BioCryst Announces Designs for REDEEM-1 and REDEEM-2 Pivotal Trials with BCX9930 as Oral Monotherapy for Patients with PNH
July 15 2021 - 7:00AM
BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced the
designs for REDEEM-1 and REDEEM-2, two upcoming pivotal trials with
its oral Factor D inhibitor, BCX9930, in patients with paroxysmal
nocturnal hemoglobinuria (PNH).
REDEEM-1 is a randomized, open-label, active,
comparator-controlled comparison of the efficacy and safety of
BCX9930 (500 mg bid) monotherapy in approximately 81 PNH patients
with an inadequate response to a C5 inhibitor. In part 1 of this
trial, patients who have not had an adequate response to a C5
inhibitor will be randomized 2:1 to discontinue their C5 inhibitor
and receive BCX9930 as monotherapy or to continue receiving their
C5 inhibitor for 24 weeks. All patients will receive BCX9930 in
part 2 (weeks 25-52) to assess the long-term safety, tolerability
and effectiveness of BCX9930. Patients who are randomized to C5
inhibitor therapy in part 1 will discontinue that therapy at the
week 24 visit and start BCX9930 as monotherapy for part 2.
REDEEM-2 is a randomized, placebo-controlled
trial to evaluate the efficacy and safety of BCX9930 (500 mg bid)
as monotherapy versus placebo in approximately 57 PNH patients not
currently receiving complement inhibitor therapy. In part 1 of this
trial, patients will be randomized 2:1 to receive BCX9930 or
placebo under double-blind conditions for 12 weeks. All patients
will receive BCX9930 in part 2 (weeks 13-52) to assess the
long-term safety, tolerability and effectiveness of BCX9930, with
patients randomized to placebo in part 1 switching to BCX9930 at
the week 12 visit.
The primary endpoint for both trials is the
change from baseline in hemoglobin (Hb), assessed at weeks 12
to 24 in REDEEM-1 and at week 12 in REDEEM-2.
Key secondary endpoints for both trials are the
proportion of subjects who are transfusion-free, the number of
units of packed red blood cells (pRBC) transfused and change from
baseline in FACIT-Fatigue score. In REDEEM-2 the percent change
from baseline in lactate dehydrogenase also is a key secondary
endpoint. Other secondary endpoints in both trials include:
percent reduction in the rate of pRBC units transfused, change from
baseline in total PNH RBC clone size, ratio of total PNH RBC clone
size to PNH white blood cell clone size, reticulocyte count and the
proportion of subjects with Hb ≥12 g/dL. In REDEEM-1,
reduction in C3 opsonization of red blood cells also is a secondary
endpoint.
Trial site start-up activities are now underway
at sites around the world and both pivotal trials are expected to
begin enrolling patients in the second half of 2021.
In a dose-ranging trial of BCX9930, the company
has previously reported that BCX9930 (at doses of 400 mg or 500 mg
bid) increased hemoglobin from baseline by a mean of 3.2 g/dL in C5
inadequate response patients and 3.5 g/dL in treatment-naïve
patients and reduced or eliminated transfusions in PNH patients.
BCX9930 was safe and generally well-tolerated in the trial.
“Based on the excellent control of both
intravascular and extravascular hemolysis we have seen with BCX9930
in PNH patients in the clinical program to date, we have advanced
rapidly into PNH pivotal trials with BCX9930. Our goal is to
achieve a broad indication for BCX9930 as oral monotherapy for
patients with PNH,” said Dr. William 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.
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, the European
Union, Japan and the United Kingdom for the prevention of HAE
attacks in adults and pediatric patients 12 years and older.
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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