PHILADELPHIA, March 01, 2022 (GLOBE NEWSWIRE) — Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on the discovery and progression of mobile treatments specific to patients with autoimmune diseases, today announced that the U. S. The Food and Drug Administration (FDA) has granted Fast Track designation to MuSK-CAART, or chimeric mobile muscle kinase autoantibodies receptor (MuSK) (MuSK-CAART), to improve activities of daily living and muscle strength in patients with MuSK antibodies – positive myasthenia gravis. MuSK-CAART is being evaluated as a possible remedy for patients with MuSK-related myasthenia gravis (MG).
Cabaletta’s investigational new drug (IND) application was recently approved by the FDA under the 30-day review regimen consistent with the period. Based on the clinical trial design and initial insights from Cabaletta’s DesCAARTes™ trial comparing mobile cure DSG3-CAART as a potential remedy for mucosal pemphigus vulgaris, the planned study to compare MuSK-CAART as a potential remedy for MuSK-associated MG begins with a cohort of one hundred million rolling doses, surpassing the 20 million rolling dose cohort used in the DesCAARTes™ trial, gets rid of the dose-fractionation scheme consistent with administering all doses in a single infusion and reduces the expected number of patients in each cohort from 3 to two patients in the absence of any dose-limiting toxicity. Cabaletta plans to start a first human clinical trial in 2022 for MuSK-CAART. The trial will be an open-label examination consisting of two parts: 1) dose escalation to determine the maximum tolerated dose with two patients planned according to the cohort and 2) expansion of the cohort to the final dose decided; and is expected to enroll approximately 20 patients at multiple clinical sites in the United States.
“Anti-MuSK autoantibodies are seen in a subset of patients diagnosed with MG, and the limited treatment functions for those patients underscore the need for new and more effective medical care,” said David J. Chang, MD, medical director of Caballete. The FDA’s resolution to grant Fast Track designation underscores the need for a remedy that can potentially provide deep and lasting responses to patients living with MUSK-associated MG. We look forward to launching our first human trial later this year. “
MuSK-CAART is designed in particular to target B cells that differentiate into antibody-secreting cells, which produce autoantibodies opposed to the kinase expressed in muscle, a transmembrane protein discovered in muscle cells and mandatory for the formation and maintenance of neuromuscular junction. Studies, MuSK-CAART demonstrated selective and express targeted in vitro interaction with no evidence of off-target toxicity to date. Studies in animal models recommend that MuSK-CAART interact with the target in vivo through the removal of the target anti-MuSK cells.
About Fast Track Designation The FDA’s Fast Track procedure aims to facilitate the accelerated progression and review of curative products to treat serious or life-threatening situations and address unmet medical needs. Companies earning fast track designation are eligible for several potential benefits, and choice of meetings and interactions more common with FDA clinical progression, as well as eligibility for accelerated approval and/or precedence review, if applicable criteria are met. Companies may also be able to submit sections of their biological license application on an ongoing basis.
About MuSK MuSK-related myasthenia gravis is an autoimmune disease mediated by autoantibodies that target the neuromuscular junction (NMJ), which can lead to life-threatening muscle weakness. Generalized MG (gMG) is characterized by profound muscle weakness in the body, which can lead to motor impairment, paralyzing fatigue, shortness of breath due to respiratory muscle weakness, and episodes of respiratory failure. gMG affects approximately 50,000 to 80,000 patients in the United States. Most patients who expand gMG have autoantibodies against the NMJ component that are known to be pathogenic. Between 80% and 90% of gMG patients have detectable serum acetylcholine receptor autoantibodies and receive regular treatment with acetylcholinesterase inhibitors as first-line therapy. About 6% to 7. 5% of gMG patients have autoantibodies against muscle-specific kinase (MuSK), which is another target on the muscle membrane surface. Patients with MuSK-related MG may not respond to acetylcholinesterase inhibitors and are usually treated with corticosteroids, systemic immunosuppressants, intravenous immunoglobulins, plasmapheresis, and rituximab. However, those strategies require ongoing treatment and have been associated with significant side effects and continued dependence on corticosteroids, highlighting the need for additional effective and safe treatments for patients diagnosed with MuSK-related GD.
About Cabaletta BioCabaletta Bio (Nasdaq: CABA) is a clinical-stage biotechnology company focused on the discovery and progression of engineered T-cell treatments that have the potential to provide a deep and lasting, likely curative treatment for patients with autoimmune diseases. . The CABA™ platform, in combination with Cabaletta Bio’s patented technology, has a complex line of development that lately includes possible remedies for patients with mucous pemphigus vulgaris, MuSK-associated myasthenia gravis, PLA2R-associated membranous nephropathy, mucocutaneous pemphigus vulgaris and hemophilia A with FVIII alloantibodies. Cabaletta Bio is based in Philadelphia, Pennsylvania. For more information, visit www. cabalettabio. com and follow us on LinkedIn.
Forward-Looking StatementsThis press release comprises Cabaletta Bio’s “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, adding, without limitation, explicit or implied statements relating to expectations relating to: expectations relating to the intended incentives conferred through the Fast Track designation for MuSK-CAART for activities of daily living and muscle strength in patients with MuSK antibody myasthenia gravis; the expectation that Cabaletta Bio is likely to result for patients with MuSK MG; plans to initiate patient dosing in an open-label Phase 1 clinical trial to evaluate the protection and tolerance of MuSK-CAART in muSK MG patients in 2022; the ability of MuSK-CAART to target B cells that differentiate into antibody-secreting cells, which produce autoantibodies opposed to the muscle-specific kinase; and the progress and effects of its Phase 1 DesCAARTes™ trial, adding Cabaletta Bio’s ability to enroll the required number of patients, dose each dosing cohort as planned, and advance the trial as planned.
All forward-looking statements contained in this news release are based on management’s existing expectations and ideals regarding long-term events, and are subject to a number of threats and uncertainties that may also cause actual effects to differ. materially and adversely from those stated or implied by such forward-looking statements. These threats and uncertainties include, but are not limited to: Cabaletta’s ability to retain and recognize the intended incentives conferred by the Fast Track designation for MuSK-CAART to improve activities of daily living and muscle strength in patients with myasthenia gravis of MuSK antibodies; the threat that symptoms of biological activity do not indicate long-term results; Cabaletta’s ability to demonstrate sufficient evidence of protection, efficacy, and tolerability in its preclinical and clinical trials of MuSK-CAART; threats similar to the activation of clinical trial sites or decrease in expected enrollment rates; Threats similar to unexpected protection or knowledge of efficacy observed during clinical studies; threats similar to public physical activity outbreaks affecting countries or regions in which Cabaletta operates or does business, such as COVID-19; threats related to Cabaletta’s ability to protect and maintain its intellectual asset position; uncertainties related to the initiation and conduct of studies and other progression needs for its product candidates; and the threat that effects from preclinical studies or clinical studies may not be predictive of long-term effects in long-term studies. For a discussion of those and other threats and uncertainties, and other vital points, each of which may also cause the actual effects of Cabaletta to differ from those contained in the forward-looking statements, see the segment entitled “Risk Factors in the Maximum of Cabaletta”. recent annual report on Form 10-K, as well as discussions of potential threats, uncertainties and other vital points in other Cabaletta filings with the Securities and Exchange Commission. All data contained in this press release is as of the date of publication, and Cabaletta assumes no legal responsibility to update this data, unless required by law.
Contacts:
Anup MardaChief[email protected]
Sarah McCabeStern Investor Relations, Inc. [email protected]
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