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Alnylam (ALNY) Reports Positive Phase I Data on ALN-AS1
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Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) announced positive interim results from an ongoing phase I study, evaluating its pipeline candidate, ALN-AS1, an investigational RNAi therapeutic, for the treatment of acute hepatic porphyrias (enzyme deficiency in liver).
The phase I study is being carried out in three parts – parts A, B and C – on asymptomatic high excreter (ASHE) patients, who carry the genetic mutation of acute intermittent porphyria (AIP) and have elevated levels of aminolevulinic acid (ALA) and porphobilinogen (PBG) – the toxic heme intermediates that mediate porphyria attacks.
The new interim data, which demonstrated encouraging results, were from parts A and B of the ongoing phase I study. Subcutaneous administration of the drug showed durable lowering of ALA and PBG up to 95%. ALN-AS1 was also found to be generally well tolerated in the study.
Results were presented during an oral presentation at the 2016 Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium, being held from Sep 6–9 in Rome, Italy.
ALN-AS1 has been granted Orphan Drug designation by the FDA for the treatment of acute hepatic porphyrias.
Part C of the phase I study is presently ongoing in symptomatic AIP (acute intermittent porphyria) patients with recurrent porphyria attacks. Part C results are expected to be presented in late 2016.
We note that Alnylam has a collaboration agreement with Genzyme, a Sanofi (SNY) company, for ALN-AS1.
Other players in the acute hepatic porphyries treatment market include Panhematin and Normosang.
Alnylam is a development-stage biopharmaceutical company focused on the development of RNAi-based therapeutics. By the end of 2020, Alnylam expects to achieve a company profile with three marketed products, 10 RNAi therapeutic clinical programs including four in late stages of development – across its three STArs that include genetic medicines, cardio-metabolic disease and hepatic infectious disease.
Alnylam Pharmaceuticals currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in medical genetics sector include ANI Pharmaceuticals, Inc. (ANIP - Free Report) , Anika Therapeutics Inc. (ANIK - Free Report) and Cambrex Corporation . Each of these stocks sports a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
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Alnylam (ALNY) Reports Positive Phase I Data on ALN-AS1
Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) announced positive interim results from an ongoing phase I study, evaluating its pipeline candidate, ALN-AS1, an investigational RNAi therapeutic, for the treatment of acute hepatic porphyrias (enzyme deficiency in liver).
The phase I study is being carried out in three parts – parts A, B and C – on asymptomatic high excreter (ASHE) patients, who carry the genetic mutation of acute intermittent porphyria (AIP) and have elevated levels of aminolevulinic acid (ALA) and porphobilinogen (PBG) – the toxic heme intermediates that mediate porphyria attacks.
The new interim data, which demonstrated encouraging results, were from parts A and B of the ongoing phase I study. Subcutaneous administration of the drug showed durable lowering of ALA and PBG up to 95%. ALN-AS1 was also found to be generally well tolerated in the study.
Results were presented during an oral presentation at the 2016 Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium, being held from Sep 6–9 in Rome, Italy.
ALN-AS1 has been granted Orphan Drug designation by the FDA for the treatment of acute hepatic porphyrias.
Part C of the phase I study is presently ongoing in symptomatic AIP (acute intermittent porphyria) patients with recurrent porphyria attacks. Part C results are expected to be presented in late 2016.
We note that Alnylam has a collaboration agreement with Genzyme, a Sanofi (SNY) company, for ALN-AS1.
Other players in the acute hepatic porphyries treatment market include Panhematin and Normosang.
Alnylam is a development-stage biopharmaceutical company focused on the development of RNAi-based therapeutics. By the end of 2020, Alnylam expects to achieve a company profile with three marketed products, 10 RNAi therapeutic clinical programs including four in late stages of development – across its three STArs that include genetic medicines, cardio-metabolic disease and hepatic infectious disease.
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Zacks Rank & Key Picks
Alnylam Pharmaceuticals currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in medical genetics sector include ANI Pharmaceuticals, Inc. (ANIP - Free Report) , Anika Therapeutics Inc. (ANIK - Free Report) and Cambrex Corporation . Each of these stocks sports a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Confidential from Zacks
Beyond this Analyst Blog, would you like to see Zacks' best recommendations that are not available to the public? Our Executive VP, Steve Reitmeister, knows when key trades are about to be triggered and which of our experts has the hottest hand.Click to see them now>>