Arcturus Therapeutics

Pipeline

Arcturus Therapeutics Ltd. is an RNA medicines company with enabling technologies — UNA Oligomer™ chemistry and LUNAR™ nanoparticle delivery. Arcturus’ versatile RNA therapeutics platform can be applied toward all types of RNA medicines including small interfering RNA, messenger RNA, antisense RNA, microRNA and gene editing therapeutics. The company owns LUNAR™ nanoparticle delivery and unlocked nucleomonomer agent (UNA) technology including UNA Oligomers™, which are covered by its patent portfolio (77 patents and patent applications, issued in the U.S. and other countries). Arcturus’ proprietary UNA technology can be used to target any gene in the human genome, as well as viral genes, and other species for therapeutic purposes. The Company’s commitment to the development of novel RNA therapeutics has led to collaborations and license agreements with Janssen Pharmaceuticals, Inc. (A Johnson and Johnson Company) and Ultragenyx Pharmaceutical, Inc.

PreclinicalDiscoveryClinical
name

LUNAR-OTC

indication
Ornithine Transcarbamylase Deficiency
api
mRNA

WHOLLY OWNED PROGRAM

name

LUNAR-CF

indication
Cystic Fibrosis
api
mRNA

WHOLLY OWNED PROGRAM

GRANT by CFFT

Overview

We have partnered with a number of industry leaders and have a robust and diverse preclinical drug development pipeline. In collaboration with our development partners, we are leveraging the LUNAR and UNA platforms to develop nucleic acid medicines for diseases with significant unmet medical needs and accelerated clinical paths.

Our external development partners include Janssen Pharmaceuticals, Inc., Ultragenyx Pharmaceutical Inc., Takeda Pharmaceutical Company Limited, and Synthetic Genomics, Inc.



LUNAR-OTC

In a wholly owned program, Arcturus is developing a messenger RNA (mRNA) medicine to treat ornithine transcarbamylase deficiency, a life-threatening genetic disease that affects over 10,000 people worldwide.

Ornithine transcarbamylase deficiency
Ornithine transcarbamylase (OTC) deficiency is the most common urea cycle disorder. A lack of the OTC enzyme in liver cells results in high blood ammonia levels and can cause seizures, coma, and death in untreated patients. There is currently no cure for OTC deficiency.

Our LUNAR-OTC solution
Arcturus is working to develop RNA medicines that enable OTC patients to make healthy functional OTC enzyme in their liver cells. Preclinical studies have shown that our proprietary LUNAR delivery platform safely and effectively delivers OTC mRNA to liver cells in a mouse model of OTC deficiency, resulting in restoration of disease markers to normal levels.

LUNAR-CF

Arcturus is collaborating with the Cystic Fibrosis Foundation to develop a messenger RNA (mRNA) medicine to treat cystic fibrosis.

Cystic fibrosis
Cystic fibrosis is a common life-threatening genetic disease in the United States. The disease is caused by over 2,000 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. There are over 70,000 patients world-wide.

There are no FDA-approved drugs that can treat all 2,000 CFTR mutations. The FDA has approved three CFTR modulator therapies, Kalydeco®, Orkambi® and Symdeko® (Vertex Pharmaceuticals Inc.), to treat fewer than 40 cystic fibrosis-causing mutations. These drugs do not treat the underlying genetic cause of cystic fibrosis.

Our LUNAR-CF solution
We are developing an mRNA therapeutic using our LUNAR delivery platform to deliver normal CFTR mRNA into airway epithelial cells. This allows airway cells to produce functional CFTR protein using their normal cellular machinery. This approach will be the first to treat the underlying defect that causes cystic fibrosis-dysfunctional or absent CFTR protein-in all patients, regardless of mutation type.

We have completed preclinical proof of concept studies, demonstrating that LUNAR is able to deliver mRNA efficiently into lung epithelial cells in animals and is compatible with nebulization. In cell-based assays, we have also demonstrated that our lead CFTR mRNA has improved functional activity and longer duration of protein expression compared to a reference CFTR mRNA.