Arcturus' Inhaled mRNA Therapeutic In Cystic Fibrosis Study Highlights Potential Amid Competitive Landscape: Analyst

Zinger Key Points
  • The Phase 1b trial showed improvements in FEV1 in the four adults with cystic fibrosis after two inhaled administrations.
  • No bronchospasm or febrile reactions were observed.

Friday, Arcturus Therapeutics Holdings Inc ARCT presented Phase 1 results in healthy volunteers and Phase 1b interim data in people with cystic fibrosis for ARCT-032, an inhaled investigational mRNA therapeutic, at the European Cystic Fibrosis Conference.

ARCT-032 administration was generally safe and well tolerated, with no serious or severe adverse events in healthy volunteers and the first four dosed participants with cystic fibrosis.

The Phase 1b trial showed improvements in FEV1 (Forced Expiratory Volume in 1 second) in the four adults with cystic fibrosis after two inhaled administrations.

The absolute change in percent predicted FEV1 averaged +4.0% on Day 8 (5 days after 2nd dose). The relative change in FEV1 averaged +5.8% on Day 8.

The observed increases in FEV1 are encouraging and consistent with the previously reported data in the CF ferret model that demonstrated markedly improved mucociliary clearance (MCC) after a single dose of ARCT-032.

Of the four participants in Phase 1b to date, one had 2 Class I mutations, and the other three had F508del mutations and were being treated with Vertex Pharmaceuticals Inc’s VRTX Trikafta.

No bronchospasm or febrile reactions were observed. Dose-related, mild-to-moderate febrile reactions occurred in some healthy volunteers.

William Blair notes that despite important limitations such as the small patient sample size, differences in baseline characteristics, and significant within-patient variability, the overall data is encouraging in two key areas:

  • Firstly, in light of Vertex’s recent studies on its next-generation Vanza triple treatment, which did not show superior FEV1 improvement compared to Trikafta, William Blair is intrigued by the potential additional benefits observed in three patients with amendable mutations.
  • Second, the 4% absolute benefit and over 5.8% relative improvement seen in Class I null mutation patients—who currently lack treatment options—are comparable to the early Phase I results of ivacaftor during its CFTR modulator development.

The analyst says the results are also promising compared to a ferret model of cystic fibrosis, which accurately mimics key aspects of the disease, such as mucus buildup.

William Blair reiterates the Outperform rating on Arcturus, saying it is a leader in self-amplifying mRNA vaccine technology and advancing an mRNA therapeutics platform with a differentiated lipid nanoparticle delivery system.

Price Action: ARCT shares are down 27.9% at $30.72 at last check Friday.

Photo by Minerva Studio via Shutterstock

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