Olesoxime

Olesoxime is an experimental drug that was being developed by TRIGR Therapeutics as a potential treatment for spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease. Olesoxime is a neuroprotective compound designed to enhance motor neuron survival. It belongs to a class of cholesterol-like small molecules that target the mitochondrial permeability transition pore (mPTP), preventing its opening and subsequent neuronal cell death.

The drug's mechanism of action involves binding to the translocator protein (TSPO), which is located on the outer mitochondrial membrane. By interacting with TSPO and influencing the mPTP, olesoxime aims to preserve mitochondrial function and protect motor neurons from the damaging effects of cellular stress.

Clinical trials investigating olesoxime's efficacy in SMA, particularly in patients with type 2 and type 3 SMA, showed promising results in earlier phases. However, subsequent trials failed to meet their primary endpoints.

In ALS, while pre-clinical studies showed some neuroprotective effects, clinical trials did not demonstrate a statistically significant benefit for patients.

As of late 2023, development of olesoxime has been discontinued due to the lack of efficacy demonstrated in clinical trials. The drug is no longer being actively pursued as a treatment option for either SMA or ALS.