Arcturus Therapeutics Shifts Focus to Advanced mRNA Therapies, Extends Cash Runway

Arcturus Therapeutics, a prominent player in the mRNA therapeutics space, has announced a strategic realignment of its pipeline, focusing on its more advanced mRNA therapy programs while discontinuing early-stage vaccine candidates. This move, revealed alongside the company's first-quarter earnings report, is set to extend Arcturus' cash runway into 2028.

Pipeline Prioritization and Financial Strategy

Arcturus has made the decision to concentrate its research and development efforts on two key programs: cystic fibrosis (CF) and ornithine transcarbamylase (OTC) deficiency. This strategic shift involves scaling back work on early-stage candidates, particularly in the vaccine space.

Andrew Sassine, Chief Financial Officer of Arcturus, addressed the decision during the company's earnings call, stating, "It was not an easy decision. Due to the current market environment and uncertainty regarding our regulatory process, we have made the decision to focus our research and development expenditures on our CF and OTC deficiency programs exclusively."

The financial implications of this move are significant. Arcturus reported $273.8 million in cash, cash equivalents, and restricted cash at the end of the first quarter. With the pipeline restructuring, the company now projects its cash runway to extend until the first quarter of 2028, providing a substantial cushion for its focused development efforts.

Advancing mRNA Therapies

Arcturus is making notable progress in its prioritized programs. In cystic fibrosis, the company is advancing ARCT-032, an inhalable mRNA therapy currently in Phase II trials. This study, known as LUNAR-CF, is targeting patients who cannot be treated with CFTR modulators. Arcturus expects to complete enrollment by the end of the year, with data from the first two cohorts anticipated by mid-2025.

CEO Joseph Payne provided insights into the potential regulatory pathway, suggesting that a target of 100 enrollees could be "reasonable" for paving the way toward a regulatory submission. However, he emphasized that this number has not yet been confirmed by regulators and may depend on the success of the Phase II results.

In addition to CF, Arcturus is progressing with ARCT-810, its OTC deficiency candidate. This program is also in Phase II development, with data expected in the second quarter.

Impact on Collaborations and Future Outlook

While the company has not explicitly named the assets to be discontinued, Arcturus' pipeline page lists four investigational vaccines, including two Phase I influenza vaccines and two preclinical vaccines against undisclosed infectious diseases. The fate of these programs, developed in collaboration with CSL Seqirus under a 2022 deal that provided Arcturus with a $200 million upfront payment, remains unclear.

Despite the pipeline pruning, Arcturus continues to advance its COVID-19 vaccine, Kostaive, which has already gained approval in the European Union and Japan. The company plans to file for U.S. approval of Kostaive by the third quarter of this year, demonstrating its ongoing commitment to expanding the global presence of this asset.

As Arcturus Therapeutics refocuses its efforts on advanced mRNA therapies, the pharmaceutical industry will be watching closely to see how this strategic shift impacts the company's long-term prospects and the broader landscape of mRNA-based treatments.