Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as APD811
Ralinepag · 4 trials · 11 indications
The safety and tolerability of ralinepag will be evaluated by the Number of subjects with treatment-emergent adverse events
Clinical worsening events are defined as death, nonelective hospital admission for worsening PAH (further defined in clinical study protocol), initiation of parenteral or inhaled prostacyclin pathway agent for treatment of worsening PAH, disease progression (further defined in clinical study protocol), or unsatisfactory long-term clinical response (further defined in clinical study protocol).
Pulmonary vascular resistance was collected by right heart catheterization (RHC).
Cardiac output was collected by right heart catheterization (RHC).
Cardiac index was collected by right heart catheterization (RHC).
Mean pulmonary arterial pressure was collected by right heart catheterization (RHC).
Measurements of PVR from right heart catheterization were obtained prior to Day 1 of the dose titration period and at the end of the maintenance period (Week 22), approximately 4 hours after the last dose of study drug.
The 6MWT was conducted according to the modified guidelines issued by the American Thoracic Society prior to Day 1 of the dose titration period and at the end of the maintenance period (Week 22).
| Arm | Type | Description |
|---|---|---|
| Ralinepag | EXPERIMENTAL | Ralinepag once daily extended-release tablets (oral) 50, 250, and 400 mcg titrated to the highest tolerated dose. |
| Placebo | PLACEBO_COMPARATOR | Matching placebo tablets (oral) |
| Oral Ralinepag | EXPERIMENTAL | Ralinepag immediate-release (IR) capsules of 10, 20, 30, 40, and 100 mcg or extended-release (XR) tablets of 50, 250, and 400 mcg for oral administration. |
| APD811 | EXPERIMENTAL | Multiple dose titration to maximum tolerated dose. |
| Name | Type | Description |
|---|---|---|
| Ralinepag | DRUG | Active |
| Placebo | DRUG | Placebo |
| APD811 | DRUG | - |
Inclusion Criteria: 1. Evidence of a personally signed and dated informed consent form indicating that the subject has been informed of all pertinent aspects of the study prior to initiation of any study-related procedures. 2. Subjects who are willing and able to comply with scheduled visits, treat...
Ralinepag is an investigational oral small molecule being developed for the treatment of pulmonary arterial hypertension (PAH). It is not yet approved and remains in clinical development, with a New Drug Application or Biologics License Application stage reached. It is studied in adults with PAH, including connective tissue disease associated and familial forms.
Ralinepag targets PTGIR, the prostacyclin receptor, and acts as an agonist. By stimulating this receptor, it mimics the effects of prostacyclin, a natural vasodilator. This mechanism is intended to relax pulmonary blood vessels and slow disease progression in pulmonary arterial hypertension.
Ralinepag is developed by United Therapeutics Corporation, which trades on the Nasdaq under the ticker UTHR. The company is responsible for its clinical development program in pulmonary arterial hypertension.
Ralinepag has reached the NDA/BLA stage, meaning a marketing application has been submitted to regulators. It is still considered investigational and is not yet approved. The program includes completed Phase 3 and Phase 2 trials along with an ongoing long-term extension study.
Ralinepag has been studied in three registered trials. NCT03626688 was a completed Phase 3 efficacy and safety study in 687 PAH patients. NCT03683186 is an ongoing Phase 3 open-label extension enrolling 1,000 patients. NCT02279745 was a completed Phase 2 long-term safety study in 45 patients.