Core Insights - Cabaletta Bio is advancing its investigational CAR-T cell therapy, rese-cel, with plans for a Biologics License Application (BLA) submission for myositis anticipated in 2027 following FDA alignment on registrational pathways [1][11] - The company has successfully enrolled five disease-specific cohorts in the RESET™ clinical development program, with ongoing expansion phase enrollment [1][4] - Recent clinical data presented at the EULAR 2025 Congress indicate strong therapeutic potential for rese-cel, with nearly all patients off immunomodulatory medications and steroids [1][4] Clinical Development - Rese-cel is designed to treat autoimmune diseases by transiently depleting CD19-positive cells, aiming for durable clinical responses without chronic therapy [3] - Enrollment in two open-label, single-arm registrational myositis cohorts is set to begin in the second half of 2025, each consisting of approximately 15 patients [4][11] - Upcoming presentations will include complete Phase 1/2 data from the RESET-Myositis trial and initial data from RESET-PV and RESET-MG trials [4] Regulatory - The FDA has aligned with Cabaletta on key design elements for registrational cohorts in the RESET-Myositis trial, which includes patients with dermatomyositis and antisynthetase syndrome [11] - Additional regulatory discussions are planned for the RESET-SLE and RESET-SSc trials in late 2025 and early 2026 [11] Financial Overview - Cabaletta closed a $100 million public offering to support late clinical-stage development and extended its cash runway into the second half of 2026 [1][7] - For Q2 2025, research and development expenses were $37.6 million, up from $23.4 million in Q2 2024, while general and administrative expenses rose to $8.3 million from $6.9 million [12][17] - As of June 30, 2025, the company reported cash, cash equivalents, and short-term investments of $194.7 million, an increase from $164.0 million at the end of 2024 [12][19]

Rese-cel Development and Regulatory Strategy - Cabaletta Bio plans to submit a Biologics License Application (BLA) for Rese-cel in 2027 following recent alignment with the FDA [7] - The company is rapidly accelerating enrollment across a broad portfolio of indications at 67 active clinical sites [7, 14] - The initial BLA submission is planned in myositis, which affects approximately 80,000 US patients, followed by rapid expansion to SLE/LN and SSc [7] - The FDA has aligned with Cabaletta Bio on an open-label, single-arm evaluation of two registrational cohorts in the RESET-Myositis trial, with approximately 15 patients in each subtype-specific cohort [8] - Over 40% of the safety database is already enrolled across the RESET clinical development program [18] Clinical Data and Efficacy - Clinically meaningful responses have been observed with most patients off all immunomodulatory medications and steroids [8] - Deep transient systemic B cell depletion in blood and tissues has been confirmed by lymph node biopsy in an SSc patient [8] - In non-renal SLE, 3 out of 4 patients achieved DORIS (Definition of Remission in SLE) at the latest follow-up [84] - In the LN cohort, the first patient achieved CRR (Complete Renal Response) at week 44 [89] Manufacturing and Innovation - Cabaletta Bio is evaluating a whole blood process to eliminate apheresis [112] - The company has an expanded partnership for automated manufacturing with Cellares and completed the Technology Adoption Program [110]

Core Viewpoint - Cabaletta Bio, Inc. has announced a public offering of common stock and warrants, aiming to raise approximately $100 million to support its development of targeted cell therapies for autoimmune diseases [2][3]. Group 1: Offering Details - The offering consists of 39,200,000 shares of common stock and accompanying warrants, with an additional option for underwriters to purchase up to 15,000,000 more shares [1][2]. - The combined offering price for each share of common stock and accompanying warrant is set at $2.00, while the pre-funded warrants are priced at $1.99999 [1]. - The exercise price for the accompanying warrants is $2.50 per share, and they are immediately exercisable for fifteen months from the date of issuance [1]. Group 2: Company Overview - Cabaletta Bio is a clinical-stage biotechnology company focused on developing curative targeted cell therapies for autoimmune diseases [6]. - The company’s CABA™ platform includes strategies for engineered T cell therapies, with a lead investigational therapy called rese-cel currently in clinical trials [6]. - The RESET™ clinical development program for rese-cel spans multiple therapeutic areas, including rheumatology, neurology, and dermatology [6].

Core Insights - Cabaletta Bio (NASDAQ: CABA) shows promising efficacy signals in its approach, which is gaining traction in the medical community [1] Company Analysis - The company is being analyzed with cautious optimism due to its potential in the biotech sector [1] - The focus is on educating investors about the scientific basis of the company's business model [1] Industry Context - The medical community is increasingly gaining experience with the methodologies employed by Cabaletta Bio, indicating a positive trend in the industry [1]

Core Viewpoint - Cabaletta Bio, Inc. has initiated an underwritten public offering of its common stock and accompanying warrants, aiming to raise capital for its clinical-stage biotechnology operations focused on autoimmune diseases [1][2]. Group 1: Offering Details - The public offering includes shares of common stock and pre-funded warrants, with an option for underwriters to purchase an additional 15% of the offering [1][2]. - The offering is expected to close around June 12, 2025, subject to market conditions and customary closing conditions [2]. Group 2: Company Overview - Cabaletta Bio is a clinical-stage biotechnology company dedicated to developing curative targeted cell therapies for autoimmune diseases [5]. - The company’s CABA™ platform employs two strategies to advance engineered T cell therapies, with a focus on the CARTA strategy and its lead investigational therapy, rese-cel [5]. - Rese-cel is currently being evaluated in the RESET™ clinical development program, which spans multiple therapeutic areas including rheumatology, neurology, and dermatology [5].

Core Insights - Cabaletta Bio, Inc. announced promising clinical data from its ongoing RESET clinical trials for autoimmune diseases, highlighting the potential of rese-cel to provide drug-free, symptom-free lives for patients [1][2][6] Clinical Trial Results - In the RESET-Myositis trial, 7 out of 8 patients achieved clinically meaningful total improvement scores (TIS) while off all immunomodulators and steroids, with 4 patients experiencing grade 1 cytokine release syndrome (CRS) [1][2][3] - All systemic lupus erythematosus (SLE) patients without nephropathy achieved remission as per DORIS criteria, with 7 out of 7 patients responding clinically while off immunomodulators and glucocorticoids [1][2] - Both patients in the RESET-SSc trial showed significant improvements in modified Rodnan Skin Score (mRSS) after discontinuing immunomodulatory drugs, with one patient meeting the revised Composite Response Index in Systemic Sclerosis (CRISS) criteria [1][8] Safety Profile - Among 18 patients with follow-up of 4 weeks or more, 94% experienced no CRS or only Grade 1 CRS, and 89% had no immune effector cell-associated neurotoxicity syndrome (ICANS) [1][2] - In the RESET-SLE trial, 2 out of 8 patients experienced grade 1 CRS, and one ICANS event was reported [3][8] Regulatory and Development Plans - Cabaletta plans to initiate enrollment in two registrational myositis cohorts in 2025 and has scheduled discussions with the FDA regarding registrational pathways for SLE/LN in 3Q25, scleroderma in 4Q25, and myasthenia gravis in 1H26 [1][9] - The RESET clinical trial program is accelerating, with 51 patients actively enrolled across over 65 clinical sites as of May 30, 2025 [1][2][6] Company Overview - Cabaletta Bio is focused on developing curative targeted cell therapies for autoimmune diseases, with rese-cel being a key investigational therapy designed to deplete CD19-positive B cells [5][6] - The company aims to change treatment paradigms for autoimmune diseases through its innovative CARTA platform, which includes multiple disease-specific clinical trials [5][6]

Summary of Caballetta Bio's Conference Call Company Overview - **Company**: Caballetta Bio - **Event**: Jefferies Global Healthcare Conference - **CEO**: Steven Key Industry Insights - **Regulatory Alignment**: Caballetta Bio has aligned with the FDA on the registrational trial design for Reza cel, focusing on myositis as a target indication [3][4] - **Clinical Endpoint**: The primary endpoint for the trial will be the TIS score, a validated clinical endpoint previously used for IVIG approval in dermatomyositis [3][4] - **Cohort Design**: The trial will include two cohorts, one for ACEs and dermatomyositis subtypes (85% of myositis patients) and another for the necrotizing form (IMNM subtype, 15% of patients) [6][8] Efficacy and Safety Data - **Patient Enrollment**: The trial will enroll approximately 15 patients per cohort, focusing on those refractory to other therapies [4][10] - **Safety Database**: A safety database of over 100 patients will be utilized for the filing, with 35 patients having myositis-specific data [10][11] - **Weight-Adjusted Dosing**: Caballetta Bio is the only company using weight-adjusted dosing, which is believed to enhance safety and efficacy [18][19] Market Dynamics - **Competitive Landscape**: There are currently 35 cell therapies IND cleared for lupus, indicating a crowded market where payers require specific data for reimbursement [15] - **Patient Expectations**: Patients desire to eliminate symptoms and stop immunosuppressants, while physicians prioritize organ protection [22][24] Future Milestones - **Upcoming Presentations**: Caballetta Bio will present data at EULAR, including safety and efficacy results from 18 patients dosed for at least one month [31][36] - **Regulatory Alignment**: Expected regulatory alignments with the FDA for lupus and scleroderma in the second half of the year [45][46] - **Enrollment Projections**: Enrollment is exceeding aggressive forecasts, with ongoing updates expected at medical meetings [44][46] Additional Considerations - **Unmet Need**: There is a profound unmet need in the myositis and scleroderma markets, with significant patient enrollment observed [29][28] - **Cost Reduction Potential**: Collaborations with Solaris may lead to reduced manufacturing costs and increased scalability [48] Conclusion Caballetta Bio is positioned to make significant strides in the treatment of autoimmune diseases with its innovative approach to CAR T therapies, focusing on safety, efficacy, and regulatory alignment. The upcoming data presentations and regulatory milestones will be critical in shaping the company's future trajectory in the market.

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 the Jefferies Global Healthcare Conference on June 4, 2025 [1] - The lead strategy, CARTA, is centered on the investigational therapy rese-cel, which is currently in the RESET clinical development program [3] Company Overview - Cabaletta Bio is dedicated to advancing engineered T cell therapies that may provide deep and durable, potentially curative treatments for a variety of autoimmune diseases [3] - The company is headquartered in Philadelphia, PA, and has a comprehensive platform that includes two complementary strategies for therapy development [3] - Rese-cel is a fully human CD19-CAR T cell therapy that contains 4-1BB and is being evaluated across multiple therapeutic areas, including rheumatology, neurology, and dermatology [3]

Financial Performance - As of March 31, 2025, Cabaletta Bio, Inc. reported total assets of $165,141,000, a decrease of 10.8% from $185,046,000 on December 31, 2024[15]. - The company incurred a net loss of $35,943,000 for the three months ended March 31, 2025, compared to a net loss of $25,047,000 for the same period in 2024, representing a 43.5% increase in losses year-over-year[18]. - The net loss per share for the three months ended March 31, 2025, was $0.71, compared to $0.51 for the same period in 2024[18]. - The accumulated deficit as of March 31, 2025, reached $385,044,000, an increase from $349,101,000 at the end of 2024[15]. - The total stock-based compensation for the three months ended March 31, 2025, was $5.16 million, compared to $3.79 million for the same period in 2024, reflecting a 36% increase[91]. - The net loss for Q1 2025 was reported at $35,943,000, compared to a net loss of $25,047,000 in Q1 2024, indicating increased operational expenditures[71]. - For the three months ended March 31, 2025, total operating expenses increased to $37.1 million from $28.0 million in the same period of 2024, representing a change of $9.1 million[144]. - Cash used in operating activities was $30.8 million for Q1 2025, compared to $24.0 million in Q1 2024, indicating an increase of $6.8 million[161]. - As of March 31, 2025, the company had $131.8 million in cash and cash equivalents, which is expected to fund operations into the first half of 2026[149]. Research and Development - Research and development expenses increased to $29,018,000 for the three months ended March 31, 2025, up 32.3% from $21,954,000 in the prior year[18]. - Research and development expenses for Q1 2025 totaled $28,973,000, a significant increase from $18,000,000 in Q1 2024, reflecting a rise in personnel and clinical trial costs[71]. - Research and development expenses rose to $29.0 million in Q1 2025, up from $22.0 million in Q1 2024, marking an increase of $7.1 million[145]. - The company anticipates continued increases in research and development and general administrative expenses, with a need for additional funding to support operations[153]. - The company plans to raise additional capital through equity offerings, debt financings, and strategic alliances to fund its operations[32]. - The company expects to incur additional losses in the future as it continues its research and development efforts and will need to raise additional capital[32]. Clinical Trials and Product Development - The FDA granted clearance for the rese-cel IND application for systemic lupus erythematosus (SLE) treatment, affecting an estimated 320,000 patients in the U.S. and 150,000 in Europe, with approximately 40% of SLE patients experiencing lupus nephritis (LN)[103]. - The RESET-SLE Phase 1/2 clinical trial is designed to treat 12 patients, with a single weight-based dose of 1.0 x 10^6 cells/kg, and is open for enrollment across multiple sites in the U.S. and one in the EU[103]. - The FDA granted Fast Track Designation for rese-cel for SLE and LN, and the RESET-Myositis trial is actively enrolling patients with three myositis subtypes, affecting approximately 70,000 patients in the U.S. and 85,000 in Europe[104]. - The RESET-SSc trial for systemic sclerosis (SSc) is designed to treat 12 patients, with SSc affecting approximately 90,000 patients in the U.S. and 60,000 in Europe[105]. - The RESET-MG trial for generalized myasthenia gravis (gMG) is open for enrollment, targeting approximately 55,000 patients in the U.S. and 100,000 in Europe[107]. - As of May 9, 2025, 44 patients are enrolled and 23 patients have been dosed across multiple Phase 1/2 disease cohorts in the RESET clinical development program[118]. - The company is collaborating with Cellares Corp. to evaluate an automated manufacturing platform, with successful integration of the Cell Shuttle™ into the manufacturing strategy for rese-cel[120]. - The company plans to implement two registrational cohorts in the RESET-Myositis trial, each evaluating approximately 15 patients, with a focus on achieving a broad label for myositis treatment[116]. Financial Position and Liabilities - Total current liabilities rose to $33,989,000 as of March 31, 2025, compared to $27,086,000 on December 31, 2024, indicating a 25.4% increase[15]. - The company has an accumulated deficit of $385.0 million as of March 31, 2025, indicating ongoing financial challenges[152]. - Future lease payments under non-cancelable leases as of March 31, 2025, total $25,444,000 for finance leases and $5,939,000 for operating leases[79]. - The company has no off-balance sheet risks, such as foreign exchange contracts or other hedging arrangements, ensuring a straightforward financial position[37]. - The company has not recorded any income tax benefits for the three months ended March 31, 2025, due to the likelihood of not recognizing deferred tax benefits[95]. Risks and Challenges - The company has not yet established sales and marketing capabilities, which will be crucial upon obtaining regulatory approval to gain market acceptance[192]. - The regulatory approval process for the company's novel product candidates is complex and may take longer than expected, with potential delays in commercialization[190]. - Patients receiving T cell-based immunotherapies may experience serious adverse events, which could negatively affect the clinical development and commercial potential of the company's product candidates[193]. - The company faces inherent product liability risks during clinical testing, which could lead to substantial liabilities and limit commercialization efforts[209]. - The company currently does not hold product liability insurance for commercialization, which could inhibit the ability to market products if claims arise[210]. - Adverse events from CAR T cell therapies have resulted in patient deaths, indicating significant risks associated with current and future product candidates[213]. - The company is early in its development efforts and may face significant delays in clinical trials if suitable doses are not identified[188]. - The company must navigate variability in T cell quality and quantity, which could affect the reliability of manufacturing its product candidates[186]. Agreements and Collaborations - The Company is committed to pay up to $2,250,000 under the CARTA Services Agreement for cell processing manufacturing through December 31, 2025[54]. - The IASO Agreement includes an upfront payment of $2.5 million and potential total consideration of up to $162 million based on milestone achievements[123]. - The Company entered into a License and Supply Agreement with Oxford Biomedica, which includes an upfront fee and potential regulatory and sales milestone payments in the low tens of millions[124]. - The Company has entered into a Development and Manufacturing Services Agreement with Lonza for a term of 12 months, with the ability to extend, focusing on the CAR-T cell therapy product rese-cel[62]. - An Option and License Agreement with Autolus was established, requiring an upfront license fee of $1,200, with potential regulatory milestones of up to $12,000 for each licensed target and sales milestones totaling up to $15,000[59].

[Form 8-K Current Report](index=1&type=section&id=Form%208-K) [Results of Operations and Financial Condition](index=2&type=section&id=Item%202.02%20Results%20of%20Operations%20and%20Financial%20Condition) Cabaletta Bio disclosed its cash position as of March 31, 2025, was $131.8 million, down from $164.0 million at the end of 2024. The company expects its current cash reserves to fund operations into the first half of 2026 Cash and Cash Equivalents | Metric | As of March 31, 2025 | As of December 31, 2024 | | :--- | :--- | :--- | | Cash and Cash Equivalents | $131.8 million | $164.0 million | - The company projects its cash position as of March 31, 2025, will be **sufficient to fund its operating plan into the first half of 2026**[6](index=6&type=chunk) [Regulation FD Disclosure](index=2&type=section&id=Item%207.01%20Regulation%20FD%20Disclosure) On May 15, 2025, the company made two key disclosures: it posted an updated corporate presentation on its website and issued a press release announcing an anticipated 2027 BLA submission for Rese-cel in Myositis - An updated corporate presentation was posted to the "Investors & Media" section of the company's website on **May 15, 2025**[8](index=8&type=chunk) - The company issued a press release announcing its plan to **submit a BLA for Rese-cel in Myositis in 2027** after reaching an alignment with the FDA on registrational cohorts[9](index=9&type=chunk) [Financial Statements and Exhibits](index=2&type=section&id=Item%209.01%20Financial%20Statements%20and%20Exhibits) This section lists the exhibits furnished with the Form 8-K filing, which include the press release from May 15, 2025, and the Cover Page Interactive Data File - The following exhibits were filed with the report: - Exhibit 99.1: Press Release issued on May 15, 2025 - Exhibit 104: Cover Page Interactive Data File[11](index=11&type=chunk)