SMA Research Platform

Evidence graph for Spinal Muscular Atrophy

Biology-first target discovery
Christian Fischer / Bryzant Labs
14,766Targets
453Trials
84Drugs
7Datasets
6,987Sources
64,683Claims
72,052Evidence
29,649Hypotheses
DRUGdiscontinuedsmall molecule

olesoxime

Mechanism

Mitochondrial function, cholesterol binding. Positive in SMND7 mice (2014). Both survival + motor function improved.. DISCONTINUED 2018 (Roche) — Phase 3 OLEOS failed futility analysis; development halted.

Related claims (16)

TypePredicateConfSource
motor functionOlesoxime treatment was associated with changes from baseline in Motor Function Measure (MFM) Dimension 1 (D1) + Dimension 2 (D2) Score in patients with SMA (clinical trial result)65%NCT02628743_results
neuroprotectionNumerous preclinical studies have successfully proved olesoxime's neuroprotective properties in cell and animal models of spinal muscular atrophy.59%31283931
motor functionSecondary endpoints and sensitivity analyses suggest that olesoxime might maintain motor function in patients with type 2 or type 3 SMA over a period of 24 months (clinical trial result).59%28460889
drug efficacyNeuroprotective drugs as olesoxime or riluzole have shown positive effects in preclinical studies but no clear efficacy in clinical trials.53%28524214
safetyTreatment with Olesoxime was associated with adverse events and serious adverse events in patients with SMA (clinical trial result)53%NCT02628743_results
safetyOlesoxime seemed to be safe and generally well tolerated, with an adverse event profile similar to placebo (clinical trial result).53%28460889
drug efficacyPrevious treatment with olesoxime in the Phase 2 study was not protective of motor function in OLEOS (clinical trial result).53%33246887
drug efficacyOlesoxime's efficacy and safety were assessed in a Phase 2 study using data from 110 individuals with Type 2 or 3 SMA.53%32946459
drug efficacyOlesoxime has shown some improvement in treating SMA.53%39514016
motor functionOlesoxime treatment was associated with changes from baseline in MFM Total Score (D1+ D2 + Dimension 3 [D3]) Score in patients with SMA (clinical trial result)51%NCT02628743_results
drug efficacyOlesoxime at 10 mg/kg BID was evaluated for long-term safety, tolerability, and effectiveness in patients with Spinal Muscular Atrophy (clinical trial result)51%NCT02628743_results
drug efficacyVery low-certainty evidence suggested that olesoxime had little or no effect on motor function (clinical trial result)45%32006461
drug efficacyOlesoxime might help in the maintenance of motor function in patients with Types 2/3 SMA (clinical trial result).45%33246887
drug efficacyOlesoxime might provide meaningful clinical benefits for patients with SMA (clinical trial result).45%28460889
drug targetOlesoxime has emerged as a promising drug candidate for neurodegenerative diseases.45%31283931
drug efficacyOlesoxime had little or no effect on motor function in SMA types II or III in comparison to placebo (clinical trial result, very low-certainty evidence).42%PMID:32006461

Off-Target Findings (0)

No Boltz-2 / Chai-1 off-target panel claims recorded for this drug.

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