BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that
in a Phase 1 clinical trial with BCX9250, an oral activin
receptor-like kinase-2 (ALK-2) inhibitor discovered and developed
by BioCryst for the treatment of fibrodysplasia ossificans
progressiva (FOP), BCX9250 was safe and well tolerated at all doses
studied, with linear and dose-proportional exposure supporting
once-daily dosing.
FOP is an ultra-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. Patients with FOP
become bound by this irregular ossification over time, with
restricted movement and fused joints, resulting in deformities and
premature mortality. There are currently no approved treatments for
FOP.
The randomized, double-blind, placebo-controlled
dose-ranging trial evaluated safety, tolerability and
pharmacokinetics of single ascending doses (SAD) and multiple
ascending doses (MAD) of BCX9250 in healthy subjects.
The SAD study was designed to randomize four
cohorts of eight subjects each to receive oral BCX9250 (n=6) or
placebo (n=2) at dose levels of 5 mg, 10 mg, 15 mg and 25 mg.
Subjects in the 15 mg cohort also received a second single dose to
evaluate food effect on absorption of BCX9250.
The MAD study was designed to randomize four
cohorts of 12 subjects each to receive oral BCX9250 (n=10) or
placebo (n=2) at dose levels of 5 mg, 10 mg, 15 mg and 20 mg once
daily (QD) for seven days.
Drug exposure increased with dose in an
approximately linear and dose-proportional manner. Drug levels
after a high fat meal were similar to those after dosing on an
empty stomach. Drug exposure (area under the curve) at 20 mg
QD in the MAD was similar to that achieved with doses that
suppressed HO in a nonclinical model of activity of orally dosed
BCX9250. Additional data can be found in slides in the investors
section of the company’s website at https://ir.biocryst.com/.
In both the SAD and the MAD studies, oral
BCX9250 was safe and well tolerated, with no serious adverse
events, no study discontinuations due to adverse events, no grade 3
or 4 adverse events and no clinically significant changes in vital
signs, electrocardiograms or safety laboratory parameters. No
safety signals were seen.
“FOP is a devastating condition with no approved
treatments. These encouraging Phase 1 results provide hope for
patients and their families, and we look forward to speaking with
expert physicians, patient advocates and regulators about the next
steps to advance the program,” said Dr. Bill Sheridan, chief
medical officer of BioCryst.
In preclinical studies, BCX9250 demonstrated
potency for the target kinase, selectivity, safety and strong
suppression of HO in animal models.
About BCX9250
Discovered by BioCryst, BCX9250 is a novel,
oral, inhibitor of the ALK-2 enzyme. The ALK-2 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 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.
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 for the
prevention of HAE attacks in adults and pediatric patients 12 years
and older, and under regulatory review for approval in Japan and
the European Union. BioCryst has several ongoing development
programs including BCX9930, an oral Factor D inhibitor for the
treatment of complement-mediated diseases, galidesivir, a potential
treatment for COVID-19, Marburg virus disease and Yellow Fever, and
BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia
ossificans progressiva. 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, 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 for its
BCX9250 program. These statements involve known and unknown risks,
uncertainties and other factors which may cause actual results and
developments of such program 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: 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 files
periodically with the Securities and Exchange Commission; 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, EMA, PMDA or other
applicable regulatory agency may require additional studies beyond
the studies planned for 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 product candidates, may impose a clinical hold with
respect to such product candidates, or may withhold market approval
for product candidates; product candidates, if approved, may not
achieve market acceptance; BioCryst’s ability to successfully
develop and commercialize its 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:John Bluth+1 919 859
7910jbluth@biocryst.com
Catherine Collier Kyroulis+1 917 886
5586ckyroulis@biocryst.com
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