Pathalys Pharma develops upacicalcet, a novel intravenous calcimimetic designed to treat secondary hyperparathyroidism in patients with late-stage chronic kidney disease. This therapeutic addresses the critical issue of elevated parathyroid hormone levels resulting from kidney failure, which can lead to severe complications in affected patients.
Funding
$266M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
TCFounders
Product
Problem
Patients with late-stage chronic kidney disease often develop secondary hyperparathyroidism (SHPT), a condition where the parathyroid glands overproduce parathyroid hormone (PTH) due to the kidneys' inability to produce sufficient vitamin D or remove phosphorus. Elevated PTH levels can lead to severe complications, including mineral and bone disorders, cardiovascular issues, and reduced quality of life. Current treatments for SHPT may have tolerability issues and can lead to adverse events such as upper gastrointestinal issues and hypocalcemia.
Solution
Pathalys Pharma is developing upacicalcet, a novel intravenous calcimimetic, as a potentially improved treatment option for SHPT in patients with end-stage kidney disease undergoing hemodialysis. Upacicalcet mimics the action of calcium on tissues by activating calcium-sensing receptors, effectively reducing PTH levels. Administered intravenously at the end of hemodialysis via a pre-filled syringe, upacicalcet aims to provide better control of PTH levels while minimizing the risk of upper gastrointestinal adverse events and hypocalcemia associated with existing calcimimetics. The ongoing Phase 3 clinical trials are designed to assess the efficacy of upacicalcet in reducing intact parathyroid hormone (iPTH) levels by 30 percent or more in participants with SHPT.
Target Audience
The primary target audience includes patients with end-stage kidney disease (ESKD) on hemodialysis who are suffering from secondary hyperparathyroidism (SHPT), as well as the nephrologists and healthcare providers who manage their care.
Features
- Intravenous administration at the end of hemodialysis session via pre-filled syringe
- Novel calcimimetic that mimics calcium's action on tissues, activating calcium-sensing receptors
- Demonstrated effectiveness in reducing PTH levels in Phase 3 studies conducted in Japan
- Designed to provide better control of parathyroid hormone levels in SHPT
- May lower the risk of upper gastrointestinal adverse events compared to current calcimimetics
- May lower the risk of hypocalcemia compared to current calcimimetics
- Phase 3 clinical trials utilizing a 2:1 randomization to upacicalcet or placebo
- Doses are titrated to maintain iPTH in the range of 150-300 pg/mL