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1. Given the critical importance of the TETON trials for Tyvaso in pulmonary fibrosis, can you explain whether both ongoing studies need to succeed for FDA approval, or if one positive study could be sufficient?
2. With the entrance of competitors like Merck into the PAH market, have you observed any changes in patient referrals or market dynamics that might affect your growth projections?
3. Regarding capital allocation, how do you assess the potential for additional share buybacks or accelerated share repurchases in light of your investments in the xenotransplantation program?
4. Can you elaborate on the drivers behind Tyvaso DPI's performance compared to Nebulized Tyvaso this quarter, and what factors are influencing patient and physician preference?
5. While you've expressed confidence in the sustainability of your foundational products like Remodulin, Tyvaso, and Orenitram, what potential risks do you see that could impact their vitality, and how are you addressing them?

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United States

Rest of World (ROW)

Total Revenue

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United Therapeutics Corporation (UTHR) is a biotechnology company dedicated to developing and commercializing innovative products for chronic and life-threatening conditions. The company focuses on addressing unmet medical needs through pharmaceutical therapies and technologies, particularly in the areas of pulmonary arterial hypertension (PAH) and oncology. UTHR also explores organ manufacturing technologies to expand the availability of transplantable organs.

1. **Tyvaso DPI** - Provides inhalation powder therapy for pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease  **[0001082554-24-000047_uthr-20240930.htm:18]** **[0001082554-24-000005_uthr-20231231.htm:3]** **[0001082554-24-000025_uthr-20240331.htm:4]** **[0001082554-24-000017_uthr-20240429.htm:5]** .
2. **Nebulized Tyvaso** - Offers inhalation solution for treating pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease  **[0001082554-24-000047_uthr-20240930.htm:18]** **[0001082554-24-000005_uthr-20231231.htm:3]** **[0001082554-24-000025_uthr-20240331.htm:4]** **[0001082554-24-000017_uthr-20240429.htm:5]** .
3. **Remodulin** - Delivers treprostinil injection for pulmonary arterial hypertension management  **[0001082554-24-000047_uthr-20240930.htm:18]** **[0001082554-24-000005_uthr-20231231.htm:3]** **[0001082554-24-000025_uthr-20240331.htm:4]** **[0001082554-24-000017_uthr-20240429.htm:5]** .
4. **Orenitram** - Supplies extended-release tablets for pulmonary arterial hypertension treatment  **[0001082554-24-000047_uthr-20240930.htm:18]** **[0001082554-24-000005_uthr-20231231.htm:3]** **[0001082554-24-000025_uthr-20240331.htm:4]** **[0001082554-24-000017_uthr-20240429.htm:5]** .
5. **Unituxin** - Provides dinutuximab injection for high-risk neuroblastoma in pediatric patients  **[0001082554-24-000047_uthr-20240930.htm:18]** **[0001082554-24-000005_uthr-20231231.htm:3]** **[0001082554-24-000025_uthr-20240331.htm:4]** .
6. **Adcirca** - Offers tadalafil tablets for pulmonary arterial hypertension  **[0001082554-24-000047_uthr-20240930.htm:18]** **[0001082554-24-000005_uthr-20231231.htm:3]** **[0001082554-24-000025_uthr-20240331.htm:4]** **[0001082554-24-000017_uthr-20240429.htm:5]** .
7. **Other** - Includes various other products contributing to the company's therapeutic offerings  **[0001082554-24-000047_uthr-20240930.htm:18]** .

a770f0cac9505c2a5581d99e913b46f9742ef409912e49e1b7447d06be8be2d8

| Name                  | Position                                                      | Start Date          | Short Bio                                                                                                                                                                                                 |
|-----------------------|---------------------------------------------------------------|---------------------|----------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------|
| Martine Rothblatt     | Chairperson and Chief Executive Officer                       | 1996                | **Martine Rothblatt, Ph.D., J.D., M.B.A.** is the Founder, Chairperson, and CEO of United Therapeutics Corporation. She founded the company in **1996** and has served in her current roles since its inception  **[0001082554-24-000005_uthr-20231231.htm:72]** **[0001082554-24-000017_uthr-20240429.htm:47]** **[0001082554-24-000017_uthr-20240429.htm:32]** .                     |
| Michael Benkowitz     | President and Chief Operating Officer                         | 2016                | **Michael Benkowitz** joined United Therapeutics in **2011** as Executive Vice President, Organizational Development, and was promoted to President and COO in **2016**  **[0001104659-23-116505_tm2329335d4_exh1a.htm:85]** **[0001082554-24-000005_uthr-20231231.htm:72]** **[0001104659-23-116505_tm2329335d4_exh1a.htm:86]** .                                                              |
| James C. Edgemond     | Chief Financial Officer and Treasurer                         | March 2015          | **James C. Edgemond** joined United Therapeutics in January **2013** as Treasurer and Vice President, Strategic Financial Planning, and was promoted to CFO and Treasurer in March **2015**  **[0001082554-24-000017_uthr-20240429.htm:47]** **[0001082554-24-000005_uthr-20231231.htm:72]** **[0001104659-23-116505_tm2329335d4_exh1a.htm:85]** .                                          |
| Paul A. Mahon         | Executive Vice President, General Counsel, and Corporate Secretary | 2003                | **Paul A. Mahon, J.D.** has served as EVP, General Counsel, and Corporate Secretary since **2003**. He joined United Therapeutics in **1996** as General Counsel and Corporate Secretary  **[0001082554-24-000005_uthr-20231231.htm:72]** **[0001104659-23-116505_tm2329335d4_exh1a.htm:85]** **[0001308179-23-000803_uthr4146911-def14a.htm:51]** **[0001082554-24-000017_uthr-20240429.htm:48]** .                                          |
| Jan Malcolm           | Board Member                                                  | June 26, 2024       | **Jan Malcolm** was elected to the Board of Directors on **June 26, 2024**. She is a former Minnesota Commissioner of Health with a distinguished career in health policy and public health  **[0001104659-24-075375_tm2418313d1_ex99-1.htm:1]** **[0001082554-24-000017_uthr-20240429.htm:27]** .                                             |
| Christopher Causey    | Chairman of the Nominating and Governance Committee           | 2003                | **Christopher Causey** has been a director since **2003**. He was the Principal of the Causey Consortium, providing business strategy and marketing counsel to the healthcare industry until his retirement in 2021  **[0001082554-24-000017_uthr-20240429.htm:25]** **[0001308179-23-000803_uthr4146911-def14a.htm:26]** .                     |
| Christopher Patusky   | Lead Independent Director and Vice Chair of the Board of Directors | 2002                | **Christopher Patusky** has served as a Director since **2002**. He is the founding principal of Patusky Associates, LLC, and serves as an executive manager of Slater Run Vineyards, LLC  **[0001104659-23-116505_tm2329335d4_exh1a.htm:85]** **[0001308179-23-000803_uthr4146911-def14a.htm:33]** .                                               |

f56d3e7be5f1fd72f229f5469f3bc9778ebc80286cd1e6fcdcabea9f6dea4ef0

Tyvaso DPI

Nebulized Tyvaso

Total Tyvaso

Remodulin

Orenitram

Unituxin

Adcirca

Other

3e4089773529261a50c6c64a68901cba9e50e4ccf61e624e064bb99907a48609

- **Significant progress in xenotransplantation programs**: United Therapeutics is developing multiple xenotransplantation products, including the Tianjin Xeno kidney and heart, and the ThymoKidney with a single genetic modification. Preclinical evidence shows both products are effective, and the company plans to conduct clinical studies for both. This approach could provide multiple options for patients and enhance the available supply of organs.  **[UTHR_3381800_8]**
- **Strong growth in Tyvaso prescriptions among ILD physicians**: Over the last 15 months, there has been a **50% increase** in the number of interstitial lung disease physicians prescribing Tyvaso, with a **30% increase** in prescription depth measured by their "3-plus metric." The fully deployed expanded sales force is expected to further drive growth starting in the third quarter.  **[UTHR_3381800_6]**
- **Complementary relationship with new therapies like sotatercept**: The company believes sotatercept will be used alongside existing prostacyclin therapies rather than replacing them. **Nearly 70% of patients in sotatercept's clinical trials were on background prostacyclin therapy**, suggesting that patients will continue using United Therapeutics' products, potentially for longer durations, which could enhance the company's financial standing.  **[UTHR_3381800_7]**

5c75fa4a3296d9240ff982c06dd61aa913780270e5539e443e9b46327e6cdf63

- **Upcoming competition from new therapies, such as activin signaling inhibitors like sotatercept, could impact United Therapeutics' key products in pulmonary arterial hypertension (PAH), potentially reducing market share and revenues.**  **[UTHR_3374212_5]** **[UTHR_3374212_9]**
- **Significant capital expenditures, potentially several billions of dollars over the next several years, for building out facilities and investing in organ manufacturing projects, may strain financial resources and not yield expected returns.**  **[UTHR_3374212_12]**
- **Dependence on successful outcomes of clinical trials, such as the ralinepag studies and the TETON programs, introduces risk; delays in enrollment or unfavorable results could hinder future growth and impact revenue projections.**  **[UTHR_3374212_10]** **[UTHR_3374212_11]**

ca38a8d8496a14cd876e6f7c79b8e4149a63e7b0eb02f0158ea23fdf12e2ba95

- **Uncertainty in Xenotransplantation Approach and Competition:** United Therapeutics faces competition in xenotransplantation from other players using different gene modification strategies. The company acknowledges the need to conduct clinical studies to establish the safety and efficacy of their products, introducing uncertainty and risk into this key area of development.  **[UTHR_3381800_8]**
- **Potential Competitive Threat from Merck's Sotatercept:** The upcoming launch of Merck's sotatercept may pose a competitive threat to United Therapeutics' prostacyclin therapies. While the company believes sotatercept is complementary, there is a risk it could reduce the use of existing therapies, potentially impacting sales.  **[UTHR_3381800_7]**
- **Reliance on Future Clinical Trial Outcomes and Approvals:** The company's growth depends on successful clinical trials and regulatory approvals, including for their xenotransplantation products. Failure to achieve favorable results could negatively affect future prospects.  **[UTHR_3381800_8]**

f8ee5097ab6dd0397930ec73cf72e05db31f11286195441f71ce5b6428b8732d

- *Potential competition from new therapies could impact revenue growth.** United Therapeutics acknowledges questions around competitors, specifically **sotatercept and another inhaled treprostinil** that could enter the market. While management expresses confidence, the approval of these competitors could challenge UTHR's market position in PAH treatments.  **[UTHR_3363233_6]**
- *Increased sample sizes in clinical trials may indicate challenges in achieving efficacy endpoints.** The **TETON studies** for pulmonary fibrosis have increased their sample size to **576 patients each** due to variability observed in the disease, potentially reflecting difficulties in demonstrating significant results. This could delay approvals and affect future revenue projections.  **[UTHR_3363233_11]**
- *Manufacturing capacity constraints may limit near-term growth.** The company relies on manufacturing expansions at its partner MannKind to meet demand for **Tyvaso DPI**. While process improvements have been implemented, the long-term ability to fulfill increasing demand depends on new equipment coming online in the **first half of 2024**. Any delays could impact supply and sales growth.  **[UTHR_3363233_11]**

0dfe6e3130e2f52d78fdcfd9e4bfea200e6fdf0238787f321f29915c213d1f43

- United Therapeutics reported **strong underlying demand metrics** in their PAH business, including **strong referral growth, start growth, and patient shipments**, which are reflected in their revenue line.  **[UTHR_3395249_5]**
- The company is seeing **positive momentum from their sales force expansion**, with an **increase in the number of ILD prescribers writing Tyvaso**, leading to growth in both the **breadth and depth of ILD prescribers** over the last six months.  **[UTHR_3395249_4]**
- The **TETON trials in idiopathic pulmonary fibrosis (IPF)** are a top priority, with the potential to significantly expand their market opportunity, as the **IPF market is approximately three times the size** of the pulmonary hypertension market.  **[UTHR_3395249_7]**

07cabedcdf416ad9116e28f87b0b6650c95f91e95b6431cde0532dbb810d693c

- **Potential competition from Merck's sotatercept in the PAH market** could impact UTHR's growth. Management noted they wouldn't comment on Merck's product but acknowledged its presence, suggesting concerns about market dynamics.  **[UTHR_3395249_5]**
- **Uncertainty regarding FDA approval for IPF treatments may pose risks.** Both ongoing IPF studies may need to be successful for registration, and the company hasn't recently discussed the filing strategy with the FDA.  **[UTHR_3395249_7]** **[UTHR_3395249_8]**
- **Capital allocation priorities may limit future share buybacks, potentially affecting shareholder returns.** Management is focusing on the existing ASR and internal R&D initiatives, indicating uncertainty around future significant share repurchases.  **[UTHR_3395249_6]**

9fe39c75a68f355084e639eaeba7dccd820584e2ca3188a8f735ec93bc81c558

1. **IPF Filing Strategy**  
   **Q:** Do both IPF studies need to succeed for filing?  
   **A:** Management said that while the FDA typically requires **two positive studies for registration**, they will discuss options if **TETON 2 shows significant results**. They expect TETON 2 results **within a year** since enrollment was completed early.  **[UTHR_3395249_7]** 

2. **DPI Approval and Data Readout**  
   **Q:** What bridging study is needed for DPI approval in IPF?  
   **A:** They plan to confirm requirements with the FDA, possibly needing a **small sub-study in the TETON OLE program**. They will **not conduct interim analyses** but expect final TETON 2 results **within a year** after the follow-up period.  **[UTHR_3395249_8]** 

3. **Share Buyback Plans**  
   **Q:** Any plans for another significant share buyback or ASR?  
   **A:** The company is focused on executing the existing **$700 million ASR**, in process until the **end of September**. They are committed to wise capital allocation, prioritizing **R&D initiatives**, **corporate development**, and the current **share repurchase program**.  **[UTHR_3395249_6]** 

4. **Tyvaso DPI Growth Drivers**  
   **Q:** Can you elaborate on Tyvaso DPI drivers versus Nebulized Tyvaso?  
   **A:** Underlying demand continues strong with referrals and starts in both **Group 1 PAH and Group III PH-ILD**. The expanded sales force is showing impact, with an increase in **ILD prescribers** and more physicians treating patients directly due to education efforts.  **[UTHR_3395249_4]** 

5. **PAH Market Dynamics**  
   **Q:** Have you noticed changes in PAH referrals or starts?  
   **A:** The PAH business remains **solid and strong**, with **robust referral and start growth** in line with expectations, reflected in revenue. While not commenting on Merck's product, they note that most patients in Merck's trial used sotatercept **in combination with prostacyclins**, aligning with current observations.  **[UTHR_3395249_5]** 

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- **United Therapeutics has multiple shots on goal in kidney transplantation**, including the Xeno kidney, Miro kidney, and Iviva kidney implants, addressing a **large unmet need of over 500,000 patients on dialysis in the U.S.**  **[UTHR_3363233_9]**
- **Consistent growth in the Tyvaso franchise**, with net additions of approximately **500 patients per quarter**, achieving **record prescriptions and starts in Q3**, and process improvements ensuring supply meets demand.  **[UTHR_3363233_7]** **[UTHR_3363233_12]**
- **Improving operating margins due to disciplined expense management and revenue growth**, adhering to a budget algorithm limiting operating expenses to **no more than 50% of prior year revenue**, leading to better financial performance.  **[UTHR_3363233_8]**

f7e99394c9f92e4c6fe8d3ae9d6c7f5367ff5c7260d81e1a9f158064dd217691

United Therapeutics Corp (UTHR)

Financials