Latest news with #berotralstat
Yahoo
16-06-2025
- Health
- Yahoo
BioCryst Presents New Data on the Long-term Efficacy and Safety of ORLADEYO® (berotralstat) Across all Ages at EAACI
RESEARCH TRIANGLE PARK, N.C., June 16, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced new data on the long-term efficacy and safety of ORLADEYO® (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE) in patients across all age groups. 'These data from both clinical trials and real-world settings continue to strengthen the evidence that ORLADEYO is an effective and well-tolerated long-term prophylactic treatment for HAE. Importantly, patients report not only fewer and less severe attacks, but also meaningful improvements in daily functioning and emotional well-being. This contributes to the growing body of evidence supporting the use of ORLADEYO as a long-term prophylactic option for adolescents and adults living with HAE,' said Helen Thackray, chief research and development officer of BioCryst. The following four studies were presented at the European Academy of Allergy and Clinical Immunology (EAACI) meeting in Glasgow, United Kingdom, from June 13 to 16, 2025. Berotralstat Use Reduced Number of HAE Attacks Requiring Treatment or Professional Care in Pediatric Patients: Interim Results from APeX-P The ongoing open-label APeX-P study is evaluating the pharmacokinetic, safety, and effectiveness of berotralstat in children aged 2 to 11 years with HAE. Patients were enrolled into four weight-based cohorts. Cohort 1 received a 150 mg capsule once daily; cohorts 2-4 received once-daily oral granule doses of 108 mg, 96 mg and 78 mg, respectively. The median age was 8 years (range: 3–11), with disease onset typically between ages 2–6 years. All patients received standard of care (SOC) treatment for 12 weeks prior to the study. Key results Eighty-six percent reduction in attacks requiring professional care: attacks dropped from 22 during the 12-week SOC period to three following 12 weeks of berotralstat treatment Early/rapid and sustained reduction in the rate of HAE attacks requiring on-demand treatment Mean (SEM) adjusted monthly attack rate decreased from 1.28 (0.25) during SOC to 0.38 (0.13) from day one to week four of berotralstat treatment Sustained reduction for up to 48 weeks of follow-up Berotralstat was well tolerated across all cohorts; the most common treatment-emergent adverse events were nasopharyngitis, upper respiratory tract infection, and headache. Assessment of the Effectiveness and Tolerability of Berotralstat for Long-term Prophylaxis in Hereditary Angioedema: Findings from the Berolife Study The Berolife study is an open-label observational study in France which assessed the real-world tolerability and effectiveness of oral once-daily berotralstat (150 mg) in patients with HAE aged >12 years. A total of 82 patients were enrolled, with a mean (SD) age of 40.0 (17.5) years. The mean (SD) baseline attack rate was 1.1 (1.0) attacks per month (median: 0.83), based on the six months prior to enrollment. Key results Significant reduction in monthly HAE attacks was observed at six months (in patients with follow-up data (n=37)) Median monthly attack rate decreased from 1.0 to 0.44 attacks after six months of berotralstat treatment Sustained reductions in attack frequency maintained at 12, 18, and 24 months Berotralstat was well tolerated throughout the study period Adverse events were consistent with previous clinical trial data Impact of Berotralstat on Quality of Life in Patients with Hereditary Angioedema This analysis assessed the impact of berotralstat on patient-reported quality of life (QoL) outcomes compared to placebo, using pooled data from previous Phase 3 APeX-2 and APeX-J clinical trials which showed that once-daily berotralstat 150 mg significantly reduced the frequency of HAE attacks. This QoL assessment was measured using the validated Angioedema Quality of Life Questionnaire (AE-QoL), which evaluates four key domains: functioning, fatigue/mood, fears/shame, and nutrition. Key results Significant improvements in AE-QoL total and domain scores were observed with berotralstat versus placebo at week 24 Benefits sustained through week 96, indicating lasting improvement in daily life At week 24, 60 percent of patients receiving berotralstat achieved the Minimal Clinically Important Difference (MCID) in AE-QoL total score, compared to 52.4 percent in the placebo group Over time, the proportion of patients reaching MCID increased in the berotralstat cohort, suggesting progressive and cumulative improvements in QoL alongside reductions in HAE attack frequency Patients With HAE Report Positive Perceptions Following Berotralstat Treatment: Results from a Focus Group This qualitative study explored patient experiences with HAE, including their care journey and perceptions of treatment with berotralstat. Focus groups were conducted to gain insight into the real-world impact of berotralstat on patients' daily lives. Seven patients from France, aged 20-70 years, participated in the focus groups. All had been treated with berotralstat for at least six months, with a median age at HAE diagnosis of 28 years. Prior to starting berotralstat, the majority of participants (71 percent) had switched from previous treatments due to long-term tolerability concerns and guidance from health authorities. Key findings Participants reported notable improvements in disease control, including reduced frequency and severity of HAE attacks Minimal side effects were observed Berotralstat was described as less burdensome and easier to incorporate into daily routines compared to previous therapies Improved disease management was associated with reduced psychological distress and a greater sense of normalcy, contributing to enhanced quality of life 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. U.S. Indication and Important Safety Information INDICATIONORLADEYO® (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 useThe 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 INFORMATIONAn 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). Berotralstat is a substrate of P-glycoprotein (P-gp) and breast cancer resistance protein. 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 Please see full Prescribing Information. About BioCryst PharmaceuticalsBioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with hereditary angioedema and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. For more information, please visit or follow us on LinkedIn. Forward-Looking StatementsThis press release contains forward-looking statements, including statements relating to ORLADEYO safety, performance and effectiveness. 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: BioCryst's ability to successfully implement or maintain its commercialization plans for ORLADEYO; interim results of a clinical trial do not necessarily predict final results; the commercial viability of ORLADEYO, including its ability to achieve sustained market acceptance; the FDA or other applicable regulatory agency may require additional studies beyond the studies planned for products and product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may impose certain restrictions, warnings, or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, delay, or withdraw market approval for products and product candidates; and BioCryst's ability to successfully manage its growth and compete effectively. Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst's most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause the actual results to differ materially from those contained in BioCryst's forward-looking statements. BCRXW Contact:John Bluth+1 919 859 7910jbluth@ Niamh Lyons+353 87 7745000nlyons@
Associated Press
03-06-2025
- Business
- Associated Press
BioCryst Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
By BioCryst Pharmaceuticals, Inc. Published [hour]:[minute] [AMPM] [timezone], [monthFull] [day], [year] RESEARCH TRIANGLE PARK, N.C., June 03, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the compensation committee of BioCryst's board of directors granted seven newly-hired employees restricted stock units (RSUs) covering an aggregate of 47,250 shares of BioCryst common stock. The RSUs were granted as of June 2, 2025, as inducements material to each employee entering into employment with BioCryst. The RSUs were granted in accordance with Nasdaq Listing Rule 5635(c)(4). The RSUs 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. The RSUs are subject to the terms and conditions of BioCryst's Inducement Equity Incentive Plan and a restricted stock unit agreement covering the grant. About BioCryst Pharmaceuticals BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with hereditary angioedema and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class oral small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. For more information, please visit or follow us on LinkedIn . BCRXW Contact: John Bluth +1 919 859 7910 [email protected] The Associated Press is an independent global news organization dedicated to factual reporting. Founded in 1846, AP today remains the most trusted source of fast, accurate, unbiased news in all formats and the essential provider of the technology and services vital to the news business. More than half the world's population sees AP journalism every day.
Yahoo
15-05-2025
- Health
- Yahoo
FDA awards BioCryst's Orladeyo NDA for paediatric HAE
The US Food and Drug Administration (FDA) has accepted BioCryst Pharmaceuticals' new drug application (NDA) for Orladeyo (berotralstat), granting it priority review for use in children aged two to 11 with hereditary angioedema (HAE). A Prescription Drug User Fee Act target action date was set by the agency for 12 September 2025. The application was supported by the APeX-P trial's positive interim data. This trial assessed a prophylactic HAE therapy in these subjects. The results indicated that the therapy was well tolerated and maintained a consistent safety profile in this young age group. It also led to sustained and early minimisations of monthly attack rates. If approved, this plasma kallikrein inhibitor will be the inaugural oral targeted prophylactic therapy for HAE patients under 12. Beyond the US, the company has also submitted a line extension application to the European Medicines Agency for the use of the therapy oral granules in this patient population. The company plans additional regulatory filings in other worldwide territories, including Canada and Japan. BioCryst Pharmaceuticals chief research and development officer Dr Helen Thackray stated: 'As detailed in the results from APeX-P, we observed that participants experienced serious HAE attacks at a very early age, with a median age of symptom onset of two years, which suggests there is a larger burden of disease at an earlier age than has been appreciated thus far. 'If approved, we believe this oral granule formulation of Orladeyo could help children with HAE and their families better manage their condition and avoid the traumatic experience of acute attacks with emergency care or hospital stays.' In December 2020, the therapy was approved by the US regulator initially for HAE attack prevention in the adult population and in children aged 12 and above. It is currently available commercially in 30 countries. in 2024, the company secured a contract worth up to $69m from the US Department of Health and Human Services for the procurement of Rapivab to treat influenza. "FDA awards BioCryst's Orladeyo NDA for paediatric HAE" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
10-02-2025
- Business
- Yahoo
BioCryst to Report Fourth Quarter 2024 Financial Results on February 24
RESEARCH TRIANGLE PARK, N.C., Feb. 10, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the company will report its fourth quarter 2024 financial results on Monday, February 24, 2025. 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 webcast and replay of the call will be available online in the investors section of the company website at About BioCryst Pharmaceuticals BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with complement-mediated and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. For more information, please visit or follow us on LinkedIn. BCRXW Contact:John Bluth+1 919 859 7910jbluth@
