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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d)
of The Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): July 21, 2023
CABALETTA BIO, INC.
(Exact name of Registrant as Specified in its Charter)
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| Delaware |
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001-39103 |
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82-1685768 |
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(Commission File Number) |
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(I.R.S. Employer Identification No.) |
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| 2929 Arch Street, Suite 600, Philadelphia, PA |
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19104 |
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(Zip Code) |
(267) 759-3100
(Registrant’s telephone number, including area code)
Not Applicable
(Former name or former address, if changed since last report)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
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Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
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Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
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Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) |
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Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
Securities registered pursuant to Section 12(b) of the Act:
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| Title of Each Class |
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Trading
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Name of Each Exchange on Which Registered |
| Common Stock, par value $0.00001 per share |
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CABA |
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The Nasdaq Global Select Market |
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
Emerging growth company ☒
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
| Item 5.02. |
Departure of Directors or Certain Officers; Election of Directors; Appointment of Certain Officers; Compensatory Arrangements of Certain Officers. |
On July 21, 2023, upon the recommendation of its Nominating and Corporate Governance Committee (the “NCG Committee”), the Board of Directors (the “Board”) of Cabaletta Bio, Inc. (the “Company”) appointed Shawn Tomasello to join the Board, effective as of July 21, 2023 (the “Effective Date”), to fill the newly created vacancy on the Board resulting from an increase in the size of the Board from five (5) to six (6) directors. Ms. Tomasello will serve as a Class I director until her term expires at the 2026 annual meeting of stockholders of the Company at which time she will stand for election by the Company’s stockholders. The Board determined that Ms. Tomasello is independent under the applicable listing standards of The Nasdaq Stock Market (“Nasdaq”).
On the Effective Date, Ms. Tomasello was also appointed to the Compensation Committee of the Board (the “Compensation Committee”). The Board has determined that Ms. Tomasello meets the requirements for independence of compensation committee members under the applicable listing standards of Nasdaq and the Securities Exchange Act of 1934, as amended (the “Exchange Act”). In addition, effective as of the Effective Date: (i) Catherine Bollard, M.D. resigned as a member and Chair of the Compensation Committee, (ii) Mark Simon was appointed as the new Chair of the Compensation Committee and also resigned as Chair of the NCG Committee, and (iii) Scott Brun, M.D. was appointed as the new Chair of the NCG Committee.
In addition, on the Effective Date, the Board approved the formation of the Science and Technology Committee of the Board (the “S&T Committee”) to assist with the Board’s oversight of the Company’s research and development, manufacturing and technical operations and to advise the Board with respect to the Company’s scientific, pre-clinical and clinical activities. The newly created S&T Committee is composed of Dr. Bollard, Dr. Brun and Ms. Tomasello, with Dr. Bollard serving as the Chair of the S&T Committee.
As of the Effective Date, the Board’s committee composition is as follows:
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Audit Committee: Richard Henriques (Chair), Mark Simon and Scott Brun, M.D. |
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Compensation Committee: Mark Simon (Chair), Shawn Tomasello and Richard Henriques. |
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NCG Committee: Scott Brun, M.D. (Chair), Catherine Bollard, M.D. and Mark Simon. |
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S&T Committee: Catherine Bollard, M.D. (Chair), Scott Brun, M.D. and Shawn Tomasello. |
In connection with the formation of the S&T Committee, the Board approved the Company’s Second Amended and Restated Non-Employee Director Compensation Policy (the “Second A&R Director Compensation Policy”), effective as of the Effective Date, in order to establish compensation for the S&T Committee. Under the Second A&R Director Compensation Policy, the Company will pay a cash retainer of $7,500 per year to the members of the S&T Committee and a cash retainer of $15,000 to the Chair of the S&T Committee. No further changes were made to the terms of the Company’s existing amended and restated non-employee director compensation policy. The foregoing description of the terms of the Second A&R Director Compensation Policy does not purport to be complete and is qualified in its entirety by reference to the full text of the Second A&R Director Compensation Policy which will be filed with the Company’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2023.
As a non-employee director, Ms. Tomasello will receive cash compensation for her Board and committee service in accordance with the Second A&R Director Compensation Policy. In addition, under the Second A&R Compensation Policy, upon her election as a director on the Effective Date, Ms. Tomasello was granted an option to purchase 44,000 shares of the Company’s common stock at an exercise price per share of $13.48. This option shall vest in substantially equal quarterly installments over three years from the Effective Date, provided, however, that all vesting shall cease if the director ceases to have a service relationship, unless the Board determines that the circumstances warrant continuation of vesting. Ms. Tomasello is not a party to any transaction with the Company that would require disclosure under Item 404(a) of Regulation S-K, and there are no arrangements or understandings between Ms. Tomasello and any other persons pursuant to which she was selected as a director. In addition, Ms. Tomasello entered into an indemnification agreement with the Company consistent with the form of indemnification agreement entered into between the Company and its existing non-employee directors, a copy of which was filed as Exhibit 10.5 to the Company’s Registration Statement on Form S-1 (File No. 333-234017) filed with the Securities and Exchange Commission on September 30, 2019. Pursuant to the terms of this agreement, the Company may be required, among other things, to indemnify Ms. Tomasello for some expenses, including attorneys’ fees, judgments, fines and settlement amounts respectively incurred by her in any action or proceeding arising out of her respective service as one of the Company’s directors.
| Item 7.01. |
Regulation FD Disclosure. |
On July 24, 2023, the Company issued a press release announcing Ms. Tomasello’s appointment to the Board. A copy of this press release is furnished as Exhibit 99.1 to this report on Form 8-K.
On July 24, 2023, the Company also posted to the “Investors & Media” section of the Company’s website at www.cabalettabio.com an updated corporate presentation (the “Corporate Presentation”) to disclose Ms. Tomasello’s position on the Board and provide updated data from its study of desmoglein 3 chimeric autoantibody receptor T (“DSG3-CAART”) cells as a potential treatment for patients with mucosal pemphigus. A copy of the Corporate Presentation is furnished hereto as Exhibit 99.2 to this report on Form 8-K.
The information in this Item 7.01, including Exhibits 99.1 and 99.2 attached hereto, is intended to be furnished and shall not be deemed “filed” for purposes of Section 18 of the Exchange Act, or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing.
On July 24, 2023, the Company issued the Corporate Presentation reiterating guidance that it anticipates reporting 3-month clinical data on efficacy endpoints and tolerability for patients dosed with CABA-201 by the first half of 2024.
With respect to the CAART strategy, the Company included an update indicating the completion of the 1-month safety and persistence evaluation from the DesCAARTes™ trial for DSG3-CAART for the cohort in the combination sub-study where patients are pre-treated with intravenous immunoglobulin (“IVIg”) and cyclophosphamide (without fludarabine) prior to DSG3-CAART infusion. DSG3-CAART peak persistence and persistence over the initial 29 days post-infusion in the three mucosal pemphigus vulgaris subjects dosed was modestly increased by the cyclophosphamide only combination therapy. The Company announced the initiation of enrollment in an additional cohort in the combination sub-study with DSG3-CAART where patients are pretreated with IVIg, cyclophosphamide and fludarabine prior to DSG3-CAART infusion. The cohort is designed to evaluate the ability to improve DSG3-CAART engraftment and persistence.
| Item 9.01. |
Financial Statements and Exhibits. |
(d) Exhibits
SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its behalf by the undersigned, hereunto duly authorized.
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CABALETTA BIO, INC. |
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| Date: July 24, 2023 |
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By: |
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/s/ Steven Nichtberger |
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Steven Nichtberger, M.D. |
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President and Chief Executive Officer |
Exhibit 99.1
Cabaletta Bio Appoints Global Commercial Leader Shawn Tomasello to Board of
Directors
– Ms. Tomasello created and led global commercial and medical affairs functions at Kite Pharma from pre-launch through its acquisition by Gilead Sciences –
PHILADELPHIA, July 24, 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 announced the appointment of Shawn
Tomasello to its Board of Directors. Ms. Tomasello has over 35 years of experience in the life sciences industry, including specific expertise in CD19-CAR T therapy, where she most recently served as the
Chief Commercial Officer of Kite Pharma, Inc. between 2015 and 2018, leading the worldwide commercialization effort for the CD19-CAR T cell therapy,
Yescarta®, and playing a key role in its acquisition by Gilead Sciences, Inc. As part of her appointment to the Board of Directors, Ms. Tomasello will become a member of the Compensation
Committee and the newly formed Science & Technology Committee.
“Shawn is a recognized biopharmaceutical leader with a proven track record
building large-scale commercial organizations to bring transformative therapies to patients in need, including having overseen the global commercial launch of the leading approved CD19-CAR T cell
therapy,” said Steven Nichtberger, M.D., Chief Executive Officer and Co-founder of Cabaletta. “Shawn’s experience in pre-launch planning, scaling and
commercializing a CD19-CAR T therapy globally will provide important additional perspective to our Board of Directors as we continue to expand and advance our CABA-201
development program.”
Ms. Tomasello brings over three decades of experience in the life sciences industry and most recently served as the Chief
Commercial Officer of Kite Pharma, now part of Gilead Sciences, where she oversaw the global commercialization of Yescarta®, the first approved CAR-T
therapy for non-Hodgkin lymphoma. Prior to joining Kite Pharma, she was the Chief Commercial Officer of Pharmacyclics LLC, now part of AbbVie Inc., where she led both commercial and medical affairs. Before
that, Ms. Tomasello held senior leadership positions at Celgene Corporation, including President of the Americas, Hematology and Oncology, where she led the company through five successful product launches encompassing 11 indications and played
a critical role in acquisitions. Previously, she was National Director of Hematology for Rituxan® at Genentech, Inc. Earlier in her career, Ms. Tomasello held positions at Pfizer
Laboratories, Miles Pharmaceuticals, Inc. and Proctor & Gamble Company. She holds an M.B.A. from Murray State University and a B.S. in Marketing from the University of Cincinnati.
“I am excited to join Cabaletta’s Board of Directors and to support the company’s vision to develop and potentially launch the first targeted
curative cell therapies for patients with autoimmune diseases,” said Shawn Tomasello. “I look forward to applying my decades of experience building and scaling global commercial organizations in the life sciences industry to bring CABA-201 closer to patients with autoimmune disease and support development of the broader CABA™ platform.”
About Cabaletta Bio
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 fully human
CD19-CAR T, as the lead product candidate being evaluated in systemic lupus erythematosus and myositis, 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 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 its expectations regarding:
Cabaletta Bio’s ability to grow its autoimmune-focused pipeline; its plans around CABA-201, including its expectations for the expansion and advancement of the
CABA-201 development program and potential launch of CABA-201; the company’s business plans and objectives, including on a global scale; the potential curative
effect of the therapies associated with the CABATM platform; and the anticipated contribution of the members of our board of directors, specifically Ms. Tomasello, and our executives to our
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: 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; 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 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 Marda
Chief Financial Officer
investors@cabalettabio.com
William Gramig
Stern Investor Relations, Inc.
william.gramig@sternir.com
Corporate Presentation JULY 2023
Exhibit 99.2
Disclaimer The following presentation,
including any printed or electronic copy of these slides, the talks given by the presenters, the information communicated during any delivery of the presentation and any question and answer session and any document or material distributed at or in
connection with the presentation (collectively, the “Presentation”) has been prepared by Cabaletta Bio, Inc. (“we,” “us,” “our,” “Cabaletta” or the “Company”) and is made for
informational purposes only. This Presentation does not purport to be a prospectus, to be complete or to contain all of the information you may desire. Statements contained herein are made as of the date of this Presentation unless stated otherwise,
and this Presentation shall not under any circumstances create an implication that the information contained herein is correct as of any time after such date or that information will be updated or revised to reflect information that subsequently
becomes available or changes occurring after the date hereof. This Presentation may contain “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business, operations, and
financial conditions, and include, but are not limited to, express or implied statements regarding our current beliefs, expectations and assumptions regarding: our business, future plans and strategies for our CAAR T and CARTA technologies and
CABA™ platform; our ability to grow our 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; our expectations around the potential success and therapeutic benefits of CABA-201, including our belief that CABA-201 may enable an “immune system reset” and provide deep and durable responses for patients with autoimmune
diseases; our plans for (i) a Phase 1/2 clinical trial of CABA-201 in patients with SLE, including our anticipated progress, clinical trial design, ability to leverage our experience in autoimmune cell therapy and lupus product development and (ii)
a Phase 1/2 clinical trial of CABA-201 in patients with myositis, including our anticipated progress, clinical trial design and ability to leverage our experience in autoimmune cell therapy; our planned initial clinical data read-out in the first
half of 2024 for patients treated with CABA-201; our ability to enroll the requisite number of patients, dose each dosing cohort in the intended manner, and advance the trial as planned in our Phase 1/2 clinical trials of CABA-201; the timing any
planned regulatory filings for our development programs; the progress and results of our DesCAARTes™ Phase 1 trial, including the significance and impact around reported safety and clinical and translational data of cohorts from our
DesCAARTes™ trial, and our ability to advance dose escalation and initiate combination cohorts and to optimize our targeted cell therapy; our ability to implement a pre-treatment regimen, the outcomes of such pre-treatment regimen and the
potential ability to enhance in vivo DSG3-CAART exposure; the therapeutic potential and clinical benefits of our product candidates; the expectation that Cabaletta may improve outcomes for patients suffering from systemic lupus erythematosus,
myositis, mucosal pemphigus vulgaris, myasthenia gravis, or other autoimmune diseases; our ability to escalate dosing as high as 15 billion cells in cohort A6m, initiate dosing in a combination cohort or otherwise; our ability to evaluate, and the
potential significance of, the relationship between DSG3-CAART persistence and potential clinical responses in patients with mPV; our ability to safely retreat additional patients and whether we will continue to observe a lack of immune-mediated
clearance of DSG3-CAART cells after retreatment and repeat dosing of patients; our ability to successfully complete our preclinical and clinical studies for our product candidates, including our ability to enroll the requisite number of patients,
dose each dosing cohort in the intended manner, and progress the trial; the ability of MuSK-CAART to target B cells that differentiate into antibody secreting cells, which produce autoantibodies against muscle-specific kinase; our ability to obtain
and maintain regulatory approval of our product candidates, including our expectations regarding the intended incentives conferred by and ability to retain Orphan Drug Designation and Fast Track Designation for our product candidates, as applicable;
the further expansion and development of our modular CABA™ platform across a range of autoimmune diseases; our ability to contract with third-party suppliers and manufacturers, implement an enhanced manufacturing process and further develop
our internal manufacturing strategy, capabilities and facilities; our potential commercial opportunities, including value and addressable market, for our product candidates; our expectations regarding our use of capital and other financial results;
and our ability to fund operations into the fourth quarter of 2025. Words such as, but not limited to, “look forward to,” “believe,” “expect,” “anticipate,” “estimate,”
“intend,” “plan,” “would,” “should” and “could,” and similar expressions or words, identify forward-looking statements. Various risks, uncertainties and assumptions could cause actual
results to differ materially from those anticipated or implied in our forward-looking statements. Such risks and uncertainties include, but are not limited to, risks related to the success, cost, and timing of our product candidate development
activities and preclinical studies and clinical trials, risks related to our ability to demonstrate sufficient evidence of safety, efficacy and tolerability in our preclinical studies and clinical trials of CABA-201, DSG3-CAART and MuSK-CAART, the
risk that the results observed with the similarly-designed construct, including, but not limited to, due to dosing regimen, are not indicative of the results we seek to achieve with CABA-201, our plans to evaluate additional cohorts in the
DesCAARTes™ trial, including a cohort implementing a pre-treatment regimen, the risk that signs of biologic activity or persistence may not inform long-term results, the risk that persistence observed with effective CART-19 oncology studies in
combination with lymphodepletion is not indicative of, or applicable to, clinical responses in patients with mPV, risks related to clinical trial site activation or enrollment rates that are lower than expected, our ability to protect and maintain
our intellectual property position, risks related to our relationship with third parties, uncertainties related to regulatory agencies’ evaluation of regulatory filings and other information related to our product candidates, our ability to
retain and recognize the intended incentives conferred by any Orphan Drug Designation and Fast Track Designations, the risk that any one or more of our product candidates will not be successfully developed and commercialized, the risk that the
results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies, the impact of COVID-19 on the timing, progress, interpretability of data, and results of ongoing or planned clinical
trials and risks relating to as a result of extraordinary events or circumstances such as the COVID-19 pandemic, and any business interruptions to our operations or to those of our clinical sites, manufacturers, suppliers, or other vendors resulting
from the COVID-19 pandemic or similar public health crisis. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Except as required by applicable law, we do not plan to publicly
update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Although we believe the expectations reflected in such forward-looking statements are
reasonable, we can give no assurance that such expectations will prove to be correct. Accordingly, you are cautioned not to place undue reliance on these forward-looking statements. No representations or warranties (expressed or implied) are made
about the accuracy of any such forward-looking statements. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause our actual results to differ materially from those contained in the
forward-looking statements, see the section entitled "Risk Factors" in our most recent annual report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in our other and subsequent filings with the
Securities and Exchange Commission. Certain information contained in this Presentation relates to or is based on studies, publications, surveys and other data obtained from third-party sources and the Company’s own internal estimates and
research. While the Company believes these third-party sources to be reliable as of the date of this Presentation, it has not independently verified, and makes no representation as to the adequacy, fairness, accuracy or completeness of, any
information obtained from third-party sources. The Company is the owner of various trademarks, trade names and service marks. Certain other trademarks, trade names and service marks appearing in this Presentation are the property of third parties.
Solely for convenience, the trademarks and trade names in this Presentation are referred to without the ® and TM symbols, but such references should not be construed as any indicator that their respective owners will not assert, to the fullest
extent under applicable law, their rights thereto.
Develop and launch the first curative
targeted cellular therapies for patients with autoimmune diseases
Leveraging years of experience with
cellular therapy in autoimmunity to initiate CABA-201 clinical trials Cabaletta®: Pursuing cures for a broad range of autoimmune diseases CARTA – Chimeric Antigen Receptor T cells for Autoimmunity; CAART – Chimeric AutoAntibody
Receptor T cells; IND – Investigational New Drug; SLE – Systemic lupus erythematosus; DM – Dermatomyositis; ASyS – Anti-synthetase syndrome; IMNM – Immune-mediated necrotizing myopathy Mackensen, Andreas, et al.
"Anti-CD19 CAR T cell therapy for refractory systemic lupus erythematosus." Nature Medicine (2022): 1-9. Müller, Fabian, et al. "CD19-targeted CAR T cells in refractory antisynthetase syndrome." The Lancet (2023). Dai, Zhenyu, et al.
"Development and functional characterization of novel fully human anti‐CD19 CARs for T‐cell therapy." Journal of Cellular Physiology 236.8 (2021): 5832-5847. Assumes no dose-limiting toxicities are observed in the cohort and
uninterrupted enrollment occur in the trials. Advancing SLE & myositis trials with efficient designs – including starting dose & parallel cohorts 1.0 x 106 cells/kg initial dose of CABA-201 is identical to dose used in academic SLE1
and myositis2 studies Parallel cohorts with 6 patients each – SLE study with 1) LN & 2) Non-renal SLE; myositis study with 1) DM, 2) ASyS & 3) IMNM CABA-201 has been specifically engineered for patients with autoimmune diseases Fully
human CD19 binder with data in ~20 oncology patients – clinical safety profile supporting further evaluation in autoimmunity Same 4-1BB costimulatory domain and similar CD19 binder affinity3 as used in the academic SLE1 & myositis studies2
Potential to cure a broad range of autoimmune diseases where B cells have a role initiating or maintaining disease DesCAARTes™ trial of DSG3-CAART in mucosal pemphigus vulgaris Enrolling in combination sub-study cohort using pre-treatment with
IVIg, cyclophosphamide and fludarabine MusCAARTes™ trial in MuSK myasthenia gravis – leveraging insights from experience with DSG3-CAART Initiated in 4Q22; received FDA Fast Track Designation & Orphan Drug Designation CARTA Strategy
| CABA-201 (4-1BB CD19-CAR T) to be evaluated in SLE & myositis Phase 1/2 studies CAART Strategy | DSG3-CAART & MuSK-CAART clinical studies evaluating combination regimens Initial CABA-201 3-month clinical efficacy and tolerability data
expected by 1H244
One CABA™ platform, two
strategies to address autoimmune diseases Complementary strategies to optimize clinical outcomes using cellular therapies in autoimmune diseases Mackensen, Andreas, et al. "Anti-CD19 CAR T cell therapy for refractory systemic lupus erythematosus."
Nature Medicine (2022): 1-9. Müller, Fabian, et al. "CD19-targeted CAR T cells in refractory antisynthetase syndrome." The Lancet (2023). Ellebrecht, Christoph T., et al. "Reengineering chimeric antigen receptor T cells for targeted therapy of
autoimmune disease." Science 353.6295 (2016): 179-184. Fully Human Anti-CD19 Targeting Domain CARTA Chimeric Antigen Receptor T cells for Autoimmunity CAART Chimeric AutoAntibody Receptor T cells CABA-201 4-1BB Costimulatory Domain &
CD3-Zeta Signaling Domain DSG3-CAART Potential to ‘reset the immune system’ in patients with autoimmune diseases driven by B cells, through generalized transient B cell depletion and repopulation of healthy B cells1,2 In autoimmune
diseases with a limited number of well-defined pathogenic autoantibodies, permanent antigen-specific B cell depletion may provide an elegant biologic solution to disease3 Autoantigen Targeting Domain 4-1BB Costimulatory Domain & CD3-Zeta
Signaling Domain
Biologic opportunity for cure or
treatment may be possible in dozens of autoimmune diseases* CABA™ platform may apply across a range of autoimmune diseases Pemphigus Vulgaris1,2,3 Pemphigus Foliaceus1,2,3 Epidermolysis Bullosa Acquisita3 Bullous Pemphigoid1,2,3 Lupus
Nephritis3,4 Membranous Nephropathy1,2,3 Goodpasture’s Syndrome1,2,3,4 Myasthenia Gravis1,2,3,5 Multiple Sclerosis6 Neuromyelitis Optica3 CIDP (Chronic Inflammatory Demyelinating Polyneuropathy)1.2 Anti-NMDAR Encephalitis3,6 Lambert-Eaton
Syndrome5 Systemic Lupus Erythematosus3,4,5,6 Rheumatoid Arthritis2,3,4 Myositis5 Systemic sclerosis6 ANCA-Associated Vasculitis3,4,5 Immune Thrombocytopenic Purpura3 Thrombotic Thrombocytopenic Purpura1,2,3 Antiphospholipid Syndrome4,5 Autoimmune
Hemolytic Anemia3 Type 1 Diabetes3,6 Graves’ Disease3,5 Hashimoto’s Disease5 Illustrative list of autoimmune diseases where B cells may play a role in initiating or maintaining disease, and where biologic opportunity for cure or
treatment with CAART or CARTA approach may be possible. Diseases in bold represent where clinical studies are underway or plan to be initiated by Cabaletta or by Professor Schett Koneczny, Inga. "Update on IgG4-mediated autoimmune diseases: new
insights and new family members." Autoimmunity Reviews (2020): 102646. Huijbers, Maartje G., et al. "IgG4-mediated autoimmune diseases: a niche of antibody-mediated disorders." Annals of the New York Academy of Sciences 1413.1 (2018): 92. Ludwig,
Ralf J., et al. "Mechanisms of autoantibody-induced pathology." Frontiers in immunology 8 (2017): 603. Suurmond, Jolien, and Betty Diamond. "Autoantibodies in systemic autoimmune diseases: specificity and pathogenicity." The Journal of clinical
investigation 125.6 (2015): 2194-2202. Xiao, Ze Xiu, Joseph S. Miller, and Song Guo Zheng. "An updated advance of autoantibodies in autoimmune diseases." Autoimmunity Reviews (2020): 102743. Hampe, Christiane S. "B cells in autoimmune diseases."
Scientifica 2012 (2012). Dermatology Nephrology Neurology Rheumatology Hematology Endocrinology
Pipeline targeting autoimmune diseases
where cure is possible CABA-201 is being evaluated in separate clinical trials for myositis and SLE. Additional CAART pipeline candidates include PLA2R-CAART in preclinical stage for PLA2R membranous nephropathy, DSG3/1-CAART in discovery stage for
mucocutaneous pemphigus vulgaris & 2 undisclosed targets in discovery stage. Currently being evaluated in a Phase 1 trial. CABA™ Platform Indication Program Discovery Preclinical Phase 1/2 Phase 2/3 CARTA Chimeric Antigen Receptor T cells
for Autoimmunity Myositis (IIM, Idiopathic Inflammatory Myopathy)1 Dermatomyositis CABA-201 4-1BB CD19-CAR T Anti-Synthetase Syndrome Immune-Mediated Necrotizing Myopathy CARTA Chimeric Antigen Receptor T cells for Autoimmunity Systemic Lupus
Erythematous (SLE)1 Lupus Nephritis CABA-201 4-1BB CD19-CAR T Non-Renal SLE Multiple Undisclosed Indications CAART2 Chimeric AutoAntibody Receptor T cells Mucosal Pemphigus Vulgaris DSG3-CAART3 MuSK Myasthenia Gravis MuSK-CAART3 IND cleared IND
cleared
Chimeric Antigen Receptor T Cells for
Autoimmunity CABA-201
Emerging clinical data with 4-1BB
CD19-CAR T7 in… SLE – Systemic lupus erythematosus; SLEDAI-2K – Systemic Lupus Erythematosus Disease Activity Index 2000 Mackensen, Andreas, et al. "Anti-CD19 CAR T cell therapy for refractory systemic lupus erythematosus." Nature
Medicine (2022): 1-9. Taubmann, J., et al. "OP0141 Long Term Safety and Efficacy Of CAR-T Cell Treatment in Refractory Systemic Lupus Erythematosus-Data from the First Seven Patients." (2023): 93-94. Müller, Fabian, et al. "CD19-targeted CAR T
cells in refractory antisynthetase syndrome." The Lancet (2023). Taubmann, Jule, et al. "Rescue therapy of antisynthetase syndrome with CD19-targeted CAR-T-cells after failure of several B cell depleting antibodies." Rheumatology (Oxford, England)
(2023): kead330. Pecher, Ann-Christin, et al. "CD19-Targeting CAR T Cells for Myositis and Interstitial Lung Disease Associated With Antisynthetase Syndrome." JAMA 329.24 (2023): 2154-2162. Bergmann, Christina, et al. "AB0816 Treatment of a Patient
with Severe Diffuse Systemic Sclerosis (Ssc) Using CD19-targeting CAR-T-cells." (2023): 1621-1621. The construct utilized in these studies has a similar design to CABA-201, sharing the 4-1BB costimulatory domain, but is a different construct. 9
Exclusive translational research partnership with key investigator informs Cabaletta’s CD19 clinical strategy Academic data: Immune system reset in autoimmune patients1-6 Robust improvement in clinical disease activity within 3 months of
CD19-CAR T treatment Rapid and deep CD19+ B cell depletion Return of healthy B cells within 7 months Favorable safety data with CD19-CAR T regimen Clinical SLE responses up to 24 mo with no relapses reported2 7 Systemic lupus erythematosus
patients1,2 3 Anti-synthetase syndrome patients3-5 1 Systemic sclerosis patient6 SLE (N=5)1 SLEDAI-2K Myositis (N=1)2 Total improvement score
Cabaletta’s CD19 binder with
similar in vitro & in vivo activity to FMC631,2 (binder used in academic studies3,4) CABA-201: CD19-targeting CAR T therapy for autoimmune diseases SLE – Systemic lupus erythematosus; IIT – Investigator-initiated trial; Flu/Cy
– Fludarabine/Cyclophosphamide; CRS – Cytokine release syndrome; ICANS – Immune effector cell-associated neurotoxicity syndrome Peng, Binghao J, et al. “Preclinical specificity and activity of CABA-201, a fully human 4-1BB
containing CD19 CAR T therapy for treatment-resistant autoimmune disease.” Poster presented at: American Society Gene and Cell Therapy 26th Annual Meeting; 2023 May 19; Los Angeles, CA. Dai, Zhenyu, et al. "Development and functional
characterization of novel fully human anti‐CD19 chimeric antigen receptors for T‐cell therapy." Journal of Cellular Physiology 236.8 (2021): 5832-5847. Mackensen, Andreas, et al. "Anti-CD19 CAR T cell therapy for refractory systemic
lupus erythematosus." Nature Medicine (2022): 1-9. Müller, Fabian, et al. "CD19-targeted CAR T cells in refractory antisynthetase syndrome." The Lancet (2023). Evaluated as part of CT120, a dual-CD19xCD22 CAR T product candidate under
development by Nanjing IASO Biotherapeutics, Co., Ltd. (IASO Bio). Fully human anti-CD19 binder 4-1BB costimulatory domain CD3-Zeta signaling domain CABA-201 Similar in vitro & in vivo biologic activity to FMC631 Same co-stimulatory domain as
used in academic studies CD19-CAR Construct Clinical data reported by IASO using licensed CD19 binder in oncology5 Fully human binder Evaluated as dual-CAR combined with CD22 binder with standard Flu/Cy preconditioning Data reported in ~20 patients
to date B cell leukemia and lymphoma in IIT in China Safety data supports autoimmune development CABA-201 In vitro In vivo CABA-201 CD19 binder used in academic studies3,4
~66K U.S. patients with IIM
subtypes with B cell involvement; frequently severe, with limited treatment options Myositis: Strong scientific rationale & significant disease burden Lundberg, Ingrid E., et al. "Idiopathic inflammatory myopathies." Nature Reviews Disease
Primers 7.1 (2021): 86. Kronzer, Vanessa L., et al. "Incidence, Prevalence, and Mortality of Dermatomyositis: A Population‐Based Cohort Study." Arthritis Care & Research 75.2 (2023): 348-355. Badshah, Aliena, et al. "Antisynthetase
syndrome presenting as interstitial lung disease: a case report." Journal of Medical Case Reports 13.1 (2019): 1-6. Coffey, Caitryn, et al. “Incidence of Antisynthetase Syndrome and Risk of Malignancy in a Population-based Cohort
(1998-2019)” [abstract]. Arthritis Rheumatol. 2021; 73 (suppl 9). Shelly, Shahar, et al. "Incidence and prevalence of immune‐mediated necrotizing myopathy in adults in Olmsted County, Minnesota." Muscle & Nerve 65.5 (2022): 541-546.
~6K U.S. pts5 Immune-mediated necrotizing myopathy (IMNM) ~17K U.S. pts3,4 Anti-synthetase syndrome (ASyS) ~43K U.S. pts2 Dermatomyositis (DM) Key myositis subtypes based on underlying immune mechanisms & clinical characteristics1 Potential
manifestations & subtype prevalence 1 Autoimmune disease with B cell involvement 2 High burden on function & quality of life1 Idiopathic inflammatory myopathies (IIMs, or myositis) are a group of autoimmune diseases characterized by
inflammation and muscle weakness Typically, mid-adult onset & more common in females Symptoms may include weakness, fatigue, pain, shortness of breath and difficulty swallowing Mainstay of therapy is glucocorticoids with a steroid-sparing agent
(i.e. methotrexate, azathioprine, rituximab), and increasing use of IVIg (intravenous immunoglobulin) Many patients have disease that remains refractory Therapies carry potential long-term side effects High mortality rate due to interstitial lung
disease (ILD), cardiovascular disease and/or malignancy CABA-201
CABA-201 to be evaluated in
patients with active myositis, including DM, ASyS & IMNM Phase 1/2 study design for CABA-201 in patients with myositis DM – Dermatomyositis; ASyS – Anti-synthetase syndrome; IMNM – Immune-mediated necrotizing myopathy Subjects
will be treated with a standard preconditioning regimen consisting of fludarabine and cyclophosphamide prior to a single dose CABA-201. Mackensen, Andreas, et al. "Anti-CD19 CAR T cell therapy for refractory systemic lupus erythematosus." Nature
Medicine (2022): 1-9. Müller, Fabian, et al. "CD19-targeted CAR T cells in refractory antisynthetase syndrome." The Lancet (2023). Phase 1/2 Study of CABA-201 Open label trial to evaluate CABA-201 in subjects with active idiopathic inflammatory
myopathy (IIM, or myositis) CABA-201 Key Inclusion Criteria Age ≥ 18 to ≤ 65 years Evidence of active disease Disease activity despite prior or current treatment with standard of care treatments Key Exclusion Criteria Cancer-associated
myositis Significant lung or cardiac impairment Treatment with B cell depleting agent within ~6 months Treatment with biologic agent within ~3 months Parallel Cohorts SCREENING CABA-201 INFUSION MONITORING Next Patient Study Endpoint &
Objectives Primary objective: Safety & tolerability of CABA-201 in subjects with active myositis within 28 days of infusion Key secondary objectives include CABA-201 effects on: Myositis clinical disease activity; Myositis serology; and
Pharmacokinetics / pharmacodynamics Dose of 1x106 cells/kg1 Equivalent to dose of 4-1BB-containing CD19-CAR T used in recent academic studies2,3 ASyS (N = 6) IMNM (N = 6) DM (N = 6) Consistent track record of execution by team across multiple
clinical studies evaluating novel cell therapy candidates for patients with autoimmune diseases
Up to 320,000 SLE patients in the
U.S., ~40% with LN, who face increased risk of kidney failure & death SLE & Lupus Nephritis: High unmet clinical need Helmick CG, Felson DT, Lawrence RC, Gabriel S, Hirsch R, Kwoh CK, Liang MH, Kremers HM, Mayes MD, Merkel PA, Pillemer SR,
Reveille JD, Stone JH; National Arthritis Data Workgroup. Estimates of the prevalence of arthritis and other rheumatic conditions in the United States. Part I. Arthritis Rheum. 2008 Jan;58(1):15-25. Tian, J., Zhang, D., Yao, X., Huang, Y., & Lu,
Q. (2023). Global epidemiology of systemic lupus erythematosus: a comprehensive systematic analysis and modelling study. Annals of the Rheumatic Diseases, 82(3), 351-356. Hoover PJ, Costenbader KH. Insights into the epidemiology and management of
lupus nephritis from the US rheumatologist's perspective. Kidney Int. 2016 Sep;90(3):487-92. Lewis, M. J., & Jawad, A. S. (2017). The effect of ethnicity and genetic ancestry on the epidemiology, clinical features and outcome of systemic lupus
erythematosus. Rheumatology, 56(suppl_1), i67-i77. Hahn, B. H., Mcmahon, M. A., Wilkinson, A., Wallace, W. D., Daikh, D. I., Fitzgerald, J. D., ... & Grossman, J. M. (2012). American College of Rheumatology guidelines for screening, treatment,
and management of lupus nephritis. Arthritis care & research, 64(6), 797-808. CABA-201 Within 10 years of LN diagnosis: End-stage renal disease: 17%3 Mortality: 12%5 Current therapies include steroids, immunosuppressive agents and biologics3
Many patients progress and/or relapse Significant risk of adverse effects Require long-term administration SLE is a chronic autoimmune disease that affects ~160-320K1 patients in the U.S. & over 3 million people worldwide2 Potential for
life-threatening complications Disproportionately affects young women people of color3,4 Significant unmet need remains despite current therapies, which require chronic administration and carry significant treatment-related risks Lupus nephritis
(LN) is a serious complication of SLE, affecting ~40% of SLE patients3 Skin & mucosa Kidneys Heart Joints, muscles & bones Immune system abnormalities Blood Neurological Lungs Potential SLE Manifestations
CABA-201 to be evaluated in
patients with active SLE with or without renal involvement Phase 1/2 study design for CABA-201 in patients with SLE SLE – Systemic lupus erythematosus; EULAR – European League Against Rheumatism; ACR – American College of
Rheumatology Subjects will be treated with a standard preconditioning regimen consisting of fludarabine and cyclophosphamide prior to a single dose CABA-201. Mackensen, Andreas, et al. "Anti-CD19 CAR T cell therapy for refractory systemic lupus
erythematosus." Nature Medicine (2022): 1-9. Müller, Fabian, et al. "CD19-targeted CAR T cells in refractory antisynthetase syndrome." The Lancet (2023). Phase 1/2 Study of CABA-201 Open label trial to evaluate CABA-201 in subjects with active
SLE CABA-201 Key Inclusion Criteria Age ≥ 18 to ≤ 65 years Clinical SLE per the 2019 EULAR/ACR classification criteria Positive ANA titer or anti-dsDNA antibody Disease activity despite prior or current treatment with standard of care
Key Exclusion Criteria Treatment with B cell depleting agent within 6 months Treatment with biologic agent within 3 months Consistent track record of execution by team across multiple clinical studies evaluating novel cell therapy candidates for
patients with autoimmune diseases Fast Track Designation SLE with lupus nephritis (N = 6) SLE without renal involvement (N = 6) Parallel Cohorts SCREENING CABA-201 INFUSION MONITORING Next Patient Study Endpoint & Objectives Primary objective:
Safety & tolerability of CABA-201 in subjects with SLE with active LN or SLE without renal involvement within 28 days of infusion Key secondary objectives include CABA-201 effects on: SLE clinical disease activity, and SLE serology, as well as
Pharmacokinetics / pharmacodynamics Dose of 1x106 cells/kg1 Equivalent to dose of 4-1BB-containing CD19-CAR T used in recent academic studies2,3
Our product candidate, people &
partnership enable accelerated progress while integrating unique insights Accelerating development of CABA-201 for autoimmune diseases SLE – Systemic lupus erythematosus Peng, Binghao J, et al. “Preclinical specificity and activity of
CABA-201, a fully human 4-1BB containing CD19 CAR T therapy for treatment-resistant autoimmune disease.” Poster presented at: American Society Gene and Cell Therapy 26th Annual Meeting; 2023 May 19; Los Angeles, CA. Dai, Zhenyu, et al.
"Development and functional characterization of novel fully human anti‐CD19 chimeric antigen receptors for T‐cell therapy." Journal of Cellular Physiology 236.8 (2021): 5832-5847. Mackensen, Andreas, et al. "Anti-CD19 CAR T cell therapy
for refractory systemic lupus erythematosus." Nature Medicine (2022): 1-9. Müller, Fabian, et al. "CD19-targeted CAR T cells in refractory antisynthetase syndrome." The Lancet (2023). Partnership – Exclusive translational research
partnership has provided early, actionable insights Scientific and clinical data sharing has significantly impacted our timelines as well as clinical strategy and design CABA-201 Efficient clinical trial designs for CABA-201 facilitate rapid &
broad development program Product – Candidate with 4-1BB co-stim domain; similar binding activity to academic CD19-CAR T1-4 Efficient clinical trial designs across multiple autoimmune diseases CABA-201 fully human binder clinical tolerability
profile based on use in ~20 oncology patients 4-1BB co-stimulatory domain identical to that used in academic CD19-CAR T study1,2 People – Singular focus on potentially curative cell therapies for autoimmune disease since 2018 Deep
understanding and experience with logistically complicated cell therapy programs in autoimmune patients Novel insights on clinical designs from prior FDA discussions and 4 timely submitted and cleared IND filings Track record at a dozen US sites
with implementation of complicated cell therapy logistics in autoimmune patients Leadership with experience developing both SLE products that were FDA approved in the past 65 years 1 2 3
Chimeric AutoAntibody Receptor T
Cells DSG3-CAART & MuSK-CAART
Current treatments require broad
immunosuppression associated with safety risks and transient efficacy Overview of pemphigus vulgaris & current treatment landscape Image credit: D@nderm. http://www.vgrd.org/archive/cases/2004/pv/DSCN4996%20copy.JPG Joly, Pascal, et al.
"First-line rituximab combined with short-term prednisone versus prednisone alone for the treatment of pemphigus (Ritux 3): a prospective, multicentre, parallel-group, open-label randomised trial." The Lancet 389.10083 (2017): 2031-2040.
Werth, Victoria P., et al. "Rituximab versus Mycophenolate Mofetil in Patients with Pemphigus Vulgaris." New England Journal of Medicine (2021). Rituximab label, 08/2020 revision. Kushner, Carolyn J., et al. "Factors Associated With Complete
Remission After Rituximab Therapy for Pemphigus." JAMA dermatology (2019). Tony, Hans-Peter, et al. "Safety and clinical outcomes of rituximab therapy in patients with different autoimmune diseases: experience from a national registry
(GRAID)." Arthritis research & therapy 13.3 (2011): 1-14. CROT = 8+ weeks without lesions while off systemic therapy Mucosal PV1 (25%) Associated Antibody Anti-DSG3 Anti-DSG3 + Anti-DSG1 Clinical Signs Painful blisters of the mucous membranes
(mouth, nose, larynx, esophagus, eyes, genitalia, rectum) Blisters on orifices and skin U.S. Disease Prevalence 3,250 to 4,750 9,750 to 14,250 Mucocutaneous PV2 (75%) DSG3-CAART Transient remission Broad immunosuppression3,6 Rituximab plus steroids
(~3,500 mg/yr)4 Modestly effective & poorly tolerated 8+ weeks6 16+ weeks4,5 % of patients failing to achieve any 8+ or 16+ weeks without lesions or medicines Not Achieved Achieved ~30% relapse in 1 year & >50% relapse in 2 years6 Safety
risks 22% annual serious adverse event (SAE) rate4 4-9%3,4,5 annual risk of severe infection in PV ~1.9% lifetime risk of fatal infection7
Favorable tolerability to date, but
only modest persistence increase observed with IVIg & Cy Ongoing DesCAARTes™ study in patients with mucosal PV Volkov J, et al. “Correlative findings following DSG3-CAART infusion with & without combination preconditioning
therapy in patients with Pemphigus Vulgaris (DesCAARTes study).” Poster presented at: American Society Gene and Cell Therapy 26th Annual Meeting; 2023 May 18; Los Angeles, CA. * 20M, 2.5B, 5.0B to 7.5B and 10B to 15B refers to the number of
DSG3-CAART cells infused for patients in dosing cohorts A1 to A6m (M – millions; B – billions). Fast Track Designation Cohorts Combination (Pre-Treatment) Dose* A1 – A4 -- 20M to 2.5B A5 -- 5 to 7.5B A6m -- Up to 15B P4 IVIg /
Cyclophosphamide (Cy) 2.5B P4F IVIg / Cy / Fludarabine (Flu) 2.5B Primary objective: Determine the maximum tolerated dose of DSG3-CAART Primary endpoint: Adverse events, including dose-limiting toxicities (DLTs), related to DSG3-CAART within 3
months of infusion DSG3-CAART DesCAARTes™ study of DSG3-CAART Ongoing open-label Phase 1 study to determine the maximum tolerated dose & to evaluate safety of DSG3-CAART Fast Track Designation Orphan Drug Designation * Range of persistence
observed in CD19-CAR T studies in B cell malignancies * DSG3-CAART Peak Persistence1 Combination cohort (P4F) with fludarabine / Cy / IVIg currently enrolling and designed to evaluate the ability to improve DSG3-CAART engraftment & persistence
Study timelines being evaluated
based on emerging data from DesCAARTes™ study High unmet need in MuSK myasthenia gravis MusCAARTes™ study of MuSK-CAART Ongoing open-label study to determine the maximum tolerated dose & to evaluate safety of MuSK-CAART Part Cohort
Subjects A – Monotherapy Dose Escalation7 Dose increasing from 500M with 2 (+4) design A1-A3 2 (+4) per cohort A – Adaptive Combination Cohorts7 Combination cohorts8, starting at A2 dose A4+ 2 (+4) per cohort B – Expansion Expanded
subject enrollment at final selected dose B ~12 MuSK-CAART IgG4-dominant disease Autoantibody titers drop after rituximab1,2 Pathogenic B cells incompletely eliminated by rituximab and persist during relapse3 Circulating anti-MuSK antibody titers
may be ~90% lower than anti-DSG3 antibody in PV4,5,6 1 2 Study Endpoint & Objectives Primary Endpoint: Adverse Events, including DLT Secondary & Tertiary Objectives: CAART expansion/persistence, effect on serum anti-MuSK antibody titer, use
of concomitant systemic medications, effect on clinical symptoms, manufacturing success rate Compelling biologic rationale, similar to PV Differentiated market opportunity Total U.S. MG prevalence 50,000 to 80,000 patients MuSK+ MG comprises 6-7.5%
of total MG population Compared to AChR+ MG, MuSK+ disease is typically more severe with limited treatment options MuSK+ disease has early onset, 7:1 females * 500M refers to the number of MuSK-CAART cells infused for patients in dosing cohort A1 (M
– millions). Hain, Berit, et al. "Successful treatment of MuSK antibody–positive MG with rituximab." Muscle & Nerve. 33.4 (2006): 575-580. Illa, Isabel, et al. "Sustained response to Rituximab in anti-AChR and anti-MuSK positive
Myasthenia Gravis patients." Journal of neuroimmunology 201 (2008): 90-94. Jiang, Ruoyi, et al. "Single-cell repertoire tracing identifies rituximab-resistant B cells during myasthenia gravis relapses." JCI insight 5.14 (2020). Matthews, Ian, et al.
"Muscle-specific receptor tyrosine kinase autoantibodies—a new immunoprecipitation assay." Clinica chimica acta 348.1-2 (2004): 95-99. McConville, John, et al. "Detection and characterization of MuSK antibodies in seronegative myasthenia
gravis." Annals of neurology 55.4 (2004): 580-584. Marino, Mariapaola, et al. "Long-lasting rituximab-induced reduction of specific—but not total—IgG4 in MuSK-positive myasthenia gravis." Frontiers in immunology 11 (2020): 613.
A total of 6 subjects will need to have received the final selected dose in Part A of the study. Cohorts A3 and A4 will be enrolled concurrently after cohort A2 is well-tolerated with a preference for enrollment into A4. Fast Track Designation
Orphan Drug Designation
Corporate Summary
Three-stage approach allows for
efficient allocation of capital while leveraging experienced partners Manufacturing strategy Currently contracted for MuSK-CAART product candidate. Stage 3: Commercialization & Scale-Up Data-gated, staged investment Stage 1: Penn Stage 2: CDMOs
& CABA Process Cell processing capacity secured through Penn partnership SOPs previously used to develop multiple clinical stage CAR T products Clinical vector validated CDMOs for vector and cell processing with commercial support capabilities
Leasing followed by engineering and build out of Cabaletta-owned manufacturing facility, and/or Establishment of a strategic partnership to rapidly & reliably scale manufacturing, leveraging the partner’s manufacturing expertise 2021
– 2019 – 1 1
BOARD OF DIRECTORS Cabaletta Bio
leadership LEADERSHIP TEAM Anup Marda Chief Financial Officer Arun Das, M.D. Chief Business Officer David J. Chang, M.D., M.P.H., FACR Chief Medical Officer Martha O’Connor Chief HR Officer Michael Gerard General Counsel Steven Nichtberger,
M.D. President, CEO & Chairman Heather Harte-Hall Chief Compliance Officer Samik Basu, M.D. Chief Scientific Officer Gwendolyn Binder, Ph.D. President, Science & Technology SCIENTIFIC ADVISORY BOARD Track record of operational success
evaluating novel cell therapy candidates in autoimmunity across preclinical, clinical, manufacturing & regulatory domains Aimee Payne, M.D., Ph.D. Co-Founder and Co-Chair Michael C. Milone, M.D., Ph.D. Co-Founder and Co-Chair Carl June, M.D. Jay
Siegel, M.D. Brian Daniels, M.D. Steven Nichtberger, M.D. Richard Henriques, M.B.A. Scott Brun, M.D. Mark Simon, M.B.A. Catherine Bollard, M.D. Drew Weissman, M.D., Ph.D. Iain McInnes, Ph.D., FRCP, FRSE, FMedSci Georg Schett, M.D. Shawn Tomasello,
M.B.A.
Leveraging years of experience with
cellular therapy in autoimmunity to initiate CABA-201 clinical trials Cabaletta®: Pursuing cures for a broad range of autoimmune diseases CARTA – Chimeric Antigen Receptor T cells for Autoimmunity; CAART – Chimeric AutoAntibody
Receptor T cells; IND – Investigational New Drug; SLE – Systemic lupus erythematosus; DM – Dermatomyositis; ASyS – Anti-synthetase syndrome; IMNM – Immune-mediated necrotizing myopathy Mackensen, Andreas, et al.
"Anti-CD19 CAR T cell therapy for refractory systemic lupus erythematosus." Nature Medicine (2022): 1-9. Müller, Fabian, et al. "CD19-targeted CAR T cells in refractory antisynthetase syndrome." The Lancet (2023). Dai, Zhenyu, et al.
"Development and functional characterization of novel fully human anti‐CD19 CARs for T‐cell therapy." Journal of Cellular Physiology 236.8 (2021): 5832-5847. Assumes no dose-limiting toxicities are observed in the cohort and
uninterrupted enrollment occur in the trials. Advancing SLE & myositis trials with efficient designs – including starting dose & parallel cohorts 1.0 x 106 cells/kg initial dose of CABA-201 is identical to dose used in academic SLE1
and myositis2 studies Parallel cohorts with 6 patients each – SLE study with 1) LN & 2) Non-renal SLE; myositis study with 1) DM, 2) ASyS & 3) IMNM CABA-201 has been specifically engineered for patients with autoimmune diseases Fully
human CD19 binder with data in ~20 oncology patients – clinical safety profile supporting further evaluation in autoimmunity Same 4-1BB costimulatory domain and similar CD19 binder affinity3 as used in the academic SLE1 & myositis studies2
Potential to cure a broad range of autoimmune diseases where B cells have a role initiating or maintaining disease DesCAARTes™ trial of DSG3-CAART in mucosal pemphigus vulgaris Enrolling in combination sub-study cohort using pre-treatment with
IVIg, cyclophosphamide and fludarabine MusCAARTes™ trial in MuSK myasthenia gravis – leveraging insights from experience with DSG3-CAART Initiated in 4Q22; received FDA Fast Track Designation & Orphan Drug Designation CARTA Strategy
| CABA-201 (4-1BB CD19-CAR T) to be evaluated in SLE & myositis Phase 1/2 studies CAART Strategy | DSG3-CAART & MuSK-CAART clinical studies evaluating combination regimens Initial CABA-201 3-month clinical efficacy and tolerability data
expected by 1H244
Corporate Presentation JULY
2023
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