BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced
that data from the open-label extension (OLE) of the APeX-2 trial
of oral, once-daily ORLADEYO® (berotralstat) for the prophylactic
treatment of hereditary angioedema (HAE) in patients 12 years and
older have been published online by the Journal of Allergy and
Clinical Immunology: In Practice (JACI: In Practice).
“As detailed in this manuscript, long-term
prophylaxis with ORLADEYO has enabled patients with HAE to better
manage their condition, including reducing the number of HAE
attacks they experience and demonstrating clinically meaningful
improvement in their quality of life. I have seen firsthand in my
practice evidence that the longer patients remain on ORLADEYO, the
better their outcomes are,” said William R. Lumry, M.D., clinical
professor of internal medicine at the University of Texas
Southwestern Medical School.
“We are pleased to share long-term efficacy and
safety data from APeX-2 as published in JACI: In Practice. These
data further illustrate the potential lasting outcomes that can be
appreciated by patients who are treated with oral, once-daily
ORLADEYO. We continue to see long-term safety and effectiveness
data that reinforce ORLADEYO as an important treatment option for
patients with HAE, and we look forward to sharing additional
real-world evidence at upcoming medical congresses,” said Dr. Ryan
Arnold, chief medical officer of BioCryst.
APeX-2 was a Phase 3, double-blind,
placebo-controlled, parallel-group, three-part study evaluating
ORLADEYO versus placebo for the prevention of HAE attacks in 121
patients with HAE Type I or Type II. In part 3 of the study (weeks
49-96), all patients (n=81) were treated with open-label ORLADEYO
at 150 mg. The primary endpoint of the OLE was long-term safety and
tolerability, while secondary endpoints included HAE attack rates
and quality of life (QoL).
The authors concluded that ORLADEYO was
generally well tolerated, provided rapid and
sustained reductions in HAE attacks and improved QoL over
the study duration of 96 weeks. The results from part 3 of APeX-2
were first presented at the European Academy of Allergy and
Clinical Immunology (EAACI) Congress in July 2021.
Additional details can be found in the
manuscript, which is available online at jaci-inpractice.org.
JACI: In Practice is an official journal of the American Academy of
Allergy, Asthma, and Immunology (AAAAI).
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
INDICATIONORLADEYO® (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 useThe 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).
Berotralstat is a substrate of P-glycoprotein
(P-gp) and breast cancer resistance protein. 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 is a global biotechnology company with a
deep commitment to improving the lives of people living with
complement-mediated and other rare diseases. BioCryst leverages its
expertise in structure-guided drug design to develop first-in-class
or best-in-class oral small-molecule and protein therapeutics to
target difficult-to-treat diseases. BioCryst has commercialized
ORLADEYO® (berotralstat), the first oral, once-daily plasma
kallikrein inhibitor, and is advancing a pipeline of small-molecule
and protein therapies. For more information, please visit
www.biocryst.com or follow us on LinkedIn.
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 files
periodically 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 the actual results to differ materially from those contained
in BioCryst’s forward-looking statements.
BCRXW
Contact:John Bluth+1 919 859
7910jbluth@biocryst.com
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