BioCryst Eyes $1B Rare-Disease Future Through External Innovation

Key Points

• BioCryst is pivoting to external innovation, using cash flow to license or acquire earlier-stage rare-disease programs while maintaining profitability. Management targets more than $1 billion in revenue by 2030 from ORLADEYO, navenibart and potentially a third product.
• ORLADEYO remains the company’s foundation, with continued patient growth, expansion into pediatric HAE and opportunities among patients with normal C1 inhibitor levels. BioCryst expects competition from Pharvaris to affect some new patient starts but believes existing ORLADEYO users are less likely to switch.
• BioCryst’s key pipeline catalysts include a third-quarter readout for navenibart’s pivotal ALPHA-ORBIT trial and year-end data for BCX17725 in Netherton syndrome. Navenibart is intended to offer quarterly or semiannual dosing for patients currently using injectable HAE prophylaxis.

BioCryst Pharmaceuticals NASDAQ: BCRX is shifting its strategy toward external innovation as it seeks to expand beyond its ORLADEYO hereditary angioedema, or HAE, franchise, newly appointed Chief Executive Officer Charlie Gayer said at a Morgan Stanley event.

Gayer, who became CEO on Jan. 1, said the company has moved away from internal drug discovery following its transition to profitability. Instead, BioCryst plans to use its growing cash flow to acquire or license medicines that can support a broader rare-disease portfolio.

“We think the best way to go forward is through external innovation,” Gayer said. He outlined a longer-term goal of having ORLADEYO, navenibart and potentially a third product on the market by the end of 2030, generating more than $1 billion in revenue while maintaining a stable cost base and increasing profitability.

External Innovation Strategy

Gayer said BioCryst’s decision to discontinue internal discovery capabilities was driven by the opportunity to evaluate a broader universe of rare-disease programs rather than funding a limited number of internal projects. The company has added scientific leadership, including Chief R&D Officer Dr. Sandeep Menon and Chief Scientific Officer David Jenkins, to evaluate outside opportunities.

The company’s acquisition of Astria Therapeutics and its navenibart candidate was an example of the strategy, though Gayer said future transactions are more likely to involve earlier-stage Phase I or Phase II assets than similarly sized acquisitions.

BioCryst paid an enterprise value of approximately $700 million for Astria, according to Gayer. Going forward, he said the company expects potential licensing transactions in the range of $100 million to $200 million upfront, with additional payments structured later. He said BioCryst does not intend to strain its balance sheet and will maintain profitability.

The company is focused on rare diseases requiring chronic, self-administered or home-administered therapies. Gayer said BioCryst does not plan to pursue oncology, hospital-delivered products, gene therapy or cell therapy.

ORLADEYO Demand and HAE Market

Gayer said ORLADEYO remains the foundation of BioCryst’s business and has continued to add patients more than five years after launch. ORLADEYO is the only oral prophylactic treatment in the HAE market, he said, and its growth has been supported by physician experience, real-world evidence and BioCryst’s patient-access efforts.

He said approximately 60% of patients who begin ORLADEYO remain on treatment for one year, reflecting what he described as strong efficacy for a majority of patients. Gayer noted that the medicine is not appropriate for every patient.

The HAE market includes roughly 10,000 patients in the United States and has seen 11 branded products approved, with another five or six products in late-stage development, Gayer said. About 80% of diagnosed and treated HAE patients now receive prophylactic treatment, compared with a substantially lower proportion a decade ago.

BioCryst sees additional growth opportunities in patients with normal C1 inhibitor and in pediatric HAE. Gayer said patients with normal C1 inhibitor account for more than one-third of ORLADEYO users. The company recently launched a pellet formulation for children ages 2 through 12, and Gayer said early demand since the August launch has exceeded BioCryst’s expectations.

On competition from Pharvaris’ deucrictibant, Gayer said BioCryst had modeled a market entry based on a product profile similar to results reported from the competitor’s CHAPTER-3 study. He said BioCryst expects a new oral therapy could reduce some new patient starts for ORLADEYO, but believes patients doing well on ORLADEYO are unlikely to switch.

Navenibart Pivotal Study

BioCryst views navenibart as complementary to ORLADEYO because it is designed for patients using injectable prophylactic therapies. Gayer said approximately 5,000 U.S. HAE patients use injectable prophylaxis.

Navenibart is a kallikrein inhibitor being studied at dosing intervals of every three months or every six months. Gayer said Phase II data from the ALPHA-SOLAR study showed comparable experience across the two dosing schedules, with patients reaching a mean attack rate of fewer than two per year. He also said the formulation is intended to avoid painful injections.

The pivotal ALPHA-ORBIT trial fully enrolled in June. BioCryst expects a data readout in the third quarter of the following year, after the final patient reaches 12 months of treatment. The company intends to seek labeling for both dosing options if supported by the data.

Gayer said BioCryst expects to use its existing commercial organization to launch navenibart. The company has 40 sales representatives and spends slightly less than $150 million annually on sales and marketing, an amount he said is expected to rise largely with cost-of-living increases.

BCX17725 Data Expected by Year-End

BioCryst also expects data by year-end for BCX17725, a subcutaneous KLK5 inhibitor being studied in Netherton syndrome. The rare genetic condition causes uncontrolled tissue kallikrein activity and can lead to severe skin symptoms, according to Gayer. He said there are no approved therapies for the condition and BioCryst estimates there may be more than 3,000 U.S. patients.

The three-month study includes 12 patients and uses an intravenous loading dose followed by subcutaneous dosing every two weeks. BioCryst will assess the proportion of patients achieving clinically meaningful responses, using physician and patient measures, as well as evidence of a dose response. Gayer said the results will help determine whether the program advances directly toward a pivotal study or requires additional confirmatory work.

About BioCryst Pharmaceuticals (NASDAQ:BCRX)

BioCryst Pharmaceuticals, Inc is a biotechnology company that discovers, develops and commercializes orally administered medicines for rare diseases. The company focuses on treatments that address significant unmet medical needs, using expertise in small-molecule drug discovery and development.

BioCryst's primary commercial product is ORLADEYO (berotralstat), an oral, once-daily medication used to prevent hereditary angioedema attacks in adults and pediatric patients ages 12 and older.

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