Arcturus Therapeutics

Arcturus Therapeutics is an American biotech company focused on the discovery, development and commercialization of therapeutics for rare diseases with focus on RNA. Arcturus has developed a novel, potent and safe RNA therapeutics platform called LUNAR®, a proprietary lipid-enabled delivery system for RNA medicines including small interfering RNA, messenger RNA, gene editing, DNA, antisense, and microRNA oligotherapeutics. The company’s pipeline includes RNA therapeutics towards rare diseases such as ornithine transcarbamylase deficiency (OTCD), and respiratory diseases such as cystic fibrosis.

Arcturus Therapeutics
Public company
Traded asNASDAQ: ARCT
IndustryPharmaceutical industry
Founded2013 (2013)
HeadquartersSan Diego, CA, US
ProductsARCT-810 for treatment of OTCD; received FDA Orphan Drug Status on 27 July 2019
Websitewww.arcturusrx.com

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 patients body.[1] The company has a portfolio of at least 10 RNA-based therapeutics in development.

On June 27th 2019, the FDA granted Orphan Drug 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.[2]

Platform

Arcturus Therapeutics primary technology platform for RNA therapeutics is called LUNAR® [3][4] a novel Lipid-enabled nanoparticle. 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 cells cytosol. Once release of the RNA into the cytosol occurs, the normal translational machinery of the cell can interact with the RNA to make functional protein that delivers a therapeutic effect.

ARCT-810, the company’s lead product utilizes Arcturus' LUNAR® lipid-mediated delivery platform to safely and effectively deliver OTC messenger RNA to liver cells. Research using the LUNAR platform has also been independently published in PNAS by the Salk Institute in La Jolla, California who demonstrated the successful use of LUNAR and confirmed it to be a safe, reproducible, and effective LNP mRNA delivery platform to treat diseases requiring protein replacement[5].  Researchers at the MD Anderson Cancer Center also showed the unique application of microRNAs delivered in nanoparticles using the LUNAR platform as novel immune therapeutics for the treatment of malignancy[6]

Products in development

The company has at least 10 programs in development for novel RNA therapeutics, both wholly owned and in collaboration with other Pharmaceutical companies.

LUNAR-OTC

The company’s lead development program: an mRNA medicine to treat ornithine transcarbamylase deficiency, a life-threatening genetic disease caused by lack of the OTC enzyme in liver cells. OTC can cause seizures, coma, and death in untreated patients. There is currently no cure for OTC deficiency. In 2019, LUNAR OTC received FDA Orphan Drug Status.[7]

LUNAR-CF

In collaboration with the Cystic Fibrosis Foundation to treat cystic fibrosis.[8]

LUNAR-GSD   

In collaboration with Ultragenyx to treat Glycogen Storage Disease type III.[9]

LUNAR-Rare

In collaboration with Ultragenyx to develop therapeutic candidates for certain rare disease targets.

LUNAR-HBV

In collaboration with Janssen to develop medicines for the treatment of hepatitis B virus infection.[10]

LUNAR-NASH

In collaboration with Takeda to develop medicines for the treatment of NASH and other gastrointestinal disorders.[11]

LUNAR-RPL

In collaboration with Synthetic Genomics to develop improved technology for vaccines and therapeutics.[12]

LUNAR-AH

In collaboration with Synthetic Genomics to develop Infectious Disease Prophylactic vaccines

LUNAR-CV

A wholly owned program focused on Rare Cardiovascular Disease

LUNAR-MD

A wholly owned program focused on Rare Metabolic Disease

LUNAR-COV19

In 2020 Arcturus Therapeutics partnered with Duke–NUS Medical School to develop a COVID-19 vaccine using STARR technology.[13] The company also partnered with Catalent, a contract development and manufacturing organization, who will manufacture multiple batches of Arcturus’ COVID-19 mRNA vaccine candidate.[14]

References
  1. Encyclopedia of Information Science and Technology, Fourth Edition. edited by Khosrow-Pour.
  2. "Designating an Orphan Product: Drugs and Biological Products". FDA Website. 24 Jul 2019.
  3. Teruki, Yanagi (28 June 2016). "Lipid Nanoparticle-mediated siRNA Transfer Against PCTAIRE1/PCTK1/Cdk16 Inhibits In Vivo Cancer Growth". Molecular Therapeutic Nucleic Acids. 5(6): e327.
  4. Stein, Richard (26 May 2017). "Better Targeting, Delivery of RNAi Therapies". Genetic Engineering & Biotechnology News. 37 (11): 1, 14–17. doi:10.1089/gen.37.11.02.
  5. Ramaswamy, Suvasini (7 March 2017). "Systemic delivery of factor IX messenger RNA for protein replacement therapy". PNAS. 114 (10): E1941–E1950. doi:10.1073/pnas.1619653114. PMC 5347596. PMID 28202722.
  6. Yaghi, Nasser (1 March 2017). "Immune modulatory nanoparticle therapeutics for intracerebral glioma". Neuro-Oncology. 9 (3): 372–382. doi:10.1093/neuonc/now198. PMC 5464307. PMID 27765835.
  7. "Arcturus Therapeutics Receives Orphan Drug Designation". Drug Development and Delivery.
  8. Lopez PhD., Jose Marques (25 June 2018). "RNA Therapy for CF shows promise in preclinical studies". Cystic Fibrosis News Today.
  9. "mRNA therapeutics - Arcturus Therapeutics/Ultragenyx Pharmaceutical". Adis Insights, Springer. 21 Jun 2019.
  10. Adams, Ben (19 Oct 2017). "Johnson & Johnson, RNA biotech Arcturus combine for hepatitis B work". Fierce Biotech.
  11. Elvidge, Suzanne. "Mixed results in NASH for Gilead". BioPharma Drive.
  12. Kotok, Allan (31 Oct 2017). "RNA Meds Companies Partner on Human, Animal Vaccines". Science Business.
  13. "Coronavirus: Clinical trials for Singapore's vaccine project could start in August". The Straits Times. Retrieved 27 April 2020.
  14. "With Arcturus, Catalent bags another COVID project - Bioprocess Insider". BioProcess International. 2020-05-06. Retrieved 2020-05-08.
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