New long-term data reinforcing promising safety and efficacy profile of brain-penetrant tolebrutinib presented at ECTRIMS 2021
- One-year results from Phase 2b extension study of brain-penetrant tolebrutinib showed 98 percent of patients remained on treatment
- After 48 weeks, mean MRI lesion activity remained low in patients who started on or switched to tolebrutinib 60mg
- Data from in vitro studies in human microglia extended previous observations that BTK-dependent inflammatory signalling can be modulated by tolebrutinib
“Understanding the ability of a brain-penetrant therapy to slow disability accumulation has the potential to bring new hope to people suffering from difficult-to-treat MS. For nearly two decades,
Ninety-eight percent (122/125) of LTS-treated patients remained in the Phase 2b extension study through Week 48. The extension study was designed to evaluate the safety of tolebrutinib and provided the opportunity to evaluate efficacy parameters and report MRI outcomes. The LTS consisted of Part A, a double-blind treatment period where patients continued the same tolebrutinib dose as administered in the dose-finding study (5, 15, 30 or 60mg/day) and Part B, where all participants switched to the 60mg tablet (5/60mg, 15/60mg, 30/60mg, 60/60mg), which is the dose being tested in the Phase 3 trials.
“Results showed favorable safety and efficacy for tolebrutinib, and nearly all patients remained enrolled at the one-year mark of the long-term extension study,” says
Safety and Efficacy Outcomes:
- Safety data showed continued favorable tolerability of tolebrutinib and no new safety signals. The most frequent AEs were headache (10%), COVID-19 (9%), upper respiratory tract infection (8%) and nasopharyngitis (7%).
- At baseline, mean Expanded Disability Status Scale (EDSS) scores across treatment groups ranged from 2.18 to 2.65. Over 48 weeks of treatment, mean EDSS scores remained relatively stable in all treatment groups. For the 60/60mg treatment group, mean (SD) score was 2.65 (1.22) at baseline and 2.45 (1.31) at Week 48.
- Patients treated with tolebrutinib 60mg experienced low annualized relapse rate (ARR) of 0.17 (95% CI: 0.10, 0.29) over the 48-week treatment period. The majority of patients (89.5%) were free of relapses during this period. The relapse rate for these patients was 1.23 in the year prior to the Phase 2b study.
MRI Outcomes:
- At Week 48 of the extension study, the mean number of new Gd-enhancing lesions/scan remained low (<0.4) in the 60/60mg arm. Patients who switched to 60mg in Part B (Weeks 15-47) of the LTS experienced a reduction in Gd-enhancing lesions, approaching values observed in the 60/60mg treatment arm.
The company also presented data on the effect of tolebrutinib on human microglia that support its capacity to modulate neuroinflammatory processes directly within the central nervous system (CNS). Results from this study extended upon previous findings in mouse microglial cells to show that BTK-dependent inflammatory signalling in human microglia and tri-cultures can be modulated using tolebrutinib in vitro. This research contributes to an improved understanding of BTK signalling in neuroinflammation and how BTK inhibitors target the neuroinflammation believed to contribute to disability progression in people with MS. Tolebrutinib is the only BTK inhibitor in development for MS which has been shown to directly modulate microglia, based on publicly available information.
About tolebrutinib:
Tolebrutinib is an investigational brain-penetrant Bruton’s tyrosine kinase inhibitor that achieves CSF concentrations needed for targeting B lymphocytes and microglial cells, modulating neuroinflammation. Tolebrutinib is being evaluated in Phase 3 clinical trials for the treatment of relapsing forms of MS (RMS), non-relapsing secondary progressive MS (nrSPMS), and primary progressive MS (PPMS), and its safety and efficacy have not been confirmed by any regulatory authority worldwide. For more information on tolebrutinib clinical trials, please visit www.clinicaltrials.gov.
About
With more than 100,000 people in 100 countries,
Media Relations Contact
Tel.: +1 (781) 264-1091
Sally.Bain@sanofi.com
Investor Relations Contacts Paris
Investor Relations
Tel.: +33 (0)1 53 77 45 45
investor.relations@sanofi.com
https://www.sanofi.com/en/investors/contact
Sanofi Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words “expects”, “anticipates”, “believes”, “intends”, “estimates”, “plans” and similar expressions. Although Sanofi’s management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of
Attachment
Source:
2021 GlobeNewswire, Inc., source