BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today announced that
data from part 1 of a clinical trial of its broad-spectrum
antiviral, galidesivir, showed that galidesivir was safe and
generally well tolerated in patients infected with SARS-CoV-2, the
virus that causes COVID-19. The trial was not designed or sized to
demonstrate clinical efficacy and no clinical efficacy benefit with
galidesivir treatment compared to placebo treatment was observed in
the trial.
Based on the company’s ongoing discussions with
the National Institute of Allergy and Infectious Diseases (NIAID),
part of the National Institutes of Health, a major funding partner
for the program, BioCryst expects NIAID to continue their support
for the development of galidesivir with a focus on biodefense
threats, such as Marburg virus disease, and to discontinue the
pursuit of a COVID-19 indication for galidesivir.
“We are encouraged by the first patient data
with galidesivir and the growing body of evidence that galidesivir
could be an important broad-spectrum antiviral. We appreciate the
government’s continued investment to investigate galidesivir as a
biodefense drug,” said Jon Stonehouse, chief executive officer of
BioCryst.
BioCryst is engaged in ongoing discussions with
NIAID to define specific further galidesivir studies that NIAID
would support.
Part 1 Trial DesignPart 1 of
the trial enrolled 24 hospitalized adults diagnosed with moderate
to severe COVID-19 confirmed by PCR. Three cohorts of eight
patients were randomized to receive intravenous galidesivir (n=6)
or placebo (n=2) every 12 hours for seven days.
Each dosing regimen began with a single 10 mg/kg
or 20 mg/kg loading dose, followed by a maintenance dose of 2 mg/kg
or 5 mg/kg administered twice daily. The three dosing regimens
evaluated were 10 mg/kg then 2 mg/kg (cohort 1), 10 mg/kg then 5
mg/kg (cohort 2) and 20 mg/kg then 5 mg/kg (cohort 3).
The trial was conducted in Brazil under a U.S.
investigational new drug application. The protocol also was
approved by the Agência Nacional de Vigilância Sanitária (ANVISA)
and the Brazilian National Ethics Committee (CONEP).
Part 1 ResultsThe primary
objective of part 1 of the dose-ranging study was to evaluate the
safety of galidesivir. No safety signals were identified, and all
three dose levels were equally safe.
Secondary objectives were to evaluate the effect
of galidesivir on the clinical course of COVID-19 and on SARS-CoV-2
infection in the respiratory tract.
Galidesivir treatment was associated with a more
rapid decline in viral RNA levels in the respiratory tract in an
apparent dose-dependent
manner. A separate
study of galidesivir in a COVID-19 animal model showed that early
administration of galidesivir reduced SARS-CoV-2 viral burden in
lung tissue (1.4-1.6 log lower tissue viral burden) and was
associated with a significant reduction in damage to lung tissue,
compared to vehicle control treated animals. These results suggest
that early antiviral treatment of SARS-CoV-2 infection may protect
against developing severe COVID-19 lung disease.
NIAID is a major funding partner of the galidesivir program
under contracts 75N93020C00055 and HHSN272201300017C.
About Galidesivir
(BCX4430)Galidesivir, a broad-spectrum antiviral drug, is
an adenosine nucleoside analog that acts to block viral RNA
polymerase. It is in advanced development for the treatment of
Marburg virus disease. In animal studies, galidesivir has
demonstrated activity against a variety of serious pathogens,
including, Ebola, Marburg, Yellow Fever and Zika viruses.
Galidesivir has also demonstrated broad-spectrum activity in vitro
against more than 20 RNA viruses in nine different families,
including coronaviruses, filoviruses, togaviruses, bunyaviruses,
arenaviruses, paramyxoviruses, and flaviviruses. BioCryst is
developing galidesivir in collaboration with U.S. government
agencies and other institutions.
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 for the prevention
of HAE attacks in adults and pediatric patients 12 years and older,
and under regulatory review for approval in Japan and the European
Union. BioCryst has several ongoing development programs including
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 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 and Korea. 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
BioCryst’s plans and expectations for its galidesivir development
program. These statements involve known and unknown risks,
uncertainties and other factors which may cause actual results and
developments of such program to be materially different from any
future results or developments expressed or implied by the
forward-looking statements. These statements reflect our current
views and are based on assumptions and 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: NIAID’s ability to determine not to exercise
available options under its contracts with BioCryst or to terminate
such contracts at any time, causing BioCryst not to realize the
aggregate value of the contracts; funding for galidesivir under
government contracts is dependent on the progress toward, and the
achievement of, developmental milestones; the ongoing COVID-19
pandemic 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 herein or in the documents
BioCryst files periodically with the Securities and Exchange
Commission; developing and manufacturing any product candidate,
including galidesivir, may take longer or may be more expensive
than planned; funding for the continued development and manufacture
of galidesivir may not be available; ongoing and future preclinical
and clinical studies with galidesivir may not have positive
results; BioCryst may not be able to enroll the required number of
subjects in planned clinical trials of product candidates,
including galidesivir; BioCryst may not advance human clinical
trials with product candidates, including galidesivir, as expected;
and the FDA, 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 a clinical hold
with respect to such product candidates, or may withhold 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 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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