Avalo Therapeutics Announces First Patient Dosed in the AVTX-803 Pivotal Trial (LADDER) for the Treatment of Leukocyte Adhesion Deficiency Type II (LAD II)
August 02 2022 - 07:30AM
Avalo Therapeutics, Inc. (Nasdaq: AVTX), announced the first
patient has been dosed in the pivotal LADDER trial (A Phase 3,
Randomized, Double-blind, Two-period, Crossover, Withdrawal Study
to Assess the Efficacy and Safety of AVTX-803 in Subjects with
Leukocyte
Adhesion
Deficiency Type II (LA
D II)
ER). AVTX-803 is a therapy developed and studied
for the treatment of leukocyte adhesion deficiency type II (LAD II,
also known as SLC35C1-CDG). AVTX-803 has been granted Rare
Pediatric Disease Designation (RPDD), Orphan Drug Designation (ODD)
and Fast Track Designation (FTD).
“We are excited to announce the first patient was
dosed in the LADDER pivotal trial. This substantially advances the
program toward an approved treatment option for patients and also a
potential PRV,” said Garry A. Neil, MD, President and Chief
Executive Officer, Avalo Therapeutics.
AVTX-803The active pharmaceutical
ingredient (API) in AVTX-803 is L-fucose. L-fucose is a
plant-derived, naturally occurring monosaccharide with high
solubility in water and is isolated as a white crystalline powder.
AVTX-803 is an oral formulation of L-fucose that enhances
fucosylation of proteins in the absence of a functioning GDP-fucose
transporter, partially restoring protein function. AVTX-803 was
granted FTD, ODD and RPDD, making it potentially eligible for a
Priority Review Voucher (PRV).
Pivotal TrialThe LADDER trial is a
pivotal, 16-week randomized, double-blind, two-period crossover,
withdrawal study to assess the efficacy and safety of AVTX-803 in
patients with LAD II (n=2). The primary endpoint is the comparison
of leukocyte function as determined by sialyl Lewis-X (SLx) antigen
expression on leukocytes between treatment periods. The trial will
conclude with the End of Study/Early Termination Visit at which
time subjects will be permitted to enroll into a long-term
open-label, safety and efficacy study.
LAD IILAD II is an ultra-rare,
inherited congenital disorder of glycosylation (CDG), previously
known as CDG-IIc and also referred to as SLC35C1-CDG. The disease
is autosomal recessive and is characterized by reduced or absent
fucosylation of certain glycoproteins. Patients with LAD II present
with a multi-system phenotype that may include: leukocytosis,
neutrophilia, recurrent infections, reduced selectin expression and
binding, Bombay blood group, hypotonia, poor feeding, short
stature, coarse facial features, global developmental delay and
psychomotor retardation. In some cases, these symptoms lead to
death, and in most LAD II patients, the disorder negatively impacts
quality of life.About Avalo Therapeutics Avalo
Therapeutics is a leading clinical-stage precision medicine company
that discovers, develops, and commercializes targeted therapeutics
for patients with significant unmet clinical need in immunology and
rare genetic diseases. The Company has built a diverse portfolio of
innovative therapies to deliver meaningful medical impact for
patients in urgent need. The Company’s clinical candidates commonly
have a proven mechanistic rationale, biomarkers and/or an
established proof-of-concept to expedite and increase the
probability of success.
For more information about Avalo, please visit
www.avalotx.com.
Forward-Looking Statements This
press release may include forward-looking statements made pursuant
to the Private Securities Litigation Reform Act of 1995.
Forward-looking statements are statements that are not historical
facts. Such forward-looking statements are subject to significant
risks and uncertainties that are subject to change based on various
factors (many of which are beyond Avalo’s control), which could
cause actual results to differ from the forward-looking statements.
Such statements may include, without limitation, statements with
respect to Avalo’s plans, objectives, projections, expectations and
intentions and other statements identified by words such as
“projects,” “may,” “might,” “will,” “could,” “would,” “should,”
“continue,” “seeks,” “aims,” “predicts,” “believes,” “expects,”
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similar expressions (including their use in the negative), or by
discussions of future matters such as: the development of product
candidates or products; timing and success of trial results and
regulatory review; potential attributes and benefits of product
candidates; the future financial and operational outlook; and other
statements that are not historical. These statements are based upon
the current beliefs and expectations of Avalo’s management but are
subject to significant risks and uncertainties, including: drug
development costs, timing and other risks, including reliance on
investigators and enrollment of patients in clinical trials, which
might be slowed by the COVID-19 pandemic; Avalo's cash position and
the potential need for it to raise additional capital; reliance on
key personnel, including as a result of recent management changes;
regulatory risks; general economic and market risks and
uncertainties, including those caused by the COVID-19 pandemic and
tensions in Ukraine; and those other risks detailed in Avalo’s
filings with the SEC. Actual results may differ from those set
forth in the forward-looking statements. Except as required by
applicable law, Avalo expressly disclaims any obligations or
undertaking to release publicly any updates or revisions to any
forward-looking statements contained herein to reflect any change
in Avalo’s expectations with respect thereto or any change in
events, conditions or circumstances on which any statement is
based.
For media and investor
inquiries
Christopher Sullivan, CFOAvalo Therapeutics,
Inc.ir@avalotx.com410-803-6793
or
Chris BrinzeyICR
WestwickeChris.brinzey@westwicke.com339-970-2843
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