Cabaletta Bio Receives FDA Clearance of IND Application for CABA-201 for Treatment of Systemic Lupus Erythematosus
March 31 2023 - 7:00AM
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 Investigational New Drug (IND)
application for CABA-201, a 4-1BB-containing fully human CD19-CAR T
cell investigational therapy, has been cleared by the U.S. Food and
Drug Administration (FDA). The Company plans to initiate a Phase
1/2 clinical trial of CABA-201 for the treatment of systemic lupus
erythematosus (SLE) in patients with active lupus nephritis (LN) or
active SLE without renal involvement.
“We believe the clearance of this IND application within 6
months of licensing the binder for CABA-201 is an important
milestone for patients with autoimmune disease. The efficient
clinical trial design was informed by the data package we
submitted, including clinical safety data with the CABA-201 binder,
our experience from prior autoimmune cell therapy IND applications
and our exclusive translational research partnership with the
senior author of the Nature Medicine paper, which demonstrated 5/5
durable remissions throughout the follow-up period up to 17 months
in patients with refractory SLE,” said Steven Nichtberger, M.D.,
Chief Executive Officer and Co-founder of Cabaletta. “The Phase 1/2
clinical trial will begin in patients with either active LN or SLE
without renal involvement. Based on its similarity to the product
used in the Nature Medicine paper, we believe CABA-201 has the
potential to provide deep and durable responses for patients with
SLE and possibly other autoimmune diseases where B cells play a
role to initiate or sustain disease pathology. By achieving a
timely IND clearance, we believe we are well positioned to generate
3-month clinical data on efficacy endpoints and tolerability
for patients dosed with CABA-201 by the first half of 2024.”
SLE is a chronic, potentially severe, autoimmune disease, most
commonly impacting young women between the ages of 15 and 40 with
higher frequency and more severity in people of color, where the
immune system attacks healthy tissue throughout the body. It is
characterized by abnormal B cell function and autoantibody
production resulting in a range of clinical manifestations
including end organ damage and an increased risk of death. It
affects an estimated 160,000-320,000 patients in the U.S. in total.
LN is the most common end-organ manifestation of SLE, affecting
approximately 40% of SLE patients. Among these patients, the risk
of end-stage renal disease is approximately 17% and the risk of
death is approximately 12%, each within 10 years of diagnosis.
CABA-201 is designed to be given as a one-time infusion, with
the potential to transiently, but fully, eliminate B cells, thus
enabling an “immune system reset” and durable remission in patients
with SLE. The Phase 1/2 clinical trial is an open-label dose
evaluation study designed to evaluate CABA-201 in SLE subjects with
active LN or active SLE without renal involvement. Subjects will be
treated with a standard preconditioning regimen consisting of
fludarabine and cyclophosphamide prior to CABA-201 infusion. This
represents the first trial that employs Cabaletta’s CARTA (Chimeric
Antigen Receptor T cells for Autoimmunity) strategy.
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 lupus nephritis and systemic lupus erythematosus
without renal involvement, 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
may offer potentially curative therapies 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 expectations regarding: Cabaletta’s ability to
grow its autoimmune-focused pipeline; the ability to capitalize on
and potential benefits resulting from the translational research
partnership with Professor Georg Schett and the exclusive license
agreement with IASO Bio; the Company’s business plans and
objectives; Cabaletta Bio’s expectations around the potential
success and therapeutic benefits of CABA-201, including its belief
that CABA-201 may enable an “immune system reset” and provide deep
and durable responses for patients with SLE and potentially for
patients diagnosed with other autoimmune disease; the Company’s
plans to initiate a Phase 1/2 clinical trial of CABA-201 in
patients with SLE, including its anticipated progress, clinical
trial design, ability to leverage its experience in autoimmune cell
therapy and lupus product development; the Company’s planned
initial clinical data read-out in the first half of 2024;
Cabaletta’s ability to enroll the requisite number of patients,
dose each dosing cohort in the intended manner in its Phase 1/2
clinical trial of CABA-201; and the ability to accelerate
Cabaletta’s pipeline and develop meaningful therapies for patients,
including in collaboration with academic and industry partners and
the ability to optimize such collaborations on its development
programs.
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: 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 Nature Medicine publication 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; risks related to the
impact of public health epidemics affecting countries or regions in
which Cabaletta has operations or does business, such as COVID-19;
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
Sarah McCabeStern Investor Relations,
Inc.212-362-1200sarah.mccabe@sternir.com
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