Arcturus Therapeutics: Clinical Trials for OTC

Founded in 2013, Arcturus Therapeutics, is a United States based pharmaceutical company, which is focused on developing life-changing therapies and vaccines for those with rare disorders, including OTC deficiency. Arcturus develops medicines made with a molecule known as messenger RNA (mRNA). Each mRNA is uniquely designed to treat a specific disease.

Arcturus is developing ARCT-810, an investigational mRNA therapy that is designed to address the underlying cause of OTC deficiency. When a person has OTC deficiency, the body is not able to process ammonia in the same way and a buildup of ammonia can occur, causing distress and illness.

 The mRNA in ARCT-810 is designed to be a template for making a healthy OTC enzyme, which removes excess ammonia from the body through a process known as the urea cycle. Arcturus uses its lipid nanoparticle technology called LUNAR to deliver the mRNA template for OTC to the liver, where the OTC enzyme is made. Arcturus believes that mRNA and the LUNAR lipid nanoparticle work together to make a functioning OTC enzyme, which removes excess ammonia from the body.

ARCT-810-04 will be an open-label study that will enroll up to nine individuals 12 years and older with OTC deficiency. ARCT-810 is an investigational mRNA treatment designed to treat the root cause of OTC deficiency. The mRNA in ARCT-810 acts as a template for making the OTC enzyme. The goal of the study is to evaluate the safety and tolerability of multiple dosing levels of ARCT-810 and its efficacy in restoring urea cycle function.

The study is being conducted at Uncommon Cures which is located in Chevy Chase, MD (right outside Washington, D.C) and eligible study participants will receive 5 doses of ARCT-810 via intravenous (IV) infusion every two weeks at one of three dose levels. There is no placebo in this study, and participants will have approximately 13 study visits over a 16-week period, including the screening visit.

The ARCT-810-04 study will cover for eligible participants all study-related costs and arrange accommodations to fit your schedule. This includes flights, lodging, transportation, meals, and other necessary expenses. Participants will also receive a stipend to help with missed workdays, and support is available for caregivers or family members traveling with you.

International participation is allowed and translation services are available.

If you or someone you know are interested in learning about the ARCT-810-03 study, please visit https://otcmrnastudy.com

For all other inquiries, contact community@arcturusrx.com or visit www.arcturusrx.com