Drugs Currently Being Tested in Clinical Trials
Drugs Currently Being Tested in Clinical Trials
Your clinical team will know of any UK clinical trials you or your child might qualify for. Please be aware, however recruitment is often global with are a very limited number of places.
Some of the drugs currently in clinical trials are being tested as ‘combination therapies’. The currently available SMA treatments (Nusinersen, Zolgensma and Risdiplam) aim to increase the amount of survival motor neuron (SMN) in the body. They are referred to as ‘SMN-targeted therapies‘.
Combining an SMN-targeted therapy with a second approach that targets muscles may result in a complementary or added benefit. The idea is that this combined approach treat both the underlying cause of SMA and the symptoms of the condition.
The ASCEND trial will evaluate whether treatment with a higher dose of nusinersen has the potential to improve clinical outcomes and address unmet medical needs in people with later-onset SMA who were previously treated with risdiplam. Up to 135 later-onset, non-ambulatory people with SMA aged 5-39 years will be enrolled. All participants must have been treated with the maximum dose of 5 mg of risdiplam before joining the study and be willing and able to change their treatment regimen to a higher dose of nusinersen.
Read more about trials of nusinersen and any updates, including information about access in the UK in our Nusinersen section >
BIIB115 is similar to Spinraza/nusinersen in that it is an "antisense oligonucleotide" (ASO), which is a small snippet of synthetic genetic material. Like Spinraza/nusinersen, BIIB115 is designed to target the SMN2 gene to increase the amount of SMN protein that it produces. A Phase 1 clinical trial of BIIB115 in healthy volunteers was initiated in late 2022, and is expected to be completed in mid 2027.
This is a randomised, double-blind, sham-controlled Phase 3 study with a primary objective to evaluate the clinical efficacy, safety, and tolerability of a one-time intrathecal (IT) dose of Zolgensma in treatment naïve children and young people with Type 2 SMA who are between 2 and 18 years of age, able to sit, but have never walked.
Read more about trials of zolgensma and any updates, including information about access in the UK in our Zolgensma section >
To aid delivery, a tablet form of Risdiplam has been created. In late 2023, a Phase 1 study was conducted in healthy volunteers to compare two different formulations of 5 mg risdiplam (dispersible tablets) versus the current risdiplam oral solution formulation. In results presented at the 2024 SMA Europe congress, it was shown that the tablet resulted in the same concentration of risdiplam in the body as the currently available oral solution, and no new safety signals were identified. In the first half of 2024 Roche plans to apply to the European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA) for the tablet formulation to be considered for licencing.
Read more about trials of risdiplam and any updates, including information about access in the UK in our Risdiplam section >
SRK-015 / Apitegromab is a muscle-directed therapy that aims to reverse or restrict the muscle atrophy and weakness experienced by SMA patients. The drug is an antibody that very specifically targets an important protein called myostatin. Myostatin is naturally present in our muscles, where it plays an important role in limiting muscle growth. By inhibiting myostatin function, Apitegromab can potentially release the breaks on muscle cell growth leading to an increase in muscle size and function. If this is successful, this could benefit people who have SMA.
Read more about trials and any updates, including information about access in the UK >
GYM329 is an investigational anti-myostatin antibody that is designed to target skeletal muscles (i.e., those used for voluntary movement), potentially increasing their size and growth. Myostatin plays an important role in the regulation of skeletal muscle size by controlling growth. Inhibiting myostatin may help muscles grow in size and strength. GYM329 in combination with risdiplam, which is designed to increase the amount of SMN protein throughout the body, has the potential to further improve motor function and outcomes for people living with SMA Type 2.
Read more, including information about proposed access in the UK >
Taldefgrobep alfa is a protein designed to bind to and inhibit the function of myostatin. Myostatin is naturally present in our muscles, where it plays an important role in limiting muscle growth. By inhibiting myostatin function, taldefgrobep alfa can potentially release the breaks on muscle cell growth leading to an increase in muscle size and function. If this is successful, this could benefit people who have SMA.
Read more, including information about proposed access in the UK >
NMD670 is an orally-bioavailable, small molecule that specifically targets a protein called ClC-1 that is found almost exclusively on the surface of muscles cells. The function of ClC-1 is to dampen the excitability of muscles and prevent them from becoming overstimulated. By inhibiting ClC-1 function, NMD670 can increase and improve the passage of signals from motor neurons to muscles, which can increase how easily muscles are able to contract.
NMD670 is being tested in a phase 2, randomised, double-blind, placebo-controlled study in 50 ambulatory participants with SMA Type 3 that is estimated to be completed in late 2024.