Tolperisone is a medicine widely prescribed outside of the U.S. for acute muscle spasms and spasticity. In the U.S., tolperisone is being investigated as a novel, non-opioid treatment for acute and painful muscle spasms without drowsiness or impact on cognitive function. Tolperisone is predicted to have analgesic activity in addition to muscle relaxation because of its centrally-acting dual voltage gated calcium and sodium channel blocker mechanism of action.
Neurana completed a Phase 1 driving simulation study which demonstrated that tolperisone was similar to placebo in driving performance. In comparison, cyclobenzaprine, a commonly used muscle relaxant, significantly impacted driving performance. Most individuals performed similar to those with a blood alcohol concentration above 0.05%, which is considered above the legal limit in most states. In addition, most patients on cyclobenzaprine were unaware of their impairment.
Neurana completed a positive Phase 2 dose-ranging clinical study of tolperisone, the STAR Study, in September 2019. This double-blind, randomized, placebo-controlled, parallel-group clinical trial evaluated the efficacy and safety of tolperisone administered three times a day in patients experiencing acute back pain and/or stiffness due to acute and painful muscle spasms. The primary endpoint measurement was subject-rated pain due to acute back spasm using a Numerical Rating Scale from no pain to worst possible pain. The STAR Study enrolled 415 patients ages 18 to 65 years across 38 sites in the United States. Topline data demonstrated the overall efficacy of tolperisone, with the 600 mg per day dose being highly statistically significant (p=0.004). Tolperisone was generally well tolerated, with most adverse events mild to moderate and no serious adverse events reported. Headache was the only adverse event reported in more than 5% of patients. The rates of drowsiness were similar among the treatment groups compared with placebo. In addition, a secondary endpoint demonstrated no change in sleepiness with tolperisone compared to placebo.
Composition of matter and process patents have been issued by the US Patent and Trademark Office (USPTO) providing Orange Book listable patent protection through 2032.