Alnylam Pharmaceuticals, Inc. ALNY declared results from the Global Open-Label Extension (OLE) study of Onpattro (patisiran), an RNAi therapeutic, for the treatment of the polyneuropathy of hereditary ATTR (hATTR) amyloidosis. Results were presented at the 2019 Peripheral Nerve Society (PNS) Annual Meeting. The interim results evaluated Onpattro’s long-term efficacy and safety in eligible patients (N=211), who completed the phase II OLE (N=25) and phase III APOLLO (N=186) studies.
We remind investors that the company’s only approved drug, Onpattro, is a first-of-its-kind RNAi therapeutic for the treatment of the polyneuropathy of hATTR amyloidosis in adults. The drug is the first and only FDA-approved treatment for this indication.
The data presented were as of the cut off date of Sep 24, 2018. As of Sep 24, 2018, patients in the Global OLE study received a mean of 20.5 months of Onpattro, with more than 6,000 doses administered. Serum transthyretin (TTR) levels were reduced about 80% at 6 months in patients in the placebo arm of APOLLO, who started treatment with Onpattro, and were durably maintained over time. Patients on treatment for 30-36 months showed sustained improvement in neuropathy impairment and quality of life relative to corresponding parent study baselines. The safety of Onpattro was consistent with that observed and previously reported in APOLLO and phase II OLE studies, with an encouraging tolerability profile.
Based on its unique mechanism of action with potent knockdown of serum TTR and an encouraging tolerability profile, these long-term data continue to highlight the potential for meaningful clinical benefit with Onpattro treatment
Shares of the company dipped 1.6% year to date against the industry’s growth of 4.6%.
Further, the company also reported new analyses from the APOLLO phase III study of Onpattro, and results of an indirect treatment comparison of Onpattro and inotersen. The indirect treatment comparison revealed favorable treatment effects of the candidate relative to inotersen across all endpoints evaluated.
Further, Alnylamalso announced results on the impact of Onpattro in patients, who received tafamidis (a TTR tetramer stabilizer) treatment prior to enrolling in APOLLO. About one-third of patients enrolled in APOLLO were previously treated with tafamidis. Of these patients, 34% discontinued treatment with tafamidis due to disease progression. Patients with prior tafamidis use, who received Onpattro treatment for 18 months in APOLLO, experienced significant improvement from baseline in polyneuropathy and QOL compared with placebo, similar to that observed in the overall APOLLO population. Additional analyses from APOLLO showed that patients previously treated with tafamidis experienced improvements in both neuropathy impairment and quality of life with Onpattro.
These results highlight the need to avoid any delays in treatment to prevent accumulation of greater disease burden in hATTR amyloidosis patients with polyneuropathy.
Alnylam Pharmaceuticals, Inc. Price
Alnylam Pharmaceuticals, Inc. price | Alnylam Pharmaceuticals, Inc. Quote
Zacks Rank and Stocks to Consider
Alnylam currently has a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Anika Therapeutics Inc. ANIK, Applied Genetics Technologies Corp. AGTC and Acorda Therapeutics Inc. ACOR, each carrying a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Anika’s earnings per share estimates have moved up from $1.21 to $1.31 for 2019 and from $1.21 to $1.33 for 2020 in the past 60 days. The company beat estimates in the trailing four quarters, with the average being 72.00%.
Applied Genetics’ loss per share estimates have narrowed from $1.25 to 1 cent for 2019 and from $2.39 to $2.15 for 2020 in the past 60 days. The company surpassed estimates in three of the trailing four quarters, with average positive surprise of 83.47%.
Acorda’s loss per share estimates have narrowed from $3.84 to $3.59 for 2019 and from $3.32 to $3.09 for 2020 in the past 60 days. The company outpaced estimates in the trailing four quarters, with the average being 79.32%.
Looking for Stocks with Skyrocketing Upside?
Zacks has just released a Special Report on the booming investment opportunities of legal marijuana. Ignited by new referendums and legislation, this industry is expected to blast from an already robust $6.7 billion to $20.2 billion in 2021. Early investors stand to make a killing, but you have to be ready to act and know just where to look.
See the pot trades we're targeting>>
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
Acorda Therapeutics, Inc. (ACOR) : Free Stock Analysis Report
Applied Genetic Technologies Corporation (AGTC) : Free Stock Analysis Report
Alnylam Pharmaceuticals, Inc. (ALNY) : Free Stock Analysis Report
Anika Therapeutics Inc. (ANIK) : Free Stock Analysis Report
To read this article on Zacks.com click here.