July 2018

23 Jul New Analyses of Clinical Results from APOLLO Phase 3 Study of Patisiran at PNS Annual Meeting

We presented new analyses from the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic for the treatment of hereditary ATTR (hATTR) amyloidosis, at the 2018 Peripheral Nerve Society (PNS) Annual Meeting, held July 22-25, 2018 in Baltimore, MD. Read our press release Ajroud-Driss et al. - "Impact of Patisiran on Overall Health Status in hATTR Amyloidosis: Results from the APOLLO Trial" Polydefkis et al. - "Infusion Related Reactions in Patients with hATTR Amyloidosis Treated with Patisiran" Dyck et al. - "Neuropathy Progression in Patients with hATTR Amyloidosis: Analysis of the APOLLO Placebo Arm" Coelho et al. - "Longitudinal Changes in mNIS+7 are Associated with Changes in Ambulatory Status in Hereditary Transthyretin-Mediated Amyloidosis" Coelho et al. - "Indirect Comparison of Patisiran and Tafamidis for Treatment of Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy" Gonzalez-Duarte et al. - "Changes in Neuropathy Stage in Patients with Hereditary Transthyretin-Mediated Amyloidosis Following Treatment with Patisiran, an Investigational RNAi Therapeutic: An Analysis from the Phase 3 APOLLO Study"

08 Jul New Clinical Results from APOLLO Phase 3 Study of Patisiran at ICNMD

We presented new results from the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic for the treatment of hereditary ATTR (hATTR) amyloidosis, at the International Congress on Neuromuscular Diseases (ICNMD), held July 6-10, 2018 in Vienna, Austria. Obici et al. – “APOLLO Phase 3...

04 Jul APOLLO Phase 3 Results for Patisiran Published in “The New England Journal of Medicine”

We have published results from the APOLLO Phase 3 trial of patisiran in The New England Journal of Medicine in a paper titled, “Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis.” Read the paper in The New England Journal of Medicine

16 Jun New Clinical Results for Patisiran at EAN

We presented additional results from the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic for the treatment of hereditary ATTR (hATTR) amyloidosis, at the 4th Congress of the European Academy of Neurology (EAN), held June 16-19, 2018 in Lisbon, Portugal. Coelho et al. –...

08 Jun New Positive Clinical Results for Lumasiran at OxalEurope

We presented new positive results from the Phase 1/2 study with lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of Primary Hyperoxaluria Type 1 (PH1), at the OxalEurope, European Hyperoxaluria Consortium, held June 8, 2018 in Naples, Italy. Read our press release View the presentation

29 May New Clinical Results from APOLLO Phase 3 Study of Patisiran at ESC-HF

We presented new results from the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic for the treatment of hereditary ATTR (hATTR) amyloidosis, at the European Society of Cardiology Heart Failure 2018 Congress, held May 26-29, 2018 in Vienna, Austria. Slama et al. - "Analysis of NT-proBNP Baseline Levels in APOLLO as a Predictor of Survival in Hereditary Transthyretin-Mediated (hATTR) Amyloidosis" Merlini et al. - "Impact of Patisiran on Norfolk Quality of Life Questionairre Diabetic Neuropathy in Patients with Hereditary Transthyretin-Mediated Amyloidosis: Results from the Cardiac Subpopulation in the Phase 3 APOLLO Study"

17 May Nonclinical Research Published in Nature Biotechnology Describing REVERSIR Platform for Potent and Rapid Reversal of siRNA Activity for Tailored Control of RNAi Pharmacology

One of the main features of our ESC GalNAc-siRNA conjugates platform is potent and durable target knockdown in the liver sustained for several months in humans. In some cases, RNAi therapeutics may benefit from a technology that enables reversal of target mRNA knockdown and provides finer control over pharmacology, a desired attribute for therapeutic entities. REVERSIR^TM molecules are GalNAc-conjugated, short, single-stranded, high affinity oligonucleotide constructs designed to recognize and bind to the complementary, RISC-bound antisense strand of siRNAs, thereby leading to rapid and complete reversal of RNAi-mediated target gene silencing activity in vivo. Read article in Nature Biotech