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RPC1063

Phase 3

Multiple Sclerosis | Small molecule | Immunology |Bristol-Myers Squibb Company|Last Updated: Jan 30, 2024

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedCONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment2,516
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02576717A Multi-Site, Open-Label Extension Trial of Oral RPC1063 in Relapsing Multiple SclerosisPHASE3 COMPLETED 2,494Oct 16, 2015Jan 5, 2023Jan 30, 2024175 United States, Belarus +24
NCT02797015Pharmacokinetics and Pharmacodynamics Study of RPC1063 in RMSPHASE1 COMPLETED 22Jun 23, 2016Oct 20, 2017Mar 27, 20186 United States
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Study Endpoints
Primary Endpoints
Number of Participants Experiencing Adverse Events (AEs)
From first dose to 90-days post last dose (an average of 65 months up to a max of 85 months)

An AE is any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease temporarily associated with the use of medicinal product, whether or not considered related to the investigational medicinal product.

Number of Participants Experiencing Serious Adverse Events (SAEs)
From first dose to 90-days post last dose (an average of 65 months up to a max of 85 months)

A Serious Adverse Event (SAE) is any untoward medical occurrence that at any dose results in death, is life-threatening (defined as an event in which the subject was at risk of death at the time of the event; it does not refer to an event which hypothetically might have caused death if it were more severe), requires inpatient hospitalization or causes prolongation of existing hospitalization.

Number of Participants Experiencing Adverse Events (AEs) Leading to Discontinuation
From first dose to 90-days post last dose (an average of 65 months up to a max of 85 months)

An AE is any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease temporarily associated with the use of medicinal product, whether or not considered related to the investigational medicinal product.

Number of Participants Experiencing Adverse Events (AEs) Leading to Withdrawal
From first dose to 90-days post last dose (an average of 65 months up to a max of 85 months)

An AE is any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease temporarily associated with the use of medicinal product, whether or not considered related to the investigational medicinal product.

Number of Participants Experiencing Adverse Events (AEs) of Special Interest
From first dose to 90-days post last dose (an average of 65 months up to a max of 85 months)

An AE is any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease temporarily associated with the use of medicinal product, whether or not considered related to the investigational medicinal product.

Number of Participants With Abnormalities in Blood Absolute Lymphocyte Count (ALC)
From first dose up until last dose of study treatment (up to approximately 82 months)

An absolute lymphocyte count (ALC) is a part of a blood test that measures the number of lymphocytes, a type of white blood cell, in the blood. Lymphocytes help fight infections and diseases. Reductions in ALC levels for participants in this study is expected and is a primary pharmacodynamic effect of RPC1063. LLN = Lower limit of normal

Number of Participants With Abnormalities in White Blood Cell Count (WBC)
From first dose up until last dose of study treatment (up to approximately 82 months)

A white blood cell count is a part of a blood test that measures the number of white blood cells in the blood. White blood cells help fight infections and diseases. LLN = Lower limit of normal

Number of Participants With Abnormalities in Blood Absolute Neutrophil Count (ANC)
From first dose up until last dose of study treatment (up to approximately 82 months)

An absolute neutrophil count is a part of a blood test that measures the number of neutrophils, a type of white blood cell, in the blood. Neutrophils help fight infections and diseases.

Number of Participants With Abnormalities in Specific Liver Function Tests
From first dose up until last dose of study treatment (up to approximately 82 months)

The number of participants with laboratory abnormalities in specific liver tests above ULN by category. ULN = Upper Limit of Normal

Number of Participants With Electrocardiogram (ECG) Result Abnormalities
From first dose to 28-days post last dose (an average of 63 months up to a max of 83 months)

An electrocardiogram (ECG) measures electrical activity of the heart to detect cardiac problems.

Number of Participants With Clinically Relevant Abnormalities in Vital Signs
At baseline and 60 months after first dose of study therapy

Vital signs included body temperature, sitting heart rate/pulse (HR), sitting systolic blood pressure (SBP), sitting diastolic blood pressure (DBP). Baseline refers to assessments made on or before the first day participants received study treatment.

Number of Participants With Physical Examination Abnormalities
At baseline and every 12 months thereafter up until 84 months post first dose.

The number of participants with abnormal physical examination results. The assessments included abdominal, extremity, head, heart, lungs, neck, neurological non-MS, other and skin assessments. Baseline refers to assessments made on or before the first day participants received study treatment.

Number of Participants Self-Identifying Suicidality by Columbia-Suicide Severity Rating Scale (C-SSRS)
At baseline and every 3 months thereafter up until 78 months post first dose.

The Columbia-Suicide Severity Rating Scale (C-SSRS) is a unique suicide risk assessment tool that supports suicide risk assessment through a series of simple, plain-language questions. The answers help users identify whether someone is at risk for suicide, assess the severity and immediacy of that risk, and gauge the level of support that the person needs. Results are displayed as the number of participants who answered "Yes" to at least one of the 10 questions in the suicidal ideation or suicidal behavior section. Ideation from 1 (wishing to be dead) - 5 (Active suicidal ideation with specific plan and intent) Behavior from 6 (Preparatory acts or behavior) - 10 (Completed suicide). Baseline refers to assessments made on or before the first day participants received study treatment.

Number of Participants With Changes in Suicidality From Last Day on Treatment Per the Columbia-Suicide Severity Rating Scale (C-SSRS)
1, 4, 7, 14, 21, 28, and 90 days post last dose.

The Columbia-Suicide Severity Rating Scale (C-SSRS) is a unique suicide risk assessment tool that supports suicide risk assessment through a series of simple, plain-language questions. The answers help users identify whether someone is at risk for suicide, assess the severity and immediacy of that risk, and gauge the level of support that the person needs. Results are displayed as the number of participants who answered "Yes" to at least one of the 10 questions in the suicidal ideation or suicidal behavior section. Ideation from 1 (wishing to be dead) - 5 (Active suicidal ideation with specific plan and intent) Behavior from 6 (Preparatory acts or behavior) - 10 (Completed suicide).

Change in Physician's Withdrawal Checklist (PWC-20) Total Score From Last Day on Treatment
1, 4, 7, 14, 21, and 90 days post last dose.

The PWC-20 is a rater-administered 20-item scale to assess signs and symptoms of withdrawal. Twenty items are rated on a 4-point scale as not present (0 points), mild (1 point), moderate (2 points), or severe (3 points). The points from all items are calculated as a total score. Higher scores indicate more severe withdrawal symptoms.

Change in Hospital Anxiety and Depression Scale (HADS) Score From Last Day on Treatment
1, 4, 7, 14, 21, and 90 days post last dose.

The HADS is a validated patient reported outcome for assessing anxiety and depression. It consists of 14 items in total, 7 items related to anxiety and 7 items related to depression. For each item patients select a statement (valued at 0 to 3 points) that closest matches their own feeling over the past week. Separate total scores for anxiety and depression are derived by adding up points. Total scores can range from 0 to 21 points. Higher scores indicate more severe anxiety and depression and scores of 8 to 10 are generally considered indicative of borderline anxiety/depression disorders and scores of 11 and higher are generally considered indicative of anxiety/depression disorders.

Changes in Epworth Sleepiness Scale (ESS) Score From Last Day on Treatment
1, 4, 7, 14, 21, and 90 days post last dose.

The ESS is a validated self administered questionnaire with 8 questions. Respondents rate on a 4-point scale (0 to 3) their chances of dozing off or falling asleep while engaged in 8 different activities. The ESS score is the sum of 8 item scores and can range from 0 to 24 points. Higher scores indicate more daytime sleepiness.

Changes in Vital Sign Values From Last Day on Treatment
1, 4, 7, 14, 21, 28, and 90 days post last dose.

Vital signs included sitting systolic blood pressure (SBP), sitting diastolic blood pressure (DBP).

Maximum plasma concentration (Cmax)
24 hours after the last RPC1063 dose on Day 85
Area under the plasma concentration-time curve (AUC)
Approximately 3 months
Secondary Endpoints
Annualized Relapse Rate (ARR)
From first dose up until last dose of study treatment or data-cutoff date, whichever occurred first (up to approximately 87 months)
Time to First Relapse (TFR)
Overall: From first dose to first relapse, last dose, or data-cutoff date, whichever occurred first (up to approx 87 months); Visits: 2 weeks post first dose, 3 months post first dose, and every 3 months thereafter up until 81 months post first dose.
Number of Participants Who Were Relapse Free
From first dose to last dose of study treatment or data-cutoff date, whichever occurred first (up to approximately 87 months)
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
1 mg RPC1063 (Ozanimod) oral capsuleEXPERIMENTAL1 mg RPC1063 (Ozanimod) oral capsule daily
1 mg RPC1063EXPERIMENTAL1 mg RPC1063 oral capsule daily
0.5 mg RPC1063EXPERIMENTAL0.5 mg RPC1063 oral capsule daily
Interventions
NameTypeDescription
RPC1063DRUG -
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Eligibility Criteria
Age Range18 Years — 55 Years
SexALL
Healthy VolunteersNo
Study Sites175

Eligibility Criteria: To be eligible to participate in this trial, patients must meet all of the following criteria: 1. Completed one of the parent trials 2. Does not have a condition that would require withdrawal from one of the parent trials 3. Has no conditions requiring treatment with a prohib...

Countries:United StatesBelarusBelgiumBosnia and HerzegovinaBulgariaCroatiaEstoniaGeorgiaGermanyGreeceHungaryItalyLatviaLithuaniaMoldovaNew ZealandPolandPortugalRomaniaSerbiaSlovakiaSouth AfricaSpainSwedenUkraineUnited Kingdom
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