PureTech Health announced plans to present groundbreaking results from its Phase 2b ELEVATE IPF trial of deupirfenidone (LYT-100) at the upcoming American Thoracic Society International Conference in San Francisco, taking place May 16-21, 2025. The late-breaking oral presentation will showcase data demonstrating unprecedented efficacy in stabilizing lung function decline in patients with idiopathic pulmonary fibrosis (IPF).
The global, randomized, double-blind trial evaluated 257 participants who received either 550 mg of deupirfenidone, 825 mg of deupirfenidone, 801 mg pirfenidone, or placebo three times daily for 26 weeks. According to the company, deupirfenidone demonstrated the potential to stabilize lung function decline over the treatment period while maintaining favorable safety and tolerability profiles.
"The Phase 2b ELEVATE IPF trial results represent a major advancement for the treatment of IPF," said Bharatt Chowrira, Ph.D., J.D., Chief Executive Officer of PureTech. "Deupirfenidone demonstrated the potential to stabilize lung function decline over 26 weeks as a monotherapy—something not achieved by marketed or investigational IPF therapies, to our knowledge."
A Potential New Standard of Care for IPF
Idiopathic pulmonary fibrosis is a rare, progressive, and fatal lung disease characterized by irreversible scarring of lung tissue. With a median survival of just two to five years following diagnosis, IPF affects more than 230,000 people across the United States and EU5 countries (France, Germany, Italy, Spain, and the United Kingdom).
Currently, only two FDA-approved treatments exist for IPF: pirfenidone and nintedanib. Despite their availability, nearly three out of four people with IPF in the United States have never received either treatment. The current standard-of-care therapies only modestly slow lung function decline, with tolerability issues limiting the ability to achieve higher, potentially more effective doses.
Deupirfenidone, a deuterated form of pirfenidone, aims to overcome these limitations. The Phase 2b trial measured the rate of decline in Forced Vital Capacity (FVC)—a standard measurement in IPF clinical trials that assesses disease progression and predicts mortality—for the combined deupirfenidone arms versus placebo over the 26-week treatment period.
Innovative Trial Design
The ELEVATE IPF trial employed a prespecified Bayesian analysis to assess the primary endpoint, providing a posterior probability of superior efficacy for deupirfenidone compared to placebo. This approach allowed for augmentation of the placebo arm with data from historical IPF trials, enabling a more patient-centric clinical trial design by minimizing the number of participants exposed to placebo—a critical consideration given the progressive and fatal nature of IPF.
Professor Toby Maher, M.D., Ph.D., Director of Interstitial Lung Disease at Keck School of Medicine, University of Southern California, will present the detailed findings on May 20, 2025, at 2:15 PM Pacific Time during the conference session focused on advances in interstitial lung disease and pulmonary hypertension.
Regulatory Path Forward
PureTech is actively planning its regulatory strategy to advance deupirfenidone into Phase 3 development. The company has scheduled a meeting with the U.S. Food and Drug Administration by the end of the third quarter of 2025 to discuss the Phase 2b results and align on a potential registrational pathway.
"The additional data we plan to highlight at ATS provide further confidence in the robust and durable efficacy and favorable tolerability of deupirfenidone," Chowrira added. "We believe deupirfenidone has the potential to set a new standard of care and make a transformative difference for patients living with this devastating disease."
PureTech aims to initiate a Phase 3 trial by the end of 2025 and anticipates providing further guidance later this year following the finalization of the trial design and FDA interactions.
Broader Therapeutic Potential
Beyond IPF, PureTech believes deupirfenidone may address multiple underserved fibrotic diseases, including progressive fibrosing interstitial lung diseases and other fibrotic conditions. This potential for broader application could significantly expand the therapeutic impact of the drug if it continues to demonstrate positive results in future trials.
The upcoming presentation at the ATS International Conference represents a significant milestone in the development of deupirfenidone and potentially offers new hope for patients suffering from IPF, a condition with substantial unmet medical needs despite existing treatments.
For IPF patients and the medical community, these results could signal a meaningful advance in treatment options for a disease that currently has limited effective therapies and poor long-term outcomes.
