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Cabaletta Bio Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Business Update Company expecting Investigational New Drug (IND) clearance in the first half of 2023 for CABA-201, a 4-1BB-containing

Key Takeaway: Cabaletta Bio reported its financial results for the fourth quarter and full year of 2022, highlighting expectations for Investigational New Drug (IND) clearance for CABA-201 in the first half of 2023. The company is focused on developing engineered T cell therapies targeting autoimmune diseases and anticipates generating initial clinical data for CABA-201 by mid-2024. Cabaletta has financial resources to support operations through the first quarter of 2025 and is actively progressing its pipeline with ongoing trials for other product candidates. However, risks associated with regulatory approvals and clinical trial outcomes persist.

Market Sentiment Analysis

POSITIVE FACTORS

  • Cabaletta expects IND clearance for CABA-201 in the first half of 2023.
  • Anticipates initial clinical data generation by the first half of 2024.
  • The company has adequate financial resources to fund operations until Q1 2025.
  • They are making progress with other clinical-stage candidates.

CONCERNS & RISKS

  • There are inherent risks related to regulatory filings and potential clearance delays.
  • Concerns about whether initial results from clinical studies will predict future outcomes.
  • Low patient enrollment rates could impact trial timelines.
  • Uncertainties around maintaining relationships with collaboration and manufacturing partners.

Full Press Release Details

Cabaletta Bio Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Business Update
Company expecting Investigational New Drug (IND) clearance in the first half of 2023 for
CABA-201, a 4-1BB-containing fully human CD19-CAR T cell therapy, with potential to
generate initial clinical data by the first half of 2024
PHILADELPHIA, March 16, 2023 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 reported financial results for the fourth quarter and full year ended
December 31, 2022, and provided a business update.
As we seek FDA clearance for CABA-201 in the next
few months, we believe that our specifically designed product candidate for autoimmune patients, our experience with efficient autoimmune cell therapy clinical trial design coupled with timely implementation of complicated autoimmune cell therapy
trials and our exclusive translational research partnership with Georg Schett, M.D., which is currently delivering actionable clinical insights, provide us with the opportunity to deliver potentially transformative outcomes for patients with a broad
range of autoimmune diseases, said Steven Nichtberger, M.D., Chief Executive Officer and Co-founder of Cabaletta. In parallel, we continue to make progress on our pipeline of clinical-stage CAART
legacy product candidates, with 1-month safety and persistence data for the combination sub-study in the
DesCAARTes trial for DSG3-CAART anticipated in the first half of 2023 and recruitment in the MusCAARTes trial for MuSK-CAART
ongoing. Looking ahead, we are confident in our ability to advance our autoimmune-focused pipeline for patients with serious unmet need and deliver on multiple upcoming value-creating milestones.
Recent Operational Highlights and Upcoming Milestones
Chimeric Antigen Receptor T cells for Autoimmunity (CARTA) Strategy
CABA-201: Autologous, engineered T cells with a chimeric antigen receptor containing a fully human CD19
binder and a 4-1BB co-stimulatory domain as a potential treatment for a broad range of autoimmune diseases where B cells contribute to the initiation and/or maintenance
Chimeric AutoAntibody Receptor T (CAART) cells Strategy
DSG3-CAART: Desmoglein 3 chimeric autoantibody receptor T (DSG3-CAART) cells as a potential treatment for patients with mucosal pemphigus vulgaris
Muscle-specific kinase (MuSK) chimeric autoantibody receptor T (MuSK-CAART) cells as a potential treatment for patients with MuSK-associated myasthenia gravis.
Corporate Highlights
Cabaletta will participate in the upcoming 22nd Annual Needham Virtual Healthcare Conference, which is being held from April 17 20,
Fourth Quarter and Full Year 2022 Financial Results
The Company expects that its cash, cash equivalents and
investments as of December 31, 2022, will enable it to fund its operating plan into the first quarter of 2025.
Cabaletta 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 CD19-CAR T, as the
lead product candidate, 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 are located in Philadelphia, PA.
Forward-Looking Statements
This 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 Georg Schett, M.D., and the exclusive license agreement with IASO Bio;
the company s business plans and objectives; the timing of its IND clearance for CABA-201, initiation of clinical evaluation of CABA-201 and generation of initial
clinical data for CABA-201; statements regarding anticipated significance of, and timing of release of, safety and persistence data and combination cohort data; statements regarding regulatory filings for its
development programs, including the planned timing of such regulatory filings and potential review by such regulatory authorities; the expectation that Cabaletta Bio may improve outcomes for patients suffering from mPV, MG, or other autoimmune
diseases; the progress and results of its DesCAARTes Phase 1 trial and MusCAARTes Phase 1 trial, including Cabaletta s ability
to enroll the requisite number of patients, dose each dosing cohort in the intended manner, and progress the trial; 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; use of capital, expenses, future accumulated deficit and other financial results in the future; ability to fund operations into the first
quarter of 2025; and the anticipated contribution of the members of Cabaletta s executives to the company s operations and progress.
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 Lancet Rheumatology publications 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 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.
SELECTED FINANCIAL DATA
(unaudited; in thousands, except share and per share data)
Statements of Operations
Three months ended December 31, Year Ended December 31,
2022 2021 2022 2021
Unaudited
Operating expenses:
Research and development 12,400 9,919 39,300 32,494
General and administrative 3,902 3,974 14,839 13,819
Total operating expenses 16,302 13,893 54,139 46,313
Loss from operations (16,302 ) (13,893 ) (54,139 ) (46,313 )
Other income
Interest income 610 5 1,164 24
Net loss (15,692 ) (13,888 ) (52,975 ) (46,289 )
Net loss per voting and non-voting share, basic and diluted $ (0.52 ) $ (0.49 ) $ (1.81 ) $ (1.80 )
Selected Balance Sheet Data
December 31,
2022 2021
Unaudited
Cash, cash equivalents and investments $ 106,547 $ 122,222
Total assets 116,968 126,336
Total liabilities 12,448 8,380
Total stockholders equity 104,520 117,956
Chief Financial Officer
Stern Investor Relations, Inc.

Frequently Asked Questions

What is CABA-201?

CABA-201 is a fully human CD19-CAR T cell therapy designed for autoimmune diseases.

When is the expected IND clearance for CABA-201?

The IND clearance for CABA-201 is expected in the first half of 2023.

What diseases can DSG3-CAART treat?

DSG3-CAART cells are aimed at treating mucosal pemphigus vulgaris.

What financial period does the report cover?

The report covers the fourth quarter and full year ended December 31, 2022.

How long can Cabaletta fund operations?

Cabaletta's funds are expected to sustain operations into the first quarter of 2025.

Last updated: Mar 16, 2023