Long-term (48-week) Data Show Treatment with Berotralstat Provides Robust and Durable Reductions in HAE Attacks and Improveme...
June 06 2020 - 3:00AM
BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today announced new
data from the APeX-2 and APeX-S clinical trials, which showed that
hereditary angioedema (HAE) patients taking oral, once-daily
berotralstat experienced sustained decreases in their attack
frequency and improvements in quality of life (QoL) scores over 48
weeks. Berotralstat was also safe and generally well-tolerated over
48 weeks in both APeX-2 and APeX-S.
The data were presented at the European Academy
of Allergy and Clinical Immunology (EAACI) Digital Congress.
“As HAE patients continue in our long‑term
clinical trials, we are seeing reductions in attack rate and
increases in QoL scores which highlight the impact oral, once-daily
berotralstat could have in reducing the burden of disease for
patients currently dependent on injectable or infused prophylaxis
options,” said Dr. William Sheridan, chief medical officer of
BioCryst.
- In APeX-2, 31 patients who were
randomized to 150 mg of oral, once-daily berotralstat at the
beginning of the study and completed 48 weeks of therapy had a mean
baseline attack rate of 2.9 attacks per month, which declined to
1.5 attacks per month after one month and to 1.0 attack per month
at 12 months.
- In APeX-S, patients completing 48
weeks of treatment on 150 mg of berotralstat (n=73) had a median
attack rate of zero attacks per month in six of the 12 months,
including month 12 (week 48).
- The low attack rate experienced by
HAE patients on 150 mg of oral, once-daily berotralstat reduced the
burden of disease and translated into clinically meaningful
improvements in mean angioedema quality of life (AE-QoL) total
score, as measured by the disease-specific AE-QoL questionnaire.
This persisted through month 12 (week 48) in the APeX-S
trial.
- Improvements in mean change from
baseline AE-QoL total score exceeding the minimum clinically
important difference (MCID) of six points were observed by week
four and at week 48 the mean AE-QoL total score for the
berotralstat 150-mg treatment group had decreased by 14.7 points
compared with baseline.
An integrated 48-week analysis across both
APeX-2 and APeX-S showed no new safety findings. Berotralstat was
safe and generally well tolerated in a total of 342 patients with a
total of 232 patient-years of daily oral dosing. The most common
adverse event was the common cold, which occurred with similar
frequency in berotralstat and placebo patients. Gastrointestinal
events led to discontinuation of berotralstat in 3.2 percent of
patients. Drug-related serious adverse events occurred in three of
342 subjects (0.9 percent) and resolved after stopping or
interrupting berotralstat dosing.
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 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: 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.
BCRXW
Contact:John Bluth+1 919 859
7910jbluth@biocryst.com
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