Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
RCT2100 · 1 trial · 1 indication
Safety and tolerability as assessed by number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Safety and tolerability of multiple-ascending doses of inhaled RCT2100 administered to participants with CF
To assess the safety and tolerability of RCT2100 co-administered with ivacaftor in participants with CF.
| Arm | Type | Description |
|---|---|---|
| RCT2100 (Part 1) | EXPERIMENTAL | RCT2100 single dose |
| Placebo (Part 1) | PLACEBO_COMPARATOR | Placebo single dose |
| RCT2100 (Part 2) 4 week | EXPERIMENTAL | RCT2100 multiple dose |
| RCT2100 (Part 2) 12 week | EXPERIMENTAL | RCT2100 multiple dose |
| Experimental: RCT2100 (Part 3) 6 week | EXPERIMENTAL | RCT2100 multiple dose |
| Name | Type | Description |
|---|---|---|
| RCT2100 | DRUG | RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer |
| Placebo | OTHER | Placebo of similar volumes to experimental dose strengths administered via oral inhalation using nebulizer |
| Ivacaftor | DRUG | ivacaftor administered orally for 6 weeks |
Part 1 Major Inclusion Criteria: * Healthy, adult, male or female, 18-55 years of age, inclusive, at screening. * Body weight greater than or equal to 50 kg and body mass index (BMI) between 16-32 kg/m2, inclusive * The participant has a forced expiratory volume in one second (FEV1) of at least 80%...
| 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 |
RCT2100 is an investigational small molecule being developed for cystic fibrosis. It is currently in Phase 2 clinical development. The drug is being studied in a randomized, double-blind, placebo-controlled trial that includes healthy participants and participants with cystic fibrosis.
RCT2100 is being developed by cbdMD, Inc., a company traded on the NYSE American under the ticker symbol YCBD. The company is conducting a Phase 2 clinical trial of RCT2100 for cystic fibrosis.
RCT2100 is in Phase 2 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The ongoing Phase 2 trial is active but not recruiting participants, with a planned enrollment of 128.
RCT2100 is being evaluated in a Phase 2 study with the identifier NCT06237335. This randomized, double-blind, placebo-controlled trial is assessing the safety and tolerability of RCT2100 in healthy participants and in participants with cystic fibrosis. The study is being conducted in the United States, France, Netherlands, New Zealand, and the United Kingdom.
RCT2100 is a small molecule that targets the cystic fibrosis transmembrane conductance regulator (CFTR) mRNA. It is designed to address the underlying cause of cystic fibrosis by modulating CFTR function. The drug is being studied in a biomarker-selected patient population.