Arcturus Begins Phase 2 Dosing for CF and OTC Deficiency
Arcturus Therapeutics Holdings Inc., a pioneering company in mRNA medicine, announced that it has initiated Phase 2 multiple ascending dose studies for two major conditions—cystic fibrosis (CF) and ornithine transcarbamylase (OTC) deficiency. These studies began in December 2024, marking an important milestone for the company’s investigational mRNA therapies, ARCT-032 and ARCT-810.
The Phase 2 study for cystic fibrosis (CF) is part of Arcturus’ efforts to address the significant unmet medical needs in the CF community. ARCT-032, an inhaled investigational mRNA therapy, aims to help individuals who do not benefit from existing treatments, including those who either cannot qualify for or do not respond to CFTR modulator therapies. Dr. Pad Chivukula, Chief Scientific Officer of Arcturus Therapeutics, expressed his optimism, noting that ARCT-032 could significantly improve the quality of life for CF patients who have limited treatment options.
Each participant in the Phase 2 CF study (NCT06747858) will receive daily doses of ARCT-032 for a period of 28 days. This phase builds on previous studies and continues to advance the investigation into how ARCT-032 may restore normal CFTR protein function in the lungs of individuals with CF, addressing the lung disease that leads to the progressive deterioration of lung function. ARCT-032 has already received Orphan Medicinal Product Designation from the European Medicines Agency (EMA) and Orphan Drug Designation from the U.S. Food and Drug Administration (FDA).
In parallel, Arcturus has initiated dosing in its Phase 2 clinical study for OTC deficiency with the first participant receiving ARCT-810, an investigational mRNA therapy. ARCT-810 is designed to address the underlying cause of OTC deficiency by enabling liver cells to produce the functional OTC enzyme. This disorder, which is the most common urea cycle disorder, can result in severe neurological consequences, including seizures and coma, due to the inability to remove toxic ammonia from the body. ARCT-810 aims to correct this deficiency by providing the mRNA blueprint for producing the functional OTC enzyme.
The first participant in the OTC deficiency study received a dose of 0.5 mg/kg ARCT-810, marking the start of the multiple ascending dose study in the United States. Participants in this study will receive five intravenous infusions over two months. This expansion follows the completion of an earlier placebo-controlled European study with 8 participants (0.3 mg/kg dose). As a result, Arcturus is now advancing its clinical program for OTC deficiency into the U.S., with an open-label, multiple-dose design to further evaluate ARCT-810.
Both ARCT-032 and ARCT-810 are critical to advancing therapies for rare diseases that currently have no cure. The CF study is particularly relevant for the approximately 15% of CF patients who do not respond to CFTR modulator treatments, and ARCT-810 offers a potential life-changing alternative for those suffering from OTC deficiency, a disorder that typically requires a liver transplant. ARCT-810 is the only mRNA therapy currently in clinical trials that aims to restore OTC enzyme function in the liver, offering a potentially transformative treatment for these patients.
Arcturus is well positioned in the field of mRNA therapeutics, with a broad portfolio that extends beyond CF and OTC deficiency. The company has developed KOSTAIVE®, the world’s first self-amplifying mRNA (sa-mRNA) COVID-19 vaccine, which has been approved. Arcturus is also collaborating globally, notably with CSL Seqirus on mRNA vaccines, and has a joint venture, ARCALIS, in Japan for the production of mRNA vaccines and therapeutics. In addition to CF and OTC deficiency, Arcturus’ pipeline includes RNA-based treatments for various diseases, using its advanced LUNAR® lipid-mediated delivery platform, which allows for precise mRNA delivery to targeted cells.
The company’s commitment to advancing RNA-based medicines is supported by a robust intellectual property portfolio, with over 400 patents and patent applications worldwide. Arcturus’ cutting-edge technologies and partnerships position it as a leader in the growing mRNA therapeutics and vaccine sector, with the potential to significantly impact the treatment of rare diseases like CF and OTC deficiency, as well as infectious diseases such as COVID-19 and influenza.
With its progress in Phase 2 studies for CF and OTC deficiency, Arcturus continues to push the boundaries of what is possible in the field of mRNA medicine, aiming to offer new hope to patients with unmet medical needs.
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