Financial Data and Key Metrics Changes - Revenue for Q4 2021 was $47.2 million, with $46.2 million from net sales of ORLADEYO, marking a significant increase from $122.6 million in total revenue for the year [24][6]. - The company expects ORLADEYO revenue to grow to no less than $250 million in 2022, with a peak sales target of $1 billion [25][6]. - Operating expenses for Q4 2021 were $90.8 million, with a projected range of $440 million to $480 million for full year 2022 [24][25]. Business Line Data and Key Metrics Changes - ORLADEYO's launch has been successful, achieving $122.6 million in net revenue in its first year, and the company anticipates doubling sales in 2022 [6][9]. - The patient retention rate for ORLADEYO was reported at 70%, with nearly 80% of patients switching from Takhzyro remaining on therapy for at least six months [10][11]. Market Data and Key Metrics Changes - The company has broad coverage for ORLADEYO, with at least 80% of the HAE population in the U.S. having access to coverage [11]. - The U.S. is expected to account for the majority of ORLADEYO sales, with the company already launching in seven other countries and anticipating further regulatory approvals [13][25]. Company Strategy and Development Direction - The company aims to continue growing ORLADEYO in the U.S. and globally while advancing its pipeline, particularly focusing on the oral Factor D inhibitor, BCX9930 [8][21]. - The strategy includes investing in the Factor D program, which has the potential for multiple indications, and expanding the pipeline to develop new drugs [21][26]. Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving $1 billion in peak global sales for ORLADEYO, supported by strong market demand for oral therapies [6][13]. - The company is focused on maximizing value creation through strategic investments in its pipeline and enhancing the ORLADEYO launch [26][25]. Other Important Information - The company ended Q4 2021 with approximately $518 million in cash and plans to draw down an additional $75 million debt tranche midyear 2022 [25][24]. - Management highlighted the importance of setting patient expectations to improve retention rates for ORLADEYO [30][31]. Q&A Session Summary Question: What best practices can improve retention rates for ORLADEYO? - Management emphasized the importance of preparing patients for potential GI effects and setting realistic expectations for therapy [30][31]. Question: What is the expected timing for data from the renal basket study? - Management indicated that the study is enrolling independently and aims to achieve proof-of-concept by the end of 2023 [33][34]. Question: Will Q1 sales for ORLADEYO be lower sequentially from Q4? - Management confirmed that Q1 is expected to have little to no growth due to seasonal reimbursement dynamics but anticipates stronger growth in subsequent quarters [37][40]. Question: How does the company view competition from gene therapy and gene editing? - Management believes that while gene therapies may be beneficial for hard-to-treat patients, ORLADEYO's once-daily oral administration will remain competitive for those controlled on the drug [66][67]. Question: What is the growth expectation for operating expenses in 2022? - Management indicated that the majority of the growth in operating expenses will be driven by R&D investments, particularly for the Factor D program [52][53].

Financial Data and Key Metrics Changes - For Q3 2021, the company reported revenue of $41 million, with $37 million coming from net sales of ORLADEYO [29] - The company expects full-year net revenue from ORLADEYO to be between $115 million and $120 million [8][30] - Operating expenses for the quarter were $72.5 million, excluding non-cash stock compensation [29] Business Line Data and Key Metrics Changes - ORLADEYO has shown strong growth, with more than half of new patients switching from other prophylactic treatments [11][76] - The prescriber base for ORLADEYO grew by 25% in Q3, with nearly half of the top 500 HAE treaters now prescribing the drug [11] - The average patient now receives paid product within 30 days, indicating improved access [12][46] Market Data and Key Metrics Changes - The company is launching ORLADEYO in multiple international markets, including the UK, Germany, France, Japan, and the UAE [16][30] - The UK market is expected to see a significant uptake due to favorable NICE recommendations, allowing broader patient access [16][35] - The company anticipates that international revenue will become more meaningful in 2022 [30] Company Strategy and Development Direction - The company aims to leverage its discovery platform to develop more oral drugs for rare diseases, with BCX9930 as the next candidate [9][23] - The strategy includes expanding the use of ORLADEYO globally while continuing to advance the Factor D program [30] - The company is focused on building a strong pipeline of therapies for rare diseases, with ongoing clinical trials for BCX9930 [23][60] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in exceeding the global peak sales target of $500 million for ORLADEYO [9] - The company noted that the launch of ORLADEYO has exceeded expectations, even during the pandemic [7] - Management highlighted the importance of in-person interactions and live meetings to enhance physician engagement and patient education [68] Other Important Information - The company ended Q3 with $204 million in cash, providing a strong financial position to support global launches and R&D efforts [30] - The gross-to-net adjustments for ORLADEYO are improving, with a target of 15% to 20% of peak sales [29][52] Q&A Session Summary Question: What is the current status of ORLADEYO sales in Europe? - Management indicated that sales are starting in Germany and France, with expectations for a slower ramp-up due to COVID impacts on patient-doctor interactions [34] Question: How is enrollment for the PNH studies proceeding? - Management noted that enrollment is on track, with no significant barriers anticipated, but the competitive landscape makes it hard to predict exact timelines [37] Question: Can you provide more details on the prescriber base growth? - The prescriber base grew by 25% in Q3, with a doubling of repeat prescriptions among top prescribers, indicating strong adoption [43] Question: What is the current access situation for ORLADEYO? - Payer access is improving, with most patients receiving paid product within 30 days, and continued progress is expected into the next year [46] Question: What percentage of patients are currently on free drug? - As of Q3, about one-third of patients are on free drug, with expectations that more than half will transition to paid products over time [85]

Financial Performance - Total revenues for the three months ended September 30, 2021, were $40,994,000, a significant increase from $6,102,000 in the same period of 2020[25]. - Product sales reached $39,141,000 for the three months ended September 30, 2021, compared to $2,478,000 in the same period of 2020, reflecting a growth of approximately 1,479%[25]. - The net loss for the nine months ended September 30, 2021, was $166,283,000, compared to a net loss of $122,321,000 in the same period of 2020, indicating a 36% increase in losses[27]. - The company reported a comprehensive loss of $58,774,000 for the three months ended September 30, 2021, compared to a comprehensive loss of $46,109,000 in the same period of 2020, an increase of 27.5%[25]. - The total revenues for the nine months ended September 30, 2021, were $110,012, compared to $13,796 for the same period in 2020, indicating an increase of about 696%[64]. - The total operating expenses for the nine months ended September 30, 2021, were $235,555,000, compared to $136,148,000 in 2020, reflecting a 73.2% increase[25]. - The company expects its 2021 expenses to exceed its 2021 revenues, continuing to incur operating losses and negative cash flows until revenues reach a sufficient level[36]. Cash and Liabilities - Cash, cash equivalents, and restricted cash at the end of the period were $203,893,000, down from $274,348,000 at the beginning of the period, reflecting a decrease of 25.6%[27]. - Total current liabilities increased to $133,608,000 in 2021 from $105,654,000 in 2020, marking a rise of 26.4%[24]. - As of September 30, 2021, the total stockholders' equity (deficit) was $(147,044) thousand, reflecting a net loss of $(58,801) thousand for the quarter[31]. - The company may need to raise additional capital in the future to fund operations, which could impact its operational flexibility[9]. - The company has access to an additional $75 million from its existing credit facility, which it believes will fund operations into 2023[36]. Research and Development - Research and development expenses for the nine months ended September 30, 2021, were $145,279,000, up from $87,610,000 in 2020, representing a 65.7% increase[25]. - The company integrates biology, crystallography, medicinal chemistry, and computer modeling to develop small molecule pharmaceuticals[35]. - Research and development expenses include direct and indirect costs, with significant reliance on third-party contract research organizations (CROs) for clinical studies[78]. Product Development and Sales - The company is focused on advancing product candidates through clinical trials, with a particular emphasis on ORLADEYO® (berotralstat) and other candidates like BCX9930 and BCX9250[5]. - The Company’s principal sources of product sales include ORLADEYO, which began shipping in December 2020, and sales of peramivir and RAPIVAB[65]. - The company has not recorded any reserves or allowances for receivables from product sales as of September 30, 2021, indicating a positive outlook on collection[46]. - The company estimates rebates to third-party payors, which are deducted from total gross product revenues, impacting current liabilities[67]. Risks and Challenges - BioCryst incurred losses since inception and expects to continue incurring losses, indicating potential challenges in achieving profitability[9]. - The ongoing COVID-19 pandemic poses risks that could disrupt operations, including delays and increased expenses[9]. - The company relies heavily on third-party relationships for product development and commercialization, and any failure in these relationships could adversely affect business[11]. - The company faces intense competition, which could reduce demand for its products and impact future revenue generation[12]. - The company is subject to various laws and regulations, and non-compliance could result in penalties and harm its reputation[14]. Collaborations and Funding - BioCryst anticipates potential funding from contracts with BARDA/HHS and NIAID/HHS for the development of galidesivir, which could impact financial performance[5]. - The company has collaborations with partners for product commercialization, which are critical for its business model and revenue generation[5]. - The Company received an upfront payment of $22,000 from Torii Pharmaceutical Co., Ltd. for the commercialization of ORLADEYO in Japan, along with a $15,000 milestone payment triggered by regulatory approval[142]. - The Company completed a $30,000 financing transaction to monetize future royalty and milestone payments under the Shionogi Agreement, receiving net proceeds of $22,691 after transaction costs[94]. Stock and Compensation - Stock-based compensation expense for the nine months ended September 30, 2021, was $26,140,000, significantly higher than $8,907,000 in 2020, representing a 194% increase[27]. - The weighted average grant date fair value per share of stock option awards granted during the first nine months of 2021 was $8.11, compared to $3.35 for the same period in 2020[130]. - As of September 30, 2021, the Company had approximately $60,420 of total unrecognized compensation expense related to non-vested stock option awards[126].

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2021 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ______________________ to ______________________ Commission File Number 000-23186 BIOCRYST PHARMACEUTICALS, INC. (Exact name of registrant as specified in its charter) De ...

ORLADEYO Update - ORLADEYO获得了欧盟的批准，并在日本和德国上市[8] - 公司在美国报告了ORLADEYO销售额的第一季度/完整季度收入[8] BCX9930 Update - BCX9930的PNH剂量范围研究已完成[6] - BCX9930作为C5抑制剂疗法反应不足的PNH患者的口服单药治疗的关键试验正在进行中[10] - REDEEM-1试验有90%的把握检测到血红蛋白平均变化量≥ 2 g/dL的差异[12] - REDEEM-2试验有90%的把握检测到血红蛋白平均变化量≥ 2.15 g/dL的差异[14] - 在C5抑制剂初治患者中，所有9名患者继续从BCX9930中获益，在400 mg或500 mg BID BCX9930给药期间，没有发生RBC输血[23] - 在对C5抑制剂反应不足的6名患者中，5名在基线时PNH RBCs上有高水平C3调理素的患者继续从BCX9930中获益[24] Financial Update - 截至2020年12月31日，现金、现金等价物、限制性现金和投资为3.03亿美元[25] - 截至2021年6月30日，现金、现金等价物、限制性现金和投资为2.23亿美元[25] - 公司拥有一笔来自Athyrium Capital Management的高级信贷融资，本金为1.25亿美元，为期5年，仅付息[25] - 公司预计目前的现金流将持续到2023年[25]

Financial Data and Key Metrics Changes - The company reported $50 million in total revenue for Q2 2021, with $28.5 million coming from ORLADEYO net revenue [7][16] - Operating expenses for the quarter, excluding non-cash stock compensation, were $72 million, and the company ended Q2 with $223 million in cash [16][17] - The company expects ORLADEYO sales to reach no less than $100 million for the year, with a potential trajectory to exceed $500 million in global peak sales [9][18] Business Line Data and Key Metrics Changes - ORLADEYO's launch has been successful, with a 50% increase in prescribers in Q2 and 60% of new patients switching from other prophylactic therapies [11][12] - The APeX-2 trial data showed an 80% reduction in attacks for patients on ORLADEYO after 96 weeks, indicating strong efficacy [11][46] - The company is advancing the BCX9930 program into pivotal trials for PNH, with expectations of significant benefits over existing therapies [10][22] Market Data and Key Metrics Changes - The company has made significant progress in payer coverage, with approximately 70% of the HAE market having coverage policies for ORLADEYO [14][33] - Launches of ORLADEYO are underway in Japan and Germany, with plans for additional launches in the UK, France, and other markets over the next 12 months [15][56] Company Strategy and Development Direction - The company aims to continue growing ORLADEYO while pursuing approvals and launches for BCX9930 across various complement-mediated diseases [10][29] - The focus is on developing a pipeline of oral medicines for rare diseases, demonstrating commercial capabilities in this area [10][30] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving at least $100 million in sales for ORLADEYO, despite uncertainties related to patient visits and COVID-19 impacts [18][71] - The company is monitoring the effects of summer vacation schedules and COVID-19 on patient uptake and physician interactions [71][72] Other Important Information - The company has established a strong foundation for the second half of the year, with cash runway extending into 2023 [17][18] - Management highlighted the importance of patient retention and compliance, with current trends aligning with clinical trial data [46][47] Q&A Session Summary Question: Commentary on patient starts and pacing - Management noted strong and consistent new patient starts since launch, with 70% of patients now on reimbursed product [32][33] Question: On-demand versus prophylactic therapy outlook - Management indicated a shift towards prophylactic therapy, with potential market share settling at 80/20 or even 90/10 in favor of prophylactic options [37] Question: Enrollment timing for pivotal trials - Management emphasized the importance of selecting high-quality sites for enrollment in pivotal trials, with enthusiasm from leading hematologists [84] Question: Gross to net adjustments - Management reported improving gross to net adjustments as more patients transition to reimbursed products, expecting to reach 15% to 20% range [42][56] Question: Impact of COVID-19 on patient uptake - Management acknowledged variability in COVID-19 prevalence affecting face-to-face interactions but did not observe direct trends impacting patient uptake [74]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Form 10-K ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2020 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _____ to _____ Commission File Number 000-23186 BIOCRYST PHARMACEUTICALS, INC. (Exact name of registrant as specified in its charter) Delaware (State or other jurisdiction of in ...