Update on Novartis drug LMI070

18 August 2016

The pharmaceutical company Novartis have recently provided an update on their SMN2 splice-modifying drug LMI070.
LMI070 is being tested in a Phase II clinical trial conducted with SMA patients aged 6 months or less. However, in May 2016, safety concerns with the treatment caused Novartis to pause enrolment in the trial. Animals treated with LMI070 every day for a year (as a safety precaution) showed unexpected complications with their blood vessels, kidneys, spinal cord and peripheral nerves, such as the motor neurons (click here for more information).
In addition to pausing enrolment, as an added precaution Novartis have lowered the dose of LMI070 for patients already enrolled in the study.
Perhaps as a consequence of this, about eight weeks after the dose reduction, worsening of motor function of varying severity was reported in some of the enrolled patients.
With the approval of a team of independent experts, the Data Monitoring Committee (DMC), patients and their families are now being offered the option of returning to the original higher dose of LMI070.
Novartis are hopeful that enrolment will resume once additional toxicology experiments are performed and patients have been monitored for an extended period of time.
Further Information
LMI070 clinical trial page