An investigative drug developed by Kadmon called KD025 was shown to be safe and effective to prevent lung function decline in patients with idiopathic pulmonary fibrosis (IPF), according to Phase 2 trial data.
The ongoing IPF trial (NCT02688647) is expected to include about 36 IPF patients who have previously been treated with Esbriet (pirfenidone) or Ofev (nintedanib), or both. Participants were randomized to receive 400 mg KD025 orally once daily or standard-of-care treatment.
Data collected after 24 weeks of 20 KD025-treated patients, and nine patients receiving standard of care, showed that the investigational drug could effectively improve lung function. Treatment with KD025 was seen to prevent the median decline of forced vital capacity (FVC; a measure of lung function) by 73%, and the median decline in FVC predicted compared to standard of care by 50%.
In addition, KD025 was found to prevent IPF progression as determined by improved annualized FVC decline experienced by the patients compared to the previous year.
In general, the investigative drug was well-tolerated, with no serious drug-related adverse events reported. Approximately 90% of participants who received KD025 during the 24 weeks of the trial decided to continue KD025 treatment beyond this time period.
“In this proof-of-concept trial, KD025 has demonstrated clinical activity at 24 weeks and was well tolerated, with no apparent safety signals, potentially offering a new option for patients with IPF,” Kevin F. Gibson, MD, professor and medical director at the Dorothy P. and Richard P. Simmons Center for Interstitial Lung Disease at University of Pittsburgh Medical Center and lead investigator of the trial, said in a press release.
KD025 is a selective inhibitor of the ROCK2 enzyme. Preclinical studies have shown that blocking ROCK2 activity can modulate fibrotic processes and abnormal immune responses, as the enzyme is known to regulate the molecular pathway involved in wound healing.
“We are pleased with today’s results, which demonstrate the activity and tolerability of KD025 in IPF, including in patients who have received prior therapy with approved agents,” said Harlan W. Waksal, MD, president and CEO at Kadmon.
“These findings support the therapeutic potential of ROCK inhibition in IPF and further validate Kadmon’s ROCK inhibitor platform, which is being applied across programs in fibrotic diseases as well as inflammatory diseases,” Waksal added.
The company is planning to present top-line data of the IPF Phase 2 trial at the American Thoracic Society (ATS) International Conference May 18-23 in San Diego, California.
Kadmon is currently evaluating the safety and effectiveness of it’s drug candidate in three Phase 2 trials for the treatment of IPF; graft versus host disease (NCT02841995); and psoriasis (NCT02852967).