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Also known as RE-021 (Sparsentan), RE-021
RE-021, sparsentan · 2 trials · 2 indications
Primary efficacy objective is to determine the change in UP/C in FSGS patients receiving RE-021 (Sparsentan) from baseline to 8 weeks over a range of dose levels compared to treatment with irbesartan as active control.
Evaluation of, at minimum, the maximum concentration (Cmax) parameters for sparsentan following a single dose of sparsentan as an oral suspension in the fed or fasted state.
Evaluation of, at minimum, the area under the concentration-time curve from dosing time 0 (AUC(0-last)) parameters for sparsentan following a single dose of sparsentan as an oral suspension in the fed or fasted state.
Evaluation of, at minimum, the area under the concentration curve to infinite (AUC(0-inf)) time parameters for sparsentan following a single dose of sparsentan as an oral suspension in the fed or fasted state.
Evaluation of the ratio, fed/fasted, for the Cmax
Evaluation of the ratio, fed/fasted, for the AUC(0-last)
Evaluation of the ratio, fed/fasted, for the AUC(0-inf)
Evaluation of, at minimum, the following pharmacokinetics parameters for sparsentan following multiple doses of sparsentan as an oral suspension in the fed state: Cmax from a single dose for Study days 18 and 25 (Period 3, Days 7 and 14)
Evaluation of, at minimum, the following pharmacokinetics parameters for sparsentan following multiple doses of sparsentan as an oral suspension in the fed state: Area under the plasma concentration-time curve over the last 24-h dosing interval (AUC(0-24)) from a single dose for Study days 18 and 25 (Period 3, Days 7 and 14)
Evaluation of, at minimum, the following pharmacokinetics parameters for sparsentan following multiple doses of sparsentan as an oral suspension in the fed state: temporal change parameter (TCP) (AUC(0-24)/AUC(0-inf)) from a single dose for Study days 18 and 25 (Period 3, Days 7 and 14)
| Arm | Type | Description |
|---|---|---|
| RE-021 (Sparsentan) 200 mg - Double-Blind Period | EXPERIMENTAL | RE-021 (Sparsentan) will be administered as a single oral morning dose. In this ARM the RE-021 (Sparsentan) dose will be 200mg. Patients at \</= 50kg will receive half of the RE-021 (Sparsentan) dose for the 8 week duration. |
| RE-021 (Sparsentan) 400 mg - Double-Blind Period | EXPERIMENTAL | RE-021 (Sparsentan) will be administered as a single oral morning dose. In this ARM the RE-021 (Sparsentan) dose will be 400mg. Patients at \</= 50kg will receive half of the RE-021 (Sparsentan) dose for the 8 week duration. |
| RE-021 (Sparsentan) 800 mg - Double-Blind Period | EXPERIMENTAL | RE-021 (Sparsentan) will be administered as a single oral morning dose. In this ARM the RE-021 (Sparsentan) dose will be 800mg. Patients at \</= 50kg will receive half of the RE-021 (Sparsentan) dose for the 8 week duration. |
| Irbesartan 300 mg - Double-Blind Period | ACTIVE_COMPARATOR | The control will be administered irbesartan as a single oral dose of 150mg for the first week before escalating to 300mg for the remaining 7 weeks. Patients at \</= 50kg will receive 150mg irbesartan for the 8 week duration. |
| RE-021 (Sparsentan) - Open-Label Extension Period | EXPERIMENTAL | Includes all subjects who completed the Double-Blind period and enrolled in the Open-Label Extension period of the study. All subjects who completed the Double-Blind period were evaluated for response and safety at the Week 8 visit to determine eligibility for continued treatment on their assigned doses in an Open-Label Extension period for up to 496 additional weeks. Subjects treated with irbesartan during the Double-Blind period were offered sparsentan treatment at the dose they would have received according to the Double-Blind dose cohort in which they were enrolled. |
| Fasted State | EXPERIMENTAL | Sparsentan will be administered in 3-dose level in healthy subjects. Subjects will be randomized to 1 of 3 dose levels (200mg, 400mg and 800 mg) and will receive a single dose of sparsentan in the fasted state on Period 1, Day 1 (Study Day 1) |
| Fed State | EXPERIMENTAL | Sparsentan will be administered in 3-dose level in healthy subjects. Subjects will have a single dose of sparsentan (200mg, 400mg and 800 mg) in the fed state (high-fat breakfast) on Period 2, Day 1 (Study Day 8) |
| Fed State - Multiple | EXPERIMENTAL | Sparsentan will be administered in 3-dose level in healthy subjects. Subjects will have multiple doses of sparsentan (200mg, 400mg and 800 mg) in the fed state on Period 3, Days 1 to 14 (Study days 12 to 25) |
| Name | Type | Description |
|---|---|---|
| RE-021 (Sparsentan) | DRUG | Oral, once-daily |
| Irbesartan | DRUG | Oral, once-daily |
| RE-021, sparsentan | DRUG | RE-021, sparsentan - Subjects will be randomized 1 of 3 dose level |
Inclusion Criteria 1. Biopsy-proven FSGS OR documentation of a genetic mutation in a podocyte protein associated with the disease. 2. Urine protein/creatinine ratio (Up/C) at or above 1.0 g/g. 3. Estimated glomerular filtration rate (eGFR) \>30. 4. Mean seated blood pressure (BP) \>100/60 mmHg and ...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Apellis Pharmaceuticals, Inc. | APLS | 1 | PHASE2 | APL-2 |
| Sanofi SA Sponsored ADR | SNY | 1 | PHASE2 | Frexalimab, Brivekimig, rilzabrutinib |
| Novartis AG Sponsored ADR | NVS | 1 | PHASE2 | Atrasentan |
| Akebia Therapeutics, Inc. | AKBA | 1 | PHASE2 | Praliciguat |
| Travere Therapeutics, Inc. | TVTX | 1 | PHASE2 | Sparsentan |
| Vera Therapeutics, Inc. Class A | VERA | 1 | PHASE2 | Atacicept |
RE-021, also known as sparsentan, is being studied for focal segmental glomerulosclerosis, a kidney condition that scars the glomeruli and leads to protein in the urine. It has also been evaluated in healthy subjects in a pharmacokinetic study of an oral suspension. It is an investigational small molecule in the nephrology therapeutic area.
RE-021 (sparsentan) is being developed by Travere Therapeutics, Inc., which trades on the Nasdaq under the ticker TVTX. The company is the sponsor behind the clinical program evaluating the small molecule in focal segmental glomerulosclerosis and in healthy subjects.
RE-021 (sparsentan) is in Phase 2 development for focal segmental glomerulosclerosis. A Phase 2 randomized, double-blind, active-controlled safety and efficacy study has been completed. A separate Phase 1 pharmacokinetic study of an oral sparsentan suspension in healthy subjects has also been completed. The drug remains investigational and is not approved.
Two completed trials are registered for RE-021 (sparsentan). NCT01613118 was a randomized, double-blind, active-controlled Phase 2 safety and efficacy study in focal segmental glomerulosclerosis that enrolled 109 patients in the United States, Czechia, and Italy. NCT05562362 was a Phase 1 pharmacokinetic study of oral sparsentan suspension in 47 healthy subjects in the United Kingdom.
Yes, RE-021 and sparsentan are the same investigational drug. RE-021 is the earlier code name used in the Phase 2 focal segmental glomerulosclerosis trial, while sparsentan is the name used in later study titles, including the Phase 1 pharmacokinetic trial of the oral suspension. Both names refer to the same small molecule developed by Travere Therapeutics.
Across the two completed trials, 156 participants have been enrolled for RE-021 (sparsentan). The Phase 2 focal segmental glomerulosclerosis study NCT01613118 enrolled 109 patients aged 8 years and older, and the Phase 1 pharmacokinetic study NCT05562362 enrolled 47 healthy adult subjects. The Phase 2 trial was randomized, double-blind, and active-controlled.