Cabaletta Bio Receives FDA Clearance of IND Application for Treatment of Systemic Sclerosis with CABA-201
02 Ottobre 2023 - 1:00PM
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 third 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 systemic sclerosis (SSc). The Company
plans to initiate a Phase 1/2 clinical trial of CABA-201 across two
parallel SSc cohorts – one cohort of six patients with severe skin
manifestations and a separate cohort of six patients with severe
organ involvement associated with systemic sclerosis. Consistent
with the previously announced CABA-201 IND clearances for lupus and
myositis, the starting dose for the trial, 1 x 106 cells/kg, was
informed by the high degree of similarity between CABA-201 and the
CD19-CAR T construct administered to a patient with severe, diffuse
SSc in the recent Annals of Rheumatic Diseases publication.
“As we remain on track to deliver three-month clinical data from
the initial patients treated with CABA-201 by the first half of
2024, the clearance of our third IND application for CABA-201
within the past 6 months demonstrates our relentless focus on
developing CABA-201 for a broad portfolio of potential indications
in patients with autoimmune diseases,” said Steven Nichtberger,
M.D., Chief Executive Officer and Co-founder of Cabaletta. “Based
on published, third-party academic clinical data and a recently
released abstract by the same academic group at the upcoming
American College of Rheumatology Convergence 2023 meeting reporting
administration of CD19-CAR T cells to treat additional patients
with systemic sclerosis, we believe CABA-201 has the potential to
slow or halt the progression of this autoimmune disease, thereby
providing an important treatment option for these patients who
currently have very few available treatments.”
SSc is a rare and potentially fatal chronic autoimmune disease
characterized by progressive skin and internal organ fibrosis that
can be life-threatening, including interstitial lung disease,
pulmonary hypertension, and scleroderma renal crisis. Although the
etiology of SSc is not well understood, the pathogenic role of
autoantibodies and B cells in SSc provides a rationale for studying
CAR T therapy in this population. SSc affects approximately 88,000
patients in the U.S., and typically affects middle-aged
individuals, particularly women. Standard treatment options, which
have modest effects, include generalized immunosuppressive agents
or drugs targeted to specific symptomatic manifestations.
Autologous hematopoietic stem cell transplant may provide some
benefits in organ involvement, but carries significant risks,
including mortality, infertility, and secondary autoimmune disease,
limiting its potential to be applied broadly. Due to the lack of
adequate treatments, the risk of mortality in systemic sclerosis
remains high, with an average survival of approximately 12 years
following diagnosis.
About the Phase 1/2 Clinical Trial of CABA-201 in
SScThe Phase 1/2 clinical trial will be an open-label
study of CABA-201 in subjects with SSc across two parallel cohorts.
The severe skin cohort will include six patients with severe skin
involvement, and the organ cohort will include six patients who
meet the pulmonary, cardiac, or renal involvement criteria
regardless of skin involvement. Subjects will receive a one-time
infusion of CABA-201, using the same dose being used in the lupus
and myositis 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), evidence of significant skin,
pulmonary, renal, or cardiac involvement, and significant organ
involvement despite use of immunosuppressants. Key exclusion
criteria include a primary diagnosis of another rheumatic
autoimmune disease, treatment with a B cell depleting agent within
six months or treatment with a biologic agent within three months.
As the third 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, potentially
enabling an immune system reset associated with a slowing or
halting of active inflammatory disease progression in patients with
SSc.
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, and systemic
sclerosis, 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 and a recently released abstract
by the same academic group at the upcoming American College of
Rheumatology Convergence 2023 meeting; the anticipated market
opportunities for CABA-201 in SSc patients; the Company’s business
plans and objectives; Cabaletta Bio’s expectations around the
potential success and therapeutic benefits of CABA-201; the
Company’s plans to initiate separate Phase 1/2 clinical trials of
CABA-201 in subjects with SSc, SLE and myositis, including its
anticipated progress, clinical trial design, ability to leverage
its experience in autoimmune cell therapy and autoimmune disease
product development for each clinical trial; the Company’s planned
initial clinical data read-out from the CABA-201 program in the
first half of 2024; 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; 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: 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 Nature Medicine and Annals of
Rheumatic Diseases 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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