BioCryst’s Oral Factor D Inhibitor (BCX9930) Shows High Potency and Specificity for Alternative Pathway of Complement
December 06 2020 - 10:00AM
BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that
preclinical data on BCX9930, an oral Factor D inhibitor under
development as monotherapy for paroxysmal nocturnal hemoglobinuria
(PNH) and other complement-mediated diseases, showed complete in
vitro blockade of both hemolysis of PNH erythrocytes and
accumulation of C3 fragments on PNH erythrocytes, indicating that
BCX9930 monotherapy has the potential to inhibit both intravascular
and extravascular hemolysis.
In the study, BCX9930 was highly specific for
the alternative pathway and, after oral dosing of BCX9930 in
primates, alternative pathway activity was completely
suppressed.
The data were presented at the 62nd American
Society of Hematology (ASH) Annual Meeting being held as a virtual
event, December 5-8, 2020.
“These data demonstrate that BCX9930 is a highly
potent and specific orally bioavailable Factor D inhibitor with
potential for treatment of patients with PNH and other alternative
pathway mediated diseases,” said Dr. William Sheridan, chief
medical officer of BioCryst.
“The preclinical results have translated as
expected into the clinic, where we have seen oral monotherapy with
BCX9930 drive rapid and dose-dependent reductions in key
biomarkers, including LDH, and increase hemoglobin levels in PNH
patients. We look forward to completing our proof of concept study
in PNH and progressing into advanced development next year across
multiple hematology and nephrology complement- mediated diseases,”
Sheridan added.
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. The
U.S. Food and Drug Administration (FDA) has granted both Fast Track
status and Orphan Drug designation for BCX9930 in PNH. In an
ongoing dose ranging trial of BCX9930 in patients with PNH, BCX9930
was safe and well tolerated, with no drug-related serious adverse
events. As a Factor D inhibitor, BCX9930 is designed as an oral
monotherapy that can address both intravascular and extravascular
hemolysis in PNH patients. Treatment-naïve PNH patients who have
received more than six weeks of therapy at a monotherapy dose of
400 mg bid showed rapid and dose-dependent reductions in key
biomarkers, including LDH, and increases in hemoglobin levels that
were maintained without transfusions.
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,
an oral Factor D inhibitor for the treatment of complement-mediated
diseases, galidesivir, a potential treatment for COVID-19, 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, 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: the ongoing COVID-19 pandemic, which
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 below or in the documents BioCryst files
periodically with the Securities and Exchange Commission; ongoing
and future preclinical and clinical development of BCX9930 may not
have positive results; BioCryst may not be able to enroll the
required number of subjects in planned clinical trials of product
candidates; BioCryst may not advance human clinical trials with
product candidates as expected; the FDA, EMA, PMDA 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 certain restrictions, warnings, or other
requirements on product candidates, may impose a clinical hold with
respect to such product candidates, or may withhold market approval
for product candidates; product candidates, if approved, may not
achieve market acceptance; BioCryst’s ability to successfully
commercialize its product candidates, manage its growth, and
compete effectively; risks related to the international expansion
of BioCryst’s business; and actual financial results may not be
consistent with expectations, including that 2020 operating
expenses and cash usage may not be within management's expected
ranges. 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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