BioCryst reports efficacy of HAE drug across age groups

RESEARCH TRIANGLE PARK, N.C. - BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX), a pharmaceutical company with a market capitalization of $2.28 billion and impressive revenue growth of 41.67% over the last twelve months, today shared new data demonstrating the effectiveness of its hereditary angioedema (HAE) treatment, berotralstat, in reducing symptom days in children and maintaining a strong safety and efficacy profile across all ages. According to InvestingPro data, the company’s stock is trading near its 52-week high of $11.11, reflecting growing investor confidence in its clinical developments. The data was presented at the 14th C1-Inhibitor Deficiency & Angioedema Workshop in Budapest, Hungary, occurring from May 29 to June 1, 2025.

The APeX-P trial, an ongoing study, showed promising interim results for berotralstat in children aged 2 to 11. Patients reported fewer missed school days due to HAE, and at least half remained attack-free monthly. The percentage of days with symptoms fell from 11 percent pre-study to 4 percent over a 12-week period, with improvements sustained up to 48 weeks. Common adverse events included nasopharyngitis and headache. The company’s strong gross profit margin of 64.25% supports its continued investment in clinical development. For detailed financial analysis and 14 additional key insights about BCRX, visit InvestingPro.

In addition, findings from the Italian Expanded Access Program (EAP) highlighted the consistent low attack rate and improved quality of life among patients transitioning to commercially available ORLADEYO, BioCryst’s marketed prophylactic treatment for HAE.

A subgroup analysis of the Berolife study in France focused on adolescents aged 12 to 17 who received berotralstat. Results indicated a significant drop in median monthly attack rates from baseline after six months of treatment, with tolerability aligning with previous clinical trials.

ORLADEYO (berotralstat) is the first oral, once-daily medication designed to prevent HAE attacks in patients 12 years and older. It functions by decreasing the activity of plasma kallikrein. However, the drug is not recommended for acute HAE attacks or at doses higher than 150 mg daily due to potential QT prolongation.

BioCryst emphasizes its commitment to improving the lives of those with HAE and other rare diseases through its structure-guided drug design approach. With five analysts recently revising earnings estimates upward and net income expected to grow this year, the company’s financial outlook appears promising. Access comprehensive analysis and the full Pro Research Report, along with real-time financial metrics and expert insights, through InvestingPro’s extensive coverage of over 1,400 US stocks.

The press release statement provided the basis for this report.

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 Orladeyo, which brought in $134 million, marking a 51% year-over-year increase. The company’s earnings per share also outperformed expectations, coming in at $0.00 against a predicted loss of $0.07. Additionally, BioCryst raised its annual revenue guidance for Orladeyo to between $580 million and $600 million, citing a higher-than-expected payment rate from Medicare patients.

Jefferies maintained a Buy rating on BioCryst and raised its price target from $12.00 to $14.00, attributing this to the robust performance of Orladeyo. The firm noted that upcoming pipeline data updates, particularly concerning treatments for Netherton syndrome and diabetic macular edema, could further enhance the company’s growth prospects. Furthermore, BioCryst shared new evidence at a recent conference showing that ORLADEYO significantly reduces hereditary angioedema attack rates, providing additional confidence for physicians and patients.

These developments underscore BioCryst’s strategic focus on expanding its market potential through new product developments and strategic initiatives. The company also announced it expects to achieve full-year profitability in 2025, a year ahead of schedule, driven by its strong financial performance and strategic debt reduction efforts.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.