PureTech's Deupirfenidone Receives FDA Backing for Phase 3 Trial in Idiopathic Pulmonary Fibrosis
Key Insights
PureTech Health completed a successful end-of-phase-2 meeting with the FDA for deupirfenidone, a deuterated form of pirfenidone, receiving support for advancement into a pivotal phase 3 trial.
The FDA endorsed a streamlined 505(b)(2) regulatory pathway and indicated that successful results from a single phase 3 study could be sufficient to support potential registration.
The phase 3 SURPASS-IPF (search) trial will compare deupirfenidone 825 mg three times daily to pirfenidone 801 mg three times daily in adults with IPF, with initiation planned for the first half of 2026.
PureTech Health announced the successful completion of its end-of-phase-2 meeting with the U.S. Food and Drug Administration (search) regarding deupirfenidone (LYT-100), a next-generation treatment for idiopathic pulmonary fibrosis (search). The FDA provided supportive feedback for advancing the drug candidate into a pivotal phase 3 trial and endorsed the use of a streamlined 505(b)(2) regulatory pathway.
The regulatory milestone positions deupirfenidone, a deuterated form of the approved IPF (search) drug pirfenidone, for potential registration based on results from a single phase 3 study if successful. The drug is being developed by Celea Therapeutics (search), a PureTech-founded entity that expects to finalize financing in early 2026 to support trial initiation.
Phase 3 Trial Design and Timeline
The pivotal phase 3 SURPASS-IPF (search) trial will be a global, randomized, double-blind, head-to-head study comparing deupirfenidone 825 mg three times daily to pirfenidone 801 mg three times daily in adults with IPF who are not receiving background therapy. The primary efficacy endpoint will measure the change from baseline in absolute forced vital capacity (search) at week 52, assessing the superiority of deupirfenidone compared with pirfenidone.
"Our discussion with the FDA was productive and provided helpful feedback on key elements of our Phase 3 program and the overall data expectations for registration," said Sven Dethlefs, Chief Executive Officer of Celea Therapeutics (search). The trial is scheduled to begin in the first half of 2026, following completion of financing arrangements.
Strong Phase 2b Foundation
The regulatory advancement builds on results from the global phase 2b ELEVATE IPF (search) trial, which demonstrated deupirfenidone's potential to stabilize lung function decline. In that study, participants treated with deupirfenidone 825 mg three times daily experienced slower rates of lung function decline compared to those receiving pirfenidone 801 mg three times daily or placebo.
Specifically, the change from baseline in forced vital capacity (search) at 26 weeks was -21.5 mL for deupirfenidone, compared to -51.6 mL for pirfenidone and -112.5 mL for placebo, representing a 91 mL difference between deupirfenidone and placebo. In the open-label extension, more than 90% of participants enrolled, and those continuing deupirfenidone treatment maintained a robust treatment effect with an overall FVC (search) decline of -32.8 mL over 52 weeks.
Addressing Unmet Medical Need
Idiopathic pulmonary fibrosis (search) is a rare, progressive, and fatal lung disease characterized by irreversible scarring of lung tissue that leads to steady decline in lung function. Median survival following diagnosis is estimated to be two to five years, with no current cure available.
The uptake and adherence to approved antifibrotics has historically been limited by modest efficacy and tolerability issues, with only approximately 25% of people with IPF (search) in the U.S. having received treatment as of 2019. Deupirfenidone aims to overcome these limitations by potentially offering improved efficacy while maintaining a favorable safety and tolerability profile.
Market Response and Future Outlook
PureTech shares rose 3.0% to 125.60 pence following the announcement. The company's Founded Entity model enables advancement of promising medicines through dedicated structures backed by external capital, accelerating their path to patients while creating value for shareholders.
Beyond IPF (search), deupirfenidone may also address multiple underserved fibrotic conditions, including progressive fibrosing interstitial lung diseases (search), potentially expanding its therapeutic applications in the future.