Arcturus Therapeutics Begins Phase 2 Studies for CF Treatment

Arcturus Therapeutics Initiates Dosing in Phase 2 Studies

SAN DIEGO -- Arcturus Therapeutics Holdings Inc. (the Company, Arcturus; Nasdaq: ARCT), a pioneer in messenger RNA medicines, is thrilled to announce the initiation of dosing for participants in its Phase 2 multiple ascending dose studies targeting Cystic Fibrosis (CF) and Ornithine Transcarbamylase (OTC) Deficiency.

Progress in Phase 2 CF Study

In an exciting development for the CF community, the first participants in the Phase 2 clinical trial are set to commence treatment with ARCT-032. The regimen includes daily treatments over a 28-day duration designed to assess the safety and efficacy of this innovative therapeutic approach.

Details of the Study

This study, labeled as NCT06747858, aims to trial the effectiveness of ARCT-032 in individuals with cystic fibrosis, a life-threatening genetic condition. Expectations are high that ARCT-032 will meet the significant medical needs for patients who do not respond to current treatment options.

OTC Deficiency Treatment Study

The research expands further into the realm of metabolic disorders with the commencement of the inaugural dosing for an OTC deficient participant, who will be receiving 0.5 mg/kg of ARCT-810. This individual started their treatment in the United States. The regimen consists of five intravenous infusions spread over a two-month window.

Advancing Innovations

Previously, the company completed a dosing phase in a European placebo-controlled study involving eight participants administered 0.3 mg/kg of ARCT-810. With the expansion of its clinical program in the United States, the research will help ascertain the therapeutic potential of this groundbreaking treatment.

Insights from Leadership

Dr. Pad Chivukula, Chief Scientific Officer of Arcturus Therapeutics, expressed enthusiasm regarding the progress of these Phase 2 studies. He emphasized, "ARCT-032 has the potential to address the significant unmet medical need in the CF community for those who do not qualify for or benefit from available treatment options."

Understanding Cystic Fibrosis

Cystic fibrosis is a genetic disorder that severely impacts respiratory function. It is caused by mutations in the CFTR gene, resulting in the malfunction of the CFTR protein, essential for maintaining the balance of salt and water in tissues. Currently, around 40,000 individuals in the U.S. confront this condition, with over 105,000 living with it globally.

Standard Treatments

Numerous patients with cystic fibrosis benefit from CFTR modulators, but approximately 15% do not due to the absence of functional CFTR proteins or intolerance to existing therapies. Thus, innovative solutions like ARCT-032 are critical in filling this therapeutic gap.

A Closer Look at ARCT-032

ARCT-032 represents a novel medicinal approach, administrating mRNA therapeutically designed to generate functional CFTR in the lungs of patients. With Orphan Medicinal Product Designation from the European Medicines Agency and Orphan Drug Designation from the FDA, it is tailored to restore CFTR function, making strides to mitigate the disease's progression.

Potential Impact

The approach of targeting lung disease, a primary mortality factor for CF patients, carries potential for substantial improvements in quality of life and survival rates, essential as the need for functional CFTR proteins becomes paramount.

Focus on Ornithine Transcarbamylase Deficiency

OTC deficiency is a severe genetic disorder disrupting the body's ability to eliminate ammonia, a toxic waste product. The disease results from mutations in the OTC gene, affecting critical metabolic processes. Current treatment options often involve maintaining strict dietary controls and nitrogen scavenging medications, yet these do not tackle the root causes of the disorder.

ARCT-810's Role

ARCT-810, an investigational mRNA therapy given intravenously, aims to express the normal OTC enzyme within the liver cells of affected individuals. By addressing the root deficiency in OTC enzyme production, it holds the promise of revolutionizing care for OTC deficiency patients.

About Arcturus Therapeutics

Founded in 2013, Arcturus Therapeutics, located in San Diego, California, focuses on advancing mRNA-based medicines and vaccines. Their diverse pipeline demonstrates promise in treating conditions such as cystic fibrosis and OTC deficiency, utilizing their proprietary delivery platforms for optimized therapeutic outcomes.

Looking Ahead

As mRNA technologies continue to evolve, Arcturus’ dedication to pioneering these innovative therapies reinforces its commitment to addressing pressing healthcare needs around the globe. Their endeavor to restore health through cutting-edge science solidifies their role at the forefront of modern medicine.

Frequently Asked Questions

What is the main goal of Arcturus's Phase 2 studies?

The primary goal is to evaluate the safety and effectiveness of ARCT-032 for cystic fibrosis and ARCT-810 for OTC deficiency.

How many participants are included in these studies?

The studies include multiple ascending dose participants, with specific dosing regimens established for each treatment.

What are ARCT-032 and ARCT-810 targeting?

ARCT-032 targets cystic fibrosis by restoring CFTR protein function, while ARCT-810 focuses on replenishing the OTC enzyme in the liver.

What challenges do cystic fibrosis patients face?

Many patients struggle with inadequate treatment options, with around 15% not benefiting from existing medicines due to their specific genetic variations.

How is Arcturus positioned in the mRNA therapeutic field?

Arcturus is at the forefront, pioneering mRNA technologies with a focus on rare diseases, leveraging their proprietary platforms and extensive patent portfolio.

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