Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
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 | 9 | PHASE3 | VX-121/TEZ/D-IVA |
| Sionna Therapeutics, Inc. | SION | 2 | PHASE2 | SION-719 |
| BiomX Inc. | PHGE | 1 | PHASE2 | BX004 |
| Arcturus Therapeutics Holdings, Inc. | ARCT | 1 | PHASE2 | ARCT-032 |
| 4D Molecular Therapeutics, Inc. | FDMT | 1 | PHASE2 | 4D-710 |
| Krystal Biotech, Inc. | KRYS | 1 | PHASE1 | KB407 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |