BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that
its oral Factor D inhibitor, BCX9930, significantly increased
hemoglobin and reduced transfusions in an ongoing dose-ranging
trial in treatment-naïve (no prior treatment with C5 inhibitors)
paroxysmal nocturnal hemoglobinuria (PNH) patients, and in PNH
patients with an inadequate response to C5 inhibitors. BCX9930 was
safe and generally well-tolerated in the trial.
Based on these results, and recent interactions
with U.S. and European regulators, the company plans to advance
directly into pivotal trials in PNH and proof of concept trials in
renal complement-mediated diseases in the second half of 2021.
PNH patients in the trial also experienced
reductions in key laboratory biomarkers, such as reticulocyte
count, lactate dehydrogenase (LDH) (treatment-naïve patients) and
percentage of C3 opsonization (patients with inadequate C5
response) following dosing at 400 mg bid or 500 mg bid.
“The significant reduction in transfusions and
increases in hemoglobin seen in this trial with an oral medicine
address an unmet need for patients and physicians -- a PNH therapy
that can maximize hematological benefit through the control of both
intravascular and extravascular hemolysis,” said Antonio Risitano,
M.D., Ph.D., San Giuseppe Moscati Hospital, Avellino, Italy, and
principal investigator of the trial.
“I am very encouraged by these results which may
position proximal inhibitors, and in particular this oral
anti-Factor D agent, as medications changing the treatment paradigm
of PNH, and possibly of other alternative pathway mediated
diseases,” he added.
About the TrialIn the trial, 10
treatment-naïve PNH patients received oral BCX9930 monotherapy and
six PNH patients who had an inadequate response on a C5 inhibitor
had oral BCX9930 added to their intravenous C5 therapy regimen.
Fifteen patients, nine treatment-naïve and six who had an
inadequate response on a C5 inhibitor, received BCX9930 at doses of
400 mg bid or 500 mg bid for at least eight weeks. Some patients
have now been on study drug for almost a year.
Treatment with BCX9930 at doses of 400 mg bid or
500 mg bid for at least eight weeks resulted in significant
improvements in clinical symptoms and laboratory biomarkers of
disease in both the treatment-naïve and C5 inadequate-responder
cohorts.
- Through their last study visit, 100
percent of treatment-naïve patients and 83 percent of C5 inhibitor
inadequate response patients were transfusion-free. Prior to the
trial, 22 percent of treatment-naïve patients and 17 percent of C5
inadequate responders were transfusion-free.
- Hemoglobin levels increased by a
mean of 3.5 g/dL in treatment-naïve patients and 3.2 g/dL in C5
inhibitor inadequate response patients; at last visit, mean
hemoglobin levels were 11.8 g/dL and 12.2 g/dL,
respectively.
- Relative red blood cell clone size,
a marker of hemolytic control, also increased from 53 to 92 percent
in treatment-naïve patients and from 50 to 80 percent in C5
inhibitor inadequate response patients.
- C3 opsonization, a key marker of
extravascular hemolysis, declined to less than two percent by the
eight-week visit in five of six C5 inadequate response patients
following BCX9930 therapy.
- In C5 inhibitor naïve patients,
clinical chemistry biomarkers of intravascular hemolysis, including
lactate dehydrogenase (LDH), aspartate aminotransferase (AST) and
bilirubin, all declined. Mean LDH x ULN declined by 73 percent from
7.5x ULN at baseline to 2.0x ULN.
The most common drug-related treatment-emergent
adverse events (TEAEs) were transient headache (in 10 of 16
patients) soon after starting treatment, and a benign drug rash (in
six of 16 patients) that resolved in all patients through continued
study drug administration. TEAEs of hemolysis occurred in two of 16
patients and resolved without any changes to dosing. There were no
discontinuations or drug interruptions due to related adverse
events. No safety signals were seen in routine monitoring of
adverse events, vital signs, electrocardiograms, or laboratory
evaluations of hematology, clinical chemistry, coagulation, or
urinalysis.
“The data we are seeing at 400 mg and 500 mg
show meaningful disease control in both treatment-naïve and C5
inadequate response patients, and an excellent safety profile. Our
goal is to bring an oral monotherapy to patients and we now have
the data to rapidly advance an optimal dose into pivotal trials in
PNH and proof of concept trials in other complement-mediated
diseases,” said Dr. Bill Sheridan, chief medical officer of
BioCryst.
The U.S. Food and Drug Administration has
granted both Fast Track status and Orphan Drug Designation to
BCX9930 for PNH.
Virtual R&D Day
TodayResults from the trial, including slides with
additional data and new market research assessing PNH patient
demand for an oral therapy, will be presented and discussed at
BioCryst’s virtual R&D day today from 9:00 am ET to 11:00 am
ET, and will be available in the Investors section of BioCryst’s
website at http://www.biocryst.com. The live video webcast and
replay of the virtual R&D day may be accessed at:
https://onlinexperiences.com/Launch/QReg/ShowUUID=0108DF33-EA47-4C52-B45E-A49134DC75CF.
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 Japan
for the prevention of HAE attacks in adults and pediatric patients
12 years and older, and under regulatory review for approval in the
European Union and United Kingdom. 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), a viral neuraminidase inhibitor for the treatment of
influenza, has received regulatory approval in the U.S., Canada,
Australia, Japan, Taiwan and Korea. 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 and expectations for its BCX9930 program.
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; the FDA, EMA, or other applicable
regulatory agency may require additional studies beyond the studies
planned for products and 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 products and product candidates, may impose a
clinical hold with respect to product candidates,
or may withhold, delay, or withdraw market approval for
products and product candidates; product candidates, if
approved, may not achieve market acceptance; BioCryst’s ability to
successfully commercialize its products and 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 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
Contact:InvestorsJohn Bluth+1 919
859 7910jbluth@biocryst.com
MediaCatherine Collier
Kyroulis+1 917 886 5586ckyroulis@biocryst.com
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