BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced
updates from its drug discovery process and additional therapies
from its pipeline that the company will highlight at a Research and
Development (R&D) Day today at 1:00p ET at the company’s
Discovery Center of Excellence in Birmingham, Alabama. A live
webcast of the event will be available online in the investors
section of the company website at www.biocryst.com.
“The success we are achieving with ORLADEYO
demonstrates that when we deliver a first-in-class or best-in-class
medicine, we can change patients’ lives. The focus of our discovery
efforts is to leverage our 30-plus years of experience to bring
more first-in-class or best-in-class medicines to patients living
with complement-mediated and other rare diseases. The team is
excited to share our approach, and our expanded pipeline of new
programs, at our R&D Day,” said Jon Stonehouse, president and
chief executive officer of BioCryst.
“We have significantly diversified our pipeline
and we continue to be disciplined around decisions to invest
further only when a program meets the high bar we have set of
first-in-class or best-in-class. We believe the combination of
ORLADEYO, our exciting pipeline and our financial strength,
position us to continue to make a big difference in patients’
lives, and that leads to sustainable growth for years to come,”
Stonehouse added.
PROGRAM UPDATES AT R&D
DAY
BCX17725 — Fusion Protein KLK5 Inhibitor
for treatment of Netherton syndrome
Netherton syndrome is a rare, lifelong genetic
disorder that often presents in neonates or infancy. The disease is
caused by the deficiency of a natural inhibitor (SPINK5) of
KLK5, a serine protease responsible for regulating skin shedding.
Patients may have red, scaly and inflamed skin and susceptibility
to recurrent immune reactions. Netherton syndrome can be life
threatening, especially during infancy when patients are vulnerable
to dehydration and recurrent infections. Currently, there is no
approved treatment for Netherton syndrome.
BCX17725 is a potent and selective
investigational fusion protein KLK5 inhibitor designed to provide
best-in-class, potentially disease-modifying treatment for people
with Netherton syndrome.
At the R&D Day, the company plans to
showcase how it applied its structure-guided drug design expertise
to create this fusion protein, and share nonclinical data showing
distribution of the molecule to the epidermis following
intraperitoneal (IP) administration.
The company expects to begin clinical trials of
BCX17725 in 2024.
Avoralstat — Plasma kallikrein inhibitor
combined with Clearside’s SCS
Microinjector® for treatment of
diabetic macular edema (DME) in patients with sub-optimal response
to anti-VEGF therapy
DME is the most common cause of vision loss in
individuals with diabetes and at least one-third of patients have
persistent DME despite anti-VEGF therapies, which are administered
via monthly injection. Data have shown that elevated plasma
kallikrein may be a cause of non-response to anti-VEGF therapy.
Delivering avoralstat directly into the
suprachoroidal space of the eye with the SCS Microinjector could
allow avoralstat to inhibit plasma kallikrein at the sites of edema
formation in DME disease, the retinal and choroidal vascular
endothelium. Avoralstat has low solubility, which supports
evaluation of a suspension depot formulation for ophthalmic
injection. With low solubility, the drug could persist in the eye
at the site of disease for a long duration, resulting in less
frequent injections.
At the R&D Day, the company plans to present
nonclinical data showing high levels of avoralstat are maintained
in the eye for at least 90 days following suprachoroidal
injection.
Avoralstat was previously studied in an oral
formulation in a phase 3 trial in patients with HAE. In the HAE
clinical trial program in 276 individuals, avoralstat was safe and
well tolerated with an adverse event profile similar to
placebo.
The company believes that combining a potent,
low-solubility kallikrein inhibitor that has a pre-existing
systemic safety database with the only FDA-approved approach to
access the suprachoroidal space provides the potential to deliver a
best-in-class medicine for DME patients inadequately controlled
with anti-VEGF therapy.
The company expects to conduct formulation and
nonclinical work in 2024 and begin clinical trials in 2025.
Oral C5 Inhibitor — Treatment of
generalized myasthenia gravis (and other complement-mediated
diseases)
Generalized myasthenia gravis (gMG) is a chronic
autoimmune, neuromuscular disease that causes muscle weakness that
worsens after periods of activity. It is caused when autoantibodies
(anti-AChR) form that activate the complement system, causing the
immune system to attack the neuromuscular junctions, or connections
between muscles and nerves.
BioCryst is developing a first-in-class oral C5
inhibitor that could be the first targeted oral therapy with
competitive efficacy to currently approved injected and infused
anti-C5 therapies, such as eculizumab and ravulizumab. A drug with
this profile could enable gMG patients to switch from infused
therapy and address their disease earlier in the treatment
paradigm.
At the R&D Day, the company plans to show
data from the nonclinical program highlighting the potency,
selectivity, oral bioavailability and pharmacokinetic parameters of
its lead molecule candidates.
The company expects to complete lead
optimization in 2024 and begin clinical trials in 2025.
Bifunctional complement inhibitor —
simultaneously inhibiting multiple complement pathways to better
address complex renal complement-mediated diseases like IgAN and
lupus nephritis
BioCryst is developing a bifunctional complement
inhibitor anti-C2 monoclonal antibody that also inhibits the
alternative pathway. This investigational candidate could be a
first-in-class combined inhibitor of the classical, lectin and
alternative pathways of the complement system to best treat complex
renal complement-mediated diseases like progressive IgAN and lupus
nephritis, which are influenced by multiple complement
pathways.
At the R&D Day, the company plans to share
nonclinical data demonstrating the strong potency and ability of
the company’s bifunctional monoclonal antibody to inhibit multiple
pathways in the complement system.
The company expects to select a lead molecule in
2024 and begin clinical trials in 2025.
Oral C2 Inhibitor — classical and lectin
pathway complement inhibitor to treat autoimmune hemolytic anemias,
including cold agglutinin disease (CAD) and warm autoimmune
hemolytic anemia (wAIHA)
The limited approved options for treating
diseases like CAD and wAIHA are injectable or infused. An oral C2
inhibitor from BioCryst would be first-in-class and allow patients
to switch from infused therapy and address their disease earlier in
the treatment paradigm.
Inhibiting C2 could decrease red cell
destruction (hemolysis) in autoimmune hemolytic anemias by blocking
the classical and lectin pathways.
At the R&D Day, the company plans to
describe the data targets for potency, selectivity and
bioavailability of its lead compounds.
The company expects to select a lead molecule in
2025.
BCX10013 — Once-daily, oral Factor D
inhibitor for renal complement-mediated diseases
BCX10013 is a potent and selective Factor D
inhibitor that, if it achieves comparable or superior efficacy to
twice-daily alternative pathway inhibitors in development, would
offer a best-in-class profile as the first and only oral
alternative pathway inhibitor with once-daily dosing.
At the R&D Day, the company plans to present
data from the recently completed 160 mg cohort of its multiple
ascending dose healthy volunteer trial which highlights the
strength and duration of alternative pathway suppression achieved
at this dose level, supporting once-daily clinical dosing.
The company expects to report data from its
ongoing proof-of-concept trial in 2024.
ORLADEYO®
(berotralstat) — Pediatric formulation for the prevention
of HAE attacks in patients 2 to <12 years of age
ORLADEYO, the first oral, once-daily plasma
kallikrein inhibitor, is approved in the United States and many
global markets for people with HAE age 12 and older. The ongoing
APeX-P clinical trial is assessing an oral granule formulation of
ORLADEYO in pediatric HAE patients who are 2 to <12 years of
age.
Approximately 40 percent of HAE patients have
their first attack by five years of age and there are no current
targeted oral therapies available for prophylaxis in children
<12 years old.
The company expects to submit a U.S.
supplemental new drug application for the pediatric use of ORLADEYO
in 2025.
2024 R&D Investment
Guidance The company’s focus on rigorous best-in-class or
first-in-class investment criteria, and the lower investment cost
of earlier stage programs, are expected to limit 2024 R&D
spending to a total of between $230 million and $240 million across
all programs. This represents an increase from projected 2023
R&D spending of $25 million to $35 million, and represents a
decrease from R&D spending in 2022 of $13 million to $23
million. These numbers include non-cash stock compensation. At the
R&D Day, the company will describe its disciplined approach to
capital allocation.
About BioCryst
PharmaceuticalsBioCryst Pharmaceuticals is a global
biotechnology company with a deep commitment to improving the lives
of people living with complement-mediated and other rare diseases.
BioCryst leverages its expertise in structure-guided drug design to
develop first-in-class or best-in-class oral small-molecule and
protein therapeutics to target difficult-to-treat diseases.
BioCryst has commercialized ORLADEYO® (berotralstat), the first
oral, once-daily plasma kallikrein inhibitor, and is advancing a
pipeline of small-molecule and protein therapies. For more
information, please visit www.biocryst.com or follow us on
LinkedIn.
Forward-Looking Statements
This press release contains forward-looking
statements, including statements regarding BioCryst’s plans and
expectations for its pipeline development. 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, 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 files
periodically with the Securities and Exchange Commission;
BioCryst’s ability to successfully progress its pipeline
development plans as described herein; the results of BioCryst’s
partnerships with third parties may not meet BioCryst’s current
expectations; ongoing and future preclinical and clinical
development of product candidates may take longer and be more
expensive than expected and 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; 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 revenue, 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, which identify important factors that could
cause actual results to differ materially from those contained in
BioCryst’s projections and forward-looking statements.
BCRXW
Contact:John Bluth+1 919 859
7910jbluth@biocryst.com
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