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Alnylam Completes Enrollment in Phase III Givosiran Study
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Alnylam Pharmaceuticals Inc. (ALNY - Free Report) announced that it has completed enrollment in the phase III study of its investigational RNA interference (RNAi) therapeutic — givosiran. The candidate is targeting aminolevulinic acid synthase 1 or ALAS1 for the treatment of acute hepatic porphyrias (AHP).
Enrollment in the ENVISION study was completed ahead of schedule with 94 AHP patients, exceeding the target of about 75 patients due to high patient demand.
The ENVISION phase III study is a randomized, placebo-controlled, global, multi-center study to evaluate the efficacy and safety of givosiran in patients with a documented diagnosis of AHPs. Patients were randomized on a 1:1 basis to receive 2.5 mg/kg of givosiran or placebo subcutaneously administered monthly, over a 6-month treatment period.
The primary endpoint is the annualized rate of porphyria attacks requiring hospitalization, urgent healthcare visit or hemin administration at home over the 6-month treatment period. All patients completing the 6-month treatment period are eligible to continue on an open-label extension (OLE) study in which they will receive treatment with givosiran for up to 30 months.
As expected, the company intends to report topline results of the interim analysis by the end of September in support of a potential accelerated approval. The interim analysis is based on lowering of urinary aminolevulinic acid (ALA) levels in approximately 30 patients at three months of treatment as a surrogate biomarker that is reasonably likely to predict clinical benefit. The company expects to report topline results of the primary endpoint of annualized attack rate after six months of treatment in early 2019.
Pending company and FDA review of the program at the time of interim analysis and assuming positive results and acceptable safety, the company continues to expect to submit a new drug application (NDA) at or around year-end 2018, seeking an accelerated approval.
So far this year, shares of Alnylam declined 23.3% compared with the industry’s fall of 5.9%.
We remind investors that earlier this month, the company received the FDA approval for a first-of-its-kind RNAi therapeutic -- Onpattro (patisiran) lipid complex injection -- for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. Onpattro is the first and only FDA-approved treatment for this indication.
Alnylam is also developing other candidates. The company along with its partner The Medicine Company is evaluating inclisiran in phase III ORION studies for hypercholesterolemia. The company is also conducting fitusiran's ATLAS phase III study for the treatment of patients with hemophilia A or B with and without inhibitors. The company expects phase III readouts for fitusiran and inclisiran in 2019, leading to potential approvals in 2020.
Gilead’s earnings per share estimates have increased from $6.15 to $6.57 for 2018 and from $6.33 to $6.48 for 2019 over the past 30 days. The company delivered a positive earnings surprise in three of the trailing four quarters with an average beat of 6.43%. The stock has rallied 2.3% so far this year.
Celgene’s earnings per share estimates have increased from $8.54 to $8.76 for 2018 and from $10.19 to $10.60 for 2019 over the past 30 days. The company delivered a positive earnings surprise in all of the trailing four quarters with an average beat of 2.38%.
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Alnylam Completes Enrollment in Phase III Givosiran Study
Alnylam Pharmaceuticals Inc. (ALNY - Free Report) announced that it has completed enrollment in the phase III study of its investigational RNA interference (RNAi) therapeutic — givosiran. The candidate is targeting aminolevulinic acid synthase 1 or ALAS1 for the treatment of acute hepatic porphyrias (AHP).
Enrollment in the ENVISION study was completed ahead of schedule with 94 AHP patients, exceeding the target of about 75 patients due to high patient demand.
The ENVISION phase III study is a randomized, placebo-controlled, global, multi-center study to evaluate the efficacy and safety of givosiran in patients with a documented diagnosis of AHPs. Patients were randomized on a 1:1 basis to receive 2.5 mg/kg of givosiran or placebo subcutaneously administered monthly, over a 6-month treatment period.
The primary endpoint is the annualized rate of porphyria attacks requiring hospitalization, urgent healthcare visit or hemin administration at home over the 6-month treatment period. All patients completing the 6-month treatment period are eligible to continue on an open-label extension (OLE) study in which they will receive treatment with givosiran for up to 30 months.
As expected, the company intends to report topline results of the interim analysis by the end of September in support of a potential accelerated approval. The interim analysis is based on lowering of urinary aminolevulinic acid (ALA) levels in approximately 30 patients at three months of treatment as a surrogate biomarker that is reasonably likely to predict clinical benefit. The company expects to report topline results of the primary endpoint of annualized attack rate after six months of treatment in early 2019.
Pending company and FDA review of the program at the time of interim analysis and assuming positive results and acceptable safety, the company continues to expect to submit a new drug application (NDA) at or around year-end 2018, seeking an accelerated approval.
So far this year, shares of Alnylam declined 23.3% compared with the industry’s fall of 5.9%.
We remind investors that earlier this month, the company received the FDA approval for a first-of-its-kind RNAi therapeutic -- Onpattro (patisiran) lipid complex injection -- for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. Onpattro is the first and only FDA-approved treatment for this indication.
Alnylam is also developing other candidates. The company along with its partner The Medicine Company is evaluating inclisiran in phase III ORION studies for hypercholesterolemia. The company is also conducting fitusiran's ATLAS phase III study for the treatment of patients with hemophilia A or B with and without inhibitors. The company expects phase III readouts for fitusiran and inclisiran in 2019, leading to potential approvals in 2020.
Alnylam Pharmaceuticals, Inc. Price
Alnylam Pharmaceuticals, Inc. Price | Alnylam Pharmaceuticals, Inc. Quote
Zacks Rank & Stocks to Consider
Alnylam has a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Gilead Sciences Inc. (GILD - Free Report) and Celgene Corp. . While Gilead carries a Zacks Rank #1 (Strong Buy), Celgene carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Gilead’s earnings per share estimates have increased from $6.15 to $6.57 for 2018 and from $6.33 to $6.48 for 2019 over the past 30 days. The company delivered a positive earnings surprise in three of the trailing four quarters with an average beat of 6.43%. The stock has rallied 2.3% so far this year.
Celgene’s earnings per share estimates have increased from $8.54 to $8.76 for 2018 and from $10.19 to $10.60 for 2019 over the past 30 days. The company delivered a positive earnings surprise in all of the trailing four quarters with an average beat of 2.38%.
More Stock News: This Is Bigger than the iPhone!
It could become the mother of all technological revolutions. Apple sold a mere 1 billion iPhones in 10 years but a new breakthrough is expected to generate more than 27 billion devices in just 3 years, creating a $1.7 trillion market.
Zacks has just released a Special Report that spotlights this fast-emerging phenomenon and 6 tickers for taking advantage of it. If you don't buy now, you may kick yourself in 2020.
Click here for the 6 trades >>