RCS - PureTech Health PLC - PRTC's Deupirfenidone Granted FDA & EU ODD for IPF

Puretech HealthAnnouncement

19/02/2026 17:15

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RNS Number : 7309T  PureTech Health PLC  19 February 2026

19 February 2026

 

PureTech Health plc

 

PureTech Announces Orphan Drug Designations Granted by the U.S. Food and Drug
Administration and European Commission for Deupirfenidone (LYT-100) in
Idiopathic Pulmonary Fibrosis

 

Designations provide market exclusivity as well as other development
incentives

 

PureTech Health plc (https://puretechhealth.com/) (Nasdaq: PRTC, LSE: PRTC)
("PureTech" or the "Company"), a hub-and-spoke biotherapeutics company
dedicated to giving life to science and transforming innovation into value,
today announced that the U.S. Food and Drug Administration (FDA) and European
Commission have granted Orphan Drug Designation to deupirfenidone (LYT-100)
for the treatment of idiopathic pulmonary fibrosis (IPF), a rare,
progressive, and fatal lung disease. Deupirfenidone is being advanced by Celea
Therapeutics, a Founded Entity established by PureTech to lead its
late-stage development and potential commercialization.

 

Orphan Drug Designation is intended to support the development of therapies
for rare diseases, defined as conditions affecting fewer than 200,000 people
in the United States or fewer than 5 in 10,000 individuals in the European
Union. These designations provide sponsors with a range of incentives
intended to encourage the development of medicines for diseases with high
unmet medical needs.

 

"Orphan Drug Designation from both the FDA and European Commission underscores
the urgent need for more effective therapies for people living with IPF," said
Robert Lyne, Chief Executive Officer of PureTech Health. "Critically, only a
minority of patients with this progressive and fatal disease have ever been
treated with currently approved therapies, largely due to the tradeoff between
tolerability challenges and modest efficacy. We believe deupirfenidone
represents a potentially transformative option for this underserved population
and is a compelling example of how PureTech's model can advance differentiated
medicines toward meaningful patient impact."

 

"The Phase 2b data for deupirfenidone suggest a new benchmark for efficacy in
IPF, with slowing of lung function decline to a level that more closely
mirrors healthy aging, without compromising tolerability," said Sven Dethlefs,
PhD, Chief Executive Officer of Celea Therapeutics. "Orphan Drug Designation
further validates both the seriousness of this disease and the importance of
advancing our program, which we believe has the potential to redefine
treatment expectations for patients living with IPF."

 

Results from the global Phase 2b randomized, double-blind, active- and
placebo-controlled, dose-ranging ELEVATE IPF trial underscored the
differentiated profile of deupirfenidone. In that trial, participants treated
with deupirfenidone 825 mg three times a day (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 the
FDA-approved dose of pirfenidone 801 mg TID or placebo (-21.5 mL vs. -51.6 mL
vs. -112.5 mL, respectively), with a 91 mL difference between deupirfenidone
825 mg and placebo at 26 weeks (p = 0.02). Following the completion of the
blinded portion of the trial, 90% of trial completers (170 participants)
enrolled in the open-label extension. Those who continued treatment with
deupirfenidone 825 mg TID maintained a robust treatment effect and experienced
an overall FVC decline of -32.8 mL over a 52-week period, 1  (#_ftn1) which is
similar to the expected natural decline in lung function in healthy older
adults over that time (approximately -30.0 mL to -50.0 mL). 2  (#_ftn2)

PureTech's Founded Entity, Celea Therapeutics, intends to finalize financing
in the first half of 2026 to support the initiation of the Phase 3 SURPASS-IPF
trial in the first half of 2026. SURPASS-IPF will compare deupirfenidone 825
mg TID to pirfenidone 801 mg TID in a head-to-head study powered to test for
superiority. Based on feedback from the FDA
(https://news.puretechhealth.com/news-releases/news-release-details/puretech-announces-successful-end-phase-2-meeting-fda)
and other global health authorities, PureTech believes that the results from
this single Phase 3 trial, if successful, and supported by the totality of
data from the overall deupirfenidone development program, could complete the
data package required to support potential registration of deupirfenidone.

 

About Orphan Drug Designation

Orphan Drug Designation is intended to encourage the development of medicines
for rare diseases that affect relatively small patient populations and often
lack effective treatment options. Regulatory authorities such as the U.S. Food
and Drug Administration (FDA) and European Commission provide orphan
designation to improve the feasibility of rare disease drug development
through enhanced regulatory interactions, financial incentives, and, upon
approval, defined periods of market exclusivity.

 

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 next
generation antifibrotic and a deuterated form of pirfenidone, one of three
FDA-approved therapies for IPF. The uptake of and adherence to approved
antifibrotics has historically been limited by a tradeoff between modest
efficacy and tolerability, and only ~25% of people with IPF in the U.S. had
ever received treatment as of 2019. 3  (#_ftn3)

 

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 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. 4  (#_ftn4)

 

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 and is currently a wholly-owned subsidiary of 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.

 

About PureTech Health

PureTech Health is a hub-and-spoke biotherapeutics company dedicated to giving
life to science and transforming innovation into value. We do this through a
proven, capital-efficient R&D model focused on opportunities with
validated pharmacology and untapped potential to address significant patient
needs. This strategy has produced dozens of therapeutic candidates, including
three that have received U.S. FDA approval. By identifying, shaping, and
de-risking these high-conviction assets, and scaling them through dedicated
structures backed by external capital, we accelerate their path to patients
while creating sustainable value for shareholders.

 

For more information, visit www.puretechhealth.com or connect with us on
LinkedIn (https://www.linkedin.com/company/puretech-health/) and 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)

Investor Relations
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)

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

 1  (#_ftnref1) Integrated analysis of double-blind (26 weeks) and initial
open-label extension data from Phase 2b ELEVATE IPF trial as of May 9, 2025,
using 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.

 2  (#_ftnref2) 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)

 3  (#_ftnref3) 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.

 4  (#_ftnref4) 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)

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