To report initial safety, B-cell depletion, and disability outcomes with azer-cel in people with progressive multiple sclerosis.
Approach:
Study: The ongoing Phase I TG-Azercel-101 study enrolled 26 people with primary progressive or secondary progressive MS and recent disability progression despite high-efficacy treatment; all had previously received anti-CD20 therapy.
Treatment and follow-up: Participants received bendamustine lymphodepletion followed by one infusion of 30 million to 500 million donor-derived, CD19-directed CAR T cells. Median follow-up at the September 3 cutoff was 4.9 months.
Key Findings:
Peripheral B-cell counts fell below 10 cells/µL in 25 of 26 participants.
Among 21 participants with paired cerebrospinal fluid samples, median CD19-positive B-cell events declined 52.3% by day 10.
At day 90, disability scores were stable or improved in 16 of 18 evaluable participants; three improved by at least two points. Seven of eight assessed at six months remained stable or improved.
No dose-limiting toxicities, grade 3 or higher cytokine release syndrome, grade 2 or higher immune effector cell-associated neurotoxicity syndrome, graft-versus-host disease, or immune effector cell-associated hemophagocytic syndrome were reported. Two partici…
Interpretation:
These are initial findings from an ongoing Phase I study. TG Therapeutics CEO Michael Weiss described the results as encouraging given participants’ progressive disability despite prior high-efficacy treatment.
Limitations:
The study is ongoing, with a median follow-up of 4.9 months at the reported cutoff.
Disability outcomes were available for 18 participants at day 90 and eight at six months.
The context provides no comparator group or detailed results beyond the initial report.
