AB Science, a French pharmaceutical company, announced on Monday that it has been granted a patent in Japan for the use of its lead compound, masitinib, in the treatment of amyotrophic lateral sclerosis (ALS). This patent strengthens AB Science’s intellectual property position in ALS, securing protection for masitinib until 2037, and follows similar patents granted in 11 other regions, including the US, Europe, Israel, and China.
ALS is a fatal motor neuron disorder that gradually progresses to the loss of both upper and lower motor neurons at the spinal or bulbar level. In ALS, motor neurons start to degenerate and eventually die, interrupting the transfer of messages to the muscles. The disorder belongs to a group of degenerative diseases that progress over time, making it vital that scientists and pharmaceutical companies continue to research and develop effective treatment options.
Masitinib, AB Science’s lead compound, has received orphan drug designation for ALS from the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA). This recognition provides market exclusivity for 10 and seven years in Europe and the US, respectively, following product approval.
In addition to the patent granted in Japan, AB Science has also secured patents in Europe, the US, Israel, China, South Korea, Australia, Eurasia, Mexico, Singapore, New Zealand, and South Africa. Most recently, AB Science received a Notice of Allowance (NOA) for the patent in Japan, demonstrating its commitment to develop innovative drugs that target diseases with high unmet medical needs.
The newly granted patent provides a broad range of protection for masitinib, and other compounds in its class, for the treatment of ALS. This protection is particularly significant, as it includes a patient population initially selected for treatment based on disease aggressiveness, measured by functional progression rate, which is consistent with the clinical development program of masitinib in ALS.
AB Science claims that this patient population was used in Study AB10015, a randomized, placebo-controlled phase 2/3 clinical trial that investigated the efficacy and safety of masitinib as an add-on therapy to riluzole over 48 weeks, compared with a placebo add-on therapy to riluzole in patients with ALS. The primary efficacy endpoint was the change in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) from baseline to week 48 between treatment groups in the “Normal Progressor” population.
The study found that patients who received masitinib at a dose of 4.5 mg/kg/day with a progression rate of less than 1.1 points/month – “Normal Progressors” – showed a significant benefit compared with those who received a placebo. A significant slowing in the rate of functional decline was observed, corresponding to a 27% reduction in the rate. Additionally, a sensitivity analysis in ALS patients early in the course of their disease found a significant reduction in the risk of death, with a long-term survival follow-up.
“Our lead compound masitinib has shown encouraging results in treating amyotrophic lateral sclerosis (ALS), a debilitating disease with no cure,” said Alain Moussy, CEO and co-founder of AB Science. “We continue to strengthen the intellectual property portfolio of masitinib and the company’s commitment to developing innovative drugs that target diseases with high unmet medical needs.”
In conclusion, the recent patent grant in Japan, together with the 11 others, further strengthens AB Science’s intellectual property position in the key regions where masitinib could be marketed. The company hopes to continue its work in developing innovative drugs that address unmet medical needs and provide effective treatment options in the fight against serious and fatal diseases.
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