Alnylam Pharmaceuticals, Inc. announced that it has presented key pre-clinical proof-of-concept data from its RNAi therapeutic program targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of porphyria including acute intermittent porphyria (AIP). The new research findings were presented at the International Congress of Porphyrins and Porphyrias being held May 16 - 18, 2013 in Lucerne, Switzerland. Specifically, Alnylam scientists and collaborators at the Icahn School of Medicine at Mount Sinai in New York City presented data from pre-clinical models of the human disease showing that RNAi therapeutics targeting ALAS-1 can completely block the abnormal production of toxic intermediates of the heme biosynthesis pathway that cause the symptoms and disease pathology of AIP. Alnylam's AIP drug candidate, ALN-AS1, is part of the company's 'Alnylam 5x15' product development and commercialization strategy, in which the company aims to advance five genetic disease target programs into clinical development, including programs in late stages, by the end of 2015. The new research results support the advancement of RNAi therapeutics as a promising strategy for the prevention and/or treatment of acute attacks in patients with AIP. In the new studies, Alnylam scientists and collaborators at Icahn School of Medicine at Mount Sinai presented findings from a mouse model of AIP. Prophylactic administration of an ALAS-1 specific siRNA completely protected the mice from phenobarbital-induced up-regulation of hepatic ALAS-1 mRNA and the resulting accumulation of the neurotoxic ALA and PBG heme biosynthesis precursors. This protective effect was dose responsive and durable, with a single dose administration resulting in a protective effect that lasted for at least two weeks. Further, in a treatment model, a single dose of ALAS-1 siRNA rapidly reduced the high levels of plasma ALA and PBG that were elevated during a phenobarbital-induced acute attack. Further, preliminary comparative studies show that ALAS-1 siRNA administration was more effective than heme administration in the treatment of an acute attack. Finally, the company presented results from its ongoing GalNAc-siRNA conjugate efforts enabling subcutaneous dose administration. In particular, a prototype GalNAc-siRNA targeting ALAS-1 was shown to be effective in blocking ALA and PBG production in both prophylactic and treatment models of AIP. The company is on track to designate a GalNAc-siRNA development candidate, ALN-AS1, in late 2013 resulting in an investigational new drug (IND) filing in 2014.

Alnylam Pharmaceuticals, Inc. Presents at UBS Global Healthcare Conference, May-22-2013 08:00 AM. Venue: Sheraton New York Hotel, 811 Seventh Avenue, New York, New York, United States. Speakers: Michael P. Mason, Principal Financial Officer, Principal Accounting Officer, Vice President of Finance and Treasurer.

Alnylam Pharmaceuticals, Inc. reported unaudited consolidated earnings results for the first quarter ended March 31, 2013. For the quarter, the company's net revenues from research collaborators were $18,642,000 compared to $20,587,000 in the prior year period. Loss from operations was $99,804,000 against $10,893,000 a year ago. Loss before income taxes was $9,575,000 against $11,368,000 a year ago. Net loss was $9,013,000 or $0.15 per basic and diluted share against $11,368,000 or $0.25 per basic and diluted share a year ago. Interest income was $200,000. The company expects net GAAP revenues from research collaborators to decrease for the remainder of 2013.