On August 4, 2025 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the second quarter ended June 30, 2025, and provided a corporate update (Press release, BioCryst Pharmaceuticals, AUG 4, 2025, View Source [SID1234654722]).

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"The financial performance this quarter is the best in the company’s history resulting from better-than-expected revenue growth and very meaningful operating profit. In the fifth year since approval, ORLADEYO revenue and demand have never been stronger, and this is driven by outstanding execution and increasing confidence in the product. Our accelerating operating profit and the sale of our European ORLADEYO business strengthen our financial position to deliver even greater value, and our pipeline remains on track for initial data later this year in two clinical programs," said Jon Stonehouse, chief executive officer of BioCryst.

ORLADEYO (berotralstat): Oral, Once-daily Treatment for Prevention of Hereditary Angioedema (HAE) Attacks

ORLADEYO net revenue in the second quarter of 2025 was $156.8 million (+45 percent year-over-year (y-o-y)).

New patient prescriptions in the second quarter were the highest ever in a quarter, beating those in the first quarter of the launch by over 10 percent.

The number of new prescribers of ORLADEYO in the U.S. in the second quarter increased to 69, up from 59 in the first quarter.

Patient discontinuations in the U.S. were lower in the first half of 2025 than in the first half of 2024, despite the larger base of patients taking ORLADEYO.

New real-world data from over 350 patients with HAE with normal C1 inhibitor showed substantial reductions in attack rates with ORLADEYO, reinforcing its value for a historically underserved patient segment and providing strong evidence to close both treatment and reimbursement gaps.

Sales from the U.S. contributed 89.5 percent of global ORLADEYO net revenues in the second quarter.

"ORLADEYO continued its upward trajectory in the second quarter, delivering our strongest quarter yet for new patient prescriptions and revenue. Growth was fueled by increasing demand in the U.S. and internationally, improved efficiency in getting paid shipments, fewer discontinuations, gross-to-net improvements, and continued impact of our real-world evidence generation—especially for patients with HAE with normal C1 inhibitor. With this momentum, we are confident in meeting our prior full-year guidance, even when factoring in the expected removal of European ORLADEYO sales in the fourth quarter," said Charlie Gayer, president and chief commercial officer of BioCryst.

Rare Disease Pipeline

Our goal is to build on our success with ORLADEYO by bringing additional selected, highly differentiated products to patients with rare diseases.

The Prescription Drug User Fee Act goal date for the company’s new drug application for ORLADEYO granules in children with HAE aged 2 to 11 is December 12, 2025. ORLADEYO would be the first targeted oral prophylactic therapy for children with HAE.

BCX17725, an investigational KLK5 inhibitor for the treatment of Netherton syndrome, is enrolling a phase 1 trial in healthy volunteers and patients. The company expects initial data from this program by the end of the year.

Avoralstat, an investigational plasma kallikrein inhibitor for the treatment of diabetic macular edema (DME), is enrolling a phase 1 trial in patients. The company expects initial data from this program by the end of the year.

Second Quarter 2025 Financial Results

For the three months ended June 30, 2025, total revenues were $163.4 million, compared to $109.3 million in the second quarter of 2024 (+50 percent y-o-y). The increase was primarily due to $156.8 million in ORLADEYO net revenue in the second quarter of 2025, compared to $108.3 million in ORLADEYO net revenue in the second quarter of 2024 (+45 percent y-o-y).

Research and development expenses for the second quarter of 2025 increased to $43.4 million from $37.6 million in the second quarter of 2024 (+15 percent y-o-y), primarily due to an increase in preclinical and early clinical work for avoralstat and BCX17725, investigational new drug application-enabling activities for early phase pipeline programs, and stock-based compensation. These increases were partially offset by the discontinuation and close-out of the Factor D programs and ORLADEYO-related regulatory, safety, quality, and manufacturing expenses, previously recorded in research and development, that are now recorded in selling, general, and administrative to reflect the program’s commercial progression.

Selling, general and administrative expenses for the second quarter of 2025 increased to $87.4 million, compared to $61.2 million in the second quarter of 2024 (+43 percent y-o-y). Approximately $10.7 million of the increase was driven by deal-related costs and stock-based compensation. Approximately $6.5 million was driven by ORLADEYO-related regulatory, safety, quality, and manufacturing expenses, previously recorded in research and development, that are now recorded in selling, general, and administrative to reflect the program’s commercial progression. The remainder was driven by the growth of ORLADEYO and general and administrative expenses.

Operating income for the second quarter of 2025 was $29.8 million, compared to $8.8 million for the second quarter of 2024. Non-GAAP operating income, excluding stock-based compensation expense and deal-related costs, was $57.0 million for the second quarter of 2025, compared to $21.9 million for the second quarter of 2024.

Interest expense was $21.6 million in the second quarter of 2025, compared to $24.7 million in the second quarter of 2024 (-13 percent y-o-y). The decrease was primarily the result of the $75 million partial prepayment on the outstanding principal amount under the Pharmakon Term Loan in April 2025, and the decrease in the effective interest rate related to the Pharmakon Loan Agreement.

Net income for the second quarter of 2025 was $5.1 million, or $0.02 per share, compared to a net loss of $12.7 million, or $0.06 per share, for the second quarter of 2024. Non-GAAP net income, excluding stock-based compensation expense and deal-related costs, was $32.3 million, or $0.15 per share, for the second quarter of 2025, compared to $0.5 million, or $0.00 per share, for the second quarter of 2024.

Cash, cash equivalents, restricted cash and investments totaled $287.1 million at June 30, 2025, of which $15.1 million of cash and cash equivalents are held within the company’s European business and is reflected in current assets held for sale, compared to $338.1 million at June 30, 2024. Net cash utilization for the second quarter of 2025 was $30.4 million, which was driven by the $75 million Pharmakon prepayment made in April 2025. Excluding this prepayment, there was $44.6 million of cash generated during the quarter, primarily driven by ORLADEYO sales.

In July, the company paid down an additional $50 million on the outstanding principal amount under the Pharmakon term loan, leaving a remaining principal balance of $199 million. Upon the expected closing of the sale of its European business in early October, the company intends to retire all its remaining term debt.

Financial Outlook for 2025
The company is maintaining its outlook for full year 2025 global net ORLADEYO revenue to between $580 million and $600 million, even when excluding fourth quarter European revenue after the expected closing of the sale of its European business.

Excluding stock-based compensation expense and deal-related costs, and without removal of fourth quarter European operating expenses, the company expects 2025 non-GAAP operating expenses to be between $440 million and $450 million. The company plans to provide updated 2025 operating expense guidance on its 3Q 2025 earnings call, after the expected closing of the sale of its European business.

The company remains on track to deliver net income and positive cash flows for full year 2025. Positive cash flow refers to the improvement in cash, cash equivalents, restricted cash and investments from year end 2024 to year end 2025, not including the impact of $125 million in Pharmakon prepayments made in 2025.

Conference Call and Webcast
BioCryst management will host a conference call and webcast at 8:30 a.m. ET today to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 1-844-481-2942 for domestic callers and 1-412-317-1866 for international callers. A live webcast and replay of the call will be available online in the investors section of the company website at www.biocryst.com.