Cabaletta Bio
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- Public
- Established
- 2017-06-01
- Employees
- 103
- Market Cap
- $281.3M
- Website
- http://www.cabalettabio.com
- Introduction
Cabaletta Bio, Inc. is a clinical-stage biotechnology company, which engages in the discovery and development of engineered T cell therapies for B cell-mediated autoimmune diseases. Its proprietary technology utilizes chimeric autoantibody receptor to selectively bind and eliminate B cells that produce disease-causing autoantibodies. The company was founded by Michael C. Milone, Steven Nichtberger, and Aimee Payne in April 2017 and is headquartered in Philadelphia, PA.
CAR-T Cell Therapy Funding Surges to $141.2 Billion as Industry Expands Globally
• The CAR-T cell therapy industry has raised over $141.2 billion through various financing mechanisms, with estimates suggesting total industry funding could reach $281.7 billion when including undisclosed deals.
• More than 170 companies worldwide are developing CAR-T products with 1,944 therapies in development, while 13 CAR-T cell therapies have received regulatory approval globally since 2017.
• Despite a slowdown in IPOs and M&A activity in 2024, venture capital funding remains strong with 89 CAR-T companies securing $7.7 billion since 2014, supporting advancement in both blood cancer and solid tumor applications.
FDA's Cell and Gene Therapy Champion Peter Marks Departs, Leaving Industry at Critical Juncture
• Peter Marks, head of FDA's Center for Biologics Evaluation and Research since 2016, has resigned, leaving cell and gene therapy developers without their biggest regulatory advocate during a challenging investment period.
• Under Marks' leadership, the FDA approved dozens of cell and gene therapies including the first gene therapy, first cellular treatment for cancer, and first CRISPR gene editing medicine, establishing flexible regulatory frameworks for these novel treatments.
• Despite concerns about regulatory uncertainty following Marks' departure, incoming FDA Commissioner Marty Makary has signaled support for conditional approval pathways for rare disease treatments where randomized controlled trials aren't feasible.
Cellares Revolutionizes Cell Therapy Manufacturing with Automated Platform for Autoimmune and Cancer Treatments
• Cellares has successfully completed a Technology Adoption Program with Cabaletta Bio, demonstrating their Cell Shuttle platform can automate and scale manufacturing of rese-cel, a CAR-T therapy for autoimmune diseases including myositis, scleroderma, and lupus nephritis.
• The company has also formed a strategic partnership with the University of Wisconsin School of Medicine and Public Health to automate production of a CRISPR-edited GD2 CAR-T therapy for solid tumors, addressing manufacturing bottlenecks in clinical development.
• Cellares' Integrated Development and Manufacturing Organization (IDMO) Smart Factories in the US, Europe, and Japan aim to reduce manufacturing costs by up to 50% while increasing production capacity tenfold compared to conventional CDMOs.
Cabaletta Bio's Rese-cel Shows Promising Results in Multiple Autoimmune Diseases, Achieving Drug-Free Remission
• Clinical trials demonstrate significant efficacy of rese-cel across multiple autoimmune conditions, with three SLE patients achieving DORIS remission and successful discontinuation of immunosuppressants and steroids.
• Safety profile remains favorable among first 10 treated patients, with 90% experiencing either no or mild cytokine release syndrome and minimal neurotoxicity concerns.
• Treatment shows consistent deep B-cell depletion in all patients, with confirmed tissue-resident B cell elimination and successful recruitment across 50 clinical sites in US and Europe.
EPCORE NHL-2 Trial Shows Promise Combining Chemotherapy and Immunotherapy for DLBCL
• A phase Ib/II clinical trial led by Mount Sinai researchers demonstrates successful outcomes in combining chemotherapy with immunotherapy for diffuse large B-cell lymphoma treatment.
• The groundbreaking research, published in Blood journal, was conducted under the leadership of Dr. Joshua Brody, director of the Lymphoma Immunotherapy Program at the Tisch Cancer Institute.
• The study represents a significant advancement in lymphoma treatment, supported by the Leukemia & Lymphoma Society's research initiatives, which have invested over $600 million in hematologic oncology research.
IASO Bio's CAR-T Therapy, Equecabtagene Autoleucel, Advances in Hong Kong and Singapore for Multiple Myeloma
• IASO Bio's Equecabtagene Autoleucel (FUCASO) NDA has been accepted by the Hong Kong Department of Health for relapsed/refractory multiple myeloma.
• The CAR-T therapy, already approved in China, is the first fully human CAR-T product and has shown remarkable efficacy and safety.
• Singapore Health Sciences Authority (HSA) has also accepted the NDA for Equecabtagene Autoleucel, marking a key step in IASO Bio's global expansion.
• IASO Bio plans to implement a 'Manufactured in China, supplied overseas' model upon approval, expanding access to CAR-T therapies.
Cabaletta Bio Advances Novel CAR T-Cell Therapy for Multiple Sclerosis into Phase 1/2 Trial
• Cabaletta Bio has received FDA clearance to proceed with RESET-MS trial, testing their B-cell-targeting CAR T-cell therapy rese-cel in relapsing and progressive multiple sclerosis patients.
• The innovative cell therapy, rese-cel (formerly CABA-201), aims to reset the immune system by targeting CD19-positive B-cells, potentially offering a curative approach for autoimmune diseases.
• The trial will enroll adults aged 18-60 with either relapsing MS who experienced recent relapses or progressive MS patients showing worsening disability, following specific preconditioning treatment.
Cabaletta Bio's Rese-cel Shows Promising Safety and Efficacy in Autoimmune Disease Trials
• Cabaletta Bio will present updated clinical data on rese-cel at upcoming scientific meetings in February 2025, showcasing its potential in autoimmune diseases.
• Early data from the first 10 patients dosed with rese-cel show a favorable safety profile with minimal cytokine release syndrome or neurotoxicity.
• Cabaletta Bio plans to meet with the FDA in the first half of 2025 to discuss the registrational pathway for rese-cel.
• The company is expanding clinical trials of rese-cel across multiple autoimmune indications, including myositis, SLE, systemic sclerosis and multiple sclerosis.
CAR-T Cell Therapy Industry Surges to $141.2 Billion in Funding as Clinical Success Drives Investment
• The CAR-T cell therapy industry has raised over $141.2 billion through various financing mechanisms, with potential total value reaching $281.7 billion when including undisclosed deals.
• Since 2014, 89 CAR-T companies have secured $7.7 billion in venture capital funding, while 42 companies have raised $6.4 billion through IPOs, demonstrating sustained investor confidence.
• Despite a slowdown in M&A activity in 2024, the sector continues to expand with 170+ companies developing 1,944 therapies, including 244 trials targeting solid tumors beyond the current blood cancer focus.
CAR T-Cell Therapy Shows Promise in Treating Autoimmune Diseases: Clinical Trials and Future Directions
• UChicago Medicine launched a Phase 2 clinical trial to explore CAR T-cell therapy for systemic lupus erythematosus, inflammatory myositis, and systemic sclerosis.
• CAR T-cell therapy, initially for blood cancers, is being adapted to autoimmune diseases, potentially offering a new solution for resistant cases.
• BMS reported promising Phase 1 trial results using CD19 CAR T-cells for severe autoimmune diseases, showing significant disease activity reduction.
• Researchers are optimistic about CAR T-cell therapy's potential to 'reset' the immune system, but emphasize the need for long-term data and safety monitoring.
Lupus Nephritis Clinical Trial Landscape Shows Active Development
• The lupus nephritis clinical trial pipeline is robust, featuring over 30 companies developing more than 35 therapies.
• Key players like AstraZeneca, Roche, and Novartis are advancing drugs such as Anifrolumab and Ianalumab through clinical trials.
• Recent trial activities include Adicet Bio dosing the first patient in a Phase I trial of ADI-001 and Kyverna presenting updated clinical data on KYV-101.
• Various therapeutic modalities, including monoclonal antibodies and cell therapies, are being explored across different clinical stages.
Sana Biotechnology's SC291 Receives FDA Fast Track for Lupus Treatment
• The FDA has granted Fast Track designation to Sana Biotechnology's SC291 for relapsed/refractory systemic lupus erythematosus (SLE).
• SC291, a HIP-modified CD19-directed allogeneic CAR T therapy, is being evaluated in the GLEAM trial for B-cell mediated autoimmune diseases.
• Sana Biotechnology anticipates sharing initial clinical data from the GLEAM trial in 2025, highlighting the potential of SC291.
• Fast Track designation aims to expedite the development and review of drugs addressing serious conditions with unmet medical needs.
CAR-T Therapy Shows Promise in Autoimmune Diseases with Minimal Toxicities
• A phase 1/2 study of CD19 CAR-T therapy in autoimmune diseases, including lupus, myositis, and systemic sclerosis, demonstrates significant clinical responses.
• The CASTLE study reports minimal toxicities, with low-grade cytokine release syndrome and no major hematotoxicities or neurotoxicity observed in patients.
• Cabaletta Bio's CABA-201 shows potential for immunosuppressant-free clinical responses in refractory autoimmune diseases, with ongoing trials and planned FDA meetings.
• Next-generation manufacturing processes for CAR-T therapies, like that used for CC-97540, offer faster production times and require fewer cells for infusion.
Adicet Bio's ADI-001 Receives FDA Fast Track for Refractory SLE, Expands Autoimmune Pipeline
• Adicet Bio's ADI-001 receives FDA Fast Track designation for refractory systemic lupus erythematosus (SLE) with extrarenal involvement, expediting its development.
• A Phase 1 trial is underway, evaluating ADI-001 across six autoimmune indications, including lupus nephritis (LN), SLE, systemic sclerosis (SSc), and others, with preliminary data expected in 2025.
• ADI-001, an allogeneic gamma delta CAR T-cell therapy targeting CD20, has shown promising B-cell depletion in preclinical studies, suggesting potential as an off-the-shelf treatment.
• Adicet Bio is also advancing ADI-270, a CAR T-cell therapy for metastatic clear cell renal cell carcinoma (ccRCC), with Phase 1 data anticipated in the first half of 2025.
Biogen and UCB's Dapirolizumab Pegol Shows Promise in Phase III Lupus Trial
• Dapirolizumab pegol (DZP) plus standard of care demonstrated a statistically significant 49.5% response rate in SLE patients, compared to 34.6% with standard care alone.
• The Phase III PHOENYCS GO trial met its primary endpoint, with a treatment difference of 14.6% and a p-value of 0.0110, indicating clinical benefit.
• DZP also achieved key secondary endpoints, including a 1.8-fold greater improvement in SLEDAI-2000 scores (p=0.0001) and more patients achieving Lupus Low Disease Activity State.
• A second Phase III trial is planned to support regulatory submission, following positive safety and tolerability data from the PHOENYCS GO trial.
Cabaletta Bio Advances RESET Program for Autoimmune Diseases; Reports Q3 2024 Financials
• Cabaletta Bio's RESET program, featuring CABA-201, is actively enrolling patients across multiple clinical trials targeting autoimmune diseases like myositis, SLE, and SSc.
• The company has expanded its clinical trial program into Europe, securing EMA approval for the RESET-SLE trial, and is actively recruiting at 40 U.S. sites.
• Cabaletta Bio anticipates a meeting with the FDA in 2025 to discuss program designs for CABA-201, a cell therapy aimed at resetting the immune system in autoimmune disorders.
• With $183.0 million in cash reserves as of September 30, 2024, Cabaletta Bio expects to fund operations into the first half of 2026, supporting ongoing clinical development and strategic expansion.
Cabaletta Bio Announces Positive Clinical Updates for CABA-201 in Autoimmune Diseases
• Cabaletta Bio reported clinical data from RESET-Myositis™, RESET-SLE™, and initial data from RESET-SSc™ trials at ACR Convergence 2024, showcasing CABA-201's potential in autoimmune disease treatment.
• Sixteen patients have been enrolled, with ten dosed across the RESET™ clinical development program, and 40 U.S. clinical sites are actively recruiting patients for CABA-201 trials.
• Cabaletta anticipates a meeting with the FDA in 2025 to discuss potential registrational program designs for CABA-201, pending data outcomes.
• EMA has authorized clinical trials for CABA-201 in Europe for lupus, expanding the clinical development program internationally.
Cabaletta Bio Advances CABA-201 Trials, Expands to Europe, and Reports $183M Cash Position
• Cabaletta Bio reports 40 active U.S. clinical sites recruiting for its RESET trials, with 16 patients enrolled and 10 dosed as of November 12.
• The company received EMA CTA authorization for CABA-201 in lupus, facilitating expansion into Europe and broadening the clinical development program.
• Clinical data from RESET-Myositis, RESET-SLE, and initial RESET-SSc trials will be presented at ACR Convergence 2024, highlighting potential efficacy.
• Cabaletta Bio's cash position stands at $183.0 million as of September 30, 2024, expected to fund operations into the first half of 2026.
Cabaletta Bio to Present New CABA-201 Clinical Data at ACR Convergence 2024
• Cabaletta Bio will present new clinical data on CABA-201, a CD19-CAR T cell therapy, at the ACR Convergence 2024 in Washington, D.C.
• The presentations will cover the safety and efficacy of CABA-201 in patients with immune-mediated necrotizing myopathy and systemic lupus erythematosus.
• Correlative studies of CABA-201 in myositis and SLE will also be presented, offering insights into the therapy's mechanism of action.
• CABA-201 is being evaluated across multiple autoimmune conditions, holding promise for durable remission without chronic therapy.
CAR-T Cell Therapy Shows Promising Results in Lupus and Autoimmune Diseases at EULAR Conference
• Early clinical trial data presented at EULAR demonstrates CAR-T cell therapy's potential to induce remission in lupus and other autoimmune conditions, with some patients maintaining benefits for up to three years.
• Multiple companies including Kyverna Therapeutics, Cabaletta Bio, and Novartis reported encouraging safety profiles and positive patient responses, though some participants still require maintenance medications.
• While showing promise, experts emphasize the need for more extensive testing, particularly in diverse populations, and acknowledge challenges including manufacturing complexity and cost considerations.
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