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UNITED THERAPEUTICS CORPORATION / AS OF APRIL 29, 20201
UNITED THERAPEUTICS CORPORATIONA Balanced, Value-creating Biotechnology Model with 6 Therapeutic Platforms
Dinutuximab
61 2 543
Organ Manufacturing
NCE/Novel Biologics(1)
(1) NCE = New Chemical Entity.
UNITED THERAPEUTICS CORPORATION / AS OF APRIL 29, 20202
REMODULIN® THERAPEUTIC PLATFORMNEXT-GENeration Enabling Technologies for Remodulin®
IMPLANTABLE SYSTEM FOR
REMODULIN® (ISR)(4)
New & Innovative SMART PUMP TECHNOLOGY
The world’s first pre-filled, pre-programmed infusion system containing treprostinil
Prodrug of treprostinil that is being developed to reduce site pain associated with subcutaneous delivery of treprostinil
TREVYENT®(2,3)REMUNITY™ REMOPRO™ (2)
FDA CLEARED(1) FDA APPROVED
Remodulin® subcutaneous pump refills: SIMPLIFIED
(1) FDA clearance of pharmacy-filled version. U.S. launch targeted July 2020. (2) Trevyent® and RemoPro™ are development stage products not approved for sale in any jurisdiction. (3) Evaluating FDA complete response letter to NDA. (4) FDA requires that certain conditions of Medtronic’s PMA approval of the Implantable System for Remodulin must be satisfied prior to launch or sale of the Implantable System for Remodulin; accordingly, Implantable System for Remodulin labeling may be revised in the process of satisfying such conditions of approval.
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UNITED THERAPEUTICS CORPORATION / AS OF APRIL 29, 20203
TYVASO® THERAPEUTIC PLATFORMNEXT-GENeration Enabling Clinical Trials and Technologies for Tyvaso®
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INCREASEPhase III clinical trial in PH WHO Group 3
TYVASO® for PH ILD(1,2)
(1) Tyvaso® is not approved for PH WHO Group 3 patients. (2) PH ILD = Pulmonary Hypertension associated Interstitial Lung Disease. (3) Tyvaso increased six-minute walk distance by 21 meters versus placebo (p=0.0043, Hodges-Lehmann estimate) after 16 weeks of treatment. Additionally, INCREASE met all secondary endpoints (i.e., Change in NT-proBNP from baseline to week 16; Time to first clinical worsening event; Change in peak 6MWD from baseline to week 12; Change in trough 6MWD from baseline to week 15); FDA label supplement to be filed mid-2020. (4) Treprostinil Technosphere® is a development-stage product not approved for sale in any jurisdiction. (5) PH COPD = Pulmonary Hypertension associated with Chronic Obstructive Pulmonary Disease.
PERFECT Phase III clinical trial in PH WHO Group 3
TYVASO® for PH COPD(1,5)
TREPROSTINIL TECHNOSPHERE® (4)
Oral inhalation technology in PH WHO Group 1
ALL ENDPOINTS MET (3)
UNITED THERAPEUTICS CORPORATION / AS OF APRIL 29, 20204
ORENITRAM® THERAPEUTIC PLATFORM
Oral prodrug being developed to provide increased tolerability and convenience through a once-daily dosing regimen
ORENIPRO™ (1)
ONCEDAILY
NEXT-GENeration Enabling Clinical Trials for Orenitram®
(1) OreniPro™ is a developmental stage product not approved for sale in any jurisdiction.
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UNITED THERAPEUTICS CORPORATION / AS OF APRIL 29, 20205
UNEXISOME™(1)
Phase I asset for the treatment of BPD(3)
A novel biologic for the treatment of BPD
Therapeutic Platform
SAPPHIRE(1)
Phase III gene therapy clinical trial in PH WHO Group 1
Rebuilding lung blood vessels destroyed by PAH
(1) Unexisome™, Ralinepag, the Sapphire gene therapy product, LNG01, and GEMS are development-stage products not approved for sale in any jurisdiction. (2) Program formerly referred to SM04646. (3) BPD = Bronchopulmonary Dysplasia. (4) IPF = Idiopathic Pulmonary Fibrosis.
NEW CHEMICAL ENTITIES (NCE) & NOVEL BIOLOGICS
RALINEPAG(1)
Phase III clinical trials in PH WHO Group 1
A small molecule WNT inhibitor to treat IPF
Genetically-Engineered Mesenchymal stem cells (MSCs) for the treatment of sepsis and PAH
LNG01(1)(2)
Phase I asset in IPF(4)
Next-generation, oral, selective and potent prostacyclin receptor agonist
GEMS(1)
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O
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O
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UNITED THERAPEUTICS CORPORATION / AS OF APRIL 29, 20206
DINUTUXIMAB THERAPEUTIC PLATFORM
UNITUXIN®
Potential label expansion for pediatric patients with relapsed or refractory neuroblastoma(1)
HUMANIZED DINUTUXIMAB
Humanized dinutuximab for high GD2 expression tumors(2)
NEXT-GENeration Dinutuximab Programs
(1) Unituxin is not approved for pediatric patients with relapsed or refractory neuroblastoma. (2) Humanized dinutuximab is not an approved product in any jurisdiction.
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UNITED THERAPEUTICS CORPORATION / AS OF APRIL 29, 20207
» EX-VIVO Lung Perfusion (EVLP)
» XENO Transplantation
» 3D Organ Scaffold Printing
» Regenerative Medicine
~1.0 MILLION PEOPLE IN THE U.S. who have end-stage organ disease and may
need a heart, kidney or lung transplant
ORGAN MANUFACTURING THERAPEUTIC PLATFORMTransform the Marketplace to Make More Organs Available for Transplant
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