RESEARCH TRIANGLE PARK, N.C., Feb. 23, 2024 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced new analyses of real-world use of oral, once-daily ORLADEYO® (berotralstat) that showed patients who initiated ORLADEYO experienced rapid, substantial and sustained reductions in attack rates through 18 months of treatment regardless of the severity of their disease, their history of prior prophylaxis or their C1-inhibitor (C1-INH) level and function. The data are being presented in ...

RESEARCH TRIANGLE PARK, N.C., Feb. 21, 2024 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the company will present at the TD Cowen 44th Annual Health Care Conference in Boston on Wednesday, March 6, 2024, at 10:30 a.m. ET and the Barclays 26th Annual Global Healthcare Conference in Miami on Tuesday, March 12, 2024, at 7:30 a.m. ET. Links to the live audio webcasts and replays of the presentations may be accessed in the Investors & Media section of BioCryst's website ...

RESEARCH TRIANGLE PARK, N.C., Feb. 19, 2024 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the Italian Medicines Agency, Agenzia Italiana del Farmaco (AIFA), has finalized reimbursement and recommended ORLADEYO® (berotralstat) for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in eligible patients 12 years and older. With this recommendation, HAE patients in Italy now have access to the first oral, once-daily therapy for the routine prevent ...

RESEARCH TRIANGLE PARK, N.C., Feb. 12, 2024 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the company will report its fourth quarter 2023 financial results on Monday, February 26, 2024. BioCryst management will host a conference call and webcast at 8:30 a.m. ET that day 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 webca ...

RESEARCH TRIANGLE PARK, N.C. , Feb. 05, 2024 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the company will present five abstracts featuring new analyses of real-world use of oral, once-daily ORLADEYO® (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE) in patients 12 years and older at the 2024 American Academy of Allergy, Asthma & Immunology (AAAAI) annual meeting. The meeting will take place at the Walter E. Washington Convention Center in ...

Core Insights - BioCryst Pharmaceuticals will present five abstracts on the real-world use of ORLADEYO® (berotralstat) for hereditary angioedema (HAE) at the 2024 AAAAI annual meeting [1] - ORLADEYO is the first oral therapy specifically designed to prevent HAE attacks in patients aged 12 and older [2][3] Group 1: Abstract Presentations - The first abstract discusses how berotralstat prophylaxis reduces HAE attack rates regardless of baseline attacks [1] - The second abstract highlights consistently low HAE attack rates with berotralstat, irrespective of prior prophylaxis [1] - The third abstract presents interim analysis from the Berolife Study on the tolerability and effectiveness of berotralstat for long-term prophylaxis [1] - The fourth abstract evaluates adherence to berotralstat through a prospective survey in community pharmacies [1] - The fifth abstract assesses the real-world effectiveness of berotralstat in HAE patients with and without C1-inhibitor deficiency [1] Group 2: Product Information - ORLADEYO is indicated for the prophylaxis of HAE attacks in adults and pediatric patients aged 12 years and older [3] - The drug works by decreasing the activity of plasma kallikrein, preventing HAE attacks with a once-daily capsule [2] - Safety information indicates that the effectiveness of ORLADEYO for acute HAE attacks has not been established, and it should not be used for such treatment [4] Group 3: Safety and Efficacy - Common adverse reactions in patients receiving ORLADEYO include abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease [5] - A reduced dosage of 110 mg is recommended for patients with moderate or severe hepatic impairment [5] - The drug is a moderate inhibitor of CYP2D6 and CYP3A4, necessitating monitoring for concomitant medications [6]

Company Overview - BioCryst Pharmaceuticals is a global biotechnology company focused on improving the lives of individuals with complement-mediated and other rare diseases [3] - The company specializes in structure-guided drug design to develop first-in-class or best-in-class oral small-molecule and protein therapeutics targeting difficult-to-treat diseases [3] - BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies [3] Stock Options and RSUs Grant - BioCryst's board of directors granted stock options to nine newly-hired employees, allowing them to purchase a total of 32,100 shares, along with restricted stock units (RSUs) covering 21,450 shares [1] - The options have an exercise price of $5.30 per share, equal to the closing price of BioCryst common stock on the grant date [2] - The options and RSUs will vest in four equal annual installments starting on the one-year anniversary of the grant date, contingent on the employees' continued service with the company [2]

Part I. Financial Information [Item 1. Financial Statements](index=8&type=section&id=Item%201.%20Financial%20Statements) The unaudited condensed consolidated financial statements for Q3 2023 reflect increased revenues, narrower net loss, and balance sheet changes [Condensed Consolidated Balance Sheets](index=8&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Condensed Consolidated Balance Sheets (in thousands) | Account | Sep 30, 2023 | Dec 31, 2022 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $150,905 | $304,767 | | Investments | $246,685 | $119,543 | | Total current assets | $502,466 | $516,500 | | Total assets | $522,924 | $550,000 | | **Liabilities and Stockholders' Deficit** | | | | Total current liabilities | $90,763 | $105,514 | | Royalty financing obligations | $535,186 | $501,655 | | Secured term loans | $297,995 | $231,624 | | Total stockholders' deficit | ($410,986) | ($294,597) | | Total liabilities and stockholders' deficit | $522,924 | $550,000 | [Condensed Consolidated Statements of Comprehensive Loss](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Comprehensive%20Loss) Condensed Consolidated Statements of Comprehensive Loss (in thousands, except per share data) | Metric | Three Months Ended Sep 30, 2023 | Three Months Ended Sep 30, 2022 | Nine Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2022 | | :--- | :--- | :--- | :--- | :--- | | Revenues | $86,742 | $75,827 | $238,011 | $191,282 | | Total operating expenses | $98,663 | $93,272 | $299,050 | $293,406 | | Loss from operations | ($11,921) | ($17,445) | ($61,039) | ($102,124) | | Net loss | ($36,149) | ($42,520) | ($164,808) | ($175,575) | | Basic and diluted net loss per common share | ($0.19) | ($0.23) | ($0.87) | ($0.95) | [Condensed Consolidated Statements of Cash Flows](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Condensed Consolidated Statements of Cash Flows (in thousands) | Cash Flow Activity | Nine Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | ($86,223) | ($138,001) | | Net cash used in investing activities | ($103,025) | ($205,453) | | Net cash provided by financing activities | $35,658 | $83,828 | | Decrease in cash, cash equivalents and restricted cash | ($153,750) | ($259,391) | | Cash, cash equivalents and restricted cash at end of period | $152,489 | $248,343 | [Notes to Condensed Consolidated Financial Statements](index=13&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) - The company believes its financial resources as of September 30, 2023, are **sufficient to fund operations for at least the next 12 months**, despite expecting 2023 expenses to exceed revenues[43](index=43&type=chunk) Revenue Breakdown (in thousands) | Revenue Source | Three Months Ended Sep 30, 2023 | Three Months Ended Sep 30, 2022 | Nine Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2022 | | :--- | :--- | :--- | :--- | :--- | | ORLADEYO (U.S.) | $75,268 | $60,106 | $208,934 | $162,743 | | ORLADEYO (Outside U.S.) | $10,416 | $5,866 | $26,173 | $18,155 | | **Total ORLADEYO** | **$85,684** | **$65,972** | **$235,107** | **$180,898** | | Other revenues | $1,058 | $9,855 | $2,904 | $10,384 | | **Total revenues** | **$86,742** | **$75,827** | **$238,011** | **$191,282** | - On April 17, 2023, the company entered into a new **$450 million Loan Agreement** with Pharmakon, drawing an initial **$300 million**; proceeds were used to repay the existing **$241.8 million Athyrium Credit Agreement**[133](index=133&type=chunk)[223](index=223&type=chunk) - Upon repayment of the Athyrium Term Loans, the company incurred a **one-time loss on extinguishment of debt of $29.0 million** for the nine months ended September 30, 2023, which included prepayment fees and the write-off of unamortized financing costs[150](index=150&type=chunk) - Subsequent to the quarter end, on October 23, 2023, Baker Bros. Advisors LP exercised all remaining pre-funded warrants, resulting in the issuance of **14,997 common shares**; **no warrants remain outstanding**[182](index=182&type=chunk) - On November 3, 2023, the company entered into a license agreement with Clearside Biomedical, Inc. to develop avoralstat for diabetic macular edema, involving a **$5 million upfront fee** and potential future milestones and royalties[182](index=182&type=chunk)[183](index=183&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=37&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses rare disease strategy, ORLADEYO commercialization, financial results, and pipeline advancements [Overview and Recent Developments](index=37&type=section&id=Overview%20and%20Recent%20Developments) - The company's strategy focuses on developing and commercializing **first-in-class or best-in-class oral therapeutics** for rare diseases, with a key product being **ORLADEYO for hereditary angioedema (HAE)**[186](index=186&type=chunk)[187](index=187&type=chunk) - BioCryst anticipates the global commercial market for ORLADEYO has the potential to reach a peak of **$1 billion in annual net revenues**, with **70-80% expected from the U.S.**[188](index=188&type=chunk) - The company is advancing its complement program, including **BCX10013 for renal complement-mediated diseases**, and has introduced **several new pipeline candidates**[190](index=190&type=chunk)[192](index=192&type=chunk) - **Oral C5 Inhibitor:** For generalized myasthenia gravis (gMG), with clinical trials expected to begin in 2025 - **Bifunctional Complement Inhibitor:** An anti-C2 monoclonal antibody for complex renal diseases, with clinical trials expected in 2025 - **Oral C2 Inhibitor:** For autoimmune hemolytic anemias, with a lead molecule selection expected in 2025 - **BCX17725:** A KLK5 inhibitor for Netherton syndrome, with clinical trials expected to begin in 2024 - **Avoralstat:** Licensed with Clearside's microinjector for diabetic macular edema (DME)[200](index=200&type=chunk)[201](index=201&type=chunk)[202](index=202&type=chunk)[203](index=203&type=chunk)[204](index=204&type=chunk) [Results of Operations](index=41&type=section&id=Results%20of%20Operations) Comparison of Results of Operations (in millions) | Metric | Q3 2023 | Q3 2022 | Change | 9M 2023 | 9M 2022 | Change | | :--- | :--- | :--- | :--- | :--- | :--- | :--- | | Total Revenues | $86.7 | $75.8 | +14.4% | $238.0 | $191.3 | +24.4% | | R&D Expenses | $46.9 | $52.7 | -11.0% | $146.5 | $180.1 | -18.7% | | SG&A Expenses | $50.6 | $36.9 | +37.1% | $149.5 | $109.2 | +36.9% | | Interest Expense | $27.3 | $24.8 | +10.1% | $83.7 | $72.6 | +15.3% | - The increase in Q3 and 9-month 2023 revenue was primarily driven by higher ORLADEYO net revenue, which grew by **$19.7 million and $54.2 million** respectively, partially offset by a decrease in other revenues due to a 2022 RAPIVAB stockpiling sale to HHS[207](index=207&type=chunk)[214](index=214&type=chunk) - **R&D expenses decreased** in 2023 due to reduced investment following the discontinuation of the BCX9930 (Factor D) and BCX9250 (FOP) programs in late 2022[209](index=209&type=chunk)[216](index=216&type=chunk) - **SG&A expenses increased significantly** due to investment in expanding the U.S. commercial team for ORLADEYO and growing international operations[210](index=210&type=chunk)[217](index=217&type=chunk) [Liquidity and Capital Resources](index=43&type=section&id=Liquidity%20and%20Capital%20Resources) - As of September 30, 2023, the company had principal liquidity sources of approximately **$150.9 million in cash and cash equivalents** and **$246.7 million in available-for-sale investments**[226](index=226&type=chunk) - On April 17, 2023, the company entered into a new **$450 million loan agreement** with Pharmakon, drawing an initial **$300 million** to repay its previous Athyrium credit facility and for general corporate purposes; **three additional tranches of $50 million each** are available at the company's option through September 30, 2024[223](index=223&type=chunk) - Management believes that based on current revenue and expense expectations, the company's financial resources are **sufficient to fund operations for at least the next 12 months**[230](index=230&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=50&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company faces interest rate and foreign currency risks, with no material impact from inflation on results - The company is subject to **interest rate risk** from its Pharmakon Loan Agreement, which bears interest at a **variable rate** based on the three-month SOFR plus a margin; as of September 30, 2023, the **effective interest rate was 13.24%**[267](index=267&type=chunk) - **Foreign currency risk** exists due to commercial sales in Europe (Euros, British Pound) and royalties from Japan (Japanese yen), as well as operations in other regions; the company **does not currently engage in foreign currency hedging**[272](index=272&type=chunk)[273](index=273&type=chunk) [Controls and Procedures](index=51&type=section&id=Item%204.%20Controls%20and%20Procedures) Disclosure controls were ineffective due to material weaknesses in internal financial reporting from staffing issues; remediation is underway - The CEO and CFO concluded that disclosure controls and procedures were **not effective** as of September 30, 2023, due to **material weaknesses in internal control over financial reporting**[276](index=276&type=chunk) - The material weaknesses stem from **staffing issues and insufficient resources** in the accounting function, leading to **inadequate review** of manual journal entries and general ledger account reconciliations[276](index=276&type=chunk)[277](index=277&type=chunk) - Planned remediation includes **hiring more accounting personnel**, **engaging a third-party specialist**, and **updating policies, procedures, and training** related to internal controls[278](index=278&type=chunk) - Despite the material weaknesses, management believes the condensed consolidated financial statements in the report are **fairly presented in all material respects in conformity with U.S. GAAP**[280](index=280&type=chunk) Part II. Other Information [Risk Factors](index=53&type=section&id=Item%201A.%20Risk%20Factors) The company outlines significant business, financial, development, regulatory, and market risks impacting its operations - **Business Risks:** The company has a **history of losses** and may **need to raise additional capital**; its success depends on **advancing its pipeline**, particularly **BCX10013**, and the **commercial success of ORLADEYO**, which faces **intense competition**[290](index=290&type=chunk)[292](index=292&type=chunk)[296](index=296&type=chunk)[339](index=339&type=chunk) - **Financial & Control Risks:** The company has identified **material weaknesses in its internal control over financial reporting**, which could **harm investor confidence and the stock price**; the business is also subject to **significant indebtedness** under the Pharmakon Loan Agreement, which contains **restrictive covenants**[414](index=414&type=chunk)[389](index=389&type=chunk) - **Development & Regulatory Risks:** Drug development is **complex, lengthy, and expensive**, with a **high risk of failure**; the company **relies heavily on third parties** for manufacturing and clinical trials, and obtaining and maintaining regulatory approvals is **uncertain and subject to extensive requirements**[298](index=298&type=chunk)[305](index=305&type=chunk)[312](index=312&type=chunk) - **International & Market Risks:** International expansion exposes the company to **varied legal, political, and financial risks**, including **foreign currency fluctuations** and **complex reimbursement environments**; **unpredictable market and economic conditions**, such as **inflation, rising interest rates, and geopolitical conflicts**, could adversely affect operations and access to capital[396](index=396&type=chunk)[398](index=398&type=chunk)[431](index=431&type=chunk) [Other Information](index=80&type=section&id=Item%205.%20Other%20Information) During Q3 2023, no director or officer adopted or terminated Rule 10b5-1 or non-Rule 10b5-1 trading arrangements - During the three months ended September 30, 2023, **no director or officer adopted or terminated a Rule 10b5-1 trading arrangement or a non-Rule 10b5-1 trading arrangement**[437](index=437&type=chunk) [Exhibits](index=81&type=section&id=Item%206.%20Exhibits) This section provides a comprehensive list of all exhibits filed with the Form 10-Q

BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) Q3 2023 Earnings Conference Call November 2, 2023 8:30 AM ET Company Participants John Bluth - Investor Relations Jon Stonehouse - Chief Executive Officer Charlie Gayer - Chief Commercial Officer Jinky Rosselli - Chief Data & Insights Officer Anthony Doyle - Chief Financial Officer Conference Call Participants Tazeen Ahmad - Bank of America Stacy Ku - Cowen & Co. Liisa Bayko - Evercore ISI Serge Belanger - Needham & Company Operator Good morning and welcome to th ...

Table of Contents Title of each class Trading Symbol(s) Name of each exchange on which registered Common Stock BCRX Nasdaq Global Select Market UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q x QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2023 or o TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ______________________ to ...