Biocryst Begins Enrollment of Phase 1 Trial of BCX9930, an Oral Factor D Inhibitor for Complement-Mediated Diseases
June 27 2019 - 4:15PM
BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that
the company has begun enrollment of a Phase 1 trial of BCX9930, an
oral Factor D inhibitor discovered and developed by BioCryst, for
the treatment of complement-mediated diseases.
The objectives of the trial are to evaluate the
safety and tolerability of single and multiple ascending doses of
BCX9930 in healthy subjects and to characterize the pharmacokinetic
and pharmacodynamic profiles of BX9930 in single and multiple
ascending doses of BCX9930 in healthy subjects.
The company expects to report data from the
trial in the fourth quarter of 2019.
“An oral Factor D inhibitor would meet a
significant unmet medical need for patients with many
complement-mediated diseases. The preclinical profile of BCX9930
showed high potency, excellent specificity for Factor D, complete
suppression of complement-mediated hemolysis after oral dosing, and
a wide safety margin, so we look forward to seeing the clinical
results from this Phase 1 trial,” said Dr. William Sheridan, chief
medical officer of BioCryst.
“We are very excited to begin enrollment of our
Phase 1 trial of BCX9930, another proprietary BioCryst oral
medicine for rare diseases. With complement-mediated diseases, we
can rapidly gather important data from our Phase 1 trial regarding
the effect on the complement system, and we will use these results
to direct our plans to generate proof of concept data in PNH
patients next year,” said Jon Stonehouse, chief executive officer
of BioCryst.
Preclinical Profile of
BCX9930
- In preclinical oral dosing studies, BCX9930 completely
suppressed complement-mediated hemolysis in laboratory assays of
complement activity.
- In preclinical in-vitro studies of red blood cells from
patients with paroxysmal nocturnal hemoglobinuria (PNH), BCX9930
completely suppressed complement-mediated hemolysis, and completely
blocks the deposition of C3 fragments on PNH red blood
cells.
- In preclinical in-vitro studies, BCX9930 demonstrated
significant activity at low drug concentrations in several
well-established complement activity assays.
- In preclinical in-vitro assays, BCX9930 was more potent on
Factor D by approximately 200-fold to more than 3000-fold compared
with other serine protease enzymes outside the complement
pathway.
- In preclinical oral dosing studies, drug exposure increased in
proportion to dose, and high drug levels were achieved.
- In preclinical in-vivo safety pharmacology and toxicology
studies, drug concentrations at the no observed adverse event level
(NOAEL) doses of BCX9930 were more than 500-fold greater than the
estimated therapeutic target level.
About Complement-Mediated
Diseases
The complement system is part of the body’s
natural immune system and is responsible for helping the body
eliminate microbes (including viral and bacterial infections) and
damaged cells. It is comprised of proteins which are primarily
produced in the liver and circulate in the blood. Once activated,
the complement system stimulates inflammation, phagocytosis and
cell lysis.
Excessive or uncontrolled activation of the
complement system can cause severe, and potentially fatal, immune
and inflammatory disorders.
The complement system comprises biological
cascades of amplifying enzyme cleavages involving more than 30
proteins and protein fragments, and may be activated through three
pathways: the classical pathway (initiated by antibody-antigen
complexes), the lectin pathway (initiated by lectin binding) and
the alternative pathway (initiated by microbial surfaces).
The alternative pathway also provides a critical
amplification loop for all three pathways, regardless of the
initiating mechanism. Factor D is an essential enzyme in the
alternative pathway, thus making Factor D an attractive target to
address complement-mediated diseases.
About BCX9930
Discovered by BioCryst, BCX9930 is a novel,
oral, potent and selective small molecule inhibitor of
Factor D currently in Phase 1 clinical development for the
treatment of complement-mediated diseases. Patients with
complement-mediated diseases, many of which can cause death or
severe morbidity, currently either have no treatments available, or
are limited to repeated intravenous infusion treatments. The
company plans to report results from the ongoing Phase 1
trial in the fourth quarter of 2019.
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 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 developing BCX9930 may take longer
or may be more expensive than planned; that ongoing and future
preclinical and clinical development of BCX9930 may not advance as
expected, enroll the required number of subjects or have positive
results; that the FDA, EMA or other applicable regulatory agency
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 BCX9930. 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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