Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
PTI-428 · 2 trials · 1 indication
Safety and tolerability will be assessed by adverse events (AEs), safety labs, electrocardiograms (ECGs), physical examinations and vital signs.
| Arm | Type | Description |
|---|---|---|
| PTI-428 dose level 1 | ACTIVE_COMPARATOR | - |
| PTI-428 dose level 2 | ACTIVE_COMPARATOR | - |
| Placebo PTI-428 | PLACEBO_COMPARATOR | - |
| PTI-428 | ACTIVE_COMPARATOR | Subjects will receive once daily dosing of PTI-428 or placebo for 14 days. |
| Placebo | PLACEBO_COMPARATOR | Subjects will receive once daily dosing of PTI-428 or placebo for 14 days. |
| Name | Type | Description |
|---|---|---|
| PTI-428 | DRUG | Active |
| Placebo | DRUG | Placebo |
Inclusion Criteria: * Confirmed diagnosis of CF with the F508del/F508del genotype on record * On tezacaftor/ivacaftor dosing for both label indication and per label dosing for a minimum of 1 month on Day 1 * Forced expiratory volume in 1 second (FEV1) 40-90% predicted, inclusive * Clinically stable...
| 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 |
PTI-428 is an investigational small molecule being developed for the treatment of cystic fibrosis, a respiratory condition. It is currently in Phase 2 clinical development and has been studied in clinical trials enrolling adult patients with cystic fibrosis.
PTI-428 is being developed by cbdMD, Inc., a company traded on the stock exchange under the ticker symbol YCBD. The drug is currently in Phase 2 clinical development for the treatment of cystic fibrosis.
PTI-428 is currently in Phase 2 clinical development. It has completed two clinical trials, including a Phase 1 study and a Phase 2 study, both of which have been completed. The drug is investigational and has not been approved by regulatory authorities.
PTI-428 has been studied in two completed clinical trials. The first, NCT03258424, was a Phase 1 study assessing safety, tolerability, and pharmacokinetics in patients with cystic fibrosis on KALYDECO as background therapy. The second, NCT03591094, was a Phase 2 study assessing safety, tolerability, pharmacokinetics, and effect in patients with cystic fibrosis.
PTI-428 is not FDA approved. It is an investigational drug currently in Phase 2 clinical development for the treatment of cystic fibrosis. Both of its clinical trials have been completed, but the drug has not received regulatory approval.