Second UHeart™ recipient recovering after a successful
transplant
UThymoKidney™ demonstrates normal function during a 61-day
study
Human xenotransplant clinical studies could begin in 2025
United Therapeutics Corporation (Nasdaq: UTHR), a public
benefit corporation, today announced the achievement of two
milestones for its xenotransplantation programs in September: the
second transplant of a UHeart™ xenoheart into a living person, and
a 61-day study of the UThymoKidney™ xenokidney and thymus in a
human pre-clinical model.
United Therapeutics’ organ manufacturing efforts consist of
seven investigational programs: xenoheart, xenokidney,
xenothymokidney, regenerative lungs, 3D-printed lungs, 3D-printed
livers, and 3D-printed kidneys. These groundbreaking programs are
intended to address the ongoing shortage of transplantable organs
for patients with end stage organ disease.
According to the U.S. Health Resources and Services
Administration, around 110,000 Americans are currently waiting for
an organ transplant, and more than 6,000 patients – 17 every day –
die each year before receiving one. Almost 89,000 patients are
waiting for kidneys, over 10,000 for livers, over 3,300 for hearts,
and almost 1,000 for lungs, with many more patients suffering from
end-stage organ failure who are ineligible for the strict organ
transplant waiting list who could benefit from a readily available
supply of organs on demand.
United Therapeutics initiated its xenotransplantation research
work in 2011 and currently employs more than 50 scientists and
support staff advancing this program. The company is currently
finalizing construction of a $100 million clinical-scale designated
pathogen free facility in Christiansburg, VA to support future
clinical xenotransplantation studies with a capacity of
approximately 125 organs per year.
To date, 10 xenotransplantation procedures using United
Therapeutics' xenohearts and xenokidneys have been performed in
living and brain-dead human recipients: two living human recipients
of xenohearts, six brain-dead xenokidney/thymokidney recipients,
and two brain-dead xenoheart recipients. United Therapeutics has
built on its history of innovation in xenotransplantation with
strong partnerships with top academic medical centers including the
University of Maryland Medicine (UMM), NYU Langone Health
(NYU), and the University of Alabama at Birmingham
(UAB).
Second Successful Xenoheart Transplant Conducted at the
University of Maryland Medical Center
In the second procedure of its kind, surgeons affiliated with
UMM last Wednesday transplanted a UHeart from a 10-gene modified
pig into Mr. Lawrence Faucette, under an emergency approval
from the U.S. Food and Drug Administration (FDA). According
to the transplant team, a xenoheart transplant was the only option
available for Mr. Faucette, who has end-stage heart disease and was
deemed ineligible for a traditional transplant with a human heart
due to his pre-existing peripheral vascular disease and
complications with internal bleeding. Mr. Faucette was facing
near-certain death from heart failure.
United Therapeutics’ UHeart is a heart from a pig with 10
genetic modifications to support organ functioning in the human
body. Six human genes were added to the pig genome to facilitate
immune acceptance of the organ, while four genes were knocked out:
three that contribute to porcine organ rejection in humans and one
that can cause organ growth beyond what is normal for humans.
United Therapeutics is also developing the UKidney™, a kidney from
the same 10-gene modified pig.
“The second transplantation of a UHeart into a living person and
the extended study of a UThymoKidney in a decedent, coupled with
other ground-breaking research by our collaborators at the
University of Alabama at Birmingham, University of Maryland
Medicine, and NYU Langone Health, form the basis of knowledge we
are sharing with the FDA ahead of commencing future clinical
studies of xeno organs in humans,” said Leigh Peterson,
Ph.D., Executive Vice President, Product Development &
Xenotransplantation at United Therapeutics. “We look forward to
continuing our dialogue with the FDA with the goal of starting
clinical studies in 2025.”
Dr. Peterson continued: “We are grateful for the courageous
contribution of Mr. Faucette and his family to the advancement of
this important science. The entire United Therapeutics family is
pulling for Mr. Faucette and hopeful for a smooth recovery.”
United Therapeutics is preparing for clinical trials of its
xenoheart and xenokidney products, following completion of ongoing
preclinical studies required by the FDA.
Xenokidney Demonstrates the Longest-Documented Case of Xeno
Organ Function in a Human Body at NYU Langone Medical
Center
In a 61-day study that concluded September 13th, surgeons at NYU
Langone Medical Center evaluated the function of a UThymoKidney in
what is now the longest-documented case of a xeno organ functioning
in a human body. The kidney recipient, with his family’s consent,
was a deceased 58-year-old man who had been on a ventilator after
being declared dead by neurologic criteria before the
xenotransplant.
United Therapeutics’ UThymoKidney is a kidney from a one-gene
modified pig along with the pig’s thymus. The gene that is
inactivated is responsible for the synthesis of alpha-gal, a sugar
on the surface of cells that can cause the immediate rejection of
the organ in a human body. The pig’s thymus is intended to help
teach the recipient’s immune system to recognize the kidney as
“self” and prevent rejection.
“We at United Therapeutics congratulate our research
collaborators at University of Maryland Medicine and NYU for these
two historic achievements,” said David Ayares, Ph.D.,
President and Chief Scientific Officer of United Therapeutics’
Revivicor subsidiary. “We also recognize the contributions of the
patients and families associated with these procedures that help us
move forward with this important science. In addition, I want to
recognize the decades of work and dedication by the Revivicor team
to bring this technology so close to its potential to revolutionize
the way we treat end stage organ disease.”
United Therapeutics: Enabling Inspiration
At United Therapeutics, our vision and mission are one. We use
our enthusiasm, creativity, and persistence to innovate for the
unmet medical needs of our patients and to benefit our other
stakeholders. We are bold and unconventional. We have fun; we do
good. We are the first publicly traded biotech or pharmaceutical
company to take the form of a public benefit corporation. Our
public benefit purpose is to provide a brighter future for patients
through the development of novel pharmaceutical therapies; and
technologies that expand the availability of transplantable
organs.
You can learn more about what it means to be a PBC here:
unither.com/PBC.
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, statements
regarding progress toward developing our organ manufacturing
programs, including our plans to complete required preclinical
studies and commence clinical trials of one or more
xenotransplantation products in 2025, our planned construction of a
clinical-grade xenotransplantation manufacturing facility and its
anticipated 125 organ per year capacity, and our goals of
innovating for the unmet medical needs of our patients and to
benefit our other stakeholders and furthering our public benefit
purpose of developing novel pharmaceutical therapies and
technologies that expand the availability 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. In particular, our plans to commence
clinical studies of one or more xenotransplantation products in
2025 are subject to regulatory clearance, including the completion
of preclinical studies to the satisfaction of the FDA, and many
other factors that we cannot control. 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 September 22, 2023, 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.
UHEART, UKIDNEY, and UTHYMOKIDNEY are trademarks of United
Therapeutics Corporation.
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version on businesswire.com: https://www.businesswire.com/news/home/20230922830369/en/
Dewey Steadman at (202) 919-4097
https://ir.unither.com/contact-uthr/
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