FDA Grants Fast Track Designation for BioCryst’s ALK-2 Inhibitor, BCX9250
08 June 2022 - 9:00PM
BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced
the U.S. Food and Drug Administration (FDA) has granted Fast Track
designation for BCX9250 for the prevention of heterotopic
ossification (HO) in patients with fibrodysplasia ossificans
progressiva (FOP).
According to the FDA, the purpose of the Fast Track designation
is to get important new drugs to patients earlier by facilitating
the development, and expediting the review, of drugs to treat
serious conditions and fill an unmet medical need. Companies that
receive this designation are eligible for benefits including more
frequent meetings with and written communication from the
FDA, eligibility for accelerated approval and priority review,
if relevant criteria are met, and rolling review of the new drug
application (NDA).
“We are pleased with the FDA’s decision to grant
Fast Track designation to BCX9250, as there is a significant unmet
need among patients living with FOP. With this designation
following the EMA’s recent decision to grant PRIME eligibility for
BCX9250 in Europe, we believe the non-clinical data and the
first-in-human Phase 1 safety, tolerability and pharmacokinetics
study in healthy subjects support the potential of our ALK-2
inhibitor program to be a meaningful therapeutic advance for the
FOP community,” said Dr. Helen Thackray, chief research and
development officer of BioCryst.
FOP is an ultra-rare, severely disabling genetic
disorder characterized by 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.
BCX9250 is 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 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 StatementsThis press release
contains forward-looking statements, including statements regarding
BioCryst’s plans and expectations for BCX9250 and the potential
benefits associated with an FDA Fast Track 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: Fast Track
designation by the FDA may not lead to a faster development,
regulatory review, or approval process with the FDA 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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