Arcturus Therapeutics Updates on OTC Deficiency and CF Programs

Arcturus Therapeutics Holdings Inc. has shared updates on two of its investigational mRNA therapeutics: ARCT-810 and ARCT-032. ARCT-810 is designed to treat ornithine transcarbamylase (OTC) deficiency, while ARCT-032 aims to provide a treatment for cystic fibrosis (CF).

ARCT-810 (OTC Deficiency) Update

The Phase 2 study of ARCT-810 in Europe has reached a key milestone by completing the enrollment of eight participants at a dose level of 0.3 mg/kg. The study involves adolescents and adults, who are randomized in a 3:1 ratio to receive either the investigational drug or a placebo. Participants are administered six doses every two weeks. The safety and full biomarker data from this study are expected to be available later this year.

Following this, ARCT-810's clinical program is expanding to the United States to include patients with more severe OTC deficiency. Screening for this new phase has already begun, and further details about the U.S. trial and interim data will be shared later this year.

ARCT-032 (CF) Update

Arcturus plans to submit an Investigational New Drug (IND) application within the next 60 days for a Phase 2 study of ARCT-032. This study will involve multiple ascending doses to establish a safe and effective treatment for CF patients, particularly those with Class I mutations who do not benefit from current CFTR modulators. The Phase 2 study is supported by data from safety and tolerability trials in 32 healthy volunteers and an ongoing Phase 1b trial where no serious adverse events or febrile reactions have been observed within the targeted dose range. Notably, one participant with Class I mutations showed an improvement in lung function after two administrations of ARCT-032.

About Ornithine Transcarbamylase Deficiency

OTC deficiency is the most prevalent urea cycle disorder, primarily affecting males due to its X-linked inheritance. This metabolic disorder hampers the body’s ability to eliminate toxic waste products generated during protein digestion, leading to dangerous levels of ammonia in the blood, which can cause severe neurological damage or even death if untreated. Current treatments focus on a low-protein diet and ammonia scavengers, which are difficult to maintain and do not address the root cause of the disease. Liver transplants are the only definitive treatment but carry significant risks.

About ARCT-810

ARCT-810 is an intravenously administered mRNA therapeutic aimed at producing a functional OTC enzyme in the liver. The drug has received various designations from the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA), including Orphan Drug and Rare Pediatric Disease designations. The mRNA therapy seeks to correct the enzyme deficiency, thereby potentially preventing metabolic crises and neurocognitive damage associated with elevated ammonia levels.

About Cystic Fibrosis

Cystic fibrosis is a genetic disorder that affects approximately 40,000 people in the U.S. and 105,000 globally. It is caused by mutations in the CFTR gene, leading to insufficient chloride transport in the airways. This results in thick mucus that clogs the airways, causing respiratory infections, inflammation, and failure. While CFTR modulators provide relief for many, about 15% of patients do not respond to these treatments due to specific mutations or drug intolerance.

About ARCT-032

ARCT-032 is an inhaled mRNA therapeutic designed to deliver functional CFTR protein to the lungs. It employs Arcturus' LUNAR® lipid-mediated platform for aerosolized delivery. This therapeutic approach has shown promise in preclinical studies and has received Orphan Drug and Rare Pediatric Disease designations from the FDA. If successful, ARCT-032 could restore CFTR function in the lungs, potentially mitigating the progressive lung damage characteristic of cystic fibrosis.

Arcturus Therapeutics continues to advance these promising therapies with the goal of addressing significant unmet medical needs in rare liver and respiratory diseases.

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