BioCryst Advancing Potential Treatment for Rare and Severely Debilitating Fibrodysplasia Ossificans Progressiva
January 05 2018 - 6:00AM
BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) announced today that
it has advanced a program exploring activin receptor-like kinase-2
(ALK2) inhibitors for treatment of Fibrodysplasia Ossificans
Progressiva (FOP). Investigational New Drug Application (IND)
enabling nonclinical development of optimized lead candidates
BCX9250 and BCX9499 has been initiated with the goal of progressing
to Phase 1 clinical trials in the first half of 2019.
FOP is a very rare disease that affects approximately 1 in 2
million people worldwide. In patients with FOP, minor trauma can
result in rapid development of painful inflammatory masses. These
progress over several weeks resulting in the replacement of the
affected soft tissue by permanent bone masses. There is no cure,
and there are no approved treatments for FOP.
“We are thrilled that our drug discovery culture has succeeded
in bringing forward attractive oral ALK2 inhibitors that have the
potential to treat patients with FOP,” said Dr. William P.
Sheridan, Senior Vice President and Chief Medical Officer. “At
BioCryst, we constantly strive to create medicines that not only
treat serious rare diseases, but do so in a way that retains the
best quality of life possible for patients and, ultimately, their
families and caregivers as well. We expect our core integrated drug
discovery capabilities, refined over years of research and applied
in new ways, to impact the BioCryst pipeline in an accelerating
fashion in coming periods.”
ALK2 enzyme is a part of the normal signaling pathway for bone
formation and responds to binding its specific ligands (bone
morphogenic proteins, BMPs), by stimulating normal bone growth and
renewal in healthy children and adults. Specific activating
mutations of the ALK2 gene are seen in all cases of FOP. An
activating mutation in ALK2 is necessary for the disease to occur,
making the ALK2 kinase an ideal drug target for treatment of FOP
with an ALK2 kinase inhibitor.
The goal of the ALK2 inhibitor project at BioCryst is to
discover and develop orally administered kinase inhibitor drug
candidates that are able to slow or prevent the progressive
formation of bone in soft tissues, also known as heterotopic
ossification (HO). The two lead candidate molecules dramatically
reduced HO in an experimental model of ALK2-driven HO in laboratory
rats, with up to 89 percent reduction in volume of HO compared to
controls.
“I am encouraged by pharmaceutical company efforts directed at
ALK2, the central target of FOP,” stated Dr. Frederick Kaplan, the
Isaac & Rose Nassau Professor of Orthopedic Molecular Medicine
and Co-Director of The Center for Research in FOP & Related
Disorders at The Perelman School of Medicine at The University of
Pennsylvania.
The lead candidates for the FOP program, BCX9250 and BCX9499,
were selected from a number of potential candidates based on
potency for the target kinase, selectivity, and safety screening
criteria that included industry-standard in vitro panels and in
vivo PK and safety studies in laboratory animals. BioCryst plans to
complete IND-enabling manufacturing and nonclinical safety studies
to support Phase 1 trials beginning in 2019, and as early as
possible thereafter, clinical trials in patients with FOP.
About Fibrodysplasia Ossificans Progressiva
(FOP)
FOP is a rare, severely disabling condition characterized by the
irregular formation of bone outside the normal skeleton, also known
as heterotopic ossification (HO). HO can occur in muscles, tendons
and soft tissue. FOP patients progressively become bound by this
irregular ossification, with restricted movement and fused joints,
resulting in deformities and premature mortality. There are
currently no approved treatments for FOP. More information on this
disease can be found at the International Fibrodysplasia Ossificans
Progressiva Association and OrphaNet websites.
About BioCryst Pharmaceuticals
BioCryst Pharmaceuticals designs, optimizes and develops novel
small-molecule medicines that address both common and rare
conditions. BioCryst has several ongoing development programs
including an oral product candidate for hereditary angioedema, and
galidesivir, a potential treatment for filoviruses. 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, Japan, Taiwan
and Korea. Post-marketing commitments for RAPIVAB are ongoing, as
well as activities to support regulatory approvals in other
territories. 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: that
developing any FOP drug candidate may take longer or may be more
expensive than planned; that ongoing and future preclinical and
clinical development of FOP candidates may not have positive
results; that BioCryst may not be able to enroll the required
number of subjects in planned clinical trials of product
candidates; that the Company may not advance human clinical trials
with product candidates as expected; that the FDA and EMA
may require additional studies beyond the studies planned for
product candidates, or may not provide regulatory clearances which
may result in delay of planned clinical trials, or may impose a
clinical hold with respect to such product candidate, or withhold
market approval for product candidates; that the Company may not be
able to continue development of ongoing and future development
programs; that such development programs may never result in future
products. 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
projections and forward-looking statements.
BCRXW
CONTACT: Thomas Staab, BioCryst Pharmaceuticals,
+1-919-859-7910
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