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4 Drug/Biotech Stocks Making a Mark in RNAi Therapeutics

Ekta Bagri

Biotech bigwig Regeneron Pharmaceuticals, Inc.’s REGN recently extended collaboration agreement with Alnylam Pharmaceuticals, Inc. ALNY brought ribonucleic acid interference (RNAi) therapeutics in focus.

The companies will work together to discover, develop and commercialize RNAi therapeutics for a broad range of diseases by addressing disease targets expressed in the eye and central nervous system (CNS) in addition to a select number of targets expressed in the liver. Regeneron will make a $400-million upfront payment to Alnylam. It will also purchase $400 million of Alnylam equity at $90.00 per share. Additionally, Alnylam is eligible to receive up to an additional $200 million in milestone payments.

The collaboration with Alnylam will give Regeneron an option to have a pipeline based on RNAi technology.

In 2018, the FDA approved Alnylam’s Onpattro for the treatment of polyneuropathy of hATTR amyloidosis in adults in the United States and in adults with Stage 1 or Stage 2 polyneuropathy in the EU. This is the first FDA-approved treatment for patients with polyneuropathy caused by hATTR, a rare, and often fatal genetic disease. It is also the first FDA approval of a new class of drugs called small interfering RNA (siRNA) treatment.

What is RNAi?

RNAi is a naturally occurring biological pathway within cells for sequence-specific silencing and regulation of gene expression. A revolutionary approach, RNAi therapeutics comprises siRNA and works by silencing messenger RNA, or mRNA, that encode for disease-causing proteins. 

Consequently, a lot of companies are investing a sizeable chunk of their R&D budget to develop treatments using RNAi based technology.

4 Companies Developing Candidates Using RNAi Technology

Here we take a look at some other companies that have promising candidates:

Arrowhead Pharmaceuticals Inc. ARWR develops therapies that trigger the RNA interference mechanism. The company has a broad portfolio of RNA chemistries. ARO-AAT, an RNAi therapeutic candidate for the treatment of liver disease associated with alpha-1 antitrypsin deficiency, is one of the key candidates. The company completed a phase I randomized, double-blind, placebo controlled single-ascending dose (SAD) and multiple-ascending dose (MAD) study to evaluate the safety, tolerability, pharmacokinetics, and effect of subcutaneous doses of ARO-AAT on serum alpha-1 antitrypsin levels in healthy adult volunteers in 2018 and plans to initiate a phase II study in 2019.

Other candidates include ARO-APOC3 for hypertriglyceridemia, ARO-ANG3 for dyslipidemia, ARO-ENaC for cystic fibrosis, and ARO-HIF2 for renal cell carcinoma. In 2016, the company out licensed ARO-LPA (AMG 890) for cardiovascular disease to Amgen Inc. The company also has a license and collaboration agreement with Amgen for ARO-AMG1 for an undisclosed genetically validated cardiovascular target. ARO-HBV for chronic hepatitis B virus was out-licensed to Janssen Pharmaceuticals, Inc. in October 2018.


Dicerna Pharmaceuticals, Inc. DRNA is another biopharmaceutical company focused on the discovery and development of innovative, subcutaneously delivered RNAi-based therapeutics using its GalXC RNAi platform intended for the treatment of diseases of the liver, including rare diseases, viral infectious diseases, chronic liver diseases, and cardiovascular diseases. Key candidates include DCR PHXC for the treatment of primary hyperoxaluria (“PH”), currently in a phase I trial; DCR HBVS for the treatment of chronic hepatitis B virus (“HBV”), currently in a phase I trial; and an undisclosed product candidate against a serious rare liver disease, currently in Investigational New Drug application (“IND”) enabling studies.

The company has strategic collaborations with Eli Lilly and Company, Alexion Pharmaceuticals, Inc. and Boehringer Ingelheim International GmbH.


Arbutus Biopharma ABUS is another company, which is developing RNAi therapeutics. The company is developing a second-generation RNAi agent, AB-729 that targets a single RNAi trigger sequence that spans all HBV transcripts and reduces all viral antigens, including hepatitis B surface antigen (HBsAg). A phase II clinical trial combining AB-729, Arbutus’ oral capsid inhibitor — AB-506 and an approved nucleoside analogue (NA) is expected to be initiated in the first half of 2020. 

Arbutus currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.


Moderna, Inc. MRNA is another clinical stage biotechnology company developing messenger RNA (mRNA) therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases and cardiovascular diseases. Moderna currently has 20 mRNA development candidates in its portfolio, with 11 in clinical studies. The company has collaborations with AstraZeneca, Merck & Co., and Vertex Pharmaceuticals.


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Alnylam Pharmaceuticals, Inc. (ALNY) : Free Stock Analysis Report
Regeneron Pharmaceuticals, Inc. (REGN) : Free Stock Analysis Report
Arbutus Biopharma Corporation (ABUS) : Free Stock Analysis Report
Dicerna Pharmaceuticals, Inc. (DRNA) : Free Stock Analysis Report
Moderna, Inc. (MRNA) : Free Stock Analysis Report
Arrowhead Pharmaceuticals, Inc. (ARWR) : Free Stock Analysis Report
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