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SAN DIEGO - Arcturus Therapeutics Holdings Inc. (NASDAQ:ARCT), a $375 million market cap biotech company whose stock has seen a -21% return year-to-date, announced positive interim results from Phase 2 trials of ARCT-810, an mRNA therapeutic candidate designed to treat ornithine transcarbamylase (OTC) deficiency, a rare liver disorder. According to InvestingPro data, the company maintains a strong financial position with more cash than debt on its balance sheet.
According to the company’s press release statement, the treatment significantly reduced glutamine levels, an important biomarker of urea cycle function, to within normal range across both Phase 2 studies. In the combined analysis of eight patients, glutamine levels decreased significantly during treatment (p-value = 0.0055). While the clinical results appear promising, InvestingPro analysis shows the company is currently burning through cash rapidly, with a negative free cash flow of nearly $90 million in the last twelve months.
The U.S. Phase 2 open-label study also showed significant improvements in relative ureagenesis function (RUF), increasing from a baseline of 29.0% to 43.7% at 28 days post-fifth dose (p-value = 0.026). Two of three participants achieved RUF greater than 50%, which the company described as a clinically meaningful improvement.
Ammonia levels, which can reach toxic levels in OTC deficiency patients, remained stable and within normal range in patients receiving ARCT-810 following at least two doses and stayed stable for approximately 28 days after treatment completion.
The therapy has been generally safe and well-tolerated across single-dose Phase 1/1b and multi-dose Phase 2 studies involving 40 participants to date, including 20 with OTC deficiency. No serious infusion-related reactions were observed using the improved 3-hour intravenous regimen.
OTC deficiency is the most common urea cycle disorder, affecting approximately 10,000 people in Europe and the U.S. The condition can lead to toxic ammonia buildup in the blood, potentially causing seizures, neurocognitive impairment, coma, and death.
ARCT-810 has received Orphan Drug Designation and Fast Track Designation from the FDA, along with Orphan Medicinal Product Designation from the European Medicines Agency.
The company is continuing its Phase 2 trials and suggests these results could provide a path toward a multi-biomarker driven pivotal study. Analysts maintain an optimistic outlook, with six analysts recently revising their earnings estimates upward for the upcoming period. For deeper insights into Arcturus Therapeutics’ financial health and growth prospects, investors can access comprehensive analysis through InvestingPro, which offers exclusive access to over 30 key metrics and professional research reports.
In other recent news, Arcturus Therapeutics reported its first-quarter 2025 financial results, revealing a significant revenue shortfall with earnings per share (EPS) at a loss of $0.52, missing the forecasted profit of $3.59. Revenue was reported at $29.4 million, substantially below the anticipated $205.21 million. Despite these results, the company has extended its cash runway to the first quarter of 2028, focusing on its mRNA therapeutics pipeline for cystic fibrosis (CF) and ornithine transcarbamylase (OTC) deficiency. Analyst firms have shown mixed reactions to these developments. Scotiabank initiated coverage with a Sector Outperform rating and a $32 price target, citing potential upside in Arcturus’ therapeutic pipeline. Cantor Fitzgerald maintained an Overweight rating, suggesting confidence in the company’s operational efficiency despite financial challenges. Canaccord Genuity adjusted its price target to $66 from $68, reiterating a Buy rating and highlighting the significance of the upcoming CF program data. Meanwhile, Citi also maintained a Buy rating with a $44 target, expressing optimism about Arcturus’ strategic focus on CF and OTC treatments. These recent developments reflect a complex landscape for Arcturus Therapeutics as it navigates financial hurdles and strategic shifts.
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