- Prothena (NASDAQ:PRTA) reports 9-month results from long-term extension portion of the Phase 1 study evaluating PRX004, an anti-amyloid immunotherapy for ATTR amyloidosis, a rare inherited disorder characterized by the buildup of amyloid protein in tissues and organs.
- Results included slowing of neuropathy progression for all seven evaluable patients, evidenced by a +1.29 point mean change in neuropathy impairment score, was more favorable than expected progression of +9.2 points in untreated and placebo-treated patients. 3 of 7 patients demonstrated improvement with a mean change in score of –3.33 points at 9 months.
- PRX004 also demonstrated improvement in cardiac systolic function for all 7 evaluable patients, with a mean change in GLS, a sensitive marker that can detect subtle myocardial dysfunction, of –1.21% at 9 months.
- Shares halted premarket
- https://seekingalpha.com/news/3642865-prothenas-immunotherapy-show-slowing-neuropathy-progression-in-long-term-amyloidosis-study
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