BioCryst reports ORLADEYO reduces severe HAE attack rates

RESEARCH TRIANGLE PARK, N.C. - BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX), which has seen its stock surge over 45% in the past six months and currently trades near $10.21, has shared new real-world evidence demonstrating significant reductions in hereditary angioedema (HAE) attack rates in adolescents and patients with severe HAE, following treatment with ORLADEYO® (berotralstat). The company, with a market capitalization of $2.1 billion, has demonstrated strong revenue growth of nearly 42% over the last twelve months. According to InvestingPro analysis, the stock is currently trading at Fair Value levels. The data were presented at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) conference in Montreal, which concluded today.

The findings are based on a retrospective pre-post study, which included data from BioCryst’s sole-source pharmacy between December 15, 2020, and January 8, 2024. The study observed 56 U.S. patients with HAE-C1-INH, who experienced a substantial decrease in monthly attack rates from 7.78-8.23 at baseline to 1.24-1.90 after starting ORLADEYO. At 12 and 18 months, the reduction in attacks per month was statistically significant, with 6.25 and 6.43 fewer attacks, respectively.

Additionally, 99 U.S. adolescent patients aged 12-17 years reported significant and sustained reductions in HAE attack rates during each 90-day follow-up period after initiating ORLADEYO treatment. The attack rates dropped from a mean baseline of 2.07-2.30 attacks per month to 0.36-0.76, with the number of attacks decreasing by 1.56 per month after 12 months and by 1.85 per month after 18 months.

Dr. Raffi Tachdjian, from the University of California Los Angeles, commented on the real-world impact of ORLADEYO, suggesting that these findings should offer physicians and their HAE patients additional confidence in controlling their attacks. Dr. Donald S. Fong, chief medical officer of BioCryst, emphasized the effectiveness of ORLADEYO across a broad spectrum of HAE patients.

ORLADEYO® (berotralstat) is the first oral therapy specifically designed to prevent HAE attacks in adult and pediatric patients 12 years and older. It operates by reducing the activity of plasma kallikrein, a component that contributes to swelling in HAE.

While the medication has been celebrated for its prophylactic use, it is not recommended for the treatment of acute HAE attacks. The most common adverse reactions in patients taking ORLADEYO include abdominal pain, vomiting, and back pain, among others. The drug’s safety and efficacy in pediatric patients under 12 years of age have not been established.

This news is based on a press release statement from BioCryst Pharmaceuticals, which remains committed to improving the lives of people with rare diseases through the development of small-molecule and protein therapeutics. Five analysts have recently revised their earnings estimates upward, with expectations of profitability this year. Detailed financial analysis and future growth projections are available in the comprehensive Pro Research Report on InvestingPro, part of the platform’s coverage of over 1,400 US equities.

In other recent news, BioCryst Pharmaceuticals reported a significant increase in Q1 2025 revenue, reaching $145.5 million, which exceeded the forecasted $127.9 million. This growth was largely driven by strong sales of its product, Orladeyo. The company also reported earnings per share of $0.00, surpassing the anticipated loss of $0.07. BioCryst raised its annual revenue guidance for Orladeyo to a range of $580-600 million, reflecting a growth of 33-37% over the previous year. Jefferies maintained a Buy rating on BioCryst, raising the price target from $12.00 to $14.00, citing the strong performance of Orladeyo and potential future growth from its product pipeline. The company’s financial outlook for 2025 has improved, with expectations of achieving profitability a year ahead of schedule. BioCryst has also made strides in its product development pipeline, with upcoming data releases expected for treatments targeting Netherton syndrome and diabetic macular edema. These developments have contributed to a positive market response and investor optimism about the company’s future prospects.

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