—Based on ZENITH-1 data, company plans to
commence Phase 3 trial of single-dose 750 mg oral BCX7353 for acute
treatment of hereditary angioedema (HAE) attacks in summer
2019—
BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today reported
additional topline data from the Phase 2 ZENITH-1 trial, including
new data from the 250 mg and 500 mg dose cohorts. Data from the now
complete trial confirm previously reported results showing a single
dose of oral 750 mg BCX7353 was well-tolerated and superior to
placebo (p<0.05) against the majority of efficacy endpoints
evaluated in HAE patients suffering an acute attack, and
demonstrate a clear dose response across the three dose levels.
Based on the results of ZENITH-1, the company
plans to meet with the U.S. Food and Drug Administration (FDA) in
the second quarter, and to commence a Phase 3 trial with the 750 mg
dose of oral BCX7353 in the summer of 2019.
“The results of ZENITH-1, with onset of action
within one hour, duration of effect of a single dose over 24 hours,
and a robust efficacy dose response across all dose levels are very
exciting for patients who have an urgent need for an oral treatment
option for acute attacks,” said Dr. William Sheridan, chief medical
officer of BioCryst.
“Based on these excellent results, we plan to
quickly advance 750 mg oral BCX7353 into a Phase 3 trial that will
be designed to support approval in the U.S. and European Union,”
Sheridan added.
Efficacy and tolerability data for the 750 mg
dose cohort were previously reported by the company in a September
4, 2018 press release. With the 750 mg dose, compared to placebo,
improvement in symptoms and Visual Analog Scale (VAS) scores was
seen as early as one hour after oral BCX7353 dosing, and was
sustained through 24 hours. Through 24 hours, standard of care
(SOC) medication use was reduced by 31.6 percent after BCX7353
compared with placebo (p=0.0029), and no or mild symptoms were
reported in 64.1 percent of attacks treated with BCX7353 compared
with 32.3 percent of attacks treated with placebo (p=0.0038).
In the additional data reported today at the
AAAAI annual meeting, a clear dose response was observed across the
250 mg to 750 mg range. Across dose levels, BCX7353 was
generally safe and well-tolerated with no notable differentiation
from the adverse event profile of placebo.
The prior press release from September 4, 2018
containing safety and efficacy data from the 750 mg dose cohort,
and the poster containing the additional results presented today at
the AAAAI annual meeting, including figures highlighting dose
response and tolerability across all three dose levels, can be
found on the investor relations section of the company’s website
at: http://ir.biocryst.com/.
ZENITH-1 Trial Design
ZENITH-1 was a double-blind, placebo-controlled,
randomized, cross-over, dose-ranging trial of oral BCX7353 for
acute treatment of angioedema attacks in patients with HAE. A total
of 63 patients were randomized and 58 received at least one dose of
blinded study drug: 11 in the 250 mg cohort, 14 in the 500 mg
cohort and 33 in the 750 mg cohort.
ZENITH-1 was designed for compatibility with
modern treatment guidelines for at-home self-administered drug
administration as early as possible after onset of symptoms. The
goals of the trial were to identify activity against clinically
meaningful endpoints that the company could use to construct a
Phase 3 registration trial, to identify the dose or doses the
company could advance, and to assess safety and tolerability.
Adults with HAE Type I or II self-administered a
dose of blinded study drug for three attacks; two treated with
active drug and one with placebo, in a randomized sequence.
Subjects were asked to administer blinded study medicine within one
hour of onset of symptoms. Subjects were free to use approved
prescribed acute medications but were asked to wait at least four
hours post-study drug if possible. Patient completed trial diaries
to collect information on symptoms, VAS scores and use of SOC
medicines prior to dosing and at 1, 2, 3, 4, 8 and 24 hours after
study drug administration. In the 250 mg, 500 mg and 750 mg cohorts
respectively, a total of 21, 25 and 64 attacks were treated with
BCX7353 and 11, 11 and 31 attacks were treated with placebo.
About BCX7353Discovered by
BioCryst, BCX7353 is a novel, oral, once-daily, selective inhibitor
of plasma kallikrein currently in advanced clinical development for
the prevention and treatment of angioedema attacks in patients with
HAE. BCX7353 was generally safe and well tolerated in the Phase 2
APeX-1 clinical trial. BioCryst is currently conducting the Phase 3
APeX-2 clinical trial and the long-term safety APeX-S clinical
trial, each evaluating two dosage strengths of BCX7353 administered
orally once-daily as a preventive treatment to reduce the frequency
of attacks in patients with HAE. BioCryst has also completed the
ZENITH-1 clinical trial. ZENITH-1 was a proof-of-concept Phase 2
clinical trial testing oral BCX7353 for the treatment of acute
angioedema attacks.
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 of oral drugs for rare diseases including
BCX7353, an oral plasma kallikrein inhibitor for treatment of
hereditary angioedema; a preclinical program with an oral ALK-2
inhibitor for treatment of fibrodysplasia ossificans progressive,
and intravenous galidesivir, a broad-sepctrum viral RNA polymerase
inhibitor, as a potential treatment for Marburg virus disease and
Yellow Fever, under contracts from NIAID and HHS/BARDA. RAPIVAB®
(peramivir injection), an intravenous influenza virus 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 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 ongoing and future preclinical and
clinical development of HAE second generation drug candidates
(including APeX-2, APeX-S and APeX-J) may not have positive
results, may be more expensive or may not move as quickly as
planned; that the FDA, EMA or other applicable regulatory agency
may not provide regulatory clearances which may result in delay of
planned clinical trials or failure to achieve market approval for
product candidates. 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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