Clinical Research Alert: Phase 3 Study of Salanersen in Older Teens and Adults with SMA

Researchers at Biogen are conducting the phase 3 SOLAR study to learn more about spinal muscular atrophy (SMA) and to evaluate salanersen, an investigational therapy being studied for the treatment of SMA in individuals 15-60 years old. Salanersen is investigational and has not been approved by regulatory authorities.

About the SOLAR study

The SOLAR study is evaluating the efficacy and safety of salanersen in individuals 15-60 years old with SMA.

In most people living with SMA, the SMN1 gene is either missing or changed, leading to a lower amount of overall SMN protein in the body. Without enough of this protein, motor neurons and muscles lose their ability to work properly. A similar gene called SMN2 can help replace some of the lost SMN protein in the body. Salanersen is designed to help the SMN2 gene to make more SMN protein.

The SOLAR study is open-label, meaning that all participants will receive salanersen. Salanersen will be administered through an intrathecal injection, which means that the drug will be given through a needle into the fluid around the spinal cord and brain. Salanersen will be given once every year for a total of five doses throughout the study.

The study includes two groups of participants: a group who has not previously taken any treatment for SMA and a group who has been taking risdiplam. Participation will last a little over five years and includes a screening period of up to 28 days. Participants will complete a total of 17 clinic visits and 9 phone calls over the course of the study. Study visits are more frequent earlier in the study and become less frequent over time.

The effects of salanersen will be evaluated using a number of assessments including but not limited to:

• Physical and neurological examinations
• Blood and urine sample collections
• Vital sign measurements
• Electrocardiograms (ECGs)
• Assessments of strength, function, and movement
• Assessment of nerve and muscle health
• Questionnaires about how the participants are feeling

Study criteria

Individuals may be eligible for the SOLAR study if they meet the following key criteria:

• Be 15 to 60 years of age, inclusive
• Have a confirmed diagnosis of SMA
• Be able to sit without using support for at least 10 seconds
• EITHER have not received any prior medication specifically designed to treat the underlying cause of SMA OR be currently receiving risdiplam, willing to stop taking it during the study, and not have been treated with another medication specifically designed to treat the underlying cause of SMA

Additional eligibility criteria also apply and will be reviewed by the study team. Please visit this link for the full list of inclusion and exclusion criteria.

Interested in participating?

Travel support and reimbursement will be available for eligible participants and their families.

Individuals interested in learning more about the SOLAR study should visit the sponsor’s trial website.

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