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Arcturus—A Clinical-Stage Mrna Therapeutics and Vaccines Company

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Arcturus—A Clinical-Stage Mrna Therapeutics and Vaccines Company ADVERTISEMENT FEATURE Arcturus Therapeutics Holdings Inc. arcturusrx.com Arcturus—a clinical-stage mRNA therapeutics and vaccines company With its unique mRNA therapeutics platform, Arcturus Therapeutics is a leading clinical- stage mRNA medicines company focused on the development of infectious disease vaccines and significant opportunities within liver and respiratory rare diseases. Arcturus Therapeutics, based in San Diego, has Low dose → Up to 40 lower dose than currently authorized vaccines proprietary enabling technologies: (i) LUNAR lipid- Single shot → Convenient for patients and healthcare workers mediated delivery, (ii) self-transcribing and repli- cating mRNA (STARR) technology, and (iii) innova- Lyophilized → No need for ultra-low temperature storage tive manufacturing of mRNA drug substance and Non-viral → Redosable capabilities for COVID-19 endemic phase the LUNAR-formulated drug product. Cost eective → Due to low dose and reduced material needs, Arcturus’ lead clinical stage product candidates materials and manufacturing sites can be reduced include a first-in-class, self-amplifying mRNA Safe → Lower dose means lower amount of product being vaccine for COVID-19 and an mRNA replacement administered potentially improving safety and tolerability therapy for ornithine transcarbamylase (OTC) Variant coverage- → T cells may be highly protective against variants and deficiency (Fig. 1). Preclinical programs include cellular immunity increase durability wholly owned mRNA therapeutics for cystic fibro- Fig. 2 | Arcturus’ COVID-19 vaccine candidate. sis and influenza, along with partnered programs in glycogen storage disease type 3, hepatitis B, and lyophilized, non-viral vector vaccine that potentially Clinical stage mRNA therapeutics non-alcoholic steatohepatitis. provides extended variant coverage and can be used for rare diseases Arcturus’ versatile RNA therapeutics platforms can for periodic re-dosing (Fig. 2). Due to its up to 40-fold Arcturus’ platform has great potential for helping be applied toward multiple types of nucleic acid medi- smaller dose requirement, ARCT-021 requires less treat rare diseases, including metabolic diseases. cines including mRNA, siRNA, replicon RNA, asRNA, manufacturing capacity than other currently autho- The company’s ARCT-810 program addresses OTC DNA, and gene editing therapeutics. Arcturus’ tech- rized mRNA vaccines for emergency use. deficiency, a rare genetic disease that can lead nologies are covered by a portfolio of 209 patents Interim data from phase 1 and phase 2 studies in to life threatening accumulation of ammonia in and patent applications, issued in the US, Europe, adults showed dose-related increases in binding the blood. ARCT-810 uses the LUNAR platform to Japan, China and other regions. Arcturus’ develop- and neutralizing antibody responses, and specific deliver mRNA into liver cells to produce function- ment partners include Janssen Pharmaceuticals, T cell activation following single dose administra- ing OTC with disease-modifying effects. ARCT-810 Ultragenyx, Takeda, CureVac, Synthetic Genomics, tion. No vaccine-related serious adverse events has received orphan drug designation from the US Duke-National University of Singapore (NUS) were reported. A phase 3 clinical trial application Food and Drug Administration. In a phase 1 study, Medical School, and the Cystic Fibrosis Foundation. is aimed for submission in Q2 2021 for ARCT-021 ARCT-810 demonstrated favorable safety, toler- to evaluate efficacy and safety. ability and PK profiles. Arcturus has now initiated STARR-based COVID-19 vaccine A key challenge for RNA-based therapeutics is a phase 1b study with ARCT-810 and is planning Arcturus’ COVID-19 vaccine candidate, ARCT-021, handling the drug product from its manufacture a phase 2 multiple-dose study in the second half developed in conjunction with Duke–NUS Medical until it is delivered into patients. While other mRNA of 2021. School, elicits an immune response to the SARS- vaccines are distributed as frozen liquids, ARCT-021 “Our key proprietary technology generates a CoV-2 virus spike protein. ARCT-021 is a single shot, is a lyophilized vaccine that preserves therapeutic one-dose, lyophilized drug product that addresses efficacy while not requiring an ultra-cold (i.e. less the major hurdles in RNA therapeutics develop- Product name Indication Stage than -70°C) supply chain. ment, namely the effective and safe delivery of Vaccines The platform is highly scalable, and with a library RNA molecules to disease-relevant target tissues,” of more than 250 proprietary lipids, Arcturus can said Joseph Payne, President and CEO of Arcturus. LUNAR-COV19 COVID-19 Clinical customize the LUNAR particles for specific indica- “We believe the versatility of our platform to tar- (ARCT-021) tions and targets. Following endocytosis, the acidic get multiple tissues, its compatibility with vari- LUNAR-FLU Influenza Preclinical environment in the endosomes triggers the LUNAR ous nucleic acid therapeutics, and our expertise Hepatic particles to release their cargo into the cytoplasm. in developing scalable manufacturing processes LUNAR-OTC Ornithine Clinical STARR directs the cellular protein production puts us in a position to deliver the next generation (ARCT-810) transcarbamylase machinery to either generate a protective immune of nucleic acid medicines.” deficiency response or to produce therapeutic proteins of interest. Compared to other non-self-replicating Respiratory mRNA-based therapeutic platforms, the sus- LUNAR-CF Cystic fibrosis Preclinical tained protein expression provided by the STARR Neda Safarzadeh, Sr. Director (ARCT-032) mRNA brings reduced dosing requirements and Head of IR, PR & Marketing Fig. 1 | Arcturus’ pipeline of mRNA-based improved results. STARR mRNA is delivered with Arcturus Therapeutics Holdings Inc. therapeutics. Arcturus has developed a diverse Arcturus’LUNAR delivery system. By combining CONTACT San Diego, CA, USA pipeline of mRNA-based therapeutics to address these two technologies, Arcturus is developing a Tel: +1-858-900-2682 infectious disease vaccines and opportunities vaccine product that addresses both the pandemic Email: [email protected] within liver and respiratory rare diseases. and the endemic phases. ADVERTISER RETAINS SOLE RESPONSIBILITY FOR CONTENT www.nature.com/biopharmdeal | June 2021 | B9.
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