CABA Cabaletta Bio Inc.

11.6
+0.34  (+3%)
Previous Close 11.26
Open 11.05
52 Week Low 7
52 Week High 16.38
Market Cap $328,647,001
Shares 28,331,638
Float 24,179,748
Enterprise Value $199,754,243
Volume 53,900
Av. Daily Volume 172,458
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Upcoming Catalysts

Drug Stage Catalyst Date
DSG3-CAART
Pemphigus vulgaris
Phase 1
Phase 1
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Drug Pipeline

Drug Stage Notes
MuSK-CAART
MuSK-associated myasthenia gravis
Phase 1
Phase 1
IND submission in 4Q 2021 and Phase 1 trial planned to commence in 2022.

Latest News

  1. PHILADELPHIA, Nov. 09, 2021 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (NASDAQ:CABA), a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies for patients with B cell-mediated autoimmune diseases, today announced that the Company will participate in two upcoming investor conferences in November:

    • Guggenheim Securities 3rd Annual Neuro/Immunology Conference: Steven Nichtberger, M.D., President and Chief Executive Officer, will participate in a fireside chat on Tuesday, November 16, 2021 at 8:15 a.m. ET. Members of management will also be available for one-on-one meetings.

    • Evercore ISI 4th Annual HealthCONx Conference: Steven Nichtberger, M.D., President and Chief Executive Officer, will participate…

    PHILADELPHIA, Nov. 09, 2021 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (NASDAQ:CABA), a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies for patients with B cell-mediated autoimmune diseases, today announced that the Company will participate in two upcoming investor conferences in November:

    • Guggenheim Securities 3rd Annual Neuro/Immunology Conference: Steven Nichtberger, M.D., President and Chief Executive Officer, will participate in a fireside chat on Tuesday, November 16, 2021 at 8:15 a.m. ET. Members of management will also be available for one-on-one meetings.



    • Evercore ISI 4th Annual HealthCONx Conference: Steven Nichtberger, M.D., President and Chief Executive Officer, will participate in a fireside chat on Tuesday, November 30, 2021 at 12:35 p.m. ET. Members of management will also be available for one-on-one meetings.

    Both presentations will be available on the News and Events section of the company's website at www.cabalettabio.com. Replays will be available on the website for 90 days.

    About Cabaletta Bio

    Cabaletta Bio is a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies, and exploring their potential to provide a deep and durable, perhaps curative, treatment, for patients with B cell-mediated autoimmune diseases. The Cabaletta Approach to selective B cell Ablation (CABA™) platform, in combination with Cabaletta's proprietary technology, utilizes Chimeric Auto Antibody Receptor (CAAR) T cells that are designed to selectively bind and eliminate only specific autoantibody-producing B cells while sparing normal antibody-producing B cells, which are essential for human health. The Company's lead product candidate, DSG3-CAART, is being evaluated in the DesCAARTes™ Phase 1 clinical trial as a potential treatment for patients with mucosal pemphigus vulgaris, a prototypical B cell-mediated autoimmune disease. The FDA granted Fast Track Designation for DSG3-CAART in May 2020. For more information about the DesCAARTes™ Phase 1 clinical trial, please visit our website (DesCAARTes™ Phase 1 Trial). The Company's lead preclinical product candidate, MuSK-CAART, is in IND-enabling studies and is designed as a potential treatment for patients with MuSK-associated myasthenia gravis. For more information, visit www.cabalettabio.com.

    Contacts:

    Anup Marda

    Chief Financial Officer

    Sarah McCabe

    Stern Investor Relations, Inc.

    212-362-1200



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  2. PHILADELPHIA, Nov. 09, 2021 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (NASDAQ:CABA), a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies for patients with B cell-mediated autoimmune diseases, today announced the appointments of Samik Basu, M.D. to Chief Scientific Officer (CSO) and Heather Harte-Hall to Chief Compliance Officer (CCO). In these new roles, Dr. Basu and Ms. Harte-Hall will continue to report to Gwendolyn Binder, Ph.D., Cabaletta Bio's Executive Vice President of Science & Technology.

    "Samik and Heather are both highly respected and accomplished leaders at Cabaletta and we are thrilled to announce their well-deserved promotions to these new roles. Samik has demonstrated…

    PHILADELPHIA, Nov. 09, 2021 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (NASDAQ:CABA), a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies for patients with B cell-mediated autoimmune diseases, today announced the appointments of Samik Basu, M.D. to Chief Scientific Officer (CSO) and Heather Harte-Hall to Chief Compliance Officer (CCO). In these new roles, Dr. Basu and Ms. Harte-Hall will continue to report to Gwendolyn Binder, Ph.D., Cabaletta Bio's Executive Vice President of Science & Technology.

    "Samik and Heather are both highly respected and accomplished leaders at Cabaletta and we are thrilled to announce their well-deserved promotions to these new roles. Samik has demonstrated superior leadership guiding the preclinical research and translational medicine teams through the discovery and development of our deep pipeline of precision therapies for patients with B cell-mediated autoimmune diseases. Heather has established quality and compliance programs, which along with her insights and strategic guidance, have proven invaluable as we have advanced our pipeline, most notably our lead product candidate DSG3-CAART, in mucosal pemphigus vulgaris," said Dr. Binder.

    "The addition of Samik and Heather to our leadership team positions us well to deliver on the next phase of our growth including the expansion of our laboratories, continued quality and compliance around our expanding manufacturing efforts and multiple clinical and corporate milestones related to our portfolio of potentially curative products for patients with B cell mediated autoimmune diseases," said Steven Nichtberger, M.D., Chief Executive Officer and Co-founder of Cabaletta.

    Dr. Basu joined Cabaletta in December 2019 and was most recently Vice President of Preclinical Research and Translational Medicine before assuming his new role as Chief Scientific Officer. Prior to joining Cabaletta, Dr. Basu was Head of Translational Sciences (Medicine) at Adaptimmune Therapeutics, plc, where he led research efforts focused on understanding the mechanisms of resistance and response to T-cell receptor based adoptive immunotherapies to inform next generation approaches and clinical strategies. Before that, Dr. Basu co-led preclinical development efforts for Keytruda® (pembrolizumab) at Merck Research Laboratories. He is a physician-scientist with 17 years of industry and academic experience in adoptive immunotherapy, translational research, autoimmunity, and tumor immunology with prior roles at the National Institutes of Health, Albert Einstein College of Medicine, and the University of Pennsylvania. Dr. Basu holds an M.D. from Temple University and completed residency training in Clinical Pathology.

    Ms. Harte-Hall joined Cabaletta in March 2019 and was most recently Vice President of Quality and Compliance before assuming her new role as Chief Compliance Officer. Previously, she was Head of Clinical Quality and Compliance at Adaptimmune Therapeutics, plc, where she built a compliance program and helped establish a culture of integrity to drive ethical behavior in the management and conduct of clinical trials. Ms. Harte-Hall developed and implemented quality and compliance programs focusing on risk management and compliance functions to improve activities such as regulatory monitoring, auditing, and managing alignment with policies, procedures and controls. Ms. Harte-Hall has over 15 years of experience in pharmaceutical and healthcare management and has held various positions in quality and compliance at Centocor Biotech, Inc., Wyeth, LLC and Pfizer Inc. She currently serves as a committee member for the Alliance for Regenerative Medicine (ARM) Accelerator Group and a member of the Society of Corporate Compliance and Ethics (SCCE). Ms. Harte-Hall holds a Master of Science in Psychology from Capella University and B.A. in Business Administration from St. Leo University.

    About Cabaletta Bio

    Cabaletta Bio is a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies, and exploring their potential to provide a deep and durable, perhaps curative, treatment, for patients with B cell-mediated autoimmune diseases. The Cabaletta Approach to selective B cell Ablation (CABA™) platform, in combination with Cabaletta's proprietary technology, utilizes Chimeric Auto Antibody Receptor (CAAR) T cells that are designed to selectively bind and eliminate only specific autoantibody-producing B cells while sparing normal antibody-producing B cells, which are essential for human health. The Company's lead product candidate, DSG3-CAART, is being evaluated in the DesCAARTes™ Phase 1 clinical trial as a potential treatment for patients with mucosal pemphigus vulgaris, a prototypical B cell-mediated autoimmune disease. The FDA granted Fast Track Designation for DSG3-CAART in May 2020. For more information about the DesCAARTes™ Phase 1 clinical trial, please visit our website (DesCAARTes™ Phase 1 Trial). The Company's lead preclinical product candidate, MuSK-CAART, is in IND-enabling studies and is designed as a potential treatment for patients with MuSK-associated myasthenia gravis. For more information, visit www.cabalettabio.com.

    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 the progress and results of its DesCAARTes™ Phase 1 trial; Cabaletta Bio's business plan and objectives; the expectation that Cabaletta Bio may improve outcomes for patients suffering from mucosal pemphigus vulgaris (mPV); the safety, efficacy and tolerability of DSG3-CAART for the treatment of mPV; the impact of COVID-19 on the timing, progress, interpretability of data, and results of ongoing or planned clinical trials; the ability to develop and advance deep pipeline of precision therapies for patients with B cell-mediated autoimmune diseases; and the anticipated contribution of Cabaletta Bio's executives to its operations and progress.

    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 Bio'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 affecting countries or regions in which we have operations or do 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 DSG3-CAART for the treatment of pemphigus vulgaris; 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; 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.

    Contacts:

    Anup Marda

    Chief Financial Officer

    Sarah McCabe

    Stern Investor Relations, Inc.

     



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  3. – Dose dependent increase in DSG3-CAART persistence observed in the third dose cohort with 500 million DSG3-CAART cells relative to the first two low dose cohorts in DesCAARTes™ Phase 1 clinical trial throughout the 28 days following infusion –

    – No dose limiting toxicities (DLTs) or clinically relevant adverse events observed as of October 31, 2021, in the first three dose cohorts –

    Dosing initiated in fourth patient cohort at a dose of 2.5 billion DSG3-CAART cells. DesCAARTes™ trial advancing toward key milestones; top-line biologic activity data from first two low dose cohorts expected to be announced in 4Q21 –

    – Lead preclinical program, MuSK-CAART, Investigational New Drug (IND) submission on track for 4Q21; PLA2R-CAART preclinical

    – Dose dependent increase in DSG3-CAART persistence observed in the third dose cohort with 500 million DSG3-CAART cells relative to the first two low dose cohorts in DesCAARTes™ Phase 1 clinical trial throughout the 28 days following infusion –

    – No dose limiting toxicities (DLTs) or clinically relevant adverse events observed as of October 31, 2021, in the first three dose cohorts –

    Dosing initiated in fourth patient cohort at a dose of 2.5 billion DSG3-CAART cells. DesCAARTes™ trial advancing toward key milestones; top-line biologic activity data from first two low dose cohorts expected to be announced in 4Q21 –

    – Lead preclinical program, MuSK-CAART, Investigational New Drug (IND) submission on track for 4Q21; PLA2R-CAART preclinical data to be presented at the American Society of Nephrology's Kidney Week that show potential as a precision therapy for patients with PLA2R membranous nephropathy –

    – Ended the quarter with $119.3M in cash, extending the cash runway to fund operations through at least 1Q23 –

    PHILADELPHIA, Nov. 01, 2021 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (NASDAQ:CABA), a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies for patients with B cell-mediated autoimmune diseases, today reported financial results for the third quarter ended September 30, 2021, and provided a business update.

    "The DesCAARTes™ trial for DSG3-CAART for patients with mucosal-dominant pemphigus vulgaris has demonstrated encouraging momentum, with continued strong patient enrollment as well as new site and investigator engagement. Dose dependent increases in DSG3-CAART persistence in the third cohort through 28 days following infusion have been observed, as well as the continued absence of any DLTs or clinically relevant adverse events for the first three cohorts as of October 31, 2021. Our next anticipated data readout will include top-line biologic activity data from the first two low dose cohorts, which we expect to announce in the fourth quarter of 2021. We look forward to continuing to generate data on potential biologic activity as we proceed to higher dosing cohorts, with the goal of providing a targeted, highly effective, and potentially curative, therapy without generalized immunosuppression," said Steven Nichtberger, M.D., Chief Executive Officer and Co-founder of Cabaletta. "We are operating under a strengthened balance sheet as a result of $32 million in additional gross proceeds through our "at-the-market" (ATM) equity offering program. In addition to advancing the DesCAARTes™ trial, we are also focused on growing our novel pipeline. To that end, we expect to progress our two lead preclinical programs in the balance of the year with the submission of an IND to the FDA for MuSK-CAART being developed for patients with the MuSK form of myasthenia gravis, and a pre-IND interaction with the FDA to align on a development path for PLA2R-CAART being developed for patients with PLA2R-associated membranous nephropathy."

    Autoimmune Disease-Focused 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 (mPV).

    • Observance of dose dependent DSG3-CAART persistence and favorable safety profile through cohort three of the DesCAARTes™ Phase 1 trial: The Company announced today that three patient cohorts in the DesCAARTes™ Phase 1 trial have completed DSG3-CAART dosing as of October 31, 2021. The Company observed a dose dependent increase in persistence of DSG3-CAART in the third 500 million cell cohort relative to the first two low dose cohorts throughout the 28 days following infusion. In addition, no clinically relevant adverse events or DLTs were observed during the 28-day monitoring period post-infusion. These safety data were observed without preconditioning, and in the presence of circulating anti-DSG3 antibodies. This safety profile builds off 28-day safety data from three patients in the second cohort that the Company reported in August 2021.



    • New site activations driving patient enrollment: As of October 31, 2021, three additional clinical sites were opened for recruitment, doubling the total number of activated DesCAARTes™ trial sites to six.



    • Trial advancing through fourth patient cohort: Dosing was initiated in the fourth patient cohort at a dose of 2.5 billion DSG3-CAART cells. The Company anticipates announcing 28-day safety data for the fourth dose cohort in the first quarter of 2022.



    • Near-term biologic activity data expected for the first two low dose cohorts: Cabaletta plans to announce top-line biologic activity data from the first two low dose cohorts in the fourth quarter of 2021.

    MuSK-CAART: Muscle Specific Kinase (MuSK) chimeric autoantibody receptor T (MuSK-CAART) cells as a potential treatment for patients with MuSK-associated myasthenia gravis.

    • IND studies ongoing and Phase 1 trial planned for 2022: IND-enabling studies consistent with U.S. Food and Drug Administration (FDA) guidance received during the pre-IND meeting are ongoing. The Company remains on track to submit an IND to the FDA in the fourth quarter of 2021. This IND submission will incorporate clinical trial design and data insights from the DesCAARTes™ trial, including starting dose and dose fractionation regimen.



    • GMP manufacturing secured with WuXi: The Company has implemented its manufacturing process with WuXi Advanced Therapies, Inc., its GMP manufacturing partner for the planned MuSK-CAART clinical study.

    PLA2R-CAART: Phospholipase A2 receptor (PLA2R) chimeric autoantibody receptor T (PLA2R-CAART) cells as a potential treatment for patients with PLA2R-associated membranous nephropathy.

    • Early preclinical validation of PLA2R-Chimeric AutoAntibody Receptor T cell candidates will be presented at ASN Kidney Week 2021: Preclinical data demonstrated that Chimeric Auto Antibody Receptor (CAAR) T cells specifically recognized and eliminated anti-PLA2R antibody-expressing B cells and membrane proteome arrays screened with PLA2R CAAR candidates did not identify off-target interactions. These data will be presented as an oral abstract by University of Pennsylvania professor Aimee Payne, M.D., Ph.D., Cabaletta Bio co-founder and Scientific Advisory Board co-chair, at the American Society of Nephrology (ASN) Kidney Week 2021.



    • PLA2R-CAART advancing toward clinical development: Cabaletta expects to conduct a pre-IND interaction with the FDA in the fourth quarter of 2021. The Company expects to discuss the future development path and determine its potential IND submission timing for the program.

    Corporate Highlights

    • Expanded executive leadership team with key appointment to support future growth: In September 2021, Michael Gerard was appointed general counsel. Mr. Gerard joined Cabaletta with a wide range of experience in strategic legal and corporate matters within the life sciences industry. Most recently, Mr. Gerard served as associate general counsel at Spark Therapeutics, Inc., where he supported the global gene therapy Manufacturing, Business Development, Technical Development, Supply Chain, Quality, Alliance Management, Real Estate, IT and Facilities teams.

    Upcoming Events in the Fourth Quarter of 2021

    • Cabaletta will participate in a fireside chat at the Guggenheim Securities 3rd Annual Neuro/Immunology Conference in November 2021.



    • Cabaletta will participate in a fireside chat at the 4th Annual Evercore ISI HealthCONx Conference in November 2021.

    Third Quarter 2021 Financial Results

    The Company expects that its cash, cash equivalents and investments as of September 30, 2021, will enable it to fund its operating plan through at least the first quarter of 2023.

    • Research and development expenses for the three months ended September 30, 2021, were $8.2 million, compared to $5.7 million for the same period in 2020.



    • General and administrative expenses for the three months ended September 30, 2021, were $3.4 million, compared to $2.8 million for the same period in 2020.



    • As of September 30, 2021, Cabaletta had cash, cash equivalents and investments of $119.3 million, compared to $108.7 million as of December 31, 2020. This increase primarily reflects net proceeds of $34.7 million from sales of common stock under Cabaletta's ATM offering program in the nine months ended September 30, 2021, partially offset by cash used in operations. In October 2021, the Company sold an additional 600,000 shares of its common stock through its ATM program, generating additional net proceeds of approximately $6.3 million.

    About Cabaletta Bio

    Cabaletta Bio is a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies, and exploring their potential to provide a deep and durable, perhaps curative, treatment, for patients with B cell-mediated autoimmune diseases. The Cabaletta Approach to selective B cell Ablation (CABA™) platform, in combination with Cabaletta's proprietary technology, utilizes CAAR T cells that are designed to selectively bind and eliminate only specific autoantibody-producing B cells while sparing normal antibody-producing B cells, which are essential for human health. The Company's lead product candidate, DSG3-CAART, is being evaluated in the DesCAARTes™ phase 1 clinical trial as a potential treatment for patients with mucosal pemphigus vulgaris, a prototypical B cell-mediated autoimmune disease. The FDA granted Fast Track Designation for DSG3-CAART in May 2020. For more information about the DesCAARTes™ Phase 1 clinical trial, please visit our website (DesCAARTes™ Phase 1 Trial). The Company's lead preclinical product candidate, MuSK-CAART, is in IND-enabling studies and is designed as a potential treatment for patients with MuSK-associated myasthenia gravis. For more information, visit www.cabalettabio.com.

    University of Pennsylvania Financial Disclosure

    Dr. Payne is a University of Pennsylvania (Penn) faculty member, scientific collaborator, key advisor, and co-founder of Cabaletta Bio. As such, she holds an equity stake in the Company, her laboratory at Penn receives sponsored research funding from Cabaletta Bio, and as an inventor of the licensed technology she may receive additional future financial benefits under licenses granted by Penn to Cabaletta Bio. The University of Pennsylvania may also receive future financial benefit under licenses it has granted to Cabaletta Bio.

    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: 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 the fourth dose cohort in the first quarter of 2022 and top-line biologic activity data for the first two low dose cohorts in the fourth quarter of 2021; the expectation that Cabaletta may improve outcomes for patients suffering from mPV; the progress of its MuSK-CAART program, including the completion and expected results of its ongoing IND-enabling studies and plans to submit an IND application or equivalent regulatory filing for MuSK-CAART in the fourth quarter of 2021; Cabaletta's plans to conduct a pre-IND interaction with the FDA for PLA2R-CAART in the fourth quarter of 2021; 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; the impact of COVID-19 on the timing, progress, interpretability of data, and results of ongoing or planned preclinical and clinical trials; statements regarding 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 at least the first 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 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 Fast Track Designation for DSG3-CAART for improving healing of mucosal blisters in patients with mucosal pemphigus vulgaris, respectively; 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; 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.

     
    CABALETTA BIO, INC.

    SELECTED FINANCIAL DATA

    (unaudited; in thousands, except share and per share data)
     
    Statements of Operations
     
      Three Months Ended

    September 30,
     Nine Months Ended

    September 30,
       2021   2021   2021   2020 
             
                     
      unaudited unaudited
    Operating expenses:        
    Research and development $8,169  $5,650  $22,575  $15,601 
    General and administrative  3,394   2,766   9,845   8,902 
    Total operating expenses  11,563   8,416   32,420   24,503 
    Loss from operations  (11,563)  (8,416)  (32,420)  (24,503)
    Other income:        
    Interest income  3   23   19   473 
    Net loss  (11,560)  (8,393)  (32,401)  (24,030)
    Net loss per share of voting and non-voting

    common stock, basic and diluted
     $(0.45) $(0.36) $(1.31) $(1.09)



    Selected Balance Sheet Data
     
      September 30,

    2021
     December 31,

    2020
           
      (unaudited)
         
    Cash, cash equivalents and investments $        119,260 $        108,662
    Total assets  122,638  114,724
    Total liabilities         6,023  5,180
    Total stockholders' equity                  116,615  109,544
           

    Contacts:

    Anup Marda

    Chief Financial Officer

     

    Sarah McCabe

    Stern Investor Relations, Inc.

    212-362-1200

     



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  4. – Dose dependent increase in DSG3-CAART persistence observed in the third dose cohort relative to the first two low dose cohorts throughout the 28 days following infusion –

    – No dose-limiting toxicities (DLTs) or clinically relevant adverse events observed as of October 31, 2021, in the first three dose cohorts, dosing up to 500 million DSG3-CAART cells –

    – Dosing initiated in fourth patient cohort at a dose of 2.5 billion DSG3-CAART cells with 28-day safety data anticipated in 1Q22 –

    – Top-line biologic activity data for the first two low dose cohorts anticipated to be announced in 4Q21 –

    PHILADELPHIA, Nov. 01, 2021 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (NASDAQ:CABA), a clinical-stage biotechnology company focused on the discovery and…

    – Dose dependent increase in DSG3-CAART persistence observed in the third dose cohort relative to the first two low dose cohorts throughout the 28 days following infusion –

    – No dose-limiting toxicities (DLTs) or clinically relevant adverse events observed as of October 31, 2021, in the first three dose cohorts, dosing up to 500 million DSG3-CAART cells –

    – Dosing initiated in fourth patient cohort at a dose of 2.5 billion DSG3-CAART cells with 28-day safety data anticipated in 1Q22 –

    – Top-line biologic activity data for the first two low dose cohorts anticipated to be announced in 4Q21 –

    PHILADELPHIA, Nov. 01, 2021 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (NASDAQ:CABA), a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies for patients with B cell-mediated autoimmune diseases, today announced 28-day clinical data from the third dose cohort using 500 million DSG3-CAART cells in the DesCAARTes™ Phase 1 clinical trial for the treatment of patients with mucosal-dominant pemphigus vulgaris (mPV).

    As of October 31, 2021, three patient cohorts in the DesCAARTes™ Phase 1 trial have completed DSG3-CAART dosing. The Company observed a dose dependent increase in DSG3-CAART persistence in the third cohort relative to the first two low dose cohorts throughout the 28 days following infusion. In addition, no clinically relevant adverse events or DLTs were observed during the 28-day monitoring period post-infusion. These safety data were observed without preconditioning, and in the presence of circulating anti-DSG3 antibodies.

    "We are highly encouraged by the observation of dose dependent increases in persistence as well as the continued absence of any DLTs or clinically relevant adverse events for DSG3-CAART across the first three cohorts, particularly in the presence of circulating anti-DSG3 antibodies and without lymphodepletion," said David J. Chang, M.D., Chief Medical Officer of Cabaletta. "The rapid pace of the clinical trial has been possible due to the enthusiasm and engagement of patients, investigators and patient advocacy groups. With a 100% manufacturing success rate to date, we look forward to continuing to advance the trial until we identify a maximum tolerated dose and dosing regimen that has the potential to achieve a durable response while maintaining a favorable tolerability profile for patients suffering with mPV."

    As of October 31, 2021, three additional clinical sites have opened for recruitment, doubling the total number of activated DesCAARTes™ trial sites to six. Dosing of patients in the fourth cohort at a treatment dose of 2.5 billion DSG3-CAART cells has been initiated. The Company anticipates announcing 28-day safety data for the fourth dose cohort in the first quarter of 2022. Top-line biologic activity data for the first two low dose cohorts are anticipated to be announced in the fourth quarter of 2021.

    About the DesCAARTes™ Clinical Trial

    Cabaletta's DesCAARTes™ Phase 1 trial is an open-label, multi-center study of DSG3-CAART in adults with mucosal-dominant pemphigus vulgaris (mPV). The trial is designed to evaluate the safety and tolerability of DSG3-CAART as well as to identify evidence of target engagement and early signs of efficacy. The study consists of three parts: 1) dose escalation to determine the maximum tolerated dose, 2) dose consolidation, and 3) expansion at the final selected dose and schedule. The trial is expected to enroll approximately 30 patients across multiple clinical sites throughout the United States. Visit our website (DesCAARTes™ Phase 1 Trial) for more information.

    About Pemphigus Vulgaris

    mPV is a rare autoimmune blistering disease that is characterized by the loss of adhesion between cells of the skin or mucous membranes. mPV is caused by the production of autoantibodies that disrupt structural proteins within the skin and/or mucosa that connect with other proteins to enable the skin and/or mucosal cells to connect with each other. The autoantibodies can target DSG3 and/or desmoglein 1 (DSG1), which are primarily expressed in the mucosal membranes and skin, respectively. mPV is characterized by autoantibodies against DSG3 only whereas mucocutaneous PV (mcPV) is characterized by autoantibodies against DSG3 and DSG1.

    About CAAR T Cell Therapy

    Chimeric AutoAntibody Receptor (CAAR) T cells are designed to selectively bind and eliminate only disease-causing B cells, while sparing the normal B cells that are essential for human health. CAAR T cells are based on the chimeric antigen receptor (CAR) T cell technology. While CAR T cells typically contain a CD19-targeting molecule, CAAR T cells express an autoantibody-targeted antigen on their surface. The co-stimulatory domain and the signaling domain of both a CAR T cell and a CAAR T cell carry out the same activation and cytotoxic functions. Thus, Cabaletta's CAARs are designed to direct the patient's T cells to kill only the pathogenic cells that express disease-causing autoantibodies on their surface, potentially leading to complete and durable remission of disease while sparing all other B cell populations that provide beneficial immunity from infection.



    About Cabaletta Bio

    Cabaletta Bio is a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies, and exploring their potential to provide a deep and durable, perhaps curative, treatment, for patients with B cell-mediated autoimmune diseases. The Cabaletta Approach to selective B cell Ablation (CABA™) platform, in combination with Cabaletta's proprietary technology, utilizes CAAR T cells that are designed to selectively bind and eliminate only specific autoantibody-producing B cells while sparing normal antibody-producing B cells, which are essential for human health. The Company's lead product candidate, DSG3-CAART, is being evaluated in the DesCAARTes™ phase 1 clinical trial as a potential treatment for patients with mucosal pemphigus vulgaris, a prototypical B cell-mediated autoimmune disease. The FDA granted Fast Track Designation for DSG3-CAART in May 2020. For more information about the DesCAARTes™ Phase 1 clinical trial, please visit our website (DesCAARTes™ Phase 1 Trial). The Company's lead preclinical product candidate, MuSK-CAART, is in IND-enabling studies and is designed as a potential treatment for patients with MuSK-associated myasthenia gravis. For more information, visit www.cabalettabio.com.

    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 the progress and results of its DesCAARTes™ Phase 1 trial, including: Cabaletta's ability to enroll the requisite number of patients and dose each dosing cohort in the intended manner and time; the expected timing and significance around the announcement of 28-day safety for the fourth dose cohort in the first quarter of 2022 and top-line data on biologic activity for the first two low dose cohorts in the fourth quarter of 2021; the expectation that Cabaletta may improve outcomes for patients suffering from mPV; the effectiveness and timing of product candidates that Cabaletta may develop, including in collaboration with academic partners; the safety, efficacy and tolerability of DSG3-CAART for the treatment of mPV; the significance of data Cabaletta may announce regarding certain efficacy outcomes assessed in the DesCAARTes™ trial; the impact of preclinical data on the future development of CAAR T therapies in our pipeline portfolio; the impact of COVID-19 on the timing, progress, interpretability of data, and results of ongoing or planned clinical trials, including the DesCAARTes™ Phase 1 trial; and statements regarding regulatory filings regarding 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 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 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 the treatment of pemphigus vulgaris or for improving healing of mucosal blisters in patients with mucosal pemphigus vulgaris, respectively; 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; 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.

    Contacts:

    Anup Marda

    Chief Financial Officer

    Sarah McCabe

    Stern Investor Relations, Inc.



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  5. – Chimeric AutoAntibody Receptor (CAAR) T cells specifically recognized and eliminated anti-PLA2R antibody-expressing B cells in vitro –

    – Membrane proteome arrays screened with PLA2R CAAR candidates did not identify off-target interactions –

    – Pre-IND interaction with the Food and Drug Administration (FDA) is planned for later this year for PLA2R-CAART preclinical candidate –

    PHILADELPHIA, Oct. 15, 2021 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (NASDAQ:CABA), a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies for patients with B cell-mediated autoimmune diseases, today announced that data from in vitro studies supporting the early preclinical validation of PLA2R-Chimeric AutoAntibody…

    – Chimeric AutoAntibody Receptor (CAAR) T cells specifically recognized and eliminated anti-PLA2R antibody-expressing B cells in vitro –

    – Membrane proteome arrays screened with PLA2R CAAR candidates did not identify off-target interactions –

    – Pre-IND interaction with the Food and Drug Administration (FDA) is planned for later this year for PLA2R-CAART preclinical candidate –

    PHILADELPHIA, Oct. 15, 2021 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (NASDAQ:CABA), a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies for patients with B cell-mediated autoimmune diseases, today announced that data from in vitro studies supporting the early preclinical validation of PLA2R-Chimeric AutoAntibody Receptor T (CAART) cell candidates will be presented at ASN Kidney Week 2021. The data will be presented as an oral abstract by Aimee Payne, M.D., Ph.D., Professor of Dermatology at the University of Pennsylvania's Perelman School of Medicine and co-chair of the Scientific Advisory Board and co-founder at Cabaletta Bio at the American Society of Nephrology (ASN) Kidney Week 2021 being held virtually from November 4-7, 2021.

    "Current therapeutic strategies for PLA2R membranous nephropathy patients include generalized immune suppression, including B cell depletion with rituximab, which often requires repeat treatments and may cause serious infections in certain patients. Building on the platform for CAART product candidates that are more advanced in development, including DSG3-CAART and MuSK-CAART, we aim to develop a highly specific therapy that provides safety, efficacy, and durability for patients suffering from this autoimmune disease," said Dr. Payne "This initial preclinical data is promising, as it demonstrates the in vitro function of PLA2R-CAART cells against autoantibody-binding epitopes relevant to pathogenic B cells from patients with primary membranous nephropathy, with no evidence of off-target interactions."

    For this study, the Payne Laboratory generated multiple CAARs comprised of the PLA2R epitopes targeted by autoantibodies in PLA2R membranous nephropathy patients, as an extracellular decoy on the surface of T cells. These CAARs were observed to direct specific cytolysis of B cells expressing each of the anti-PLA2R autoantibodies tested. The data suggest that PLA2R-CAAR T cell therapy has the potential to target pathogenic B cells expressing autoantibodies against PLA2R while sparing healthy B cells, which could provide a novel precision therapeutic approach for patients with PLA2R membranous nephropathy. Membrane proteome arrays screened against leading PLA2R CAAR candidates did not identify any off-target interactions.

    "These discovery and early preclinical studies conducted by our co-founder Dr. Aimee Payne and colleagues at the University of Pennsylvania are a meaningful step for Cabaletta Bio, as they further support the breadth of the CABA™ platform for patients with B cell-mediated autoimmune diseases and high unmet clinical need," said Gwendolyn Binder, Ph.D., EVP Science and Technology at Cabaletta Bio. "These data provide the scientific rationale to commit our lead PLA2R-CAART candidate to preclinical development for patients with PLA2R membranous nephropathy."

    Oral Abstract Presentation Details
    TitleChimeric autoantibody receptor (CAAR) T cells as a precision therapy for antigen-specific B cell depletion in PLA2R membranous nephropathy
    Abstract NumberFR-OR32
    SessionGlomerular Diseases: Antibodies, Complement, and Inflammatory Mediators
    Session Date/TimeFri, Nov 5, 4:30 PM - 6:00 PM PDT
    FormatPre-recorded, virtual with live Q&A after presentation

    PLA2R-CAART is one of seven CAAR T programs that have emerged from the Cabaletta Approach to selective B cell Ablation (CABA™) platform. Cabaletta's lead product candidate, DSG3-CAART, is currently being evaluated in the DesCAARTes™ Phase 1 clinical trial as a potential treatment for patients with mucosal pemphigus vulgaris (mPV). The lead preclinical product candidate, MuSK-CAART, is designed as a potential treatment for patients with MuSK-associated myasthenia gravis, with an IND submission planned by the end of 2021. Cabaletta has four additional discovery programs derived from the CABA™ platform.

    About Membranous Nephropathy

    Primary membranous nephropathy (MN) is a B cell-mediated autoimmune disease that affects the kidneys. Approximately 70-80% of the 15,000 primary MN patients in the U.S. have autoantibodies directed to the phospholipase A2 receptor (PLA2R) on kidney podocytes. Since the discovery of these anti-PLA2R autoantibodies in 2009, evidence has shown that these autoantibodies accumulate as immune deposits in the glomeruli of the kidney and damage the filtration barrier, leading to nephrotic syndrome as characterized by proteinuria as well as risk for progression to kidney failure and dialysis and potential need for transplant. Immunosuppressive therapies are commonly used, but high unmet need remains as a significant fraction of patients either relapse or fail to respond or experience risk of serious infection following treatment with immunosuppressive agents.

    About CAAR T Cell Therapy

    Chimeric AutoAntibody Receptor (CAAR) T cells are designed to selectively bind and eliminate only disease-causing B cells, while sparing the normal B cells that are essential for human health. CAAR T cells are based on the chimeric antigen receptor (CAR) T cell technology. While CAR T cells typically contain a CD19-targeting molecule, CAAR T cells express an autoantibody-targeted antigen on their surface. The co-stimulatory domain and the signaling domain of both a CAR T cell and a CAAR T cell carry out the same activation and cytotoxic functions. Thus, Cabaletta's CAARs are designed to direct the patient's T cells to kill only the pathogenic cells that express disease-causing autoantibodies on their surface, potentially leading to complete and durable remission of disease while sparing all other B cell populations that provide beneficial immunity from infection.



    About Cabaletta Bio

    Cabaletta Bio is a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies, and exploring their potential to provide a deep and durable, perhaps curative, treatment, for patients with B cell-mediated autoimmune diseases. The Cabaletta Approach to selective B cell Ablation (CABA™) platform, in combination with Cabaletta's proprietary technology, utilizes CAAR T cells that are designed to selectively bind and eliminate only specific autoantibody-producing B cells while sparing normal antibody-producing B cells, which are essential for human health. The Company's lead product candidate, DSG3-CAART, is being evaluated in the DesCAARTes™ phase 1 clinical trial as a potential treatment for patients with mucosal pemphigus vulgaris, a prototypical B cell-mediated autoimmune disease. The FDA granted Fast Track Designation for DSG3-CAART in May 2020. For more information about the DesCAARTes™ Phase 1 clinical trial, please visit our website (DesCAARTes™ Phase 1 Trial). The Company's lead preclinical product candidate, MuSK-CAART, is in IND-enabling studies and is designed as a potential treatment for patients with MuSK-associated myasthenia gravis. For more information, visit www.cabalettabio.com.

    University of Pennsylvania Financial Disclosure

    Dr. Payne is a Penn faculty member, scientific collaborator, key advisor, and co-founder of Cabaletta Bio. As such, she holds an equity stake in the Company, her laboratory at Penn receives sponsored research funding from Cabaletta Bio, and as an inventor of the licensed technology she may receive additional future financial benefits under licenses granted by Penn to Cabaletta Bio. The University of Pennsylvania may also receive future financial benefit under licenses it has granted to Cabaletta Bio.

    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 the progress and results of its DesCAARTes™ Phase 1 trial, including Cabaletta Bio's ability to enroll the requisite number of patients; the expectations regarding the preliminary results from in vitro data in anti-PLA2R antibody-expressing B cells; the timing of Cabaletta's planned initiation of investigational new drug (IND) enabling studies for MuSK-CAART; the effectiveness and timing of additional product candidates and discovery programs that Cabaletta may develop from the CABA™ platform, including in collaboration with academic partners; the safety, efficacy and tolerability of DSG3-CAART for the treatment of mPV, MuSK-CAART for the treatment of MuSK-associated myasthenia gravis, and PLA2R-CAART for the treatment of PLA2R membranous nephropathy; the impact of preclinical data on the future development of CAAR T therapies in Cabaletta's pipeline portfolio; expectations of the potential impact of COVID-19 on strategy, future operations, and the timing of Cabaletta's clinical trials, including the potential impacts on its DesCAARTes™ Phase 1 trial; and statements regarding regulatory filings regarding 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: Cabaletta Bio's ability to demonstrate sufficient evidence of safety, efficacy and tolerability in its preclinical and clinical trials of DSG3-CAART, MuSK-CAART and PLA2R-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 our planned regulatory submissions and developments; risks related to the impact of public health epidemics affecting countries or regions in which we have operations or do business, such as COVID-19; 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; and the risk that the 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.

    Contacts:

    Anup Marda

    Chief Financial Officer

    Sarah McCabe

    Stern Investor Relations, Inc.



    Primary Logo

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