PHILADELPHIA - Cabaletta Bio, Inc. (NASDAQ: NASDAQ:CABA), a clinical-stage biotechnology company, announced new clinical data on its investigational therapy CABA-201, showing potential for drug-free clinical responses in autoimmune diseases. The data, presented at the American College of Rheumatology Convergence 2024, included eight patients across the ongoing Phase 1/2 RESET clinical trials.
The patients, all with active, refractory autoimmune diseases, discontinued all immunosuppressants prior to and throughout the follow-up period after receiving CABA-201 infusion. Safety profiles indicated no cytokine release syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS) in the majority, with low-grade CRS in three patients and one previous ICANS event reported.
CABA-201 led to consistent and complete B cell depletion within a month post-infusion. Early signs of immune system reset were observed, with evidence of transitional naïve B cell repopulation as early as eight weeks in the initial two patients.
Clinical outcomes showed promising signs of efficacy. One patient with immune-mediated necrotizing myopathy (IMNM) maintained an improved clinical response six months post-treatment without immunosuppressants. In the systemic lupus erythematosus (SLE) cohort, all patients exhibited clinical responses, and one patient with lupus nephritis (LN) showed significant improvement in proteinuria levels.
The RESET clinical development program is actively recruiting at 40 U.S. clinical sites, with 16 patients enrolled and 10 dosed with CABA-201 as of November 12, 2024. The company plans to discuss potential registrational trial designs with the FDA in 2025, aiming to advance the therapy's development.
CABA-201, a 4-1BB-containing fully human CD19-CAR T cell therapy, is designed for a one-time infusion to deplete CD19-positive B cells, potentially resetting the immune system for durable remission without ongoing immunosuppression.
Cabaletta Bio is set to host a live investor conference call and webcast today at 8:00 a.m. ET to review the clinical data and provide updates on the RESET program. This announcement is based on a press release statement.
In other recent news, Cabaletta Bio has experienced significant developments. Guggenheim maintained a Buy rating on Cabaletta Bio, with a target of $23, following a considerable acceleration in patient enrollment for clinical studies. The company also reported plans for discussions with the FDA in 2025 regarding a potential registrational program for CABA-201. UBS initiated coverage on Cabaletta Bio, issuing a Buy rating and setting a price target of $10.00, while highlighting the potential of CABA-201 in treating autoimmune diseases like systemic lupus erythematosus (SLE) and myositis.
Cabaletta Bio has secured lease terms through August 2026 for its Philadelphia location, providing a stable operational base for its research and development activities. The company's lead candidate, CABA-201, has shown promising data and received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration for systemic sclerosis treatment. Analysts from H.C. Wainwright, Stifel, and Citi have maintained their Buy ratings on Cabaletta Bio, with price targets of $25.00, $26.00, and $30.00 respectively.
These recent developments highlight the company's progress in clinical trials and favorable analyst ratings, underscoring the potential of its therapeutic candidate, CABA-201.
InvestingPro Insights
As Cabaletta Bio (NASDAQ: CABA) presents promising clinical data for its CABA-201 therapy, investors should consider some key financial metrics and insights from InvestingPro.
According to InvestingPro data, Cabaletta Bio's market capitalization stands at $148.1 million, reflecting its status as a clinical-stage biotechnology company. The company's stock price has experienced significant volatility, with a 32.06% decline in the past week and an 83.13% drop over the last year. This volatility aligns with the nature of early-stage biotech companies, where stock performance can be heavily influenced by clinical trial results and regulatory developments.
InvestingPro Tips highlight that Cabaletta Bio holds more cash than debt on its balance sheet, which is crucial for funding ongoing clinical trials and research. However, the company is quickly burning through cash, a common characteristic of pre-revenue biotech firms investing heavily in R&D. This cash burn rate underscores the importance of the positive clinical data for CABA-201, as successful trial outcomes could potentially lead to partnerships or additional funding opportunities.
It's worth noting that Cabaletta Bio is not currently profitable, with a negative P/E ratio of -1.42 for the last twelve months as of Q3 2024. This is not unusual for clinical-stage biotech companies, but it emphasizes the importance of the company's pipeline progress, particularly with CABA-201.
For investors seeking a more comprehensive analysis, InvestingPro offers 14 additional tips for Cabaletta Bio, providing a deeper understanding of the company's financial health and market position.
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