Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
DNL343 · 1 trial · 1 indication
Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score using a Bayesian repeated measures model that accounts for loss to follow-up due to mortality. Each of 12 questions assessing distinct functional ability is scored from 4(normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Participants with higher scores have more physical function. Note that only participants who survived to their Week 24 visit contribute to the estimate.
The Mortality Rate, presented as mean deaths per month, was estimated from a Bayesian shared-parametric model that assumed exponentially distributed survival times. Mortality is defined as death or death equivalent. A participant is determined to meet the criteria of death equivalent if permanent assisted ventilation (PAV) is used for more than 22 hours per day for more than seven days in a row.
| Arm | Type | Description |
|---|---|---|
| DNL343 | EXPERIMENTAL | - |
| Matching Placebo | PLACEBO_COMPARATOR | - |
| Name | Type | Description |
|---|---|---|
| DNL343 | DRUG | DNL343 is administered orally once daily per day for 24 weeks. |
| Matching Placebo | DRUG | Matching placebo is administered orally once daily per day for 24 weeks. |
Inclusion Criteria: * No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683). Exclusion Criteria: * The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683). 1. Diagn...
DNL343 is an investigational small molecule being studied for the treatment of Amyotrophic Lateral Sclerosis (ALS). It is being evaluated in a Phase 2 clinical trial as part of the HEALEY ALS Platform Trial, Regimen G. The drug is intended to address the underlying disease process in ALS, though its specific mechanism has not been disclosed.
DNL343 is being developed by Merit Medical Systems, Inc., a company traded on the NASDAQ under the ticker symbol MMSI. The company is conducting clinical research on this investigational therapy for Amyotrophic Lateral Sclerosis.
DNL343 is in Phase 2 clinical development. It is an investigational drug, not yet approved by regulatory authorities. The Phase 2 trial, NCT05842941, has been completed, and the drug remains under investigation for the treatment of Amyotrophic Lateral Sclerosis.
DNL343 is being studied in the HEALEY ALS Platform Trial, Regimen G, under the clinical trial identifier NCT05842941. This Phase 2, randomized, double-blind, placebo-controlled trial enrolled 249 participants with Amyotrophic Lateral Sclerosis in the United States. The trial has been completed.
DNL343 is not known to be the same as any other drug. It is a distinct investigational small molecule being developed by Merit Medical Systems, Inc. for Amyotrophic Lateral Sclerosis. No alternative names for this drug have been reported.