02 Nov, 2017 APOLLO Phase 3 Results for Patisiran Presented at EU-ATTR Amyloidosis Meeting
We presented positive complete results from the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic being developed for patients with hereditary ATTR (hATTR) amyloidosis with polyneuropathy, at the 1st European ATTR Amyloidosis Meeting for Patients and Doctors, held November 2-3, 2017 in Paris, France.
Berk et al. – “Long-Term, Open-Label Clinical Experience with Patisiran, an Investigational RNAi Therapeutic for Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy”
- Patisiran met its primary endpoint with a 34.0 point mean difference relative to placebo and a negative 6.0 point mean change (improvement) relative to baseline in modified neuropathy impairment score (mNIS+7) at 18 months.
- Patisiran met all secondary endpoints, including a 21.1 point mean difference relative to placebo and a negative 6.7 point mean change (improvement) relative to baseline in Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN) score at 18 months.
- Significant effects observed on certain exploratory cardiac biomarker and echocardiographic endpoints in pre-specified cardiac subpopulation relative to placebo at 18 months.
- Encouraging safety profile with up to 18 months of dosing. The most commonly reported adverse events that occurred more frequently in patisiran patients were peripheral edema (29.7%) and infusion-related reactions (18.9%).