SMA Drug Pipeline
SMA Drug Pipeline
Page last updated: August 2023
Using this drug pipeline, we are tracking the progress of SMA treatments currently being tested in human clinical trials.
The diagram is split into:
- drugs that increase SMN protein levels (SMN-dependent)
- combined treatments that deliver SMN-dependent with SMN-independent drugs (those that do not affect SMN protein levels)
Download the diagram below to see how each drug is progressing through clinical trials and follow links to further information about each drug on our website. You can also find listings for any related global trial on the ClinicalTrials.gov website, if applicable. Each listing includes details such as eligibility criteria, where in the world the trial is taking place, and whether the trial is currently recruiting.
The diagram will be regularly updated to map the development and progress of each drug programme.
Last updated: 4th January 2023
CK-2127107 / reldesemtiv (Cytokinetics)
CK-2127107 / reldesemtiv is a drug designed to increase the force generated by skeletal muscle and delay the onset and extent of muscle fatigue. It is being trialled for Amyotrophic lateral sclerosis (ALS) also known as Motor neurone disease (MND) but not now for SMA.
Amifampridine (Catalyst Pharmaceuticals)
Catalyst, a biopharmaceutical company focused on developing innovative therapies for people with rare debilitating neuromuscular and neurological diseases, was evaluating amifampridine phosphate, a neuronal potassium channel blocker, for the treatment of SMA.
LMI070 / Branaplam (Novartis)
Similar to RG7916, LMI070 was a small molecule drug that increases the amount of SMN protein made by the SMN2 gene. Development of Branaplam was discontinued in mid 2021.
Olesoxime (Trophos / Roche)
Olesoxime was a neuro-protective drug that supports the nervous system under stressful conditions. Development of Olesoxime was discontinued in mid 2018.