Hereditary Angioedema Patients Report Breakthrough Attacks on Current Injectable/Infused Prophylaxis Medication

June 6, 2020

—Data presented at European Academy of Allergy and Clinical Immunology (EAACI) Digital Congress—

RESEARCH TRIANGLE PARK, N.C., June 06, 2020 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today presented findings from two patient surveys conducted to gain insights into patients’ current hereditary angioedema (HAE) treatment expectations, experience and satisfaction. Patient-reported attack history shows patients treated with current injectable or infused prophylactic medications (Takhzyro®, Haegarda®, Cinryze®) continue to experience breakthrough attacks with a mean ranging from 0.9 to 1.8 attacks over the three months prior to survey.

In addition, the majority of patients surveyed did not expect to have zero attacks even when taking prophylaxis therapy.

The data were presented at the European Academy of Allergy and Clinical Immunology (EAACI) Digital Congress.

“While the injectable and infused medications introduced over the past 12 years have provided added prophylactic attack control for HAE patients, most patients in the study continue to experience breakthrough attacks, and medication adherence continues to be a challenge. HAE patients have indicated an interest in new preventative treatment options, with the majority agreeing that they would prefer an oral medication, despite liking their current prophylactic medication,” said Jinky Rosselli, vice president of global business analysis and operations at BioCryst, who conducted the research.  

Study Methods and Results
Study participants were U.S. adult patients with a diagnosis of Type I or Type II HAE.  Patients participated in surveys in 2018 (n=75) and 2019 (n=100). Two patients participated in both surveys. The patient surveys were conducted anonymously in compliance with the EphMRA code of conduct. 

  • In the 2019 survey, 85 percent of patients reported taking at least one medicine to prevent HAE attacks, compared to 64 percent in the 2018 survey.
  • The majority (89 percent) of patients using prophylaxis in the 2019 survey reported using at least one of the three most commonly prescribed injectable or infused prophylactic therapies.
  • Over the three months prior to the survey, these patients reported a mean of 0.9, 1.6 and 1.8 attacks while taking Haegarda®, Cinryze® and Takhzyro®, respectively.  Patients taking these injectable or infused therapies reported similar attack rates in the 2018 study.

  • In the 2019 survey, 21 percent of patients using prophylaxis reported the expectation to have zero attacks over the next 12 months.

About BioCryst Pharmaceuticals
BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. BioCryst has several ongoing development programs including berotralstat (BCX7353), an oral treatment for hereditary angioedema, BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, galidesivir, a potential treatment for COVID-19, Marburg virus disease and Yellow Fever, and BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva. RAPIVAB® (peramivir injection), a viral neuraminidase inhibitor for the treatment of influenza, is BioCryst's first approved product and has received regulatory approval in the U.S., Canada, Australia, Japan, Taiwan, Korea and the European Union. Post-marketing commitments for RAPIVAB are ongoing. For more information, please visit the Company's website at

Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding future results, performance or achievements. These statements involve known and unknown risks, uncertainties and other factors which may cause BioCryst’s actual results, performance or achievements to be materially different from any future results, performances or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: that the ongoing COVID-19 pandemic could create challenges in all aspects of our business, including without limitation delays, stoppages, difficulties and increased expenses with respect to our and our partners’ development, regulatory processes and supply chains, could negatively impact our ability to access the capital or credit markets to finance our operations, or could have the effect of heightening many of the risks described below or in the documents we file periodically with the Securities and Exchange Commission; that developing any HAE product candidate may take longer or may be more expensive than planned; that ongoing and future preclinical and clinical development of BCX9930, BCX9250 and galidesivir may not have positive results; that BioCryst may not be able to enroll the required number of subjects in planned clinical trials of product candidates; that BioCryst may not advance human clinical trials with product candidates as expected; that the FDA, EMA, PMDA or other applicable regulatory agency may require additional studies beyond the studies planned for product candidates, or may not provide regulatory clearances which may result in delay of planned clinical trials, or may impose a clinical hold with respect to such product candidates, or withhold market approval for product candidates; that actual financial results may not be consistent with expectations, including that 2020 operating expenses and cash usage may not be within management's expected ranges.  Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst’s most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, all of which identify important factors that could cause the actual results to differ materially from those contained in BioCryst’s projections and forward-looking statements.


John Bluth
+1 919 859 7910