BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that
the randomized (n=121), double-blind, placebo-controlled, Phase 3
APeX-2 trial of once-daily, oral BCX7353 for the prevention of
hereditary angioedema (HAE) attacks achieved its primary endpoint
for both dose levels (110 mg and 150 mg), with the 150 mg dose
reducing the attack rate in HAE patients by 44 percent (p<0.001)
compared to placebo.
Fifty percent of patients receiving 150 mg
BCX7353 in APeX-2 had a ≥ 70 percent reduction in their HAE attack
rate compared to baseline, compared to 15 percent of placebo
patients (p=0.002).
In patients on the 150 mg dose with a baseline
attack rate of < 2 attacks per month, BCX7353 reduced the HAE
attack rate by 66 percent compared to placebo (p=0.009). In
patients with a baseline attack rate of ≥ 2 attacks per month, the
attack rate was reduced by 40 percent (p=0.005).
Of 108 patients who completed 24 weeks of study
drug treatment, 100 percent continued into the ongoing 48 week
extension phase of the trial.
In APeX-2, both the 110 mg and 150 mg dose
levels of once-daily oral BCX7353 were generally safe and
well-tolerated. No drug-related serious adverse events were
reported.
The most common drug-related adverse events
reported in at least five percent of patients in APeX-2 were:
nausea (9.8% 110 mg, 7.5% 150 mg, 15.4% placebo), dyspepsia (9.8%
110 mg, 7.5% 150 mg, 5.1% placebo) and diarrhea (7.3% 110 mg, 10%
150 mg, 0% placebo).
“HAE patients around the world desperately want
access to a cost-effective, convenient, oral therapy to manage
their disease. Given the profile of the 150 mg dose of BCX7353 in
APeX-2, with half of patients experiencing at least a 70 percent
reduction in attack rate, we have a new oral therapy that patients
will want to try,” said Jon Stonehouse, chief executive officer of
BioCryst.
“With successful results from APeX-2, BioCryst
is committed to making it easy for HAE patients around the world to
access this potentially life-changing oral therapy, and we believe
BCX7353 is positioned to become a front-line therapy option,”
Stonehouse added.
The results from APeX-2 support the submission
of a new drug application (NDA) to the U.S. Food and Drug
Administration (FDA). BioCryst plans to submit an NDA to the FDA in
the fourth quarter of 2019 and a Marketing Authorization
Application (MAA) to the European Medicines Agency (EMA) in the
first quarter of 2020.
“The additional clinical information we now have
from APeX-2 confirms that this is an oral kallikrein inhibitor that
is effective at preventing HAE attacks in a large segment of the
HAE patient population while having a very attractive tolerability
profile. Based on this profile, and the consistent observation that
real world efficacy has been higher than clinical trial efficacy
with HAE therapies, I expect many patients will want to try this
oral option to see how well it works for them,” said Bruce Zuraw,
M.D., professor of medicine and chief of the Division of
Rheumatology, Allergy and Immunology at the University of
California School of Medicine, and principal investigator of the
APeX-2 trial.
The company plans to submit detailed results
from the APeX-2 trial for peer‑reviewed publication and
presentation.
About APeX-2APeX-2 is a
randomized, double-blind, placebo-controlled, three-arm trial
testing two dose levels of orally administered once-daily BCX7353
(110 mg and 150 mg) for prevention of angioedema attacks. The trial
enrolled 121 patients with Type I and II HAE in the United States,
Canada and Europe.
The primary efficacy endpoint of APeX-2 is the
rate of investigator confirmed angioedema attacks over 24 weeks of
study drug administration. Following study qualification during a
run-in period of 14 to 56 days, patients were randomized 1:1:1 to
receive placebo or one of the two doses of BCX7353. Randomization
was stratified on a baseline attack rate of <2/month or
≥2/month. Forty-one patients were randomized to 110 mg BCX7353, 40
to 150 mg BCX7353, and 40 to placebo. There was a clear dose
response, with the 110 mg dose of BCX7353 reducing HAE attack rate
by 30 percent (p=0.024) compared to placebo. As noted above, the
150 mg dose reduced attack rate by 44 percent (p<0.001) compared
to placebo.
To qualify for the trial, patients were required
to have a specified number of investigator-confirmed HAE attacks
during the run-in period of a maximum of 56 days from the screening
visit. The average baseline attack rate prior to randomization was
3.0 per 28 days.
Following completion of the 24 week analysis
period, patients continued on study drug in an ongoing extension
phase of APeX-2 through 48 weeks. Patients randomized to placebo
for 24 weeks were re-randomized to receive one of the two doses of
study drug in the extension phase of the trial. Patients who
complete 48 weeks may continue in the trial on open-label BCX7353
for up to 96 weeks.
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 3
APeX-2 and Phase 2 APeX-1 clinical trials. In APeX-2, BCX7353 (150
mg) reduced the HAE attack rate by 44 percent (p<0.001) compared
to placebo, and reduced the HAE attack rate by ≥ 70 percent in 50
percent of patients. BioCryst is currently conducting APeX-S, a
long-term safety clinical trial, and plans to submit an NDA to the
FDA in the fourth quarter of 2019 and an MAA to the EMA in the
first quarter of 2020. 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.
Conference Call and
WebcastBioCryst management will host a conference call and
webcast today, Tuesday, May 21, at 8:30 a.m. ET today to discuss
the APeX-2 results. The live call may be accessed by dialing
877-303-8027 for domestic callers and 760-536-5165 for
international callers and using conference ID # 8068168. A live
webcast of the call and any slides will be available online at the
investors section of the company website at www.biocryst.com. A
telephone replay of the call will be available by dialing
855-859-2056 for domestic callers or 404-537-3406 for international
callers and entering the conference ID# 8068168.
About BioCryst Pharmaceuticals
BioCryst 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
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 Marburg virus
disease and Yellow Fever, and a preclinical program to develop oral
ALK-2 inhibitors 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
developing BCX7353 may take longer or may be more expensive than
planned; that ongoing and future preclinical and clinical
development of BCX7353 may not advance as expected; that future
studies may not enroll the required number of subjects or have
positive results; and that the FDA, EMA or other applicable
regulatory agencies may require additional studies beyond the
studies planned, may not provide regulatory clearances, may impose
a clinical hold or may withhold market approval with respect to
BCX7353. 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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