‘We’re not afraid of competition’: BioCryst CEO sets out strategy after scrapping internal discovery

Now accepting nominations for the Fierce Innovation Awards - Submit your application today Biotech ‘We’re not afraid of competition’: BioCryst CEO sets out strategy after scrapping internal discovery By Will Maddox Aug 14, 2026 10:07am BioCryst Pharmaceuticals Licensing deals hereditary angioedema Orladeyo BioCryst Pharmaceuticals has been on a journey this year. The North Carolina-based biotech—which markets the plasma kallikrein inhibitor Orladeyo for a rare genetic disorder called hereditary angioedema (HAE)—entered 2026 with a clinical-stage pipeline that included another kallikrein inhibitor for the eye disease diabetic macular edema (DME), as well as a KLK5 inhibitor in phase 1 trials for a rare, severe autosomal recessive genetic disorder called Netherton syndrome. But BioCryst turned heads in May when the company revealed that it was scrapping the DME therapy, called avoralstat, citing a focus on rare diseases.

The following month, the biopharma announced that it would shutter its internal discovery programs, including closing the company’s Discovery Center of Excellence facility in Birmingham, Alabama. From now on, any new additions to BioCryst’s pipeline will come solely from external deals. The goal is to add two new programs in the next 18 months.

When Fierce caught up with Charlie Gayer to better understand what comes next, the BioCryst CEO was keen to emphasize that the company is “not getting away from science and R&D.” “That's still a part of BioCryst,” said Gayer, who served as the company’s chief commercial officer until he took on the CEO role in January. The hunt for fresh programs to license will be partly bankrolled by Orladeyo. Having initially been approved in 2020 as the first daily oral pill to prevent HAE attacks in patients aged 12 and older, the drug’s reach was expanded last year to include an oral pellet formulation for those aged between two and 12 years.

The med brought in $158.2 million for the second quarter of 2026, with Evercore analysts pointing to a “steady increase in demand” in a recent note to investors. “We realized the industry is moving a lot faster right now,” Gayer told Fierce in an interview. “Now that we’re a profitable company, the best way for us to bring more drugs to rare disease patients and to do it efficiently and improve our probability of success is [by] looking externally.” In fact, this search for external innovation predates avoralstat’s failure.

BioCryst acquired Astria Therapeutics last year, which netted the company another kallikrein inhibitor for HAE in the form of navenibart. The company has since sold the European rights to the drug to Neopharmed Gentili for $70 million upfront. A phase 3 readout for navenibart is pencilled in for the third quarter of 2027.

Before that, phase 1 data for the Netherton syndrome therapy, dubbed BCX17725, is expected by the end of this year. Though the company has previously had a focus on HAE—a rare genetic condition that causes recurrent, unpredictable and potentially life-threatening swelling of the face, limbs, gastrointestinal tract and airways—Gayer said BioCryst is keeping its options open when it comes to hunting for new rare disease therapies. While we shouldn’t expect BioCryst to dive into cell or gene therapies anytime soon, the CEO said the company would be on the lookout for small molecules or protein therapeutics, with “chronic self-administration being key.” “Serving patients on a consistent basis, being able to find them … being able to help with diagnosis, being nimble and accurate has been part of our sweet spot—and there are a lot of rare diseases in different spaces that fit that,” he said.

Whatever drug candidate BioCryst opts for, it will likely have already entered human trials while not being so advanced that the deal would “stress the balance sheet,” Gayer said. With the company open to so many different modalities and indications, BioCryst has been building out a team ready for the challenge. In April, Sandeep Menon, M.D., Ph.D., was brought in as chief research and development officer, having previously served as chief development officer at Alnylam following over a decade at Pfizer.

Last month, BioCryst appointed David Jenkins, Ph.D., former head of research and external innovation at Ipsen, into the newly created position of chief scientific officer. “[Jenkins] brings great drug hunter experience,” Gayer explained. “As we evaluate different areas, we’re bringing in experts to help us think this through,” added the CEO, who pointed out that the company is “also beefing up our internal development expertise.” Gayer suggested one of BioCryst’s strengths has been “really understanding the patients and their needs.” “When you go back to the dawn of Orladeyo, prior to launch—quite frankly, our pivotal data weren't that great, and there were a lot of people back in 2019 asking, ‘Is this an approvable drug?

Is it going to go anywhere?’” he remembered. “We really dug deep into the research and patient insights …. and found that there was such an overwhelming desire and need at the time for oral therapy,” Gayer said.

The company has taken a similar approach with BCX17725, using large language models to analyze the Electronic Medical Record to better understand how many patients may actually have Netherton syndrome. “Going deeper has given us confidence that they're not just a few hundred—like people had thought before—but there are actually probably more than 3,000 patients in the U.S.,” Gayer said. The plan for now is to take BCX17725 into a phase 2 trial in 2027 after getting expected proof-concept data by the end of this year.

By the end of next year, BioCryst also hopes to have submitted navenibart to the FDA for approval in HAE. While the CEO explained that the current pipeline strategy is based on quality rather than quantity of assets, he’s hoping that a few more drugs can brought on board to counteract the fact that “not every early-stage program is going to be successful.” “If we could bring in a couple of programs over the next 12 or 18 months, that would be fantastic,” Gayer said. With experience in HAE, BioCryst is no stranger to finding success in a crowded market.

Since 2008, there have been more than 10 approved treatments for the rare genetic disease, including Orladeyo. BioCryst’s drug already has competition in the kallikrein inhibitor space courtesy of KalVista Pharmaceuticals' Ekterly, which was approved last year for patients with HAE and was seen as the big prize of Chiesi Group's acquisition of the company, which was announced this spring. There could be more on the way, including a potential one-time gene editing treatment from Intellia Therapeutics that has been described as “paradigm-shifting” by analysts.

“There are a number of companies focused on rare disease but there haven't been that many that stay consistently focused on rare disease and have the ability to commercialize the way that we do,” Gayer said. BioCryst is on the lookout for differentiated products to license, added the CEO, stressing that the biopharma is “not afraid of competition.” “We'd love to be the first or best kind of product in a disease state like Netherton, where there's high need and it's an open field,” Gayer said. “But those are harder to come by these days—so we're going to look well beyond that.” BioCryst Pharmaceuticals Licensing deals hereditary angioedema Orladeyo immunology Biotech