FDA Grants Orphan Drug Designation for BioCryst’s ALK-2 Inhibitor, BCX9250, for the Treatment of Fibrodysplasia Ossificans Progressiva
August 31 2022 - 7:00AM
BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced
the U.S. Food and Drug Administration (FDA) has granted orphan drug
designation for BCX9250 for the treatment of fibrodysplasia
ossificans progressiva (FOP).
The FDA has the option to grant orphan drug
designation to applicants for the purpose of supporting the
development of a drug or biologic intended to treat a disease or
condition that affects fewer than 200,000 individuals in the U.S.
Companies that receive this designation are eligible for
incentives, including tax credits for qualified clinical trials,
exemption from user fees and up to seven years of market
exclusivity after approval.
“We appreciate the FDA’s decision to grant
orphan drug designation to BCX9250 as we work toward our goal of
bringing this important oral investigational therapy to FOP
patients. The benefits available to us through this designation –
and the designations we previously received from the FDA and the
EMA – have the potential to advance our ALK-2 inhibitor program as
efficiently as possible as we move towards beginning trials in FOP
patients next year,” said Dr. Helen Thackray, chief research and
development officer of BioCryst.
FOP is an ultra-rare, severely disabling genetic
disorder characterized by heterotopic ossification (HO), or the
irregular formation of bone outside the normal skeleton. HO can
occur in muscles, tendons, ligaments and other connective tissues.
Patients with FOP become bound by this irregular ossification over
time, with restricted movement and fused joints, resulting in
deformities, restricted mobility and premature mortality.
Earlier this year, BioCryst announced that
BCX9250 has received Fast Track designation from the FDA, in
addition to orphan drug designation and PRIME designation from the
European Medicines Agency (EMA).
BioCryst will present three posters featuring
data from non-clinical studies and first-in-human Phase 1 safety,
tolerability and pharmacokinetics study in healthy subjects from
the BCX9250 program at the American Society for Bone and Mineral
Research Annual Meeting, which is being held in Austin, Texas, from
Sept. 9-12, 2022. As the company previously reported in the Phase 1
study, BCX9250 was safe and well tolerated at all doses studied,
with linear and dose-proportional exposure supporting the potential
for once-daily dosing.
About BCX9250BCX9250 is a
novel, oral activin receptor-like kinase-2 (ALK-2) inhibitor
designed to inhibit the ALK-2 enzyme, which 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 ALK-2 gene are seen in all
cases of fibrodysplasia ossificans progressiva (FOP). An activating
mutation in ALK-2 is necessary for the disease to occur, making the
ALK-2 enzyme an ideal drug target for treatment of FOP. In a Phase
1 clinical trial in healthy subjects, BCX9250 was safe and well
tolerated at all doses studied, with linear and dose-proportional
exposure supporting the potential for once-daily dosing.
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 Statements
This press release contains forward-looking
statements, including statements regarding BioCryst’s plans and
expectations for BCX9250 and the potential benefits associated with
an FDA orphan drug designation. These statements involve known and
unknown risks, uncertainties and other factors which may cause any
actual results or benefits 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: orphan drug, Fast Track, and other designations by
the FDA or other applicable regulatory agency may not lead to a
faster development, regulatory review, or approval process with
such agency and does not increase the likelihood that BCX9250 will
receive marketing approval; ongoing and future preclinical and
clinical development of BCX9250 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 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; and 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 above or in the
documents BioCryst periodically files with the Securities and
Exchange Commission. 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 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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