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Riociguat

Phase 3

Hypertension, Pulmonary | Small molecule | Cardiovascular |Merck & Company, Inc.|Last Updated: May 12, 2026

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
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment24
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02562235Riociguat in Children With Pulmonary Arterial Hypertension (PAH)PHASE3 ACTIVE NOT_RECRUITING 24Oct 29, 2015Aug 3, 2027May 12, 202616 Colombia, Germany +7
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Study Endpoints
Primary Endpoints
Number of Participants With Any Treatment-emergent Adverse Events
From start of study drug up to 2 days after the last dose of study drug in the main study part, up to 24 weeks plus/minus 5 days.

An adverse event (AE), including AE in relation to a medical device (i.e. Raumedic dosing pipette), is any untoward medical occurrence in a participant administered with a pharmaceutical product and does not necessarily have to have a causal relationship with this treatment. A serious AE (SAE) is any untoward medical occurrence that at any dose is resulting in death, is lifethreatening, requires hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity. AEs occurring between start of study drug and up to 2 days after the last dose were defined as treatment-emergent AEs (TEAEs).

Change in Heart Rate From Baseline
Baseline and Week 24 (plus/minus 5 days)

Mean change in heart rate from baseline is reported.

Change in Blood Pressure From Baseline
Baseline and Week 24 (plus/minus 5 days)

Mean changes in systolic blood pressure (SBP) and diastolic blood pressure (DBP) from baseline are reported.

Change in Respiratory Rate From Baseline
Baseline and Week 24 (plus/minus 5 days)

Mean change in respiratory rate from baseline is reported.

Number of Subjects With Transitions From Baseline in Bone Age Compared to Chronological Age
Baseline and Week 24 (plus/minus 5 days)

X-ray of left hand was performed for each participant and bone age was determined centrally by a specialist. For each participant, the bone age was compared to the chronological age and assigned to one of the categories - "delayed", "in accordance" or "advanced", indicating the advancement or delay in the growth of the bone. Number of participants who transitioned to another category different from baseline was calculated and is reported.

Change in Hematology Parameters (Platelets) From Baseline
Baseline and Week 24 (plus/minus 5 days)

Hematology parameters were collected. Parameters with a decrease or increase in the mean value compared to baseline are reported in this data set.

Change in Hematology Parameters (Lymphocytes/Leucocytes Ratio) From Baseline
Baseline and Week 24 (plus/minus 5 days)

Hematology parameters were collected. Parameters with a decrease or increase in the mean value compared to baseline are reported in this data set.

Change in Hematology Parameter (Neutrophils/Leucocytes Ratio) From Baseline
Baseline and Week 24 (plus/minus 5 days)

Hematology parameters were collected. Parameters with a decrease or increase in the mean value compared to baseline are reported in this data set.

Change in Clinical Chemistry (Alanine Aminotransferase) From Baseline
Baseline and Week 24 (plus/minus 5 days)

Clinical chemistry parameters were collected and analyzed. Parameters with a trend to lower or higher mean values from baseline are reported in this data set.

Change in Clinical Chemistry (Aspartate Aminotransferase) From Baseline
Baseline and Week 24 (plus/minus 5 days)

Clinical chemistry parameters were collected and analyzed. Parameters with a trend to lower or higher mean values from baseline are reported in this data set.

Change in Clinical Chemistry (Sodium) From Baseline
Baseline and Week 24 (plus/minus 5 days)

Clinical chemistry parameters were collected and analyzed. Parameters with a trend to lower or higher mean values from baseline are reported in this data set.

Change in Clinical Chemistry (Blood Urea Nitrogen) From Baseline
Baseline and Week 24 (plus/minus 5 days)

Clinical chemistry parameters were collected and analyzed. Parameters with a trend to lower or higher mean values from baseline are reported in this data set.

Change in Clinical Chemistry (eGFR) From Baseline
Baseline and Week 24 (plus/minus 5 days)

Clinical chemistry parameters were collected and analyzed. Parameters with a trend to lower or higher mean values from baseline are reported in this data set. eGFR = estimated glomerular filtration rate

Change in Clinical Chemistry (Urea) From Baseline
Baseline and Week 24 (plus/minus 5 days)

Clinical chemistry parameters were collected and analyzed. Parameters with a trend to lower or higher mean values from baseline are reported in this data set.

Change in Clinical Chemistry (Gamma Glutamyl Transferase) From Baseline
Baseline and Week 24 (plus/minus 5 days)

Clinical chemistry parameters were collected and analyzed. Parameters with a trend to lower or higher mean values from baseline are reported in this data set.

Plasma Concentration of Riociguat at Week 0
Week 0 (30-90 minutes post-dose; 2.5-4 hours post-dose)

For each participant, one blood sample was collected at one given time point. Values below lower limit of quantification (LLOQ) were substituted by 1/2 LLOQ for the calculation in statistics. Means at any time were only calculated if at least 2/3 of the individual data were measured and were above the limit of quantification (LOQ). Geometric mean and percentage geometric coefficient of variation (%CV) are reported. W = Week.

Plasma Concentration of Riociguat at Week 4
Week 4 (pre-dose)

For each participant, one blood sample was collected at one given time point. Values below lower limit of quantification (LLOQ) were substituted by 1/2 LLOQ for the calculation in statistics. Means at any time were only calculated if at least 2/3 of the individual data were measured and were above the limit of quantification (LOQ). Geometric mean and percentage geometric coefficient of variation (%CV) are reported.

Plasma Concentration of Riociguat at Week 8
Week 8 (pre-dose)

For each participant, one blood sample was collected at one given time point. Values below lower limit of quantification (LLOQ) were substituted by 1/2 LLOQ for the calculation in statistics. Means at any time were only calculated if at least 2/3 of the individual data were measured and were above the limit of quantification (LOQ). Geometric mean and percentage geometric coefficient of variation (%CV) are reported.

Plasma Concentration of BAY60-4552 at Week 0
Week 0 (30-90 minutes post-dose; 2.5-4 hours post-dose)

BAY60-4552 is riociguat's active metabolite. For each participant, one blood sample was collected at one given time point and in that sample both riociguat and BAY60-4552 were measured. Values below lower limit of quantification (LLOQ) were substituted by 1/2 LLOQ for the calculation in statistics. Means at any time were only calculated if at least 2/3 of the individual data were measured and were above the limit of quantification (LOQ). Geometric mean and percentage geometric coefficient of variation (%CV) are reported. W = Week

Plasma Concentration of BAY60-4552 at Week 4
Week 4 (pre-dose)

BAY60-4552 is riociguat's active metabolite. For each participant, one blood sample was collected at one given time point and in that sample both riociguat and BAY60-4552 were measured. Values below lower limit of quantification (LLOQ) were substituted by 1/2 LLOQ for the calculation in statistics. Means at any time were only calculated if at least 2/3 of the individual data were measured and were above the limit of quantification (LOQ). Geometric mean and percentage geometric coefficient of variation (%CV) are reported.

Plasma Concentration of BAY60-4552 at Week 8
Week 8 (pre-dose)

BAY60-4552 is riociguat's active metabolite. For each participant, one blood sample was collected at one given time point and in that sample both riociguat and BAY60-4552 were measured. Values below lower limit of quantification (LLOQ) were substituted by 1/2 LLOQ for the calculation in statistics. Means at any time were only calculated if at least 2/3 of the individual data were measured and were above the limit of quantification (LOQ). Geometric mean and percentage geometric coefficient of variation (%CV) are reported.

Secondary Endpoints
Change in 6-minute Walking Distance From Baseline
Baseline and Week 24 (plus/minus 5 days)
Number of Subjects With Change in WHO Functional Class From Baseline
Baseline and Week 24 (plus/minus 5 days)
Change in NT-proBNP From Baseline
Baseline and Week 24 (plus/minus 5 days)
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
RiociguatEXPERIMENTALParticipants with age ≥6 to \<18 years received riociguat up to 2.5 mg three times a day (titration between 1.0 mg and 2.5 mg) for up to 8 weeks during the individual dose titration (IDT) phase, and followed with the last dose administered in the IDT phase for up to 16 weeks during the maintenance phase. Down-titration (up to 0.5 mg) of the dose for safety reasons was allowed at any time.
Interventions
NameTypeDescription
Riociguat (Adempas, BAY63-2521)DRUGFor children with body-weight \<50 kg at screening: body-weight adjusted dose equivalent to the exposure of (0.5 mg) 1.0 - 2.5 mg three times a day, IDT in adults treated for PAH; oral suspension. For children ≥50 kg at screening: 1.0 to 2.5 mg three times a day; oral tablet.
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Eligibility Criteria
Age Range6 Years — 17 Years
SexALL
Healthy VolunteersNo
Study Sites16

Inclusion Criteria: * Children from 6 years to less than 18 years of age with pulmonary arterial hypertension (PAH) * Diagnosed with PAH : * Idiopathic (IPAH) * Hereditable (HPAH) * PAH associated with (APAH) * Connective tissue disease * Congenital heart disease with shunt closure ...

Countries:ColombiaGermanyHungaryItalyJapanMexicoPolandTaiwanTurkey (Türkiye)
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Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT02562235studyFirstPostDate: changed