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Cabaletta Bio Announces FDA Granted Orphan Drug Designation to CABA-201 for Treatment of Systemic Sclerosis | ||
By: GlobeNewswire - 20 Mar 2024 | Back to overview list |
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PHILADELPHIA, March 20, 2024 (GLOBE NEWSWIRE) -- 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 that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CABA-201, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy, for the treatment of systemic sclerosis (SSc). CABA-201 is in development as a potential treatment for autoimmune diseases driven by B cells. The RESET™ (REstoring SElf-Tolerance) clinical trial program includes four Phase 1/2 trials advancing for the evaluation of CABA-201 across multiple autoimmune conditions, including the Phase 1/2 RESET-SSc™ trial. “Patients diagnosed with systemic sclerosis, a rare and life-threatening chronic autoimmune disease characterized by progressive skin and internal organ fibrosis, face limited treatment options as current therapies provide only modest effects and focus on treating the complications associated with the disease. With an average patient survival of 12 years following diagnosis, there is a significant unmet need for new treatment options that focus on eliminating the root cause of the disease to prevent further organ damage for patients,” said David J. Chang, M.D., Chief Medical Officer of Cabaletta. “Based on the role of B cells and the recently published academic clinical data with CD19-CAR T therapy in systemic sclerosis, we believe CABA-201 may transform the treatment for systemic sclerosis. Orphan Drug Designation is an important recognition for investigational therapies for rare diseases and provides us with potentially valuable benefits as we develop CABA-201 for patients with systemic sclerosis.” The FDA grants Orphan Drug Designation to drugs or biologics intended to treat or prevent rare diseases or conditions that affect fewer than 200,000 individuals in the United States. This designation qualifies Cabaletta for certain incentives, which may include partial tax credit for clinical trial expenditures, waived user fees and potential eligibility for seven years of marketing exclusivity. About the RESET-SSc™ Trial About CABA-201 About Systemic Sclerosis About Cabaletta Bio Forward-Looking Statements 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 CABA-201; the risk that the results observed with the similarly-designed construct employed in the recent academic publications, including due to the dosing regimen, are not indicative of the results we seek to achieve with 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 and public health crises; 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 other 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 William Gramig |
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