Pathalys Pharma and Launch Therapeutics have announced that the first patient has been enrolled in the PATH (PTH Attenuation Trial in Hemodialysis) clinical programme, consisting of two identical phase 3 trials, to assess upacicalcet for the treatment of secondary hyperparathyroidism (SHPT) in end-stage kidney disease (ESKD) patients on haemodialysis.
The PATH clinical trial program is the output of Pathalys’ previously disclosed intent to initiate two identical phase 3 clinical trials to evaluate upacicalcet. Upacicalcet is a novel calcimimetic that performed “exceptionally well”, according to a company press release, in phase 3 studies in Japan, where it was approved in 2021 for the treatment of SHPT in patients on dialysis.
“We have enrolled our first patient for this programme ahead of schedule. With the successful conclusion of this trial, we hope to accelerate upacicalcet toward registration in the USA with the goal of making this therapy available for dialysis patients with SHPT,” said Theodore Danoff, chief medical officer and senior vice president of clinical development at Pathalys. “The advancement of an intravenous calcimimetic like upacicalcet may allow patients on dialysis better control of parathyroid hormone levels in SHPT in a convenient and compliant way while also lowering risk of upper gastrointestinal adverse events and hypocalcemia, which continue to be an issue with current calcimimetics.”
Jessica Kendrick (University of Colorado School of Medicine, Aurora, USA), the principal investigator for the studies, commented: “Advancing upacicalcet will be a very meaningful step for patients and enrolling the first patient ahead of schedule is promising given that the sooner we can dose our first patient, the closer we will be to obtaining the needed data for FDA review and hopefully approval.”
The PATH study program is designed to assess the efficacy of upacicalcet as measured by its ability to reduce intact parathyroid hormone (iPTH) by 30% or more in participants with secondary hyperparathyroidism (SHPT) and currently on haemodialysis.
Each trial will randomise approximately 375 patients (2:1) to upacicalcet or placebo. Eligible patients will be adults receiving in-centre haemodialysis three times weekly and whose iPTH values are 450pg/mL or greater. Upacicalcet will be titrated based on iPTH and serum corrected calcium (cCa) levels with dose steps between 25 and 300 mcg as a bolus IV injection given at the end of a dialysis session. Doses will be titrated to maintain iPTH in the range of 150–300pg/mL.
