SAN DIEGO - Arcturus Therapeutics (NASDAQ:ARCT) Holdings Inc. (NASDAQ:ARCT), a biotechnology firm specializing in mRNA-based treatments, has announced significant progress in its clinical programs for ARCT-810 and ARCT-032, targeting rare diseases ornithine transcarbamylase (OTC) deficiency and cystic fibrosis (CF), respectively.
The Phase 2 study of ARCT-810, an investigational mRNA therapeutic for OTC deficiency, has completed enrollment in Europe and the UK, with eight subjects receiving the 0.3 mg/kg dose level. The study is ongoing, with a 3:1 randomization of ARCT-810 to placebo, and safety and biomarker data are expected later in the year. Additionally, the company has initiated screening in the U.S. for patients with more severe OTC deficiency to expand its clinical program.
OTC deficiency, a genetic disorder resulting in high ammonia levels and potential neurotoxicity, currently has no cure other than liver transplant. ARCT-810 aims to restore normal OTC enzyme function in the liver, potentially preventing metabolic crises. The treatment has received Orphan Medicinal Product Designation from the European Medicines Agency (EMA) and Orphan Drug Designation from the U.S. Food and Drug Administration (FDA).
For cystic fibrosis, Arcturus plans to submit an Investigational New Drug (IND) application within the next 60 days for a Phase 2 study of ARCT-032, an inhaled mRNA therapeutic designed to express functional CFTR protein in the lungs. The upcoming study follows encouraging results from Phase 1 trials, including a CF participant with Class I mutations showing improved lung function after two administrations. ARCT-032 has also been granted Orphan Medicinal Product and Orphan Drug Designations by the EMA and FDA.
Cystic fibrosis, characterized by dysfunctional CFTR protein leading to severe respiratory complications, affects nearly 40,000 people in the U.S. and over 105,000 worldwide. Approximately 15% of CF patients do not benefit from current CFTR modulator medications.
Arcturus's commitment to addressing unmet medical needs in liver and respiratory rare diseases is underscored by its proprietary technologies, including the LUNAR® lipid-mediated delivery platform. The company, founded in 2013, is known for developing the first self-amplifying mRNA COVID vaccine approved globally.
This update is based on a press release statement from Arcturus Therapeutics Holdings Inc.
In other recent news, Arcturus Therapeutics Holdings Inc. has made significant strides in its operations. The biotechnology company has reported a net loss of $26.8 million for Q1 2024, while promising interim results from their Phase 1b trial of ARCT-032, an inhaled mRNA therapeutic for cystic fibrosis, have been disclosed. The trial showed an average absolute increase of +4.0% in forced expiratory volume on Day 8.
Investment firms Piper Sandler and Citi have maintained their positive ratings on Arcturus, with price targets of $140.00 and $48.00 respectively. These ratings were influenced by the promising Phase 1b trial results and the anticipation of significant developments in the company's four main programs in the second half of 2024.
ARK ETFs, managed by Cathie Wood, also showed a bullish stance on Arcturus, acquiring 58,363 shares through its ARKG ETF, amounting to a $1,857,110 investment. This move signals a strengthening position in the company's portfolio.
Additionally, Arcturus Therapeutics announced the appointment of Dr. Moncef Slaoui to its Board of Directors. Dr. Slaoui, recognized for his leadership in Operation Warp Speed and his tenure at GlaxoSmithKline (NYSE:GSK), brings extensive experience in pharmaceutical innovation and vaccine development to the company.
InvestingPro Insights
Arcturus Therapeutics Holdings Inc. (NASDAQ:ARCT) has been in the spotlight for its innovative mRNA-based therapies, with notable advancements in their clinical programs. As investors consider the potential of Arcturus's pipeline, it's imperative to examine the company's financial health and market performance to understand the full picture.
InvestingPro data indicates that Arcturus has a market capitalization of $639.09 million, with a negative Price-to-Earnings (P/E) ratio of -5.92, reflecting challenges in profitability. The company's revenue has seen a significant decline over the last twelve months as of Q1 2024, with a decrease of 55.69%. Additionally, the gross profit margin stands at a concerning -55.1%, indicating that the company spends more on the cost of goods sold than it earns in revenue.
Despite these financial headwinds, an InvestingPro Tip reveals a silver lining: Arcturus holds more cash than debt on its balance sheet, which may provide some financial flexibility as it continues to invest in its clinical programs. Moreover, the company's liquid assets exceed its short-term obligations, suggesting a stable position to meet its immediate financial liabilities.
However, investors should be aware of the stock's recent performance, which has been quite volatile. According to another InvestingPro Tip, the stock has taken a significant hit over the last week and has fared poorly over the last month. This volatility may be a factor for risk-averse investors to consider.
For those looking to dive deeper into Arcturus's financials and stock performance, InvestingPro offers additional insights. There are 11 more InvestingPro Tips available, which could help investors make a more informed decision. To gain access to these valuable tips, use the coupon code PRONEWS24 to get up to 10% off a yearly Pro and a yearly or biyearly Pro+ subscription.
Arcturus's progress in developing treatments for rare diseases like OTC deficiency and cystic fibrosis is commendable, and its proprietary LUNAR® delivery platform could be a game-changer. As the biotech firm continues its journey, keeping an eye on financial metrics and market performance will be crucial for stakeholders.
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