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Cabaletta Bio Reports Fourth Quarter and Full Year 2021 Financial Results and Provides Business Update DesCAARTes trial progressing in cohort A5 with presentation of DSG3-CAART clinical and translational data from cohort

Key Takeaway: Cabaletta Bio Reports Fourth Quarter and Full Year 2021 Financial Results and Provides Business Update DesCAARTes trial progressing in cohort A5 with presentation of DSG3-CAART clinical and translational data from cohorts A3 and A4 and 28-day safety data for cohort A5 expected

Full Press Release Details

Cabaletta Bio Reports Fourth Quarter and Full Year 2021 Financial Results and Provides Business Update
DesCAARTes trial progressing in cohort A5 with presentation
of DSG3-CAART clinical and translational data from cohorts A3 and A4 and 28-day safety data for cohort A5 expected at upcoming scientific meetings in mid-2022
MuSK-CAART Investigational New Drug (IND) application cleared and Fast Track Designation granted by the U.S. Food and Drug
Administration (FDA); planning to initiate first-in-human trial in 2022
Ended 2021 with $122.2 million in cash on hand to fund operations through 3Q 2023
PHILADELPHIA, March 17, 2022 Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on the
discovery and development of targeted cell therapies for patients with autoimmune diseases, today reported financial results for the fourth quarter and full year ended December 31, 2021, and provided a business update.
We are encouraged by early data from the DesCAARTes trial in patients with mucosal
pemphigus vulgaris, including the dose-dependent increase in persistence seen in cohort A3 relative to the two low dose cohorts throughout the 28 days following DSG3-CAART infusion and the absence of dose limiting toxicities observed through cohort
A4 as well as continued investigator engagement and patient interest. We look forward to reporting DSG3-CAART clinical and translational data from the middle dose cohorts A3 and A4 along with 28-day safety
data from cohort A5 at scientific meetings in mid-2022, said Steven Nichtberger, M.D., Chief Executive Officer and Co-founder of Cabaletta. Learnings from
the DesCAARTes trial have provided platform-based insights for our growing autoimmune-focused pipeline, including the
MusCAARTes trial for patients with MuSK-associated myasthenia gravis. MuSK-CAART was recently granted Fast Track Designation by the FDA to improve activities of daily living and muscle
strength in patients with MuSK-associated myasthenia gravis, and with our now-cleared IND application, we plan to initiate the MusCAARTes trial in
2022 as we continue to advance our mission of delivering deep, durable, and potentially curative, responses for patients with autoimmune diseases.
Pipeline Highlights and Anticipated Upcoming Milestones
DSG3-CAART: Desmoglein 3 chimeric autoantibody receptor T (DSG3-CAART) cells as a potential treatment for patients with mucosal pemphigus vulgaris
MuSK-CAART: Muscle-specific kinase (MuSK) chimeric autoantibody receptor T (MuSK-CAART) cells as a potential treatment for patients with
MuSK-associated myasthenia gravis.
PLA2R-CAART: Phospholipase A2 receptor (PLA2R) chimeric autoantibody receptor T (PLA2R-CAART) cells as a potential
treatment for patients with PLA2R-associated membranous nephropathy.
Corporate Highlights
Fourth Quarter and Full Year 2021 Financial Results
The Company expects that its cash and cash equivalents as of December 31, 2021, will enable it to fund its operating plan through the third quarter of
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, in combination with Cabaletta Bio s proprietary technology, has advanced a growing pipeline that currently includes potential treatments for patients with mucosal
pemphigus vulgaris, MuSK-associated myasthenia gravis, PLA2R-associated membranous nephropathy, mucocutaneous pemphigus vulgaris and hemophilia A with FVIII alloantibodies. Cabaletta Bio s headquarters are located in Philadelphia, PA. For more
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 progress and results of its DesCAARTes 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 expected timing and
significance around the announcement of 28-day safety for cohort A5 and biologic activity data for cohorts A3 and A4 in mid-2022; the expected timing and significance
around additional clinical data updates from the DesCAARTes trial at scientific meetings throughout 2022 and 2023; the expectation that Cabaletta may improve outcomes for patients suffering
from mPV; the ability of Oxford
Biomedica to supply Cabaletta with a sufficient quantity and/or quality of lentiviral vector; expectations regarding the intended incentives conferred by Fast Track Designation for MuSK-CAART to
improve activities of daily living and muscle strength in patients with MuSK antibody-positive myasthenia gravis; the expectation that Cabaletta Bio may improve outcomes for patients suffering from MuSK MG; plans to initiate patient dosing in an
open-label Phase 1 clinical trial to evaluate MuSK-CAART safety and tolerability in MuSK MG patients in 2022; Cabaletta s ability to enroll the requisite number of patients, dose each dosing cohort in the intended manner, and progress the
MusCAARTes trial; the ability of MuSK-CAART to target B cells that differentiate into antibody secreting cells, which produce autoantibodies against muscle-specific kinase; the ability
of WuXi Advanced Therapies to supply sufficient quality and quantity of MuSK-CAART for the planned MusCAARTes trial; Cabaletta s plans to advance development of its preclinical
pipeline; the effectiveness and timing of product candidates that Cabaletta may develop, including in collaboration with academic partners; presentation of additional data at upcoming scientific conferences, and other preclinical data; expectations
regarding the design, implementation, timing and success of its current and planned clinical trials and the successful completion of nonclinical studies; planned potential timing and advancement of its preclinical studies and clinical trials and
related regulatory submissions; ability to continue its growth and realize the anticipated contribution of the members of its board of directors and executives to its operations and progress; ability to optimize the impact of its collaborations on
its development programs; the impact of COVID-19 on the timing, progress, interpretability of data, and results of ongoing or planned preclinical and clinical trials; statements regarding the timing of
regulatory filings regarding its development programs; use of capital, expenses, future accumulated deficit and other financial results in the future; and ability to fund operations through the third quarter of 2023.
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 may not inform long-term results; Cabaletta s ability to demonstrate sufficient evidence of safety, efficacy and tolerability in its preclinical and clinical trials of DSG3-CAART; 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 the impact of public health epidemics, such as the ongoing COVID-19 pandemic, affecting countries or regions in which we have operations or do business; Cabaletta s ability to retain and recognize the intended incentives conferred by Orphan Drug Designation and Fast
Track Designation for DSG3-CAART for improving healing of mucosal blisters in patients with mucosal pemphigus vulgaris; Cabaletta s ability to retain and recognize the intended incentives conferred by Fast Track Designation for MuSK-CAART to
improve activities of daily living and muscle strength in patients with MuSK antibody-positive myasthenia gravis; Cabaletta s ability to demonstrate sufficient evidence of safety, efficacy and tolerability in its preclinical and clinical trials
of DSG3-CAART and MuSK-CAART; risks related to fostering and maintaining successful relationships with Cabaletta s manufacturing partners; risks related to Cabaletta s ability to protect and maintain its intellectual property position;
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 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,
2021 2020 2021 2020
Unaudited
Operating expenses:
Research and development 9,919 5,775 32,494 21,376
General and administrative 3,974 3,555 13,819 12,457
Total operating expenses 13,893 9,330 46,313 33,833
Loss from operations (13,893 ) (9,330 ) (46,313 ) (33,833 )
Other income
Interest income 5 21 24 494
Net loss (13,888 ) (9,309 ) (46,289 ) (33,339 )
Net loss per voting and non-voting share, basic and diluted $ (0.49 ) $ (0.40 ) $ (1.80 ) $ (1.44 )
Selected Balance Sheet Data
December 31,
2021 2020
Unaudited
Cash, cash equivalents and investments $ 122,222 $ 108,662
Total assets 126,336 114,724
Total liabilities 8,380 5,180
Total stockholders equity 117,956 109,544
Chief Financial Officer
Stern Investor Relations, Inc.
Last updated: Mar 17, 2022