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Ralinepag

Phase 3

PAH | Small molecule | Immunology |United Therapeutics Corporation|Last Updated: Jun 16, 2026

Target and mechanism

Molecular targetPTGIR
Target classAgonist
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials2
Total Enrollment1,687

FDA Designations

No designations recorded

Clinical trial landscape

Ralinepag · 3 trials · 11 indications

Phase 3 2Phase 2 1
NCT03683186A Study Evaluating the Long-Term Efficacy and Safety of Ralinepag in Subjects With PAH Via an Open-Label ExtensionPAH
ENROLLING BY_INVITATION1,000 Analytics
NCT03626688A Study Evaluating the Efficacy and Safety of Ralinepag to Improve Treatment Outcomes in PAH PatientsPAH
COMPLETED687 Analytics
PHASE3ENROLLING BY_INVITATION
A Study Evaluating the Long-Term Efficacy and Safety of Ralinepag in Subjects With PAH Via an Open-Label Extension
PAHUnlock trial analytics
PHASE3COMPLETED
A Study Evaluating the Efficacy and Safety of Ralinepag to Improve Treatment Outcomes in PAH Patients
PAHUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of subjects with treatment-emergent adverse events [Safety and Tolerability]
Up to 6 years

The safety and tolerability of ralinepag will be evaluated by the Number of subjects with treatment-emergent adverse events

Time from randomization to the first adjudicated protocol-defined clinical worsening event
The study duration was event-based. This parameter was assessed from randomization until the conclusion of the study, up to 3 years

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).

Change From Baseline in Pulmonary Vascular Resistance
At 1 or 2 years after the subject enrolled into the study, pending their last RHC prior to Protocol Amendment 2.

Pulmonary vascular resistance was collected by right heart catheterization (RHC).

Change From Baseline in Cardiac Output
At 1 or 2 years after the subject enrolled into the study, pending their last RHC prior to Protocol Amendment 2.

Cardiac output was collected by right heart catheterization (RHC).

Change From Baseline in Cardiac Index
At 1 or 2 years after the subject enrolled into the study, pending their last RHC prior to Protocol Amendment 2.

Cardiac index was collected by right heart catheterization (RHC).

Change From Baseline in Mean Pulmonary Arterial Pressure
At 1 or 2 years after the subject enrolled into the study, pending their last RHC prior to Protocol Amendment 2.

Mean pulmonary arterial pressure was collected by right heart catheterization (RHC).

Secondary Endpoints

Change from Baseline in N-terminal pro-brain natriuretic peptide (NT-proBNP)
Baseline to Week 28
Change from Baseline in 6-minute walk distance (6MWD)
Baseline to Week 28
Change from Baseline in WHO/New York Heart Association (NYHA) Functional Class
Baseline to Week 28
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
RalinepagEXPERIMENTALRalinepag once daily extended-release tablets (oral) 50, 250, and 400 mcg titrated to the highest tolerated dose.
PlaceboPLACEBO_COMPARATORMatching placebo tablets (oral)
Oral RalinepagEXPERIMENTALRalinepag 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.

Interventions

NameTypeDescription
RalinepagDRUGActive
PlaceboDRUGPlacebo
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites190

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...

Countries:United StatesArgentinaAustraliaAustriaBelgiumBrazilBulgariaCanadaChileChinaCroatiaCzechiaDenmarkFranceGermanyGreeceHungaryIsraelItalyMexicoNetherlandsPolandPortugalRomaniaSerbiaSingaporeSouth KoreaSpainSwedenTaiwanTurkey (Türkiye)UkraineUnited KingdomSlovakia
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Recent Changes (Last 90 Days)

MEDIUMJul 17, 2026NCT03626688TRIAL_REMOVED: changed
MEDIUMJul 17, 2026NCT03626688TRIAL_REMOVED: changed
MEDIUMJul 17, 2026NCT03626688TRIAL_REMOVED: changed

Frequently asked questions about Ralinepag

What is Ralinepag used for in PAH?

Ralinepag is an investigational small molecule being developed for pulmonary arterial hypertension (PAH). It is designed to treat PAH by targeting the prostacyclin receptor. Ralinepag is currently in Phase 3 clinical development and is not yet approved by regulatory authorities.

What does Ralinepag target?

Ralinepag targets the prostacyclin receptor (PTGIR) and acts as an agonist. By activating this receptor, it aims to produce effects relevant to treating pulmonary arterial hypertension. The drug is being studied for its potential to improve treatment outcomes in PAH patients.

Who makes Ralinepag?

Ralinepag is being developed by United Therapeutics Corporation, a biopharmaceutical company listed on the NASDAQ under the ticker symbol UTHR. The company is conducting clinical trials to evaluate the drug's efficacy and safety for pulmonary arterial hypertension.

What phase is Ralinepag in?

Ralinepag is in Phase 3 clinical development for pulmonary arterial hypertension. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory agencies. The Phase 3 program includes studies evaluating its efficacy, safety, and long-term outcomes.

What clinical trials is Ralinepag in?

Ralinepag has been studied in clinical trials including NCT02279745, a completed Phase 2 long-term safety and efficacy study, and NCT03626688, a completed Phase 3 efficacy and safety study. An open-label extension trial, NCT03683186, is enrolling by invitation to evaluate long-term safety and efficacy.

Is Ralinepag the same as any other drug?

Ralinepag is a distinct investigational compound and is not known to be the same as any other marketed drug. It is a small molecule prostacyclin receptor agonist being developed specifically for pulmonary arterial hypertension, with no alternative names reported.