Arcturus Therapeutics Holdings Inc. (NASDAQ: ARCT) on Wednesday revealed interim Phase 2 trial data for ARCT-810, its investigational treatment for ornithine transcarbamylase (OTC) deficiency.
Following the announcement, the stock gained more than 6%.
OTC deficiency is a rare genetic disorder that stops the body from properly breaking down and removing nitrogen, leading to a dangerous buildup of ammonia in the blood.
Furthermore, the biotechnology firm debuted LUNAR 2.0, a next-generation mRNA delivery platform, alongside updates on its expanding liver disease pipeline.
Strategic myNEO Acquisition
In a move to expand its technological capabilities, the company signed a definitive agreement to purchase myNEO, a partner since 2024. Arcturus expects to finalize the transaction in October.
The buyout secures a unique computational engine backed by proprietary intellectual property.
Arcturus plans to use the technology to strengthen its artificial intelligence infrastructure, upgrade mRNA design quality, and improve overall target identification.
The company reported $191.5 million in cash and cash equivalents as of June 30, 2026.
ARCT-810 Phase 2 Progress
Interim findings from the Phase 2 ARCT-810 study showed the treatment remains generally safe and well-tolerated among participants.
Patients successfully maintained or reduced their first-morning fasting ammonia levels within normal limits, even when they increased their protein consumption.
The therapy also lowered glutamine levels effectively. Specifically, participants receiving the 0.5 mg/kg dose achieved normal average glutamine levels during treatment.
Additionally, researchers observed weight gain across all participants.
Introducing LUNAR 2.0
Arcturus launched LUNAR 2.0, an advanced mRNA delivery system featuring upgraded lipids and a refined formulation process.
In non-human primate testing, the platform showed a 40-fold expression improvement over the original LUNAR 1.0 platform.
Furthermore, LUNAR 2.0 demonstrated 38 times the potency of ATX-95, the primary lipid used in ARCT-810.
The upgraded technology opens doors for new therapies targeting conditions previously considered inaccessible, including phenylketonuria and gout.
ARCT-2601 Pipeline Advancement
Utilizing the LUNAR 2.0 system, Arcturus developed ARCT-2601 as its next-generation investigational treatment for OTC deficiency.
Preclinical studies indicate ARCT-2601 shares a similar safety profile with ARCT-810.
Arcturus aims to begin dosing OTC deficiency patients aged 12 and older with ARCT-2601 by the end of the year.
The company will integrate this new drug into the existing Phase 2 study through an amended protocol.
Based on earlier data, ARCT-2601 could offer patients the advantage of lower or less frequent dosing.
ARCT Price Action: Arcturus Therapeutics shares were up 6.54% to $14.17 at the time of publication on Thursday, according to market data.
