9 October
2024
PureTech Health plc
PureTech Presents Research
Highlighting Burden of Idiopathic Pulmonary Fibrosis (IPF) and Use
of a Bayesian Statistical Analysis for LYT-100 (Deupirfenidone) at
CHEST 2024 Annual Meeting
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.
Contact:
PureTech
Public Relations
publicrelations@puretechhealth.com
UK/EU Media
Ben Atwell, Rob Winder
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Justin Chen
+1-609-578-7230
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