BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today announced data from a cross-sectional study among patients, caregivers and physicians capturing the burden of injectable prophylactic therapy experienced by hereditary angioedema (HAE) patients and caregivers, and differences in perceptions between physicians and HAE patients.

The three abstracts were presented at the 2020 Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI), which is being conducted virtually from November 13-15.

Poster presentations:

• Patient Perspectives on the Treatment Burden of Injectable Medication Administration for Hereditary Angioedema (#160)
• Prophylactic Treatment Burden: Assessment by Caregivers of Patients with Hereditary Angioedema (#161)
• Understanding Differences in Perceptions of Hereditary Angioedema Treatment Burden May Improve Patient-Physician Treatment Care Dialogue (#162)

“These data are consistent across HAE patients, caregivers and treating physicians showing many patients experience a significant treatment burden associated with current prophylactic HAE therapies. New therapies with easier routes of administration may meet a significant unmet need for HAE patients seeking improved quality of life,” said study lead Cristine Radojicic, M.D., assistant professor of medicine at Duke University School of Medicine.

Overall, the burden of treatment reported across all groups surveyed suggests an unmet need still remains in HAE clinical management. These study findings collectively highlight the opportunity to strengthen the shared decision making between patients and physicians with more effective dialogue about the burden of treatment and patients' individual needs and preferences.

Following is a brief summary of the data from the cross-sectional study conducted via three double-blinded surveys with HAE patients (n=75), caregivers (n=30) and physicians (n=109), respectively:

• Almost nine in 10 patients with HAE report they have learned to tolerate difficult aspects of their treatment and 58 percent report they are tired of their injections. Even though patients are satisfied with their current prophylactic medications, 86 percent are still interested in a less burdensome route of administration.
• Over 50 percent of caregivers agree it was challenging to learn how to administer HAE treatment, specifically gaining comfort with using needles and learning how to self-administer. Seventy-one percent of caregivers agree that patients experience needle fatigue with their HAE prophylactic medications and an even greater proportion of caregivers believe a once-daily pill would provide the patient more freedom (86 percent), independence (85 percent), and reduce caregivers’ burden.
• Most physicians (94 percent) and patients (84 percent) agree there is a need for newer and more novel HAE treatments. In addition, 86 percent of caregivers believe that, while their patient is satisfied with current treatment, the patient would still be interested in one that is easier to administer.
• Over 70 percent of physicians surveyed believe that starting prophylaxis treatment was overwhelming, becoming comfortable with needles was intimidating, and learning how to self-administer was challenging for their patients. The study also shows that physicians tend to underestimate time required for preparation and administration of prophylaxis medications. Importantly, despite recognition of the burden with current treatments, there is discordance between patients and physicians regarding the person initiating conversations about medication challenges, suggesting an opportunity to improve the dialogue to help with an individualized approach to the management of HAE.

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 ORLADEYO™ (berotralstat), 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 www.BioCryst.com.

Forward-Looking StatementsThis 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: the ongoing COVID-19 pandemic, which could create challenges in all aspects of BioCryst’s business, including without limitation delays, stoppages, difficulties and increased expenses with respect to BioCryst’s and its partners’ development, regulatory processes and supply chains, negatively impact BioCryst’s ability to access the capital or credit markets to finance its operations, or have the effect of heightening many of the risks described below or in the documents BioCryst files periodically with the Securities and Exchange Commission; developing and commercializing ORLADEYO or any HAE product candidate may take longer or may be more expensive than planned; BioCryst may not be able to enroll the required number of subjects in planned clinical trials of product candidates; BioCryst may not advance human clinical trials with product candidates as expected; the FDA, EMA, PMDA or other applicable regulatory agency may require additional studies beyond the studies planned for product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may impose certain restrictions, warnings, or other requirements on product candidates, may impose a clinical hold with respect to such product candidates, or may withhold market approval for product candidates; product candidates, if approved, may not achieve market acceptance; BioCryst’s ability to successfully commercialize its product candidates, manage its growth, and compete effectively; risks related to the international expansion of BioCryst’s business; and 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 forward-looking statements.

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Contact:John Bluth+1 919 859 7910jbluth@biocryst.com

Catherine Collier Kyroulis+1 917 886 5586ckyroulis@biocryst.com