PureTech Health plc (Nasdaq: PRTC, LSE: PRTC), a clinical-stage biotherapeutics company, today announced positive results from the Phase 2b ELEVATE IPF trial. The study evaluated deupirfenidone (LYT-100) in patients with idiopathic pulmonary fibrosis (IPF). In this randomized, double-blind, placebo-controlled trial, patients were administered two doses of deupirfenidone (550 mg and 825 mg) three times daily (TID) for 26 weeks.
Dr. Toby Maher, lead investigator, highlighted that deupirfenidone 825 mg TID significantly reduced lung function decline compared to placebo, achieving near-physiologic levels. The treatment demonstrated an effect size 50% greater than that of pirfenidone, the current standard of care. The trial met its primary endpoint, showing a 98.5% probability of superiority for deupirfenidone in slowing lung function decline, measured by forced vital capacity (FVC) at 26 weeks. Additionally, a dose-dependent response was observed.
The results showed that deupirfenidone 825 mg TID achieved a statistically significant reduction in FVC decline compared to placebo (-21.5 mL vs. -112.5 mL; p=0.02), representing an 80.9% improvement. Pirfenidone 801 mg TID showed a 54.1% improvement (-51.6 mL vs. -112.5 mL). The secondary endpoint, which assessed FVC percent predicted (FVCpp), also showed significant benefit with deupirfenidone 825 mg TID (-0.43% vs. -3.43%; p=0.01).
Both doses of deupirfenidone were well-tolerated. Gastrointestinal (GI) side effects were lower with deupirfenidone 825 mg TID compared to pirfenidone, including reduced incidence of nausea, dyspepsia, diarrhea, and constipation. Abdominal pain was the only GI issue more common with deupirfenidone (14.1% vs. 7.9%). No deaths were attributed to treatment, with five deaths in the pirfenidone arm and one in each of the deupirfenidone arms.
A total of 170 patients (more than 90%) opted to continue in an open-label extension study of deupirfenidone, which showed consistent tolerability and promising long-term results.
Dr. Eric Elenko, President of PureTech, expressed satisfaction with the trial’s results, emphasizing the favorable tolerability and enhanced efficacy of deupirfenidone, especially at the higher dose. CEO Dr. Bharatt Chowrira added that these results, if supported by Phase 3 trials, could represent a major advancement in IPF treatment.
PureTech plans to discuss the Phase 2b findings with regulatory authorities to determine the next steps. Further data will be presented at upcoming scientific meetings.
About the ELEVATE IPF Trial
The Phase 2b ELEVATE IPF trial assessed deupirfenidone’s efficacy and safety in 257 IPF patients. The primary endpoint was the rate of decline in FVC over 26 weeks. FVC is a key measure of lung function and disease progression in IPF.
About Deupirfenidone
Deupirfenidone is a deuterated version of pirfenidone, aimed at improving tolerability while maintaining efficacy for IPF treatment.
About IPF
IPF is a rare and fatal lung disease with limited treatment options. Despite available therapies, adherence is often low due to side effects. Deupirfenidone aims to offer a more effective and tolerable alternative.
About PureTech Health
PureTech is a clinical-stage biotherapeutics company focused on developing novel therapies to address serious diseases. The company has advanced a diverse pipeline of therapeutic candidates, including those for IPF.
