Arcturus Therapeutics Presents New Data at 47th European Cystic Fibrosis Conference

Arcturus Therapeutics, a company specializing in messenger RNA (mRNA) medicines, revealed new clinical data on its investigational therapeutic, ARCT-032, at the 47th European Cystic Fibrosis Conference in Glasgow, Scotland. The data highlights the results from Phase 1 clinical trials involving both healthy volunteers and individuals with cystic fibrosis (CF).

Clinical Trial Findings

ARCT-032, an inhaled mRNA therapeutic, demonstrated a favorable safety profile in these trials. Among the 36 study participants, which included four adults with CF, no serious adverse events were reported. The Phase 1b interim analysis provided encouraging results, particularly in participants with Class 1 mutations and those with F508del mutations who were concurrently receiving Trikafta® treatment.

A noteworthy trend in improved lung function was observed. Specifically, the average absolute increase in Forced Expiratory Volume in 1 second (FEV1) was +4.0%, reaching up to +9% at the highest end, with a relative change of +5.8% FEV1 on Day 8 after two doses of ARCT-032. These results align well with pre-clinical data derived from CF ferret models, which also showed significant improvement in mucociliary clearance post-administration of ARCT-032.

Detailed Participant Data

In the Phase 1b trial, the four CF participants showed varied improvements in their lung function metrics:

- A 24-year-old female with F508del mutation on Trikafta® showed an increase in FEV1 from 83% to 85%.
- A 43-year-old male with F508/G85E mutations on Trikafta® saw an increase from 72% to 81%.
- A 27-year-old female with F508del mutation on Trikafta® experienced an increase from 68% to 69%.
- A 40-year-old female with G542X (Class I) mutation not on Trikafta® exhibited an increase from 45% to 49%.

No incidences of bronchospasm or febrile reactions were detected in the CF participants. In healthy volunteers, mild to moderate febrile reactions were observed, which included symptoms such as elevated temperature, headache, muscle aches, back pain, or nausea. These reactions were dose-related and transient declines in FEV1 were mitigated through pretreatment with albuterol, a standard bronchodilator. Importantly, no serious adverse events or dose-limiting toxicities were encountered across any dose levels.

Understanding Cystic Fibrosis

Cystic fibrosis is a severe genetic disorder that affects the respiratory system. Mutations in the CFTR gene lead to a dysfunctional CFTR protein, impairing chloride transport in the airways. This results in thick, sticky mucus that obstructs airways and leads to chronic respiratory infections, inflammation, and potentially fatal respiratory failure. Current CFTR modulator therapies aim to enhance CFTR function but are not effective for all mutation types.

About ARCT-032

ARCT-032 is an inhaled mRNA therapeutic designed to address CF by delivering CFTR mRNA to the lungs using Arcturus' proprietary LUNAR® lipid-mediated platform. This approach aims to restore CFTR protein function, thereby mitigating the progression of lung disease in CF patients. The therapeutic has received Orphan Medicinal Product Designation from the European Medicines Agency (EMA) and Orphan Drug Designation from the U.S. FDA.

About Arcturus Therapeutics

Established in 2013 and headquartered in San Diego, California, Arcturus Therapeutics is a leader in developing mRNA-based medicines and vaccines. The company's technology portfolio includes LUNAR® lipid-mediated delivery and STARR® mRNA Technology, making advances in treating diseases like cystic fibrosis and developing vaccines for infectious diseases such as COVID-19 and influenza.

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