BioCryst Presents Data Demonstrating >99 Percent Suppression of Alternative Pathway Complement Activity with BCX9930 in C3G Patients
August 26 2022 - 7:00AM
BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced
that its oral Factor D inhibitor, BCX9930, demonstrated >99
percent suppression of the alternative pathway (AP), and that
>98 percent suppression was maintained for 24 hours post-dosing,
in patients with C3 glomerulopathy (C3G), a rare renal disease that
is characterized by dysregulation of the AP of the complement
system.
The data, which are the first data with BCX9930
in patients with C3G, are being presented in a poster session at
the 18th European Meeting on Complement in Human Disease, which is
being held in Bern, Switzerland, from August 26-29, 2022.
“The analyses from this study add to the body of
evidence that show Factor D inhibition can correct dysregulation of
the alternative pathway and has the potential to be a meaningful
treatment approach for multiple complement-mediated diseases. The
data are consistent using both commercially available assays and
sophisticated proprietary assays our team has developed, which we
can also utilize with our next-generation pipeline programs to
quickly assess complement activation in vivo early in development,”
said Dr. Yarlagadda S. Babu, chief discovery officer of
BioCryst.
BioCryst EMCHD 2022 Presentation Highlights
BCX9930, an Oral Factor D Inhibitor,
Suppresses Complement Alternative Pathway C3 Convertase
Activity in vitro, and in
Patients with Complement 3 Glomerulopathy (Poster
#80); Sunday, August 28, 2022; 11:45am-1:30pm CET; Hintere Gasse,
Building Fab8
- The study assessed the effects of
oral BCX9930 on AP activity in ex vivo activated serum from healthy
volunteers (n=16) and C3G patients (n=6) who participated in a
phase 1, open-label study to evaluate the pharmacokinetics and
pharmacodynamics of a single dose of BCX9930.
- One assay was used to assess the
ability of BCX9930 to inhibit formation of AP C3 convertase in
vitro (Elisa-detecting properdin (P)-bound C3 convertase), two
assays were used to assess the ability of BCX9930 to inhibit AP
complement activity ex vivo (AP Wieslab kit and multiplex assays),
and another assay was used to assess the ability of BCX9930 to
inhibit formation of AP C3 convertase ex vivo (immunofixation
electrophoresis).
- The analyses found that a single
oral dose (600 mg) of BCX9930:
- Demonstrated >99 percent
(median) suppression of the AP, and that >98 percent (median)
suppression was maintained for 24 hours post-dosing, in C3G
patients.
- Achieved rapid (within two hours)
and maximal (median >99 percent relative to pre-dose levels)
suppression of the generation of C3a, a product of C3 convertase
activity, in both healthy volunteers and C3G patients.
- These data support further
development of Factor D inhibitors for the treatment of
complement-mediated diseases, including C3G and other diseases
driven by dysregulation of the AP.
In the study, a single dose of BCX9930 600 mg
was generally safe and well-tolerated. Based on prior work with
BCX9930, the proposed clinical dose of 400 mg (bid) provides a
similar level of AP suppression as the 600 mg single dose used in
this study in C3G patients.
Currently, BioCryst is conducting the RENEW
proof-of-concept basket study evaluating BCX9930 in C3G,
immunoglobulin A nephropathy (IgAN) and primary membranous
nephropathy (PMN), all rare renal diseases caused by dysregulation
of the alternative pathway.
BioCryst is also currently conducting the
REDEEM-1 and REDEEM-2 pivotal trials evaluating BCX9930 in
paroxysmal nocturnal hemoglobinuria (PNH).
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 and multiple global
markets. BioCryst has several ongoing development programs
including BCX9930, an oral Factor D inhibitor for the treatment of
complement-mediated diseases, BCX9250, an ALK-2 inhibitor for the
treatment of fibrodysplasia ossificans progressiva, and
galidesivir, a potential treatment for Marburg virus disease and
yellow fever. RAPIVAB® (peramivir injection) is approved in the
U.S. and multiple global markets, with post-marketing commitments
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 actual results, performance or
achievements to be materially different from any future results,
performance 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 periodically files 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; 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 certain restrictions, warnings, or
other requirements on product candidates, may impose a clinical
hold with respect to product candidates, or may withhold or delay
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, which identify important factors that could
cause the actual results to differ materially from those contained
in BioCryst’s forward-looking statements.
BCRXW
Investor Contact:John Bluth+1
919 859 7910jbluth@biocryst.com
Media Contact:Catherine
Collier Kyroulis+1 917 886 5586ckyroulis@biocryst.com
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