Arcturus Therapeutics provides update on OTC deficiency and CF programmes
Betsy Goodfellow | July 2, 2024 | News story | Research and Development | Arcturus Therapeutics, OTC, Pharmacy, clinical trials, cystic fibrosis
Arcturus Therapeutics has announced mid-year updates for ARCT-810, an investigational mRNA therapeutic to treat ornithine transcarbamylase (OTC) deficiency, and ARCT-032, an investigational inhaled mRNA therapeutic for the treatment of cystic fibrosis (CF).
The update surrounding ARCT-810 for OTC deficiency surrounded the completion of enrollment into a double-blind phase 2 study for adolescents and adults at a 0.3mg/kg dose level. The patients will be randomised at a ratio of 3:1 and will be given six doses of the drug or placebo every 14 days. Treatment and follow-up are expected later this year, with full safety and biomarker data.
As for ARCT-032 for CF treatment, the company is intending to submit an investigational new drug (IND) application for a phase 2 multiple ascending dose study within the next 60 days. This study is expected to identify a safe and effective dose in Class 1 (null) and other CF patients who do not benefit from CFTR modulators.
Dr Juergen Froehlich, chief medical officer at Arcturus Therapeutics, commented: “CFTR replacement using mRNA therapeutics is an area of significant medical interest, as it may benefit null patients and potentially many other CF patients. The safety profile of ARCT-032 observed in phase 1 and after two administrations in phase 1b for patients dosed to date, including the trend of lung function improvement in the Class I participant, is encouraging. These data are supportive of expanding our clinical program to the US and other regions with a phase 2 multiple ascending dose protocol, and I look forward to a successful conduct and outcome of this planned study. We are also excited to have started patient screening for the expansion of our ARCT-810 clinical programme in the US to more severely affected OTC deficiency patients.”
Betsy Goodfellow
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