United Therapeutics Corporation · NASDAQ:UTHR
United Therapeutics — Technical Presentation (March 2026)
21 slides from United Therapeutics's technical presentation, Mar 2, 2026
Slide 1: Phase 3 ADVANCE OUTCOMES Results Webcast
United Therapeutics Corporation Phase 3 ADVANCE OUTCOMES Results Webcast MARCH 2, 2026
Slide 2: Safe Harbor Statement
INTRODUCTION 2 2 Safe Harbor Statement All statements in this presentation are made as of March 2, 2026. We undertake no obligation to publicly update or revise these statements, whether as a result of new information, future events, or otherwise. Statements included in this presentation that are no
Slide 3: Today's Speakers
3 INTRODUCTION Today’s Speakers Dr. Martine Rothblatt Dr. Derek Solum Chairperson and Chief Executive Senior Director, Product Development and Officer Lead for the Global ADVANCE OUTCOMES Program Michael Benkowitz Dr. Daniel Lachant, DO, MSCI, President and Chief Operating ATSF Officer Associate Pro
Slide 4: Dr. Martine Rothblatt
4 Dr. Martine Rothblatt CHAIRPERSON AND CHIEF EXECUTIVE OFFICER
Slide 5: ADVANCE OUTCOMES Headline Conclusions
5 ADVANCE OUTCOMES RESULTS ADVANCE OUTCOMES Headline Conclusions 1 2 3 Ralinepag reduced the The study also met Treatment with ralinepag risk of a clinical several important was well-tolerated, and the safety profile was worsening event versus secondary endpoints, consistent with known placebo by 55
Slide 6: Dr. Derek Solum
6 Dr. Derek Solum SENIOR DIRECTOR, PRODUCT DEVELOPMENT AND LEAD FOR THE GLOBAL ADVANCE OUTCOMES PROGRAM
Slide 7: ADVANCE OUTCOMES Study Design
ADVANCE OUTCOMES DESIGN 7 ADVANCE OUTCOMES Study Design1 PRIMARY ENDPOINT STUDY VISIT SCHEDULE Time from randomization to the first adjudicated protocol-defined clinical worsening event All participants receiving PAH standard of care or PAH- SECONDARY ENDPOINTS specific background therapy were rando
Slide 8: ADVANCE OUTCOMES Study Design: Clinical Worsening Definition
ADVANCE OUTCOMES DESIGN 8 ADVANCE OUTCOMES Study Design1 CLINICAL WORSENING DEFINITION A protocol-defined clinical worsening event is defined as 1 of the following: • Death (all causes) • Nonelective hospital admission lasting at least 24 hours and/or right heart failure inclusive of lung transplant
Slide 9: Baseline Demographics of Enrolled Subjects
9 ADVANCE OUTCOMES BASELINE Baseline Demographics of Enrolled Subjects Ralinepag Placebo Overall N=350 N=337 N=687 Age at Study Entry (years), median (range) 54.0 (18, 83) 52.0 (19, 84) 53.0 (18, 84) Female (%)/Male (%) 256 (73.1)/94 (26.9) 264 (78.3)/73 (21.7) 520 (75.7)/167 (24.3) Time Since Diagn
Slide 10: Baseline Demographics Cont'd
10 ADVANCE OUTCOMES BASELINE Baseline Demographics Cont’d Ralinepag Placebo Overall N=350 N=337 N=687 Weight (kg), mean (SD) 73.2 (16.3) 73.8 (17.9) 73.5 (17.1) Background Therapy One or No Background Therapy, n (%) 68 (19.4) 71 (21.1) 139 (20.2) Dual Background Therapy, n (%) 282 (80.6) 266 (78.9)
Slide 11: Primary Endpoint
ADVANCE OUTCOMES RESULTS 11 Primary Endpoint Time to Adjudication Clinical Worsening (Weeks); Kaplan-Meier Estimates 1. P value is calculated with log rank test stratified by background PAH therapy and baseline 6MWD category. 2. Hazard ratio, 95% CI, and P value are calculated with proportional haza
Slide 12: Primary Endpoint
ADVANCE OUTCOMES RESULTS 12 Primary Endpoint Adjudicated Clinical Worsening Events Ralinepag Placebo N=350 % N=337 % Clinical worsening All events 64 18.3% 121 35.9% event category as 11 3.1% 10 3.0% Death (all causes) the first event n(%) Hospitalization due to PAH 19 5.4% 20 5.9% Disease Progressi
Slide 13: Primary Endpoint
ADVANCE OUTCOMES RESULTS 13 Primary Endpoint Time to Disease Progression; Kaplan-Meier Estimates
Slide 14: Subgroup Analyses Time to First Clinical Worsening Event
ADVANCE OUTCOMES RESULTS 14 Subgroup Analyses Time to First Clinical Worsening Event Subgroup Ralinepag Placebo Hazard Ratio (95% CI) # of Events / # of Patients Overall 64/350 121/337 0.45( 0.33, 0.62) Baseline 6MWD <400 m 41/114 67/112 0.52( 0.35, 0.76) >=400 m 23/236 54/225 0.41( 0.25, 0.67) Etio
Slide 15: Subgroup Analyses Time to First Clinical Worsening Event
ADVANCE OUTCOMES RESULTS 15 Subgroup Analyses Time to First Clinical Worsening Event Subgroup Ralinepag Placebo Hazard Ratio (95% CI) # of Events / # of Patients Baseline WHO FC WHO FC II 32/261 56/223 0.46( 0.30, 0.72) WHO FC III or IV 32/89 65/114 0.50( 0.32, 0.77) PAH Therapy at Baseline None 2/8
Slide 16: Subgroup Analyses Time to First Clinical Worsening Event
ADVANCE OUTCOMES RESULTS 16 Subgroup Analyses Time to First Clinical Worsening Event Subgroup Ralinepag Placebo Hazard Ratio (95% CI) # of Events / # of Patients Baseline mean PAP <median (44 mmHg) 20/167 45/168 0.40( 0.23, 0.67) >=median (44 mmHg) 44/182 76/169 0.48( 0.33, 0.70) Baseline PVR <media
Slide 17: Summary of Most Commonly Reported Adverse Events by Preferred Term
17 ADVANCE OUTCOMES RESULTS Summary of Most Commonly Reported Adverse Events by Preferred Term Ralinepag Placebo N=350 N=337 Preferred Term n (%) n (%) Any Event 346 (98.9%) 320 (95.0%) Headache 284 (81.1%) 140 (41.5%) Diarrhea 204 (58.3%) 95 (28.2%) Nausea 158 (45.1%) 86 (25.5%) Myalgia 126 (36.0%)
Slide 18: ADVANCE OUTCOMES Headline Conclusions
18 ADVANCE OUTCOMES RESULTS ADVANCE OUTCOMES Headline Conclusions 1 2 3 Ralinepag reduced the The study also met Treatment with ralinepag risk of a clinical several important was well-tolerated, and the safety profile was worsening event versus secondary endpoints, consistent with known placebo by 5
Slide 19: Dr. Daniel Lachant, DO, MSCI, ATSF
19 Dr. Daniel Lachant, DO, MSCI, ATSF ASSOCIATE PROFESSOR OF MEDICINE, UNIVERSITY OF ROCHESTER
Slide 20: Dr. Martine Rothblatt
20 Dr. Martine Rothblatt CHAIRPERSON & CHIEF EXECUTIVE OFFICER
Slide 21: Q&A
21 Q&A Dr. Martine Rothblatt Chairperson and Chief Executive Officer Michael Benkowitz President and Chief Operating Officer Dr. Leigh Peterson EVP, Product Development and Xenotransplantation Dr. Derek Solum Senior Director, Product Development and Lead for the Global ADVANCE OUTCOMES Program Dr. D
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