BioCryst Advancing BCX9930, an Oral Factor D Inhibitor for Complement-Mediated Diseases, into Phase 1 Development
March 04 2019 - 7:00AM
—BCX9930 shows high potency, specificity,
suppression of hemolysis and wide safety margin in preclinical
studies—
BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that
the company is advancing BCX9930, an oral Factor D inhibitor
discovered and developed by BioCryst, into Phase 1 clinical
development in the second quarter of 2019 for the treatment of
complement-mediated diseases.
The company plans to initiate a Phase 1 trial to
study single and multiple ascending doses of oral BCX9930 in
healthy subjects in the second quarter of 2019, and to report the
results in the fourth quarter of 2019.
“Based on its outstanding preclinical profile,
with high potency, excellent specificity for Factor D, complete
suppression of complement-mediated hemolysis after oral dosing, and
a wide safety margin, we are excited to advance oral BCX9930 into
clinical development,” said Dr. William Sheridan, chief medical
officer of BioCryst.
“The BioCryst R&D team has established a
proven and proprietary expertise to discover and develop first in
class or best in class oral medicines for rare diseases.
These are difficult targets to design oral medicines for and we
have now successfully developed multiple compounds for three rare
diseases, hereditary angioedema (HAE), complement-mediated diseases
and fibrodysplasia ossificans progressiva (FOP),” said Jon
Stonehouse, chief executive officer of BioCryst.
Preclinical Profile of
BCX9930
- In preclinical in-vivo studies of complement activity in the
blood, oral dosing of BCX9930 completely suppressed
complement-mediated hemolysis.
- In preclinical in-vitro studies of red blood cells from
patients with paroxysmal nocturnal hemoglobinuria (PNH), BCX9930
completely suppressed complement-mediated hemolysis, and completely
blocked deposition of complement enzyme 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.
“Existing IV infusion therapy for
complement-mediated diseases currently generates more than $3
billion in annual global sales with approvals in just a few of the
many potential disease areas. With full global rights to a
BioCryst-invented oral Factor D inhibitor, and relatively quick
proof of concept, we look forward to advancing BCX9930 to address
multiple complement-mediated diseases,” Stonehouse
added.
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 advancing into 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 initiate a Phase 1 clinical trial of oral BCX9930
in the second quarter of 2019 and to report the results in the
fourth quarter of 2019.
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 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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