Cabaletta Bio Receives FDA Clearance of CABA-201 IND Application for Treatment of Generalized Myasthenia Gravis
06 Noviembre 2023 - 6:00AM
Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology
company focused on developing and launching the first curative
targeted cell therapies for patients with autoimmune diseases,
today announced that the Company’s fourth Investigational New Drug
(IND) application for CABA-201, a 4-1BB-containing fully human
CD19-CAR T cell investigational therapy, has been allowed to
proceed by the U.S. Food and Drug Administration (FDA) for a Phase
1/2 study in patients with generalized myasthenia gravis (gMG). The
Company plans to initiate a Phase 1/2 clinical trial of CABA-201
across two parallel gMG cohorts based on autoantibody status – one
cohort of six patients with acetylcholine receptor (AChR)
antibody-positive gMG and a second cohort of six patients with AChR
antibody-negative gMG. Consistent with the previously announced
CABA-201 IND application clearances for lupus, myositis and
systemic sclerosis, the starting dose for the Phase 1/2 trial
evaluating CABA-201 in gMG will be 1 x 106 cells/kg.
“While we remain on track to deliver initial clinical data from
CABA-201 treated patients with lupus and/or myositis in the first
half of 2024, we are continuing to expand our CABA-201 portfolio
beyond rheumatology and into neurology. The announcement of our
fourth CABA-201 IND clearance is also our first IND clearance for
the product candidate in a predominantly autoantibody mediated
disease. Consistent with previously announced CABA-201 trials, the
Phase 1/2 clinical trial design for CABA-201 in gMG includes the
same starting dose as used in the previously cleared clinical
trials with CABA-201 and parallel cohorts of six patients. While
prioritizing delivery of initial clinical data in the first half of
2024 from our myositis and/or lupus trials, our clinical operations
team is well positioned to execute the gMG program based on
experience with MG sites in our legacy CAART platform,” said Steven
Nichtberger, M.D., Chief Executive Officer and Co-founder of
Cabaletta. “Despite recent advances with chronic, broadly
immunosuppressive therapies, we believe there is an unmet need for
a treatment option like CABA-201 that may provide a deep and
durable, perhaps curative, outcome with a single dose in patients
with gMG.”
Myasthenia gravis (MG) is a rare autoimmune disease
characterized by autoantibodies that interfere with signaling at
the neuromuscular junction (NMJ), leading to potentially
life-threatening muscle weakness. The majority of patients with MG
have autoantibodies known to be pathogenic based on their
interference with proteins in the NMJ, of which the majority target
AChR. Generalized MG affects approximately 85% of the between
50,000 and 80,000 estimated MG patients in the United States.
Symptoms of gMG include profound muscle weakness throughout the
body, disabling fatigue, shortness of breath due to respiratory
muscle weakness and risk for episodes of respiratory failure.
Standard of care therapies include cholinesterase inhibitors,
steroids, immunomodulators, and biologics, which often provide
modest clinical effect and require chronic administration,
increasing the risk of serious long-term side effects.
About the Phase 1/2 Clinical Trial of CABA-201 in
gMGThe Phase 1/2 clinical trial will be an open-label
study of CABA-201 in subjects with gMG across two parallel cohorts.
The first cohort will include six patients with AChR
antibody-positive gMG, and the second will include six patients
with AChR antibody-negative gMG. Participants will receive a
one-time infusion of CABA-201, using the same dose being used in
the lupus, myositis and systemic sclerosis clinical trials of
CABA-201, 1 x 106 cells/kg, preceded by a standard preconditioning
regimen of fludarabine and cyclophosphamide. Key inclusion criteria
include patients between ages 18 and 70 (inclusive), a diagnosis of
gMG, and disease activity despite standard of care therapies. Key
exclusion criteria include myasthenia gravis with only ocular
manifestations, an active or untreated thymoma, and treatment with
a B cell depleting agent within six months. As the fourth trial
within Cabaletta’s CARTA (Chimeric Antigen Receptor T cells for
Autoimmunity) strategy, this study is intended to evaluate the
potential ability of CABA-201 to transiently, but completely,
eliminate B cells throughout the body.
About Cabaletta BioCabaletta Bio (Nasdaq: CABA)
is a clinical-stage biotechnology company focused on the discovery
and development of engineered T cell therapies that have the
potential to provide a deep and durable, perhaps curative,
treatment for patients with autoimmune diseases. The CABA™ platform
encompasses two strategies: the CARTA (chimeric antigen receptor T
cells for autoimmunity) strategy, with CABA-201, a 4-1BB-containing
fully human CD19-CAR T, as the lead product candidate being
evaluated in systemic lupus erythematosus, myositis, systemic
sclerosis and generalized myasthenia gravis, and the CAART
(chimeric autoantibody receptor T cells) strategy, with multiple
clinical-stage candidates, including DSG3-CAART for mucosal
pemphigus vulgaris and MuSK-CAART for MuSK myasthenia gravis. The
expanding CABA™ platform is designed to develop potentially
curative therapies that offer deep and durable responses for
patients with a broad range of autoimmune diseases. Cabaletta Bio’s
headquarters and labs are located in Philadelphia, PA.
Forward-Looking StatementsThis press release
contains “forward-looking statements” of Cabaletta Bio within the
meaning of the Private Securities Litigation Reform Act of 1995, as
amended, including without limitation, express or implied
statements regarding its expectations regarding: Cabaletta’s
ability to grow its autoimmune-focused pipeline; the ability to
capitalize on and potential benefits resulting from published
third-party academic clinical data; the anticipated market
opportunities for CABA-201 in gMG patients; the Company’s business
plans and objectives; Cabaletta’s expectations around the potential
success and therapeutic benefits of CABA-201, including its belief
in the potential for CABA-201 to provide a deep and durable,
perhaps curative, outcome with a single dose in patients with gMG;
the Cabaletta clinical operations team’s ability to execute the gMG
program based on experience with MG sites in the Company’s legacy
CAART platform; the Company’s plans to initiate and progress
separate Phase 1/2 clinical trials of CABA-201 in subjects with
gMG, SSc, SLE and myositis; the Company’s planned initial clinical
data read-out from the CABA-201 program in the first half of 2024;
and Cabaletta’s ability to enroll the requisite number of patients
and dose each dosing cohort in the intended manner in its Phase 1/2
clinical trials of CABA-201.
Any forward-looking statements in this press release are based
on management’s current expectations and beliefs of future events,
and are subject to a number of risks and uncertainties that could
cause actual results to differ materially and adversely from those
set forth in or implied by such forward-looking statements. These
risks and uncertainties include, but are not limited to: risks
related to regulatory filings and potential clearance; the risk
that signs of biologic activity or persistence may not inform
long-term results; Cabaletta’s ability to demonstrate sufficient
evidence of safety, efficacy and tolerability in its preclinical
studies and clinical trials of DSG3-CAART, MuSK-CAART and CABA-201;
the risk that the results observed with the similarly-designed
construct employed in the recent academic publications, including
due to the dosing regimen, are not indicative of the results we
seek to achieve with CABA-201; risks related to clinical trial site
activation or enrollment rates that are lower than expected; risks
related to unexpected safety or efficacy data observed during
clinical studies; risks related to volatile market and economic
conditions and public health crises; Cabaletta’s ability to retain
and recognize the intended incentives conferred by Orphan Drug
Designation and Fast Track Designation for its product candidates,
as applicable; risks related to Cabaletta’s ability to protect and
maintain its intellectual property position; risks related to
fostering and maintaining successful relationships with Cabaletta’s
collaboration and manufacturing partners; uncertainties related to
the initiation and conduct of studies and other development
requirements for its product candidates; the risk that any one or
more of Cabaletta’s product candidates will not be successfully
developed and/or commercialized; and the risk that the initial or
interim results of preclinical studies or clinical studies will not
be predictive of future results in connection with future studies.
For a discussion of these and other risks and uncertainties, and
other important factors, any of which could cause Cabaletta’s
actual results to differ from those contained in the
forward-looking statements, see the section entitled “Risk Factors”
in Cabaletta’s most recent annual report on Form 10-K as well as
discussions of potential risks, uncertainties, and other important
factors in Cabaletta’s other and subsequent filings with the
Securities and Exchange Commission. All information in this press
release is as of the date of the release, and Cabaletta undertakes
no duty to update this information unless required by law.
Contacts:
Anup MardaChief Financial Officerinvestors@cabalettabio.com
William GramigStern Investor Relations,
Inc.william.gramig@sternir.com
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