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 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
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 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
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
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.
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, please visit www.puretechhealth.com
