BioCryst Pharmaceuticals : Q1 2026 Earnings Presentation

Published: 2026-05-06 07:25:21 ET BCRX

Published on 05/06/2026 at 07:25 am EDT

First Quarter 2026 Results Call

CONFIDENTIAL & PROPRIETARY

C or p or a te U p d a te & F i n a nc i a l Re s u l ts May 6, 2026

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 non-GAAP measures, which show our results with certain items adjusted, is valuable and useful since they can provide greater transparency into the financial results of core, ongoing operations and improve comparability across reporting periods. These non-GAAP measures also correspond with the way we expect investors and financial analysts to compare our results. Our non-GAAP measures should be considered only as supplements to, and not as substitutes for or in isolation from, our other measures of financial information prepared in accordance with GAAP, such as GAAP revenue or operating income.

Our references to non-GAAP operating profit and non-GAAP ORLADEYO revenue constitute non-GAAP financial measures. These non-GAAP financial measures are calculated using our GAAP results, adjusted to show the results without including, as applicable, revenues and expenses associated with our European ORLADEYO business, stock-based compensation, and expenses incurred in connection with our acquisition of Astria Therapeutics, Inc. A reconciliation between each first quarter non-GAAP financial measure and its respective closest equivalent GAAP financial measure is provided in the tables in the appendix. In addition, we provide non-GAAP ORLADEYO revenue for the full year 2025. This refers to our GAAP ORLADEYO revenue of

$602M for the full year 2025, adjusted to exclude $39M in revenue associated with our European ORLADEYO business.

We also provide our non-GAAP operating expense outlook for full year 2026, which refers to our expected GAAP operating expense, excluding stock-based compensation, restructuring and transaction-related costs. We have not provided a reconciliation against the comparable forward-looking GAAP measure because we are unable to predict with reasonable certainty the full amount of stock-based compensation expense or restructuring or transaction-related costs for the full year 2026 without unreasonable effort. Stock-based compensation expense is uncertain and depends on various factors, including our future hiring and retention needs, as well as the future fair market value of our common stock, which is difficult to predict and subject to change. In addition, we are unable to predict with reasonable certainty the full amount of restructuring and transaction-related costs as the related costs are dependent on various factors that have not yet or have only recently occurred. The actual amount of stock-based compensation, restructuring and transaction-related costs for the full year 2026 could have a material impact on GAAP reported results for the guidance period.

Charlie Gayer

President and Chief Executive Officer

Dr. Sandeep Menon

Chief Research and Development Officer

Babar Ghias

Chief Financial Officer

AGENDA

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 high cash flow visibility

Sustainable $1B peak revenue opportunity for ORLADEYO®

>80% contribution margin1

IP runway into 20402

Maximizing potential of rare disease pipeline

Netherton syndrome: high unmet need and potential for best-in-class therapy

Targeted rare disease-focused discovery

Externalizing non-core assets

Via strategic business development

Focus on de-risked late-stage rare disease assets

Near-term value creation

Leveraging existing operating infrastructure

Contribution margin defined as revenue minus direct costs (COGS + S&M)

Our pipeline

A SSE T P RO G RAM P RE - C L IN IC A L P H A SE 1 P H A SE 2 P H AS E 3 / P I V O T AL AP P RO V E D / C O MMER C I A L

CORE PROGRAMS

ORLADEYO® (berotralstat) Hereditary

Oral Plasma Kallikrein Inhibitor Angioedema (HAE)

ORLADEYO® (berotralstat) Hereditary

Oral Plasma Kallikrein Inhibitor in Pediatrics Angioedema (HAE)

Navenibart Hereditary

Monoclonal Antibody Plasma Kallikrein Inhibitor Angioedema (HAE)

BCX17725 Netherton Syndrome

Protein Therapeutic

Undisclosed Rare Diseases

NON-CORE PROGRAMS

RAPIVAB® Infectious Diseases

(peramivir injection)

STAR-0310 Atopic Dermatitis

Monoclonal Antibody OX40 Antagonist

ORLADEYO: the first and only approved targeted oral for HAE prophylaxis

ORLADEYO (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of HAE in patients ages 2+

Discovered in BioCryst labs

Approved in US Dec 2020; now in 6th year of launch

Pediatric formulation (pellets) approved Dec 12, 2025

IP through May 20401

At year-end 2025:

>1,600

patients on therapy2

>3,500

patients have tried since launch

>1,500

unique prescribers

(figures reflect ORLADEYO metrics in US market)

Pediatric extension through May 2040; composition of matter patent

All patients including Quick Start, paid, and patient assistance program (PAP) 7

ORLADEYO offers proven long-term attack control, demonstrated by strong real-world efficacy data

Great efficacy + differentiated convenience Strong retention over time 3

2.8

0.3

1.1

0.6

0.7

0.9

HAE monthly attack rate

2.5

1.5

0.5

Leads to

(% of patients persistent at various time points)

76%

61%

86%

BL¹ 1 3 6 9 12 15 18 21 24

Months of treatment with ORLADEYO²

3 months 6 months 12 months

ORLADEYO efficacy is consistent with ~80-90% reduction in attack rate provided by injectable therapies

BL: baseline

Data from APeX-2 open label extension study; 150mg, n=21 completers; Kiani-Alikhan S, et al. J Allergy Clin Immunol Pract. 2024;12(3):733-743.e10.

Zuraw BL, et al. Allergy Asthma Proc. 2025 May 1;46(3):209-217. Persistence is defined as having no gap in treatment ≥45 days after the treatment initiation

ORLADEYO: highly achievable path to $1B peak revenue

$1B Peak

$563M

$625-645M

Key drivers to peak

~150 net patient adds per year (adult + pediatric)

Paid rate improvement toward 85% at YE 2029 vs. 81% at YE

20252

Modest annual price increases

Contribution from ex-US geographies

2025¹ 2026 2029

1. Non-GAAP revenue excluding EU for FY 2025

Navenibart: a long-acting plasma kallikrein inhibitor for HAE prophylaxis

Trusted mechanism & modality

Monoclonal antibody inhibitor of plasma kallikrein

Compelling efficacy data

High affinity and potency with fast onset delivers rapid, effective attack prevention

Infrequent dosing schedule

YTE modification for extended half-life

Pain-free subcutaneous administration

Citrate-free, high-concentration formulation, delivered via autoinjector

Potential to be the first therapy for HAE prophylaxis with dosing every 3 or 6 months

Obtained through acquisition of Astria Therapeutics in January 2026

Currently in pivotal Phase 3 studies

IP through 2042

Program is on track to support US regulatory filing by end of 2027

Navenibart: potential for strong efficacy with optimal dosing profile

100%

Mean attack rate reduction

90%

80%

70%

Navenibart (Q3M)

92% mean attack reduction

(Q4W)

13.3 months mean follow up n=17

Navenibart (Q6M)

90% mean attack reduction

11.9 months mean follow up n=12

60%

50%

(Q8W)

40%

30%

0 4 8 12 16 20 24 28

Dosing interval (weeks)

Q3M/Q6M, 3/6-month dosing

NOTE: Efficacy data presented are derived from different clinical trials conducted at different times by different sponsors, with differences in trial design and patient populations. As a result, cross-trial comparisons cannot be made, and no head-to-head clinical trials have been conducted. ANDEMBRY: US Prescribing Information (2025). TAKHZYRO; US Prescribing Information (2025). Dawnzera; US Prescribing Information (2025). Navenibart data from: Long-Term, Sustained, Robust Hereditary Angioedema Attack Suppression with Navenibart Administered Every 3 and 6 Months: ALPHA-SOLAR Interim Results; presented at the 2026 American Academy of Allergy, Asthma &

BioCryst positioned to offer the leading oral and injectable

Combined portfolio to reach distinct, durable, and growing segments, covering full spectrum of patient preference

Prefer an

oral therapy

Prefer an

njectable

therapy

HAE patients receiving prophylaxis primarily fall into two categories:

Navenibart

Navenibart's profile maps precisely to the remaining unmet needs in the injectable market

Oral preference

Has steadily increased since 2023

Consistent growth in ORLADEYO demand reflects this dynamic

Injectable preference

Many patients are satisfied on existing injectables, although there is a clear unmet need

Remaining unmet needs:

Q3M or longer dosing

Administration improvements

Reduced injection site pain

Opportunity to offer the most patient-friendly options, optimally serving the HAE patient community

Significant addressable opportunity in HAE

Estimated US patients on prophylaxis

Navenibart opportunity: 5,000+ patients

9,700

9,100

8,400

7,600

ORLADEYO

predicted share: 2,000+ stable patients

2025 2026 2027 2028 2029 2030 2031 2032 2033 2034

The durability of the ORLADEYO patient base is driven by strong, injectable-like efficacy and the convenience of oral administration

Source: BioCryst Internal Market Research Study (Conducted Jun 2025), 2018-2023 administrative claims data

BioCryst HAE franchise expected to generate double digit revenue growth into the next decade

HAE portfolio revenue1

$1.8B+

$1B+

$563M

~15% CAGR

$1B+

cash balance by 2029

2025E ex-EU³ 2029E 2033E

Significant revenue growth Operating leverage

Strong profitability and cash flow generation

& synergies with Astria

Enhanced financial capacity for future BD

Implied projections for navenibart based on Wall Street analyst research

Non-GAAP operating expenses expected to grow at a mid-single digit CAGR

Licensing agreement for European rights to navenibart

Transaction Overview

Strategic Rationale

BioCryst entered into a license agreement with an Irish affiliate of Neopharmed Gentili for exclusive European commercial rights to navenibart for HAE prophylaxis.

Upfront payment of $70M, with additional payments of up to $275M in future regulatory and sales milestones.

BioCryst will also receive tiered royalties on net sales ranging from 18% to 30%.

Builds on existing relationship with a proven European partner with deep regional expertise.

Optimizes commercial focus and portfolio coordination: both companies can offer ORLADEYO + navenibart in their respective regions.

Strengthens BioCryst's financial position while retaining meaningful upside.

Reinforces ongoing focus on core, supporting commitment to scale in a capital efficient manner

BCX17725: a targeted KLK5 inhibitor for Netherton syndrome (NS)

What is Netherton syndrome?

Aligned with core rare disease focus

High unmet need

Potential for market expansion over time due to misdiagnosis or underdiagnosis

Prescriber landscape comparable to HAE's: addressable by small sales team

In-house analytics expertise primed to aid diagnosis and market development

Clear biology

•

Validated target and known cause of disease: SPINK5 gene variant causes KLK5 overactivity BCX17725 is a systemically-administered KLK5 inhibitor

Potential for BioCryst to be first mover

BCX17725 could be the first-in-class targeted systemic therapy

A severe, rare, genetic disorder with widespread skin involvement and systemic complications

Causes premature separation of skin layers, severe inflammation, and infection risk

Diagnosed US population of ~1,6001 with potential to grow to 3,000-5,000 with greater diagnosis and treatment

No approved targeted therapies

1. Based on healthcare claims analysis

Why Netherton syndrome?

KLK5

KLK14

KLK7

Degrades epithelial junctions

PAR2

Downstream consequences of KLK5 activation

Skin barrier dysfunction

Inflammatory cascade

Atopy

KLK5 initiates the pathologic protease cascade (KLK7, KLK14) and inflammation (via PAR2) in the skin

BCX17725 designed to stop KLK5 overactivity at the top of the pathway

BCX17725 targets KLK5, the key player in Netherton syndrome

BCX17725

Petrova E and Hovnanian A. Exp Opin Orphan Drugs (2020) 8(11): 455-487

PAR2: protease-activated receptor 2

Targeted therapies drive diagnosis of rare diseases

Change in US Prevalence After Launch of Targeted Therapy

+70%

+214%

+67%

+200%

Est. Prevalence (000's)

20 +67%

Netherton Syndrome Population Assumptions

Initial healthcare claims analysis indicates a diagnosed US prevalence of ~1,600

Ongoing analysis leveraging NLP models, EMR data, and rare disease analogs suggest potential for US diagnosed population to increase to greater than 3,000

Fabry HAE PNH C3G Pompe

HAE, hereditary angioedema; PNH, paroxysmal nocturnal hemoglobinuria; C3G, Complement 3 glomerulopathy; NLP, natural language processing; EMR, electronic medial records

SAD

MAD

Completed

Multiple Dose

4 weeks of treatment, open-label, safety and PK

Multiple Dose

12 weeks of treatment, open-label, safety and efficacy

Part 4:

NS patients N = up to 12

Part 3:

NS patients N = 1-3

anticipated

This study is designed to help us understand:

Preliminary safety

Systemic exposure

Distribution into skin

Early efficacy signals

Dosing for pivotal study

BCX17725 Phase 1 study design

Parts 1 & 2:

Healthy volunteers

Data from Part 4 expected by YE 2026

Goal of study: inform and plan for pivotal study in 2027

Finance summary

(Figures in millions)

RESULTS1, 2

Q1 2026

Q1 2025

Q1 2026

Q1 2025

ORLADEYO revenue

$148

$134

$148

$123

Operating profit (loss)

($702)

$21

$54

$43

FY 2026 GUIDANCE3

AS OF

MAY 6, 2026

FEB 26, 2026

ORLADEYO revenue

Unchanged

$625-645

Total revenue

Unchanged

$635-660

Non-GAAP operating expenses

Unchanged

$450-470

OTHER ITEMS4, 5

MAR 31, 2026

PRO FORMA

MAR 31, 2026

Cash and investments

$261

$331

Senior credit facility (Blackstone loan at SOFR + 4.5%)

$400

Non-GAAP ORLADEYO revenue excludes revenues associated with the European ORLADEYO business which was sold to Neopharmed Gentili S.p.A. on October 1, 2025.

Non-GAAP operating profit excludes revenues and expenses associated with the European ORLADEYO business, stock-based compensation, and expenses incurred in connection with our acquisition of Astria, including acquired in-process research and development expense of $697.8 million, assembled workforce amortization, severance and retention related costs, and the portion of the Astria stock option payout attributable to post-combination service.

Non-GAAP operating expenses exclude stock-based compensation, restructuring, and transaction-related costs.

Cash and investments includes cash equivalents, restricted cash, and short-term investments as of March 31, 2026. Pro forma cash and investments includes the prior items plus net proceeds of

$70 million from the license agreement with Neopharmed Gentili for European rights to navenibart after quarter end.

Senior credit facility reflects the Blackstone loan drawn on January 23, 2026.

ORLADEYO direct revenue has reached zero-royalty tier

(Figures in millions)

Illustrative Royalty Dynamic

(Total net royalties paid and payable1)

Royalty terms

$85

Royalty Pharma

$350M-550M: 4.5%

$0-350M: 9.5%

Over $550M:

None

$43

$86

Mid-$80s

$74

Mid-$40s

2024

2025

2026E

At Peak

post-OMERS²

$0-350M:

OMERS $350M-550M:

Over $550M:

10%

None

1.55x ($233M)

Total royalty burden has reached a soft cap now that revenues are over the $550M tier4

Overall royalties expected to remain relatively flat until the OMERS royalty hits its cumulative payback cap (expected in 2029), at which point the total amount will fall approximately in half.

Note: At Peak royalty estimates are for illustrative purposes only.

2026E and At Peak figures illustrate royalties net of royalties on European sales because Neopharmed Gentili S.p.A remits license revenue to BioCryst for these amounts.

Assumes the OMERS royalty has reached its cap.

Direct sales include the United States, key European markets and other markets where ORLADEYO is sold directly or through distributors.

Appendix: Non-GAAP reconciliations

Reconciliation of Non-GAAP Income From Operations

Three Months Ended March 31, 2026 Three Months Ended March 31, 2025

U.S. GAAP

Non-GAAP

Adjustments1 Non-GAAP U.S. GAAP

Non-GAAP

Adjustments1 Non-GAAP

ORLADEYO

148,347 $

- $

148,347

ORLADEYO

134,243 $

11,536 $

122,707

Other revenues

5,050 - 5,050

Other revenues

11,291 - 11,291

Cost of product sales - ORLADEYO

2,696

Cost of product sales - ORLADEYO

1,994

665

1,329

Acquired in-process research and development

697,761

Research and development (excluding stock-based compensation)

28,742

157

28,585

Sales and marketing (excluding stock-based compensation)

42,953

5,482

37,471

General and administrative (excluding stock-based compensation)

21,959

2,417

19,542

Stock-based compensation

16,027 16,027 -

Total operating expenses

124,307 33,800 90,507

Income from operations $ 21,227 $ (22,264) $ 43,491

Total operating expenses 858,011 755,838 102,173

Stock-based compensation 21,368 21,368 -

General and administrative (excluding stock-based compensation) 42,393 21,088 21,305

Sales and marketing (excluding stock-based compensation) 47,670 9,193 38,477

Research and development (excluding stock-based compensation) 53,500 15,480 38,020

Cost of product sales - peramivir 2,574 - 2,574

Cost of product sales - peramivir 2,681 - 2,681

Expenses:

Total revenues 145,534 11,536 133,998

Total revenues 156,413 - 156,413

License revenue - - -

License revenue 3,016 - 3,016

Revenues:

(Loss) income from operations $ (701,598) $ (755,838) $ 54,240

1 Reflects the following non-GAAP adjustments for the three months ended March 31, 2026:

Expenses incurred in connection with the acquisition of Astria Therapeutics, Inc. on January 23, 2026:

1 Represents revenues and expenses associated with our European ORLADEYO business which was sold to Neopharmed Gentili S.p.A. on October 1, 2025 and consolidated stock-based compensation.

Acquired in-process research and development related to navenibart $ 697,761

Assembled workforce amortization $ 600

Expense associated with severance and retention award agreements $ 12,321

Portion of stock option payout attributable to post-combination service $ 29,129

Stock-based compensation $ 16,027

Disclaimer

BioCryst Pharmaceuticals Inc. published this content on May 06, 2026, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on May 06, 2026 at 11:23 UTC.