IPF patient survey provides new insights into
disease burden and patient experience one decade after the approval
of the first antifibrotics for IPF
Clinical abstract reviews the ability of
Bayesian analysis to maximize statistical power and reduce the
number of patients on placebo in Phase 2b ELEVATE IPF trial of
LYT-100
Topline data from the Phase 2b ELEVATE IPF
trial of LYT-100 expected by the end of 2024
PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) (“PureTech” or the
“Company”), a clinical-stage biotherapeutics company dedicated to
changing the lives of patients with devastating diseases, presented
two oral presentations and one poster supporting its clinical and
patient engagement strategies related to LYT-100 (deupirfenidone)
for the treatment of idiopathic pulmonary fibrosis (IPF) at the
CHEST 2024 Annual Meeting in Boston, Massachusetts.
“This month marks a decade since the first two antifibrotics for
the treatment of IPF were approved, and since then, limited
therapeutic advances have been made and people with IPF still face
substantial challenges,” said Camilla Graham, MD, MPH, Vice
President of Medical Affairs at PureTech Health. “At PureTech, we
are investing in changing this paradigm and this research
highlights ongoing gaps with symptom management and supportive care
in IPF. We hope this research will help improve communications
between people with IPF and their healthcare teams. It also informs
our work to develop a new IPF treatment option, which we believe
will address key limitations of the current standard-of-care
medicines.”
PureTech presented qualitative and quantitative research that
highlights both the burden of IPF as well as gaps in disease
management that exacerbate the quality of life for people with IPF.
The 90-person survey found that the majority of participants
experience a high burden of disease that interferes with their
normal activities, including shortness of breath (86%), fatigue
(78%) and cough (77%). In addition to the burden of the disease
itself, comorbidities, side effects of antifibrotic treatment and
the use of supplemental oxygen interfere with patients’ quality of
life, suggesting a need for improved interventions to manage
symptoms.
Beyond this, PureTech’s research revealed commonalities and
differences in the patient experience for those receiving care at
interstitial lung disease (ILD) centers (n=45) versus community
pulmonary practices (CPP) (n=45). When asked about their top
resources for IPF information, nearly all people in both care
settings listed their pulmonologist as their primary source, and
the majority in each were very satisfied with their communications
about their IPF diagnosis. Antifibrotic treatment rates at ILD
centers and CPPs were similar, but differences existed in perceived
communication around antifibrotic treatment options. 76% of people
receiving care in ILD centers reported that their pulmonologist had
discussed both FDA-approved antifibrotics with them, while this was
only true of 56% of those receiving care at CPPs. Across a series
of quality-of-life parameters, patients at CPPs indicated a higher
impact and severity of their disease than patients at ILD centers,
reflecting an important discrepancy that merits further
investigation.
PureTech also presented a clinical abstract reviewing its plan
to evaluate the primary outcome of the Phase 2b ELEVATE IPF
clinical trial using a prespecified Bayesian approach. ELEVATE IPF
is PureTech’s randomized, double-blind, placebo-controlled,
dose-finding study designed to evaluate the efficacy, tolerability,
safety and dosing regimen of LYT-100 in patients with IPF compared
to placebo. The trial will also assess the relative efficacy of two
doses of LYT-100. Participants have been randomized in a ratio of
1:1:1:1 to receive either 550 mg of LYT-100, 825 mg of LYT-100,
pirfenidone or placebo three times a day (TID) for up to 26 weeks
and have the option to enroll in an open-label extension. The
primary endpoint is the rate of decline in Forced Vital Capacity
(FVC) for the combined LYT-100 arms versus placebo over the 26-week
treatment period using a Bayesian linear mixed effects model
including dynamic borrowing. This approach augments the placebo arm
sample size with external placebo data from historical IPF trials.
A Bayesian approach has the advantage of enhancing overall
statistical power and improving decision-making while limiting the
number of patients required to be treated with placebo in a fatal
disease. This approach has been used previously in Phase 2 trials
of novel IPF therapeutics.
“Bayesian dynamic borrowing allows us to leverage historical
trial data for the placebo arm to maximize the number of patients
exposed to active treatment arms and minimize the number exposed to
placebo,” said Carol Ann Satler, MD, PhD, Senior Director, Clinical
Development at PureTech. “IPF is a rare, fatal disease,
underscoring the importance of efficient clinical trial design.
Bayesian analyses have previously been leveraged in IPF studies for
this reason, especially given the substantial historical placebo
dataset in IPF, and we look forward to sharing results from our
Phase 2b trial by the end of this year.”
Topline results from the Phase 2b ELEVATE IPF trial are expected
by the end of 2024. A streamlined development program for LYT-100
is planned using the same endpoints that have supported past IPF
product approvals. Pending positive clinical outcomes and
regulatory feedback, the program will advance into a Phase 3
clinical trial. PureTech believes the results of the Phase 2b
trial, together with a successful Phase 3 trial, could serve as the
basis for registration in the U.S. and other geographies.
About Idiopathic Pulmonary Fibrosis (IPF)
IPF is a rare, progressive and fatal lung disease with a median
survival of 2-5 years.1 Pirfenidone is one of only two drugs
approved to treat IPF, and for those patients able to tolerate
treatment, it has been shown to improve survival by approximately
2.5 years compared to supportive care alone.1 However, tolerability
issues with both of the standard-of-care drugs result in patients
discontinuing treatment or reducing their dose. This contributes to
nearly three out of every four people with IPF choosing to forego
treatment with these otherwise efficacious medicines.2
About LYT-100 (Deupirfenidone)
LYT-100 (deupirfenidone) is being advanced for the treatment of
conditions involving inflammation and fibrosis, including IPF. It
is a deuterated form of pirfenidone that is designed to retain the
beneficial pharmacology and clinically-validated efficacy of
pirfenidone with a highly differentiated PK profile. LYT-100 has
also demonstrated favorable tolerability across multiple clinical
studies in more than 400 individuals.
Pirfenidone is one of the two standard-of-care treatments
approved for IPF, along with nintedanib, both of which are
efficacious but associated with significant tolerability issues.
These tolerability issues result in treatment discontinuations
and/or dose reductions below the FDA-approved dose, thereby
limiting the effectiveness of these otherwise efficacious
medicines. With LYT-100, PureTech aims to deliver better outcomes
for patients by enabling individuals to maintain comparable or
higher pirfenidone-equivalent doses for longer. PureTech believes
LYT-100 has the potential both to supplant the current
standard-of-care treatments and to serve a larger market of
patients who are unable to tolerate current therapies. Topline data
for the global Phase 2 ELEVATE IPF trial are expected by the end of
2024.
About PureTech Health
PureTech is a clinical-stage biotherapeutics company dedicated
to giving life to new classes of medicine to change the lives of
patients with devastating diseases. The Company has created a broad
and deep pipeline through its experienced research and development
team and its extensive network of scientists, clinicians and
industry leaders that is being advanced both internally and through
its Founded Entities. PureTech's R&D engine has resulted in the
development of 29 therapeutics and therapeutic candidates,
including three that have been approved by the U.S. Food and Drug
Administration. A number of these programs are being advanced by
PureTech or its Founded Entities in various indications and stages
of clinical development, including registration enabling studies.
All of the underlying programs and platforms that resulted in this
pipeline of therapeutic candidates were initially identified or
discovered and then advanced by the PureTech team through key
validation points.
For more information, visit www.puretechhealth.com or connect
with us on X (formerly Twitter) @puretechh.
Cautionary Note Regarding Forward-Looking Statements
This press release contains statements that are or may be
forward-looking statements within the meaning of the Private
Securities Litigation Reform Act of 1995. All statements contained
in this press release that do not relate to matters of historical
fact should be considered forward-looking statements, including
without limitation those related to the LYT-100 development program
and development plans, its potential benefits to patients, the
timing for results from the Phase 2b clinical trial of LYT-100, the
advancement of the program into a Phase 3 trial, and our future
prospects, developments and strategies. The forward-looking
statements are based on current expectations and are subject to
known and unknown risks, uncertainties and other important factors
that could cause actual results, performance and achievements to
differ materially from current expectations, including, but not
limited to, those risks, uncertainties and other important factors
described under the caption "Risk Factors" in our Annual Report on
Form 20-F for the year ended December 31, 2023, filed with the SEC
and in our other regulatory filings. These forward-looking
statements are based on assumptions regarding the present and
future business strategies of the Company and the environment in
which it will operate in the future. Each forward-looking statement
speaks only as at the date of this press release. Except as
required by law and regulatory requirements, we disclaim any
obligation to update or revise these forward-looking statements,
whether as a result of new information, future events or
otherwise.
_________________________ 1 Fisher, M., Nathan, S. D., Hill, C.,
Marshall, J., Dejonckheere, F., Thuresson, P., & Maher, T. M.
(2017). Predicting Life Expectancy for Pirfenidone in Idiopathic
Pulmonary Fibrosis. Journal of Managed Care & Specialty
Pharmacy, 23(3-b Suppl), S17-S24.
https://doi.org/10.18553/jmcp.2017.23.3-b.s17 2 Dempsey TM, Payne
S, Sangaralingham L, Yao X, Shah ND, Limper AH. Adoption of the
Antifibrotic Medications Pirfenidone and Nintedanib for Patients
with Idiopathic Pulmonary Fibrosis. Ann Am Thorac Soc. 2021
Jul;18(7):1121-1128
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PureTech Public Relations
publicrelations@puretechhealth.com
UK/EU Media Ben Atwell, Rob Winder +44 (0) 20 3727 1000
puretech@fticonsulting.com
US Media Justin Chen +1-609-578-7230
jchen@tenbridgecommunications.com
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