In May, we shared with you news of the difficult decision to pause enrollment for our study of LMI070 for the treatment of Type 1 Spinal Muscular Atrophy (SMA). This decision was made because 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.
We would like to bring you up to date on what has been happening since then.
In addition to pausing enrollment, we also lowered the dose of LMI070 for enrolled patients in an abundance of caution. About eight weeks after this reduction, we received confirmed reports of worsening of motor skills in some patients varying in degree of severity. We are extremely saddened to note that one patient died following an upper respiratory infection with contribution of increasing respiratory muscle weakness.
Following these reports, and with the approval of the Data Monitoring Committee (DMC), a team of independent experts who monitor the trial’s safety results, we have decided to offer patient’s families the option to return to the original dose. This has allowed parents and investigators to make the best decision for each individual patient.
We remain committed to the safety and wellbeing of children in this trial. We hope to resume enrollment in the study when we have fully evaluated the new toxicology findings, the additional monitoring of patients, and the feedback from the regulatory authorities, investigators, and DMC members.
We will continue to provide updates as new information is available.
The Novartis Team