Deupirfenidone showed a slower rate of lung function decline compared to existing therapies in a Phase 2b trial for idiopathic pulmonary fibrosis.
RT’s Three Key Takeaways:
- Regulatory Milestones: The Food and Drug Administration (FDA) and European Commission granted Orphan Drug Designation to deupirfenidone for the treatment of idiopathic pulmonary fibrosis.
- Phase 2b Results: Clinical trial data showed that deupirfenidone 825 mg significantly slowed the decline of forced vital capacity compared to both placebo and the current FDA-approved dose of pirfenidone.
- Phase 3 Initiation: A head-to-head superiority study comparing deupirfenidone to pirfenidone is scheduled to begin in the first half of 2026.
The FDA and European Commission have granted Orphan Drug Designation to deupirfenidone (LYT-100) for the treatment of idiopathic pulmonary fibrosis (IPF), according to PureTech Health.
IPF is a rare and progressive lung disease characterized by irreversible scarring of lung tissue. Orphan Drug Designation is provided by regulatory authorities to encourage the development of therapies for rare diseases, which are defined as conditions affecting fewer than 200,000 people in the US or fewer than 5 in 10,000 individuals in the European Union. These designations provide incentives such as financial benefits, enhanced regulatory interactions, and periods of market exclusivity upon approval.
“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, in a news release. “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.”
Clinical Trial Data
The regulatory designations follow results from the global Phase 2b ELEVATE IPF trial. The randomized, double-blind study evaluated the efficacy of deupirfenidone 825 mg administered three times a day (TID) against a placebo and the FDA-approved dose of pirfenidone 801 mg TID.
At 26 weeks, participants receiving deupirfenidone 825 mg experienced a slower rate of lung function decline as measured by Forced Vital Capacity (FVC). The decline for the deupirfenidone group was -21.5 mL, compared to -51.6 mL for the pirfenidone group and -112.5 mL for the placebo group. Data from an open-label extension suggested the treatment effect was maintained over 52 weeks, with an overall FVC decline of -32.8 mL. This rate is similar to the natural decline of lung function expected in healthy older adults, which typically ranges from -30.0 mL to -50.0 mL annually.
“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, chief executive officer of Celea Therapeutics, in a news release. “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.”
Future Development
Deupirfenidone is a deuterated form of pirfenidone, which is one of the three current FDA-approved therapies for IPF. While antifibrotics are the standard of care, PureTech reports that only approximately 25% of people with IPF in the US had received treatment as of 2019 due to issues with efficacy and patient adherence.
Celea Therapeutics, the entity established by PureTech to lead the development of the drug, intends to initiate the Phase 3 SURPASS-IPF trial in the first half of 2026. The study will compare deupirfenidone 825 mg TID directly to pirfenidone 801 mg TID in a head-to-head trial designed to test for superiority.
Based on feedback from the FDA and other global authorities, the company believes that successful results from this single Phase 3 trial, combined with existing data, could support the registration of deupirfenidone as a new treatment option for the healthcare industry. Beyond IPF, deupirfenidone is also being explored for other fibrotic conditions, including progressive fibrosing interstitial lung diseases.
