united therapeutics corporation

26
J.P. Morgan Healthcare Conference January 13, 2020 UNITED THERAPEUTICS CORPORATION

Upload: others

Post on 26-Feb-2022

2 views

Category:

Documents


0 download

TRANSCRIPT

J.P. Morgan Healthcare ConferenceJanuary 13, 2020

UNITED THERAPEUTICS CORPORATION

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

UTHR OVERVIEW

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

UTHR MARKETS

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).

UTHR PIPELINE

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

APPENDIX

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