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Alnylam to Present New Clinical Results at 58th Annual Meeting of the American Society of Hematology (ASH)
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11-3-16 9:00 AM EDT | Email Article
Alnylam to Present New Clinical Results at 58th Annual Meeting of the American Society of Hematology (ASH)


– New Data to be Presented from Clinical Studies with Fitusiran for the Treatment of Hemophilia, ALN-AS1 for the Treatment of Acute Hepatic Porphyrias, and ALN-CC5 for the Treatment of Complement-Mediated Diseases –

Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics company, today announced that Alnylam scientists and collaborators will present new results from clinical studies of three of its investigational RNAi therapeutics – fitusiran, ALN-AS1 and ALN-CC5 – at the 58th Annual Meeting of the American Society of Hematology (ASH) being held December 3-6, 2016 in San Diego, California.

“The data being presented at ASH demonstrate our commitment to the development of novel treatment options for patients living with rare hematologic diseases, an area of high unmet need,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “We look forward to presenting updated safety and clinical activity data from studies of fitusiran and ALN-CC5 and interim data from the ALN-AS1 phase 1 study in acute hepatic porphyria patients with recurrent porphyria attacks, for which there are limited treatment options.”

Presentations include:

Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results from a Phase 1 and Phase 1/2 Extension Study in Patients with Inhibitors
Author: K. John Pasi, Royal London Haemophilia Centre
Session: 322. Disorders of Coagulation or Fibrinolysis: Poster I
Date/Time: Saturday, December 3, 5:30 – 7:30 p.m. PT
Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results from a Phase 1 and Phase 1/2 Extension Study in Patients without Inhibitors
Author: Margaret V. Ragni, University of Pittsburgh and Hemophilia Center of Western Pennsylvania
Session: 322. Disorders of Coagulation or Fibrinolysis: Poster II
Date/Time: Sunday, December 4, 6:00 – 8:00 p.m. PT
Interim Data from a Randomized, Placebo Controlled, Phase 1 Study of ALN-AS1, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyria
Author: Eliane Sardh, Karolinska University Hospital
Session: 801. Gene Therapy and Transfer: Poster I
Date/Time: Saturday, December 3, 5:30 – 7:30 p.m. PT
A Subcutaneously Administered Investigational RNAi Therapeutic (ALN-CC5) Targeting Complement C5 for Treatment of PNH and Complement-Mediated Diseases: Preliminary Phase 1/2 Study Results in Patients with PNH
Author: Anita Hill, University of Leeds
Session: 508. Bone Marrow Failure: Poster III
Date/Time: Monday, December 5, 6:00 – 8:00 p.m. PT
About Fitusiran
Fitusiran is a subcutaneously administered, investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia A and B and rare bleeding disorders (RBD) currently in early stage clinical development. Fitusiran is designed to lower levels of AT with the goal of promoting sufficient thrombin generation to restore hemostasis and prevent bleeding in patients with hemophilia and RBD. AT, also known as "antithrombin III" and "SERPINC1" is a liver-expressed plasma protein and member of the "serpin" family of proteins that acts by inactivating thrombin and other coagulation factors. AT plays a key role in normal hemostasis by helping to limit the process of fibrin clot formation. However, in hemophilia, insufficient thrombin generation results in impaired fibrin clot formation. Lowering AT in the hemophilia setting may promote the generation of sufficient levels of thrombin needed to form an effective fibrin clot and prevent bleeding. This rationale is supported by human genetic data suggesting that co-inheritance of thrombophilic mutations, including AT deficiency, may ameliorate bleeding in hemophilia. Lowering of AT is a unique and innovative strategy for restoring hemostasis in people with hemophilia. Fitusiran utilizes Alnylam's ESC-GalNAc conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index.

About ALN-AS1
ALN-AS1 is a subcutaneously administered, investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias, including acute intermittent porphyria (AIP), currently in early stage clinical development. AIP is an ultra-rare autosomal dominant disease caused by loss of function mutations in porphobilinogen deaminase (PBGD), an enzyme in the heme biosynthesis pathway that can result in accumulation of toxic heme intermediates, including aminolevulinic acid (ALA) and porphobilinogen (PBG). Patients with AIP can suffer from acute and/or recurrent life-threatening attacks characterized by severe abdominal pain, neuropathy (affecting the central, peripheral or autonomic nervous system), and neuropsychiatric manifestations. ALN-AS1 is an ESC-GalNAc-siRNA conjugate targeting ALAS1, a liver-expressed, rate-limiting enzyme upstream of PBGD in the heme biosynthesis pathway. Inhibition of ALAS1 is known to reduce the accumulation of heme intermediates that cause the clinical manifestations of AIP. ALN-AS1 has the potential to be a prophylactic approach for the prevention of recurrent attacks, as well as for the treatment of acute porphyria attacks.

About ALN-CC5
ALN-CC5 is a subcutaneously administered, investigational RNAi therapeutic targeting the C5 component of the complement pathway, currently in early stage clinical development for the treatment of complement-mediated diseases. The complement system plays a central role in immunity as a protective mechanism for host defense, but its dysregulation results in life-threatening complications in a broad range of human diseases including paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic-uremic syndrome (aHUS), myasthenia gravis, neuromyelitis optica, and membranous nephropathy, amongst others. Complement component C5, which is predominantly expressed in liver cells, is a genetically and clinically validated target; loss-of-function human mutations are associated with an attenuated immune response against certain infections and intravenous anti-C5 monoclonal antibody (mAb) therapy has demonstrated clinical activity and tolerability in a number of complement-mediated diseases. A subcutaneously administered RNAi therapeutic that silences C5 represents a novel approach to the treatment of complement-mediated diseases. ALN-CC5 utilizes Alnylam's ESC-GalNAc conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index.

The safety and efficacy of fitusiran, ALN-AS1, and ALN-CC5 have not been evaluated by the U.S. Food and Drug Administration or any other health authority.

Sanofi Genzyme Alliance
In January 2014, Alnylam and Sanofi Genzyme, the specialty care global business unit of Sanofi, 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 Sanofi Genzyme obtained the right to access certain programs in Alnylam's current and future Genetic Medicines pipeline in the rest of the world (ROW) through the end of 2019, together with certain broader co-development/co-commercialization rights and global rights for certain products. In the case of fitusiran, Sanofi Genzyme has elected to opt into the program for its ROW rights, while retaining its further opt-in right to co-develop and co-promote fitusiran with Alnylam in North America and Western Europe, subject to certain restrictions.

About RNAi
RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today which was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.
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