Pelecopan

—Q1 2025 ORLADEYO net revenue of $134.2 million (+51 percent y-o-y)— —Full year 2025 ORLADEYO revenue guidance increased to $580 million to $600 million— —Company now expects to be profitable for full year 2025, a year ahead of schedule— —Company pays down $75 million of debt; saves approximately $23.5 million over life of loan— —NDA submitted for ORLADEYO oral granules for children with HAE aged 2-11— —U.S. IND open for BCX17725 for Netherton syndrome— RESEARCH TRIANGLE PARK, N.C., May 05, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today reported financial results for the first quarter ended March 31, 2025, and provided a corporate update. “We started 2025 with another quarter of outstanding performance. ORLADEYO revenue growth was driven by moving ORLADEYO patients from free drug to paid at a much faster rate than we expected, resulting in a substantial increase to our annual guidance as we also move closer to peak sales of $1 billion. This increased financial strength accelerates our path to profitability and enables us to start paying down our debt, while continuing to invest in and advance our pipeline,” said Jon Stonehouse, president and chief executive officer of BioCryst. ORLADEYO® (berotralstat): Oral, Once-daily Treatment for Prevention of Hereditary Angioedema (HAE) Attacks ORLADEYO net revenue in the first quarter of 2025 was $134.2 million (+51 percent year-over-year (y-o-y)).The total percentage of all ORLADEYO patients on paid drug has increased to approximately 84 percent (compared to 73.5 percent at end of 2024), generating higher than expected ORLADEYO revenue that the company expects will continue through the full year.First quarter prescriptions were strong, above the quarterly average in 2024, and the percentage of U.S. HAE patients who describe a strong preference for an oral prophylaxis therapy increased to 70 percent, up from 50 percent in 2023, in the company’s latest market survey of HAE patients. Sales from the U.S. contributed 89.5 percent of global ORLADEYO net revenues in the first quarter. The number of patients treated with ORLADEYO outside the U.S. continued to grow in new and existing markets. “By driving a dramatic increase in the rate of paid ORLADEYO patients through the prescription reauthorization period, on top of continued very strong new patient demand, our team has achieved in four months what we had expected would take three years, getting the rate of paid patients nearly to our long-term goal of 85 percent. As a result, we will capture significantly more revenue opportunity in 2025 and subsequent years and our path to peak sales of $1 billion is even more profitable than we had projected,” said Charlie Gayer, chief commercial officer of BioCryst. Rare Disease Pipeline The goal with our pipeline is to build on our success with ORLADEYO by bringing additional selected, highly differentiated rare disease products to patients. The company has submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) to expand the ORLADEYO label to children with HAE aged 2 to 11 using an oral granule formulation. The company also expects to submit regulatory filings in 2025 in global territories, including Europe, Japan and Canada. ORLADEYO would be the first targeted oral prophylactic therapy for children with HAE.The FDA has cleared the company’s investigational new drug application (IND) which will enable its clinical trial of BCX17725, an investigational KLK5 inhibitor for the treatment of Netherton syndrome, to enroll patients in the United States. This phase 1 trial is also open in Australia. The company expects initial data from the program in 2025. Netherton syndrome is a serious, rare, lifelong genetic disorder causing disruption of the skin barrier with premature separation of the skin layers, chronic inflammation and vulnerability to serious infections, caused by lack of normal function of a natural inhibitor of KLK5. People with Netherton syndrome often have itchy, red, scaly, inflamed skin, fragile hair, and are more likely to develop severe food allergies, asthma and eczema. Netherton syndrome can be life threatening, especially during infancy when patients are vulnerable to dehydration and recurrent infections. Currently, there are no approved treatments that target the underlying cause of Netherton syndrome. BCX17725 is designed to replace missing functions of the natural KLK5 inhibitor, which could restore the normal skin barrier and result in improved skin function, including protection from severe inflammatory and infectious complications of the disease. The first clinical trial with suprachoroidal delivery of avoralstat, the company’s investigational plasma kallikrein inhibitor for the treatment of diabetic macular edema (DME), has been granted authorization to proceed in Australia. The company expects initial data from DME patients in 2025. DME is an important cause of vision loss in diabetes and is due to leakage of fluid from the blood vessels in the retina. While current treatments focus on vascular endothelial growth factor (VEGF) inhibition, DME can develop from other mechanisms, such as the kallikrein-bradykinin pathway. This is supported by observations that many DME patients have an incomplete response to intravitreal anti-VEGF therapies that are administered every four to eight weeks. Avoralstat targets the kallikrein-bradykinin system on the retinal vascular endothelial cells and may result in less vascular leakage and less edema. Avoralstat, delivered to the suprachoroidal space, is designed to provide long-lasting exposure to the retinal vessels, which could result in less frequent injections and a reduced burden on patients and the healthcare system. “Today we are reporting significant milestones for each of the next three programs in our pipeline, demonstrating our focus on advancing medicines with the potential to provide differentiated outcomes for patients of all ages. As we move closer to delivering an urgently needed oral prophylactic therapy to children with HAE, we are simultaneously progressing two clinical-stage programs in Netherton syndrome and DME towards the first patient data later this year,” said Dr. Helen Thackray, chief research and development officer of BioCryst. First Quarter 2025 Financial Results For the three months ended March 31, 2025, total revenues were $145.5 million (+$52.7 million y-o-y), compared to $92.8 million in the first quarter of 2024 (+56.8 percent y-o-y). The increase was primarily due to $134.2 million (+$45.3 million y-o-y) in ORLADEYO net revenue in the first quarter of 2025, compared to $88.9 million in ORLADEYO net revenue in the first quarter of 2024 (+51.0 percent y-o-y). Research and development expenses for the first quarter of 2025 decreased to $37.3 million from $46.5 million in the first quarter of 2024 (-19.8 percent y-o-y), primarily due to decreased expenses driven by the discontinuation and close-out of the Factor D programs, BCX10013 and BCX9930. These reductions were partially offset by an increase in other research, preclinical and development costs, comprised of avoralstat and other early-phase pipeline programs, and a change in general and administrative expense allocations. Selling, general and administrative expenses for the first quarter of 2025 increased to $82.5 million, compared to $59.5 million in the first quarter of 2024 (+38.7 percent y-o-y). The increase was primarily due to increased commercial investment to support our growing ORLADEYO revenue, our newly launched regions, expanded international operations and global commercial support activities. Additionally, there was an increase to general and administrative expenses, and an offsetting reduction to research and development expenses, due to a change in the general and administrative expense allocations in 2025. Operating income for the first quarter of 2025 was $21.2 million, compared to an operating loss of $14.5 million for the first quarter of 2024. Non-GAAP operating income, excluding stock-based compensation expense, was $42.6 million for the first quarter of 2025, compared to a non-GAAP operating loss of $0.8 million for the first quarter of 2024. Interest expense was $23.5 million in the first quarter of 2025, compared to $24.5 million in the first quarter of 2024 (-4.1 percent y-o-y). The decrease was primarily due to a decrease in interest expense associated with the interest accrued under the Pharmakon Loan Agreement. Net income for the first quarter of 2025 was $32 thousand, or $0.00 per share, compared to a net loss of $35.4 million, or $0.17 per share, for the first quarter of 2024. Cash, cash equivalents, restricted cash and investments totaled $317.3 million at March 31, 2025, compared to $338.4 million at March 31, 2024. Net cash utilization for the first quarter of 2025 was $25.5 million, which was driven by debt service, royalty payments and one-time first quarter compensation expense. Early in the second quarter, the company paid down $75 million of the outstanding Pharmakon debt, which will result in approximately $23.5 million in interest savings over the life of the loan. Financial Outlook for 2025The company is increasing its outlook for full year 2025 global net ORLADEYO revenue to be between $580 million and $600 million (previously $535 million to $550 million). The company now expects full year 2025 operating expenses will be $440 million to $450 million (previously $425 million to $435 million). This operating expense outlook does not reflect non-cash stock compensation expense. The company is accelerating its expectation for sustainable profitability and positive cash flows by a year. The company now expects to deliver net income and positive cash flows for full year 2025 (previously expected both for full year 2026). 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 the $75 million Pharmakon prepayment made in April 2025. Conference Call and WebcastBioCryst 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. About BioCryst Pharmaceuticals BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with complement-mediated and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class oral small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. For more information, please visit www.biocryst.com or follow us on LinkedIn. Forward-Looking Statements This press release contains forward-looking statements, including statements regarding future results, performance or achievements, and expectations regarding pipeline development timing. These statements involve known and unknown risks, uncertainties and other factors which may cause BioCryst’s actual results, performance, achievements or pipeline development timing to be materially different from any future results, performance, achievements, or timing expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: BioCryst’s ability to successfully implement or maintain its commercialization plans for ORLADEYO; BioCryst’s ability to successfully progress its pipeline development plans as described herein, including meeting the expected timelines; the results of BioCryst’s partnerships with third parties may not meet BioCryst’s current expectations; risks related to government actions, including that decisions and other actions, including as they relate to pricing, may not be taken when expected or at all, or that the outcomes of such decisions and other actions may not be in line with BioCryst’s current expectations; the commercial viability of ORLADEYO, including its ability to achieve sustained market acceptance and demand; ongoing and future preclinical and clinical development of product candidates may take longer than expected and may not have positive results; the outcome of preclinical testing and early clinical trials may not be predictive of the success of later clinical trials, and interim results of a clinical trial do not necessarily predict final results; BioCryst may not be able to enroll the required number of subjects in planned clinical trials of product candidates; BioCryst may not advance human clinical trials with product candidates as expected; the FDA or other applicable regulatory agency may require additional studies beyond the studies planned for products and product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may not review regulatory filings on our expected timeline, may impose certain restrictions, warnings, or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, delay or withdraw market approval for products and product candidates; product candidates, if approved, may not achieve market acceptance; BioCryst’s ability to successfully commercialize its products and product candidates; BioCryst’s ability to successfully manage its growth and compete effectively; risks related to the international expansion of BioCryst’s business; timing for achieving and sustainability of profitability and positive cash flow may not meet management’s expectations; statements and projections regarding financial guidance and goals and the attainment of such goals may differ from actual results based on market factors and BioCryst’s ability to execute its operational and budget plans; and actual financial results may not be consistent with expectations, including that revenue, operating expenses and cash usage may not be within management’s expected ranges. Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst’s most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause actual results to differ materially from those contained in BioCryst’s projections and forward-looking statements. Non-GAAP Financial MeasuresThe information furnished in this release 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” or “adjusted.” We believe providing these non-GAAP measures, which show our results with these items adjusted, is valuable and useful since they allow management and investors to better understand the company’s financial performance in the absence of certain non-cash items such as stock-based compensation and allow investors to more accurately understand our current and past period results and more easily compare them to future results. 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, operating income, net income, and earnings per share. Our reference to our first quarter 2024 and 2025 “non-GAAP” financial measure of non-GAAP operating income (loss) constitutes a non-GAAP financial measure. It refers to our GAAP results, adjusted to show the results without including non-cash stock compensation expense. A reconciliation between GAAP and non-GAAP operating income (loss) is provided in the table below. We also provide our non-GAAP operating expense outlook for full year 2025, which refers to our expected GAAP operating expense, excluding stock-based compensation expense. 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 for full year 2025 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. The actual amount of stock-based compensation expense for the full year 2025 could have a material impact on GAAP reported results for the guidance period. BCRXW Contact:John Bluth+1 919 859 7910jbluth@biocryst.com BIOCRYST PHARMACEUTICALS, INC.CONSOLIDATED FINANCIAL SUMMARY(In thousands, except per share) Statements of Operations (unaudited) Three Months Ended March 31, 2025 2024 Revenues: ORLADEYO$134,243 $88,867 Other 11,291 3,894 Total revenues 145,534 92,761 Expenses: Cost of product sales 4,568 1,265 Research and development 37,270 46,493 Selling, general and administrative 82,469 59,491 Total operating expenses 124,307 107,249 Income (loss) from operations 21,227 (14,488) Other income (expense): Interest income 3,024 4,031 Interest expense (23,494) (24,506)Foreign currency gains (losses), net 1 (51)Total other expense (20,469) (20,526) Income (loss) before income taxes 758 (35,014)Income tax expense 726 365 Net income (loss)$32 $(35,379) Net income (loss) per common share: basic$0.00 $(0.17)Weighted average shares of common stock outstanding: basic 208,882 206,064 Net income (loss) per common share: diluted$0.00 $(0.17)Weighted average shares of common stock outstanding: diluted 215,261 206,064 Balance Sheet Data (in thousands) March 31, 2025 December 31, 2024 (unaudited) Note 1Cash, cash equivalents and investments$315,640 $341,173 Restricted cash 1,691 1,610 Receivables 93,394 79,069 Total assets 480,047 490,420 Secured term loan 315,413 314,869 Royalty financing obligation 500,918 513,729 Accumulated deficit (1,770,008) (1,770,040)Stockholders’ deficit (451,927) (475,934)Shares of common stock outstanding 209,208 208,543 Note 1: Derived from audited financial statements. Reconciliation of Adjusted Income (Loss) from Operations (in thousands) Three Months Ended March 31, 2025 2024 GAAP income (loss) from operations$21,227 $(14,488)Less: Stock-based compensation expense (21,368) (13,652)Adjusted income (loss) from operations$42,595 $(836)

—Q2 2024 ORLADEYO net revenue grows 34 percent y-o-y to $108.3 million— —Full-year 2024 ORLADEYO revenue guidance increased to $420-$435 million (previously $390-$400 million)— —Company generates GAAP operating profit of $8.8 million in second quarter ($21.9 million non-GAAP operating profit)— —Pipeline continues to advance— RESEARCH TRIANGLE PARK, N.C., Aug. 05, 2024 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today reported financial results for the second quarter ended June 30, 2024, and provided a corporate update. “The first half of 2024 has been outstanding for BioCryst due to the success we are having in the marketplace with ORLADEYO. As a result, we are increasing our full year guidance for ORLADEYO, advancing multiple programs toward the clinic in the next 18 months and moving the company closer to profitability,” said Jon Stonehouse, president and 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 2024 was $108.3 million (+34 percent year-over-year (y-o-y)).Operational improvements drove revenue above expectations for the second quarter. This included a three percent increase in the overall rate of paid patients relative to the end of 2023, which now stands at 74.4 percent.New patient starts continued at the same high rate seen in the prior two quarters, matching the new patient demand seen in the first three quarters of the launch in 2021, and patient retention remained strong. We continue to see more than 60 percent of patients on paid therapy staying on for at least a year.A recent market research study showed that 52 percent of allergist/immunologists are extremely likely to prescribe ORLADEYO to more patients, up from 29 percent in early 2023.With additional recent approvals and reimbursement authorizations in Europe and Latin America, ORLADEYO is now commercially available to patients in more than 20 countries. Ex-U.S. ORLADEYO revenue in the second quarter increased 51 percent y-o-y and accounted for 11 percent of global ORLADEYO net revenues. “Our second quarter revenue growth and increased full year guidance for ORLADEYO reflect strong underlying demand and favorable patient outcomes, combined with our continuing focus on improving patient services and market access operations. The real-world experience of patients who respond to ORLADEYO is consistent with the 91 percent reduction in attacks from baseline that we saw in long-term clinical trials. The potential for patients to have that high level of HAE attack control in a once-daily pill is a major differentiator in the marketplace that makes ORLADEYO’s trajectory toward $1 billion in peak sales increasingly clear,” said Charlie Gayer, chief commercial officer of BioCryst. Rare Disease Pipeline The goal with our pipeline is to build on our success with ORLADEYO by bringing additional selected, highly differentiated rare disease products to patients. The company remains on track to submit a regulatory filing in 2025 to expand the ORLADEYO label to enable children as young as two years of age to receive an oral granule formulation of ORLADEYO. ORLADEYO would be the first oral prophylactic therapy for children with HAE.The company has completed its clinical evaluation of its oral Factor D inhibitor, BCX10013. The drug was safe and well tolerated at all doses studied, however the level of clinical activity observed was less than other therapies on the market and potential partners have declined to make the additional investment required to evaluate higher doses. BioCryst plans to discontinue development, consistent with its previously announced plans.The company expects to advance BCX17725, its KLK-5 inhibitor for the treatment of Netherton syndrome, into the clinic by the end of 2024. Netherton syndrome is a serious, rare, lifelong genetic disorder affecting the skin, hair and immune system. People with Netherton syndrome often have red, scaly, inflamed skin, fragile hair, and are more likely to develop skin infections, allergies, asthma and eczema. Netherton syndrome can be life threatening, especially during infancy when patients are vulnerable to dehydration and recurrent infections. Currently, there is no approved treatment for Netherton syndrome. In 2025, the company plans to advance avoralstat, a plasma kallikrein inhibitor, into a clinical trial of patients with diabetic macular edema (DME). DME is an important cause of vision loss in diabetes and is due to leakage from the blood vessels in the retina. While current treatments focus on VEGF inhibition, DME can develop from other mechanisms, such as the kallikrein-bradykinin pathway. This is supported by observations that many DME patients have an incomplete response to intravitreal anti-VEGF therapies that are administered every four to eight weeks. Avoralstat targets the kallikrein-bradykinin system on the retinal vascular endothelial cells and may result in less vascular leakage and less edema. Avoralstat, delivered to the suprachoroidal space as a depot formulation, is designed to provide high dose levels to the retinal vessels with long-lasting exposure, which could result in less frequent injections and a reduced burden on patients and the healthcare system. Second Quarter 2024 Financial Results For the three months ended June 30, 2024, total revenues were $109.3 million, compared to $82.5 million in the second quarter of 2023 (+32.5 percent year-over-year (y-o-y)). The increase was primarily due to $108.3 million in ORLADEYO net revenue in the second quarter of 2024, compared to $81.0 million in ORLADEYO net revenue in the second quarter of 2023 (+33.7 percent y-o-y). R&D expenses for the second quarter of 2024 decreased to $37.6 million from $51.2 million in the second quarter of 2023 (-26.6 percent y-o-y), primarily due to decreased spending on BCX10013 and the discontinuation of the BCX9930 program. These reductions were partially offset by increased investment in BCX17725, avoralstat and other discovery programs, and our ongoing ORLADEYO pediatric trial. Selling, general and administrative expenses for the second quarter of 2024 increased to $61.2 million, compared to $51.0 million in the second quarter of 2023 (+20.0 percent y-o-y), primarily due to an increase in commercial expenses to support growing revenue, newly launched regions and expanded international operations. In addition, there was an increase in general and administrative expenses, primarily related to an increase in resourcing to support our accounting and IT functions. Total operating expenses were $100.6 million for the second quarter of 2024, compared to $103.2 million in the second quarter of 2023 (-2.5 percent y-o-y). Non-cash stock compensation was $13.2 million for the second quarter of 2024. Total operating expenses, not including non-cash stock compensation for the second quarter of 2024 were $87.4 million, compared to $90.4 million in the second quarter of 2023 (-3.3 percent y-o-y). The company generated a GAAP operating profit of $8.8 million in the second quarter of 2024. This compares to a GAAP operating loss of $20.7 million in the second quarter of 2023. Adjusted for non-cash stock compensation, the non-GAAP operating profit was $21.9 million in the second quarter of 2024, compared to a non-GAAP operating loss of $7.9 million in the second quarter of 2023. Interest expense was $24.7 million in the second quarter of 2024, compared to $28.9 million in the second quarter of 2023 (-14.5 percent y-o-y). The decrease was primarily due to a decrease in the amortization of interest associated with our royalty financing obligations. Net loss for the second quarter of 2024 was $12.7 million, or $0.06 per share, compared to a net loss of $75.3 million, or $0.40 per share, for the second quarter of 2023. In the second quarter of 2023, there was a $29.0 million one-time debt extinguishment fee related to the close-out of the Athyrium debt facility. Excluding this one-time event, non-GAAP net loss for the second quarter of 2023 was $0.24 per share. Cash, cash equivalents, restricted cash and investments totaled $338.1 million at June 30, 2024, compared to $415.7 million at June 30, 2023. Operating cash use for the second quarter of 2024 was $0.2 million. Non-GAAP Pro forma Financial Measures The information furnished in this release includes non-GAAP pro forma 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” or “adjusted.” We believe providing these non-GAAP measures, which show our pro forma results with these items adjusted, is valuable and useful since they allow the company and investors to better understand the company’s financial performance in the absence of these one-time events and allow investors to more accurately understand our second quarter 2023 and second quarter 2024 results and more easily compare them to future results. These non-GAAP pro forma measures also correspond with the way we expected Wall Street analysts to compare our results. Our non-GAAP pro forma 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, operating income, net income, operating profit and earnings per share. Our references to our second quarter 2023 and first six months 2023 “non-GAAP pro forma” financial measures of adjusted net loss and adjusted earnings per share constitute non-GAAP financial measures. They refer to our GAAP results, adjusted to show the results without the one-time loss on the extinguishment of the Athyrium term loans. Our reference to our second quarter 2024 and first six months 2024 “non-GAAP pro forma” financial measure of non-GAAP operating profit constitutes a non-GAAP financial measure. It refers to our GAAP results, adjusted to show the results without including non-cash stock compensation expense. Financial Outlook for 2024 Based on the operational improvements and strong patient and physician demand for ORLADEYO seen in the first half of 2024, the company is raising its outlook for full year 2024 global net ORLADEYO revenue to be between $420 million and $435 million (previously $390 million to $400 million). The company maintains its prior operating expense outlook, and expects full year 2024 operating expenses to be between $365 million and $375 million, flat to full year 2023 operating expenses. This operating expense outlook does not reflect non-cash stock compensation expense, or one-time expenses related to the previously announced workforce reduction implemented in the first quarter of 2024. Based on the company’s disciplined approach to capital allocation, and the increased revenue guidance for ORLADEYO, the company is confident that it will achieve a full-year operating profit in 2024 (not including non-cash stock compensation), be approaching quarterly positive earnings per share (EPS) and positive cash flow in the second half of 2025 (not including non-cash stock compensation) and be profitable on an EPS basis, with positive cash flow, for full year 2026. The company expects it can achieve these financial milestones without raising additional funds and does not intend to draw the additional $150 million of debt available to it from Pharmakon. Conference Call and WebcastBioCryst 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. About BioCryst Pharmaceuticals BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with complement-mediated and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class oral small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. For more information, please visit www.biocryst.com or follow us on LinkedIn. Forward-Looking StatementsThis press release contains forward-looking statements, including statements regarding future results, performance or achievements. These statements involve known and unknown risks, uncertainties and other factors which may cause BioCryst’s actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: BioCryst’s ability to successfully implement or maintain its commercialization plans for ORLADEYO; risks related to the planned discontinuation of the development of BCX10013; BioCryst’s ability to successfully progress its pipeline development plans as described herein; risks related to the reduction in size of BioCryst’s R&D organization; the results of BioCryst’s partnerships with third parties may not meet BioCryst’s current expectations; risks related to government actions, including that decisions and other actions, including as they relate to pricing, may not be taken when expected or at all, or that the outcomes of such decisions and other actions may not be in line with BioCryst’s current expectations; the commercial viability of ORLADEYO, including its ability to achieve sustained market acceptance; ongoing and future preclinical and clinical development of product candidates may take longer than expected and may not have positive results; the FDA or other applicable regulatory agency may require additional studies beyond the studies planned for products and product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may impose certain restrictions, warnings, or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, delay or withdraw market approval for products and product candidates; product candidates, if approved, may not achieve market acceptance; BioCryst’s ability to successfully commercialize its products and product candidates; BioCryst’s ability to successfully manage its growth and compete effectively; risks related to the international expansion of BioCryst’s business; timing for achieving profitability and positive cash flow may not meet management’s expectations; statements and projections regarding financial guidance and goals and the attainment of such goals may differ from actual results based on market factors and BioCryst’s ability to execute its operational and budget plans; and actual financial results may not be consistent with expectations, including that revenue, operating expenses and cash usage may not be within management’s expected ranges. Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst’s most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause actual results to differ materially from those contained in BioCryst’s projections and forward-looking statements. BCRXW Contact:John Bluth+1 919 859 7910jbluth@biocryst.com BIOCRYST PHARMACEUTICALS, INC.CONSOLIDATED FINANCIAL SUMMARY(In thousands, except per share) Statements of Operations (unaudited) Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Revenues: ORLADEYO $108,288 $81,009 $197,155 $149,423 Other 1,044 1,482 4,938 1,846 Total revenues 109,332 82,491 202,093 151,269 Expenses: Cost of product sales 1,699 894 2,964 1,825 Research and development 37,623 51,247 84,116 99,635 Selling, general and administrative 61,214 50,997 120,578 98,864 Royalty 35 56 162 63 Total operating expenses 100,571 103,194 207,820 200,387 Income (loss) from operations 8,761 (20,703) (5,727) (49,118) Interest income 3,554 3,750 7,585 7,128 Interest expense (24,733) (28,915) (49,239) (56,311)Foreign currency (losses) gains, net (84) 301 (135) 72 Loss on extinguishment of debt — (29,019) — (29,019)Loss before income taxes $(12,502) $(74,586) $(47,516) $(127,248)Income tax expense 172 740 537 1,411 Net loss $(12,674) $(75,326) $(48,053) $(128,659) Basic and diluted net loss per common share $(0.06) $(0.40) $(0.23) $(0.68) Weighted average shares outstanding 206,425 189,118 206,244 188,815 Balance Sheet Data (in thousands) June 30, 2024 December 31, 2023 (unaudited) (Note 1)Cash, cash equivalents and investments $336,344 $388,987 Restricted cash 1,795 1,804 Receivables 68,759 56,950 Total assets 472,419 516,960 Secured term loan 313,822 303,231 Royalty financing obligation 523,633 531,599 Accumulated deficit (1,729,212) (1,681,159)Stockholders’ deficit (475,606) (455,528)Shares of common stock outstanding 206,629 205,771 Note 1: Derived from audited financial statements. Reconciliation of Adjusted Net Loss and Adjusted Diluted Earnings Per Share (in thousands) Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 GAAP net loss $(12,674) $(75,326) $(48,053) $(128,659)Less: One-time R&D restructuring expense — — (1,264) — Less: One-time loss on extinguishment of Athyrium term loans — (29,019) — (29,019)Adjusted net loss $(12,674) $(46,307) $(46,789) $(99,640) GAAP basic and diluted net loss per common share $(0.06) $(0.40) $(0.23) $(0.68) Adjusted basic and diluted net loss per common share $(0.06) $(0.24) $(0.23) $(0.53) Reconciliation of Adjusted Income (Loss) from Operations (in thousands) Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 GAAP income (loss) from operations $8,761 $(20,703) $(5,727) $(49,118)Less: Stock-based compensation expense (13,173) (12,841) (26,825) (26,848)Adjusted income (loss) from operations $21,934 $(7,862) $21,098 $(22,270)