Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
MRT5005 · 1 trial · 1 indication
An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that did not necessarily had a causal relationship with the study treatment. A serious adverse event (SAE) was any untoward medical occurrence (whether considered to be related to study treatment or not) that at any dose: resulted in death; or was life-threatening; or required inpatient hospitalization or prolongation of existing hospitalization; or resulted in persistent or significant disability/incapacity; or was a congenital abnormality/birth defect; or was an important medical event. The TEAEs were defined as events that were newly reported or reported to worsen in severity after the start of study treatment up to 48 weeks (end of follow-up period) after the last dose of study treatment administration.
| Arm | Type | Description |
|---|---|---|
| Part A - SAD Group 1: MRT5005 8 mg | EXPERIMENTAL | Participants received single dose of MRT5005 8 milligrams (mg) by nebulization on Day 1. |
| Part A - SAD Group 2: MRT5005 16 mg | EXPERIMENTAL | Participants received single dose of MRT5005 16 mg by nebulization on Day 1. |
| Part A - SAD Group 3: MRT5005 20 mg | EXPERIMENTAL | Participants received single dose of MRT5005 20 mg by nebulization on Day 1. |
| Part A - SAD Group 4: MRT5005 24 mg | EXPERIMENTAL | Participants received single dose of MRT5005 24 mg by nebulization on Day 1. |
| Part A - SAD Groups: Pooled Placebo | PLACEBO_COMPARATOR | Participants received single dose of placebo (normal saline) by nebulization on Day 1. Data were analyzed as a pooled population for all participants who received placebo in Part A SAD groups and reported in this arm. |
| Part B - MAD Group 1: MRT5005 8 mg | EXPERIMENTAL | Participants received MRT5005 8 mg once weekly by nebulization for 5 weeks (i.e., on Day 1, Day 8, Day 15, Day 22 and Day 29). |
| Part B - MAD Group 2: MRT5005 12 mg | EXPERIMENTAL | Participants received MRT5005 12 mg once weekly by nebulization for 5 weeks (i.e., on Day 1, Day 8, Day 15, Day 22 and Day 29). |
| Part B - MAD Group 3: MRT5005 16 mg | EXPERIMENTAL | Participants received MRT5005 16 mg once weekly by nebulization for 5 weeks (i.e., on Day 1, Day 8, Day 15, Day 22 and Day 29). |
| Part B - MAD Group 4: MRT5005 20 mg | EXPERIMENTAL | Participants received MRT5005 20 mg once weekly by nebulization for 5 weeks (i.e., on Day 1, Day 8, Day 15, Day 22 and Day 29). |
| Part B - MAD Groups: Pooled Placebo | PLACEBO_COMPARATOR | Participants received placebo (normal saline) once weekly by nebulization for 5 weeks (i.e., on Day 1, Day 8, Day 15, Day 22 and Day 29). Data were analyzed as a pooled population for all participants who received placebo in Part B MAD groups and reported in this arm. |
| Part D: MRT5005 4 mg | EXPERIMENTAL | Participants received MRT5005 4 mg once daily by nebulization from Day 1 to Day 5. |
| Part D: Placebo | PLACEBO_COMPARATOR | Participants received placebo (normal saline) once daily by nebulization from Day 1 to Day 5. |
| Name | Type | Description |
|---|---|---|
| MRT5005 | DRUG | Nebulization of MRT5005 |
| Normal saline | DRUG | Normal Saline for Inhalation |
Inclusion Criteria: * Confirmed diagnosis of CF as defined by both of the following: * Two CF disease-causing cystic fibrosis transmembrane conductance regulator (CFTR) mutations in Class I or II (genotype confirmed at the screening visit). * Chronic sinopulmonary disease and/or gastrointestin...
| 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 |
MRT5005 is an investigational small molecule being developed for the treatment of cystic fibrosis. It is administered by nebulization and is currently in clinical development for adults with this condition.
The specific molecular target of MRT5005 has not been disclosed. It is a small molecule modality being studied for its potential to treat cystic fibrosis, a genetic respiratory condition.
MRT5005 is being developed by Sanofi, a global biopharmaceutical company listed on the stock exchange under the ticker SNY.
MRT5005 is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. A Phase 1 trial has been completed to evaluate its safety and tolerability.
MRT5005 has been studied in one completed Phase 1 clinical trial, identified as NCT03375047. This trial evaluated the safety and tolerability of MRT5005 administered by nebulization in 42 adults with cystic fibrosis in the United States.
No alternative names for MRT5005 have been reported. It is identified solely by its developmental code name MRT5005 in clinical trial records.