Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
IONIS-ENaCRx · 1 trial · 2 indications
| Arm | Type | Description |
|---|---|---|
| IONIS-ENaCRx | EXPERIMENTAL | Ascending single and multiple doses of IONIS-ENaCRx inhaled or nebulized. |
| Placebo | PLACEBO_COMPARATOR | Placebo comparator calculated volume to match active comparator inhaled or nebulized. |
| Name | Type | Description |
|---|---|---|
| IONIS-ENaCRx | DRUG | Ascending single and multiple doses of IONIS-ENaCRx inhaled or nebulized. |
| Placebo | DRUG | Placebo comparator calculated volume to match active comparator inhaled or nebulized. |
Inclusion Criteria (Healthy Volunteers) 1. Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal. 2. Males must be surgically sterile or, abstinent or, if engaged in sexual relations with a woman of child-bearing potential, the subject or the subject's non...
Top 6 of 7 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 8 | PHASE3 | Alyftrek, Trikafta, Ivacaftor, VX-581, VX-272 |
| 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 |
| Roivant Sciences Ltd. | ROIV | 1 | PHASE1 | SP-101 |
IONIS-ENaCRx is an investigational small molecule developed by Ionis Pharmaceuticals, Inc. for cystic fibrosis, a respiratory disease. It has been studied in a Phase 1/2a clinical trial that enrolled 98 participants, including healthy volunteers and patients with cystic fibrosis. The drug remains in clinical development and is not approved.
IONIS-ENaCRx is being developed as a treatment for cystic fibrosis, a respiratory condition. Its clinical testing has involved both healthy volunteers and patients with cystic fibrosis. Because the program is at the Phase 1 stage, IONIS-ENaCRx is investigational and has not been established as a marketed therapy for cystic fibrosis.
Ionis Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker IONS, is the developer of IONIS-ENaCRx. The company is responsible for the clinical program evaluating the drug in cystic fibrosis and in healthy volunteers.
IONIS-ENaCRx is in Phase 1 development. Its single recorded trial, NCT03647228, was a Phase 1/2a study, and that trial is now completed. The drug is investigational and has not been approved for any indication.
IONIS-ENaCRx has been evaluated in one clinical trial, NCT03647228, titled a Phase 1/2a study of single and multiple doses in healthy volunteers and patients with cystic fibrosis. That trial is completed, enrolled 98 participants, was randomized, double blind, and placebo controlled, and ran in Germany and the United Kingdom.
IONIS-ENaCRx is not approved. It is an investigational drug that has completed a Phase 1/2a clinical trial in healthy volunteers and patients with cystic fibrosis. Further clinical development would be required before any regulatory approval could be considered.