U.S. Markets close in 53 mins

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

Stock Monitor: ACADIA Pharma Post Earnings Reporting

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:

www.active-investors.com/registration-sg

Active-Investors.com is currently working on the research report for ACADIA Pharmaceuticals Inc. (NASDAQ: ACAD), 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=ACAD

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

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.

Active-Investors:

Active-Investors (A-I) produces regular sponsored and non-sponsored reports, articles, stock market blogs, and popular investment newsletters covering equities listed on NYSE and NASDAQ and Canadian stocks. A-I has two distinct and independent departments. One department produces non-sponsored analyst certified content generally in the form of press releases, articles and reports covering equities listed on NYSE and NASDAQ and the other produces sponsored content (in most cases not reviewed by a registered analyst), which typically consists of compensated investment newsletters, articles and reports covering listed stocks and micro-caps. Such sponsored content is outside the scope of procedures detailed below.

A-I has not been compensated; directly or indirectly; for producing or publishing this document.

PRESS RELEASE PROCEDURES:

The non-sponsored content contained herein has been prepared by a writer (the "Author") and is fact checked and reviewed by a third-party research service company (the "Reviewer") represented by a credentialed financial analyst. For further information on analyst credentials, please email info@active-investors.com. Rohit Tuli, a CFA® charterholder (the "Sponsor"), provides necessary guidance in preparing the document templates. The Reviewer has reviewed and revised the content, as necessary, based on publicly available information which is believed to be reliable. Content is researched, written and reviewed on a reasonable-effort basis. The Reviewer has not performed any independent investigations or forensic audits to validate the information herein. The Reviewer has only independently reviewed the information provided by the Author according to the procedures outlined by A-I. A-I is not entitled to veto or interfere in the application of such procedures by the third-party research service company to the articles, documents or reports, as the case may be. Unless otherwise noted, any content outside of this document has no association with the Author or the Reviewer in any way.

NO WARRANTY

A-I, the Author, and the Reviewer are not responsible for any error which may be occasioned at the time of printing of this document or any error, mistake or shortcoming. No liability is accepted whatsoever for any direct, indirect or consequential loss arising from the use of this document. A-I, the Author, and the Reviewer expressly disclaim any fiduciary responsibility or liability for any consequences, financial or otherwise arising from any reliance placed on the information in this document. Additionally, A-I, the Author, and the Reviewer do not (1) guarantee the accuracy, timeliness, completeness or correct sequencing of the information, or (2) warrant any results from use of the information. The included information is subject to change without notice.

NOT AN OFFERING

This document is not intended as an offering, recommendation, or a solicitation of an offer to buy or sell the securities mentioned or discussed, and is to be used for informational purposes only. Please read all associated disclosures and disclaimers in full before investing. Neither A-I nor any party affiliated with us is a registered investment adviser or broker-dealer with any agency or in any jurisdiction whatsoever. To download our report(s), read our disclosures, or for more information, visit http://active-investors.com/legal-disclaimer/.

CONTACT

For any questions, inquiries, or comments reach out to us directly. If you're a company we are covering and wish to no longer feature on our coverage list contact us via email and/or phone between 09:30 EDT to 16:00 EDT from Monday to Friday at:

Email: info@active-investors.com

Phone number: 73 29 92 6381

Office Address: 6, Jalan Kia Peng, Kuala Lumpur, 50450 Kuala Lumpur, Wilayah Persekutuan Kuala Lumpur, Malaysia

CFA® and Chartered Financial Analyst® are registered trademarks owned by CFA Institute.

SOURCE: Active-Investors