Lexeo Therapeutics Granted FDA Fast Track Designation and Orphan Drug Designation for LX2020, an AAV-Based Gene Therapy Candidate for PKP2 Arrhythmogenic Cardiomyopathy (ACM)
December 18 2023 - 7:00AM
Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic
medicine company dedicated to pioneering treatments for genetically
defined cardiovascular diseases and APOE4-associated Alzheimer's
disease, today announced the U.S. Food and Drug Administration
(FDA) has granted Fast Track designation and Orphan Drug
designation to LX2020, the company’s AAVrh10-based gene therapy
candidate designed to intravenously deliver a functional PKP2 gene
to cardiac muscle for the treatment of arrhythmogenic
cardiomyopathy (ACM) caused by mutations in the PKP2 gene
(PKP2-ACM).
“Receiving both Orphan Drug and Fast Track designations from the
FDA for LX2020 further validates the importance of progressing a
potential one-time treatment option for patients suffering from
PKP2-ACM,” commented R. Nolan Townsend, Chief Executive Officer of
Lexeo Therapeutics. “PKP2-ACM is one of the most prevalent diseases
in the genetic cardiovascular space and with no approved
therapeutic options, we look forward to the possibility of
delivering a first and best in class treatment option for
patients.”
The planned Phase 1/2 trial, HEROIC-PKP2, is a first in human,
52-week open-label, dose-escalating, multicenter trial to determine
the safety and tolerability of LX2020 in adult patients with
PKP2-ACM. Preliminary efficacy measures will evaluate myocardial
protein expression, biomarkers measuring cardiac structure and
function, and arrhythmia burden. LX2020 will be administered as a
one-time intravenous infusion to patients in two ascending-dose
cohorts, evaluating the 2.0x1013 vg/kg and 6.0x1013 vg/kg dose
levels with three patients in each cohort, and the potential for
cohort expansion. Long-term safety and efficacy will be evaluated
for an additional four years following completion of the initial
trial.
The FDA grants Orphan Drug designation status to support the
development of medicines for rare disorders that affect fewer than
200,000 people in the U.S. Orphan Drug designation provides certain
benefits, such as market exclusivity upon regulatory approval,
exemption of FDA application fees, as well as tax credits for
qualified clinical trials. Fast Track designation allows more
frequent FDA interactions to facilitate development and expedite
the review process for novel drug candidates that treat serious or
life-threatening diseases and address unmet medical needs.
About LX2020LX2020 is an AAVrh10-based gene
therapy candidate designed to intravenously deliver a functional
PKP2 gene to cardiac muscle for the treatment of PKP2-ACM. PKP2
mutations are associated with approximately 75% of all genetic
cases of ACM, estimated to affect approximately 60,000 patients in
the United States. PKP2 mutations can cause fibro-fatty replacement
of heart muscle and severe abnormal heart rhythms, or arrhythmias,
that cause cardiac dysfunction and can result in sudden cardiac
death. Currently there are no approved treatments to slow, prevent
or reverse disease progression in patients with PKP2-ACM. LX2020 is
designed to increase desmosomal PKP2 protein levels, reassemble
desmosomes and restore myocardial cell function.
About Lexeo TherapeuticsLexeo Therapeutics is a
New York City-based, clinical stage genetic medicine company
dedicated to transforming healthcare by applying pioneering science
to fundamentally change how genetically defined cardiovascular
diseases and APOE4-associated Alzheimer’s disease are treated.
Using a stepwise development approach, Lexeo is leveraging early
proof-of-concept functional and biomarker data to advance a
pipeline of cardiovascular and APOE4-associated Alzheimer’s disease
programs.
Forward looking StatementsCertain statements in
this press release may constitute “forward-looking statements”
within the meaning of the federal securities laws, including, but
not limited to, our expectations and plans regarding our current
product candidates and programs, including the anticipated timing
of the initiation of and results from our clinical trials,
expectations regarding the time period over which our capital
resources will be sufficient to fund our anticipated operations and
estimates regarding Lexeo’s financial condition. Words such as
“may,” “might,” “will,” “objective,” “intend,” “should,” “could,”
“can,” “would,” “expect,” “believe,” “design,” “estimate,”
“predict,” “potential,” “develop,” “plan” or the negative of these
terms, and similar expressions, or statements regarding intent,
belief, or current expectations, are forward-looking statements.
While Lexeo believes these forward-looking statements are
reasonable, undue reliance should not be placed on any such
forward-looking statements. These forward-looking statements are
based upon current information available to the company as well as
certain estimates and assumptions and are subject to various risks
and uncertainties (including, without limitation, those set forth
in Lexeo’s filings with the U.S. Securities and Exchange Commission
(the SEC)), many of which are beyond the company’s control and
subject to change. Actual results could be materially different
from those indicated by such forward looking statements as a result
of many factors, including but not limited to: risks and
uncertainties related to global macroeconomic conditions and
related volatility; expectations regarding the initiation,
progress, and expected results of our preclinical studies, clinical
trials and research and development programs; the unpredictable
relationship between preclinical study results and clinical study
results; delays in submission of regulatory filings or failure to
receive regulatory approval; liquidity and capital resources; and
other risks and uncertainties identified in our Quarterly Report of
10-Q for the quarterly period ended September 30, 2023, filed with
the SEC and subsequent future filings we may make with the SEC. New
risks and uncertainties may emerge from time to time, and it is not
possible to predict all risks and uncertainties. Lexeo claims the
protection of the Safe Harbor contained in the Private Securities
Litigation Reform Act of 1995 for forward-looking statements. Lexeo
expressly disclaims any obligation to update or alter any
statements whether as a result of new information, future events or
otherwise, except as required by law.
Media Response:Janine Bogris(201)
245-6838janine.bogris@canalecomm.com
Investor Response:Laurence Watts(619)
916-7620laurence@gilmartinir.com
Lexeo Therapeutics (NASDAQ:LXEO)
Historical Stock Chart
From Jun 2024 to Jul 2024
Lexeo Therapeutics (NASDAQ:LXEO)
Historical Stock Chart
From Jul 2023 to Jul 2024