BioCryst Presents New Data at ACAAI Highlighting Burden of HAE on Pediatric Patients and Caregivers and Sustained Reduction of Attacks in Children Treated with One Year of ORLADEYO® (berotralstat)

–  Findings highlight negative psychosocial impact of HAE attacks and resulting ED/hospital visits on pediatric patients and their caregivers –

–  ORLADEYO oral granules demonstrated early and sustained reduction in HAE attacks over one year of treatment in second interim analysis of APeX-P –

–  ORLADEYO oral granules are currently under review with U.S. FDA for ages 2 to <12 with decision expected by end of year; would be first and only targeted oral prophylactic therapy for patients with HAE age 2 to <12 –

RESEARCH TRIANGLE PARK, N.C., Nov. 06, 2025 (GLOBE NEWSWIRE) --  BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced new data demonstrating the early and negative psychosocial impact of hereditary angioedema (HAE) and resulting emergency department (ED) and hospital visits on pediatric patients and their caregivers, as well as new one-year data from the ongoing APeX-P clinical trial showing early and sustained reductions in monthly attack rates over one year in pediatric patients with HAE aged 2 to <12 years treated with once-daily ORLADEYO^® (berotralstat).

The New Drug Application (NDA) for an oral granule formulation of once-daily ORLADEYO as prophylaxis in pediatric patients with HAE aged 2 to <12 years is currently under review with the U.S. Food and Drug Administration (FDA), with a target action date of December 12, 2025. The safety and effectiveness of ORLADEYO in pediatric patients <12 years of age have not been established. A capsule formulation of ORLADEYO was approved by the FDA in December 2020 as prophylaxis for patients with HAE aged 12 years and older.

Data are part of presentations at the Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI), which is taking place in Orlando, Fla. from November 6-10, 2025.

"Our psychosocial data elucidate that HAE attacks typically begin well before puberty and can have a profoundly negative impact on the mental health and quality of life of both children and their caregivers. Currently there are only injectable treatments approved for prevention of attacks in patients under 12, and findings highlight the clear and urgent need for a safe and effective long-term prophylactic treatment with a child-friendly method of administration," said Dr. Donald S. Fong, chief medical officer of BioCryst. "ORLADEYO would be the first and only targeted oral prophylactic therapy for patients with HAE aged 2 to less than 12 years, if approved. We believe that the one-year data from APeX-P reinforce the role that ORLADEYO oral granules could potentially play in preventing attacks in patients 2 to less than 12 years, and we look forward to the FDA's decision on our application later this year."

Reduction in Pediatric HAE Attack Rates with ORLADEYO: APeX-P Second Interim Analysis

Hereditary Angioedema Attack Rates in Children 2-12 with Oral Prophylactic Berotralstat Over One Year; ePoster #R089; Friday, November 7, 2:50 – 3:05 p.m. ET; Monitor #15, West Exhibit Hall

Poster #R089 explores one year safety and effectiveness of ORLADEYO in children aged 2 to <12 years with HAE in the largest pediatric HAE prophylaxis trial, APeX-P, and captures extended treatment exposure (mean 63.9 weeks).

Findings

• ORLADEYO demonstrated early and sustained attack rate reduction, with 65.5% of patients attack-free at month 1 and 70.4% of patients attack-free at month 12. Median attack rate was 0 beginning at month 1 and remained 0 for 11 out of 12 months.
• ORLADEYO demonstrated high continuation rates: nearly all (93.1%) patients completed ≥48 weeks of treatment with ORLADEYO.
• HAE attacks start early in life: median age at diagnosis was 2.0 (0.2-10.0) years with most (82.8%) experiencing HAE symptoms before age 6.
• ORLADEYO was safe and well tolerated, with no new safety signals identified. The most commonly reported treatment-emergent adverse event (TEAE) was nasopharyngitis. Please see below for full safety information for ORLADEYO.

"Treatment with ORLADEYO oral granules showed a sustained reduction in HAE attack rates over one year, and the high continuation rate suggests meaningful disease benefit and strong tolerability in this cohort," said Dr. Jolanta Bernatoniene, Paediatric Infectious Disease Department, Bristol Royal Hospital for Children, Bristol, UK. "These findings reinforce the potential of ORLADEYO to provide an important prophylactic treatment option for pediatric patients with HAE 2 to less than 12 years of age."

Methods

In the APeX-P trial, a 12-week standard-of-care period was followed by a subsequent open-label ORLADEYO treatment period lasting up to a total of 144 weeks. HAE attack rates were assessed over 48 weeks, with safety monitoring continuing for up to 144 weeks. Participants (n=29) were placed into four cohorts by body weight at baseline. The data presented here are from a second interim data cut taken at the time all participants remaining in the study had completed at least 48 weeks of ORLADEYO treatment.

Impacts of HAE on Pediatric Patients and Caregivers: Psychosocial Data

Posters #R102, #R109 and #R117 explore psychosocial impacts and disease burden of HAE, as well as HAE treatment experiences, preferences and needs based on feedback collected from caregivers together with their children (31), adolescents (19) and healthcare providers (HCPs) (109) via a combination of video ​in-depth interviews (VIDIs) and online discussions and panels.

"These findings drive home the negative impact of HAE experiences in childhood. From disruption in schooling and routines to mental health challenges, frequent and stressful ED/hospital visits and anxiety about injectable or IV medication administration, there are substantial psychosocial consequences for patients and their caregivers," said Dr. Daniel Soteres, MD, MPH, Asthma & Allergy Associates P.C. "In addition, most young patients with HAE were shown to have symptoms starting in early childhood. Continuing to understand the far-reaching impacts is important in helping HCPs optimize treatment plans."

Psychosocial Impact of Hereditary Angioedema on Young Patients and Their Caregivers; ePoster #R102; Friday, November 7, 2:50 – 3:05 p.m. ET; Monitor #16, West Exhibit Hall

Findings

• HAE negatively impacts children and their caregivers in multiple ways: adolescents and caregivers reported that HAE negatively impacts young patients' mental health, communication with others, and participation in school, sports, and social activities. Compared with caregivers, adolescents indicated a greater impact of HAE on their daily life. Caregivers also reported high negative impact on their own health, including anxiety, stress, disrupted sleep, and neglected self-care.
• HCPs recognize the negative impacts of HAE: 90% of HCPs reported that HAE attacks distress caregivers and patients, disrupt caregiver and patient daily routines, and negatively impact patients' quality of life. Most HCPs are concerned that young children with HAE will develop anxiety (84%) or depression (76%) and experience social isolation (76%) or academic underachievement (64%).

Burden of Attack for Young Patients with Hereditary Angioedema and Their Caregivers; ePoster #R109; Saturday, November 8, 11:30 –11:45 a.m. ET; Monitor #13, West Exhibit Hall

Findings

• HAE attacks start early in life: caregivers and adolescents reported symptoms starting at age 5, on average, and HCPs reported that 80% of patients started experiencing symptoms by age 8.
• HAE attacks occur frequently: caregivers reported that attacks occur about every 3 months and rated HAE as moderately severe (mean of 4.0 on a scale of 1-7).
• HAE attacks are stressful: negative emotions during attacks (fear, pain, worry) or the limitations associated with them were commonly reported by children and their caregivers.
  
  

Emergency Department Experiences of Young Patients with Hereditary Angioedema and Their Caregivers; ePoster #R117; Sunday, November 9, 12:00 – 12:15 p.m. ET; Monitor #13, West Exhibit Hall

Findings

• ED/hospital visits are common for young patients with HAE: over three-quarters of adolescents (11/14) and nearly half of caregivers (11/23) reported going to the ED or hospital at least once before age 12. Visits ranged from a few hours to a few days in length.
• Most adolescents and caregivers have experienced negative ED/hospital experiences: these visits were characterized by treatment delays, medication unavailability, challenges with medication administration, HCPs unfamiliar with HAE, and heightened stress due to the ED/hospital environment. These negative ED experiences are associated with long-term fear of hospitals or needles for some adolescents and children.
• HCPs also worry about the impact of these ED/hospital visits: nearly three quarters of HCPs surveyed reported concern about their patients receiving inadequate care, treatment-related trauma, and the long-term impact of ED/hospital visits on their patients' mental health.
  
  

In addition to displaying in the exhibit hall at the noted times, ePosters are accessible online and on demand to registered attendees on ACAAI's website.

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

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).

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 www.fda.gov/medwatch.

Please see full Prescribing Information.

About BioCryst Pharmaceuticals

BioCryst is a global biotechnology company focused on developing and commercializing medicines for hereditary angioedema (HAE) and other rare diseases, driven by its deep commitment to improving the lives of people living with these conditions. BioCryst has commercialized ORLADEYO^® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of potential first-in-class or best-in-class oral small-molecule and injectable protein therapeutics for a range of rare diseases. For more information, please visit www.biocryst.com or follow us on LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements, including statements regarding BioCryst's expectations relating to the oral granule formulation of once-daily ORLADEYO as prophylaxis in pediatric patients with HAE aged 2 to <12 years, including effectiveness and potential regulatory approval. 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: interim results of a clinical trial do not necessarily predict final results; the FDA or other applicable regulatory agency may not approve ORLADEYO for use in pediatric patients with HAE aged 2 to <12 years within the timeframe expected, or at all, may ultimately determine that there are deficiencies in the development program or execution thereof, may require additional information or studies, may disagree with our safety and efficacy conclusions, may impose certain restrictions, warnings, or other requirements, may impose a clinical hold with respect to ORLADEYO, or may withhold, delay, or withdraw market approval for ORLADEYO; and the oral granule formulation of once-daily ORLADEYO, if approved, may not achieve market acceptance. 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 actual results to differ materially from those contained in BioCryst's forward-looking statements.

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