RCS - PureTech Health PLC - PureTech Showcases Differentiated IPF Strategy

Puretech HealthAnnouncement

21/08/2025 12:00

For best results when printing this announcement, please click on link below:
https://newsfile.refinitiv.com/getnewsfile/v1/story?guid=urn:newsml:reuters.com:20250821:nRSU2057Wa&default-theme=true

RNS Number : 2057W  PureTech Health PLC  21 August 2025

21 August 2025

PureTech Health plc

 

PureTech Showcases Differentiated Development Strategy, Including New Patient
Preference Insights, and Spotlights Phase 2b Data Positioning Deupirfenidone
as a Potential New Standard of Care in IPF

 

Deupirfenidone to be advanced by PureTech's newly launched Founded Entity,
Celea Therapeutics

 

PureTech Health plc (https://puretechhealth.com/) (Nasdaq: PRTC, LSE: PRTC)
("PureTech" or the "Company"), a clinical-stage biotherapeutics company
dedicated to changing the lives of patients with devastating diseases,
demonstrated its growing leadership in idiopathic pulmonary fibrosis (IPF) at
the 2025 IPF Summit this week in Boston. The Company highlighted the strength
of its Phase 2b ELEVATE IPF trial data supporting the advancement of
deupirfenidone (LYT-100) into Phase 3 by its newest Founded Entity, Celea
Therapeutics ("Celea"), and shared new patient preference data and insights
shaped by deep engagement across the IPF treatment ecosystem.

 

"With deupirfenidone demonstrating compelling efficacy, we are taking a
holistic approach to Phase 3 planning - one that is informed by a deep
understanding of the needs of patients, clinicians, and payers alike," said
Camilla Graham, MD, MPH, Senior Vice President of Medical Affairs
at PureTech. "After more than a decade without meaningful therapeutic
advancement, the IPF treatment landscape is beginning to shift. To deliver
real impact, innovation must extend beyond the molecule - into clinical trial
design, patient and provider education, and access. That's the bar we've set
with deupirfenidone."

 

Balancing Efficacy and Safety of Treatment: Perspectives of People Living with
IPF

As part of its commitment to developing therapies that address real-world
needs, PureTech shared new findings from structured interviews with 16
individuals living with IPF, designed to understand how patients weigh the
trade-offs between efficacy and tolerability when considering novel
treatments. The data highlight that patients are not only open to new options,
they are primarily motivated by the potential for better outcomes. Key
findings included the following:

 

·      69% of participants preferred a hypothetical treatment offering
greater efficacy over one with improved tolerability.

·      94% said they would proactively initiate a conversation with
their physician if a new treatment offered a differentiated profile.

 

"We've seen real hesitation around initiating currently available therapies -
even among patients with progressive disease," said Tejaswini Kulkarni, MD,
MPH, University of Alabama at Birmingham and co-author on the research. "These
findings reflect a shift in mindset. People with IPF want better outcomes, and
they're willing to accept manageable tolerability trade-offs in pursuit of a
more effective treatment. That perspective is essential to designing trials
and therapies that truly resonate with those who matter most."

 

De-risking Late-Stage Development through Sophisticated Trial Design

Across two expert panels at the IPF Summit, PureTech shared insights into the
clinical development strategy behind deupirfenidone, emphasizing how the
design of its Phase 2b ELEVATE IPF trial was purposefully built to address
persistent challenges in the development of new IPF therapies.

 

"The ELEVATE IPF trial stands out for its thoughtful design and execution,"
said Toby Maher, MD, PhD, Professor of Medicine and Director of Interstitial
Lung Disease at Keck School of Medicine, University of Southern California,
Los Angeles, and lead investigator in the ELEVATE IPF trial. "By selecting a
26-week treatment period, including an active comparator, and applying
rigorous data quality controls, the study was designed to generate the type of
robust and reliable data that has often been difficult to achieve in
early-stage IPF therapeutic development. Just as importantly, deupirfenidone
builds on more than a decade of clinical and real-world experience with
pirfenidone, reinforcing confidence in the ELEVATE data and its potential to
be replicated in Phase 3."

 

The trial incorporated key features to generate high-confidence data that
position deupirfenidone for a Phase 3 program with reduced clinical risk,
including:

·      Trial Duration: Most Phase 2 IPF trials last just 12 weeks. While
this design can help establish an early efficacy signal, it often fails to
capture the strength and durability of a treatment effect over time.
PureTech's Phase 2b ELEVATE IPF trial spanned 26 weeks, enabling a more robust
assessment of lung function decline and a clearer view of long-term
therapeutic potential. Importantly, open-label extension data further support
the durability of deupirfenidone's treatment effect through at least 52 weeks
while reinforcing safety and tolerability.

·      Data Quality: Spirometry, the primary efficacy measure in IPF
trials, is inherently variable. To ensure data integrity, ELEVATE IPF employed
training to 2019 ATS standards and centrally-read spirometry with rigorous
quality control systems, resulting in clean, consistent data.

·      Active Comparator Arm: ELEVATE IPF was the first
industry-sponsored trial to include an approved antifibrotic agent as an
active comparator. In the trial, both pirfenidone and placebo performed in
line with historical expectations, reinforcing the credibility and
reproducibility of the deupirfenidone data.

·      Consistency of Development Plan: PureTech intends to discuss a
Phase 3 trial design with the U.S. Food and Drug Administration that
recapitulates key aspects of ELEVATE IPF, thereby minimizing technical risk
from protocol changes.

 

Together, these elements reinforce the strength and reliability of the Phase
2b data and underscore why deupirfenidone is poised to set a new benchmark as
it advances into Phase 3.

 

Deupirfenidone: A Potential New Standard of Care

In a dedicated clinical update at the IPF Summit, PureTech presented data from
its global, randomized, double-blind, active- and placebo-controlled Phase 2b
ELEVATE IPF trial, which support the advancement of deupirfenidone into Phase
3 as a potential new standard of care. The trial met its primary endpoint and
demonstrated a statistically significant and clinically meaningful reduction
in lung function decline at 26 weeks with deupirfenidone 825 mg three times a
day (TID) compared to placebo.

 

Patients treated with deupirfenidone 825 mg TID experienced a slower rate of
lung function decline, as measured by change from baseline of Forced Vital
Capacity (FVC), at 26 weeks versus those who were treated with pirfenidone 801
mg TID or placebo (-21.5 mL vs. -51.6 mL vs. -112.5 mL, respectively). 1 
(#_ftn1) Based on their respective reductions in lung function decline versus
placebo, the treatment effect with deupirfenidone 825 mg TID was approximately
50% greater than that of pirfenidone 801 mg TID (80.9% vs. 54.1%). This effect
is supported by pharmacokinetic data showing that treatment with
deupirfenidone 825 mg TID achieved ~50% higher drug exposure than pirfenidone
801 mg TID, without a corresponding increase in tolerability challenges.

 

Notably, initial data from the ongoing open-label extension (OLE) study showed
that the treatment effect with deupirfenidone 825 mg TID was sustained out to
at least 52 weeks. As of May 9, 2025, a total of 101 patients had received at
least 52 weeks of treatment with deupirfenidone. Those in the deupirfenidone
825 mg TID arm experienced a decline in FVC of -32.8 mL over the 52-week
period, 2  (#_ftn2) which is similar to the expected natural decline in lung
function in healthy older adults over one year (approximately -30.0 mL to
-50.0 mL). 3  (#_ftn3) These longer-term data support the durability of the
treatment effect observed with this dose and reinforce its potential
improvements over current IPF treatments to stabilize lung function decline
over time, while maintaining favorable safety and tolerability.

 

Additional data, including outcomes in participants who transitioned to
deupirfenidone in the OLE after initially receiving placebo or pirfenidone
during the blinded portion of the trial, will be presented at the upcoming
European Respiratory Society (ERS) International Congress in September 2025.

 

Celea, PureTech's newly launched Founded Entity focused on respiratory
diseases, is advancing the development and potential commercialization of
deupirfenidone. The company is led by Sven Dethlefs, PhD, who has played a
central role in driving the program forward over the past year and brings deep
expertise in respiratory therapeutics and commercial execution.

 

"The ELEVATE IPF trial has delivered some of the most compelling data the IPF
field has seen, and it's a privilege to now be leading the effort to advance
deupirfenidone into Phase 3 through PureTech's newest Founded Entity, Celea
Therapeutics," said Dr. Dethlefs. "With a foundation of rigorous science,
differentiated efficacy, and strong stakeholder alignment, we believe
deupirfenidone has the potential to become a new standard of care for people
with IPF."

 

About Deupirfenidone (LYT-100)

Deupirfenidone (LYT-100) is in development as a potential new standard of care
for the treatment of idiopathic pulmonary fibrosis (IPF). It is a deuterated
form of pirfenidone, which - along with nintedanib - is one of the two
FDA-approved treatments for IPF.  Both approved therapies offer only modest
efficacy in slowing lung function decline, largely due to tolerability
challenges that limit the ability to achieve higher doses that could
significantly improve patient outcomes. These limitations have contributed to
low treatment uptake and poor adherence, with approximately 25% of people with
IPF in the U.S. ever receiving either drug. 4  (#_ftn4) Despite this, combined
peak global sales exceed $5 billion, representing a significant market
opportunity in IPF and other fibrotic lung diseases.  5  (#_ftn5)

 

Deupirfenidone may overcome these limitations. In the global Phase 2b ELEVATE
IPF trial, deupirfenidone demonstrated the potential to stabilize lung
function decline over at least 26 weeks as a monotherapy while maintaining a
favorable safety and tolerability profile. Initial data from an ongoing
open-label extension study suggest that this effect may be sustained through
at least 52 weeks. These findings support the potential for deupirfenidone to
offer a meaningful advance for people living with this progressive and deadly
disease. Beyond IPF, deupirfenidone may also address multiple underserved
fibrotic conditions, including progressive fibrosing interstitial lung
diseases.

 

About Idiopathic Pulmonary Fibrosis (IPF)

Idiopathic pulmonary fibrosis (IPF) is a rare, progressive, and fatal lung
disease characterized by irreversible scarring of lung tissue that leads to a
steady decline in lung function. Median survival following diagnosis is
estimated to be two to five years, and currently there is no cure. 6  (#_ftn6)

 

About Celea Therapeutics

Celea Therapeutics is dedicated to advancing transformative treatments for
people with serious respiratory diseases. Drawn from the Latin word for "sky,"
the name reflects the company's mission to rise above the status quo and
deliver therapies that change lives. The company's lead program,
deupirfenidone (LYT-100), is a Phase 3-ready therapeutic candidate with the
potential to set a new standard of care for idiopathic pulmonary fibrosis
(IPF) and other fibrotic lung diseases.

 

Celea was founded by PureTech Health plc (Nasdaq: PRTC, LSE: PRTC), a
biotherapeutics company dedicated to giving life to science. PureTech's
innovative R&D model drives the creation of Founded Entities like Celea,
enabling the advancement of highly promising medicines to patients in a
capital-efficient manner. For more information, please visit www.celeatx.com
and www.puretechhealth.com.

 

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 portfolio 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 portfolio 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
(http://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 statements that relate to continued development
of and regulatory interactions related to deupirfenidone, the potential of
deupirfenidone in IPF and other indications, our expectations around our
therapeutic candidates and approach towards addressing major diseases, our
plans to advance our programs and deliver on our milestones, our future plans,
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, 2024 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.

 

PureTech
Public Relations
publicrelations@puretechhealth.com (mailto:publicrelations@puretechhealth.com)
(mailto:publicrelations@puretechhealth.com)
Investor Relations
IR@puretechhealth.com (mailto:IR@puretechhealth.com)
(mailto:IR@puretechhealth.com)

(mailto:IR@puretechhealth.com)
UK/EU Media
Ben Atwell, Rob Winder
+44 (0) 20 3727 1000
puretech@fticonsulting.com (mailto:puretech@fticonsulting.com)
(mailto:puretech@fticonsulting.com)

(mailto:puretech@fticonsulting.com)
US Media
Justin Chen
jchen@tenbridgecommunications.com (mailto:jchen@tenbridgecommunications.com)

 

 

 

 

 1  (#_ftnref1) The efficacy analysis used a random coefficient regression
model with absolute FVC including baseline as response variable and week,
treatment, and interaction between week and treatment as fixed effect. The
analysis was performed based on the predefined Full Analysis Set. The rate of
FVC decline at week 26 with deupirfenidone 825 mg TID compared to placebo was
statistically significant (-21.5 mL vs. -112.5 mL, respectively; p=0.02). p
value is two-sided and has not been corrected for multiplicity. The rate of
FVC decline at week 26 with pirfenidone 801 mg TID compared to placebo was
consistent with previously reported pirfenidone clinical trial data (Roche).
ELEVATE-IPF was not designed to demonstrate the superiority of deupirfenidone
825 mg TID vs. pirfenidone 801 mg TID.

 2  (#_ftnref2) Analysis conducted using the same methodology to assess rate
of decline in FVC at 26 weeks from the double-blind portion of the trial.
Efficacy analysis used a random coefficient regression model with absolute FVC
including baseline as response variable and week, treatment and interaction
between week and treatment as fixed effect. The analysis was performed based
on the predefined Full Analysis Set.

 3  (#_ftnref3) Valenzuela, C., Bonella, F., Moor, C., Weimann, G., Miede, C.,
Stowasser, S., & Maher, T. (2024, September). Decline in forced vital
capacity (FVC) in subjects with idiopathic pulmonary fibrosis (IPF) and
progressive pulmonary fibrosis (PPF) compared with healthy references [Poster
presentation]. European Respiratory Society International Congress, Vienna,
Austria; and Luoto, J., Pihlsgård, M., Wollmer, P., & Elmståhl, S.
(2019). Relative and absolute lung function change in a general population
aged 60-102 years. European Respiratory Journal, 53(3), 1701812.
https://doi.org/10.1183/13993003.01812-2017
(https://doi.org/10.1183/13993003.01812-2017)

 4  (#_ftnref4) Dempsey, T. M., Payne, S., Sangaralingham, L., Yao, X., Shah,
N. D., & Limper, A. H. (2021). Adoption of the antifibrotic medications
pirfenidone and nintedanib for patients with idiopathic pulmonary fibrosis.
Annals of the American Thoracic Society, 18(7), 1121-1128.

 5  (#_ftnref5) Esbriet peak sales (2020) per Roche 2021 Financial Results
& Ofev peak sales (2024) per Boehringer Ingelheim 2024 Financial Results.
Ofev sales include those for all approved indications - IPF, PF-ILD, and
systemic sclerosis-associated interstitial lung disease (SSc-ILD).

 6  (#_ftnref6) 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
(https://doi.org/10.18553/jmcp.2017.23.3-b.s17)

This information is provided by Reach, the non-regulatory press release distribution service of RNS, part of the London Stock Exchange. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact
rns@lseg.com (mailto:rns@lseg.com)
 or visit
www.rns.com (http://www.rns.com/)
.

RNS may use your IP address to confirm compliance with the terms and conditions, to analyse how you engage with the information contained in this communication, and to share such analysis on an anonymised basis with others as part of our commercial services. For further information about how RNS and the London Stock Exchange use the personal data you provide us, please see our
Privacy Policy (https://www.lseg.com/privacy-and-cookie-policy)
.   END  NRABFLBLEVLEBBQ