united therapeutics corporation€¦ · 17/08/2020 · next milestones of a robust pipeline...
TRANSCRIPT
August 2020
UNITED THERAPEUTICS CORPORATION
2 AUGUST 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 AUGUST 2020
OUR FOCUS
DEVELOPING NOVEL, LIFE-EXTENDING TECHNOLOGIES FOR PATIENTS IN THREE CORE AREAS:
ONCOLOGY
1 2 3
LUNG DISEASE ORGAN MANUFACTURING
5 AUGUST 2020AUGUST 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
AUGUST 2020AUGUST 20206
A DECADE OF BALANCE AND VALUE CREATION, TAKING ON EARTHSHOTS TO ONE DAY FIND A CURE
7 AUGUST 2020
TREPROSTINIL REVENUE STABLE IN THE FACE OF GENERICS
Revenue as reported in SEC Forms 10K.
IN MILLIONS ORENITRAM® TYVASO® REMODULIN®
0
300
600
900
1200
1500
2006 2007 2008 2010 2011 2013 2014 2015 2016 2018 2019201720122009
8 AUGUST 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 AUGUST 2020
PAH PATIENTS
PH PATIENTS
CHRONIC FIBROSING ILD
PATIENTS
PH ILD PH COPD
CHRONIC FIBROSING ILD
(in U.S. as of 2019) (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. ~100,000 PATIENTS IN U.S.
PREVALENCE OF PH ILD & PH COPD IN PH WHO GROUP 3(2)
PREVALENCE OF CHRONIC FIBROSING ILD(2)
PREVALENCE OF PH(1) WHO GROUP 1(2)
CURRENT PAH LANDSCAPE NEW PH OPPORTUNITIES OPPORTUNITIES BEYOND PH
30,000 100,000
100,00017%
42%
6%
35%
FC I
FC II
FC III
FC IV
11 AUGUST 2020AUGUST 2020
3X THE NUMBER OF PATIENTS CURRENTLY ON OUR PH THERAPIES(1)
REMODULIN® Implantable System for Remodulin® (ISR), Remunity™, RemoPro™, Trevyent®
PH WHO Group 1, FC III & IV
TYVASO® Treprostinil Technosphere® (TreT), INCREASE, PERFECT, TETON
PH WHO Groups 1 & 3,Chronic Fibrosing ILD
ORENITRAM® M/M Reduction Label, QD Formulation PH 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 U.S. 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 AUGUST 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 PAH-related healthcare-costs were 67% higher for
selexipag patients than for Orenitram patients
DELAY DISEASE
PROGRESSION
IMPROVE KEY PROGNOSTIC
MEASURES OF RISK
INDICATE A POSITIVE IMPACT
ON SURVIVAL(1)
REDUCE PAH-ASSOCIATED
HEALTHCARE COSTS RELATIVE TO SELEXIPAG(2,3)
61% 37%
ORENITRAM® THERAPEUTIC PLATFORM
FREEDOM-EV and other studies 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). Difference in risk of death was not statistically significant at the end of the randomized treatment period or OLE. (2) Comparison of products does not imply clinical comparisons of safety or efficacy. (3) Dean BB, Saundankar V, Stafkey-Mailey D, et al. Medication Adherence and Healthcare Costs Among Patients with Pulmonary Arterial Hypertension Treated with Oral Prostacyclins: A Retrospective Cohort Study. Drugs – Real World Outcomes. 2020 Mar 6. Dean BB. Healthcare costs lower with treprostinil versus selexipag for PAH. PharmacoEcon Outcomes News. 2020;849:12.
UTHR PIPELINE
14 AUGUST 2020
NEXT MILESTONES OF A ROBUST PIPELINE
Remunity™ (treprostinil)
Tyvaso® Therapeutic Platform
UNDER FDA REVIEW LAUNCH PREPARATIONS UNDERWAY
PREPARING NDA RESUBMISSION
Remodulin® Therapeutic Platform
INCREASE (treprostinil)
21
Trevyent® (treprostinil sodium)
Remodulin® Therapeutic Platform
3
Implantable System for Remodulin® (1) (treprostinil) Remodulin® Therapeutic Platform
4
(1) 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.
TYVASO® THERAPEUTIC PLATFORM
15 AUGUST 2020AUGUST 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
HIT PRIMARY AND ALL SECONDARY ENDPOINTS; sNDA ACCEPTED; APRIL 2021 PDUFA DATE
TYVASO®(1) for PH ILD(2)
16
TYVASO® THERAPEUTIC PLATFORM
AUGUST 2020
INCREASE – ALL ENDPOINTS MET
(1) Treatment with Tyvaso of up to 12 breaths per session, four times daily, in the INCREASE study was well tolerated and the safety profile was consistent with previous Tyvaso studies in PAH and known prostacyclin-related adverse events.
IMPROVEMENT IN six-minute-walk-distance (6MWD).
Tyvaso increased 6MWD by 21 meters versus placebo (p=0.0043, Hodges-Lehmann estimate) after 16 weeks of treatment or by 31 meters when analyzed using Mixed Model Repeated Measurement (p<0.001).
» The cardiac biomarker NT-proBNP » Time to first clinical worsening event » Change in peak 6MWD at Week 12,
and change in trough 6MWD at week 15.
SIGNIFICANT IMPROVEMENTS IN:
Primary endpoint:
Secondary endpoints:
17 AUGUST 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)(1)
TREVYENT®(3,4)
REMUNITY™ REMOLIFE(3)
OF PAH PATIENTS REFUSE PARENTERAL THERAPY
FDA APPROVED
FDA CLEARED(2) LAUNCH PREP UNDERWAY
1
2
(1) 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. (2) FDA clearance of pharmacy-filled version. U.S. launch preparations underway. (3) Trevyent® and RemoLife are development-stage product not approved for sale in any jurisdiction. (4) Preparing NDA resubmission.
18 AUGUST 2020
NEXT GENERATION CLINICAL TRIALS AND TECHNOLOGIES
NCE/NOVEL BIOLOGICS(1)
ORGAN MANUFACTURING
INCREASE (treprostinil)
BREEZE Treprostinil
Technosphere® (treprostinil)
PERFECT (treprostinil)
TETON (treprostinil)
OreniPro (oral treprostinil
prodrug)
hu14.18K322A(humanized
dinutuximab)
Unexisome™ (exosomes)
ADVANCE Studies Ralinepag
(IP receptor agonist)SAPPHIRE
Aurora-GT™ eNOS Gene Therapy
GEM (Genetically-Enhanced
Mesenchymal stem cells)LNG01
(Wnt inhibitor)
Accelerated Total Artificial Lung (TAL1)
Centralized Ex-Vivo Lung Evaluation System (EVLP)
Unilobe (Lung Lobes)Unikidney (xenokidney)
Total Artificial Lung (TAL) Uniheart
(xenoheart) U-Lung
(3DAP Lungs)(1) NCE = New Chemical Entity.
Remunity™ (treprostinil) Trevyent®
(treprostinil sodium)
Implantable System for Remodulin®
(treprostinil)
RemoLife (treprostinil)RemoPro™
(treprostinil prodrug)
1 2 3 4 5 6
19 AUGUST 2020AUGUST 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 AUGUST 2020AUGUST 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 AUGUST 2020
152 60%70%15%
>6moPATIENTS LIVES
SAVED
LUNG FUNCTION
RECELLULARIZATION OF LUNG 3DAP PRINTED
LIFE SUPPORTING ORGANS IN
PRECLINICAL MODELS
EVLP XENO KIDNEY LUNG LOBES 3DAP LUNG
ORGAN MANUFACTURING THERAPEUTIC PLATFORM
ORGAN MANUFACTURING PROGRESS TO DATE
22 AUGUST 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 AUGUST 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)(1)
TREVYENT®(3,4)
REMUNITY™(2) REMOLIFE(3)
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
FDA APPROVED
FDA CLEARED(2) LAUNCH PREP UNDERWAY(1) 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. (2) FDA clearance of pharmacy-filled version. U.S. launch preparations underway. (3) Trevyent® and RemoLife are development-stage product not approved for sale in any jurisdiction. (4) Preparing NDA resubmission.