIAL & PROPRIETARY
Slide 2: Forward-looking statements
Forward-looking statements This presentation contains forward-looking statements, including statements regarding, among other things, preliminary, unaudited results and future results, performance or achievements, expectations regarding pipeline development, including timing of clinical trial result
Slide 3: Non-GAAP Financial Measures
Non-GAAP Financial Measures This presentation includes non-GAAP financial measures that differ from measures calculated in accordance with generally accepted accounting principles in the United States of America (“GAAP”), including financial measures labeled as “non-GAAP.” We believe providing these
Slide 4: What BioCryst accomplished in 2025
What BioCryst accomplished in 2025: ORLADEYO demand growth 2025 was the strongest year since launch for new patient prescriptions Paid rate reached 84% at end of Q1 and 81% at YE; approaching long- Paid rate improvement term goal of 85%1 Sale of EU ORLADEYO business at compelling valuation; European
Slide 5: Strong Q4 and 2026 outlook
Strong Q4 and 2026 outlook Commercial Highlights PRELIMINARY RESULTS (in millions) Q4 2025 FY 2025 ORLADEYO revenue $151 $601 • 2025 was the strongest year since launch for new patient prescriptions ORLADEYO revenue, excluding EU1 $563 Cash, cash equivalents, restricted cash & investments at YE $338
Slide 6: BioCryst: delivering sustainable growth in rare disease
BioCryst: delivering sustainable growth in rare disease Value creation through three key strategic growth pillars Commercial Product Internal R&D External Opportunities Steady growth with Maximizing potential of Via strategic business high cash flow visibility rare disease pipeline development • Sus
Slide 7: ORLADEYO: the first and only approved targeted oral for HAE prophylaxis
ORLADEYO: the first and only approved targeted oral for HAE prophylaxis • ORLADEYO (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks >1,600 of HAE in patients ages 2+ patients on therapy2 • Discovered in BioCryst labs • Approved in US Dec 2020; now in 6th y
Slide 8: ORLADEYO offers proven long-term attack control, strong real-world efficacy data
ORLADEYO offers proven long-term attack control, demonstrated by strong real-world efficacy data Great efficacy + Strong retention over time differentiated convenience (% of patients persistent at various time points)3 3 86% 2.5 Leads to 76% HAE monthly attack rate 2 61% 1.5 2.8 1 0.5 1.1 1 1 0.9 0.
Slide 9: ORLADEYO: highly achievable path to peak
ORLADEYO: highly achievable path to peak Key drivers to $1B peak $1B Peak • ~150 net patient adds per year (adult + pediatric) • Paid rate improvement toward $625-645M 85% at YE 2029 vs. 81% at YE $563M 20252 • Modest annual price increases • Contribution from ex-US geographies 2025¹ 2026 2029 1. Pr
Slide 10: BioCryst to acquire Astria for ~$700M TEV
BioCryst to acquire Astria for ~$700M TEV Addition of navenibart to expand and strengthen presence in HAE while transforming growth profile Strong strategic fit Compelling LTP asset Enhances financial profile 10+ yr double digit portfolio CAGR Differentiated injectable profile Profitability ma
Slide 11: Significant addressable opportunity in HAE
Significant addressable opportunity in HAE Astria deal to augment HAE portfolio with the potential best-in-class option for any route of administration preference Estimated US patients on prophylaxis 9,700 9,100 8,400 7,600 Other Navenibart opportunity: 5,000+ patients Injectable prophy ORLADEYO ORL
Slide 12: BioCryst's commercial footprint primed for next product
BioCryst’s commercial footprint primed for next product Commercial strategy for ORLADEYO is a repeatable playbook for additional products Patients Physicians & HAEA & KOLs Robust market understanding Experienced rare disease sales team Strong stakeholder relationships Differentiated Proven patie
Slide 13: Navenibart could become the 1st choice injectable therapy
Navenibart could become the 1st choice injectable therapy Trusted mechanism & modality 100% Navenibart Monoclonal antibody inhibitor of plasma (Q3M) Navenibart kallikrein 90% (Q6M) Mean Attack Rate Reduction Compelling efficacy data 80% Phase 1b/2 data highlight navenibart’s potential to match H
Slide 14: Positioned to offer leading oral and injectable therapies
Positioned to offer leading oral and injectable therapies Combined portfolio to reach distinct, durable, and growing segments, covering full spectrum of patient preference HAE patients receiving prophylaxis primarily fall into two categories: Navenibart Oral preference Prefer an Injectable preferenc
Slide 15: BioCryst HAE franchise expected to generate double digit revenue growth
BioCryst HAE franchise expected to generate double digit revenue growth into the next decade HAE portfolio revenue1 Illustrative operating expenses2 $1.8B+ ~15% $1B+ ~15% $1B+ CAGR CAGR cash balance $563M by 2029 2025E ex-EU³ 2029E 2033E Early 2027 - navenibart pivotal topline results expected Stron
Slide 16: Our pipeline
Our pipeline ASSET PROGRAM PRE-CLINICAL PHASE 1 PHASE 2 PHASE 3/PIVOTAL APPROVED / COMMERCIAL CORE PROGRAMS ORLADEYO® (berotralstat) Hereditary Angioedema Oral Plasma Kallikrein (HAE) Inhibitor ORLADEYO® (berotralstat) Hereditary Angioedema Oral Plasma Kallikrein (HAE) Inhibitor in Pediatrics BCX177
Slide 17: BCX17725: a targeted KLK5 inhibitor for Netherton syndrome
BCX17725: a targeted KLK5 inhibitor for Netherton syndrome (NS) What is Netherton syndrome? Why Netherton syndrome? • A severe, rare, genetic disorder with widespread skin involvement and systemic complications Aligned with core rare disease focus • High unmet need • Causes premature separation of s
Slide 18: Targeted therapies drive diagnosis of rare diseases
Targeted therapies drive diagnosis of rare diseases Change in US Prevalence After Netherton Syndrome Launch of Targeted Therapy Population Assumptions 25 +67% 20 Initial healthcare claims analysis indicates a Est. Prevalence (000’s) +70% diagnosed US prevalence of ~1,600 15 +214% 10 +67% +200% 5 Ong
Slide 19: 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 PAR2 KLK5 overactivity at the top of the pathway Downs
Slide 20: BCX17725 in healthy volunteers: distribution to epidermis after IV dosing
BCX17725 in healthy volunteers: encouraging distribution to epidermis after IV dosing Pre-dose Post-dose: BCX17725 12 mg/kg IV 80 µm dermis dermis epidermis epidermis Notes: Immunofluorescence (IF) using capture antibody specific to BCX17725 and fluor-tagged (green) detection antibody. Cell nuclei –
Slide 21: BCX17725 Phase 1 study design
BCX17725 Phase 1 study design Parts 1 & 2: SAD Completed Healthy volunteers MAD Completed This study is designed to help us understand: 1. Preliminary safety Part 3: NS patients Multiple Dose 2. Systemic exposure N = 1-3 3. Distribution into skin 4 weeks of treatment, open-label, safety and PK antic
Slide 22: Key upcoming milestones
Key upcoming milestones January 2026 Acquisition of Astria Therapeutics: expected close YE 2026 BCX17725 in NS: Proof of concept results Early 2027 Navenibart in HAE: Pivotal topline results 22
Slide 23: BioCryst Pharmaceuticals, Inc.
BioCryst Pharmaceuticals, Inc. J.P. Morgan Healthcare Conference January 12, 2026 CONFIDENTIAL & PROPRIETARY
BioCryst Pharmaceuticals — J.P. Morgan Healthcare Conference 2026 on 6ix