BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that
hereditary angioedema (HAE) patients who were randomized to receive
150 mg of oral, once-daily ORLADEYO® (berotralstat) at the start of
the APeX-2 trial had an 80 percent average reduction in their mean
attack rate per month during weeks 25-96 of the trial, compared to
baseline. Median attack rates also decreased from 2.7 attacks/month
at baseline to 0.0 attacks per month in 16 of 17 months through the
same period.
ORLADEYO was generally well-tolerated during the
treatment period with fewer drug-related adverse events
reported in part 3 (weeks 49-96) as compared to part 1 (weeks 0-24)
and part 2 (weeks 25-48). Eighty-one percent of the patients who
entered part 3 completed the trial.
“The long-term data we now see from two years of
therapy in the clinical program reinforces the substantial,
sustained reduction in HAE attacks patients experienced with
ORLADEYO. These results are consistent with the experience many
patients are having in the real world since we launched ORLADEYO,
which is a key driver for the strong patient demand we are seeing
as patients switch from injectable prophylactic agents and
injectable acute-only therapies to oral, once-daily ORLADEYO for
control of their HAE attacks,” said Dr. William Sheridan, chief
medical officer of BioCryst.
The data are scheduled to be presented in an oral presentation
on Monday, July 12, 2021 at 2:15pm CET at the EAACI Hybrid Congress
2021.
Additional Presentations of New ORLADEYO
Data at EAACI
On-demand medication use was reduced in HAE patients
treated with ORLADEYO (150 mg) in APeX-2
- In HAE patients taking oral,
once-daily ORLADEYO 150 mg who had a ≥50 percent reduction in their
rate of investigator-confirmed attacks relative to their baseline
attack rate, there was a 78 percent reduction in the use of
on-demand medication (doses/month) from baseline to week 24,
leading to 2.1 fewer doses of on-demand medication per month. In
patients who had a ≥70 percent reduction in their rate of
investigator-confirmed attacks relative to their baseline attack
rate, there was an 85 percent reduction in the use of on-demand
medication (doses/month) from baseline to week 24, leading to 2.2
fewer doses of on-demand medication per month.
- The data were presented in poster #250 at the EAACI Hybrid
Congress 2021.
ORLADEYO demonstrated consistently low attack rates in
adolescent patients in APeX-S
- In an analysis of adolescent patients (ages 12-17) treated with
oral, once-daily ORLADEYO 150 mg in the open-label safety study,
APeX-S, the mean (SEM) attack rate at week 4 was 0.4 attacks/month,
which was generally sustained through week 48.
- Median attack rates in these adolescents were 0.0 attacks/month
throughout the 48 weeks of treatment.
- Greater than 70 percent of patients were attack-free in weeks 4
to 48.
- ORLADEYO was generally well-tolerated in APeX-S.
- The data were presented in poster #336 at the EAACI Hybrid
Congress 2021.
ORLADEYO demonstrated consistently low HAE attack rates
during COVID-19
- Stress is a documented trigger for
HAE attacks and recently published physician and patient survey
data shows an increase in patient-reported HAE attack rates due to
an increase in stress related to the COVID-19 pandemic (pre-COVID:
1.5 attacks/3-months vs during COVID: 4.4 attacks/3-months).1
- In an analysis of HAE patients
taking oral, once-daily ORLADEYO 150 mg in the APeX-S trial,
monthly HAE attack rates, pre-COVID and during COVID, remained
consistently low, <1 attack/month, for patients treated with
ORLADEYO. Patients receiving ORLADEYO maintained low attack rates
during this time of high societal stress and disruption.
- The data were presented in poster #166 at the EAACI Hybrid
Congress 2021.
About ORLADEYO®
(berotralstat)
ORLADEYO® (berotralstat) is the
first and only oral therapy designed specifically to prevent
attacks of hereditary angioedema (HAE) in adults 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) 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, the European Union, Japan and the United Kingdom for the
prevention of HAE attacks in adults and pediatric patients 12 years
and older. BioCryst has several ongoing development programs
including BCX9930, an oral Factor D inhibitor for the treatment of
complement-mediated diseases, BCX9250, an ALK-2 inhibitor for the
treatment of fibrodysplasia ossificans progressiva, and
galidesivir, a potential treatment for Marburg virus disease and
Yellow Fever. RAPIVAB® (peramivir injection), a viral
neuraminidase inhibitor for the treatment of influenza, has
received regulatory approval in the U.S., Canada, Australia, Japan,
Taiwan and Korea. 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 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, EMA,
PMDA 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 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.
BCRXW
Contact:InvestorsJohn Bluth+1 919
859 7910jbluth@biocryst.com
MediaCatherine Collier
Kyroulis+1 917 886 5586ckyroulis@biocryst.com
_____________________________
1 Soteres DF, et al. Update on the impact of COVID-19 in the
care of patients with Hereditary Angioedema (HAE): Results of a
Patient and HCP Survey. Presented at Academy of Managed Care
Pharmacy; April 12-16, 2021; Virtual. Abstract D21.
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