PureTech Reports Publication of Phase 2b ELEVATE IPF Trial Results in Leading Respiratory Medicine Journal
PureTech Health plc, a clinical-stage biotherapeutics company focused on translating cutting-edge science into transformative therapies, has announced the publication of results from its Phase 2b ELEVATE IPF clinical trial in The American Journal of Respiratory and Critical Care Medicine. The study evaluated deupirfenidone, an investigational therapy, for the treatment of Idiopathic Pulmonary Fibrosis (IPF), a chronic and progressive interstitial lung disease characterized by irreversible scarring of lung tissue and declining respiratory function.
The publication marks an important milestone for PureTech Health and its affiliated entity, Celea Therapeutics, as it underscores both the scientific rigor of the ELEVATE IPF trial and the potential of deupirfenidone to redefine treatment expectations in a disease area where therapeutic options remain limited. Importantly, the findings from this Phase 2b study have directly informed the design of the upcoming Phase 3 SURPASS-IPF trial, which is expected to commence in the first half of 2026, pending completion of financing efforts.
Advancing a Next-Generation Antifibrotic Therapy
Idiopathic pulmonary fibrosis remains a devastating condition with a poor prognosis, often leading to progressive respiratory failure. Current standard-of-care treatments, including pirfenidone, can slow disease progression but are not curative and provide only modest preservation of lung function. Against this backdrop, the development of deupirfenidone represents an effort to improve both efficacy and tolerability compared to existing therapies.
The ELEVATE IPF trial stands out in the clinical research landscape because it incorporated an active comparator arm using pirfenidone, allowing investigators to assess the investigational therapy within a real-world clinical context. According to Toby Maher, Professor of Medicine and Director of Interstitial Lung Disease at the Keck School of Medicine at the University of Southern California, and lead author of the study, this trial design provides valuable insights into how deupirfenidone performs relative to an established therapy.
Maher emphasized that such direct comparisons are rare in IPF research and enhance confidence in interpreting both efficacy and safety outcomes. He further noted that the magnitude of treatment effect observed in the study raises the possibility that lung function decline in IPF patients could be slowed to levels approaching those seen in normal aging—a prospect that, if confirmed in Phase 3 trials, could significantly alter the treatment paradigm.
Key Efficacy Outcomes Demonstrate Robust Clinical Benefit
The ELEVATE IPF trial successfully met its primary and key secondary endpoints, demonstrating strong evidence of efficacy for deupirfenidone. Using a prespecified Bayesian statistical framework, the therapy achieved a 98.5% posterior probability of superiority over placebo in slowing the decline of forced vital capacity (FVC), a critical measure of lung function. Additionally, a 99.6% posterior probability of superiority was observed for forced vital capacity percent predicted (FVCpp), reinforcing the consistency of the treatment effect.
At 26 weeks, patients receiving deupirfenidone 825 mg three times daily (TID) as monotherapy experienced a statistically significant and clinically meaningful reduction in lung function decline compared with placebo. Specifically, the adjusted difference in mean FVC decline was 91 mL (p=0.02), while secondary analyses of FVCpp also demonstrated statistically significant improvements (p=0.01).
One of the most compelling findings from the study was the extent to which deupirfenidone appeared to normalize the rate of lung function decline. Patients treated with deupirfenidone exhibited a mean FVC decline of just -21.5 mL over 26 weeks, a rate that closely approximates the physiological decline expected in healthy older adults. This contrasts sharply with the more rapid deterioration typically observed in untreated IPF patients.
Further insights from an ongoing open-label extension of the ELEVATE IPF study—though not included in the AJRCCM publication—suggest that this benefit may be sustained over longer durations. Data indicate that over 52 weeks, patients receiving deupirfenidone experienced an FVC decline of approximately -32.8 mL, again aligning with the expected range for normal aging. These findings suggest the potential for durable disease modification, a highly sought-after goal in IPF treatment.
Delaying Disease Progression
In addition to preserving lung function, deupirfenidone demonstrated a significant impact on delaying disease progression. The study defined progression as an absolute decline in FVCpp of at least 5% or death within 26 weeks. Patients treated with deupirfenidone showed a substantially reduced risk of progression compared with those receiving placebo, with a hazard ratio of 0.439 (p=0.0023).
This reduction in progression risk highlights the therapy’s potential to not only slow physiological decline but also improve clinically meaningful outcomes that directly affect patient quality of life and survival. Delaying disease progression is particularly important in IPF, where even small declines in lung function can translate into significant morbidity.
Favorable Pharmacokinetics and Tolerability Profile
A key differentiator for deupirfenidone lies in its pharmacokinetic profile. Data from the ELEVATE IPF trial show that the 825 mg TID dosing regimen results in approximately 50% greater drug exposure compared with pirfenidone at its highest approved dose of 801 mg TID. This increased exposure is believed to contribute to the enhanced efficacy observed in the study.
Crucially, the improved exposure does not come at the expense of tolerability. The overall incidence of adverse events (AEs) in patients receiving deupirfenidone was comparable to that observed in the pirfenidone arm (85.9% versus 84.1%, respectively). Most AEs were mild to moderate in severity, and treatment discontinuation rates were low.
Patient adherence also remained high, with 78.1% of participants in the deupirfenidone group remaining on therapy for the full 26-week study period. This is comparable to the 80.0% adherence rate observed in the placebo group, suggesting that the therapy is well tolerated over extended use.
Together, these findings indicate that deupirfenidone may offer a favorable balance between efficacy and safety, addressing a key limitation of current antifibrotic therapies.
Paving the Way for Phase 3 Development
The encouraging results from the ELEVATE IPF trial have laid the groundwork for the Phase 3 SURPASS-IPF study, which will directly compare deupirfenidone 825 mg TID monotherapy with pirfenidone 801 mg TID in a head-to-head trial designed to test for superiority. This ambitious study aims to provide definitive evidence of clinical benefit and potentially establish deupirfenidone as a new standard of care in IPF treatment.
Sven Dethlefs, Chief Executive Officer of Celea Therapeutics, highlighted the importance of the AJRCCM publication in validating the trial’s design and execution. He noted that the results provide a strong scientific and clinical foundation for advancing deupirfenidone into late-stage development.
Dethlefs emphasized that the company’s goal is to build on these promising findings to deliver a next-generation antifibrotic therapy capable of meaningfully improving outcomes for patients living with IPF. He also confirmed that Celea Therapeutics is actively working to secure the necessary financing to initiate the Phase 3 trial in the first half of 2026.
Broader Implications for IPF Treatment
The publication of the ELEVATE IPF trial results represents more than just a milestone for PureTech and Celea Therapeutics—it signals a potential shift in the treatment landscape for idiopathic pulmonary fibrosis. By demonstrating the possibility of achieving near-normal rates of lung function decline, deupirfenidone challenges long-standing assumptions about what is achievable in this disease.
If the results are replicated in Phase 3, deupirfenidone could redefine therapeutic goals in IPF, moving beyond modest slowing of disease progression toward meaningful preservation of lung function. This would have profound implications for patients, clinicians, and healthcare systems alike.
Moreover, the trial’s innovative design, including the use of an active comparator and Bayesian statistical methods, may serve as a model for future studies in IPF and other chronic diseases. By providing more robust and clinically relevant data, such approaches can accelerate the development of effective therapies and improve decision-making in clinical practice.
The Phase 2b ELEVATE IPF trial results, now published in a leading peer-reviewed journal, highlight the promise of deupirfenidone as a potentially transformative therapy for idiopathic pulmonary fibrosis. With strong efficacy data, a favorable safety profile, and the potential to significantly alter disease progression, the therapy is well positioned as it advances into Phase 3 development.
As PureTech Health and Celea Therapeutics prepare for the next stage of clinical evaluation, the medical community will be watching closely. The success of the upcoming SURPASS-IPF trial could mark a turning point in the fight against IPF, offering new hope to patients facing this challenging and life-limiting disease.
About the Phase 2b ELEVATE IPF Trial
The Phase 2b ELEVATE IPF trial was a global, randomized, double-blind, active- and placebo-controlled, dose-ranging trial designed to evaluate the efficacy, tolerability, safety, and dosing regimen of deupirfenidone (LYT-100) in patients with IPF compared to placebo. 257 participants were randomized in a ratio of 1:1:1:1 to receive either 550 mg of deupirfenidone, 825 mg of deupirfenidone, 801 mg of pirfenidone or placebo three times a day (TID) for 26 weeks. Participants who completed the trial had the option to enroll in an open-label extension, which is ongoing.
The primary endpoint of the trial was the rate of decline in Forced Vital Capacity (FVC) for the combined deupirfenidone arms versus placebo over the 26-week treatment period. FVC is a measure of the maximum amount of air (in mL) that an individual can forcibly exhale after fully inhaling. It is a standard measurement in clinical trials for IPF and is used to assess disease progression as well as to predict mortality.
A prespecified Bayesian analysis was utilized to assess the primary endpoint and provided a posterior probability, which is the probability of superior efficacy for deupirfenidone compared to placebo. This also allowed for augmentation of the placebo arm with placebo data from historical IPF trials. This approach enabled a more patient-centric clinical trial design by minimizing the number of trial participants exposed to placebo – a key consideration since IPF is progressive and fatal – while delivering a robust, placebo-controlled dataset.
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 20194.
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 years5, and currently there is no cure.
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.
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