Avenue Therapeutics, Inc. (Nasdaq: ATXI) (“Avenue” or the
“Company”), a specialty pharmaceutical company focused on the
development and commercialization of therapies for the treatment of
neurologic diseases, today announced that the first patient has
been dosed in the Phase 1b/2a clinical trial of AJ201 for the
treatment of spinal and bulbar muscular atrophy (“SBMA”), also
known as Kennedy's Disease. A recent study used genetic analysis to
estimate disease prevalence of 1:6,887 males.1 AJ201 is currently
the lead drug candidate in the clinic for SBMA and enrollment in
the trial is expected to be complete by the end of 2023 or early
2024 with potential topline data in 2024.
“We are excited to announce that the first
patient has been dosed in our Phase 1b/2a clinical trial evaluating
AJ201 for the treatment of SBMA, a progressive and devastating
neurodegenerative disease that currently has no approved treatments
available,” said Alexandra MacLean, M.D., Chief Executive Officer
of Avenue. “We are encouraged by the Phase 1 clinical data of AJ201
that demonstrate the drug’s excellent safety profile in healthy
volunteers. Additionally, compelling preclinical data in a mouse
model showed efficacy signals, including improvement in motor
function, robust degradation of mutant androgen receptors (“AR”), a
disease-signaling protein, and activation of the Nrf1 and Nrf2
pathways. In this Phase 1b/2a clinical trial, we aim to demonstrate
how AJ201’s novel, multi-fold mechanism of action reduces
accumulation of mutant AR aggregates to potentially decrease
neuroinflammation, protect cells from oxidative stress, and
ultimately, improve clinical outcomes for SBMA patients. We look
forward to advancing this much needed drug, as we continue to
deliver on our mission of bringing impactful therapies to people
suffering from neurologic diseases.”
The 12-week, multicenter, randomized,
double-blind Phase 1b/2a clinical trial of AJ201 is expected to
enroll approximately 24 patients, randomly assigned to AJ201 (600
mg/day) or placebo. The primary endpoint of the study is to assess
safety and tolerability of AJ201 in subjects with clinically and
genetically defined SBMA. Secondary endpoints include
pharmacodynamic data measuring change from baseline in mutant AR
protein levels in skeletal muscle and changes in the fat and muscle
composition as seen on MRI scans, which are believed to be
biomarkers indicating likelihood for longer term clinical
improvement. Further details about this study can be found
at ClinicalTrials.gov (Identifier: NCT05517603).
About Spinal and Bulbar Muscular
AtrophySpinal and bulbar muscular atrophy (“SBMA”) is a
rare, X-linked genetic neuromuscular disease primarily affecting
men. The condition is caused by the trinucleotide CAG repeat
expansion in the androgen receptor (“AR”) which leads to production
of a mutant polyglutamine (“polyQ”) AR protein that forms
aggregates responsible for muscular atrophy focused in the limbs
and bulbar region of the body. The weakening of the bulbar muscles
affects chewing, speech and swallowing, with patients prone to
choking or inhaling foods or liquids, resulting in airway
infection. SBMA also affects muscles in the limbs, leading to
difficulty walking and injury caused by falling. Although there is
a range of cited prevalence rates in scientific literature, a
recent study used genetic analysis to estimate disease prevalence
of 1:6,887 males.1 Currently, there are no treatments approved by
the U.S. Food and Drug Administration or European Medicines Agency
available for patients. For more information about SBMA, also known
as Kennedy’s Disease, please visit
https://kennedysdisease.org/.
About AJ201AJ201 is a novel,
first-in-class asset in development for the treatment of spinal and
bulbar muscular atrophy. It was designed to modify SBMA through
multiple mechanisms including degradation of the abnormal androgen
receptor protein and by stimulating the Nrf1 and Nrf2 pathways,
which are involved in protecting cells from oxidative stress which
can lead to cell death. A first-in-human Phase 1 study of AJ201 in
72 healthy volunteers revealed an excellent safety and
pharmacokinetic profile. It is currently being studied in a Phase
1/2a multicenter, randomized, double-blind clinical trial in six
clinical sites across the U.S., which aims to evaluate the safety,
PK/PD data and clinical response of AJ201 in patients suffering
from SBMA. AJ201 has been granted Orphan Drug Designation by the
FDA for multiple polyQ diseases, including SBMA, Huntington’s
disease and spinocerebellar ataxia. Avenue exclusively licensed
AJ201 from AnnJi Pharmaceuticals in the United States, Canada,
European Union, Great Britain, and Israel.
About Polyglutamine
diseasesPolyglutamine diseases are a group of
neurodegenerative disorders caused by expanded CAG repeats encoding
a long polyQ tract in the affected proteins. To date, a total of
nine polyQ disorders have been described. Mutant protein
aggregation in affected tissues is the pathological hallmark of
polyQ diseases. Neuroinflammation, oxidative stress and
dysregulated protein quality control are thought to be key
pathological factors that are either direct results of mutant
protein aggregations and/or exacerbate the severity and progression
of the diseases. Modulating multiple cellular pathways in enhancing
degradation of mutant AR aggregates, inducing antioxidant and heat
shock responses, and increasing proteasome expression
simultaneously provide the rationale to develop AJ201 for the
treatment of SBMA and potentially other polyQ diseases.
About Avenue TherapeuticsAvenue
Therapeutics, Inc. (Nasdaq: ATXI) is a specialty pharmaceutical
company focused on the development and commercialization of
therapies for the treatment of neurologic diseases. It is currently
developing three assets including AJ201, a first-in-class oral
small molecule for spinal and bulbar muscular atrophy, BAER-101, an
oral small molecule selective GABA-A α2/3 receptor positive
allosteric modulator for CNS diseases, and IV Tramadol, which is in
Phase 3 clinical development for the management of
moderate-to-moderately-severe pain in adults in a medically
supervised healthcare setting. Avenue is headquartered in Miami, FL
and was founded by Fortress Biotech, Inc. (Nasdaq: FBIO). For more
information, visit www.avenuetx.com.
Forward-Looking StatementsThis
press release contains predictive or “forward-looking statements”
within the meaning of the Private Securities Litigation Reform Act
of 1995. All statements other than statements of current or
historical fact contained in this press release, including
statements that express our intentions, plans, objectives, beliefs,
expectations, strategies, predictions or any other statements
relating to our future activities or other future events or
conditions are forward-looking statements. The words “anticipate,”
“believe,” “continue,” “could,” “estimate,” “expect,” “intend,”
“may,” “plan,” “predict,” “project,” “will,” “should,” “would” and
similar expressions are intended to identify forward-looking
statements. These statements are based on current expectations,
estimates and projections made by management about our business,
our industry and other conditions affecting our financial
condition, results of operations or business prospects. These
statements are not guarantees of future performance and involve
risks, uncertainties and assumptions that are difficult to predict.
Therefore, actual outcomes and results may differ materially from
what is expressed or forecasted in, or implied by, the
forward-looking statements due to numerous risks and uncertainties.
Factors that could cause such outcomes and results to differ
include, but are not limited to, risks and uncertainties arising
from: expectations for increases or decreases in expenses;
expectations for the clinical and pre-clinical development,
manufacturing, regulatory approval, and commercialization of our
pharmaceutical product candidate or any other products we may
acquire or in-license; our use of clinical research centers and
other contractors; expectations for incurring capital expenditures
to expand our research and development and manufacturing
capabilities; expectations for generating revenue or becoming
profitable on a sustained basis; expectations or ability to enter
into marketing and other partnership agreements; expectations or
ability to enter into product acquisition and in-licensing
transactions; expectations or ability to build our own commercial
infrastructure to manufacture, market and sell our product
candidates; acceptance of our products by doctors, patients or
payors; our ability to compete against other companies and research
institutions; our ability to secure adequate protection for our
intellectual property; our ability to attract and retain key
personnel; availability of reimbursement for our products;
estimates of the sufficiency of our existing cash and cash
equivalents and investments to finance our operating requirements,
including expectations regarding the value and liquidity of our
investments; the volatility of our stock price; expected losses;
expectations for future capital requirements; and those risks
discussed in our filings which we make with the SEC. Any
forward-looking statements speak only as of the date on which they
are made, and we undertake no obligation to publicly update or
revise any forward-looking statements to reflect events or
circumstances that may arise after the date of this press release,
except as required by applicable law. Investors should evaluate any
statements made by us in light of these important factors.
Contact: Jaclyn Jaffe Avenue Therapeutics, Inc.
(781) 652-4500ir@avenuetx.com
——————————————1 M. Zanovello et al., Unexpected frequency of the
pathogenic ARCAG repeat 2 expansion in the general population.
Brain, 2023 Jul 3;146(7):2723-2729.
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