BioCryst’s Oral Factor D Inhibitor, BCX9930, Shows Clinical Benefit as Monotherapy Through 400 mg bid in Treatment-naïve P...
September 30 2020 - 07:00AM
BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today announced new
data from treatment-naïve (no prior treatment with C5 inhibitors)
paroxysmal nocturnal hemoglobinuria (PNH) patients receiving doses
through 400 mg bid of its oral Factor D inhibitor, BCX9930, as
monotherapy in an ongoing dose-ranging trial.
Oral BCX9930 is driving rapid and dose-dependent
reductions in key biomarkers, including LDH, and increasing
hemoglobin levels in all PNH patients in the trial. Increases in
hemoglobin levels were maintained without transfusions.
BCX9930 has been safe and well tolerated at all
doses in the trial. No drug-related serious adverse events have
been reported.
“We are thrilled with the clinical benefits and
safety profile of oral BCX9930 monotherapy that we continue to see
in PNH patients with dosing up to 400 mg bid. With these excellent
results, we plan to initiate advanced development trials next year
in multiple complement-mediated hematology and nephrology
diseases,” said Dr. William Sheridan, chief medical officer of
BioCryst.
The FDA has granted both Fast Track status and
Orphan Drug Designation to BCX9930 for PNH. BioCryst has confirmed
meetings with regulators in the 4th quarter of 2020 to discuss the
advanced development program for BCX9930.
Updated Data Through
400 mg bid
- All seven PNH patients in the trial
were severely ill, with pre-treatment LDH from 3.8 to 11 × ULN,
indicating active hemolysis. Four patients had a history of
compromised bone marrow function, and two had thrombotic, lung or
kidney complications from PNH.
- New data from the four
treatment-naïve PNH patients who have received more than six weeks
of therapy at 400 mg bid show significant clinical benefits for
these patients.º Hemoglobin levels increased by a mean of 3.8
g/dL from baseline. These increases are being maintained without
transfusions.º Three of four patients have responded with
hemoglobin levels >11 g/dL to date. Hemoglobin in the fourth
patient, who has compromised bone marrow function from aplastic
anemia PNH, responded with an increase from 6 g/dL at baseline to
9.5 g/dL on treatment.º In all four patients, the size of the
PNH red blood cell clone approached that of the PNH granulocyte
clone, indicating near-complete control of complement-mediated
hemolysis. The mean relative (red blood cells/granulocytes) PNH red
blood cell clone size increased from 48 percent at baseline to 94
percent on treatment.º All four patients have shown
reductions in LDH. Three of four patients show average serial LDH
of <1.5 × ULN. In the fourth patient, the LDH has decreased
from a pretreatment baseline of 11 × ULN to 2.2 × ULN on treatment
to date.º All four patients have continued on therapy with
BCX9930 based on the investigators’ assessment of clinical
benefit.
- The most common
adverse event was mild to moderate headache lasting one to three
days. One patient had a mild rash that resolved after continued
BCX9930 dosing at 100 mg bid. One patient had a mild rash at
200 mg bid that is resolving during uninterrupted dosing after dose
escalation to 400 mg bid.
In addition to the ongoing dose-ranging trial in
treatment-naïve PNH patients, the company plans to report data from
PNH patients with an inadequate response to C5 inhibitors receiving
at least 400 mg bid of BCX9930 by the end of 2020.
Additional details can be found on slides, which
can be accessed at the Investors’ section of BioCryst’s website at
http://www.biocryst.com.
Conference Call and
WebcastBioCryst management will host a conference call and
webcast at 8:30 a.m. ET today to discuss the new data. The live
call may be accessed by dialing 877-303-8027 for domestic callers
and 760-536-5165 for international callers and using conference ID
# 6798951. A live webcast of the call and any slides will be
available online at the investors section of the company website at
www.biocryst.com. A telephone replay of the call will be available
by dialing 855-859-2056 for domestic callers or 404-537-3406 for
international callers and entering the conference ID #6798951.
About the
Alternative Pathway and
Complement-Mediated DiseasesThe complement system
is part of the body’s natural immune system and is responsible for
helping the body eliminate microbes and damaged cells. 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. Patients with these diseases
currently have no approved treatments or are limited to treatment
with repeated intravenous infusions.
The alternative pathway is constantly active and
provides a critical amplification loop for all three pathways
(alternative, lectin, classical) of the complement system,
regardless of the initiating mechanism. Factor D is an essential
enzyme, and the first enzyme, in the alternative pathway, making
Factor D an attractive target to address complement-mediated
diseases.
About BCX9930Discovered 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. 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. BioCryst has several ongoing
development programs including ORLADEYO™ (berotralstat), an oral
treatment for hereditary angioedema, BCX9930, 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 ALK2 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, 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 BioCryst’s plans for its
BCX9930 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 those
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 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 it 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;
development may be more expensive than expected; and the FDA 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 and developments to differ
materially from those contained in BioCryst’s forward-looking
statements.
BCRXW
Contact:John Bluth+1 919 859
7910jbluth@biocryst.com
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