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December 8, 2013
20:29 EDTALNYAlnylam presents pre-clinical results on ALN-CC5 at ASH
Alnylam Pharmaceuticals announced that it has presented new pre-clinical data with ALN-CC5, a subcutaneously administered RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases. These data were presented at the 55th Annual Meeting of the American Society of Hematology, ASH, held December 7 – 10, in New Orleans, and demonstrate that subcutaneous administration of ALN-CC5 in non-human primates, NHPs, led to an up to 98% knockdown of serum C5 and an up to 94% inhibition of hemolytic activity. Alnylam believes that ALN-CC5 – part of the company’s “Alnylam 5x15” product strategy – represents a novel approach for the treatment of complement-mediated diseases, with a potentially competitive profile compared with intravenously administered anti-C5 monoclonal antibody therapy. In addition, Alnylam presented two separate posters with new pre-clinical data on ALN-TMP, an RNAi therapeutic targeting TMPRSS6 for the treatment of beta-thalassemia and iron-overload disorders.
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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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