Core Insights - Cabaletta Bio, Inc. is a clinical-stage biotechnology company focused on developing curative targeted cell therapies for autoimmune diseases [3] - The company will participate in a fireside chat at Guggenheim's SMID Cap Biotech Conference on February 5, 2025 [1] - A live webcast of the presentation will be available on the company's website, with replays accessible for 30 days [2] Company Overview - Cabaletta Bio's CABA™ platform includes two strategies aimed at advancing engineered T cell therapies for autoimmune diseases [3] - The lead strategy, CARTA, focuses on the investigational therapy rese-cel, which is a fully human CD19-CAR T cell therapy containing 4-1BB [3] - Rese-cel is currently being evaluated in the RESET™ clinical development program across multiple therapeutic areas, including rheumatology, neurology, and dermatology [3]

Core Insights - Cabaletta Bio is advancing its investigational therapy rese-cel for autoimmune diseases, with plans to meet the FDA in the first half of 2025 to align on registrational trial designs based on emerging clinical data and increased patient enrollment [1][4][10] Recent Pipeline and Operational Progress - As of December 31, 2024, 21 patients have been enrolled across 44 active clinical sites in the U.S. and Europe within the RESET clinical development program [3] - Among the first 10 patients dosed with rese-cel, 90% experienced either no cytokine release syndrome (CRS) or grade 1 CRS, and 90% experienced no immune effector cell-associated neurotoxicity syndrome (ICANS) [3] - The first patient has been enrolled in the RESET-PV trial, which evaluates rese-cel without preconditioning [3] - The first juvenile myositis clinical site in the RESET-Myositis trial is now open and actively recruiting [3] - The FDA has cleared the IND application for rese-cel for the RESET-MS trial in multiple sclerosis, granting it Fast Track Designation [3] Strategic Priorities and Anticipated Key Milestones for 2025 - The company aims to gain alignment with the FDA on a path to registration for rese-cel, leveraging indication-specific trials to advance registrational programs [4] - Key priorities include enrolling patients and completing dosing in multiple disease-specific cohorts across the RESET clinical development program [5] - The company plans to present new and updated clinical and translational data on rese-cel throughout 2025 [5] Financial Guidance - Cabaletta ended Q4 2024 with unaudited cash and cash equivalents of $164 million, which is expected to fund its updated operating plan into the first half of 2026 [7] About the RESET-MS Trial - The RESET-MS trial is a Phase 1/2 open-label, dose escalation study evaluating rese-cel in subjects with relapsing and progressive forms of multiple sclerosis [8] About rese-cel - Rese-cel is a 4-1BB-containing fully human CD19-CAR T cell investigational therapy designed to transiently deplete all CD19-positive cells, potentially resetting the immune system for patients with autoimmune diseases [9][13]

Industry Overview - The year 2024 marked significant advancements in cell therapies, including new engineered T cell treatments and the introduction of the first tumor-infiltrating lymphocyte product [1] - There is growing recognition of the potential of CD19-directed CAR T-cell therapies in the field [1] Analyst Background - The analyst holds a PhD in biochemistry and has extensive experience in analyzing clinical trials and biotech companies [1] - The analyst is passionate about educating investors on the science behind biotech investments and aims to help them conduct thorough due diligence [1]

Product Development and Clinical Trials - CABA-201, the lead product candidate, is designed to treat a broad range of autoimmune diseases, with clinical responses in systemic lupus erythematosus maintained off immunosuppressive therapies for up to 2.5 years[99]. - The FDA granted Fast Track Designation for CABA-201 for systemic lupus erythematosus and lupus nephritis, with an estimated 160,000-320,000 patients affected in the U.S.[101]. - The RESET-SLE Phase 1/2 clinical trial is designed to treat six patients with active lupus nephritis and six patients with active systemic lupus erythematosus without renal involvement[103]. - As of November 12, 2024, 16 patients have been enrolled with 10 patients dosed across lupus, myositis, and systemic sclerosis clinical trials, with 40 U.S. clinical sites actively recruiting patients[112]. - The FDA granted clearance for CABA-201 IND applications for treatment of idiopathic inflammatory myopathy and systemic sclerosis, affecting approximately 66,000 and 88,000 patients in the U.S., respectively[104][105]. - Initial clinical data from the RESET-SLE and RESET-Myositis trials showed complete B cell depletion by day 15 post-infusion, with no serious adverse events observed[108]. - The RESET-MG Phase 1/2 clinical trial is designed to treat patients with generalized myasthenia gravis, with an estimated 50,000 to 80,000 patients affected in the U.S.[106]. - The company is working to activate clinical trial sites for the RESET-SLE trial in Canada and Europe, following regulatory approvals[103][104]. Financial Performance and Expenses - The company has not generated any revenue from product sales and does not expect to for several years[131]. - Research and development expenses are expected to increase substantially as the company continues to invest in product candidates and clinical trials[137]. - Research and development expenses increased to $26.3 million for Q3 2024, up from $13.8 million in Q3 2023, representing a 91% increase[145]. - General and administrative expenses rose to $6.8 million in Q3 2024, compared to $4.9 million in Q3 2023, marking a 39% increase[146]. - Total operating expenses for Q3 2024 were $33.0 million, up from $18.7 million in Q3 2023, reflecting a 77% increase[143]. - Net loss for Q3 2024 was $30.6 million, compared to a net loss of $16.4 million in Q3 2023, indicating an 86% increase in losses[143]. - Research and development expenses for the nine months ended September 30, 2024, totaled $71.7 million, up from $38.0 million in the same period of 2023, a 88% increase[150]. - General and administrative expenses for the nine months ended September 30, 2024, were $19.7 million, compared to $13.5 million in the same period of 2023, a 46% increase[152]. - The company experienced a net loss of $83.3 million for the nine months ended September 30, 2024, compared to a net loss of $46.8 million for the same period in 2023[167][168]. Cash and Financing Activities - As of September 30, 2024, the company had $183.0 million in cash, cash equivalents, and investments[154]. - For the nine months ended September 30, 2024, the company reported a net cash used in operating activities of $65.1 million, compared to $37.1 million for the same period in 2023[166][168]. - Cash provided by investing activities for the nine months ended September 30, 2024, was $35.2 million, primarily from the maturity of short-term investments[169]. - Cash provided by financing activities for the nine months ended September 30, 2024, was $7.3 million, significantly lower than $94.6 million for the same period in 2023[171][173]. - The company has no credit facility or committed sources of capital, indicating potential future funding challenges[165]. Manufacturing and Partnerships - The manufacturing strategy includes leveraging academic partners for early development and partnering with CDMOs for late-stage clinical studies and commercial production[115]. - The company entered into a new technology transfer agreement with Lonza in July 2024 to improve the commercial manufacturing process for CABA-201, making it nearly fully closed and semi-automated[116]. - The company has established multiple partnerships, including with Oxford Biomedica and WuXi, to enhance manufacturing capabilities for CABA-201[123][124]. - The company plans to secure scalable manufacturing capabilities through various strategies, including establishing new CDMO relationships and building its own facility[118]. - The company has entered into a partnership with Cellares Corp. to evaluate their automated manufacturing platform for CABA-201[117]. Future Outlook and Market Risks - The company anticipates continued increases in research and development and general administrative expenses in the foreseeable future[156]. - The company may generate future revenue from potential license or collaboration agreements for its product candidates[132]. - The company is exposed to market risks, primarily interest rate sensitivity, with an immediate 100 basis point change in interest rates not expected to materially affect cash equivalents[183][184]. - The company is an emerging growth company and plans to adopt new accounting standards as they become effective[177][181].

Exhibit 99.1 Cabaletta Bio Reports Third Quarter 2024 Financial Results and Provides Business Update – Clinical data from the RESET-Myositis™ and RESET-SLE™ trials, along with initial clinical data from the RESET-SSc™ trial, to be presented this weekend in oral and poster presentations at ACR Convergence 2024 – – 16 patients enrolled with 10 patients dosed as of November 12 across the RESET™ clinical development program; 40 U.S. clinical sites actively recruiting patients – – Data permitting, anticipate mee ...

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PHILADELPHIA, Sept. 25, 2024 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinicalstage biotechnology company focused on developing and launching the first curative targeted cell therapies designed specifically for patients with autoimmune diseases, today announced two presentations at the upcoming American College of Rheumatology (ACR) Convergence 2024, which is being held at the Walter E. Washington Convention Center in Washington, D.C. from November 14-19, 2024. The presentations will featur ...

PHILADELPHIA, Aug. 28, 2024 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinicalstage biotechnology company focused on developing and launching the first curative targeted cell therapies designed specifically for patients with autoimmune diseases, today announced that the Company will participate in the following investor conferences in September: Morgan Stanley 22nd Annual Global Healthcare Conference: Fireside chat on Wednesday, September 4, 2024 at 4:50 p.m. ET in New York, NY. 2024 Wells F ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from_______________ to _______________ | --- | --- | --- | |------------------------------------------------------------------------------|--------------------------- ...

Exhibit 99.1 abaletta Bio Cabaletta Bio Reports Second Quarter 2024 Financial Results and Provides Business Update – Nine patients enrolled as of August 5, 2024 across the RESET™ clinical development program, including four since EULAR in June, with 22 U.S. clinical sites now enrolling – – Additional clinical data from the RESET-Myositis™ and RESET-SLE™ trials as well as initial clinical data from the RESET-SSc™ and RESET-MG™ trials anticipated in 2H24 – – Initial clinical and translational data from each o ...