united therapeutics corporation
TRANSCRIPT
2 JPM 2020
Remarks today concerning United Therapeutics may include forward-looking statements which represent United Therapeutics’ expectations or beliefs regarding future events. We caution that such statements involve risks and uncertainties that may cause actual results to differ materially from those in the forward-looking statements. Consequently, all such forward-looking statements are qualified by the cautionary language and risk factors set forth in United Therapeutics’ periodic and other reports filed with the SEC.
There can be no assurance that the actual results, events or developments referenced in such forward-looking statements will occur or be realized. United Therapeutics assumes no obligation to update these forward-looking statements to reflect actual results, changes in assumptions or changes in factors affecting such forward-looking statements.
The discussions during this presentation could include certain financial measures that were not prepared in accordance with U.S. Generally Accepted Accounting Principles (GAAP). Reconciliations of those non-GAAP financial measures to the most directly comparable U.S. GAAP financial measures can be found in our earnings releases filed with the SEC in Current Reports on Form 8-K for the relevant time period. These reports are available on our website at www.unither.com in the “Investor Relations Financial Information SEC Filings” section.
This presentation and any related discussions or statements are intended to educate investors about our company. Sometimes that process includes reporting on the progress and results of clinical trials or other developments with respect to our products. This presentation and any related discussions or statements are not intended to promote our products, to suggest that our products are safe and effective for any use other than what is consistent with their FDA-approved labeling, or to provide all available information regarding the products, their risks, or related clinical trial results. Anyone seeking information regarding the use of one of our products should consult the full prescribing information for the product available on our website at www.unither.com.
ORENITRAM®, PATCHPUMP®, REMODULIN®, TREVYENT®, TYVASO®, and UNITUXIN® are registered trademarks of United Therapeutics Corporation and its subsidiaries.Implantable System for Remodulin® (ISR), REMUNITY™, REMOPRO™, and UNEXISOME™ are trademarks of United Therapeutics Corporation and its subsidiaries. ADCIRCA® is a registered trademark of Eli Lilly and Company.TECHNOSPHERE® is a registered trademark of MannKind Corporation.
SAFE HARBOR STATEMENT
4 JPM 2020
OUR FOCUS
DEVELOPING NOVEL, LIFE-EXTENDING TECHNOLOGIES FOR PATIENTS IN THREE CORE AREAS:
ONCOLOGY
1 2 3
LUNG DISEASE ORGAN MANUFACTURING
5 JPM 2020
MULTIPLE APPROVED THERAPIES: 20+ YEARS OF ADDRESSING UNMET MEDICAL NEEDS
INHALATIONPAH is a rare, progressive disorder characterized by high blood pressure (hypertension) in the arteries of the lungs (pulmonary arteries) for no known cause. NB is a rare solid tumor cancer that arises in immature nerve cells and strikes primarily infants and children. Commercial products also include Adcirca® for the treatment of PAH, which went generic in August 2018.
PULMONARY ARTERIAL HYPERTENSION (PAH)
TABLETINFUSION
NEUROBLASTOMA (NB)
INFUSION
JPM 20206
A DECADE OF BALANCE AND VALUE CREATION, TAKING ON EARTHSHOTS TO ONE DAY FIND A CURE
Therapeutic Platform Indication Status Oral Inhaled Infused Transplant Device Complete
Pipeline News
Implantable System for Remodulin®(treprostinil)
RemUnity™(treprostinil)
Trevyent® (treprostinil sodium)
DISTINCT(dinutuximab)
INCREASE(inhaled treprostinil)
PERFECT(inhaled treprostinil)
BREEZETreprostinil Technosphere® (inhaled treprostinil)
RemoPro™(treprostinil prodrug)
UT-30(oral treprostinil prodrug)
SAPPHIREAurora-GT™ eNOS Gene Therapy
RemoLife(treprostinil)
LB-3,4Ex Vivo Lung Perfusion (EVLP)
Unikidney(xenokidney)
Uniheart(xenoheart) Total
Artificial Lung(TAL)
U-Lung(3DAP Lungs)
ADVANCE StudiesRalinepag(IP receptor agonist)
SM04646(Wnt inhibitor) GEMS
(Genetically-Enhanced Mesenchymal stem cells)
hu14.18K322A (humanized dinutuximab)
Unexisome™(exosomes) Unilobe
(Lung Lobes)
7 JPM 2020
TOTAL TREPROSTINIL REVENUE CONTINUES TO GROW
Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q32006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018 2019
Revenue as reported in SEC Forms 10Q and 10K. (1) Compounded Annual Growth Rate (CAGR) calculated over the period from January 1, 2006 through December 31, 2018.
$350 $350
$300 $300
$250 $250
$200 $200
$150 $150
$100 $100
$50 $50
$0 $0
TYVASO®
Revenue CAGR > 18%(1) ORENITRAM®
REMODULIN®
IN MILLIONS
8 JPM 2020
FOUNDATION FOR A GROWING REMODULIN® BRAND
SAFETY & SUPPLY CHAIN RELIABILITY
HIGHLY DIFFERENTIATED PRODUCT PLATFORM
PATIENT SUPPORT LIMITED HISTORY OF SWITCHING IN THE PAH SPACE
WE BELIEVE REMODULIN® REVENUES
WILL CONTINUE TO GROW IN FACE
OF GENERICS
10 JPM 2020
6%
35%
42%
17%FC IV
FC III
FC II
FC I
PAH PATIENTS
PH PATIENTSPH ILD PH COPD
(in U.S. as of 2019)
(in U.S. as of 2019)
(1) PH = Pulmonary Hypertension. (2) Estimated patient populations based on United Therapeutics internal market research.
MARKET OPPORTUNITY
>45,000 PATIENTS IN U.S. >130,000 PATIENTS IN U.S.
PREVALENCE OF PH ILD & PH COPD IN PH WHO GROUP 3(2)
PREVALENCE OF PH(1) WHO GROUP 1(2)
30,000 100,000
17%
42%
6%
35%
11 JPM 2020
3X THE NUMBER OF PATIENTS CURRENTLY ON OUR PH THERAPIES(1)
REMODULIN® Implantable System for Remodulin® (ISR), RemUnity™, RemoPro™, Trevyent® WHO Group 1, FC III & IV
TYVASO® Treprostinil Technosphere® (TreT), INCREASE, PERFECT WHO Groups 1 & 3
ORENITRAM® M/M Reduction Label, QD Formulation WHO Group 1
(1) Does not factor in potential patient growth in WHO Group 1 from Ralinepag and SAPPHIRE. (2) Represents an approximate total number of patients currently on United Therapeutics treprostinil therapies, including Remodulin, Tyvaso, and Orenitram.
INNOVATE GROW EXPAND
7,500(2) 25,000Current patients on therapy
5 YEAR EXPANSION GOAL
0 5,000 10,000 15,000 20,000 25,000
12 JPM 2020
With a 61% reduction in risk Including NT-proBNP and functional class (FC),
as well as overall risk status
With a 37% reduction in risk of death vs placebo
at study closure
DELAY DISEASE
PROGRESSION
IMPROVE KEY PROGNOSTIC
MEASURES OF RISK
INDICATE A POSITIVE IMPACT
ON SURVIVAL(1)
61% 37%
ORENITRAM® THERAPEUTIC PLATFORM
FREEDOM-EV showed that Orenitram can
KEY BENEFITS OF EXPANDED ORENITRAM® LABEL
(1) Participants for which data are available (89%), Orenitram® was associated with a 37% decreased risk of mortality compared with placebo (p=0.0324) at study closure (which includes additional data accrued in the open-label extension study).
14 JPM 2020
FIRST FIVE MILESTONES OF A ROBUST PIPELINE
Trevyent® (treprostinil sodium)
RemUnity™ (treprostinil)
Implantable System for Remodulin® (treprostinil)
DISTINCT (dinutuximab)
Dinutuximab Therapeutic Platform
Tyvaso® Therapeutic Platform
Remodulin® Therapeutic Platform
Remodulin® Therapeutic Platform
Remodulin® Therapeutic Platform
INCREASE (treprostinil)
3 4 51 2
15 JPM 2020
DINUTUXIMAB THERAPEUTIC PLATFORM
Antibody signals immune cells to come
GD2
Cancerous Cell
Nerve CellUnituxin Antibody White Blood Cell
Estimated U.S. SCLC incidence ~30,000(2) people
(1) SCLC = Small Cell Lung Cancer. (2) Estimated patient populations based on United Therapeutics internal market research.
DISTINCT PHASE III CLINICAL TRIAL FOR SCLC(1)
100% ENROLLED | EXPECTED READOUT 1Q20
TYVASO® THERAPEUTIC PLATFORM
16 JPM 2020
INCREASE PHASE III CLINICAL TRIAL IN PH WHO GROUP 3
(1) Tyvaso® is not approved for PH WHO Group 3 patients. (2) PH ILD = Pulmonary Hypertension associated with Interstitial Lung Disease. (3) Estimated patient populations based on United Therapeutics internal market research. (4) V = Poor Lung Ventilation; Q = Low Lung Perfusion. (5) Rubin LJ. New Engl J Med. 1997;336(2):111-117. Rubin LJ. Chest. 1993;104:236-250. Seeger W, et al. J Am Coll Cardiol. 2013;62 (25 Suppl):D109-116.e.
» No approved therapies for WHO Group 3
» Inhaled therapies may preserve V/Q(4) and prevent undesirable effects of perfusion(5)
Estimated U.S. PH-ILD prevalence ~30,000(3) people
100% ENROLLED | EXPECTED READOUT 1Q20
TYVASO®(1) for PH ILD(2)
17 JPM 2020
REMODULIN® THERAPEUTIC PLATFORM
BECAUSE OF CONCERNS
AROUND IV USE OR SUBQ PAIN
PAH PATIENTS
30-40% 3
4
A PUMP FOR EVERY PAH PATIENT
IMPLANTABLE SYSTEM FOR REMODULIN®
(ISR)
TREVYENT®(1)
REMUNITY™ REMOLIFE(1)
OF PAH PATIENTS REFUSE PARENTERAL THERAPY
(1) Trevyent® and RemoLife are development-stage product not approved for sale in any jurisdiction.
FDA APPROVED
FDA CLEARED
1
2
18 JPM 2020
NEXT GENERATION CLINICAL TRIALS AND TECHNOLOGIES
NCE/NOVEL BIOLOGICS(1)
ORGAN MANUFACTURING
INCREASE (treprostinil)
PERFECT (treprostinil)
BREEZE Treprostinil
Technosphere® (treprostinil)
UT-30 (oral treprostinil prodrug)
DISTINCT (dinutuximab)
hu14.18K322A (humanized dinutuximab)
SAPPHIRE Aurora-GT™ eNOS
Gene Therapy ADVANCE Studies
Ralinepag (IP receptor agonist)
SM04646 (Wnt inhibitor)Unexisome™
(exosomes)GEMS
(Genetically-Enhanced Mesenchymal stem cells)
Unilobe (Lung Lobes)
LB-3,4 Ex Vivio Lung Perfusion
(EVLP) Unikidney
(xenokidney) Uniheart
(xenoheart) Total Artificial Lung
(TAL) U-Lung
(3DAP Lungs)(1) NCE = New Chemical Entity.
Implantable System for Remodulin®
(treprostinil) RemUnity™ (treprostinil) Trevyent®
(treprostinil sodium)RemoLife
(treprostinil)RemoPro™
(treprostinil prodrug)
1 2 3 4 5 6
19 JPM 2020
NCE & NOVEL BIOLOGICS THERAPEUTIC PLATFORM
(1) Ralinepag is development-stage product not approved for sale in any jurisdiction.
» New drug candidate for PAH
» Phase III clinical trials enrolling for Ralinepag (IP receptor agonist),
» Evaluating impact on morbidity/mortality (ADVANCE OUTCOMES) and exercise capacity (ADVANCE CAPACITY)
O
O
a
O
OHTargeting the prostacyclin pathway
RALINEPAG(1) POTENTIAL ‘BEST-IN-CLASS’ THERAPY FOR PAH
20 JPM 2020
ORGAN MANUFACTURING THERAPEUTIC PLATFORM
TRANSFORM THE MARKETPLACE TO MAKE MORE ORGANS AVAILABLE FOR TRANSPLANT
» EVLP | Ex Vivo Lung Perfusion, also applicable to other organs
» XENO | Genetically modified pathogen-free pig organs
» LUNG LOBES | Regenerated allogenic human cellularized lung lobes on decellularized porcine scaffold
» 3DAP | 3D Autologous Printed lung using re-differentiated iPS cells from patient on 3D printed scaffold
21 JPM 2020
116 60%70%15%
>6moPATIENTS
LIVES SAVED
LUNG FUNCTION
RECELLULARIZATION OF LUNG 3DAP PRINTED
LIFESUPPORTING
ORGANS
EVLP XENO KIDNEY LUNG LOBES 3DAP LUNG
ORGAN MANUFACTURING THERAPEUTIC PLATFORM
ORGAN MANUFACTURING PROGRESS TO DATE
22 JPM 2020
ORGAN MANUFACTURING THERAPEUTIC PLATFORM
ORGAN MANUFACTURING AT UTHR
SCAL
E-U
P &
LOG
ISTI
CS C
HAL
LEN
GE
TECHNOLOGY DEVELOPMENT CHALLENGE
IMM
UN
OLOG
ICAL
CH
ALLE
NGE
3DAPFIH– LONG-TERM GOAL
XENO KIDNEYFirst In Human
(FIH) – SHORT-TERM GOAL
LUNG LOBESFIH – MID-TERM GOAL
EVLPCurrently in
the clinic
Orga
n Man
ufacturing
25 JPM 2020
REMODULIN® THERAPEUTIC PLATFORM
BECAUSE OF CONCERNS
AROUND IV USE OR SUBQ PAIN
PAH PATIENTS
30-40%
A PUMP FOR EVERY PAH PATIENT
IMPLANTABLE SYSTEM FOR REMODULIN® (ISR)
TREVYENT®(1)
REMUNITY™ REMOLIFE(2)
OF PAH PATIENTS REFUSE PARENTERAL THERAPY » Ease of use and reduced risk of
bloodstream infections (BSIs) » Once-monthly refills or longer
» First pre-filled, pre-programmed infusion system
» Simplification for subQ via disposable Patch Pump® technology
» Small, lightweight, durable subQ pump with disposable cartridges
» Flexible dosing via an acoustic volume sensing technology
» Next-generation ambulatory infusion pump with smartphone compatibility
(1) Trevyent® is a development-stage product not approved for sale in any jurisdiction. (2) RemoLife is a development-stage product not approved for sale in any jurisdiction.
FDA APPROVED
FDA CLEARED