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| Press Release

Wired News – Alnylam Retains Global Rights for its Investigational RNAi Therapeutic Lumasiran

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LONDON, UK / ACCESSWIRE / March 14, 2018 / has just released a free research report on Alnylam Pharma, Inc. (NASDAQ: ALNY). If you want access to this report all you need to do is sign up now by clicking the following link as the Company's latest news hit the wire. On March 12, 2018, the Company, which is a leading RNA interference (RNAi) therapeutics organization, declared that Sanofi Genzyme has declined its opt-in for the development and commercialization of Lumasiran (ALN-GO1), which is Alnylam's investigational RNAi therapeutic for the treatment of Primary Hyperoxaluria Type 1 (PH1). Consequently, Alnylam has decided to rapidly advance Lumasiran into a Phase-3 pivotal study in the later part of 2018. Register today and get access to over 1000 Free Research Reports by joining our site below: is currently working on the research report for Dicerna Pharmaceuticals, Inc. (NASDAQ: DRNA), 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: 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:

About Primary Hyperoxaluria Type 1 (PH1)

PH1 is an ultra-orphan disease, wherein extra production of oxalate leads to the deposition of calcium oxalate crystals in the kidneys and urinary tract. This may lead to the formation of painful and recurrent kidney stones or nephrocalcinosis. Besides, toxicity from calcium oxalate deposition in the kidneys and urinary obstruction by calcium oxalate stones also leads to renal damage. After a certain point, the excess oxalate cannot be effectively excreted, which leads to accumulation and crystallization in bones, eyes, skin, and heart, leading to severe illnesses and death.

Currently, there are very few treatment options for this condition. The existing treatment options include frequent renal dialysis or the combined organ transplantation of liver and kidneys, a procedure with high morbidity which is also limited due to organ availability. Moreover, only a small proportion of patients respond to Vitamin B6 supplementation. So far, there are no approved pharmaceutical therapies for PH1.

About Lumasiran

Lumasiran, which was formerly known as ALN-GO1, is an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of PH1. RNAi is a natural cellular process of gene silencing. It is the most promising treatment and is rapidly advancing frontiers in biology and drug development, and also has the potential to transform the care of patients with genetic and other diseases.

Lumasiran has been specifically designed to lower the hepatic levels of the GO enzyme, which depletes the substrate required for oxalate production, and subsequently contributes to the pathophysiology of PH1. Lumasiran is based on Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index.

So far, Lumasiran has received the Orphan Drug Designation from both the US and the EU. However, the US Food and Drug Administration (FDA), or any other health authority, has not yet evaluated the safety and efficacy of this drug.

Alnylam and Sanofi Genzyme Alliance

In January 2014, Alnylam entered into an alliance with Sanofi Genzyme, the specialty care global business unit of Sanofi, to accelerate the development of RNAi therapeutics as a potential new class of innovative medicines for patients around the world with rare genetic diseases. In January 2018, the Companies modified their alliance, providing Alnylam with global rights to develop and commercialize products for the treatment of ATTR amyloidosis, including investigational RNAi therapeutics Patisiran and ALN-TTRsc02; and providing Sanofi Genzyme with global rights to develop and commercialize Fitusiran, an investigational RNAi therapeutic for the treatment of hemophilia and potentially other rare bleeding disorders. However, Sanofi Genzyme retains the right to opt into other Alnylam's rare genetic disease programs for development and commercialization in areas outside of the US, Canada, and Western Europe, as well as one right to a global license through the end of 2019, and potentially for up to two years thereafter.

Sanofi Genzyme declined its opt-in for the development and commercialization of Lumasiran. Yvonne Greenstreet, MBChB, MBA, Chief Operating Officer (COO) of Alnylam, expressed her pleasure on retaining the worldwide development and commercialization rights for Lumasiran. She believes this would enhance the number of global rare disease opportunities for Alnylam. The Parts A and B of the ongoing Phase-1/2 study of Lumasiran, presented to date, prove that the drug has the potential to be a transformative treatment for patients with PH1. The clinical dataset was presented at the American Society of Nephrology (ASN) Kidney Week 2017 Annual Meeting in November 2017.

Lumasiran Receives Breakthrough Therapy Designation from FDA

Additionally, Alnylam also declared that Lumasiran has been granted a Breakthrough Therapy Designation (BTD) by the FDA. This is the third BTD that Alnylam has received for its pipeline programs. The BTD is meant to expedite the development and review of investigational drugs for the treatment of serious or life-threatening conditions. BTDs are based on preliminary clinical evidence that proves that the drug may provide a substantial improvement on the clinically significant endpoint(s) over other available therapies.

Yvonne Greenstreet believes that the FDA's decision to grant a BTD to Lumasiran highlights the drug's potential to address the severe unmet medical need for PH1 patients and their families. Alnylam now looks forward to advancing Lumasiran into the Phase-3 study in late 2018, and to commercialize Lumasiran globally, assuming its approval.

Stock Performance Snapshot

March 13, 2018 - At Tuesday's closing bell, Alnylam Pharma's stock advanced 1.49%, ending the trading session at $144.16.

Volume traded for the day: 1.91 million shares, which was above the 3-month average volume of 841.90 thousand shares.

Stock performance in the last month – up 24.36%; previous three-month period – up 10.14%; past twelve-month period – up 185.58%; and year-to-date - up 13.47%

After yesterday's close, Alnylam Pharma's market cap was at $13.51 billion.

The stock is part of the Healthcare sector, categorized under the Biotechnology industry.


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