Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
CNMAu8 · 4 trials · 2 indications
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 type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.
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. The rate of mortality was estimated from a Bayesian shared-parametric model that assumed exponentially distributed survival times.
Safety endpoints include incidence of treatment-emergent AEs, drug-related AEs, deaths, SAEs, and AEs leading to discontinuation from the study. Changes from baseline (Week 36 of the placebo controlled phase) in clinical laboratory results, physical examination findings, vital signs, ECGs, and C-SSRS will be summarized descriptively by group and timepoint.
Mean change in the average difference between active treatment and placebo from Baseline through End of Study for the MUNIXscore(4), which is the sum of the respective MUNIX values for the Abductor Digiti Minimi (ADM), Abductor Pollicis Brevis (APB), Biceps Brachii (BB), and Tibialis Anterior (TA).
Mean change in BC-LCLA from baseline to end of study across all eyes as measured by 2.5% low contrast Sloan Letter Chart.
Safety endpoint include incidence of treatment-emergent AEs.
The Motor Unit Number Index (MUNIX) is a quantitative neurophysiological measure that provides an index of the number of lower motor neurons supplying a muscle. It reflects the loss of motor neurons in patients with ALS. Mean change in the average difference between active treatment and placebo from Baseline through Week 36 for the MUNIXscore(4), which is the sum of the respective MUNIX values for the Abductor Digiti Minimi (ADM), Abductor Pollicis Brevis (APB), Biceps Brachii (BB), and Tibialis Anterior (TA). The average baseline summed values will be indexed to 100%. Changes will be calculated as the percent change from the Baseline index of 100%.
| Arm | Type | Description |
|---|---|---|
| CNM-Au8 | EXPERIMENTAL | Drug: CNM-Au8 Administration: Oral Dosage: 30 mg or 60 mg daily |
| Matching Placebo | PLACEBO_COMPARATOR | Administration: Oral Dosage: 2 bottles daily |
| Open label extension | EXPERIMENTAL | This is an unblinded open-label extension, all participants will be on active drug. |
| Active treatment with 30 mg of CNM-Au8 | EXPERIMENTAL | Highly pure elemental Au nanocrystals are suspended in deionized water buffered with 0.546 mg/mL (6.5 mM) sodium bicarbonate (NaHCO3) concentrated up to 0.5 mg/mL (500 ppm) Au. |
| Placebo | PLACEBO_COMPARATOR | The matched placebo used in this study consisted of water, sodium bicarbonate, and food coloring to match volume and color of the experimental treatment |
| 30 mg CNM-Au8 | EXPERIMENTAL | 30mg suspension of clean-surfaced, faceted, gold nanocrystals in 60ml of sodium bicarbonate buffered water |
| Name | Type | Description |
|---|---|---|
| CNM-Au8 | DRUG | Drug: CNM-Au8 Administration: Oral Dosage: 30 mg or 60 mg daily |
| Matching Placebo | DRUG | Drug: Matching Placebo Administration: Oral Dosage: 2 bottles daily |
| CNMAu8 | DRUG | CNM-Au8 is an aqueous suspension of clean surfaced faceted nanocrystals consisting of gold atoms self-organized into crystals of various geometrical shapes (hexagonal bi-pyramid, pentagonal bi-pyramid, tetrahedron, decahedron, planar spheroids). Those choosing to participate in the OLE period will orally receive 30 mg of CNM-Au8, once daily. |
| Placebo | DRUG | Placebo was liquid with identical color and taste |
Inclusion Criteria: * No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683). Exclusion Criteria: * The following exclusion criterion is in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683). 1. Histo...
CNM-Au8 is an investigational small molecule developed by Clene Inc. (ticker CLNN) for neurological diseases. It is being studied in amyotrophic lateral sclerosis (ALS) and relapsing multiple sclerosis. It has received an FDA accelerated approval designation and is in the NDA/BLA stage of development.
CNM-Au8 is being developed for amyotrophic lateral sclerosis, a progressive neurological disease. It has been studied in ALS patients across multiple Phase 2 trials, including the HEALEY ALS Platform Trial and an open-label extension study. It is investigational and has not been approved for ALS.
CNM-Au8 is developed by Clene Inc., which trades under the ticker CLNN. The company is advancing the small molecule for neurological indications including amyotrophic lateral sclerosis and relapsing multiple sclerosis, and has received an FDA accelerated approval designation for the asset.
CNM-Au8 is in the NDA/BLA stage, meaning Clene Inc. has advanced it toward a regulatory submission. It has completed Phase 2 trials in ALS and relapsing multiple sclerosis. It is investigational and has not been approved by the FDA.
CNM-Au8 has been studied in several Phase 2 trials. These include NCT04414345, the HEALEY ALS Platform Trial Regimen C; NCT04098406 in ALS; NCT04626921 in relapsing multiple sclerosis; and NCT05299658, an open-label extension in early symptomatic ALS. Total enrollment across these studies is 246 participants.