Alnylam To Present New Clinical Results At 58th Annual Meeting Of The American Society Of Hematology (ASH)

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 InhibitorsAuthor: K. John Pasi, Royal London Haemophilia CentreSession: 322. Disorders of Coagulation or Fibrinolysis: Poster IDate/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 InhibitorsAuthor: Margaret V. Ragni, University of Pittsburgh and Hemophilia Center of Western PennsylvaniaSession: 322. Disorders of Coagulation or Fibrinolysis: Poster IIDate/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 PorphyriaAuthor: Eliane Sardh, Karolinska University HospitalSession: 801. Gene Therapy and Transfer: Poster IDate/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 PNHAuthor: Anita Hill, University of LeedsSession: 508. Bone Marrow Failure: Poster IIIDate/Time: Monday, December 5, 6:00 - 8:00 p.m. PT

About FitusiranFitusiran 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-AS1ALN-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-CC5ALN-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.

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