First-ever human clinical trial of a xeno-organ intended to
allow for potential registration through the submission of a
Biologics License Application to the U.S. FDA
First xenotransplant is expected to occur around mid-year
2025
United Therapeutics Corporation (Nasdaq: UTHR), a public
benefit corporation, today announced that the U.S. Food and Drug
Administration (FDA) has cleared its Investigational New
Drug application (IND) to initiate a clinical study of the
company’s investigational UKidney™ derived from a 10 gene-edited
source pig. The study will enroll an initial cohort of six
end-stage renal disease (ESRD) patients, expanding to up to
50 participants, and is intended to support a Biologics License
Application (BLA) with the FDA.
United Therapeutics expects the first xenotransplant in this
trial to be performed around mid-year 2025.
According to the American Kidney Fund, more than 557,000
patients in the U.S. are on dialysis to filter their blood when
their kidneys are no longer able to do so1. A 2009 study estimated
that 52% of kidney transplant candidates who were at least 60 years
of age when placed on the transplant waitlist die within five years
before receiving a transplant2. United Therapeutics believes that
xenotransplantation offers a therapeutic alternative to
dialysis.
“Clearance of our IND for this first-ever clinical trial of a
xenokidney represents a significant step forward in our relentless
mission to expand the availability of transplantable organs,” said
Leigh Peterson, Ph.D., Executive Vice President, Product
Development and Xenotransplantation at United Therapeutics. “Our
goal is to increase the availability of transplantable organs to
offer a therapeutic alternative to a lifetime on dialysis for a
large population of patients who are unlikely to receive an
allogeneic3 kidney transplant.”
This first-in-human clinical study aims to assess safety and
efficacy of the UKidney in two groups of participants:
- ESRD patients who have been assessed and determined to be
ineligible for a conventional allogeneic kidney transplant for
medical reasons; and
- ESRD patients who have been on the kidney transplant waitlist
but are more likely to die or go untransplanted than receive a
deceased donor kidney transplant within five years.
“Eliminating the need for dialysis or limiting time on dialysis
may improve survival for many patients with ESRD,” said Noah
Byrd, Ph.D., RAC, Vice President, Global Regulatory Affairs at
United Therapeutics. “We appreciate the productive collaboration
with the FDA in advancing our efforts to bring this potentially
revolutionary therapeutic option to the hundreds of thousands of
ESRD patients.”
About the Study
Study Design
The study is a multicenter, open-label, safety and efficacy
study and is intended to support FDA approval of a BLA. The study
is designed as a combination phase 1/2/3 trial (sometimes referred
to as a “phaseless” study) to evaluate safety and efficacy
seamlessly without moving through separate phase 1, phase 2, and
phase 3 studies that are typically associated with conventional
drug approvals. Participants will receive a UKidney transplant
followed by a 24-week post-transplant follow-up period, including
the evaluation of all study endpoints and safety assessments. After
the 24-week post-transplant follow-up period, participants who
received a UKidney will continue to be followed for the rest of
their lives, including for survival, UKidney function, and
monitoring for zoonotic infections4.
Efficacy Endpoints
Efficacy endpoints include participant survival rate, UKidney
survival rate, change in measured glomerular filtration rate5, and
change in quality of life in participants6 at 24 weeks
post-transplant. Overall survival time of participants receiving a
UKidney and overall survival time of the UKidneys themselves are
also efficacy endpoints.
Safety Endpoints
Safety endpoints include the incidence of adverse events and
serious adverse events, all-cause mortality, and the incidence of
proteinuria7, zoonotic infections, and opportunistic
infections8.
Cohort-Based Design
The first cohort will consist of six transplants at two centers.
There will be a 12-week waiting period between the first and second
transplants. After the initial cohort reaches at least 12 weeks
post-transplant, safety and efficacy data will be reviewed by an
independent Data Monitoring Committee to determine if the study
should proceed to the next cohort. In addition, United Therapeutics
intends to engage with the FDA after the first six transplants are
completed. If safety and efficacy results are supportive, the
sample size will be increased to a total of up to 50 participants
to enable the study to support registration, with additional
transplant centers expected to be added to the study.
Additional Key Participation Criteria
Additional key participation criteria include an age of 55 to 70
years old, a diagnosis of ESRD, and at least six months on
hemodialysis. Participants will be screened using a crossmatch
assay to assess expected immunological compatibility with the
UKidney. Participants must not need multiple organ transplants;
must not have severe medical co-morbidities, including but not
limited to advanced cardiovascular disease, severe peripheral
vascular disease, severe neurological disease, chronic pulmonary
disease, and uncontrolled diabetes; and must not have a history of
medical noncompliance that may preclude adherence to the demands
and requirements of xenotransplantation.
Full inclusion and exclusion criteria for this study will be
provided in a future listing on the clinicaltrials.gov website.
About End-Stage Renal Disease
According to the American Kidney Fund, there are approximately
808,000 patients with kidney failure in the United States and more
than 557,000 patients on dialysis, approximately 93,000 of whom are
on the U.S. kidney transplant waiting list. Only 21,000 deceased
donor kidney transplants occurred in 20239. A 2021 study found that
three years after starting dialysis, only 12% of patients had been
placed on an Organ Procurement and Transplant Network kidney
transplant waitlist while more than 40% died10.
About UKidney
United Therapeutics’ xenokidney, known by the proposed trade
name UKidney, is an investigational xenokidney from a pig with 10
gene edits. Six human genes are added to the pig genome to
facilitate immunological acceptance and compatibility of the organ
in the human recipient, while four porcine genes are inactivated:
three that contribute to porcine organ rejection in humans and one
that can cause organ growth.
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
(PBC). Our public benefit purpose is to provide a brighter
future for patients through (a) the development of novel
pharmaceutical therapies; and (b) 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
concerning: the timing and enrollment of our planned UKidney
clinical trial; our plans to seek FDA approval of the UKidney
following completion of the clinical trial; the potential for the
UKidney to offer a therapeutic treatment option to ESRD patients;
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. 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 February 3, 2025, 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.
UKIDNEY is a trademark of United Therapeutics Corporation.
1 American Kidney Fund 2024
(https://www.kidneyfund.org/all-about-kidneys/quick-kidney-disease-facts-and-stats)
2 Schold J, Srinivas TR, Sehgal AR, Meier-Kriesche HU. Half of
kidney transplant candidates who are older than 60 years now placed
on the waiting list will die before receiving a deceased-donor
transplant. Clin J Am Soc Nephrol. 2009 Jul;4(7):1239-45. doi:
10.2215/CJN.01280209. Epub 2009 Jun 18. PMID: 19541814; PMCID:
PMC2709520.
3Two types of transplants are discussed in this release.
Allogeneic transplants are human-to-human transplants, while
xenotransplants are transplants from non-human animals into humans.
In this context, we refer to xenotransplants specifically from
gene-edited porcine (pig) source animals.
4 Zoonotic infections are infections transmitted from one
species to another.
5 The glomerular filtration rate, or GFR, measures how much
blood passes each minute through the glomeruli, the tiny filters in
the kidneys.
6 Quality of life will be measured using the following four
surveys: the EuroQol 5-Dimension 5-Level, the Standardized Outcomes
in Nephrology Life Participant, the Kidney Transplant
Questionnaire, and the Patient Global Impression of Change.
7 Proteinuria is a condition where an excessive amount of
protein is present in the urine. This can be caused by multiple
factors, including kidney damage.
8 Opportunistic infections are infections from pathogens that
would not normally impact healthy people. Transplant patients,
receiving either xenotransplants or allogeneic transplants, must
use immunosuppressive medications to prevent rejection of
transplanted organs. Opportunistic infections can occur in people
with suppressed immune activity.
9 American Kidney Fund 2024
(https://www.kidneyfund.org/all-about-kidneys/quick-kidney-disease-facts-and-stats)
10 US Renal Data System 2023
(https://usrds-adr.niddk.nih.gov/2023/end-stage-renal-disease/7-transplantation)
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For Further Information Contact: Dewey Steadman at (202)
919-4097 (media/investors) Harry Silvers at (301) 578-1401
(investors) https://ir.unither.com/contact-uthr/
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