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BioCryst Pharmaceuticals, Inc. (BCRX): Business Model Canvas [Dec-2025 Updated] |
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BioCryst Pharmaceuticals, Inc. (BCRX) Bundle
You're looking at BioCryst Pharmaceuticals, Inc. right now, trying to map out how a promising biotech company finally transitions to consistent profitability. Honestly, the whole model pivots on Orladeyo, their oral HAE (Hereditary Angioedema) treatment, which is set to deliver between $590 million to $600 million in net product revenue for the full year 2025, a huge milestone. This is a story about commercial execution meeting pipeline ambition. So, if you want to see the nuts and bolts-from their key partnerships like the Neopharmed Gentili deal to the high-touch customer relationships they maintain-dive into the full Business Model Canvas we've mapped out below.
BioCryst Pharmaceuticals, Inc. (BCRX) - Canvas Business Model: Key Partnerships
You're looking at the relationships BioCryst Pharmaceuticals, Inc. uses to get its products to market and advance its pipeline, so let's break down the key players and the associated financial commitments as of late 2025.
Neopharmed Gentili for European commercialization of Orladeyo
BioCryst Pharmaceuticals, Inc. completed the sale of its European ORLADEYO® (berotralstat) business to Neopharmed Gentili around October 1, 2025. This move was strategic, allowing BioCryst to focus on the U.S. market and immediately improve its operating margin. The deal was valued at up to $264 million, with an upfront payment of $250 million. An additional potential $14 million is tied to future sales milestones in Central and Eastern Europe.
This transaction is expected to result in at least $50 million in expected annual operating expense savings for BioCryst. BioCryst plans to use the proceeds to retire its remaining term debt of $249 million, which eliminates approximately $70 million in future interest payments. Following this, BioCryst projects ending 2027 with approximately $700 million in cash and no term debt. Neopharmed Gentili is taking over the European commercialization, and their rare disease business was expected to generate approximately €50 million in sales year-over-year in 2025. Even excluding the European revenue after the close, BioCryst maintained its full-year 2025 revenue guidance of $580 million to $600 million.
Proposed acquisition of Astria Therapeutics for late-stage HAE asset navenibart
BioCryst Pharmaceuticals, Inc. entered into an agreement in October 2025 to acquire Astria Therapeutics, bringing in navenibart, an injectable, long-acting plasma kallikrein inhibitor for hereditary angioedema (HAE) prophylaxis. The implied enterprise value of this acquisition is approximately $700 million, with an implied aggregate equity value of about $920 million. The consideration per share is a mix of $8.55 in cash and 0.58 or 0.59 shares of BioCryst common stock. The transaction is expected to close in the first quarter of 2026. Upon closing, Astria stockholders are projected to own about 15% of the combined company. This acquisition is intended to complement Orladeyo, which had $437.6 million in sales in 2024. BioCryst estimates its 2025 Orladeyo revenue, excluding the EU, will be $550 million, and projects the combined HAE portfolio could reach $1 billion in revenue by 2029.
Contract research organizations (CROs) for global clinical trials
BioCryst Pharmaceuticals, Inc. relies on Contract Research Organizations (CROs) to manage and conduct its preclinical and clinical trials. The financial terms for these agreements vary and can depend on milestones like patient enrollment success. The company's Research and Development expenses for the second quarter of 2025 rose to $43.4 million, up from $37.6 million in Q2 2024, partly due to early clinical work on pipeline assets like avoralstat and BCX17725. The global CRO market size was valued at $76.70 billion in 2024.
The company expects initial data from two clinical programs, including avoralstat, by the end of 2025.
Specialty distributors and pharmacies for Orladeyo distribution
In the United States, ORLADEYO distribution is managed through a specialty pharmacy provider, which started shipping in December 2020. Outside the US, sales are made to specialty distributors and to hospitals and pharmacies. The market preference data shows a significant shift; the percentage of HAE patients preferring oral prophylaxis increased from 51% in 2023 to 70% in 2025.
The net revenue from ORLADEYO in the second quarter of 2025 reached $156.8 million, marking a 45% year-over-year increase. The number of new patient prescriptions in Q2 2025 was the highest ever for a quarter, beating the launch quarter by over 10%.
Potential partners for avoralstat (DME program) after initial data
BioCryst Pharmaceuticals, Inc. is developing avoralstat, a plasma kallikrein inhibitor, for diabetic macular edema (DME) in partnership with Clearside Biomedical, using Clearside's SCS Microinjector® for suprachoroidal delivery. BioCryst expected initial data from this DME program in 2025.
The financial structure of the Clearside partnership includes specific payments:
| Payment Type | Amount/Terms | Trigger/Basis |
| Upfront License Fee (Paid by BioCryst to Clearside) | $5 million | Agreement execution |
| Clinical/Regulatory Milestones (Potential) | Up to $30 million | Trial success/Regulatory approval |
| Post-Approval Sales Milestones (Potential) | Up to $47.5 million | Global net product sales reaching up to $2 billion |
| Royalties (Paid by BioCryst to Clearside) | Tiered mid-single digit royalties | Annual global net product sales, top tier at > $1.5 billion |
This collaboration aims to leverage avoralstat's characteristics-high potency and low solubility-to potentially offer efficacy with reduced dosing frequency for DME patients.
BioCryst Pharmaceuticals, Inc. (BCRX) - Canvas Business Model: Key Activities
You're looking at the core engine driving BioCryst Pharmaceuticals, Inc. right now, late in 2025. It's all about maximizing the current asset while aggressively funding the next wave of growth. Here's the breakdown of what the company is actively doing day-to-day to execute this model.
Global commercialization and sales of Orladeyo (berotralstat)
The primary activity is pushing Orladeyo, which is the oral, once-daily treatment for preventing hereditary angioedema (HAE) attacks. The momentum is clearly there; Q3 2025 net revenue hit $159.1 million, marking a 37 percent year-over-year jump. To be fair, the U.S. market is doing the heavy lifting, representing 89 percent of that global net revenue in the third quarter. The commercial team is still adding new prescribers, bringing in 64 new prescribers in the U.S. during Q3 2025 alone. A key metric for sustained revenue is patient stickiness; patient retention at the one-year mark is holding steady around 60 percent. Management is confident enough to raise the full-year 2025 global net revenue guidance for Orladeyo to between $590 to $600 million.
Here's a quick look at the commercial ramp-up:
| Metric | Value/Amount (Late 2025 Data) | Period/Context |
| Q3 2025 Net Revenue (Orladeyo) | $159.1 million | Third Quarter 2025 |
| FY 2025 Net Revenue Guidance (Orladeyo) | $590 to $600 million | Full Year 2025 Outlook |
| U.S. Share of Global Net Revenue | 89 percent | Q3 2025 |
| New U.S. Prescribers Added | 64 | Q3 2025 |
| One-Year Patient Retention Trend | ~60 percent | Long-term trend |
Research and development (R&D) for pipeline assets like BCX17725
The R&D engine is focused on diversification beyond HAE. BCX17725, a protein therapeutic targeting Netherton syndrome, is a major current focus. This asset is in a Phase 1 clinical trial, and the company expects initial results by the end of 2025. This is a critical activity because, honestly, Netherton syndrome currently has no approved treatments. The financial commitment reflects this pipeline push; Q3 2025 R&D expenses were $44.6 million, up 9 percent year-over-year, driven by BCX17725 advancement and pre-clinical work. Still, the company showed cost discipline by cutting R&D expenses in Q1 2025 to $37.3 million after discontinuing Factor D programs.
Regulatory filings for new indications (e.g., Orladeyo granules for pediatric HAE)
Expanding the label for the existing blockbuster is a high-priority activity. BioCryst Pharmaceuticals, Inc. is actively pursuing regulatory approval for an oral granule formulation of Orladeyo specifically for pediatric HAE patients. As of early 2025, the company was on track to submit a new drug application (NDA) to the FDA in 2025, with one source noting an expected decision date of September 12, 2025. This is about capturing the entire patient population, not just the adults.
Strategic capital allocation, including debt retirement and acquisitions
Capital management is a defining activity for late 2025. The company executed a major financial cleanup by completing the sale of its European ORLADEYO business. The proceeds from that sale were immediately put to work to retire all remaining Pharmakon term debt. This deleveraging effort was already underway, with a $75 million paydown of that debt occurring in early Q2 2025. Furthermore, the company signed a definitive agreement to acquire Astria Therapeutics for approximately $700 million in enterprise value, which will be financed with about $280 million in equity and $420 million in cash. This is a clear pivot toward using operational cash flow to fund M&A for future growth.
Maintaining intellectual property (IP) and patent protection for Orladeyo until 2040
Vigorously defending the market exclusivity for Orladeyo is a non-negotiable activity. The earliest estimated generic launch date, based on current patents and exclusivities, is May 01, 2040. This protection is being actively tested; BioCryst Pharmaceuticals, Inc. initiated a lawsuit in 2025 against Annora Pharma over an ANDA filing. The legal fight centers on three specific U.S. patents set to expire in 2039, while six other patents expiring in 2035 are not being challenged in that specific action. The company is definitely investing resources here to secure that long runway.
Key IP defense metrics include:
- Estimated generic entry date: May 01, 2040.
- Patents challenged in litigation expiring: 2039.
- Uncontested Orange Book patents expiring: 2035.
- Total US patents protecting the drug: 10.
Finance: draft 13-week cash view by Friday.
BioCryst Pharmaceuticals, Inc. (BCRX) - Canvas Business Model: Key Resources
You're looking at the core assets that make BioCryst Pharmaceuticals, Inc. run right now, late in 2025. It's all about the product that's generating the cash and the financial strength to keep the pipeline moving. Here's the breakdown of what they are leaning on.
Orladeyo (berotralstat) as the primary commercial asset
Orladeyo is the engine. The third quarter of 2025 saw ORLADEYO net revenue hit $159.1 million, which was a 37 percent year-over-year increase. This performance led BioCryst Pharmaceuticals, Inc. to raise its full-year 2025 global net ORLADEYO revenue guidance to between $590 million and $600 million. The U.S. market is the dominant piece, accounting for 89 percent of global ORLADEYO net revenues in Q3 2025. Patient retention has held steady, showing a long-term trend of approximately 60 percent at one year. They added 64 new prescribers in the U.S. during Q3 2025.
Cash reserves and operating cash flow for pipeline investment
The financial position has been actively managed through strategic divestiture. At September 30, 2025, cash, cash equivalents, restricted cash, and investments totaled $269.4 million. After the sale of the European business and debt retirement, the pro forma cash balance at September 30, 2025, was $294 million. For the third quarter of 2025, net cash utilization was $17.8 million, but excluding a $50 million Pharmakon prepayment, $32.2 million of cash was generated that quarter, primarily from ORLADEYO sales. Over the last 12 months, operating cash flow was $50.20 million. The company lowered its 2025 non-GAAP operating expense guidance to between $430 million and $440 million.
Here's a quick look at the balance sheet changes following recent activity:
| Financial Metric | September 30, 2025 (Actual) | September 30, 2025 (Pro Forma) |
| Cash, cash equivalents, restricted cash & investments (in millions) | $269 | $294 |
| Senior credit facility (in millions) | $199 | $0 |
| ORLADEYO Royalty Revenues Paid and Payable (Q3 2025, in millions) | $20 | $77 (9 Months Ended) |
Intellectual property and patents protecting the drug portfolio
The protection for the drug portfolio, centered on ORLADEYO, is a foundational resource. The company is focused on maintaining exclusivity for its oral treatments in rare diseases. The strategic sale of the European ORLADEYO business was completed, which impacts future royalty structures but strengthens the core U.S. asset protection.
Clinical data and expertise in the Hereditary Angioedema (HAE) space
BioCryst Pharmaceuticals, Inc. has deep, established expertise in the HAE space, which is evidenced by the sustained commercial success of ORLADEYO. Beyond ORLADEYO, the pipeline reflects this expertise, with BCX17725 advancing into the clinic, which drove Research and development expenses up to $44.6 million in Q3 2025. Initial data from Part 3 of the BCX17725 trial is expected by the end of Q1 2026. Furthermore, the planned acquisition of Astria Therapeutics, valued at approximately $700 million in enterprise value, is intended to expand its presence in the HAE market.
Specialized rare disease commercial and medical affairs teams
The commercial success is directly tied to the execution by the specialized teams. Selling, general, and administrative expenses for the third quarter of 2025 were $83.0 million, reflecting the growth of ORLADEYO and associated regulatory costs. The team's execution resulted in 64 new U.S. prescribers added in Q3 2025.
BioCryst Pharmaceuticals, Inc. (BCRX) - Canvas Business Model: Value Propositions
You're looking at the core value BioCryst Pharmaceuticals, Inc. (BCRX) is delivering to its customers-patients and prescribers-as of late 2025. It's a mix of established oral convenience and high-potential pipeline expansion.
Orladeyo: Oral, once-daily prophylaxis for HAE, a defintely lower burden than injectables
The primary value proposition centers on ORLADEYO (berotralstat), the first oral, once-daily treatment for preventing Hereditary Angioedema (HAE) attacks. This convenience is clearly resonating; Q3 2025 ORLADEYO net revenue hit $159.1 million, a 37 percent year-over-year increase. The U.S. market is the engine, accounting for 89 percent of global net revenues in Q3 2025. Prescriber confidence is high, evidenced by 64 new prescribers added in the U.S. during Q3 2025, exceeding the two-year quarterly average. Furthermore, patient retention remains solid, holding a long-term trend of approximately 60 percent at one year. This oral option is capturing preference, with 70 percent of U.S. HAE patients in a recent survey stating a strong preference for an oral prophylactic, up from 50 percent in 2023.
The financial strength derived from this product is a value proposition in itself. BioCryst raised its full-year 2025 global net ORLADEYO revenue guidance to between $590 to $600 million. More importantly, the company is on track to achieve net income and positive cash flows for the full year 2025, a year ahead of its previous schedule. This improved financial stability is cemented by the completion of the sale of the European ORLADEYO business, the proceeds of which were used to retire all remaining Pharmakon term debt.
Potential for a second oral HAE prophylaxis with navenibart (post-Astria acquisition)
BioCryst Pharmaceuticals, Inc. is layering in a second, differentiated HAE prophylaxis through the planned acquisition of Astria Therapeutics, valued at an implied enterprise value of approximately $700 million. Navenibart, the asset, is an injectable monoclonal antibody targeting HAE, but its value proposition is ultra-infrequent dosing-potentially every 3 or 6 months. This offers a convenience premium over existing injectables, which range from every two weeks to every two months. Earlier trial data suggests it can match or beat current standards, showing an average 92 percent reduction in HAE attack rate and a 50 percent attack-free rate in Phase 1b/2 testing. The transaction is expected to close in the first quarter of 2026, and upon closing, Astria shareholders will own about 15 percent of the merged company.
Addressing ultra-rare diseases like Netherton syndrome (BCX17725)
The pipeline extends value creation into other ultra-rare spaces. BCX17725 targets Netherton syndrome, a serious, lifelong genetic disorder for which there are currently no approved treatments. This investigational protein therapeutic is designed to be a potential best-in-class therapy by inhibiting KLK5. The value here is the promise of a targeted, disease-modifying option where only supportive care exists. Initial results from the ongoing Phase 1 clinical trial are expected by the end of 2025.
You can see the current portfolio focus laid out here:
| Asset | Indication | Development Stage (Late 2025) | Key Value Differentiator |
| ORLADEYO (Berotralstat) | HAE Prophylaxis (Adult/Adolescent) | Commercialized (Global) | Oral, once-daily dosing |
| ORLADEYO (Granules) | HAE Prophylaxis (Pediatric 2-11 yrs) | NDA Review (PDUFA Dec 12, 2025) | First oral prophylactic for this age group |
| Navenibart | HAE Prophylaxis (Injectable) | Phase 3 Global Trial | Ultra-infrequent dosing (every 3-6 months) |
| BCX17725 | Netherton Syndrome | Phase 1 Trial (Initial data end of 2025) | Potential first-in-class targeted therapy |
Expanding treatment options for pediatric HAE patients (granules NDA)
For the existing ORLADEYO franchise, the value proposition is expanding convenience to younger patients. The New Drug Application (NDA) for ORLADEYO oral granules in children aged 2 to 11 years is under review, with the FDA's target action date set for December 12, 2025. This is a priority review, and interim data from the APeX-P study showed an 86 percent reduction in HAE attacks requiring professional care after just 12 weeks of treatment. If approved, this would offer the first targeted oral prophylactic for children under 12, addressing the logistical and psychological burden of current options for this vulnerable population.
BioCryst Pharmaceuticals, Inc. (BCRX) - Canvas Business Model: Customer Relationships
BioCryst Pharmaceuticals, Inc. focuses its customer relationships around the specialized needs of the Hereditary Angioedema (HAE) patient community and the specialists who treat them, primarily driven by the oral prophylactic therapy, ORLADEYO (berotralstat).
High-touch, specialized support for rare disease patient communities is evidenced by strong patient retention metrics, which suggest effective ongoing support for this rare disease population.
- 93.1% of pediatric patients completed $\ge$48 weeks of treatment with ORLADEYO.
- Patient survey findings indicated that 54% of patients preferred a daily oral therapy over biweekly or monthly injections when effectiveness was comparable.
- New real-world data from over 350 patients with HAE with normal C1 inhibitor showed substantial reductions in attack rates with ORLADEYO, reinforcing value for this segment.
Patient support programs to facilitate access and adherence to Orladeyo are supported by real-world adherence data that compares favorably to other long-term prophylactic (LTP) therapies.
| Metric (12-Month Follow-up) | ORLADEYO (n=90) | Lanadelumab (n=189) | SC-pdC1-INH (n=78) |
| Adherence Rate | 77% | 76% | 80% |
| Persistence Rate | 71% | 63% | 63% |
The data shows that 86% of ORLADEYO patients, 91% of lanadelumab patients, and 83% of SC-pdC1-INH patients filled at least two prescriptions during the 12-month follow-up period.
Direct engagement with HAE specialists and allergists is reflected in the growth of the prescribing base, indicating successful outreach and education to the core physician segment.
- New patient prescriptions for ORLADEYO in the second quarter of 2025 were the highest ever in a quarter, exceeding those in the first quarter of launch by over 10 percent.
- The number of new prescribers of ORLADEYO in the U.S. in the second quarter of 2025 increased to 69, up from 59 in the first quarter.
Educational initiatives for physicians on oral prophylaxis benefits are demonstrated by the consistent presentation of clinical and real-world efficacy data to the medical community.
- Data on sustained HAE attack rate reductions were presented at the European Academy of Allergy and Clinical Immunology (EAACI) meeting in June 2025.
- New data on attack rate reductions were presented at the Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI) in November 2025.
- In one study, adolescents on ORLADEYO experienced 1.85 fewer attacks/month at 18 months compared to baseline.
Managed access programs to transition patients from free to paid drug are supported by the company's financial performance and specific patient affordability initiatives.
BioCryst Pharmaceuticals, Inc. reported ORLADEYO net revenue of $156.8 million for the second quarter of 2025, representing a 45 percent year-over-year increase. The full year 2025 ORLADEYO net revenue guidance was increased to between $535-$550 million. The company expects to achieve positive EPS and positive cash flow in the second half of 2025 (2H 2025). As of March 31, 2025, the company held $315.6 million in cash, cash equivalents, and investments. Management noted favorable early signs that many more Medicare patients taking ORLADEYO are able to afford therapy due to lower copayments under the Inflation Reduction Act. Sales from the U.S. contributed 89.5 percent of global ORLADEYO net revenues in the second quarter of 2025.
BioCryst Pharmaceuticals, Inc. (BCRX) - Canvas Business Model: Channels
You're looking at how BioCryst Pharmaceuticals, Inc. gets its product, ORLADEYO, into the hands of patients, and how they manage their pipeline reach. It's a mix of a focused internal team and external partners, especially after the recent strategic shift in Europe.
Direct sales force in the U.S. and select international markets
The core of the commercial channel remains the U.S. direct sales force, which is clearly the engine driving the majority of the revenue. In the third quarter of 2025, U.S. sales made up a commanding 89% of global ORLADEYO net revenues. That team added 64 new prescribers in the U.S. during Q3 2025, a number that beat the company's two-year quarterly average. This direct engagement is crucial for a rare disease product like ORLADEYO, where physician education matters a lot. The long-term trend for patient retention shows stability, holding at approximately 60% at the one-year mark, which helps secure that recurring revenue stream. The company projects this U.S. channel will eventually support a steady state of over 2,000 patients by 2028.
Specialty pharmacies for distribution of Orladeyo
For distribution in the United States, BioCryst Pharmaceuticals relies on a single specialty pharmacy for the distribution of the approved drug product. This streamlined approach is key to managing a niche product. The effectiveness of this channel is reflected in the rapid conversion of patients to paid status; by the first quarter of 2025, the total percentage of all ORLADEYO patients on paid drug had climbed to approximately 84%, up from 73.5% at the end of 2024. This channel handles the logistics after the prescription is written by the physician.
Licensing and distribution agreements (e.g., Neopharmed Gentili in Europe)
The European channel underwent a major transformation in 2025. BioCryst Pharmaceuticals completed the sale of its European ORLADEYO business to Neopharmed Gentili, effective in late 2025. This divestiture was structured to immediately strengthen the balance sheet. Neopharmed Gentili paid an upfront amount of $250 million, with up to an additional $14 million possible from future milestones tied to Central and Eastern Europe sales. This move is expected to save BioCryst Pharmaceuticals at least $50 million in expected annual operating expenses. Before this sale, ORLADEYO had achieved acceptance in over 30 countries, supported by various commercial partners, including past agreements with Pint Pharma for pan-Latin America and Swixx for 15 markets in CEE.
Digital and patient advocacy channels for rare disease awareness
While direct spending on digital channels isn't explicitly detailed, the success in driving patient preference is a proxy for effective awareness efforts. Market research from 2025 shows a significant shift: the percentage of patients preferring oral prophylaxis for hereditary angioedema (HAE) has jumped to 70%, up from 50% in 2023. Conversely, the preference for injection/infusion treatments dropped to just 13% in 2025. This suggests strong communication efforts are resonating with the patient community regarding the convenience of an oral, once-daily treatment.
Clinical trial sites for pipeline drug development
The channel for pipeline development involves establishing clinical trial sites globally to advance investigational therapies. For avoralstat, the first clinical trial for diabetic macular edema (DME) was authorized to proceed in Australia, with initial data anticipated in 2025. Separately, the FDA cleared the Investigational New Drug (IND) application for BCX17725 for Netherton syndrome, allowing a Phase 1 trial to enroll patients in the United States, with this trial also open in Australia, expecting initial data in 2025.
Here's a quick look at the key commercial and financial metrics tied to these channels as of late 2025:
| Metric | Value / Amount | Period / Context |
|---|---|---|
| ORLADEYO Net Revenue | $159.1 million | Q3 2025 |
| ORLADEYO Net Revenue Growth (y-o-y) | +37 percent | Q3 2025 |
| FY 2025 ORLADEYO Revenue Guidance | $590 to $600 million | Full Year 2025 |
| U.S. Share of Global ORLADEYO Revenue | 89% | Q3 2025 |
| New U.S. Prescribers Added | 64 | Q3 2025 |
| ORLADEYO Patients on Paid Drug | Approx. 84% | Q1 2025 |
| European Business Sale Upfront Proceeds | $250 million | June 2025 Agreement |
| Estimated Annual Operating Expense Savings from EU Sale | At least $50 million | Annually |
The wholesale acquisition cost for a pack of ORLADEYO is listed at $44,484.33. Also, the company's cash position as of September 30, 2025, was reported at $269 million, with a pro forma balance of $294 million.
BioCryst Pharmaceuticals, Inc. (BCRX) - Canvas Business Model: Customer Segments
You're looking at the core groups BioCryst Pharmaceuticals, Inc. (BCRX) serves with its rare disease portfolio, primarily centered around ORLADEYO (berotralstat) for Hereditary Angioedema (HAE). These segments drive the company's revenue, which for full-year 2025 is guided to be between $580 million and $600 million in global net ORLADEYO revenue, a significant increase from the initial guidance of $535 million to $550 million.
Adult and adolescent patients with Hereditary Angioedema (HAE)
This is the established, core customer base for the capsule formulation of ORLADEYO, which is indicated for prophylaxis in patients aged 12 years and older. The commercial success in this segment is clear from the revenue figures; for instance, the third quarter of 2025 saw ORLADEYO net revenue hit $159.1 million, a 37% increase year-over-year. The U.S. market remains the largest contributor, accounting for 89.5% of global ORLADEYO net revenues in the second quarter of 2025. Furthermore, the preference for oral therapy among HAE treaters in the U.S. is strong, with 70% in a Q1 2025 survey describing a strong preference for an oral prophylaxis therapy, up from 50% in 2023.
Pediatric HAE patients aged 2-11 years old (new segment via granules)
This represents a critical expansion segment, moving beyond the initial approved age group. BioCryst Pharmaceuticals, Inc. is actively targeting this group with an oral granule formulation of ORLADEYO. The New Drug Application (NDA) for this formulation was under review with the U.S. FDA, with a Prescription Drug User Fee Act (PDUFA) goal date set for December 12, 2025. The need is evident, as HAE attacks often start early; the median age at diagnosis was 2.0 years, with 82.8% of patients experiencing symptoms before age 6.
Interim data from the APeX-P trial in children aged 2 to <12 years shows compelling efficacy for the oral granules:
| Metric | Data Point | Context/Timeframe |
|---|---|---|
| Reduction in Attacks Requiring Professional Care | 86% | After 12 weeks of treatment |
| Mean Monthly Attack Rate Change | From 1.28 to 0.38 | During the first four weeks of treatment |
| Patients Attack-Free at Month 12 | 70.4% | Ongoing APeX-P trial |
The total percentage of all ORLADEYO patients on paid drug increased to approximately 84% as of Q1 2025, up from 73.5% at the end of 2024, showing successful conversion across patient types.
HAE treating physicians, including allergists and immunologists
Physicians are the key gatekeepers who prescribe ORLADEYO and will evaluate the new pediatric granule formulation. The company is actively engaging this segment through data presentations; for example, new data was presented at the American College of Allergy, Asthma & Immunology (ACAAI) meeting in November 2025. Prescription growth indicates physician adoption. In the second quarter of 2025, the number of new ORLADEYO prescribers in the U.S. increased to 69, up from 59 in the first quarter. Also, a market tracking survey of 60 HAE treaters in early 2025 showed that 97% were considering prescribing ORLADEYO.
Government and private payers (insurance companies)
Payers control access and reimbursement, which is vital for converting free-drug patients to paid status. The strong revenue growth in 2025 is explicitly attributed to moving patients from free drug to paid status at a faster rate than expected. The company's gross profit margin, reported at 64.25%, reflects strong commercial execution which supports payer negotiations. Outside the U.S., ORLADEYO was reimbursed in all major countries in Western Europe by early 2025, except for the Netherlands, with reimbursement expected in the first half of 2025. The company expects to be net income and cash flow positive for the full year 2025, signaling financial stability that supports long-term payer relationships.
Future: Patients with Netherton syndrome
This represents a future, unmet medical need segment for BioCryst Pharmaceuticals, Inc. The company is developing BCX17725, a KLK5 inhibitor, for this ultra-rare disease. Currently, there are no approved treatments for Netherton syndrome. Initial results from the Phase 1 clinical trial evaluating BCX17725 are expected by the end of 2025. Research and development expenses for Q3 2025 increased partly due to the advancement of BCX17725.
BioCryst Pharmaceuticals, Inc. (BCRX) - Canvas Business Model: Cost Structure
You're looking at the costs that drive BioCryst Pharmaceuticals, Inc.'s operations as of late 2025. It's a mix of heavy investment in the pipeline, scaling commercial operations, and major one-time strategic expenses.
High R&D expenses for advancing BCX17725 and navenibart development
Research and development spending reflects the commitment to pipeline progression. For the third quarter of 2025, Research and development expenses rose to $44.6 million, up from $41.1 million in the third quarter of 2024. This increase is directly tied to advancing BCX17725 into the clinic and executing investigational new drug (IND)-enabling activities for pre-clinical programs. While the Astria Therapeutics acquisition, expected to close in Q1 2026, brings navenibart into the portfolio, the Q3 2025 R&D spend reflects internal pipeline work.
Selling, General, and Administrative (SG&A) costs for Orladeyo commercialization
Costs related to commercializing Orladeyo (berotralstat) are a significant component, especially as the company scales. Selling, general and administrative expenses grew by 27% year-over-year in the third quarter of 2025. Looking at the second quarter of 2025, SG&A hit $87.4 million, up from $61.2 million the prior year. This growth includes costs associated with commercial progression; for instance, approximately $6.5 million in Q2 2025 was driven by Orladeyo-related regulatory, safety, quality, and manufacturing expenses that were reclassified into SG&A to reflect the drug's commercial maturity.
The full-year 2025 non-GAAP operating expense guidance, which excludes stock-based compensation and transaction-related costs, was lowered to between $430 million and $440 million.
Here are some key operating expense metrics for the recent quarters:
| Metric | Q3 2025 Amount | Q2 2025 Amount | Q3 2024 Amount |
| Research and Development Expenses | $44.6 million | $43.4 million | $41.1 million |
| Selling, General, and Administrative Expenses | Not specified as GAAP total | $87.4 million | $61.2 million |
| Non-GAAP Operating Expenses (Excl. SBC & Transaction Costs) | Approximately $108 million | Not specified | Approximately $92 million |
Manufacturing and supply chain costs for a specialty oral drug
Costs tied to the physical product, like manufacturing and supply chain, are embedded within operating expenses. As noted above, specific Orladeyo-related manufacturing expenses, totaling about $6.5 million in Q2 2025, are now captured within SG&A due to the drug's commercial status. These costs support the global commercialization of the specialty oral drug.
Acquisition costs related to the Astria Therapeutics transaction
A major cost consideration is the definitive agreement signed on October 14, 2025, to acquire Astria Therapeutics, Inc. The total enterprise value for this transaction is approximately $700 million. The consideration per share is structured as $8.55 in cash and 0.59 shares of BioCryst common stock, implying a value of $13.00 per Astria share. BioCryst secured a debt commitment letter for up to $550 million from Blackstone-managed funds to help fund the cash portion, alongside existing cash. Transaction-related costs are explicitly excluded from the non-GAAP operating expense guidance for 2025, meaning the GAAP figures will be higher when the deal closes in Q1 2026.
Interest expense, though reduced by debt retirement from European sale proceeds
Interest expense shows a clear trend of reduction due to proactive debt management. In the third quarter of 2025, interest expense was $19.7 million, a decrease of 21% from $24.8 million in the third quarter of 2024. This reduction stems partly from a $125 million partial prepayment on the Pharmakon Term Loan made during 2025, and a lower effective interest rate. Furthermore, the sale of the European Orladeyo business was completed, and the proceeds were used in October 2025 to retire all remaining Pharmakon term debt, which was approximately $200 million outstanding at that time. This action leaves the company with $0 term debt on a pro forma basis as of September 30, 2025, significantly altering the future interest cost structure.
The interest expense for Q2 2025 was $21.6 million, following a $75 million partial prepayment in April 2025.
BioCryst Pharmaceuticals, Inc. (BCRX) - Canvas Business Model: Revenue Streams
The revenue streams for BioCryst Pharmaceuticals, Inc. are heavily concentrated around its flagship product, ORLADEYO (berotralstat), supplemented by other product sales and significant one-time financial events from strategic divestitures.
The primary driver is the net product revenue from ORLADEYO sales, which the company projects to be between $590 million to $600 million for the full-year 2025, based on the retained global business excluding the European operations for the fourth quarter of 2025. This projection was raised following the third quarter of 2025 results.
The revenue streams can be broken down as follows:
- Net product revenue from ORLADEYO sales, projected at $590 million to $600 million for full-year 2025 (retained business).
- Revenue from RAPIVAB (peramivir injection) sales, which was previously guided as part of a total revenue package that implied a range of $25 million to $45 million when factoring in earlier 2025 total revenue guidance versus ORLADEYO guidance.
- Proceeds from the sale of the European ORLADEYO business, which was completed for an upfront payment of approximately $250 million.
- Potential future milestone and royalty payments from licensed assets, including up to $14 million in future milestones tied to Central and Eastern European sales from the European business divestiture.
The company has also set clear financial targets for the year, indicating a transition to sustained profitability.
| Financial Metric | 2025 Projection/Target | Context/Source Data Point |
|---|---|---|
| FY 2025 Global Net ORLADEYO Revenue (Retained Business) | $590 million to $600 million | Raised outlook following Q3 2025 results. |
| European ORLADEYO Business Sale Proceeds (Upfront) | $250 million | Completed transaction proceeds. |
| Q3 2025 Non-GAAP Net Income | $35.6 million | Actual result for the third quarter. |
| FY 2025 Expected Outcome | Net income and positive cash flow | Full-year target, excluding debt repayment impacts. |
| Potential Future Milestones (European Sale) | Up to $14 million | Tied to future sales in Central and Eastern Europe. |
The completion of the European sale, which generated $250 million upfront, was strategically used to retire the remaining Pharmakon term loan balance of $199 million. The pro forma cash balance at September 30, 2025, was $294 million, which included the net proceeds from the European sale.
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