Cabaletta Bio, Inc. has recently gained Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for its investigational therapy, CABA-201, aimed at treating systemic sclerosis (SSc), a rare autoimmune disease characterized by progressive skin and internal organ fibrosis. This designation acknowledges the significance of CABA-201's potential in addressing a disease that affects a limited number of individuals in the United States, providing Cabaletta with certain incentives such as tax credits for clinical trial expenses and waived user fees, as well as the possibility of marketing exclusivity for seven years if the therapy is approved.

Systemic sclerosis poses significant challenges for patients, with current treatment options offering only modest effects and focusing on managing complications associated with the disease rather than addressing its root cause. CABA-201, a fully human CD19-CAR T cell therapy containing 4-1BB, holds promise in transforming the treatment landscape for SSc by targeting autoimmune diseases driven by B cells.

Cabaletta's RESET-SSc™ trial, a Phase 1/2 open-label study of CABA-201, aims to evaluate its efficacy in treating SSc. The trial comprises two cohorts focusing on severe skin involvement and organ complications, respectively. Participants will undergo a single infusion of CABA-201 following a standard preconditioning regimen. The trial seeks to assess the therapy's ability to transiently eliminate B cells, potentially leading to durable remissions and resetting of the immune system.

CABA-201's innovative design aims to deplete CD19-positive B cells transiently, offering the possibility of long-term remission without continuous therapy. The FDA has cleared Investigational New Drug (IND) applications for CABA-201 in multiple autoimmune conditions, highlighting its potential in addressing various autoimmune diseases beyond SSc.

Systemic sclerosis affects approximately 88,000 individuals in the U.S., primarily middle-aged women, and is associated with life-threatening complications such as interstitial lung disease and pulmonary hypertension. Given the limited treatment options and the urgent need for more effective therapies, CABA-201 represents a promising avenue for improving outcomes and quality of life for patients with SSc.

 

Source: globenewswire.com

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