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Wired News – Alnylam Pharma Presented New Encouraging Results from Phase-1/2 Study of Lumasiran in Patients with Primary Hyperoxaluria Type-1

Stock Monitor: Cleveland BioLabs Post Earnings Reporting

LONDON, UK / ACCESSWIRE / June 12, 2018 / If you want access to our free research report on Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY), all you need to do is sign up now by clicking the following link www.active-investors.com/registration-sg/?symbol=ALNY as the Company’s latest news hit the wire. On June 08, 2018, the Company presented updated positive results from its Phase-1/2 study assessing lumasiran for the treatment of Primary Hyperoxaluria Type-1 (PH1) at the OxalEurope, European Hyperoxaluria Consortium in Naples, Italy. Register today and get access to over 1,000 Free Research Reports by joining our site below:

www.active-investors.com/registration-sg

Active-Investors.com is currently working on the research report for Cleveland BioLabs, Inc. (NASDAQ: CBLI), which also belongs to the Healthcare sector as the Company Alnylam Pharma. Do not miss out and become a member today for free to access this upcoming report at:

www.active-investors.com/registration-sg/?symbol=CBLI

Active-Investors.com is focused on giving you timely information and the inside line on companies that matter to you. This morning, Alnylam Pharma most recent news is on our radar and our team decided to put out a fantastic report on the company that is now available for free below:

www.active-investors.com/registration-sg/?symbol=ALNY

Lumasiran Demonstrated Mean Maximal Reduction in Urinary Qxalate of 64% in PH1 Patients

Updated interim data were from Part B of the Phase-1/2 study. Cohorts 1 and 2 received three monthly doses of lumasiran at 1 mg/kg or 3 mg/kg, respectively Cohort 3 received two quarterly doses at 3 mg/kg. An additional eight patients received open-label lumasiran in expansions of each of the first two cohorts, totaling 20 patients enrolled. Lumasiran demonstrated a mean maximal reduction in urinary oxalate of 64% in patients enrolled in Cohorts 1-3. On day 85, patients receiving lumasiran maintained a mean reduction in urinary oxalate of 63%. Alnylam believes the potent and durable reductions in urinary oxalate support a once quarterly, subcutaneous dose regimen. Further, these results continue to support the hypothesis that GO inhibition has the potential to reduce and possibly normalize levels of hepatic oxalate production, thus potentially halting PH1 disease progression.

Lumasiran was generally well tolerated in all patients in the Phase-1/2 study (N=20). Majority of adverse events (AEs) were mild or moderate in severity and unrelated to study drug. The placebo patient experienced kidney stones and pyelonephritis. Lumasiran has not been associated with any clinically significant adverse laboratory findings, and there were no study discontinuations due to AEs through the data cut-off date. Dosing in Part B of the Phase-1/2 study is ongoing and eligible patients are transitioning into an open-label extension (OLE) study. The Company expects to present additional data from all cohorts as well as from the OLE study in late 2018.

Alnylam’s Alignment with the FDA on a Pivotal Study Design for Lumasiran

On May 03, 2018, the Company reached alignment with the US Food and Drug Administration (FDA) on a pivotal study design for lumasiran. The Company and the FDA aligned on a primary endpoint for the pivotal study based on reduction of urinary oxalate at six months. In addition, Alnylam and the FDA aligned on a study size of approximately 25 patients with PH1. Based on the discussions with the FDA, the Company is on track to start the Phase-3 study in mid-2018 and it expects to report topline results in 2019 and, if positive, to submit an NDA in early 2020.

Lumasiran Received European Medicines Agency PRIME Designation for Accelerated Assessment of Lumasiran

On March 26, 2018, the European Medicines Agency (EMA) granted access to its Priority Medicines (PRIME) scheme for lumasiran for the treatment of PH1. The purpose of the PRIME initiative is to bring treatments to patients faster by enhancing the EMA's support for the development of medicines for diseases where there is an unmet medical need and where early clinical data show potential to benefit patients. Lumasiran was also granted Breakthrough Therapy Designation by the FDA.

About Primary Hyperoxaluria Type-1 (PH1)

PH1 is a rare disorder that mainly affects the kidneys. It results from buildup of a substance called oxalate, which normally is filtered through the kidneys and excreted in the urine. In people with PH1, the accumulated oxalate is deposited in the kidneys and urinary tract. It combines with calcium, forming the main component of kidney and bladder stones. Symptoms of PH1 may include recurrent kidney stones; blood in the urine; and urinary tract infections. Left untreated, PH1 can result in end-stage renal disease, which is life-threatening.

About Lumasiran

Lumasiran (formerly known as ALN-GO1) is an investigational RNAi therapeutic targeting glycolate oxidase (GO) in development for the treatment of PH1. Lumasiran is designed to reduce the hepatic levels of the GO enzyme, thereby depleting the substrate necessary for oxalate production, which directly contributes to the pathophysiology of PH1. Lumasiran utilizes Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index.

About Alnylam Pharmaceuticals, Inc.

Founded in 2002 and headquartered in Cambridge, Massachusetts, Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference (RNAi). The Company is leading the translation of RNAi as a new class of innovative medicines with a core focus on RNAi therapeutics in three strategic therapeutic areas (STArs): genetic medicines, cardio-metabolic diseases, and hepatic infectious diseases.

Stock Performance Snapshot

June 11, 2018 - At Monday’s closing bell, Alnylam Pharma’s stock marginally fell 0.19%, ending the trading session at $100.05.

Volume traded for the day: 383.15 thousand shares.

Stock performance in the last month – up 1.80% and past twelve-month period – up 37.37%

After yesterday’s close, Alnylam Pharma’s market cap was at $10.20 billion.

The stock is part of the Healthcare sector, categorized under the Biotechnology industry. This sector was up 0.1% at the end of the session.

Stock Monitor: Cleveland BioLabs Post Earnings Reporting

LONDON, UK / ACCESSWIRE / June 12, 2018 / If you want access to our free research report on Alnylam Pharmaceuticals, Inc. (ALNY), all you need to do is sign up now by clicking the following link www.active-investors.com/registration-sg/?symbol=ALNY as the Company’s latest news hit the wire. On June 08, 2018, the Company presented updated positive results from its Phase-1/2 study assessing lumasiran for the treatment of Primary Hyperoxaluria Type-1 (PH1) at the OxalEurope, European Hyperoxaluria Consortium in Naples, Italy. Register today and get access to over 1,000 Free Research Reports by joining our site below:

www.active-investors.com/registration-sg

Active-Investors.com is currently working on the research report for Cleveland BioLabs, Inc. (CBLI), which also belongs to the Healthcare sector as the Company Alnylam Pharma. Do not miss out and become a member today for free to access this upcoming report at:

www.active-investors.com/registration-sg/?symbol=CBLI

Active-Investors.com is focused on giving you timely information and the inside line on companies that matter to you. This morning, Alnylam Pharma most recent news is on our radar and our team decided to put out a fantastic report on the company that is now available for free below:

www.active-investors.com/registration-sg/?symbol=ALNY

Lumasiran Demonstrated Mean Maximal Reduction in Urinary Qxalate of 64% in PH1 Patients

Updated interim data were from Part B of the Phase-1/2 study. Cohorts 1 and 2 received three monthly doses of lumasiran at 1 mg/kg or 3 mg/kg, respectively; Cohort 3 received two quarterly doses at 3 mg/kg. An additional eight patients received open-label lumasiran in expansions of each of the first two cohorts, totaling 20 patients enrolled. Lumasiran demonstrated a mean maximal reduction in urinary oxalate of 64% in patients enrolled in Cohorts 1-3. On day 85, patients receiving lumasiran maintained a mean reduction in urinary oxalate of 63%. Alnylam believes the potent and durable reductions in urinary oxalate support a once quarterly, subcutaneous dose regimen. Further, these results continue to support the hypothesis that GO inhibition has the potential to reduce and possibly normalize levels of hepatic oxalate production, thus potentially halting PH1 disease progression.

Lumasiran was generally well tolerated in all patients in the Phase-1/2 study (N=20). Majority of adverse events (AEs) were mild or moderate in severity and unrelated to study drug. The placebo patient experienced kidney stones and pyelonephritis. Lumasiran has not been associated with any clinically significant adverse laboratory findings, and there were no study discontinuations due to AEs through the data cut-off date. Dosing in Part B of the Phase-1/2 study is ongoing and eligible patients are transitioning into an open-label extension (OLE) study. The Company expects to present additional data from all cohorts as well as from the OLE study in late 2018.

Alnylam’s Alignment with the FDA on a Pivotal Study Design for Lumasiran

On May 03, 2018, the Company reached alignment with the US Food and Drug Administration (FDA) on a pivotal study design for lumasiran. The Company and the FDA aligned on a primary endpoint for the pivotal study based on reduction of urinary oxalate at six months. In addition, Alnylam and the FDA aligned on a study size of approximately 25 patients with PH1. Based on the discussions with the FDA, the Company is on track to start the Phase-3 study in mid-2018 and it expects to report topline results in 2019 and, if positive, to submit an NDA in early 2020.

Lumasiran Received European Medicines Agency PRIME Designation for Accelerated Assessment of Lumasiran

On March 26, 2018, the European Medicines Agency (EMA) granted access to its Priority Medicines (PRIME) scheme for lumasiran for the treatment of PH1. The purpose of the PRIME initiative is to bring treatments to patients faster by enhancing the EMA's support for the development of medicines for diseases where there is an unmet medical need and where early clinical data show potential to benefit patients. Lumasiran was also granted Breakthrough Therapy Designation by the FDA.

About Primary Hyperoxaluria Type-1 (PH1)

PH1 is a rare disorder that mainly affects the kidneys. It results from buildup of a substance called oxalate, which normally is filtered through the kidneys and excreted in the urine. In people with PH1, the accumulated oxalate is deposited in the kidneys and urinary tract. It combines with calcium, forming the main component of kidney and bladder stones. Symptoms of PH1 may include recurrent kidney stones; blood in the urine; and urinary tract infections. Left untreated, PH1 can result in end-stage renal disease, which is life-threatening.

About Lumasiran

Lumasiran (formerly known as ALN-GO1) is an investigational RNAi therapeutic targeting glycolate oxidase (GO) in development for the treatment of PH1. Lumasiran is designed to reduce the hepatic levels of the GO enzyme, thereby depleting the substrate necessary for oxalate production, which directly contributes to the pathophysiology of PH1. Lumasiran utilizes Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index.

About Alnylam Pharmaceuticals, Inc.

Founded in 2002 and headquartered in Cambridge, Massachusetts, Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference (RNAi). The Company is leading the translation of RNAi as a new class of innovative medicines with a core focus on RNAi therapeutics in three strategic therapeutic areas (STArs): genetic medicines, cardio-metabolic diseases, and hepatic infectious diseases.

Stock Performance Snapshot

June 11, 2018 - At Monday’s closing bell, Alnylam Pharma’s stock marginally fell 0.19%, ending the trading session at $100.05.

Volume traded for the day: 383.15 thousand shares.

Stock performance in the last month – up 1.80%; and past twelve-month period – up 37.37%

After yesterday’s close, Alnylam Pharma’s market cap was at $10.20 billion.

The stock is part of the Healthcare sector, categorized under the Biotechnology industry. This sector was up 0.1% at the end of the session.

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