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Tolebrutinib

Phase 3

Non-relapsing Secondary Progressive Multiple Sclerosis | Small molecule | Neurology |Sanofi|Last Updated: Aug 17, 2026

Target and mechanism

Molecular targetBTK
Target classInhibitor
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment1,131

FDA Designations

BREAKTHROUGH_THERAPYPRIORITY_REVIEW

Clinical trial landscape

Tolebrutinib · 10 trials · 8 indications

Phase 3 5Phase 2 1Phase 1 4
NCT06372145A Study to Investigate Long-term Safety and Tolerability of Tolebrutinib in Participants With Multiple Sclerosis.Relapsing Multiple Sclerosis
ACTIVE NOT_RECRUITING2,500 Analytics
NCT04411641Nonrelapsing Secondary Progressive Multiple Sclerosis (NRSPMS) Study of Bruton's Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168) (HERCULES)Non-relapsing Secondary Progressive Multiple Sclerosis
COMPLETED1,131 Analytics
NCT04458051Primary Progressive Multiple Sclerosis (PPMS) Study of Bruton's Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168) (PERSEUS)Primary Progressive Multiple Sclerosis
COMPLETED767 Analytics
NCT04410978Relapsing Forms of Multiple Sclerosis (RMS) Study of Bruton's Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168) (GEMINI 1)Relapsing Multiple Sclerosis
COMPLETED974 Analytics
NCT04410991Relapsing Forms of Multiple Sclerosis (RMS) Study of Bruton's Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168) (GEMINI 2)Relapsing Multiple Sclerosis
COMPLETED899 Analytics
PHASE3ACTIVE NOT_RECRUITING
A Study to Investigate Long-term Safety and Tolerability of Tolebrutinib in Participants With Multiple Sclerosis.
Relapsing Multiple SclerosisUnlock trial analytics
PHASE3COMPLETED
Nonrelapsing Secondary Progressive Multiple Sclerosis (NRSPMS) Study of Bruton's Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168) (HERCULES)
Non-relapsing Secondary Progressive Multiple SclerosisUnlock trial analytics
PHASE3COMPLETED
Primary Progressive Multiple Sclerosis (PPMS) Study of Bruton's Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168) (PERSEUS)
Primary Progressive Multiple SclerosisUnlock trial analytics
PHASE3COMPLETED
Relapsing Forms of Multiple Sclerosis (RMS) Study of Bruton's Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168) (GEMINI 1)
Relapsing Multiple SclerosisUnlock trial analytics
PHASE3COMPLETED
Relapsing Forms of Multiple Sclerosis (RMS) Study of Bruton's Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168) (GEMINI 2)
Relapsing Multiple SclerosisUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs), Adverse Events of Special Interest (AESIs) and AEs leading to permanent study intervention discontinuation
From baseline until the End of study approximately 4 years per participant
Number of Participants with Potentially clinically significant abnormalities (PCSAs)
From baseline until the End of study approximately 4 years per participant

Potentially clinically significant abnormalities (PCSAs) determined by laboratory tests, electrocardiogram (ECG), or vital signs and safety findings on MRI during the study period.

Time to Onset of 6-Month Confirmed Disability Progression (CDP) as Assessed by Expanded Disability Status Scale (EDSS)
Baseline (Day 1) up to approximately 47 months

The EDSS is a disability scale that assesses the following 7 functional domains: visual, brainstem, pyramidal \[motor\], cerebellar \[coordination\], sensory, cerebral, and bowel/bladder. The total EDSS ranges from 0 (normal) to 10 (death due to multiple sclerosis \[MS\]) (0.5 increments from 1-10; next increase after 0 is 1). Higher scores indicated increased disability. Time to onset of 6-month CDP was defined as the time from randomization to the onset of a sustained increase from baseline in EDSS score of \>=1.0 point from the baseline EDSS score when the baseline score was \<=5.0 or of \>=0.5 points when the baseline EDSS score was \>5.0 confirmed after a minimum 6-month interval.

6-month composite Confirmed Disability Progression (cCDP)
Up to approximately 60 months

Time to onset of 6-month cCDP defined as follows: Increase over at least 6 months of ≥1.0 point from the baseline expanded disability status scale (EDSS) score when the baseline score is ≤5.5, or ≥0.5 points when the baseline EDSS score is \>5.5, or ≥20% from the baseline T25-FW, or ≥20% from the baseline 9-HPT

Annualized Relapse Rate (ARR) as Assessed by Confirmed Protocol-defined Adjudicated Relapses
Baseline (Day 1) to approximately 48 months

Multiple sclerosis (MS) relapse was defined as a monophasic, acute or subacute onset of new neurological symptoms or worsening of previous neurological symptoms with an objective change on neurological examination. Symptoms were attributable to MS, lasted for \>=24 hours with or without recovery, present at normal body temperature, and preceded by \>=30 days of clinical stability.

Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
From first dose of study drug (Day 1) up to maximum exposure, 39 weeks in Part A and 222 weeks in Part B

An AE was any untoward medical occurrence in a participant or clinical study participant, temporally associated with the use of study drug, whether or not considered related to the study drug. An SAE was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent disability/incapacity, was a congenital anomaly/birth defect or was an important medical event. TEAEs were defined as AEs that developed, worsened or became serious during the respective on-treatment periods.

Assessment of PK parameters Tolebrutinib: AUC
From Day 1 to Day 4

Area under the plasma concentration (AUC) versus time curve extrapolated to infinity

Assessment of PK parameters M2: AUC
From Day 1 to Day 4
Part 1a and Part 2: Number of participants with Adverse Events (AEs) and treatment-emergent adverse events (TEAEs)
Part 1a: Day 1 to approximately Day 14 Part 2: Day 1 to approximately Day 21
Part 1b: Total (free and bound) SAR442168 concentrations in CSF
From Day 1 to Day 3
Part 1b: Total (free and bound) SAR442168 metabolite(s) concentrations in CSF
From Day 1 to Day 3
Part 1c and Part 1d: Maximum plasma concentration observed (Cmax) ratio fed/fast of SAR442168
From Day 1 to Day approximately 14
Part 1c and Part1d: Cmax ratio fed/fasted of SAR442168 metabolite(s)
From Day 1 to Day approximately 14
Part 1c and Part 1d: Area under the plasma concentration versus time curve (AUC) ratio fed/fast of SAR442168
From Day 1 to Day approximately 14
Part 1c and Part1d: AUC ratio fed/fast of SAR442168 metabolite(s)
From Day 1 to Day approximately 14
Pharmacokinetics: AUClast of SAR442168
Cohort 1: day1 period 1 to day 7 period 2; Cohort 2: day1 period 1 to day 9 period 2

AUC up to the last measurable concentration

Pharmacokinetics: AUC of SAR442168
Cohort 1: day1 period 1 to day 7 period 2; Cohort 2: day1 period 1 to day 9 period 2

Area under the curve, reflects the concentration of drug

Secondary Endpoints

Time to onset of 6-month confirmed disability worsening (CDW for RMS) or confirmed disability progression (CDP for PPMS and NRSPMS) for participants from pivotal studies
From baseline until the End of study approximately 4 years per participant
Annualized Relapse Rate (ARR) for RMS only
From baseline until the End of study approximately 4 years per participant
Number of new and/or enlarging T2-hyperintense lesions per year
From baseline until the End of study approximately 4 years per participant
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
TolebrutinibEXPERIMENTAL* Participants will receive OL tolebrunitib 60 mg once daily. * RMS participants who are not eligible for OL tolebrutinib per Health Authority and/or ethics committee decisions on the study conduct (ie, partial hold on initiation of tolebrutinib) will continue their parent study treatment assignment as per their randomization from the parent study.
TeriflunomideACTIVE_COMPARATOR* participants will receive teriflunomide 14 mg daily * RMS participants who are not eligible for OL tolebrutinib per Health Authority and/or ethics committee decisions on the study conduct (ie, partial hold on initiation of tolebrutinib) will continue their parent study treatment assignment (either tolebrutinib or teriflunomide) as per their randomization from the parent study. If unblinded to teriflunomide parent study treatment assignment, these RMS participants will continue teriflunomide in the LTS17043 study.
SAR442168EXPERIMENTAL60 mg of oral SAR442168 once daily
PlaceboPLACEBO_COMPARATORPlacebo tablet to match SAR442168 once daily
Mild hepatic impairment groupEXPERIMENTALSingle dose of tolebrutinib (SAR442168) will be administered on Day 1 under fed condition
Normal hepatic function groupEXPERIMENTALSingle dose of tolebrutinib (SAR442168) will be administered on Day 1 under fed condition
Severe Renal Impairment (RI) group (Part A only)EXPERIMENTALSingle dose of tolebrutinib (SAR442168) will be administered on Day 1 under fed condition
Normal Renal Function group (Part A and B)EXPERIMENTALSingle dose of tolebrutinib (SAR442168) will be administered on Day 1 under fed condition
Moderate RI group (Part B only conditional)EXPERIMENTALSingle dose of tolebrutinib (SAR442168) will be administered on Day 1 under fed condition
Part 1aEXPERIMENTAL3 single ascending doses of SAR442168 or placebo in fasted and fed (high-fat breakfast) conditions
Part 1bEXPERIMENTAL2 single doses of SAR442168 under fed conditions (moderate-fat breakfast).
Part 1cEXPERIMENTAL1 single dose of SAR442168 under fasting and fed conditions (high-fat breakfast).
Part 1dEXPERIMENTAL1 single dose of SAR442168 under fasting and fed conditions (Standardized high-fat breakfast).
Part 2EXPERIMENTAL3 ascending once-daily repeated single doses of SAR442168 or placebo for 14 days under fed conditions (moderate-fat breakfast)
Cohort 1EXPERIMENTALSAR442168 administered alone and together with gemfibrozil. 1 day washout for each administration of SAR442168.
Cohort 2EXPERIMENTALSAR442168 administered alone and together with rifampicin. 1 day washout for each administration of SAR442168.

Interventions

NameTypeDescription
TolebrutinibDRUGPharmaceutical form:Tablet-Route of administration:oral
PlaceboDRUGPharmaceutical form:Tablet-Route of administration:oral
TeriflunomideDRUGPharmaceutical form:Tablet-Route of administration:oral
Placebo to match TolebrutinibDRUGPharmaceutical form: Film-coated tablet Route of administration: Oral
Placebo to match TeriflunomideDRUGPharmaceutical form: Tablet Route of administration: Oral
Teriflunomide HMR1726DRUGPharmaceutical form: Tablet Route of administration: Oral
gemfibrozilDRUGTablet, taken orally
rifampicinDRUGTablet, taken orally
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites352

Inclusion Criteria: \- Participants with RMS, PPMS, or NRSPMS who completed the Phase 2b LTS (LTS16004) or 1 of the 4 Phase 3 pivotal tolebrutinib trials (EFC16033, EFC16034, EFC16645, EFC16035) on IMP. OR \- The Phase 2b LTS (LTS16004) or Phase 3 tolebrutinib pivotal trial participants who tempo...

Countries:United StatesArgentinaAustraliaAustriaBelgiumBrazilBulgariaCanadaChileChinaColombiaCroatiaCzechiaDenmarkEstoniaFinlandFranceGeorgiaGermanyGreeceHong KongHungaryIndiaIsraelItalyJapanLatviaLithuaniaMexicoNetherlandsNorwayPolandPortugalPuerto RicoRomaniaSerbiaSlovakiaSouth AfricaSouth KoreaSpainSwedenSwitzerlandTaiwanThailandTurkey (Türkiye)UkraineUnited KingdomBelarusRussiaPeru
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Recent Changes (Last 90 Days)

LOWAug 17, 2026NCT06372145lastUpdatePostDate: changed
LOWAug 17, 2026NCT06372145lastUpdatePostDate: changed

Frequently asked questions about Tolebrutinib

What is Tolebrutinib used for?

Tolebrutinib is an investigational small molecule being developed for multiple sclerosis (MS), including relapsing MS, primary progressive MS, and non-relapsing secondary progressive MS. It is also studied in renal impairment and hepatic function abnormal conditions. Tolebrutinib is not FDA approved and remains in clinical development.

What does Tolebrutinib target?

Tolebrutinib is a kinase inhibitor, belonging to the -tinib class of drugs. It targets Bruton's tyrosine kinase (BTK), a key enzyme in B-cell and microglia signaling pathways implicated in multiple sclerosis pathology. By inhibiting BTK, Tolebrutinib aims to modulate immune responses involved in MS.

Who makes Tolebrutinib?

Tolebrutinib is developed by Sanofi, a global biopharmaceutical company traded on the stock exchange under the ticker SNY. Sanofi is conducting clinical trials to evaluate the safety and efficacy of Tolebrutinib in multiple sclerosis and related conditions.

What phase is Tolebrutinib in?

Tolebrutinib is in Phase 3 clinical development for multiple sclerosis. It has received FDA Breakthrough Therapy designation and Priority Review designation. However, Tolebrutinib is still investigational and not yet approved by the FDA for any indication.

What clinical trials is Tolebrutinib in?

Tolebrutinib has been studied in four clinical trials, including NCT03996291 (Phase 2, relapsing MS, completed), NCT06064539 (Phase 1, drug interaction, completed), NCT06106074 (Phase 1, tolerability and pharmacokinetics, completed), and NCT06372145 (Phase 3, long-term safety, active not recruiting). These trials have enrolled approximately 4,498 participants.

Is Tolebrutinib the same as SAR442168?

Yes, Tolebrutinib is also known as SAR442168. Clinical trial records frequently refer to the drug as SAR442168, such as in the Phase 2 study NCT03996291 and Phase 1 studies NCT06064539 and NCT06106074. Both names refer to the same investigational compound.