Anti-Fibrotic Oral Treatment for IPF Showing Early Promise in Phase 2 Study

Treatment with PBI-4050 may be a safe and effective therapy for patients with idiopathic pulmonary fibrosis (IPF), according to preliminary results from a Phase 2 clinical trial developed by ProMetic Life Sciences.

The study (NCT02538536) has enrolled 40 IPF patients at six different sites in Canada to investigate the safety, tolerability, and efficacy of PBI-4050 on lung function and IPF disease progression.

Preliminary results cover responses observed in the first 30 patients to have completed 12 weeks of treatment. Results found that PBI-4050 was safe and well-tolerated, and showed evidence of clinical effectiveness either alone or in combination with other IPF drugs, such as Ofev (nintedanib) and Esbriet (pirfenidone).

“We are very pleased with the results to date from this study as they provide us with very important data points relating to efficacy and safety,” said John Moran, chief medical officer of ProMetic, in a news release. “There is early evidence of efficacy in the patients treated with PBI-4050 alone and in those treated with PBI-4050 in combination with one of the commercially available drugs. This [evidence] compares favourably to that of the [Esbriet] or [Ofev] treatment reported in the ASCEND trial and in the two INPULSIS trials, respectively.”

The Phase 3 ASCEND (NCT01366209) and INPULSIS (INPULSIS-1, NCT01335464, and INPULSIS-2, NCT01335477) trials investigated the effectiveness of Esbriet and Ofev, respectively, in treating IPF patients. In these trials, after 13 weeks, IPF patients receiving treatment showed a lesser decline in forced vital capacity (FVC; a measure of lung function) than patients given placebo. In the PBI-4050 study, in comparison, IPF patients treated for 12 weeks with the investigational drug, alone or in combination with Ofev/Esbriet, showed a slight improvement in FVC.

“Further good news is the fact that PBI-4050 is very well tolerated by IPF patients whether used alone or in combination with either nintedanib or pirfenidone,” Moran added.

No patients under PBI-4050 treatment experienced a decline equal to or greater than 10 percent in their FVC during the 12 weeks of treatment, or death, the company reported. In contrast, a 10 percent FVC decline or worse, or death, was reported after the 13 weeks of treatment in 2.5 percent of ASCEND trial patients given Esbriet.

“As mentioned above, we use the information generated in these three large trials to help us benchmark PBI-4050’s performance whether used alone or in combination with the commercial agents,” said Joe Parker, senior director of Clinical Development at ProMetic. “IPF is a terrible condition where patients’ lung function keeps declining even when treated by pirfenidone or nintedanib. Our preliminary results are quite encouraging and will help us define the next steps for our promising lead drug candidate PBI-4050.”

PBI-4050 is an experimental oral drug designed to treat fibrosis, a process characterized by the replacement of normal tissue by fibrotic scar tissue, which affects the workings of several organs, including the lungs.