Alnylam Pharmaceuticals, Inc. ALNY announced positive results from phase I and phase I/II open-label extension (OLE) studies of givosiran, which is being evaluated for the treatment of acute hepatic porphyrias (AHPs).
Shares of the company increased 88.1% against the industry 's decline of 8.5%.
Givosiran is an investigational, subcutaneously administered RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1).
The double-blind, placebo-controlled phase I study showed that compared with a once quarterly dose regimen, monthly dosing led to an over 80% lowering of urinary aminolevulinic acid ("ALA"), a disease biomarker, and an over 75% decrease in mean annualized porphyria attack rate, relative to placebo. Increasing the monthly dose from 2.5 mg/kg to 5.0 mg/kg did not lead to further lowering of ALA and PBG levels.
Monthly dosing regimens led to increased clinical activity as compared to quarterly dosing. A continuous relationship between greater ALA lowering and AAR reduction was also observed.
The study showed enhanced clinical activity with long-term treatment of up to 22 months in the ongoing phase I/II OLE study, with an over 90% decrease in mean annualized porphyria attack rate, relative to baseline run-in attack rate. The extent of these reductions in the OLE study exceeded that observed in response to givosiran treatment during the phase I study, suggesting that extended dosing at 2.5 mg/kg monthly potentially leads to enhanced clinical activity.
Clinical activity and safety results support accelerated efforts to bring givosiran to patients with acute hepatic porphyrias.
The company also announced that that it will continue to enroll patients in the ENVISION phase III pivotal study, which was initiated at the end of 2017. The company also expects to enroll the thirtieth patient into ENVISION in the coming weeks in support of the planned interim analysis in mid-2018, which, if positive, would support a potential new drug application filing for givosiran by end of 2018.
Alnylam Pharmaceuticals, Inc. Price
Alnylam Pharmaceuticals, Inc. Price | Alnylam Pharmaceuticals, Inc. Quote
Zacks Rank and Stocks to Consider
Alnylam carries a Zacks Rank #3 (Hold).
A few better-ranked stocks from the same space worth considering are Ligand Pharmaceuticals LGND , Protagonist Therapeutics PTGX and Catabasis Pharmaceuticals CATB . Whlle Ligand and Protagonist sport a Zacks Rank #1 (Strong Buy), Catabasis carries a Zacks Rank #2 (Buy).You can see the complete list of today's Zacks #1 Rank stocks here .
Ligand's earnings per share estimates have moved up $3.78 to $4.40 from $4.75 to $5.32 for 2018 and 2019, respectively, over the last 60 days. The company delivered a positive earnings surprise in three of the trailing four quarters, with an average beat of 24.88%. The company's shares have rallied 57% over a year.
Protagonist's loss estimates narrowed from $1.30 to 66 cents for 2018 and from $1.99 to $1.26 for 2019, over the last 30 days. The company delivered a positive earnings surprise in three of the trailing four quarters, with an average beat of 24.95%.
Catabasis' loss estimates narrowed from $1.09 to 92 cents for 2018 and from $1.76 to $1.48 for 2019, in the last 30 days. The company came up with a positive earnings surprise in all the preceding four quarters, with an average beat of 14.56%. The stock has rallied 23.9% so far this year.
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Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Alnylam Pharmaceuticals, Inc. (ALNY): Free Stock Analysis Report Ligand Pharmaceuticals Incorporated (LGND): Free Stock Analysis Report Catabasis Pharmaceuticals, Inc. (CATB): Free Stock Analysis Report Protagonist Therapeutics, Inc. (PTGX): Free Stock Analysis Report To read this article on Zacks.com click here. Zacks Investment Research