Vir and Alnylam Identify RNAi Therapeutic Development Candidate, VIR-2703 (ALN-COV), Targeting SARS-CoV-2 for the Treatment of COVID-19

− Highly Potent and Broadly Cross-Reactive RNAi Therapeutic Development Candidate Selected Only Three Months After Program Initiation −

− Companies Expect to Start Human Clinical Trials at or Around Year-End 2020 –

SAN FRANCISCO, CA, USA & CAMBRIDGE, MA, USA I May 04, 2020 I Vir Biotechnology, Inc. (Nasdaq: VIR) and Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) today announced the selection of a development candidate (DC) for VIR-2703 (also referred to as ALN-COV), an investigational RNAi therapeutic targeting the SARS-CoV-2 genome. The companies plan to soon meet with the U.S. Food and Drug Administration (FDA) and other regulatory authorities to discuss a potential accelerated path for filing an Investigational New Drug (IND) or IND equivalent application at or around year-end 2020, less than a year since program initiation. The companies plan to advance VIR-2703 as an inhalational formulation for the potential treatment and/or prevention of COVID-19.

In its discovery efforts, Alnylam synthesized over 350 small interfering RNAs (siRNAs) – the molecules that mediate RNAi – targeting highly conserved regions of the SARS-CoV-2 genome, which were then analyzed bioinformatically and assessed in in vitro potency assays. Multiple highly potent siRNAs were identified that demonstrated a 3-log reduction of viral replication in an in vitro SARS-CoV-2 live virus model conducted by Vir. In dose-response assays, VIR-2703 was shown to have an effective concentration for 50% inhibition (EC50) of less than 100 picomolar and an EC95 of less than 1 nanomolar in the SARS-CoV-2 live virus model measuring inhibition of infectious virion production. Further, VIR-2703 has predicted reactivity against greater than 99.9 percent of the over 4300 SARS-CoV-2 genomes currently available in public databases that meet analysis requirements, and is also predicted to have reactivity toward the SARS-CoV genome from the 2003 SARS outbreak. With this DC selection, Vir and Alnylam will work closely together to generate the data required to enable rapid commencement of clinical studies.

“Vir is committed to applying industry-leading technologies in our fight against COVID-19. Our fruitful and expansive collaboration with Alnylam has rapidly led to identification of a development candidate targeting SARS-CoV-2,” said George Scangos, Ph.D., CEO of Vir. “With this candidate now in hand, we will further accelerate our efforts and plan to begin studies in humans at or around year-end. Our ultimate goal would be to provide rapid worldwide access, if approved, to an effective therapeutic to combat COVID-19.”

“I’m very proud of the quality and pace of work done by our scientists and with our collaborators at Vir to identify an RNAi therapeutic development candidate targeting the SARS-CoV-2 genome. To our knowledge, this is one of the most potent direct-acting antivirals targeting SARS-CoV-2 reported to date,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “As this pandemic continues to unfold, we are committed to acting with the utmost urgency to broaden and accelerate our efforts to develop investigational RNAi therapeutics against COVID-19, and potentially future coronavirus-mediated diseases.”

In addition to the development candidate targeting the SARS-CoV-2 genome, the companies will utilize Alnylam’s recent advances in lung delivery of siRNAs, with widespread silencing of gene targets in the respiratory tract in multiple cell types, including cells in the respiratory tract that may be the targets for SARS-CoV-2 infection. In addition, the companies will employ Vir’s infectious disease expertise and established capabilities to bring forward up to three additional host factor-targeting development candidates to treat COVID-19, and potentially other coronavirus diseases.

About VIR-2703 (ALN-COV)

VIR-2703 is an inhaled SARS-CoV-2-targeting siRNA that may have utility for prevention or for treatment. It leverages Alnylam Pharmaceuticals, Inc.’s latest advances in lung delivery of siRNAs and may have applicability to other coronaviruses as well. VIR-2703 is the first development candidate selected in the company’s expanded collaboration with Alnylam for up to four RNAi potential therapeutics for COVID-19.

About RNAi

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines, known as RNAi therapeutics, is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

About Vir Biotechnology

Vir Biotechnology is a clinical-stage immunology company focused on combining immunologic insights with cutting-edge technologies to treat and prevent serious infectious diseases. Vir has assembled four technology platforms that are designed to stimulate and enhance the immune system by exploiting critical observations of natural immune processes. Its current development pipeline consists of product candidates targeting hepatitis B virus, influenza A, SARS-CoV-2, human immunodeficiency virus and tuberculosis. For more information, please visit www.vir.bio.

About Alnylam

Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform. Alnylam’s commercial RNAi therapeutic products are ONPATTRO® (patisiran), approved in the U.S., EU, Canada, Japan, Switzerland, and Brazil, and GIVLAARI® (givosiran), approved in the U.S. and EU. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its "Alnylam 2020" strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines to address the needs of patients who have limited or inadequate treatment options. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam or on LinkedIn.

SOURCE: Vir Biotechnology

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