STAT+: Arrowhead RNA treatment improves liver scarring in patients, but so does a placebo, clouding study results

An experimental RNA treatment reduced liver scarring in half of patients with an inherited disease called alpha-1 antitrypsin deficiency, or AATD, according to results from a mid-stage clinical trial reported Monday by its maker Arrowhead Pharmaceuticals.

The improvement in liver fibrosis demonstrated by the treatment, called fazirsiran, was generally in line with expectations based on previous data. However, 38% of patients treated with a placebo also showed the same liver-fibrosis improvement — a response that was higher than expected, making it more difficult to discern fazirisan’s benefit.

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