Arcturus Therapeutics

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Arcturus Therapeutics
TypePublic
Nasdaq: ARCT
IndustryPharmaceutical industry
Founded2013; 9 years ago (2013)
FounderJoseph Payne and Pad Chivukula
HeadquartersSan Diego, CA, US
Key people
Joseph Payne (President, CEO & Director of the Board), Padmanabh Chivukula (CSO & COO), Andrew Sassine (CFO)
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, Phase 2 Initiated 9 January 2021
RevenueIncrease $21 Million(2019)[1]
Number of employees
124
Websitewww.arcturusrx.com

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

The company's pipeline includes RNA therapeutics for the treatment of rare diseases such as ornithine transcarbamylase deficiency, and respiratory diseases such as cystic fibrosis. Vaccine medicines include a vaccine candidate for COVID-19, and an influenza vaccine in the preclinical development 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.[2] The company has a portfolio of eight mRNA-based therapeutics and two mRNA-based vaccines in development.

On June 27, 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.[3] 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.[4] 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.[5] 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.[6]

Platform[]

LUNAR

Arcturus Therapeutics' primary technology platform for RNA therapeutics is called LUNAR [7][8] 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 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.[9] 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.[10]

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 and, separately, influenza.

Products in development[]

The company has programs in development for novel RNA therapeutics and mRNA vaccines, both wholly owned and in collaboration with other Pharmaceutical companies and Academic Labs.

LUNAR-COV19[]

ARCT-021[]

Main article: ARCT-021

In response to the COVID-19 pandemic, Arcturus Therapeutics partnered with Duke–NUS Medical School to develop a COVID-19 vaccine 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).[11] The company also partnered with Catalent and Recipharm, contract development and manufacturing organizations, to manufacture multiple batches of Arcturus' COVID-19 mRNA vaccine candidate.[12][13] LUNAR-COV19 clinical trials in healthy volunteers began in July 2020.[14] Phase 2 clinical trials were approved in the United States and Singapore by the FDA and HSA, respectively in December 2020.[15]

Arcturus has entered into development and supply agreements with the Economic Development Board of Singapore and supply agreements with the Israel Ministry of Health for LUNAR-COV19.

Next-generations[]

Main article: ARCT-154

On 2 August 2021, ARCT-154 started a clinical trial in Vietnam for next-generation development.[16] The next day, Arcturus Therapeutics 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.[17]

LUNAR-OTC[]

A wholly owned 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.[18] Lunar OTC clinical trials in patient began June 2020.[19] In December 2020, clinical phase 1b of LUNAR-OTC began with dosing in patients.

LUNAR-CF

LUNAR-CF is a project in collaboration with the Cystic Fibrosis Foundation to treat cystic fibrosis.[20]

LUNAR-FLU[]

A wholly owned program to protect against the influenza virus. Arcturus is combining its self-replicating mRNA STARR technology with LUNAR to develop a prophylactic vaccine against influenza.

LUNAR-GSD []

LUNAR-GSD is a project in collaboration with Ultragenyx to treat glycogen storage disease type III.[21]

LUNAR-Rare[]

LUNAR-Rare is a project in collaboration with Ultragenyx to develop therapeutic candidates for certain rare disease targets.

LUNAR-HBV[]

LUNAR-HBV is a project in collaboration with Janssen to develop medicines for the treatment of hepatitis B virus infection.[22]

LUNAR-NASH[]

LUNAR-NASH is a project in collaboration with Takeda to develop medicines for the treatment of non-alcoholic steatohepatitis (NASH) and other gastrointestinal disorders.[23]

LUNAR-RPL[]

LUNAR-RPL is a project in collaboration with Synthetic Genomics to develop improved technology for vaccines and therapeutics.[24]

LUNAR-AH[]

LUNAR-AH is a project in collaboration with Synthetic Genomics to develop infectious disease prophylactic vaccines.

References[]

  1. ^ "Arcturus Therapeutics Holdings Revenue 2012-2021 | ARCT".
  2. ^ Encyclopedia of Information Science and Technology, Fourth Edition. edited by Khosrow-Pour.
  3. ^ "Designating an Orphan Product: Drugs and Biological Products". FDA Website. 24 Jul 2019.
  4. ^ "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.
  5. ^ "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.
  6. ^ Arcturus Therapeutics, Inc. (2020-10-05). "A Phase 1b Randomized, Double Blinded, Placebo Controlled, Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Single Doses of ARCT-810 in Clinically Stable Patients With Ornithine Transcarbamylase Deficiency". Retrieved 12 March 2021.
  7. ^ Teruki, Yanagi (28 June 2016). "Lipid Nanoparticle-mediated siRNA Transfer Against PCTAIRE1/PCTK1/Cdk16 Inhibits In Vivo Cancer Growth". Molecular Therapy: Nucleic Acids. 5 (6): e327. doi:10.1038/mtna.2016.40. PMC 5022131. PMID 27351680.
  8. ^ 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.
  9. ^ 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.
  10. ^ 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.
  11. ^ "Coronavirus: Clinical trials for Singapore's vaccine project could start in August". The Straits Times. 15 April 2020. Retrieved 27 April 2020.
  12. ^ "With Arcturus, Catalent bags another COVID project". Bioprocess Insider. 2020-05-06. Retrieved 2020-05-08.
  13. ^ "Recipharm signs agreement with Arcturus Therapeutics to support the manufacture of LUNAR[®]-COV19 (ARCT-021) vaccine candidate". Recipharm. Retrieved 2021-03-12.
  14. ^ "Phase 1/2 Ascending Dose Study of Investigational SARS-CoV-2 Vaccine ARCT-021 in Healthy Adult Subjects". clinicaltrials.gov. Retrieved 2020-07-27.
  15. ^ Arcturus Therapeutics, Inc. (2021-03-02). "A Phase 2 Randomized, Observer-Blind, Placebo-Controlled Study to Assess the Safety, Reactogenicity, and Immunogenicity of the SARS CoV-2 Vaccine ARCT-021 in Healthy Adult Participants". Retrieved 12 March 2021.
  16. ^ "Arcturus to start clinical trial of COVID-19 vaccine in Vietnam". Reuters. 2 August 2021. Retrieved 3 August 2021.
  17. ^ "Arcturus Announces Approval of Singapore Clinical Trial Application to Advance ARCT-154 and ARCT-165, Next Generation STARR™ mRNA Vaccines Targeting SARS-CoV-2 Variants of Concern, in a Phase 1/2 Study". Arcturus Therapeutics. 3 August 2021.
  18. ^ "Arcturus Therapeutics Receives Orphan Drug Designation". Drug Development and Delivery. 28 June 2019.
  19. ^ "Arcturus Therapeutics Announces First Healthy Volunteer Dosed in Phase 1 Study of ARCT-810 for Ornithine Transcarbamylase (OTC) Deficiency". Arcturus Therapeutics. Retrieved 2020-07-27.
  20. ^ Lopez, Jose Marques (25 June 2018). "RNA Therapy for CF shows promise in preclinical studies". Cystic Fibrosis News Today.
  21. ^ "mRNA therapeutics". Adis Insights, Springer. Arcturus Therapeutics/Ultragenyx Pharmaceutical. 21 June 2019.
  22. ^ Adams, Ben (19 October 2017). "Johnson & Johnson, RNA biotech Arcturus combine for hepatitis B work". Fierce Biotech.
  23. ^ Elvidge, Suzanne. "Mixed results in NASH for Gilead". BioPharma Drive.
  24. ^ Kotok, Allan (31 October 2017). "RNA Meds Companies Partner on Human, Animal Vaccines". Science Business.

External links[]

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