BCRX BioCryst Pharmaceuticals Inc.

13.12
+0.2  (+2%)
Previous Close 12.92
Open 12.69
52 Week Low 3.3
52 Week High 14.24
Market Cap $2,330,742,547
Shares 177,648,060
Float 157,863,149
Enterprise Value $1,876,417,280
Volume 6,449,387
Av. Daily Volume 5,427,907
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Drug Pipeline

Drug Stage Notes
BCX9930
Paroxysmal nocturnal hemoglobinuria (PNH)
Phase 1/2
Phase 1/2
Pivotal trials to commence 2H 2021.
ORLADEYO (Berotralstat)
Reduce or eliminate attacks in HAE patients
Approved
Approved
FDA approval announced December 3, 2020
Galidesivir
COVID-19
Phase 1
Phase 1
Data from Part 1 of the trial released December 22, 2020 - safe and generally well tolerated but efficacy not shown. Development for COVID-19 to be discontinued.
BCX9250
Fibrodysplasia ossificans progressiva (FOP)
Phase 1
Phase 1
Phase 1 data released December 21, 2020 - safe and well tolerated.
Galidesivir
Healthy volunteers
Phase 1
Phase 1
Phase 1 trial completion announced May 9, 2019. Generally safe and well tolerated.
BCX4161 - OPuS-2
Hereditary angioedema
Phase 2b
Phase 2b
Phase 2b data released February 2016 - primary endpoint not met
RAPIVAB (peramivir)
INFLUENZA ACUTE
Approved
Approved
Approved Dec 22 2014
RAPIVAB (peramivir injection)
Acute uncomplicated influenza to pediatric patients
Approved
Approved
Approved September 21, 2017.

Latest News

  1. —Q1 2021 ORLADEYO(berotralstat) net revenue of $10.9 million—

    —ORLADEYO now approved in U.S., Japan and EU—

    —BioCryst reaches agreement with FDA that change from baseline in hemoglobin is the primary endpoint for pivotal PNH trials of oral BCX9930 set to begin in second half of 2021—

    RESEARCH TRIANGLE PARK, N.C., May 06, 2021 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced financial results for the first quarter ended March 31, 2021, and provided a corporate update.

    "Our commercial team is off to an outstanding start with the U.S. launch of ORLADEYO.  In this highly competitive market, we are demonstrating what we have known for some time now, HAE patients have been waiting to switch to an oral, once-daily…

    —Q1 2021 ORLADEYO (berotralstat) net revenue of $10.9 million—

    —ORLADEYO now approved in U.S., Japan and EU—

    —BioCryst reaches agreement with FDA that change from baseline in hemoglobin is the primary endpoint for pivotal PNH trials of oral BCX9930 set to begin in second half of 2021—

    RESEARCH TRIANGLE PARK, N.C., May 06, 2021 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced financial results for the first quarter ended March 31, 2021, and provided a corporate update.

    "Our commercial team is off to an outstanding start with the U.S. launch of ORLADEYO.  In this highly competitive market, we are demonstrating what we have known for some time now, HAE patients have been waiting to switch to an oral, once-daily therapy to reduce their attacks and burden of therapy," said Jon Stonehouse, president and chief executive officer of BioCryst.

    "Our early launch performance is the latest piece of evidence that BioCryst's differentiated strategy to discover, develop and, now, successfully commercialize unique oral medicines for rare diseases has the potential to create greater and greater value. We are doing this first in HAE and will next apply what we have learned to patients suffering from complement-mediated diseases," Stonehouse added.

    Program Updates and Key Milestones

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

    • ORLADEYO net revenue in the first quarter of 2021, the first full quarter of launch in the United States, was $10.9 million.



    • The majority of ORLADEYO revenue in the first quarter of 2021 came from new patients who switched to ORLADEYO from either injectable/infused prophylactic medications or from acute-only treatment. The remainder came from patients transitioning from clinical trials and the company's early access program.

    European Approvals and Launches

    • On April 30, 2021, the company announced that the European Commission (EC) had approved oral, once-daily ORLADEYO for the prevention of recurrent hereditary angioedema (HAE) attacks in HAE patients 12 years and older. The EC approval of ORLADEYO is applicable to all European Union member states plus Iceland, Norway and Liechtenstein.



    • BioCryst has its European commercial team in place and expects to launch ORLADEYO in the second quarter in Germany, with launches in other European markets to follow. HAE patients in France currently have access to ORLADEYO through an Autorisation Temporaire d'Utilisation de cohorte (cohort ATU).



    • On March 2, 2021, the company announced the submission of a marketing authorization application (MAA) to the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA) seeking approval of ORLADEYO for the prevention of recurrent HAE attacks in HAE patients 12 years and older. The MAA was submitted under the MHRA's new European Commission Decision Reliance Procedure. If approved, ORLADEYO would be the first oral, once-daily therapy in the United Kingdom to treat patients with HAE.

    Japanese Approval and Launch

    • On January 22, 2021, the company announced that the Ministry of Health, Labor and Welfare (MHLW) in Japan had granted marketing and manufacturing approval for oral, once-daily ORLADEYO 150 mg for prophylactic treatment of HAE in adults and pediatric patients 12 years and older.



    • On April 14, 2021, the company announced that the Japanese National Health Insurance System (NHI) approved the addition of oral, once-daily ORLADEYO™ (berotralstat) to the NHI drug price list on April 21, 2021. This triggered a $15 million milestone payment to BioCryst from Torii Pharmaceutical Co., Ltd., the company's commercial partner in Japan.



    • ORLADEYO is the first and only prophylactic HAE medication approved in Japan. Torii launched ORLADEYO in Japan on April 23, 2021. BioCryst will receive tiered royalties ranging from 20 percent to 40 percent of Japanese net sales.

    Complement Oral Factor D Inhibitor Program – BCX9930

    • BioCryst has reached agreement with the U.S. Food and Drug Administration (FDA) that the primary endpoint for the upcoming pivotal trials in paroxysmal nocturnal hemoglobinuria (PNH) is change from baseline in hemoglobin. On March 22, 2021 the company announced that BCX9930 increased hemoglobin from baseline by a mean of 3.3 g/dL in C5 inadequate response (no prior treatment with C5 inhibitors) patients and 3.5 g/dL in treatment-naïve patients and reduced transfusions in an ongoing dose-ranging trial in PNH patients. BCX9930 was safe and generally well-tolerated in the trial.



    • In the second half of 2021, the company plans to advance directly into PNH pivotal trials with oral BCX9930, at a dose of 500 mg bid, in patients naïve to C5 inhibitors, and patients with an inadequate response to C5 inhibitors. The goal of the pivotal trials is to achieve a broad indication for BCX9930 to treat PNH as oral monotherapy. Also in the second half of 2021, the company plans to initiate a proof of concept trial of oral BCX9930 (500 mg bid) in renal complement-mediated diseases.

    Additional Updates

    • On February 3, 2021, the company announced that the FDA had approved a supplemental new drug application for RAPIVAB® (peramivir injection) expanding the patient population of RAPIVAB for the treatment of acute uncomplicated influenza to include patients six months and older who have been symptomatic for no more than two days. Prior to this approval, RAPIVAB had been indicated for patients two years and older.



    • On March 19, 2021, the company announced the appointment of Helen Thackray, M.D., FAAP, to the newly created position of chief research and development officer.

    First Quarter 2021 Financial Results

    For the three months ended March 31, 2021, total revenues were $19.1 million, compared to $4.8 million in the first quarter of 2020. The increase was primarily due to $10.9 million in ORLADEYO net revenue in the first quarter of 2021.

    Research and development (R&D) expenses for the first quarter of 2021 increased to $42.4 million from $29.9 million in the first quarter of 2020, primarily due to increased investment in the development of BCX9930  as well as other research, preclinical and development costs, offset by a reduction in spend on the ORLADEYO program following our commercial launch in December 2020.

    Selling, general and administrative (SG&A) expenses for the first quarter of 2021 increased to $22.1 million, compared to $15.9 million in the first quarter of 2020. The increase was primarily due to increased investment in to support the U.S. commercial launch of ORLADEYO and expanded international operations.

    Interest and other income in the first quarter of 2021 was $6.4 million lower than the first quarter of 2020, primarily due to the partial arbitration award in the first quarter of 2020 related to our Seqirus dispute.

    Interest expense was $12.9 million in the first quarter of 2021, compared to $3.0 million in the first quarter of 2020. The increase was due to service on the royalty and debt financings which were completed in December 2020.

    Net loss for the first quarter of 2021 was $64.3 million, or $0.36 per share, compared to a net loss of $37.6 million, or $0.24 per share, for the first quarter of 2020.

    Cash, cash equivalents, restricted cash and investments totaled $244.4 million at March 31, 2021, compared to $114.6 million at March 31, 2020. Operating cash use for the first quarter of 2021 was $60.0 million.

    Financial Outlook for 2021

    In the launch period for ORLADEYO, the company is not providing specific revenue or operating expense guidance. Based on our expectations for revenue, operating expenses, and our option to access an additional $75 million from our existing credit facility, we believe our current cash runway takes us into 2023.  

    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 877-303-8027 for domestic callers and 760-536-5165 for international callers and using conference ID # 2660434. A live webcast of the call and any slides will be available online at the investors section of the company website at www.biocryst.com. A telephone replay of the call will be available by dialing 855-859-2056 for domestic callers or 404-537-3406 for international callers and entering the conference ID # 2660434.

    About BioCryst Pharmaceuticals

    BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. Oral, once-daily ORLADEYO™ (berotralstat) is approved in the United States, European Union, and Japan for the prevention of HAE attacks in adults and pediatric patients 12 years and older, and under regulatory review for approval in the United Kingdom. BioCryst has several ongoing development programs including BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva, and galidesivir, a potential treatment for Marburg virus disease and Yellow Fever. RAPIVAB® (peramivir injection), a viral neuraminidase inhibitor for the treatment of influenza, has received regulatory approval in the U.S., Canada, Australia, Japan, Taiwan and Korea. Post-marketing commitments for RAPIVAB are ongoing. For more information, please visit the company's website at www.biocryst.com.

    Forward-Looking Statements

    This 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: the ongoing COVID-19 pandemic, which could create challenges in all aspects of BioCryst's business, including without limitation delays, stoppages, difficulties and increased expenses with respect to BioCryst's and its partners' development, regulatory processes and supply chains, negatively impact BioCryst's ability to access the capital or credit markets to finance its operations, or have the effect of heightening many of the risks described below or in the documents BioCryst files periodically with the Securities and Exchange Commission; BioCryst's ability to successfully implement its commercialization plans for, and to commercialize ORLADEYO, which could take longer or be more expensive than planned; the results of BioCryst's partnerships with third parties, including Torii, 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 market acceptance, which could also impact the amount of any related royalties BioCryst would be entitled to receive from Torii; ongoing and future preclinical and clinical development of BCX9930, BCX9250 and galidesivir may not have positive 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, EMA, PMDA 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 manage its growth and compete effectively; risks related to the international expansion of BioCryst's business; and actual financial results may not be consistent with expectations, including that 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, all of which identify important factors that could cause the actual results to differ materially from those contained in BioCryst's projections and forward-looking statements.

    BCRXW

    Investors:

    John Bluth

    +1 919 859 7910

    Media:

    Catherine Collier Kyroulis

    +1 917 886 5586



    BIOCRYST PHARMACEUTICALS, INC.   
    CONSOLIDATED FINANCIAL SUMMARY    
      (in thousands, except per share)   
              
    Statements of Operations (Unaudited)         
              
      Three Months Ended    
      March 31,    
      2021    2020  
    Revenues:         
    Product sales$17,871   $218  
    Royalty revenue (897)   1,945  
    Collaborative and other research and development 2,085    2,660  
    Total revenues 19,059    4,823  
              
    Expenses:    
    Cost of product sales 5,923    -  
    Research and development 42,435    29,867  
    Selling, general and administrative 22,114    15,865  
    Royalty (36)   69  
    Total operating expenses 70,436    45,801  
              
    Loss from operations (51,377)   (40,978) 
              
    Interest and other income 26    6,446  
    Interest expense (12,904)   (3,047) 
    Loss on foreign currency (29)   (20) 
              
    Net loss$(64,284)  $(37,599) 
              
    Basic and diluted net loss per common share$(0.36)  $(0.24) 
              
    Weighted average shares outstanding 177,343    154,156  
              
              



    Balance Sheet Data (in thousands)           
     March 31, 2021 December 31, 2020
     (Unaudited) (Note 1)
    Cash, cash equivalents and investments $   240,356   $   300,366 
    Restricted cash 4,008   2,221 
    Receivables 18,386   8,646 
    Total assets 284,431   334,715 
    Non-recourse notes payable 30,000   30,000 
    Secured term loan 123,039   119,735 
    Royalty financing obligation 131,296   124,717 
    Accumulated deficit  (1,087,726)   (1,023,442)
    Stockholders' deficit  (74,988)   (19,262)
    Shares of common stock outstanding 177,670   176,883 
                
    Note 1:  Derived from audited financial statements.           
                





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  2. RESEARCH TRIANGLE PARK, N.C., May 05, 2021 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced that the company will present at the Bank of America 2021 Healthcare Conference on Wednesday, May 12, 2021 at 12:30 p.m. ET and the 2021 RBC Capital Markets Global Healthcare Conference on Wednesday, May 19, 2021 at 1:20 p.m. ET. Both are being conducted as virtual conferences.

    Links to a live audio webcast and replay of these presentations may be accessed in the Investors section of BioCryst's website at http://www.biocryst.com.

    About BioCryst Pharmaceuticals

    BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme…

    RESEARCH TRIANGLE PARK, N.C., May 05, 2021 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced that the company will present at the Bank of America 2021 Healthcare Conference on Wednesday, May 12, 2021 at 12:30 p.m. ET and the 2021 RBC Capital Markets Global Healthcare Conference on Wednesday, May 19, 2021 at 1:20 p.m. ET. Both are being conducted as virtual conferences.

    Links to a live audio webcast and replay of these presentations may be accessed in the Investors section of BioCryst's website at http://www.biocryst.com.

    About BioCryst Pharmaceuticals

    BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. Oral, once-daily ORLADEYO™ (berotralstat) is approved in the United States, European Union,   and Japan for the prevention of HAE attacks in adults and pediatric patients 12 years and older, and under regulatory review for approval in the United Kingdom. BioCryst has several ongoing development programs including BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva, and galidesivir, a potential treatment for Marburg virus disease and Yellow Fever. RAPIVAB® (peramivir injection), a viral neuraminidase inhibitor for the treatment of influenza, has received regulatory approval in the U.S., Canada, Australia, Japan, Taiwan and Korea. Post-marketing commitments for RAPIVAB are ongoing. For more information, please visit the company's website at www.biocryst.com

    BCRXW

    Investors:

    John Bluth

    +1 919 859 7910

    Media:

    Catherine Collier Kyroulis

    +1 917 886 5586



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  3. RESEARCH TRIANGLE PARK, N.C., April 30, 2021 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced that the European Commission (EC) has approved oral, once-daily ORLADEYO (berotralstat) for the prevention of recurrent hereditary angioedema (HAE) attacks in HAE patients 12 years and older.

    "As the first targeted oral prophylactic therapy approved in Europe, ORLADEYO represents a major advance in treatment for HAE patients who have been waiting for a preventive therapy. Physicians will be delighted to discuss this new option with their patients," said Emel Aygören-Pürsün, M.D., head of the HAE Center at the University Hospital in Frankfurt.

    "ORLADEYO offers people with HAE in Europe and their physicians the first…

    RESEARCH TRIANGLE PARK, N.C., April 30, 2021 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced that the European Commission (EC) has approved oral, once-daily ORLADEYO (berotralstat) for the prevention of recurrent hereditary angioedema (HAE) attacks in HAE patients 12 years and older.

    "As the first targeted oral prophylactic therapy approved in Europe, ORLADEYO represents a major advance in treatment for HAE patients who have been waiting for a preventive therapy. Physicians will be delighted to discuss this new option with their patients," said Emel Aygören-Pürsün, M.D., head of the HAE Center at the University Hospital in Frankfurt.

    "ORLADEYO offers people with HAE in Europe and their physicians the first orally administered non-steroidal option for preventing HAE attacks and represents a vitally important and most welcome step in making more treatment options available," said Henrik Balle Boysen, executive vice president and chief operating officer of HAE International, a global non-profit network of patient associations dedicated to improving the lives of people with HAE.

    The EC approval of ORLADEYO is applicable to all European Union member states plus Iceland, Norway and Liechtenstein.

    BioCryst has its European commercial team in place and expects to launch ORLADEYO this quarter in Germany, with launches in other European markets to follow. HAE patients in France currently have access to ORLADEYO through an Autorisation Temporaire d'Utilisation de cohorte (cohort ATU).

    In the United Kingdom, HAE patients also currently have access to ORLADEYO through an approved early access to medicines scheme (EAMS). A marketing authorization application (MAA) has been submitted to the Medicines and Healthcare products Regulatory Agency (MHRA). Under the new European Commission Decision Reliance Procedure, the MHRA will aim to complete the review of the UK MAA as soon as possible following the EC approval decision.

    "Most European HAE patients today treat their disease with on-demand therapy or androgens and we believe the approval of oral, once-daily ORLADEYO provides an exciting new opportunity for these patients to reduce their burden of therapy by moving to prophylaxis with ORLADEYO," said Jon Stonehouse, president and chief executive officer of BioCryst.

    "We saw tremendous enthusiasm and participation from European HAE patients in our clinical trials and we have invested in an experienced European commercial team that is excited to bring ORLADEYO to HAE patients across Europe," he added.

    In the pivotal Phase 3 APeX-2 trial, ORLADEYO significantly reduced attacks at 24 weeks, and this reduction was sustained through 48 weeks. HAE patients who completed 48 weeks of treatment (150 mg) saw reductions in their HAE attack rates, from a mean of 2.9 attacks per month at baseline to a mean of 1.0 attacks per month after 48 weeks of therapy. In the long-term open label APeX-S trial, patients completing 48 weeks of therapy (150 mg) had a mean attack rate of 0.8 attacks per month.

    ORLADEYO was safe and well tolerated in both trials. The most frequently reported adverse reactions in patients receiving ORLADEYO compared with placebo were gastrointestinal reactions. These reactions generally occurred early after initiation of treatment with ORLADEYO, became less frequent with time and typically self-resolved.

    HAE patients note a significant treatment burden associated with existing prophylactic therapy. In addition to reducing HAE attack rate, data from APeX-2 show that patients reported meaningful improvements in both quality of life, overall patient-reported satisfaction, and significant reductions in their monthly use of standard of care on-demand medicine, while taking oral, once-daily ORLADEYO (150 mg). 1,2

    About ORLADEYO (berotralstat)

    ORLADEYO (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years and older.  One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein.

    The full European Summary of Product Characteristics (SMPC) for ORLADEYO will be available from the European Medicines Association website at www.ema.europa.eu.

    About BioCryst Pharmaceuticals

    BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. Oral, once-daily ORLADEYO™ (berotralstat) is approved in the United States, European Union and Japan for the prevention of HAE attacks in adults and pediatric patients 12 years and older, and under regulatory review for approval in the United Kingdom. BioCryst has several ongoing development programs including BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva, and galidesivir, a potential treatment for Marburg virus disease and Yellow Fever. RAPIVAB® (peramivir injection), a viral neuraminidase inhibitor for the treatment of influenza, has received regulatory approval in the U.S., Canada, Australia, Japan, Taiwan and Korea. Post-marketing commitments for RAPIVAB are ongoing. For more information, please visit the company's website at www.biocryst.com.

    Forward-Looking Statements

    This press release contains forward-looking statements, including statements regarding BioCryst's plans and expectations for ORLADEYO.  These statements involve known and unknown risks, uncertainties and other factors which may cause 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: the ongoing COVID-19 pandemic, which could create challenges in all aspects of BioCryst's business, including without limitation delays, stoppages, difficulties and increased expenses with respect to BioCryst's and its partners' development, regulatory processes and supply chains, negatively impact BioCryst's ability to access the capital or credit markets to finance its operations, or have the effect of heightening many of the risks described below or in the documents BioCryst periodically files with the Securities and Exchange Commission; BioCryst's ability to successfully implement its commercialization plans for, and to commercialize, ORLADEYO, which could take longer or be more expensive than planned; risks relating to government actions, including that decisions and other actions relating to pricing and reimbursements 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 market acceptance; the FDA, EMA, PMDA 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; BioCryst's ability to successfully manage its growth and compete effectively; risks related to the international expansion of BioCryst's business; and actual financial results may not be consistent with expectations, including that 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, all of which identify important factors that could cause the actual results to differ materially from those contained in BioCryst's forward-looking statements.

    BCRXW

    Contact:

    Investors

    John Bluth

    +1 919 859 7910

    Media

    Catherine Collier Kyroulis

    +1 917 886 5586

    1 Johnston, D.T., et al. Berotralstat Improves Patient-Reported Quality of Life Through 48 Weeks in the Phase 3 APeX-2 Trial; Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology; Poster 154, Nov (2020)

    2 Jacobs, J, et al. Berotralstat Positively Impacts Patient-Reported Satisfaction: Results from the Phase 3 APeX-2 trial; Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology; Poster 158, Nov (2020)



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  4. RESEARCH TRIANGLE PARK, N.C., April 22, 2021 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced that the company will report its first quarter 2021 financial results on Thursday, May 6, 2021.

    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 877-303-8027 for domestic callers and 760-536-5165 for international callers and using conference ID # 2660434. A live webcast of the call and any slides will be available online at the investors section of the company website at www.biocryst.com. A telephone replay of the call will be available by dialing 855-859-2056 for domestic…

    RESEARCH TRIANGLE PARK, N.C., April 22, 2021 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced that the company will report its first quarter 2021 financial results on Thursday, May 6, 2021.

    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 877-303-8027 for domestic callers and 760-536-5165 for international callers and using conference ID # 2660434. A live webcast of the call and any slides will be available online at the investors section of the company website at www.biocryst.com. A telephone replay of the call will be available by dialing 855-859-2056 for domestic callers or 404-537-3406 for international callers and entering the conference ID # 2660434.

    About BioCryst Pharmaceuticals

    BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. Oral, once-daily ORLADEYO™ (berotralstat) is approved in the United States and Japan for the prevention of HAE attacks in adults and pediatric patients 12 years and older, and under regulatory review for approval in the European Union and United Kingdom. BioCryst has several ongoing development programs including BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva, and galidesivir, a potential treatment for Marburg virus disease and Yellow Fever. RAPIVAB® (peramivir injection), a viral neuraminidase inhibitor for the treatment of influenza, has received regulatory approval in the U.S., Canada, Australia, Japan, Taiwan and Korea. Post-marketing commitments for RAPIVAB are ongoing. For more information, please visit the company's website at www.biocryst.com.

    BCRXW

    Contact:

    John Bluth

    +1 919 859 7910



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  5. —ORLADEYO is the first and only approved prophylactic therapy for HAE in Japan—

    —NHI price listing triggers $15 million milestone payment to BioCryst—   

    RESEARCH TRIANGLE PARK, N.C., April 14, 2021 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced that the Japanese National Health Insurance System (NHI) has approved the addition of oral, once-daily ORLADEYO(berotralstat) to the NHI drug price list on April 21, 2021.  

    Oral, once-daily ORLADEYO was approved in Japan in January 2021 for prophylactic treatment of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older. ORLADEYO is the first and only prophylactic HAE medication approved in Japan.

    ORLADEYO will be commercialized in Japan…

    —ORLADEYO is the first and only approved prophylactic therapy for HAE in Japan—

    —NHI price listing triggers $15 million milestone payment to BioCryst—   

    RESEARCH TRIANGLE PARK, N.C., April 14, 2021 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced that the Japanese National Health Insurance System (NHI) has approved the addition of oral, once-daily ORLADEYO(berotralstat) to the NHI drug price list on April 21, 2021.  

    Oral, once-daily ORLADEYO was approved in Japan in January 2021 for prophylactic treatment of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older. ORLADEYO is the first and only prophylactic HAE medication approved in Japan.

    ORLADEYO will be commercialized in Japan by BioCryst's partner, Torii Pharmaceutical Co., Ltd. Torii plans to launch ORLADEYO following the NHI drug price listing.

    "Our goal is to bring ORLADEYO to HAE patients around the world who want a new oral, once-daily option to prevent their attacks. In Japan, ORLADEYO is the first approved prophylactic HAE medication, which has the potential to significantly impact the lives of HAE patients," said Jon Stonehouse, president and chief executive officer of BioCryst.  

    The NHI price listing triggers a $15 million milestone payment from Torii to BioCryst. In addition, BioCryst will receive tiered royalties ranging from 20 percent to 40 percent of Japanese net sales.

    BioCryst received Orphan Drug and Sakigake designation for ORLADEYO in Japan. The APeX-J trial in Japan met its primary endpoint (p=0.003) of a reduction in HAE attacks from baseline for ORLADEYO 150 mg compared to placebo, and ORLADEYO was safe and generally well-tolerated in the trial. In APeX-2, ORLADEYO also met its primary endpoint (p<0.001) for ORLADEYO 150 mg compared to placebo and was safe and generally well-tolerated.

    About ORLADEYO™ (berotralstat)

    ORLADEYO™ (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein.

    U.S. Indication and Important Safety Information

    INDICATION

    ORLADEYO™ (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.

    Limitations of use

    The safety and effectiveness of ORLADEYO for the treatment of acute HAE attacks have not been established. ORLADEYO should not be used for the treatment of acute HAE attacks. Additional doses or dosages of ORLADEYO higher than 150 mg once daily are not recommended due to the potential for QT prolongation.

    IMPORTANT SAFETY INFORMATION

    An increase in QT prolongation was observed at dosages higher than the recommended 150 mg once-daily dosage and was concentration dependent.

    The most common adverse reactions (≥10% and higher than placebo) in patients receiving ORLADEYO were abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease.

    A reduced dosage of 110 mg taken orally once daily with food is recommended in patients with moderate or severe hepatic impairment (Child-Pugh B or C) and in patients taking chronically administered P-glycoprotein (P-gp) or breast cancer resistance protein (BCRP) inhibitors (eg, cyclosporine).

    Berotralstat is a substrate of P-gp and BCRP. P-gp inducers (eg, rifampin, St. John's wort) may decrease berotralstat plasma concentration, leading to reduced efficacy of ORLADEYO. The use of P-gp inducers is not recommended with ORLADEYO.

    ORLADEYO at a dose of 150 mg is a moderate inhibitor of CYP2D6 and CYP3A4. For concomitant medications with a narrow therapeutic index that are predominantly metabolized by CYP2D6 or CYP3A4, appropriate monitoring and dose titration is recommended. ORLADEYO at a dose of 300 mg is a P-gp inhibitor. Appropriate monitoring and dose titration is recommended for P-gp substrates (eg, digoxin) when coadministering with ORLADEYO.

    The safety and effectiveness of ORLADEYO in pediatric patients <12 years of age have not been established.

    There are insufficient data available to inform drug-related risks with ORLADEYO use in pregnancy. There are no data on the presence of berotralstat in human milk, its effects on the breastfed infant, or its effects on milk production.

    To report SUSPECTED ADVERSE REACTIONS, contact BioCryst Pharmaceuticals, Inc. at 1-833-633-2279 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

    Please see full Prescribing Information.

    About BioCryst Pharmaceuticals

    BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. Oral, once-daily ORLADEYO™ (berotralstat) is approved in the United States and Japan for the prevention of HAE attacks in adults and pediatric patients 12 years and older, and under regulatory review for approval in the European Union. BioCryst has several ongoing development programs including BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva, and galidesivir, a potential treatment for Marburg virus disease and Yellow Fever. RAPIVAB® (peramivir injection), a viral neuraminidase inhibitor for the treatment of influenza, has received regulatory approval in the U.S., Canada, Australia, Japan, Taiwan and Korea. Post-marketing commitments for RAPIVAB are ongoing. For more information, please visit the company's website at www.biocryst.com.

    Forward-Looking Statements

    This press release contains forward-looking statements, including statements regarding BioCryst's plans and expectations for ORLADEYO.  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: the ongoing COVID-19 pandemic, which could create challenges in all aspects of BioCryst's business, including without limitation delays, stoppages, difficulties and increased expenses with respect to BioCryst's and its partners' development, regulatory processes and supply chains, negatively impact BioCryst's ability to access the capital or credit markets to finance its operations, or have the effect of heightening many of the risks described below or in the documents BioCryst files periodically with the Securities and Exchange Commission; BioCryst's ability to successfully implement its commercialization plans for, and to commercialize, ORLADEYO, which could take longer or be more expensive than planned; the results of BioCryst's partnerships with Torii and OrphanPacific may not meet BioCryst's current expectations; the commercial viability of ORLADEYO, including its ability to achieve market acceptance, which could impact the amount of any related royalties BioCryst would be entitled to receive from Torii; the FDA, EMA, PMDA 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; BioCryst's ability to successfully manage its growth and compete effectively; risks related to the international expansion of BioCryst's business; and actual financial results may not be consistent with expectations, including that 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, all of which identify important factors that could cause the actual results to differ materially from those contained in BioCryst's forward-looking statements.

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