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Blog Exposure - Alnylam Pharma Attained Alignment with FDA on Pivotal Study Design for Lumasiran for Treatment of Primary Hyperoxaluria Type 1

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LONDON, UK / ACCESSWIRE / May 07, 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 May 03, 2018, the Company announced that it has reached agreement with the US Food and Drug Administration (FDA) on a pivotal study design evaluating lumasiran, an investigational RNAi therapeutic for the treatment of primary hyperoxaluria type 1 (PH1). Register today and get access to over 1,000 Free Research Reports by joining our site below:


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Details of the Agreement

  • The Company and the FDA have aligned on a primary endpoint for the pivotal study based on reduction of urinary oxalate at six months, a biomarker directly linked to the pathophysiology of PH1 and known to be well correlated with disease progression.
  • Alnylam Pharma and the FDA have aligned on a study size of approximately 25 patients with PH1. Based on the discussions with the FDA, the Company will start the Phase-3 study in mid-2018 and expects to report topline results in 2019 and, if positive, Alnylam Pharma will submit a New Drug Application to the FDA in early 2020.

Phase-1/2 Part B Study

The ongoing Phase-1/2 Part B study is designed as a randomized, single-blind, placebo-controlled trial. 20 patients with PH1, aged six and higher, were enrolled in Part B with enrollment now complete. In preliminary results presented at the American Society of Nephrology (ASN) Kidney Week 2017 Annual Meeting, lumasiran demonstrated a mean maximal reduction in urinary oxalate of 66% with monthly dosing at 1 mg/kg in the unblinded Cohort 1 of the study (N=4), with all patients achieving urinary oxalate levels at or near the normal range. Moreover, lumasiran lowered urinary oxalate excretion in all patients below a threshold well documented to be associated with reduced progression to end-stage renal disease. In all patients, lumasiran was generally well tolerated and the only drug-related adverse event (AE) reported was a mild and transient injection site reaction. Five serious AEs (SAEs) were reported in three patients, including two episodes of renal stones and a case of pyelonephritis in a patient receiving placebo. None of the SAEs were considered drug-related.

Alnylam 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 recently granted Breakthrough Therapy Designation by the US FDA.

About Primary Hyperoxaluria Type 1 (PH1)

PH1 is an inherited disease in which the lack of a particular liver enzyme causes the body to accumulate excess amounts of a substance called oxalate. This oxalate leads to a buildup of insoluble calcium salts in the kidneys and other organs. If untreated, it results in life-threatening kidney failure. People with PH1 are prone to recurrent kidney stones that can lead to kidney failure. In addition to the kidneys, PH1 also leaves insoluble calcium deposits in other body tissues. This can lead to bone pain; vision loss; tingling, numbness, or pain in the extremities; enlargement of the liver and spleen; and problems with the electrical system of the heart (heart block).

About Lumasiran

Lumasiran ((formerly known as ALN-GO1) is a subcutaneously administered, 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 Pharma'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 Pharma 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

May 04, 2018 - At Friday's closing bell, Alnylam Pharma's stock advanced 4.57%, ending the trading session at $92.35.

Volume traded for the day: 2.88 million shares, which was above the 3-month average volume of 1.01 million shares.

Stock performance in the past twelve-month period – up 68.31%

After last Friday's close, Alnylam Pharma's market cap was at $9.02 billion.

The stock is part of the Healthcare sector, categorized under the Biotechnology industry. This sector was up 0.7% at the end of the session.


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