SAN DIEGO - Arcturus Therapeutics (NASDAQ:ARCT) Holdings Inc. (NASDAQ: ARCT), a biotechnology company specializing in mRNA medicines, has reported encouraging interim results from their Phase 1b trial of ARCT-032, an inhaled mRNA therapeutic for cystic fibrosis (CF). The announcement was made following the presentation of their findings at the 47th European Cystic Fibrosis Conference in Glasgow, Scotland.
The trial involved 36 participants, including four adults with CF, and showed that ARCT-032 was generally safe and well tolerated with no serious adverse events reported. The interim data highlighted an average absolute increase of +4.0% in forced expiratory volume (FEV1) on Day 8, five days after the second dose, with a relative change of +5.8%. FEV1 is a key measure of lung function in CF patients.
The four CF participants included one with Class 1 mutations and three with F508del mutations, the latter also being treated with the CFTR modulator Trikafta®. These early findings are in line with preclinical data suggesting improved mucociliary clearance following treatment with ARCT-032.
Dr. Juergen Froehlich, Chief Medical Officer of Arcturus, expressed optimism about the results, stating, "It is encouraging to see favorable lung function improvements in all currently dosed CF participants in this early study." The company is looking forward to completing the Phase 1b trial and advancing to a larger, multiple-dose clinical study.
Cystic fibrosis is a genetic disease that affects the lungs and digestive system, leading to severe respiratory and digestive issues. Mutations in the CFTR gene cause thick, sticky mucus to accumulate in the lungs, creating an environment prone to infections and inflammation. Current treatments, such as CFTR modulators, aim to alleviate symptoms but may not be effective for all CF patients due to their specific genetic mutations.
ARCT-032 has been granted Orphan Medicinal Product Designation by the European Medicines Agency (EMA) and Orphan Drug Designation by the U.S. Food and Drug Administration (FDA). Arcturus' technology aims to deliver a functional copy of the CFTR mRNA to the lungs, potentially restoring CFTR activity and improving lung function.
The company, founded in 2013, has a diverse pipeline of RNA therapeutic candidates, including treatments for ornithine transcarbamylase deficiency and CF, as well as mRNA vaccine programs for COVID-19 and influenza. Arcturus' proprietary technologies include the LUNAR® lipid-mediated delivery and STARR® mRNA Technology.
This report is based on a press release statement issued by Arcturus Therapeutics Holdings Inc.
In other recent news, Arcturus Therapeutics has reported promising trial results for its cystic fibrosis treatment, and has retained an Overweight rating from Piper Sandler, with a steady price target of $140.00. Citi has also reaffirmed its Buy rating on Arcturus, maintaining a stock price target of $48.00. Both investment firms are awaiting additional data from Arcturus's cystic fibrosis program, expected to be released at the European CF conference.
Arcturus has also been progressing with its vaccine and flu programs, with a substantial order of approximately 4 million doses of its Kostaive vaccine in Japan, and anticipates its Arcalis manufacturing facility to be operational in the third quarter of 2024. Furthermore, the company is making strides in its OTC program, with interim data and regulatory updates expected soon.
However, Arcturus reported a net loss of $26.8 million for the first quarter of 2024, despite a strong cash position and expectations of future milestone payments to support its vaccine programs. The company's cash runway extends for at least three years, and it is preparing for commercial sales in Japan. These are all recent developments in the company's journey.
InvestingPro Insights
Arcturus Therapeutics Holdings Inc. (NASDAQ: ARCT) has recently made headlines with their promising Phase 1b trial results for ARCT-032, their mRNA therapeutic for cystic fibrosis. As investors and stakeholders look closely at the company's potential, InvestingPro data and tips provide a deeper financial perspective on Arcturus.
InvestingPro data shows that Arcturus has a market capitalization of $1.12 billion, reflecting investor confidence in the company's growth potential. Despite a challenging revenue environment with a year-over-year decrease of 55.69% in the last twelve months as of Q1 2024, Arcturus has maintained impressive gross profit margins of 87.19%. This suggests that while the company is facing headwinds in revenue growth, it is still managing to control costs effectively in its production processes.
InvestingPro Tips highlight that Arcturus holds more cash than debt on its balance sheet, indicating a solid financial position to fund ongoing research and development. Additionally, the company's liquid assets exceed its short-term obligations, providing a cushion for operational expenses.
While the company's stock price has experienced significant volatility, it has shown a strong return over the last month, with a 54.57% increase. This could be a reflection of the positive clinical trial results and the potential market opportunity for ARCT-032. However, it's important to note that analysts have revised their earnings downwards for the upcoming period, and the stock's RSI suggests it is in overbought territory, which could indicate a near-term pullback.
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