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UNITED THERAPEUTICS ANNOUNCES FIRST PATIENT ENROLLED IN PHASE 3 TETON 2 STUDY OF TYVASO IN PATIENTS WITH IDIOPATHIC PULMONARY FIBROSIS Second registration study of Tyvaso (treprostinil) Inhalation S

Key Takeaway: UNITED THERAPEUTICS ANNOUNCES FIRST PATIENT ENROLLED IN PHASE 3 TETON 2 STUDY OF TYVASO IN PATIENTS WITH IDIOPATHIC PULMONARY FIBROSIS Second registration study of Tyvaso (treprostinil) Inhalation Solution for patients with IPF SILVER SPRING, Md. and RESEARCH TRIANGLE PARK, N

Full Press Release Details

UNITED THERAPEUTICS ANNOUNCES FIRST PATIENT
ENROLLED IN PHASE 3 TETON 2 STUDY OF TYVASO IN PATIENTS WITH IDIOPATHIC PULMONARY FIBROSIS
Second registration study of Tyvaso
(treprostinil) Inhalation Solution for patients with IPF
SILVER SPRING, Md. and RESEARCH TRIANGLE PARK, N.C., October 11,
2022: United Therapeutics Corporation (Nasdaq: UTHR), a public benefit corporation, announced today that the first patient
has enrolled in the phase 3 TETON 2 study which will evaluate Tyvaso in 396 adult patients with idiopathic pulmonary fibrosis
(IPF) at sites outside the United States and Canada. This second registration study is part of the broader global TETON
program evaluating Tyvaso for the treatment of IPF. The 52-week study will evaluate the impact of Tyvaso on a key prognostic
indicator for IPF known as forced vital capacity (FVC). IPF is a progressive lung disease characterized by the loss of the
ability of the lungs to transfer oxygen into the blood, ultimately resulting in respiratory failure and death.
"Despite the availability of two approved products in this therapeutic
category, there remains a critical unmet need in IPF," said Steven Nathan, M.D., Medical Director of the Advanced Lung Disease
and Lung Transplant Program at Inova Fairfax Hospital in Falls Church, Virginia, who is also chair of the TETON program steering
committee. "As is now widely known in the pulmonology community, the safety data collected from the INCREASE study showed
a positive impact of inhaled treprostinil on FVC in IPF patients with pulmonary hypertension. In follow-up to this, the TETON 1 and
TETON 2 studies have been designed to further investigate the potential antifibrotic effects of inhaled treprostinil in IPF patients."
Tyvaso is currently approved by the U.S. Food and Drug Administration
(FDA) to treat both pulmonary arterial hypertension and pulmonary hypertension (PH) associated with interstitial lung disease
(PH-ILD). The PH-ILD indication, which includes patients with PH associated with IPF, was added to the Tyvaso label in March 2021
based on the successful results of the INCREASE study. Tyvaso is not approved in any jurisdiction for use for IPF patients without
documented PH. Additionally, Tyvaso DPI (treprostinil) inhalation powder is not being evaluated in the TETON program.
"The expansion of the TETON program demonstrates our excitement
about the potential for Tyvaso in IPF," said Peter Smith, Pharm.D., United Therapeutics' Vice President of Global Product
Development. "Like with TETON 1, the TETON 2 study is an example of our flexible development model to expand beyond
PH to potentially help us better understand the impact of treprostinil as a potential treatment option for this vulnerable group of patients."
Parallel in design to the TETON 1 study, the TETON 2
study is a 396-patient, multicenter, randomized, double-blind, placebo-controlled phase 3 registration study to evaluate the safety and
efficacy of Tyvaso in subjects with IPF over a 52-week period. This second registration study is part of the broader global TETON
program evaluating Tyvaso for the treatment of IPF and will be conducted at sites outside of the United States and Canada. The TETON
1 study is being conducted at sites in the United States and Canada.
Subjects will be randomly allocated 1:1 to receive Tyvaso or placebo.
All subjects will initiate Tyvaso or placebo at a dose of three breaths administered four times daily (QID) and will titrate to
a target dosing regimen of 12 breaths QID. Study drug doses may be titrated up as tolerated, until the target dose or maximum clinically
tolerated dose is achieved.
The primary endpoint of the study is the change in FVC from baseline
to week 52. Secondary endpoints include: (1) time to clinical worsening; (2) time to first acute exacerbation of IPF; (3) overall survival
at week 52; (4) change in percent predicted FVC from baseline to week 52; and (5) change in the King's Brief Interstitial Lung Disease
Other data collected in the study will include the plasma N-terminal
pro-brain natriuretic peptide (NT-proBNP) concentration, supplemental oxygen use, and lung diffusion capacity. Safety assessments
include the development of adverse events, serious adverse events, vital signs, clinical laboratory parameters, and electrocardiogram
IPF is a scarring disease of the lungs of an unknown (idiopathic) cause
and is the most common of the idiopathic interstitial pneumonias. IPF is characterized by the progressive loss of the ability of the lungs
to transfer oxygen into the blood, ultimately resulting in respiratory failure and death. While the precise causes of IPF remain unknown, IPF rarely presents
before age 50 and can be associated with cigarette smoking and certain genetic dispositions. In addition, some evidence suggests that
gastroesophageal reflux (acid reflux, or heartburn), certain viral infections, air pollution, and some exposures in the workplace may
be risk factors for IPF. According to recent research, IPF is estimated to affect between 0.33 and 4.51 people per 10,000 persons worldwide1.
Further, United Therapeutics estimates there are around 100,000 IPF patients in the United States alone.
About TYVASO (treprostinil) Inhalation Solution
and TYVASO DPI (treprostinil) Inhalation Powder
TYVASO (treprostinil) Inhalation Solution and TYVASO DPI (treprostinil)
Inhalation Powder are prostacyclin mimetics indicated for the treatment of:
The effects diminish over the minimum recommended dosing
interval of 4 hours; treatment timing can be adjusted for planned activities.
While there are long-term data on use of treprostinil by
other routes of administration, nearly all clinical experience with inhaled treprostinil has been on a background of an endothelin receptor
antagonist (ERA) and/or a phosphodiesterase type 5 (PDE-5) inhibitor. The controlled clinical experience with TYVASO was limited to 12
IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS
DRUG INTERACTIONS/SPECIFIC POPULATIONS
In a 12-week, placebo-controlled study (TRIUMPH I) of 235
patients with PAH (WHO Group 1 and nearly all NYHA Functional Class III), the most common adverse reactions seen with TYVASO in 4%
of PAH patients and more than 3% greater than placebo were cough (54% vs 29%), headache (41% vs 23%), throat irritation/pharyngolaryngeal
pain (25% vs 14%), nausea (19% vs 11%), flushing (15% vs <1%), and syncope (6% vs <1%). In addition, adverse reactions occurring
in 4% of patients were dizziness and diarrhea.
In a 3-week, open-label, single-sequence, safety and tolerability
study (BREEZE) conducted in 51 patients on stable doses of TYVASO who switched to a corresponding dose of TYVASO DPI, the most commonly
reported adverse events seen with TYVASO DPI in 4% of PAH patients during the 3-week treatment phase included cough (35.3%), headache
(15.7%), dyspnea (7.8%), and nausea (5.9%).
In a 16-week, placebo-controlled study (INCREASE) of 326
patients with PH-ILD (WHO Group 3), adverse reactions with TYVASO were similar to the experience in studies of PAH.
Please see Full Prescribing Information for
TYVASO or TYVASO DPI, Instructions for Use manuals for TD-100 and TD-300 TYVASO Inhalation System
and TYVASO DPI Inhalation Powder, and additional information at www.TYVASOHCP.com or call 1-877-UNITHER
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 relating our plans to conduct the TETON 1 and TETON 2 studies, including our
plan to enroll 396 patients in the TETON 2 study, the potential for inhaled treprostinil to become a treatment option for patients
with idiopathic pulmonary fibrosis, our commitment to fulfilling our purpose and promise to our patients, our employees, and all of humankind,
our ability to create value and sustain our success in the long-term, and 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 October 11, 2022 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.
TYVASO and TYVASO DPI are registered trademarks of United Therapeutics
For Further Information Contact:
Dewey Steadman at (202) 919-4097
Last updated: Oct 11, 2022