United Therapeutics Corporation · NASDAQ:UTHR
United Therapeutics — J.P. Morgan Healthcare Conference 2024
33 slides from United Therapeutics's conference presentation, Jan 8, 2024
Slide 1: United Therapeutics: Enabling Inspiration
United Therapeutics: Enabling Inspiration J.P. MORGAN HEALTHCARE CONFERENCE JANUARY 8, 2024
Slide 2: Safe Harbor Statement
2 INTRODUCTION Safe Harbor Statement All statements in this presentation are made as of January 8, 2024. 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: Our Focus
3 HOW WE OPERATE Our Focus Rare Diseases Organ Manufacturing • Pulmonary Hypertension • Ex-Vivo Lung Perfusion • Pulmonary Fibrosis • Xenotransplantation • Neuroblastoma • Regenerative Medicine • 3-D Organ Printing
Slide 4: Decades of Innovation and Revenue Growth
4 HOW WE OPERATE Decades of Innovation and Revenue Growth Six Approved Products >20% Annual Revenue CAGR4 Covering Three Indications Since First Commercial Product Launch millions USD 2,000 1,800 1,600 1,400 1,200 1,000 800 600 400 200 PAH1 PH-ILD2 NB3 0 02 03 04 05 06 07 08 09 10 11 12 13 14 15 16
Slide 5: Positioned for Multiple Waves of Growth
5 HOW WE OPERATE Positioned for Multiple Waves of Growth1 Revenue $16B Revolution Organ Manufacturing $8B Innovation Tyvaso® IPF/PPF & Ralinepag PAH $4B Foundation Current Commercial Portfolio $2B Today Future 1. Illustrative purposes only. Time UTHR: A Public Benefit Corporation
Slide 6: Foundation: Our Commercial Portfolio
Foundation: Our Commercial Portfolio
Slide 7: Solid Foundation Addressing Rare Disease
7 FOUNDATION Solid Foundation Addressing Rare Disease The most prescribed parenteral The most prescribed antibody prostacyclin in the U.S. therapy for high-risk neuroblastoma in the U.S.
Slide 8: Solid Foundation Addressing Rare Disease
8 FOUNDATION Solid Foundation Addressing Rare Disease The only prostacyclin with the The most prescribed ability to transition from infused prostacyclin in the U.S. therapies and maintain infused therapy dose exposure
Slide 9: Tyvaso DPI: Early, Easy, Effective
9 FOUNDATION Tyvaso DPI®: Early, Easy, Effective
Slide 10: Tyvaso DPI: Early, Easy, Effective
10 FOUNDATION Tyvaso DPI®: Early, Easy, Effective An option for Easy-to-use Proven efficacy and appropriate patients prostacyclin delivery1,2 safety across multiple identified early1,3,5 clinical trials1,2,3,4 1. TYVASO DPI [package insert]. Research Triangle Park, NC: United Therapeutics Corporatio
Slide 11: Recent Manuscript Ties Low Flow to Deep Lung Deposition
11 FOUNDATION Recent Manuscript1 Ties Low Flow to Deep Lung Deposition 1. Colleen McEvoy, et al. Tyvaso DPI: Drug-device characteristics and patient clinical considerations, Pulmonary Pharmacology & Therapeutics, Volume 83, 2023, 102266, ISSN 1094-5539, https://doi.org/10.1016/j.pupt.2023.102266.
Slide 12: Recent Manuscript Ties Low Flow to Deep Lung Deposition
12 FOUNDATION Recent Manuscript1 Ties Low Flow to Deep Lung Deposition While many have incorrect beliefs about high-resistance devices, research and real-world clinical evidence have shown that patients with pulmonary disease, including PH, can generate the inspiratory force needed to properly use t
Slide 13: Recent Manuscript Ties Low Flow to Deep Lung Deposition
13 FOUNDATION Recent Manuscript1 Ties Low Flow to Deep Lung Deposition The minimum [peak inspiratory pressure] needed to create adequate powder dispersion to empty the Tyvaso DPI cartridge is 2 kPa for ≥2 seconds, resulting in complete dose delivery from the device.5,6,7 5. MannKind Inhalation Techn
Slide 14: Recent Manuscript Ties Low Flow to Deep Lung Deposition
14 FOUNDATION Recent Manuscript1 Ties Low Flow to Deep Lung Deposition Low inhalation pressures generated by the user … are efficient to induce low-flow rates … through the DPI.8 8. United Therapeutics Corporation, Treprostinil Inhalation Powder Investigator’s Brochure, 2 ed., 2020.
Slide 15: Tyvaso DPI is Low Flow
15 Tyvaso DPI® is LOW FLOW low flow Drug Low flow, coupled with the FDKP carrier molecule, allows more drug deposition in the smaller airways of the lungs rather than the throat Colleen McEvoy, et al. Tyvaso DPI: Drug-device characteristics and patient clinical considerations, Pulmonary Pharmacology
Slide 16: Other DPI Devices are High Flow
16 Other DPI devices are high flow Drug HIGH FLOW High flow devices cause more particles to deposit in the throat, decreasing the amount of drug distributed to the smallest airways Colleen McEvoy, et al. Tyvaso DPI: Drug-device Drug concentration characteristics and patient clinical considerations,
Slide 17: Low Flow of Tyvaso DPI Leads to Efficient Treprostinil Delivery
17 LOW FLOW of Tyvaso DPI® leads to efficient treprostinil delivery low flow 21.2 mg powder 6.4 mg powder Drug with 106 µg Yutrepia with 64 µg Tyvaso DPI® high flow Drug ~3.3x MORE YUTREPIA POWDER REQUIRED FOR A SIMILAR TREPROSTINIL EXPOSURE Drug concentration low high 1. Tyvaso DPI label, section 2
Slide 18: Tyvaso DPI: The First and Only Low Flow DPI
18 Tyvaso DPI®: The first and only Low flow, High flow, DPI tailored for like Tyvaso DPI Drug like other Drug devices drug delivery to the distal lungs for PH Colleen McEvoy, et al. Tyvaso DPI: Drug-device Drug concentration characteristics and patient clinical considerations, Pulmonary Pharmacology
Slide 19: Tyvaso DPI: First and Only DPI Tailored for Distal Lung Delivery
19 Tyvaso DPI®: The first and only DPI tailored for drug delivery to the distal lungs for PH1 ONE BREATH per cartridge2 DEEP LUNG delivery1 NO CLEANING required3 ROOM TEMP storage5 NO MAX labeled dose4 HIGH PATIENT satisfaction6 1. Colleen McEvoy, et al. Tyvaso DPI: Drug-device characteristics and p
Slide 20: Innovation: Our Near-Term Pipeline
Innovation: Our Near-Term Pipeline
Slide 21: Development Engine Addressing Unmet Needs
21 01 INNOVATION Development Engine Addressing Unmet Needs NON-REGISTRATION REGISTRATION APPROVED Tyvaso® TETON 1 – Idiopathic Pulmonary Fibrosis – U.S. and Canada TETON 2 – Idiopathic Pulmonary Fibrosis – ROW1 TETON PPF – Progressive Pulmonary Fibrosis Ralinepag ADVANCE OUTCOMES – PAH2 Organ Manufa
Slide 22: Tyvaso TETON Program
22 INNOVATION Tyvaso® TETON program TETON 1 TETON 2 TETON PPF Progressive Indication Idiopathic pulmonary fibrosis Pulmonary Fibrosis Complete U.S. Addressable Population 100,000 patients 60,0006,7,8 Enrollment for Study Size 576 576 698 TETON 1 and 2 Targeted Study Geography U.S./Canada ROW1 Worldw
Slide 23: Ralinepag ADVANCE OUTCOMES Study
23 INNOVATION Ralinepag ADVANCE OUTCOMES Study Indication Group 1 PAH1 Trial Completion U.S. Addressable Population 50,000 patients Targeted in 20253 DSMC4 recommended continuation Study Size ~700 to 1,000 patients of both studies after an unblinded safety review of 510 patients Study Geography Glob
Slide 24: Revolution: Our Organ Manufacturing Efforts
Revolution: Our Organ Manufacturing Efforts
Slide 25: Four Platforms with Four Organs; Multiple Shots on Goal
REVOLUTION 25 Four Platforms with Four Organs; Multiple Shots on Goal 1. 3DAP = 3D autologous printed. 2. Bio-Art = Bioartificial Xeno Regen Med 3DAP1 Bio-Art2 Kidney UKidney™ IVIVA Kidney UThymoKidney™ 3DAP Kidney Mirokidney® Heart UHeart™ Lung ULobe™ ULung™ MiroliverELAP®/ Liver 3DAP Liver Miroliv
Slide 26: Xenotransplants March Toward the Clinic
26 REVOLUTION Xenotransplants March Toward the Clinic Pre-clinical progress • ”Pivotal” pre-clinical studies underway • Last non-clinical xenokidney transplant expected by mid-2024 FPO • FDA meeting to discuss IND and clinical protocol for 10-gene xeno-organs HOLD FOR AERIAL PHOTO Designated Pathoge
Slide 27: Xenokidneys Represent a Multi-Billion-Dollar Opportunity
27 REVOLUTION Xenokidneys Represent a 90,000 patients Multi-Billion-Dollar Opportunity PREVALENCE IN THE U.S. 450,000 patients U.S. KIDNEY TRANSPLANT WAIT LIST ~90k patients1 PATIENTS ON DIALYSIS NOT ON THE WAIT LIST ~450k patients2 1. Organ Procurement and Transplantation Network (OPTN) data as of
Slide 28: Xenokidneys Represent a Multi-Billion-Dollar Opportunity
28 REVOLUTION 25,000 patients Xenokidneys Represent a Multi-Billion-Dollar Opportunity INCIDENCE IN THE U.S. 100,000 patients ANNUAL KIDNEY TRANSPLANTS ~25k patients1 ANNUAL PATIENTS STARTING DIALYSIS NOT ON WAIT LIST ~100k patients2 1. Organ Procurement and Transplantation Network (OPTN) data for F
Slide 29: Xenokidneys Represent a Multi-Billion-Dollar Opportunity
29 REVOLUTION $30 billion Xenokidneys Represent a Multi-Billion-Dollar Opportunity REVENUE OPPORTUNITY IN THE U.S. FULLY ADDRESS FUTURE ANNUAL NEED $30 billion to $90 billion $51 billion ANNUAL MEDICARE SPEND FOR KIDNEY CARE $51 billion in 20201 1. The United States Renal Data System 2022 Annual Dat
Slide 30: Xenokidneys Represent a Multi-Billion-Dollar Opportunity
30 REVOLUTION Xenokidneys Represent a $900 million Multi-Billion-Dollar Opportunity ANNUAL REVENUE PER DPF1 FACILITY 1,000 organ throughput $1.8 billion $300 million to $900 million 2,000 organ throughput $600 million to $1.8 billion $2.7 billion 3,000 organ throughput $900 million to $2.7 billion 1
Slide 31: UTHR: Positioned for Multiple Waves of Growth
31 REVOLUTION UTHR: Positioned for Multiple Waves of Growth1 Revenue $16B Revolution Organ Manufacturing $8B Innovation Tyvaso® IPF/PPF & Ralinepag PAH $4B Foundation Current Commercial Portfolio $2B Today Future 1. Illustrative purposes only. Time UTHR: A Public Benefit Corporation
Slide 32: Q&A
32 Q&A Dr. Martine Rothblatt Chairperson & Chief Executive Officer Michael Benkowitz President & Chief Operating Officer FPO – clean up image James Edgemond Chief Financial Officer & Treasurer UTHR: A Public Benefit Corporation
Slide 33: Therapeutics
5) o = < =) Therapeutics
United Therapeutics — J.P. Morgan Healthcare Conference 2024 on 6ix