BioCryst to Scrap Internal Discovery

Rare Daily Staff

BioCryst Pharmaceuticals is shutting down its internal drug discovery operations and closing its Birmingham, Alabama, research facility, marking a significant strategic pivot toward externally sourced innovation as the company seeks to expand its rare disease pipeline more efficiently.

The decision follows a six-month strategic review under CEO Charlie Gayer and reflects a broader industry trend of smaller biotechs moving away from fully integrated R&D models. BioCryst said it will wind down its Discovery Center of Excellence by the end of 2026 and discontinue all internal discovery programs, redirecting resources toward in-licensing, partnerships, and other external opportunities.

Gayer framed the move as a shift toward a more “agile, targeted approach” to research, emphasizing capital efficiency and speed to clinic.

“While our internal discovery programs have played a foundational role in building the business we are today, we believe that our next phase of growth and value creation will come from a more agile, targeted approach to research,” said Gayer. “By leveraging external capabilities and partnerships, as well as our powerful rare disease commercialization engine, we can expand our opportunity set and bring new rare disease therapies to patients faster and in a more capital-efficient manner.”

BioCryst’s decision underscores a growing emphasis among commercial-stage rare disease companies on capital allocation, particularly as investors scrutinize R&D productivity and timelines. By relying on external innovation, the company is betting it can access differentiated science without the fixed costs and risks associated with early discovery.

The restructuring comes as BioCryst narrows its internal focus to two clinical-stage programs. The most advanced, navenibart, is a long-acting monoclonal antibody targeting plasma kallikrein for hereditary angioedema (HAE) prophylaxis. The company recently completed enrollment in its Phase 3 ALPHA-ORBIT trial and expects top-line data in the third quarter of 2027. If successful, navenibart could differentiate itself with dosing intervals of three or six months, with efficacy assessed over a 12-month period.

BioCryst is also advancing BCX17725, a KLK5 inhibitor in development for Netherton syndrome, a rare and severe genetic skin disorder with no approved targeted therapies. The drug is currently being evaluated in a Phase 1 study, with proof-of-concept data expected by the end of 2026.

Alongside its pipeline update, the company reported progress on its commercial portfolio. BioCryst said it has resolved a previously disclosed manufacturing delay affecting oral pellet formulations of Orladeyo for pediatric patients aged 2 to under 12 years. The product is now expected to be available in early August.

The restructuring is expected to reduce operating expenses. BioCryst lowered its 2026 non-GAAP operating expense guidance to $420 million to $440 million, down from a prior range of $450 million to $470 million, reflecting anticipated savings from the closure of the Birmingham site and the transition to a leaner R&D model.

The closure of the Birmingham facility will impact employees, though the company said it plans to support affected staff through the transition.