Allergy Publishes Results from BioCryst’s APeX-J Trial of Oral, Once-Daily Berotralstat for the Prevention of HAE Attacks
November 30 2020 - 7:00AM
BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced the
journal Allergy has published data from the APeX-J trial, a
randomized, placebo-controlled trial conducted in Japan evaluating
oral, once-daily berotralstat for the prophylactic treatment of
hereditary angioedema (HAE).
The APeX-J trial met its primary endpoint of a
reduction in the rate of HAE attacks for berotralstat 150 mg
compared to placebo during the 24-week period (p=0.003).
Berotralstat was safe and generally well-tolerated in the
trial.
These results are consistent with the global
phase 3 APeX-2 trial, where berotralstat 150 mg also reduced the
rate of HAE attacks compared to placebo (p<0.001) and was safe
and generally well-tolerated.
“APeX-J is the first placebo-controlled trial of
an HAE medicine conducted in Japan and the berotralstat data are
very exciting for patients, who currently have no approved
prophylactic treatment options. Based on the safety and efficacy
profile, I believe berotralstat, if approved, would be an important
advancement in HAE management for Japanese patients,” said Dr. Isao
Ohsawa, president of Saiyu Soka hospital and principal investigator
of the APeX-J trial.
Dr. Ohsawa and the study authors note that HAE
is estimated to affect 2,500 patients in Japan and the recognition
of HAE by physicians is low. Although two on-demand treatments are
approved, no therapies are currently approved for long-term
prophylaxis in Japan.
“Berotralstat would be the first approved
prophylactic therapy for HAE patients in Japan and we believe there
is a significant opportunity for berotralstat to accelerate the
diagnosis of HAE patients and dramatically improve the quality of
life for patients,” said Jon Stonehouse, president and chief
executive officer of BioCryst.
A new drug application (JNDA) is under review in
Japan for approval of oral, once-daily berotralstat for the
prophylactic treatment of HAE. Berotralstat is being reviewed under
Sakigake designation and the company expects a decision on approval
in December 2020.
Torii Pharmaceutical, Co., Ltd. is BioCryst’s
commercial partner in Japan for berotralstat.
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 Statements
This press release contains forward-looking
statements, including statements regarding future results,
performance or achievements. These statements are identified by use
of terms such as “believe,” “will,” “would,” “expect,” and similar
words, although some forward-looking statements may be expressed
differently. 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, 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 or delay 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; and
risks related to the international expansion of BioCryst’s
business. 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
Contacts:John
Bluth+1 919 859 7910jbluth@biocryst.com
Catherine Collier Kyroulis+1 917 886
5586ckyroulis@biocryst.com
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