Open-Label Ponsegromab Shows Promise in Cancer Cachexia
here’s a breakdown of the key data from the provided text, focusing on the study of ponsegromab and cancer-associated cachexia:
What is the study about?
* Cancer-Associated Cachexia: This is a common and serious complication in cancer patients, characterized by weight loss, muscle wasting, and loss of appetite. Currently, there are no approved drugs to treat it.
* GDF-15 & Ponsegromab: The study investigates ponsegromab, a drug that blocks the action of GDF-15. GDF-15 is believed to play a role in causing cachexia by acting on a receptor in the brain. Ponsegromab prevents this signaling.
Study Design:
* Phase 2 Randomized Trial: The study is a Phase 2 clinical trial, meaning it’s designed to assess the drug’s effectiveness and safety.
* Part A (12 weeks):
* Double-blind, placebo-controlled.
* Patients with NSCLC, pancreatic cancer, or CRC where randomized to receive placebo or ponsegromab (100mg, 200mg, or 400mg) every 4 weeks.
* Primary endpoint: Change in body weight at week 12.
* part B (52 weeks):
* Open-label extension – all patients received ponsegromab 400mg every 4 weeks.
* Evaluated long-term changes in body weight, GDF-15 levels, and safety.
Key Findings (so far):
* Initial Success: In the 12-week double-blind study (Part A), ponsegromab showed improvements in body weight, appetite, symptoms, physical activity, and muscle mass compared to placebo.
* placebo Effect in Extension: Patients initially assigned to placebo in Part A did show some weight improvement during the open-label extension (Part B),but their weight gain was less than those who received ponsegromab throughout the entire study.
* Patient Population: The study included 187 patients randomized to Part A,and 117 entered Part B. Most had advanced cancer (Stage 4) and significant weight loss.
* GDF-15 Levels: Patients had high levels of GDF-15 at the start of the study (median 3581 pg/mL).