ARCT - Arcturus Therapeutics' ARCT-032: Might Be The Next Breakthrough In Cystic Fibrosis

2024-04-23 06:01:16 ET

Summary

• Arcturus Therapeutics' ARCT-032 gene therapy for cystic fibrosis has been designated as an "orphan drug" in the U.S. and Europe.
• The therapy has the potential to generate significant revenues, similar to Vertex Pharmaceuticals' Trikafta treatment.
• ARCT's financial status is currently unstable, but the company has the potential to become profitable with the success of ARCT-032 and other therapies.

Thesis

Arcturus Therapeutics Holdings ( ARCT ) recently scored a win when their ARCT-032 was designated as an "orphan drug" in the U.S. and Europe. Cystic fibrosis ((CF)) is considered a rare condition, caused by a genetic mutation. CF is more prevalent in Caucasian descendants. In the U.S. alone, it has been estimated that around 1 in 2500 babies are diagnosed with CF, while in Europe and the United Kingdom, the rate is slightly higher, with 1 in 2000 babies being diagnosed with CF.

ARCT has not officially disclosed the specifics of their ARCT-032 therapy candidate in terms of price or dose regime (single or multiple doses). However, considering that Vertex Pharmaceuticals ( VRTX ) has been able to cash in over $8 billion in 2023 alone with their Trikafta treatment, I would expect ARCT's inhalable gene therapy, which it was sponsored by the Cystic Fibrosis Foundation , to be able to retrieve similar revenues at peak sales....

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