Sanofi’s investigational BTK inhibitor, tolebrutinib, has been shown to have a significant effect on the key central nervous system immune mediators that may drive multiple sclerosis (MS) disease progression, according to new data reported by the company.
MS is a disabling, unpredictable neurological disease in which the immune system attacks the protective myelin sheath that covers the nerves and disrupts communication between the brain and the rest of the body.
It is estimated that there are over 130,000 people living with the condition in the UK alone, with around 7,000 people newly diagnosed each year.
There is no cure for MS, with treatments focusing on helping speed the recovery from attacks, modify the course of the disease and manage symptoms.
Tim Turner, global project head for tolebrutinib at Sanofi, said: “Delivering therapies across the tightly controlled blood-brain barrier to reach disease targets thought to be responsible for driving smouldering neuroinflammation within the central nervous system has been a tremendous hurdle in advancing innovation in MS.”
The new data being presented at this year’s Americas Committee for Treatment and Research in Multiple Sclerosis Forum supports the potential for tolebrutinib to target inflammation both in the periphery and directly in the central nervous system.
The study, which saw the company collaborate with the National Institute of Neurological Disorders and Stroke, involved researchers measuring proteomic changes in the cerebrospinal fluid of MS patients treated with tolebrutinib, compared to those receiving either no treatment or the B-cell depleting therapy ocrelizumab.
Additional presentations from Sanofi include an evaluation of the central nervous system penetrability of tolebrutinib, as measured by drug concentration in the cerebrospinal fluid of healthy volunteers, as well as the phase 2 MS trial design for SAR443820, Sanofi’s investigational oral RIPK1 inhibitor.
The positive results come just six months after the US Food and Drug Administration placed a partial clinical hold on tolebrutinib phase 3 trials in MS and myasthenia gravis after cases of drug-induced liver injury were observed.
Sanofi said that all instances were seen in patients with predisposing conditions and revised the protocol for the studies to exclude those vulnerable to this sort of reaction, allowing for recruitment to resume.
The company has since discontinued its phase 3 URSA trial of tolebrutinib in patients with moderate-to-severe myasthenia gravis, as announced in its fourth-quarter results update earlier this year, citing the ‘emerging competitive treatment landscape’ for the disease.
Πηγή: pmlive.com
