PRESS RELEASE

SILVER SPRING, Md. and RESEARCH TRIANGLE PARK, N.C., Aug. 25, 2021 /PRNewswire/ -- United Therapeutics Corporation (Nasdaq: UTHR) today announced it is joining forces with former NFL player Devon Still and his daughter Leah, a survivor of high-risk neuroblastoma, to launch the educational initiative "Braving NeuroBLASToma" shining a light on the rare cancer affecting immature nerve cells called neuroblasts.^iv Neuroblastoma often develops in infants and children under the age of five, but the average age of diagnosis is between one and two years old.

Each year, about 800 children are diagnosed with neuroblastoma, accounting for seven to ten percent of all childhood cancers in the United States. ^{v, vi}  At the time of diagnosis, nearly 70 percent of children will have advanced or metastatic neuroblastoma, with only half of these patients achieving remission.^vii,viii Neuroblastoma can start to form in several places including near the abdomen, spine, chest, or adrenal glands.^ix 

"When I first learned that Leah had high-risk neuroblastoma, the sense of fear and helplessness was so overwhelming that I struggled to know where to begin," said Devon Still. "Over the years, so many in the childhood cancer community have shown us how much they care each and every step of the way, from making the tough decisions to ensuring Leah's comfort while undergoing treatment. Partnering with United Therapeutics enables us to share our personal experiences with braving neuroblastoma and lend others the support we so generously received throughout the years."

Braving NeuroBLASToma features:

• A family-friendly toolkit with a resource-rich website, including a comprehensive library of information and resources such as questions for the doctor, navigating clinical trials, understanding treatment, and caregiver advice.
• A series of four beautifully illustrated books, including the latest release of Zara Takes Off – inspiring hope and encouragement as families transition to life after treatment. The new release includes a personal foreword penned by Leah, sharing details of her personal journey.
• Each book highlights different stages of the high-risk neuroblastoma journey, beginning with The Big Adventures of Little Skivolo that helps families understand diagnosis and treatment, The Next Big Adventure of Little Skivolo that focuses specifically on the antibody therapy phase of treatment, Little Skivolo's Big Book of Fun, an activity book to help entertain kids and families during hospital stays and culminates with Zara Takes Off.  

"For many years, United Therapeutics has been working with leading researchers in pediatric neuroblastoma, including the Children's Oncology Group supported by the National Cancer Institute, to bring hope to thousands of families fighting this deadly childhood cancer," said Karren Jackson , Oncology Program Head at United Therapeutics. "We are tremendously proud to partner with Devon Still and his daughter Leah to highlight what courage, resilience and support from loved ones can do for children and their families impacted by neuroblastoma."

As treatment comes to an end, families can start to transition out of treatment mode and into more familiar routines while the healthcare team continues to monitor the child's recovery. But the neuroblastoma journey doesn't stop there, patients will continue to see their oncology team on a routine basis to monitor for long-term side effects and to ensure the cancer has not returned. This leg of the journey requires continued vigilance, support, and encouragement.

"The treatment of a child with high-risk neuroblastoma challenges both patients and caregivers to their core, with each family enduring unique and evolving medical needs," said Rochelle Bagatell , MD, Pediatric Oncologist, Children's Hospital of Philadelphia, and Neuroblastoma Committee Chair for the Children's Oncology Group. "As a care provider, I encourage families to leverage the resources available to them, so together we can ensure that each child receives the best care and support possible."

For more information visit BravingNeuroBLASToma.com, a comprehensive resource for every step in the patient journey.

About Neuroblastoma:
Neuroblastoma is the most common cancer in infants less than a year old, but is considered a rare cancer.ⁱ Each year, about 800 children are diagnosed with neuroblastoma, accounting for seven to ten percent of all childhood cancers in the United States.^v,vi This solid-tumor cancer starts in immature nerve cells called neuroblasts and often develops in infants and children under the age of five, but the average age of diagnosis is between one and two years old.^iv Neuroblastoma can form anywhere along the sympathetic nerve chain and is commonly seen in in the abdomen, spine, chest or adrenal glands.^ix At the time of diagnosis, nearly 70 percent of children will have advanced or metastatic neuroblastoma.^vii Despite advancements in the research and approval of treatments for neuroblastoma, there remains a high unmet need for high-risk neuroblastoma patients. For children with pediatric high-risk neuroblastoma, even when treated and remission is achieved, about 50-60% will relapse (disease returns) and about 10% of children with pediatric high-risk neuroblastoma will not respond to upfront chemotherapy—this is called refractory neuroblastoma.^x There is currently no cure for relapsed high-risk neuroblastoma.^xi

United Therapeutics: Enabling Inspiration
United Therapeutics Corporation focuses on the strength of a balanced, value-creating biotechnology model. We are confident in our future thanks to our fundamental attributes, namely our obsession with quality and innovation, the power of our brands, our entrepreneurial culture, and our bioinformatics leadership. We also believe that our determination to be responsible citizens – having a positive impact on patients, the environment, and society – will sustain our success in the long term.

Through our wholly owned subsidiary, Lung Biotechnology PBC, we are focused on addressing the acute national shortage of transplantable lungs and other organs with a variety of technologies that either delay the need for such organs or expand the supply. Lung Biotechnology is the first public benefit corporation subsidiary of a public biotechnology or pharmaceutical company.

Please visit unither.com to learn more.

Forward Looking Statements
Statements included in this press release that are not historical in nature are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include, among others, our ability to create value and sustain our success in the long-term, as well as our efforts to develop technologies that either delay the need for transplantable organs or expand the supply of transplantable organs. These forward-looking statements are subject to certain risks and uncertainties, such as those described in our periodic reports filed with the Securities and Exchange Commission, that could cause actual results to differ materially from anticipated results. Consequently, such forward-looking statements are qualified by the cautionary statements, cautionary language and risk factors set forth in our periodic reports and documents filed with the Securities and Exchange Commission, including our most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K. We claim the protection of the safe harbor contained in the Private Securities Litigation Reform Act of 1995 for forward-looking statements. We are providing this information as of August 25, 2021, and assume no obligation to update or revise the information contained in this press release whether as a result of new information, future events or any other reason.

Media Contact:
Lisa Warshaw
(323) 360-8778
[email protected]

ⁱAmerican Cancer Society. Key Statistics. https://www.cancer.org/cancer/neuroblastoma/about/key-statistics.html
ⁱⁱ Children's Neuroblastoma Cancer Foundation. FAQs. https://www.cncfhope.org/about-cncf/faqs/ 
ⁱⁱⁱ American Cancer Society. Types of Cancer that Develop in Children. https://www.cancer.org/cancer/cancer-in-children/types-of-childhood-cancers.html 
^iv https://www.cancer.org/cancer/neuroblastoma/about/what-is-neuroblastoma.html
^v Cancer.net. Neuroblastoma-Childhood: Statistics. https://www.cancer.net/cancer-types/neuroblastoma-childhood/statistics#:~:text=Each%20year%2C%20about%20800%20children,1%20and%202%20years%20old.
^vi St. Jude Children's Research Hospital. Neuroblastoma. https://together.stjude.org/en-us/about-pediatric-cancer/types/neuroblastoma.html 
^vii PDQ Cancer Information Summaries. Neuroblastoma Treatment. https://www.ncbi.nlm.nih.gov/books/NBK65747/ 
^viii Dana-Farber Cancer Institute. Childhood Neuroblastoma. https://www.dana-farber.org/childhood-neuroblastoma/ 
^ix Cancer. Net. Neuroblastoma-Childhood: Introduction. https://www.cancer.net/cancer-types/neuroblastoma-childhood/introduction 
^x https://www.childrensoncologygroup.org/index.php/in-treatment-for-neuroblastoma 
^xihttps://www.chop.edu/conditions-diseases/relapsed-or-refractory-neuroblastoma

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SOURCE United Therapeutics Corporation