FDA grants approval to MediciNova to begin MN-166 trial to treat ALS

The randomized, double-blind, placebo-controlled trial is divided into two treatment arms, MN-166 and matching placebo, and includes a six-month treatment period followed by a six-month open-label extension.

The randomization will occur in a 2:1 ratio (MN-166: placebo) and the trial will see enrollment of around 60 male and female patients aged between 18 and 80 years.

According to the company, 40 subjects will take part in the MN-166 group, while the remaining 20 in the placebo group.

Carolinas HealthCare System Neurosciences Institute in Charlotte director of Carolinas Neuromuscular/ALS-MDA Center Dr Benjamin Rix Brooks is the principal investigator of the trial, which is funded by both the parties.

The trial is designed to evaluate several efficacy endpoints including functional activity (ALSFRS-R), respiratory function, muscle strength, and non-invasive ventilation (NIV) utilisation of MN-166.

Additional evaluation in the trial will involve monitoring the safety and tolerability of MN-166 60mg/day versus placebo when administered in combination with riluzole in ALS patients.

MediciNova president and chief executive officer Yuichi Iwaki said the company is happy to have successfully completed the FDA review period and look forward to start patient enrollment this fall.

"The safety and efficacy data from this trial will be important to our overall development efforts and should be complementary to efforts underway for proof-of-concept trials of MN-166 in progressive MS and drug addiction in the US," Iwaki said.

"While MN-166 development is addressing unmet medical needs in all of its neurological indications, ALS may represent the largest unmet medical need."

Patients interested to participate in the trial must have a diagnosis of sporadic or familial ALS with onset of less than three years from first clinical weakness prior to screening