Arcturus Therapeutics is an American RNA medicines biotechnology company focused on the discovery, development and commercialization of therapeutics for

rare disease A rare disease is any disease that affects a small percentage of the population. In some parts of the world, an orphan disease is a rare disease whose rarity means there is a lack of a market large enough to gain support and resources for discove ...

s and infectious diseases. Arcturus has developed a novel, potent, and safe RNA therapeutics platform called Lunar, a proprietary lipid-enabled delivery system for nucleic acid medicines including small interfering RNA (siRNA), messenger RNA (mRNA), gene editing RNA, DNA, antisense oligonucleotides (ASO), and

microRNA MicroRNA (miRNA) are small, single-stranded, non-coding RNA molecules containing 21 to 23 nucleotides. Found in plants, animals and some viruses, miRNAs are involved in RNA silencing and post-transcriptional regulation of gene expression. miRN ...

. The company's pipeline includes RNA therapeutics for the treatment of rare diseases such as

ornithine transcarbamylase deficiency Ornithine transcarbamylase deficiency also known as OTC deficiency is the most common urea cycle disorder in humans. Ornithine transcarbamylase, the defective enzyme in this disorder is the final enzyme in the proximal portion of the urea cycle, re ...

,and respiratory diseases such as

cystic fibrosis Cystic fibrosis (CF) is a rare genetic disorder that affects mostly the lungs, but also the pancreas, liver, kidneys, and intestine. Long-term issues include difficulty breathing and coughing up mucus as a result of frequent lung infections. O ...

. Vaccine medicines include a vaccine candidate for COVID-19 and an influenza vaccine in the

preclinical development In drug development, preclinical development, also termed preclinical studies or nonclinical studies, is a stage of research that begins before clinical trials (testing in humans) and during which important feasibility, iterative testing and drug ...

phase.

Company history

Founded in 2013 by Joseph Payne and Dr. Pad Chivukula, Arcturus Therapeutics is headquartered in San Diego, California, USA. The company's core technology platform is a proprietary lipid nanoparticle-mediated delivery system able to deliver therapeutic RNA or DNA to target cells inside a patient's body. The company has a portfolio of eight mRNA-based therapeutics and two mRNA-based vaccines in development. In September 2017, Arcturus Therapeutics merged with Alcobra Ltd., an Israeli pharmaceutical company. In February of 2018, Joseph Payne was dismissed as president and CEO by the Arcturus Therapeutics board of directors at that time. In the aftermath, as the largest shareholder, Joseph Payne filed formal requests to hold a meeting of shareholders for the purpose of selecting new members to the Arcturus Therapeutics board of directors. Under applicable law, Arcturus’ board must have honored Mr. Payne’s request by establishing a meeting date and convening said meeting within a clearly defined timeframe. Rather than adhering to the law, the Board ignored the applicable deadlines and opted to postpone the shareholder meeting indefinitely. Joseph Payne subsequently filed legal proceedings with the Israeli Court which ruled in favor of upholding shareholder rights by sanctioning the meeting of shareholders. In April of 2018, the Arcturus Therapeutics board of directors at the time filed their own complaint against Joseph Payne and his nominees to the board. In May of 2018, a legal settlement was agreed upon: 4 new directors were appointed to the board (Dr. Peter Farrell, Mr. Andrew Sassine, Mr. James Barlow and Dr. Magda Marquet) resulting in the resignation of Dr. Stuart Collinson, Mr. Daniel Geffken, Dr. David Shapiro, and Mr. Craig Willett. Other key terms of the settlement agreement included mutual releases of all parties and the agreement by Arcturus Therapeutics and Joseph Payne to terminate all pending litigation. On June 27, 2019, the

FDA The United States Food and Drug Administration (FDA or US FDA) is a federal agency of the Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food ...

granted

Orphan Drug An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases. The assignment of ...

Designation for the company's lead product candidate ARCT-810 to treat an inherited metabolic disorder, ornithine transcarbamylase deficiency (OTCD). OTCD is the most common urea cycle disorder, which impacts an individual's ability to remove toxic waste products from the body. The orphan drug designation is granted to drugs capable of treating rare diseases that affect less than 200,000 people in the United States. Drug products approved by the FDA that have orphan drug status receive up to 7 years of marketing exclusivity. On April 13, 2020, it was announced that the company's investigational new drug (IND) application for Phase 1b study in patients with OTCD was allowed to proceed by the U.S. FDA. A Clinical Trial Application (CTA) for a Phase 1 study in healthy volunteers was approved by the New Zealand Medicines and Medical Devices Safety Authority (Medsafe). On June 5, 2020 Arcturus Therapeutics announced it dosed its first healthy volunteer in a Phase 1 study with ARCT-810. Following the success of Phase 1a, clinical phase progression continued in December 2020 with the dosing of the first patients in phase 1b of the Clinical Trial.

Platform

Lunar Arcturus Therapeutics' primary technology platform for RNA therapeutics is called Lunar a novel lipid-mediated delivery system. Lunar is a multi-component drug delivery system that enables scientists to target specific cells inside the body and deliver a payload of RNA into the cell. Once release of the RNA into the cell occurs, the normal translational machinery of the cell can interact with the RNA to make a functional protein with a therapeutic effect. ARCT-810, the company's lead product utilizes Arcturus' Lunar lipid-mediated delivery platform intended to safely and effectively deliver OTC messenger RNA to liver cells. Lunar mediated delivery to the liver has been confirmed in collaboration with the

Salk Institute for Biological Studies The Salk Institute for Biological Studies is a scientific research institute located in the La Jolla community of San Diego, California, U.S. The independent, non-profit institute was founded in 1960 by Jonas Salk, the developer of the polio vacci ...

in La Jolla, California which demonstrated the successful Lunar-mediated delivery of a Factor IX messenger RNA (FIX mRNA) to the liver as reported in a publication in PNAS, where it was confirmed be efficacious. Lunar is an effective and reproducible LNP mRNA delivery platform to potentially treat diseases requiring protein replacement. Researchers at the

MD Anderson Cancer Center The University of Texas MD Anderson Cancer Center (colloquially MD Anderson Cancer Center) is a comprehensive cancer center in Houston, Texas. It is the largest cancer center in the U.S. and one of the original three comprehensive cancer centers ...

also showed the unique application of microRNAs delivered in nanoparticles using the Lunar platform as novel immune therapeutics for the treatment of malignancy. Starr Starr technology is a combination of self-replicating mRNA with the Lunar delivery platform which increases the level and duration of expression of a therapeutic protein. The Starr technology platform is able to generate a protective immune response against infectious pathogens or increase the expression of therapeutic proteins to prevent and treat a variety of diseases. Arcturus is using the Starr technology to develop vaccines for both

COVID-19 Coronavirus disease 2019 (COVID-19) is a contagious disease caused by a virus, the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). The first known case was COVID-19 pandemic in Hubei, identified in Wuhan, China, in December ...

and, separately,

influenza Influenza, commonly known as "the flu", is an infectious disease caused by influenza viruses. Symptoms range from mild to severe and often include fever, runny nose, sore throat, muscle pain, headache, coughing, and fatigue. These symptoms ...

Products in development

Lunar-COV19

ARCT-021

In response to the

COVID-19 pandemic The COVID-19 pandemic, also known as the coronavirus pandemic, is an ongoing global pandemic of coronavirus disease 2019 (COVID-19) caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). The novel virus was first identif ...

, the form partnered with

Duke–NUS Medical School The Duke–NUS Medical School (Duke–NUS) is a graduate school, graduate medical school in Singapore. The school was set up in April 2005 as the Duke–NUS Graduate Medical School, Singapore's second medical school, after the Yong Loo Lin School ...

to develop a

COVID-19 vaccine A COVID19 vaccine is a vaccine intended to provide acquired immunity against severe acute respiratory syndrome coronavirus 2 (SARS‑CoV‑2), the virus that causes coronavirus disease 2019 ( COVID19). Prior to the COVID19 pandemic, an e ...

using Starr technology (Starr technology is a combination of self-replicating mRNA with the Lunar delivery platform which increases the level and duration of expression of a therapeutic protein). The company also partnered with Catalent and Recipharm, contract development and manufacturing organizations, to manufacture multiple batches of Arcturus'

mRNA vaccine An mRNA vaccine is a type of vaccine that uses a copy of a molecule called messenger RNA (mRNA) to produce an immune response. The vaccine delivers molecules of antigen-encoding mRNA into immune cells, which use the designed mRNA as a blueprin ...

candidate. Lunar-COV19 clinical trials in healthy volunteers began in July 2020. Phase 2 clinical trials were approved in the United States and Singapore by the FDA and HSA, respectively in December 2020.

Next-generations

On 2 August 2021, ARCT-154 started a clinical trial in Vietnam for next-generation development. The next day, the firm announced that the application of approval for the clinical trial Phase I/II in Singapore called ARCT-165. Also application of ARCT-154 for phase I/II.

Lunar-OTC

A wholly owned mRNA medicine to treat ornithine transcarbamylase deficiency, a life-threatening genetic disease caused by lack of the enzyme in liver cells; there is currently no cure . In 2019, Lunar-OTC received FDA

orphan drug An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases. The assignment of ...

status. Lunar OTC clinical trials in patients began June 2020. In December 2020, clinical phase 1b of Lunar-OTC began with dosing in patients. Other projects Lunar-CF is a project in collaboration with the

Cystic Fibrosis Foundation The Cystic Fibrosis Foundation (CFF) is a 501(c)(3) non-profit organization in the United States established to provide the means to cure cystic fibrosis (CF) and ensure that those living with CF live long and productive lives. The Foundation prov ...

to treat

. Lunar-FLU is a wholly owned program to protect against the

virus. Arcturus is combining its self-replicating mRNA Starr technology with Lunar to develop a prophylactic vaccine against influenza. Lunar-GSD is a project in collaboration with

Ultragenyx Ultragenyx is an American biopharmaceutical company involved in the Research and Development of novel products for treatment of rare and ultra-rare genetic diseases for which there are typically no approved treatments and high unmet medical need ...

to treat

glycogen storage disease type III Glycogen storage disease type III (GSD III) is an autosomal recessive metabolic disorder and inborn error of metabolism (specifically of Inborn errors of carbohydrate metabolism, carbohydrates) characterized by a deficiency in glycogen debranching ...

. Lunar-Rare is a project in collaboration with

to develop therapeutic candidates for rare disease targets. Lunar-HBV is a project in collaboration with Janssen to develop medicines for the treatment of hepatitis B virus infection. Lunar-NASH is a project in collaboration with

Takeda is a Japanese family name.1990 Census Name Files< ...

to develop medicines for

non-alcoholic steatohepatitis Non-alcoholic fatty liver disease (NAFLD), also known as metabolic (dysfunction) associated fatty liver disease (MAFLD), is excessive fat build-up in the liver without another clear cause such as alcohol use. There are two types; non-alcoholic ...

(NASH) and other gastrointestinal disorders. Lunar-RPL is a project in collaboration with Synthetic Genomics to develop improved technology for vaccines and therapeutics. Lunar-AH is a project in collaboration with Synthetic Genomics to develop infectious disease prophylactic vaccines.

References

External links

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{{authority control Companies listed on the Nasdaq Biotechnology companies of the United States COVID-19 vaccine producers American companies established in 2013