BioCryst Ends Internal Discovery Efforts, Strengthens Focus on Clinical-Stage Rare Disease Assets

BioCryst Pharmaceuticals is ending its internal drug discovery programs and closing its Discovery Center of Excellence in Birmingham, Alabama by the end of 2026 as part of a strategic shift toward externally sourced innovation. The company said the transition will allow it to allocate capital more efficiently while accelerating development of its rare disease pipeline, led by the Phase 3 hereditary angioedema candidate navenibart and the investigational Netherton syndrome therapy BCX17725.

The decision follows a comprehensive review of the company’s research strategy and pipeline priorities. Rather than investing in early-stage internal discovery, BioCryst will focus on identifying promising external assets through partnerships, licensing opportunities, and scientific evaluation, leveraging its commercial expertise in rare diseases to expand its development portfolio.

Strategic Shift Prioritizes Clinical Development

BioCryst said its revised research model is intended to build a sustainable pipeline beyond its marketed therapy ORLADEYO® (berotralstat) and current clinical-stage programs while improving long-term capital efficiency. The company expects the transition to reduce operating costs and broaden access to innovative rare disease therapies emerging from academia and biotechnology partners.

President and Chief Executive Officer Charlie Gayer said the company’s internal discovery efforts established the scientific foundation for its current business, but external innovation now offers a more agile approach to future pipeline growth. He added that combining disciplined business development with BioCryst’s established rare disease commercial infrastructure could accelerate delivery of new therapies to patients.

Chief Research and Development Officer Dr. Sandeep Menon said the new strategy expands the range of scientific opportunities available for evaluation while allowing the company to remain focused on advancing its existing clinical programs.

Phase 3 Navenibart Study Reaches Key Milestone

Despite ending internal discovery activities, BioCryst emphasized continued investment in its late-stage pipeline.

Enrollment has been completed in the Phase 3 ALPHA-ORBIT trial (NCT06842823) evaluating navenibart, an investigational long-acting plasma kallikrein inhibitor for prophylaxis of hereditary angioedema (HAE). Plasma kallikrein drives excessive bradykinin production, the primary cause of recurrent HAE attacks.

The pivotal study is evaluating both three-month and six-month dosing regimens with efficacy assessed through 12 months, making it the largest pivotal HAE trial conducted to date. BioCryst expects top-line efficacy results for both six- and twelve-month endpoints during the third quarter of 2027. If successful, navenibart could become the first HAE therapy offering both three-month and six-month dosing schedules.

The company also continues to advance BCX17725, an investigational KLK5 inhibitor for Netherton syndrome, a rare inherited skin disorder without approved targeted therapies. Part 4 of the Phase 1 study is currently enrolling up to 12 patients, with proof-of-concept data expected by the end of 2026.

ORLADEYO Pediatric Formulation Returns to Schedule

BioCryst also resolved the previously disclosed manufacturing delay affecting ORLADEYO oral pellets for children aged 2 to younger than 12 years. The pediatric formulation is expected to become commercially available in early August 2026, expanding treatment availability for younger patients requiring long-term HAE prophylaxis.

Lower Operating Expenses, Revenue Guidance Reaffirmed

The restructuring prompted BioCryst to improve its 2026 non-GAAP operating expense guidance to $420 million to $440 million, down from its previous outlook of $450 million to $470 million. The company reaffirmed its full-year revenue guidance, including ORLADEYO revenue of $625 million to $645 million and total revenue of $635 million to $660 million.

BioCryst expects its streamlined operating model to continue lowering costs beyond 2026 while supporting investment in late-stage clinical development and future externally acquired rare disease programs.

Reference

BioCryst Sharpens Scientific Focus on External Innovation with Wind Down of Internal Discovery Programs and Closure of Birmingham Research Facility | BioCryst Pharmaceuticals, Inc.