Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic
medicine company, today announced an in-license agreement with
Cornell University to expedite development of the investigational
gene therapy candidate LX2006 for the treatment of Friedreich
ataxia (FA) cardiomyopathy.
Under the license agreement, Lexeo has acquired certain rights1
including rights to current and future data generated in an ongoing
investigator-initiated Phase 1A trial of AAVrh.10hFXN to treat FA
cardiomyopathy (NCT05302271). The agreement will support Lexeo’s
efforts to develop a potentially life-changing therapy for this
unmet need.
The investigator-initiated trial is being conducted by Weill
Cornell Medicine, which has pioneered groundbreaking research on
the potential of gene therapy in FA, published preclinical data
that supported the first ever gene therapy IND clearance for FA,
and sponsored a natural history study for almost a decade to better
characterize the condition and its progression. Lexeo previously
licensed know-how relating to AAVrh.10hFXN from Weill Cornell
Medicine and collaborated with researchers there to further study
the candidate, which Lexeo refers to as LX2006. Lexeo is studying
LX2006 in the company-sponsored, open label, dose-ascending,
multicenter SUNRISE-FA Phase 1/2 trial (NCT05445323), in which four
patients have been dosed to date across cohorts 1 & 2. Weill
Cornell Medicine has dosed seven patients to date with LX2006
across dose cohorts 1 & 2 and is collecting biomarker,
structural, and functional cardiac data akin to SUNRISE-FA.
“The larger aggregate data set, combined with Orphan Drug, Rare
Pediatric Disease, and Fast Track designations from FDA, is
anticipated to facilitate an accelerated path to regulatory
engagements for LX2006,” said R. Nolan Townsend, Chief Executive
Officer of Lexeo Therapeutics. “We are excited about the
opportunity to advance research in FA cardiomyopathy, which is the
leading cause of death in FA and has no approved treatment options
today.”
The interim clinical data readout of LX2006 is expected mid-year
2024. With the newly-licensed data, the readout will now include
participants across the two studies, approximately doubling the
number of evaluable patients and including patients with a
treatment duration out to 18-months. Lexeo also expects to provide
an analysis of natural history data and baseline characteristics
for study participants from both studies to characterize the
cardiovascular disease phenotype seen in FA cardiomyopathy ahead of
the interim readout.
“This agreement with Lexeo Therapeutics builds upon years of
collaboration between Weill Cornell Medicine and Lexeo to benefit
patients with FA cardiomyopathy. It is our intention that this
license agreement will accelerate the clinical investigation and
development of LX2006 as a potential life-saving therapy for
patients with FA,” said Dr. Lisa Placanica, Senior Managing
Director, Center for Technology Licensing at Weill Cornell
Medicine.
Patients Treated with LX2006 Across Clinical Trials, as
of April 22, 2024
Dose |
Combined Enrollment Update and Months of
Follow-Up |
|
>12 months |
6-12 months |
<6 months |
Dose Cohort 11.8x1011 vg/kg |
3 |
3 |
- |
Dose Cohort 25.6x1011 vg/kg |
- |
2 |
3 |
Note: Cardiac biopsies are performed only in the SUNRISE-FA
trial; one patient in dose cohort 1 and three patients in dose
cohort 2 have undergone cardiac biopsies.
The Phase 1A study of AAVrh.10hFXN conducted by investigators at
Weill Cornell Medicine is a single-site, 52-week, dose-ascending,
open-label trial evaluating the safety and preliminary efficacy of
AAVrh.10hFXN in patients who have FA cardiomyopathy. AAVrh.10hFXN
is administered as a one-time intravenous infusion to patients in
two ascending-dose cohorts with five participants per cohort. While
cardiac biopsies are not collected in this study, key cardiac
disease measures are collected at 3, 6 and 12-month intervals and
complement data collected in SUNRISE-FA.
SUNRISE-FA is a multicenter, 52-week, dose-ascending, open-label
trial evaluating the safety and preliminary efficacy of LX2006 in
patients who have FA cardiomyopathy. LX2006 is administered as a
one-time intravenous infusion to patients in at least two
ascending-dose cohorts with the potential to escalate to a third
cohort at a dose of 1.2x1012 vg/kg. Long-term safety and efficacy
will be evaluated for five years following dosing in both
trials.
________________________ 1The license agreement includes a
package of intellectual property rights including know-how
previously licensed to the Company, patent rights related to
LX2006, and rights to current and future data generated in an
ongoing investigator-initiated Phase 1A trial of AAVrh.10hFXN to
treat FA cardiomyopathy (NCT05302271).
About LX2006LX2006 is an AAV-based gene therapy
candidate delivered intravenously for the treatment of FA
cardiomyopathy, the most common cause of mortality in patients with
FA affecting approximately 5,000 patients in the United States.
LX2006 is designed to target the cardiac manifestations of FA by
delivering a functional frataxin gene to promote the expression of
the frataxin protein and restore mitochondrial function in
myocardial cells. In preclinical studies, LX2006 reversed the
cardiac abnormalities in FA disease models and showed improvement
in cardiac function and survival while demonstrating a favorable
safety profile. The FDA has granted Rare Pediatric Disease
designation, Fast Track designation, and Orphan Drug designation to
LX2006 for the treatment of FA cardiomyopathy.
About Lexeo Therapeutics Lexeo
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.
Cautionary Note Regarding 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 statements regarding the anticipated benefits
of the license agreement between Lexeo Therapeutics and Cornell
University and the data to be provided thereunder, including the
acceleration of the development of our product candidates and the
timing of approvals, if any. 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 (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 Lexeo’s 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 Lexeo’s Annual Report on Form
10-K for the annual period ended December 31, 2023, filed with the
SEC on March 11, 2024, and subsequent future filings Lexeo 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:Carolyn Hawley(619)
849-5382Carolyn.hawley@inizioevoke.com
Investor Response:Stephen Jasper(858)
525-2047stephen@gilmartinir.com
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