Therapy Areas: Hereditary Disorders
Arcturus Receives Regulatory Approval to Proceed with Phase 2 Study of ARCT-810 mRNA Therapeutic Candidate for Ornithine Transcarbamylase Deficiency
29 July 2021 - - US-based clinical-stage messenger RNA medicines company Arcturus Therapeutics Holdings Inc. (NASDAQ: ARCT) has received approval from the UK Health Research Authority to initiate a Phase 2 clinical study for ARCT-810, a novel mRNA-based therapeutic candidate for Ornithine Transcarbamylase Deficiency, the company said.

ARCT-810 represents Arcturus' lead rare-disease asset utilizing the company's novel, systemically administered LUNAR delivery platform.

The ARCT-810 Phase 2 study is a randomized, double-blind, placebo-controlled, nested single and multiple ascending dose design for adolescents and adults with OTC deficiency.

The study will enroll up to 24 subjects across two dose levels. Safety and tolerability of ARCT-810 are the primary study objectives, and pharmacokinetic and pharmacodynamic measures of urea cycle function will be performed over a 12- week treatment period to access exposure and efficacy of this novel treatment.

Ornithine Transcarbamylase deficiency is a serious urea cycle disorder with a prevalence of approximately 10,000 people worldwide.

A lack of OTC, a critical urea cycle enzyme in liver cells, results in high blood ammonia levels that can cause diminished cognitive ability, seizures, coma, and death.

There are no approved medicines addressing the root cause of OTC deficiency.

Founded in 2013 and based in San Diego, California, Arcturus Therapeutics Holdings Inc. (NASDAQ: ARCT) is a clinical-stage mRNA medicines and vaccines company with enabling technologies: LUNAR lipid-mediated delivery, STARR mRNA Technology and mRNA drug substance along with drug product manufacturing expertise.

Arcturus' diverse pipeline of RNA therapeutic and vaccine candidates includes mRNA vaccine programs for SARS-CoV-2 (COVID-19) and Influenza, and other programs to potentially treat Ornithine Transcarbamylase Deficiency, and Cystic Fibrosis along with partnered programs including Glycogen Storage Disease Type 3, Hepatitis B Virus, and non-alcoholic steatohepatitis.

Arcturus' versatile RNA therapeutics platforms can be applied toward multiple types of nucleic acid medicines including messenger RNA, small interfering RNA, replicon RNA, antisense RNA, microRNA, DNA, and gene editing therapeutics.

Arcturus' technologies are covered by its extensive patent portfolio (222 patents and patent applications, issued in the US, Europe, Japan, China and other countries).

Arcturus' commitment to the development of novel RNA therapeutics has led to collaborations with Janssen Pharmaceuticals, Inc., part of the Janssen Pharmaceutical Companies of Johnson and Johnson, Ultragenyx Pharmaceutical, Inc., Takeda Pharmaceutical company Ltd., CureVac AG, Synthetic Genomics Inc., Duke-NUS Medical School, and the Cystic Fibrosis Foundation.


Related Headlines