BioCryst to Participate in the 2023 US HAEA National Summit
RESEARCH TRIANGLE PARK, N.C. – July 17, 2023 – BioCryst Pharmaceuticals, Inc.
(Nasdaq: BCRX) today announced its participation at the 2023 US Hereditary Angioedema Association
(HAEA) National Summit, which will include the presentation of three abstracts featuring data on oral,
once-daily ORLADEYO® (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE).
The US HAEA National Summit is being held in Orlando, Fla., from July 20-23, 2023.
“We are very excited and grateful to be a part of the US HAEA community gathering again for the
first time since the COVID pandemic. This community played a critical role in helping our company
introduce the first oral, once-daily treatment for the prevention of HAE attacks. While this is a big
step forward for patients, we are just getting started and plan to continue to work tirelessly for this
community,” said Jon Stonehouse, president and chief executive officer of BioCryst.
The three abstracts that BioCryst will present at the US HAEA National Summit are:
• Once-daily oral berotralstat led to improvements in work productivity and activity impairment in
patients with hereditary angioedema: Results from the APeX-2 randomized Phase III trial; Poster
#19; Professional Scientific Track; Friday, July 21, 2023, 7:45 am-12:20 pm ET
• Disease and Treatment Burden of Hereditary Angioedema (HAE) in Pediatric Patients:
Assessment by Caregivers; Poster #4; Professional Scientific Track; Friday, July 21, 2023, 7:45
am-12:20 pm ET
• Attack-free Status in Patients who Switched from Subcutaneous Lanadelumab to Oral
Berotralstat; Poster #1; Professional Scientific Track; Friday, July 21, 2023, 7:45 am-12:20 pm
ET
About ORLADEYO® (berotralstat)
ORLADEYO® (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of
hereditary angioedema (HAE) in adult and pediatric patients 12 years and older. One capsule of
ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein.
U.S. Indication and Important Safety Information
INDICATION
ORLADEYO® (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks
of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.
Limitations of use
The safety and effectiveness of ORLADEYO for the treatment of acute HAE attacks have not been
established. ORLADEYO should not be used for the treatment of acute HAE attacks. Additional doses or
dosages of ORLADEYO higher than 150 mg once daily are not recommended due to the potential for QT
prolongation.
IMPORTANT SAFETY INFORMATION
An increase in QT prolongation was observed at dosages higher than the recommended 150 mg once-
daily dosage and was concentration dependent.
The most common adverse reactions (≥10% and higher than placebo) in patients receiving ORLADEYO
were abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease.
A reduced dosage of 110 mg taken orally once daily with food is recommended in patients with moderate
or severe hepatic impairment (Child-Pugh B or C) and in patients taking chronically administered P-
glycoprotein (P-gp) or breast cancer resistance protein (BCRP) inhibitors (eg, cyclosporine).
Berotralstat is a substrate of P-gp and BCRP. P-gp inducers (eg, rifampin, St. John’s wort) may decrease
berotralstat plasma concentration, leading to reduced efficacy of ORLADEYO. The use of P-gp inducers
is not recommended with ORLADEYO.
ORLADEYO at a dose of 150 mg is a moderate inhibitor of CYP2D6 and CYP3A4. For concomitant
medications with a narrow therapeutic index that are predominantly metabolized by CYP2D6 or CYP3A4,
appropriate monitoring and dose titration is recommended. ORLADEYO at a dose of 300 mg is a P-gp
inhibitor. Appropriate monitoring and dose titration is recommended for P-gp substrates (eg, digoxin)
when coadministering with ORLADEYO.
The safety and effectiveness of ORLADEYO in pediatric patients <12 years of age have not been
established.
There are insufficient data available to inform drug-related risks with ORLADEYO use in pregnancy.
There are no data on the presence of berotralstat in human milk, its effects on the breastfed infant, or its
effects on milk production.
To report SUSPECTED ADVERSE REACTIONS, contact BioCryst Pharmaceuticals, Inc. at 1-
833-633-2279 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please see full Prescribing Information.
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. Oral, once-daily ORLADEYO® (berotralstat) is approved in the United States and many
global markets. BioCryst has active programs to develop oral medicines for multiple targets across the
complement system, including BCX10013, an oral Factor D inhibitor in clinical development.
RAPIVAB® (peramivir injection) is approved in the U.S. and multiple global markets, with post-
marketing commitments ongoing. For more information, please visit the company’s website at
www.biocryst.com.
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 actual results, performance or achievements to be materially different from
any future results, performance 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
periodically files with the Securities and Exchange Commission; BioCryst’s ability to successfully
implement its commercialization plans for, and to commercialize, ORLADEYO, which could take longer
or be more expensive than planned; the commercial viability of ORLADEYO, including its ability to
achieve market acceptance; the FDA or other applicable regulatory agency may require additional studies
beyond the studies planned for products and 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 products and product candidates, may impose a clinical hold with respect to product
candidates, or may withhold, delay, or withdraw market approval for products and product candidates;
BioCryst’s ability to successfully 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 revenue, 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, which identify important factors that could
cause actual results to differ materially from those contained in BioCryst’s forward-looking statements.
BCRXW
Contact:
John Bluth
+1 919 859 7910
jbluth@biocryst.com