– Plans for Phase 3 Program Presented at National Kidney Foundation’s Annual Spring Clinical Meeting –
Research Triangle Park, N.C., April 7, 2022 – Pathalys Pharma, Inc., a private, late-stage biopharma company committed to the development and commercialization of a range of best-in-class therapies for patients with end stage kidney disease (ESKD), today announced plans for a phase 3 program to support the application of upacicalcet (designated PLS240 for clinical trials), globally. Pathalys holds exclusive rights for the development and commercialization of upacicalcet, a novel calcimimetic, worldwide outside of Japan and Asia.
“This is a very exciting time for Pathalys as we set out to further understand the full clinical potential of upacicalcet in patients with ESKD through two phase 3 studies that will form the basis for our applications for approval,” said Dr. Theodore Danoff, Chief Medical Officer and SVP, Clinical Development at Pathalys. “Upacicalcet is a novel calcimimetic that performed exceptionally well in phase 3 studies in Japan where it was recently approved for the treatment of SHPT in patients on dialysis. We look forward to the opportunity to demonstrate its effectiveness for patients in the US and other markets globally.”
In a poster presented today at the National Kidney Foundation’s Annual Spring Clinical Meeting (SCM22), the company disclosed its plans to initiate two phase 3 clinical trials in 2022 to evaluate upacicalcet as a treatment alternative to currently available calcimimetics.
The two identically designed phase 3 clinical trials will assess the efficacy of upacicalcet in reducing intact parathyroid hormone (iPTH) by 30 percent or more in participants with secondary hyperparathyroidism (SHPT) and currently on hemodialysis.
Each trial will randomize approximately 375 patients (2:1) to upacicalcet or placebo. Eligible patients will be adults receiving in-center hemodialysis three times weekly and whose iPTH values are 450 pg/mL or greater. Upacicalcet will be titrated based on iPTH and serum corrected calcium (cCa) levels with dose steps between 25 and 300 µg as a bolus IV injection given at the end of a dialysis session. Upacicalcet starting dose will be 50 µg if cCa is 9.0 mg/dL or greater or 25 µg if cCa is below 9.0 mg/dL. Doses will be titrated to maintain iPTH in the range of 150-300 pg/mL.
Japanese Approval and Clinical Trial:
Upacicalcet was approved last year in Japan (under the brand name UPASITA®) based in part on a randomized, double-blind, placebo-controlled phase 3 study in hemodialysis patients with SHPT. The study evaluated upacicalcet when administered three times weekly with a starting dose of 25 or 50 μg IV based on cCa levels and was titrated from 25 to 300 μg over 24 weeks.
The primary endpoint was the proportion of patients with a mean iPTH between 60 and 240 pg/mL over weeks 22 to 24 and was achieved in 69 of 103 patients (67.0 percent) in the upacicalcet group and 4 of 50 patients (8.0 percent) in the placebo group (difference of 59.0 percent, p<0.001).
A 30 percent or greater reduction in mean iPTH over weeks 22 to 24 was achieved by 80.6 percent in the upacicalcet group compared to 8.0 percent in the placebo group (difference of 72.6 percent; p<0.001).
Upper gastrointestinal disorder adverse drug reactions (ADRs) were reported in two of 103 patients (1.9 percent) in the upacicalcet group and in three of 50 patients (6.0 percent) in the placebo group. The incidence of calcium less than 7.5 mg/dL was low with 2 events (1.9 percent).
About Pathalys Pharma, Inc.
Pathalys Pharma, Inc. is a private, late-stage clinical biopharmaceutical company committed to the development of multiple advanced therapeutics that address unmet needs in the management of ESKD. Pathalys’ initial asset is upacicalcet, a novel calcimimetic with the potential to improve the treatment of SHPT in hemodialysis patients. Beyond upacicalcet, Pathalys continues to identify other high priority needs and potential solutions for patients with ESKD. Pathalys is headquartered in Research Triangle Park, North Carolina.
For more information about Pathalys, please visit www.pathalys.com.