- In November 2024, Corvus Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, announced new preclinical data demonstrating the potential of soquelitinib, the company’s lead ITK inhibitor program, to prevent lung damage, inflammation, and pulmonary hypertension associated with systemic sclerosis. The preclinical findings, presented at ACR, confirm the drug’s potential for treating immune-mediated fibrotic diseases such as systemic sclerosis.
- In March 2024, Cabaletta Bio, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CABA-201, an investigational 4-1BB-containing fully human CD19-CAR T cell therapy, for the treatment of systemic sclerosis (SSc). CABA-201 is being developed as a potential therapy for autoimmune diseases driven by B cells.
- In February 2024, Certa Therapeutics (Certa) announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its investigational therapy, FT011, for the treatment of systemic sclerosis (scleroderma), following the earlier Orphan Drug Designation. The Fast-Track Designation was awarded bassed on the results of a previously reported Phase 2 study, which showed that 12 weeks of treatment with FT011 led to a clinically meaningful improvement in 60% of patients receiving the 400mg dose and 20% of patients receiving the 200mg dose, compared to just 10% in the placebo group.
- In October 2023, Kyverna Therapeutics announced that the U.S. Food and Drug Administration (FDA) had cleared its third Investigational New Drug (IND) application for KYV-101. This approval enables Kyverna to launch a Phase 1/2 open-label, multicenter study of KYV-101, an autologous fully human anti-CD19 Chimeric Antigen Receptor (CAR) T cell therapy, for the treatment of diffuse cutaneous systemic sclerosis (scleroderma).
- In February 2023, GSK plc announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Benlysta (belimumab), a monoclonal antibody that inhibits B-cells, for the potential treatment of systemic sclerosis. GSK intends to begin a Phase II/III trial of belimumab for systemic sclerosis-associated interstitial lung disease (SSc-ILD) in the first half of 2023.
