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May 13, 2014
08:01 EDTALNYAlnylam announces results from ongoing phase 1 trial of ALN-AT3
Alnylam Pharmaceuticals announced top-line results from its ongoing Phase 1 trial of ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin in development for the treatment of hemophilia and rare bleeding disorders. These top-line results are being presented at the World Federation of Hemophilia 2014 World Congress being held May 11 – 15, 2014 in Melbourne, Australia. In Part A of the Phase 1 study, human volunteer subjects received a single subcutaneous dose of ALN-AT3 and, per protocol, the maximum allowable level of AT knockdown was set at 40%. Initial results show that a single, low subcutaneous dose of ALN-AT3 at 0.03 mg/kg resulted in an up to 28-32% knockdown of AT at nadir that was statistically significant relative to placebo. This led to a statistically significant increase in peak thrombin generation, that was temporally associated and consistent with the degree of AT knockdown. ALN-AT3 was found to be well tolerated with no significant adverse events reported. With these data, the company has transitioned to the Multiple Ascending Dose Part B of the study in moderate-to-severe hemophilia subjects. Consistent with previous guidance, the company plans to present initial clinical results from the Phase 1 study, including results in hemophilia subjects, by the end of the year. These human study results are the first to be reported for Alnylam’s Enhanced Stabilization Chemistry-GalNAc conjugate technology, which enables subcutaneous dosing with increased potency, durability, and a wide therapeutic index. Further, these initial clinical results demonstrate a greater than 50-fold potency improvement with ESC-GalNAc conjugates relative to standard template chemistry conjugates.
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July 28, 2015
07:25 EDTALNYAlnylam Pharmaceuticals to hold a roundtable
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July 27, 2015
08:05 EDTALNYAlnylam initiates Phase 1/2 clinical trial for ALN-AAT
Alnylam Pharmaceuticals (ALNY) has initiated a Phase 1/2 clinical trial with ALN-AAT, a subcutaneously administered investigational RNAi therapeutic targeting alpha-1 antitrypsin for the treatment of AAT deficiency-associated liver disease. The Phase 1/2 trial will be conducted initially in normal healthy volunteers, and, then, in patients with alpha-1 liver disease. Initiation of this trial is based on encouraging pre-clinical data presented at the Digestive Disease Week meeting May 16 – 19, 2015. The company expects to present initial clinical data from this trial in early 2016. ALN-AAT is a subcutaneously administered investigational RNAi therapeutic that utilizes Alnylam’s proprietary ESC-GalNAc-siRNA conjugate delivery technology. ESC-GalNAc-siRNA conjugates are designed to achieve targeted delivery of RNAi therapeutics to hepatocytes through uptake by the asialoglycoprotein receptor, and enable subcutaneous dosing with increased potency and durability and a wide therapeutic index. As per the filed CTA, the Phase 1/2 trial of ALN-AAT is a randomized, single-blind, placebo-controlled study being conducted in three parts. Parts A and B are single-dose and multi-dose, dose-escalation studies, designed to enroll up to a total of 48 healthy adult volunteers. Part C will be a multi-dose study designed to enroll up to a total of 24 adults with alpha-1 liver disease and mild-to-moderate liver fibrosis. The primary objective of the study is to evaluate safety and tolerability of single and multiple subcutaneous doses of ALN-AAT. Secondary objectives include evaluation of pharmacokinetics and clinical activity for ALN-AAT as measured by knockdown of serum AAT. In addition, biopsies will be obtained from subjects with alpha-1 liver disease to quantify the effects of treatment on levels of periodic acid-Schiff-stained globules, a measure of misfolded AAT accumulation observed in the livers of alpha-1 liver disease patients. In January 2014, Alnylam and Genzyme, a Sanofi (SNY) company, formed an alliance to accelerate and expand the development and commercialization of RNAi therapeutics across the world. The alliance is structured as a multi-product geographic alliance in the field of rare diseases. Alnylam retains product rights in North America and Western Europe, while Genzyme obtained the right to access certain programs in Alnylam's current and future Genetic Medicines pipeline, including ALN-AAT, in the rest of the world. In certain defined instances, Genzyme has co-development/co-commercialization and/or global product rights. Genzyme's rights are structured as an opt-in that is triggered upon achievement of human proof-of-principle.
July 23, 2015
07:29 EDTALNYAlnylam to hold a roundtable
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July 20, 2015
08:05 EDTALNYAlnylam initiates Phase 3 open label extension study with patisiran
Alnylam announced that it has initiated its Phase 3 open-label extension study, abbreviated APOLLO-OLE, with patisiran, an investigational RNAi therapeutic targeting transthyretin for the treatment of TTR-mediated amyloidosis in patients with Familial Amyloidotic Polyneuropthy. All patients who complete the APOLLO Phase 3 trial with patisiran are eligible to enroll in APOLLO-OLE. The study will evaluate the long-term safety and efficacy of patisiran and will also measure effects of treatment toward a number of clinical endpoints, including the modified Neuropathy Impairment Score, or “mNIS+7,” which is an evaluation of muscle weakness, sensory and autonomic function, and nerve conductance. The company is also reiterating its previous guidance that – assuming positive study results in the APOLLO Phase 3 study – it expects to be in a position to file a New Drug Application for patisiran in the 2017 timeframe.

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