BCRX BioCryst Pharmaceuticals Inc.

5.13
+0.1  (+2%)
Previous Close 5.04
Open 5.14
52 Week Low 1.58
52 Week High 6.2862
Market Cap $906,664,469
Shares 176,565,622
Float 164,265,095
Enterprise Value $797,330,579
Volume 9,563,239
Av. Daily Volume 4,755,889
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Drug Stage Catalyst Date
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COVID-19
Phase 1
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BCX9250
Fibrodysplasia ossificans progressiva (FOP)
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Drug Pipeline

Drug Stage Notes
Berotralstat BCX7353
Reduce or eliminate attacks in HAE patients
Approved
Approved
FDA approval announced December 3, 2020
BCX9930
Paroxysmal nocturnal hemoglobinuria (PNH)
Phase 1
Phase 1
Phase 1 data released September 30, 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. —Significant and sustained reduction in HAE attacks—

    Oral, once-daily prophylactic option enables HAE patients to reduce burden of therapy1 

    ORLADEYO approved for adult and pediatric patients 12 years and older

    RESEARCH TRIANGLE PARK, N.C., Dec. 03, 2020 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced that the U.S. Food and Drug Administration (FDA) has approved oral, once-daily ORLADEYO™ (berotralstat) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.

    "ORLADEYO offers people with HAE and their physicians the first orally administered non-steroidal option for preventing HAE attacks and represents an important and welcome step in making…

    —Significant and sustained reduction in HAE attacks—

    Oral, once-daily prophylactic option enables HAE patients to reduce burden of therapy1 

    ORLADEYO approved for adult and pediatric patients 12 years and older

    RESEARCH TRIANGLE PARK, N.C., Dec. 03, 2020 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced that the U.S. Food and Drug Administration (FDA) has approved oral, once-daily ORLADEYO™ (berotralstat) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.

    "ORLADEYO offers people with HAE and their physicians the first orally administered non-steroidal option for preventing HAE attacks and represents an important and welcome step in making more treatment options available to physicians and patients," said Anthony J. Castaldo, president and chief executive officer of the US Hereditary Angioedema Association (HAEA). 

    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.

    "Patients and physicians acknowledge that HAE treatments can add a burden to patients' lives.  As an oral, once-daily option, ORLADEYO can provide significant attack reduction and lessen the burden associated with injections and infusions," said Marc Riedl, M.D., professor of medicine and clinical director, U.S. Hereditary Angioedema Association Center at the University of California, San Diego, and an investigator in the APeX-2 trial.

    "With this new treatment option, physicians and patients can continue to have collaborative discussions to choose the treatment that meets each patient's needs, life circumstances and preferences," Riedl added.

    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 and 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).2,3

    "The FDA approval of ORLADEYO fulfills a promise BioCryst made to HAE patients that we were committed to helping them achieve the dream of an oral, once-daily medicine to prevent and reduce the burden of their attacks," said Jon Stonehouse, president and chief executive officer of BioCryst.

    "Thank you to the HAE patients who participated in our clinical trials, to the investigators around the world who conducted these trials, to the HAEA for their patient advocacy and to our employees who never forgot that patients were waiting. We will stay focused on enabling access and providing personalized support to HAE patients and physicians," Stonehouse added.

    Commitment to Patient Access

    BioCryst is committed to supporting HAE patients taking ORLADEYO through a new program designed to streamline access to therapy. Through EMPOWER Patient Services, each HAE patient and their healthcare provider will have a single point of contact for access to ORLADEYO. A dedicated care coordinator will support access for each patient with comprehensive financial support tools and reimbursement support.

    EMPOWER Patients Services is administered by Optime Care Inc., the exclusive specialty pharmacy provider for ORLADEYO. Physicians can begin writing prescriptions for ORLADEYO immediately, with direct to patient shipments from Optime Care expected to begin by the end of December 2020.

    Additional information is available at www.ORLADEYO.com and 1-866-5-EMPOWER (1-866-536-7693).

    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 for the prevention of HAE attacks in adults and pediatric patients 12 years and older, and under regulatory review for approval in Japan and the European Union. BioCryst has several ongoing development programs including, an oral Factor D inhibitor for the treatment of complement-mediated diseases, galidesivir, a potential treatment for COVID-19, Marburg virus disease and Yellow Fever, and BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva. RAPIVAB® (peramivir injection), a viral neuraminidase inhibitor for the treatment of influenza, is BioCryst's first approved product and has received regulatory approval in the U.S., Canada, Australia, Japan, Taiwan, Korea and the European Union. 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, performances 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 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 product candidates, including to support the continued commercialization of ORLADEYO, may not provide regulatory clearances which may result in delay of planned clinical trials, may impose certain restrictions, warnings, or other requirements on product candidates, including ORLADEYO, may impose a clinical hold with respect to such product candidates, or may withhold or withdraw market approval for such 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 2020 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

    Contacts:

    John Bluth

    +1 919 859 7910

    Catherine Collier Kyroulis

    +1 917 886 5586



    ________________________

    1
    Radojicic, C, et al. Patient Perspectives on the Treatment Burden of Injectable Medication Administration for Hereditary Angioedema. Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology; Poster 160, Nov (2020)

    2 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) 

    3 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)

    A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/bf57a925-5da4-4c72-8a93-02e9c4308123



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  2. RESEARCH TRIANGLE PARK, N.C., Dec. 01, 2020 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the compensation committee of BioCryst's board of directors granted 13 newly-hired employees inducement options to purchase an aggregate of 260,500 shares of BioCryst common stock on November 30, 2020 as inducements material to each employee entering into employment with BioCryst. The options were granted in accordance with Nasdaq Listing Rule 5635(c)(4).

    The options have an exercise price of $5.11 per share, which is equal to the closing price of BioCryst common stock on the grant date. The options vest in four equal annual installments beginning on the one-year anniversary of the grant date, in each case subject…

    RESEARCH TRIANGLE PARK, N.C., Dec. 01, 2020 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the compensation committee of BioCryst's board of directors granted 13 newly-hired employees inducement options to purchase an aggregate of 260,500 shares of BioCryst common stock on November 30, 2020 as inducements material to each employee entering into employment with BioCryst. The options were granted in accordance with Nasdaq Listing Rule 5635(c)(4).

    The options have an exercise price of $5.11 per share, which is equal to the closing price of BioCryst common stock on the grant date. The options vest in four equal annual installments beginning on the one-year anniversary of the grant date, in each case subject to the new employee's continued service with the company. Each stock option has a 10-year term and is subject to the terms and conditions of BioCryst's Inducement Equity Incentive Plan and a stock option agreement covering the grant.

    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. BioCryst has several ongoing development programs including ORLADEYO (berotralstat), an oral treatment for hereditary angioedema, BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, galidesivir, a potential treatment for COVID-19, Marburg virus disease and Yellow Fever, and BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva. RAPIVAB® (peramivir injection), a viral neuraminidase inhibitor for the treatment of influenza, is BioCryst's first approved product and has received regulatory approval in the U.S., Canada, Australia, Japan, Taiwan, Korea and the European Union. 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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  3. RESEARCH TRIANGLE PARK, N.C., Nov. 30, 2020 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced the journal Allergy has published data from the APeX-J trial, a randomized, placebo-controlled trial conducted in Japan evaluating oral, once-daily berotralstat for the prophylactic treatment of hereditary angioedema (HAE).

    The APeX-J trial met its primary endpoint of a reduction in the rate of HAE attacks for berotralstat 150 mg compared to placebo during the 24-week period (p=0.003). Berotralstat was safe and generally well-tolerated in the trial.

    RESEARCH TRIANGLE PARK, N.C., Nov. 30, 2020 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced the journal Allergy has published data from the APeX-J trial, a randomized, placebo-controlled trial conducted in Japan evaluating oral, once-daily berotralstat for the prophylactic treatment of hereditary angioedema (HAE).

    The APeX-J trial met its primary endpoint of a reduction in the rate of HAE attacks for berotralstat 150 mg compared to placebo during the 24-week period (p=0.003). Berotralstat was safe and generally well-tolerated in the trial.

    These results are consistent with the global phase 3 APeX-2 trial, where berotralstat 150 mg also reduced the rate of HAE attacks compared to placebo (p<0.001) and was safe and generally well-tolerated.

    "APeX-J is the first placebo-controlled trial of an HAE medicine conducted in Japan and the berotralstat data are very exciting for patients, who currently have no approved prophylactic treatment options. Based on the safety and efficacy profile, I believe berotralstat, if approved, would be an important advancement in HAE management for Japanese patients," said Dr. Isao Ohsawa, president of Saiyu Soka hospital and principal investigator of the APeX-J trial. 

    Dr. Ohsawa and the study authors note that HAE is estimated to affect 2,500 patients in Japan and the recognition of HAE by physicians is low. Although two on-demand treatments are approved, no therapies are currently approved for long-term prophylaxis in Japan.

    "Berotralstat would be the first approved prophylactic therapy for HAE patients in Japan and we believe there is a significant opportunity for berotralstat to accelerate the diagnosis of HAE patients and dramatically improve the quality of life for patients," said Jon Stonehouse, president and chief executive officer of BioCryst.

    A new drug application (JNDA) is under review in Japan for approval of oral, once-daily berotralstat for the prophylactic treatment of HAE. Berotralstat is being reviewed under Sakigake designation and the company expects a decision on approval in December 2020.

    Torii Pharmaceutical, Co., Ltd. is BioCryst's commercial partner in Japan for berotralstat.

    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. BioCryst has several ongoing development programs including ORLADEYO (berotralstat), an oral treatment for hereditary angioedema, BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, galidesivir, a potential treatment for COVID-19, Marburg virus disease and Yellow Fever, and BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva. RAPIVAB® (peramivir injection), a viral neuraminidase inhibitor for the treatment of influenza, is BioCryst's first approved product and has received regulatory approval in the U.S., Canada, Australia, Japan, Taiwan, Korea and the European Union. 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 are identified by use of terms such as "believe," "will," "would," "expect," and similar words, although some forward-looking statements may be expressed differently. 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, performances 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; developing and commercializing ORLADEYO or any HAE product candidate may take longer or may be more expensive than planned; 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 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 product candidates, may impose a clinical hold with respect to such product candidates, or may withhold or delay market approval for product candidates; product candidates, if approved, may not achieve market acceptance; BioCryst's ability to successfully commercialize its product candidates, manage its growth, and compete effectively; and risks related to the international expansion of BioCryst's business.  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

    Contacts:

    John Bluth

    +1 919 859 7910

    Catherine Collier Kyroulis

    +1 917 886 5586

     



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  4. RESEARCH TRIANGLE PARK, N.C., Nov. 20, 2020 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced that the company will present at the Evercore ISI 3rd Annual HealthCONx on Wednesday, December 2, 2020 at 12:35 p.m. ET and at the Piper Sandler 32nd Annual Healthcare Conference. Both conferences are being conducted as virtual events.

    Piper Sandler is pre-recording company presentations at the conference and the pre-recorded presentation may be accessed on Monday, November 23, 2020 in the Investors section of BioCryst's website at http://www.biocryst.com.

    Links to a live audio webcast and replay of the presentation at the Evercore ISI conference may be accessed on December 2, 2020 in the Investors section of BioCryst's…

    RESEARCH TRIANGLE PARK, N.C., Nov. 20, 2020 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced that the company will present at the Evercore ISI 3rd Annual HealthCONx on Wednesday, December 2, 2020 at 12:35 p.m. ET and at the Piper Sandler 32nd Annual Healthcare Conference. Both conferences are being conducted as virtual events.

    Piper Sandler is pre-recording company presentations at the conference and the pre-recorded presentation may be accessed on Monday, November 23, 2020 in the Investors section of BioCryst's website at http://www.biocryst.com.

    Links to a live audio webcast and replay of the presentation at the Evercore ISI conference may be accessed on December 2, 2020 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. BioCryst has several ongoing development programs including ORLADEYO (berotralstat), an oral treatment for hereditary angioedema, BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, galidesivir, a potential treatment for COVID-19, Marburg virus disease and Yellow Fever, and BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva. RAPIVAB® (peramivir injection), a viral neuraminidase inhibitor for the treatment of influenza, is BioCryst's first approved product and has received regulatory approval in the U.S., Canada, Australia, Japan, Taiwan, Korea and the European Union. 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. RESEARCH TRIANGLE PARK, N.C., Nov. 13, 2020 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today presented new clinical data that further evaluates the attack rate reductions, patient satisfaction and quality of life of hereditary angioedema (HAE) patients in the APeX-2 trial over 48 weeks. Berotralstat is an investigational treatment for the prevention of attacks in patients with HAE.

    The data from three abstracts, including a Distinguished Industry Oral Abstract, are being presented at the 2020 Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI), which is being conducted virtually from November 13-15.

    "Presenting all available treatment options to patients is an important part of…

    RESEARCH TRIANGLE PARK, N.C., Nov. 13, 2020 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today presented new clinical data that further evaluates the attack rate reductions, patient satisfaction and quality of life of hereditary angioedema (HAE) patients in the APeX-2 trial over 48 weeks. Berotralstat is an investigational treatment for the prevention of attacks in patients with HAE.

    The data from three abstracts, including a Distinguished Industry Oral Abstract, are being presented at the 2020 Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI), which is being conducted virtually from November 13-15.

    "Presenting all available treatment options to patients is an important part of HAE clinical management. These data continue to demonstrate the potential of berotralstat as a prophylactic medication, if approved by the FDA, with sustained reduction in attacks and meaningful improvements in quality of life seen over 48 weeks of treatment. With its oral, once-daily administration, berotralstat would offer patients a therapeutic alternative for managing this chronic disease," said H. James Wedner, M.D., the Dr. Phillip and Arleen Korenblat Professor of Medicine at Washington University School of Medicine in St. Louis, and lead author of the Distinguished Industry Oral Abstract.

    Following is a brief description of the clinical data posters being presented at ACAAI.

    Berotralstat Reduces Attacks in Patients with Hereditary Angioedema (HAE): APeX-2 Trial 48 Week Results; Distinguished Industry Oral Abstract, Session A, Friday, November 13, 4:30-5:30 p.m. CT

    Patients treated with oral, once-daily berotralstat 150 mg for 48 weeks experienced a sustained reduction in mean investigator confirmed HAE attack rates through month 12.

    In patients re-randomized to berotralstat 150 mg after 24 weeks on placebo, there was a marked reduction in investigator-confirmed HAE attack rates over 24 weeks of treatment. These patients had a mean attack rate per month of 2.5 at baseline, 1.7 at month six (while on placebo), 0.6 at month seven (one month after starting berotralstat 150 mg) and 0.6 at month 12 (six months after starting berotralstat 150 mg).  

    Berotralstat was generally well-tolerated in APeX-2 through 48 weeks. The safety profile observed from weeks 24 to 48 was consistent with the data observed through the first 24 weeks. The most commonly reported treatment-related adverse events were upper respiratory tract infection, abdominal pain, diarrhea and vomiting.

    Berotralstat Positively Impacts Patient-Reported Satisfaction: Results from the Phase 3 APeX-2 trial; Poster #158

    Patient satisfaction with treatment was assessed using the validated Treatment Satisfaction Questionnaire for Medicine (TSQM), which is comprised of three specific scales (side effects, effectiveness and convenience) and is scored on a global satisfaction scale from 0-100.

    HAE patients who transitioned from placebo to berotralstat 150 mg at week 24 reported improved overall treatment satisfaction and effectiveness. These patients experienced statistically significant improvements from weeks 24 to 48, with a mean global satisfaction increase of 26 points (p=0.005) and a mean effectiveness increase of 29.6 points (p<0.001).   Convenience scores remained high through week 48, reflecting the positive experiences patients had taking an oral medication.

    Berotralstat Improves Patient-Reported Quality of Life Through 48 Weeks in the Phase 3 APeX-2 Trial; Poster #154

    Quality of life was assessed with the Angioedema Quality-of-Life (AE-QoL) questionnaire, a validated tool to measure impairment of QoL based on a total and domain (functioning, fatigue/mood, fear/shame and nutrition) scores. The minimal clinically important difference (MCID) is defined as an improvement of six points.

    Clinically meaningful improvements in mean AE-QoL total scores were observed as early as week four, with a mean improvement from baseline of 15 points at week 24. This improvement was sustained through 48 weeks of treatment with berotralstat 150 mg.

    Improvements were observed in all four domains (functioning, fatigue/mood, fear/shame, nutrition) through week 48. Notably, 77 percent of patients exceeded the MCID in total AE-QoL total scores at 48 weeks, indicating the reduction in attacks following berotralstat therapy appears to have a positive impact on patients' quality of life.

    About the APeX-2 Trial

    In APeX-2, 121 eligible HAE type 1 or type 2 patients were randomized 1:1:1 to oral, once daily berotralstat 110 mg or 150 mg or placebo for 24 weeks. At 24 weeks, patients initially randomized to 110 mg or 150 mg of berotralstat continued on that dose. Patients who initially received placebo were re-randomized 1:1 at week 24 to receive either berotralstat 110 mg or 150 mg.

    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. BioCryst has several ongoing development programs including ORLADEYO (berotralstat), an oral treatment for hereditary angioedema, BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, galidesivir, a potential treatment for COVID-19, Marburg virus disease and Yellow Fever, and BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva. RAPIVAB® (peramivir injection), a viral neuraminidase inhibitor for the treatment of influenza, is BioCryst's first approved product and has received regulatory approval in the U.S., Canada, Australia, Japan, Taiwan, Korea and the European Union. 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, performances 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; developing and commercializing ORLADEYO or any HAE product candidate may take longer or may be more expensive than planned; 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 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 product candidates, may impose a clinical hold with respect to such product candidates, or may withhold market approval for product candidates; product candidates, if approved, may not achieve market acceptance; BioCryst's ability to successfully commercialize its product candidates, 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 2020 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:

    John Bluth

    +1 919 859 7910

    Catherine Collier Kyroulis

    +1 917 886 5586

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