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ARCT-032 · 2 trials · 2 indications
Safety and tolerability of ARCT-032 assess by determining incidence, severity and dose-relationship of AEs by dose level
Safety and tolerability of ARCT-032 assessed by determining the incidence, severity and dose-relationship of AEs by dose
| Arm | Type | Description |
|---|---|---|
| Cohort 1 | EXPERIMENTAL | Dose Level A of ARCT-032, inhaled daily for 28 days |
| Cohort 2 | EXPERIMENTAL | Dose Level B of ARCT-032, inhaled daily for 28 days |
| Cohort 3 | EXPERIMENTAL | Dose Level C of ARCT-032, inhaled daily for 28 days |
| Cohort 4 | EXPERIMENTAL | Dose level B or C of ARCT-032, inhaled once daily for 12 weeks |
| ARCT-032, Healthy Adults | EXPERIMENTAL | Ascending single doses of ARCT-032 administered to healthy adults via nebulizer |
| Placebo, Healthy Adults | PLACEBO_COMPARATOR | Single doses of 0.9% Saline administered to healthy adults via nebulizer |
| ARCT-032,. Adults with Cystic Fibrosis | EXPERIMENTAL | Two doses of ARCT-032 administered to adults with Cystic Fibrosis via nebulizer |
| Name | Type | Description |
|---|---|---|
| ARCT-032 | BIOLOGICAL | CFTR mRNA formulated in lipid nanoparticles |
| Placebo | OTHER | Normal saline |
Key Inclusion Criteria: 1. Confirmed diagnosis of Cystic Fibrosis 2. Not eligible for CFTR modulator therapy or not taking CFTR modulators for at least 60 days prior to dosing (e.g. due to intolerance, poor response, or lack of access to modulators) 3. FEV1 between 40% to 100% (cohorts 1-3) and 45%...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 8 | PHASE3 | VX-121/TEZ/D-IVA |
| Sionna Therapeutics, Inc. | SION | 2 | PHASE2 | SION-719 |
| BiomX Inc. | PHGE | 1 | PHASE2 | BX004 |
| 4D Molecular Therapeutics, Inc. | FDMT | 1 | PHASE2 | 4D-710 |
| Arcturus Therapeutics Holdings, Inc. | ARCT | 1 | PHASE2 | ARCT-032 |
| Krystal Biotech, Inc. | KRYS | 1 | PHASE1 | KB407 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
ARCT-032 is an investigational small molecule being developed for the treatment of Cystic Fibrosis, a genetic condition caused by CFTR gene mutations. It is currently in Phase 2 clinical development and is not yet approved by regulatory authorities.
ARCT-032 targets the CFTR protein, which is defective in people with Cystic Fibrosis due to mutations in the CFTR gene. By modulating CFTR function, the drug aims to address the underlying cause of the disease.
ARCT-032 is being developed by Arcturus Therapeutics Holdings Inc., a biopharmaceutical company listed on the stock exchange under the ticker symbol ARCT.
ARCT-032 is currently in Phase 2 clinical development for Cystic Fibrosis. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory agencies and is still undergoing clinical trials to assess its safety and efficacy.
ARCT-032 has two clinical trials. The active Phase 2 trial, NCT06747858, is recruiting 33 participants with Cystic Fibrosis in the United States. A completed Phase 1 trial, NCT05712538, enrolled 39 healthy adults and adults with Cystic Fibrosis in New Zealand.
ARCT-032 is a distinct investigational small molecule targeting CFTR. It is not the same as other CFTR modulators, as it is being developed specifically by Arcturus Therapeutics for Cystic Fibrosis and is in early-stage clinical trials.