Regeneron Pharmaceuticals (NASDAQ:REGN) announces results from a Phase 2 clinical trial, LUMINA-1, evaluating garetosmab (REGN2477) in patients with fibrodysplasia ossificans progressiva (FOP), a rare inherited disorder in which muscle and connective tissue are gradually replaced by bone (ossification).
After 28 weeks of treatment with the Activin A inhibitor, total lesion activity dropped by 25% as measure by PET bone scans (results were not statistically significant – p=0.07). The number of new lesions decreased ~90% compared to placebo. There was a 25% relative decrease in bone lesion volume (new and existing) as measured by CT scan (also not statistically significant – p=0.37) and almost a 90% drop in new bone lesions as measured by CT. Patient-reported flare-ups decreased by 50% (p=0.03).
On the safety front, 100% of patients in both the treatment and placebo arms experienced treatment-emergent adverse events, most mild or moderate. Garetosmab-related imbalances were noted in epistaxis (acute bleeding from the nose or nasopharynx) (50.0% vs. 16.7%), skin events (madarosis [loss of eyebrows, 25.0% vs 0%], acne [30.0% vs 8.3%] and a composite of skin infections including abscess, carbuncle, folliculitis, furuncle).
Activin A is a protein that plays a key role in the development of FOP.
Detailed data will be submitted for presentation at a future medical conference and will be the basis for regulatory filings.
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