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Letter to the SMA Community on the Study of LMI070 for the Treatment of Type 1 SMA

Wednesday May 25th, 2016 – Letter from Novartis:

We have made the difficult decision to pause enrollment for our study of LMI070 for the treatment of Type 1 Spinal Muscular Atrophy (SMA). Animal safety studies were taking place in parallel with this trial, which is a common practice when studying potentially life threatening diseases to help bring medicines to patients faster. Results from an animal study, using daily dosing for a year compared to weekly dosing in the human study, showed unexpected injuries to the peripheral nerves and spinal cord, testes, and blood vessels in the kidney.

Patient safety is paramount to us and after a review of these results and in consultation with our Data Monitoring Committee, a team of independent experts who monitor the trial’s safety results, we decided to continue the trial but pause additional enrollment while we do further analysis.

All patients currently enrolled in the trial are being closely monitored and the teams at the clinical trial sites are working with their families to explain the findings, answer their questions, and help them decide whether they would like to continue. We are also working closely with our teams and health authorities to determine appropriate next steps.