Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Rapcabtagene autoleucel (YTB323), rapcabtagene autoleucel (YTB323)
Rapcabtagene autoleucel · 8 trials · 9 indications
Event-free survival (EFS) defined as the time from Randomization to the first occurrence of as per protocol defined events.
The percentage of participants with a TIS of at least 40 at the 52nd week after the start of the study, corresponding to moderate-to-major improvement.
To demonstrate the superiority of rapcabtagene autoleucel as a single infusion compared to rituximab, with respect to the proportion of participants achieving a Revised Composite Response Index in Systemic Sclerosis 50 (rCRISS50) response at Week 52. This response is assessed across 5 assessment domains: (1) modified Rodnan Skin Score (mRSS), (2) Health Assessment Questionnaire Disability Index (HAQ-DI), (3) patient global assessment (PGA), (4) physician global assessment (PhGA) and (5) percent-predicted forced vital capacity (FVC%).
Defined as: Meeting the criteria of the Definition Of Remission In Systemic Lupus Erythematosus (DORIS) or Achieving complete renal response (CRR)
Proportion of participants who achieve conversion from MRD-positive status at baseline to MRD-negative status at Day 90 (± 2 weeks) following infusion of rapcabtagene autoleucel (YTB323).
Safety parameters include vital signs, adverse events, laboratory parameters and ECG evaluation
Incidence of dose limiting toxicities (DLTs), AEs and SAEs, including changes in vital signs, electrocardiograms (ECGs), laboratory parameters, neurological status and magnetic resonance (MRI) of the brain and spinal cord qualifying and reported as AEs.
Occurrence, severity, and frequency of dose limiting toxicities (DLTs), AEs and SAEs, including changes in vital signs, electrocardiograms (ECGs), laboratory parameters, neurological status and magnetic resonance (MRI) of the brain and spinal cord qualifying and reported as AEs.
| Arm | Type | Description |
|---|---|---|
| Rapcabtagene autoleucel | EXPERIMENTAL | Single infusion of rapcabtagene autoleucel (YTB323) and concomitant glucocorticoids as per protocol |
| Active comparator | ACTIVE_COMPARATOR | Comparator and concomitant glucocorticoids as per protocol |
| Comparator | ACTIVE_COMPARATOR | Investigator choice of treatment as per protocol. (Tacrolimus, Mycophenolate mofetil, Cyclophosphamide, or Rituximab) |
| rapcabtagene autoleucel arm | EXPERIMENTAL | rapcabtagene autoleucel |
| rituximab arm | ACTIVE_COMPARATOR | rituximab |
| Observational Cohort (MRD-Negative) | NO_INTERVENTION | Participants who are minimal residual disease (MRD) negative following frontline therapy will not receive study intervention and will be followed for subsequent treatments, response, disease status, and survival. |
| Rapcabtagene Autoleucel (YTB323) Treatment Cohort | EXPERIMENTAL | Participants who are MRD-positive following frontline therapy and meet eligibility criteria will undergo leukapheresis, lymphodepleting chemotherapy, and infusion of rapcabtagene autoleucel (YTB323). Participants will be followed for MRD conversion, safety, disease status, survival, and long-term gene therapy follow-up. |
| Rapcabtagene autoleucel-rheumatoid arthritis | EXPERIMENTAL | Single infusion of Rapcabtagene autoleucel in participants with rheumatoid arthritis |
| Rapcabtagene autoleucel- Sjögren's Disease | EXPERIMENTAL | Single infusion of Rapcabtagene autoleucel in participants with Sjögren's Disease |
| YTB323 Cohort 1 | EXPERIMENTAL | Participants will receive one dose of YTB323 |
| YTB323 Cohort 2 | EXPERIMENTAL | Participants will receive one dose of YTB323 |
| YTB323 Cohort 3 | EXPERIMENTAL | Participants will receive one dose of YTB323 |
| YTB323 Cohort 4 | EXPERIMENTAL | Participants will receive one dose of YTB323 |
| Name | Type | Description |
|---|---|---|
| Rapcabtagene autoleucel | BIOLOGICAL | Single infusion of rapcabtagene autoleucel |
| Active Comparator | OTHER | Active comparator option as per protocol |
| Glucocorticoids | DRUG | Concomitant glucocorticoids as per protocol |
| Active Comparator Option | OTHER | Investigator choice of treatment as per protocol |
| rituximab | BIOLOGICAL | rituximab intravenous infusion (i.v.) as per protocol |
| Rapcabtagene autoleucel (YTB323) | BIOLOGICAL | Autologous CD19-directed chimeric antigen receptor (CAR) T-cell therapy manufactured from participant-derived T cells. Participants undergo leukapheresis for cell collection, receive lymphodepleting chemotherapy, followed by a single intravenous infusion of rapcabtagene autoleucel (YTB323). |
Key inclusion criteria: 1. Men and women, aged ≥18 and ≤ 75 years with a diagnosis of GPA or MPA according to the American College of Rheumatology/ European League Against Rheumatism 2022 (ACR/EULAR 2022) classification criteria 2. Positive test for ANCA-autoantibodies 3. GPA and MPA participants w...
Rapcabtagene autoleucel is an investigational cell therapy being studied for multiple autoimmune and inflammatory conditions, including systemic lupus erythematosus, lupus nephritis, rheumatoid arthritis, Sjögren's disease, diffuse scleroderma, idiopathic inflammatory myopathies, progressive multiple sclerosis, and ANCA-associated vasculitis.
Rapcabtagene autoleucel is a chimeric antigen receptor T-cell therapy, a type of cell therapy that is engineered to target and eliminate specific cells involved in autoimmune diseases. Its mechanism involves redirecting a patient's own T cells to attack pathogenic immune cells.
Rapcabtagene autoleucel is being developed by Novartis AG, a global pharmaceutical company traded on the New York Stock Exchange under the ticker symbol NVS.
Rapcabtagene autoleucel is in Phase 2 clinical development for systemic lupus erythematosus and lupus nephritis, and Phase 2 for myositis. It is also being studied in Phase 1 trials for relapsing multiple sclerosis and large-cell lymphoma. It remains investigational and is not yet approved.
Rapcabtagene autoleucel is being evaluated in several trials, including NCT06581198 for systemic lupus erythematosus and lupus nephritis, NCT06617793 for relapsing multiple sclerosis, NCT06665256 for myositis, and NCT07443137 for large-cell lymphoma.
Yes, Rapcabtagene autoleucel is also known as YTB323. Both names refer to the same investigational cell therapy being developed by Novartis.