BioCryst Reports Long-Term ORLADEYO Benefits and New Navenibart Data in HAE

BioCryst Pharmaceuticals presented new clinical and real-world evidence reinforcing the strength of its hereditary angioedema (HAE) portfolio at the 2026 European Academy of Allergy and Clinical Immunology (EAACI) Annual Congress.

The data underscore the durable efficacy and safety of ORLADEYO® (berotralstat), the first and only targeted oral prophylactic therapy approved for HAE patients aged two years and older, while advancing navenibart, a next-generation investigational plasma kallikrein inhibitor currently in Phase 3 development.

ORLADEYO Demonstrates Durable Pediatric Benefit

Updated 48-week findings from the ongoing APeX-P study (NCT05453968), the largest long-term prophylaxis trial in pediatric HAE, showed sustained reductions in disease burden among children aged 2 to less than 12 years receiving once-daily ORLADEYO.

The median adjusted monthly attack rate requiring on-demand treatment declined from 0.691 attacks during the standard-of-care period to 0.169 attacks during ORLADEYO treatment. Attacks requiring professional medical care decreased from 22 events under standard care to three during the first 12 weeks of ORLADEYO treatment and fell to zero between Weeks 37 and 48.

Investigators also observed a shift in healthcare utilization, with the need for higher-intensity medical interventions declining over time and care moving from emergency department and urgent care settings toward routine physician visits.

No new safety signals emerged during the 48-week treatment period, further supporting ORLADEYO’s favorable long-term safety and tolerability profile in pediatric patients.

Navenibart Shows Consistent Efficacy Across Subgroups

A post hoc analysis from the Phase 1b/2 ALPHA-STAR study demonstrated consistent reductions in overall HAE attack rates across subgroups defined by baseline attack rate, body mass index (BMI), and age.

Reductions in moderate-to-severe attacks and lower use of on-demand medications were observed across analyzed subgroups, supporting broad clinical activity across diverse patient populations. Decreases in moderate-to-severe attacks were also reported across all BMI categories.

Navenibart continued to demonstrate a favorable safety and tolerability profile, with no severe or serious treatment-emergent adverse events reported. The most common adverse events were headache, nasopharyngitis, and urinary tract infection, while injection-site reactions remained infrequent.

These findings build on previously reported ALPHA-STAR results and support the ongoing Phase 3 ALPHA-ORBIT trial (NCT07204938), which is evaluating navenibart as a long-acting prophylactic therapy with dosing intervals of every three or six months.

Real-World Evidence Reinforces Clinical Utility

Complementing the clinical trial findings, BioCryst presented multiple real-world analyses evaluating ORLADEYO in adolescent and adult patients, including individuals transitioning from other long-term prophylactic therapies.

Across studies, ORLADEYO was associated with sustained reductions in HAE attack frequency and overall disease burden. Healthcare resource utilization declined, supporting the real-world effectiveness of long-term prophylaxis with ORLADEYO.

Patients who switched from alternative prophylactic therapies maintained clinical benefit following transition, while real-world analyses also reported high levels of patient satisfaction, further supporting ORLADEYO’s practicality and effectiveness in routine clinical care.

Expanding the HAE Treatment Landscape

Commenting on the findings, Sandeep Menon, Chief Research and Development Officer at BioCryst, said the data reinforce the strength of the company’s HAE portfolio by demonstrating meaningful real-world benefits for patients currently living with HAE while advancing next-generation therapeutic approaches designed to address remaining unmet needs and align with patient treatment preferences.

Hereditary angioedema is a rare genetic disorder characterized by recurrent episodes of severe swelling that can significantly affect quality of life. The EAACI 2026 presentations add to the growing body of evidence supporting ORLADEYO’s role in long-term disease control while advancing navenibart as a potential next-generation prophylactic option with extended dosing intervals for patients living with HAE.

Reference

BioCryst Presents New Clinical Data and Real-World Evidence Demonstrating Impact of HAE Portfolio at the 2026 European Academy of Allergy and Clinical Immunology Annual Meeting | BioCryst Pharmaceuticals, Inc.