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. Vaccine medicines include a vaccine candidate for COVID-19.

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; Phase 1 Clinical Trials in Healthy Volunteers Initiated on 05 June 2020


ARCT-021 Vaccine for COVID-19; Phase 1/2 Initiated 22 July 2020
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] 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. [3] Another additional 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. [4]

Platform

Arcturus Therapeutics' primary technology platform for RNA therapeutics is called LUNAR® [5][6] 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 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 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. [7] 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.[8]


STARRTM

The STARRTM technology platform combines self-replicating mRNA with LUNAR® creating the ability to  generate a protective immune response against infectious pathogens or increasing therapeutic protein expression to potentially prevent and treat a variety of diseases. Arcturus is developing a vaccine to protect against COVID-19 utilizing STARRTM technology.

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 and Academic Labs.

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.[9] Lunar OTC clinicals trials in humans began June 2020. [10]

LUNAR-CF

In collaboration with the Cystic Fibrosis Foundation to treat cystic fibrosis.[11]

LUNAR-GSD   

In collaboration with Ultragenyx to treat Glycogen Storage Disease type III.[12]

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.[13]

LUNAR-NASH

In collaboration with Takeda to develop medicines for the treatment of NASH and other gastrointestinal disorders.[14]

LUNAR-RPL

In collaboration with Synthetic Genomics to develop improved technology for vaccines and therapeutics.[15]

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 response to the COVID-19 pandemic, Arcturus Therapeutics partnered with Duke–NUS Medical School to develop a COVID-19 vaccine using STARRTM technology[16] The company also partnered with Catalent, a contract development and manufacturing organization, to manufacture multiple batches of Arcturus’ COVID-19 mRNA vaccine candidate.[17] LUNAR-COV19 clinical trials in humans began July 2020. [18]

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. "Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With OTC Deficiency - Full Text View - ClinicalTrials.gov". clinicaltrials.gov. Retrieved 2020-07-27.
  4. "Arcturus Therapeutics Announces First Healthy Volunteer Dosed in Phase 1 Study of ARCT-810 for Ornithine Transcarbamylase (OTC) Deficiency | Arcturus Therapeutics, Inc". ir.arcturusrx.com. Retrieved 2020-07-27.
  5. 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.
  6. 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.
  7. 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.
  8. 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.
  9. "Arcturus Therapeutics Receives Orphan Drug Designation". Drug Development and Delivery.
  10. "Arcturus Therapeutics Announces First Healthy Volunteer Dosed in Phase 1 Study of ARCT-810 for Ornithine Transcarbamylase (OTC) Deficiency | Arcturus Therapeutics, Inc". ir.arcturusrx.com. Retrieved 2020-07-27.
  11. Lopez PhD., Jose Marques (25 June 2018). "RNA Therapy for CF shows promise in preclinical studies". Cystic Fibrosis News Today.
  12. "mRNA therapeutics - Arcturus Therapeutics/Ultragenyx Pharmaceutical". Adis Insights, Springer. 21 Jun 2019.
  13. Adams, Ben (19 Oct 2017). "Johnson & Johnson, RNA biotech Arcturus combine for hepatitis B work". Fierce Biotech.
  14. Elvidge, Suzanne. "Mixed results in NASH for Gilead". BioPharma Drive.
  15. Kotok, Allan (31 Oct 2017). "RNA Meds Companies Partner on Human, Animal Vaccines". Science Business.
  16. "Coronavirus: Clinical trials for Singapore's vaccine project could start in August". The Straits Times. Retrieved 27 April 2020.
  17. "With Arcturus, Catalent bags another COVID project - Bioprocess Insider". BioProcess International. 2020-05-06. Retrieved 2020-05-08.
  18. "Phase 1/2 Ascending Dose Study of Investigational SARS-CoV-2 Vaccine ARCT-021 in Healthy Adult Subjects - Full Text View - ClinicalTrials.gov". clinicaltrials.gov. Retrieved 2020-07-27.
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