お知らせ • Jun 20
Camurus Announces Topline Results from the 12-Month, Randomized, Double-Blind, Placebo-Controlled POSITANO Phase 2b Study
Camurus announced topline results from the 12-month, randomized, double-blind, placebo-controlled POSITANO Phase 2b study (NCT05281328), evaluating efficacy and safety of octreotide subcutaneous (SC) depot (CAM2029) in patients with symptomatic polycystic liver disease (PLD). PLD is a rare genetic disorder causing progressive liver cysts, leading to severe symptoms and reduced quality of life. Approximately 37,000 people in the US and EU have PLD, most of whom are women. Currently, no approved treatment exists for PLD. The corresponding relative reduction in total liver cyst volume was 8.7% (p=0.016). Treatment with CAM2029 also resulted in improvements in disease symptoms and other patient and clinical reported outcomes. CAM2029 was well tolerated with a safety profile consistent with the established safety profile of approved injectable somatostatin receptor ligands, octreotide and lanreotide. The most frequently reported adverse effects were diarrhea, and mild to moderate gastrointestinal disturbances and injection site reactions. No new or unexpected safety findings were noted. Detailed results from the POSITANO study will be presented at future scientific meetings and in publications. After completion of the randomized part of POSITANO, the treatment of patients continues with CAM2029 in a 2.5-year, open extension phase of the study where further long-term efficacy and safety data are being collected. CAM2029 has been granted Orphan Drug Designation (ODD) for the treatment of autosomal dominant PLD in the US by the U.S. Food and Drug Administration (FDA) and in the EU by the European Commission. Polycystic liver disease (PL D) is a rare genetic and chronic disorder characterized by progressive growth of fluid-filled cysts in the liver, which can cause severe symptoms such as abdominal pain and discomfort, shortness of breath (dyspnea), indigestion (dyspepsia), gastro-esophageal reflux, and limited mobility. Rare complications are hepatic cyst hemorrhage, infection or rupture. Age and gender contribute to disease severity; increasing age is positively associated with both cyst sizes and numbers, and women are highly overrepresented among symptomatic patients. Most patients with PLD are diagnosed in their 30s after reporting a sudden and accelerated increase of waist width together with PLD-related symptoms.7 There is currently no approved pharmacological treatment for PLD. Clinical studies indicate that somatostatin receptor ligand, e.g., octreotide, can slow down cyst growth, decrease fluid secretion, and reduce the liver volume.9-11. About octreotide SC depot (CAM2029). CAM2029 is a ready-to-use, long-acting subcutaneous depot of octreotide under development for the treatment of three rare disease indications: acromegaly, gastroenteropancreatic neuroendocrine tumors (GEP-NET), and polycystic liver disease (PLD). CAM2029 has been evaluated in a comprehensive clinical program, including five Phase 1 and 2 studies, two Phase 3 studies in acromegaly (ACROINNOVA 1 and 2), an ongoing Phase 3 study in patients with GEPNET (SORENTO), and the Phase 2/3 study in patients with PLD (POSITANO). CAM2029 is designed for enhanced octreotide exposure and convenient, once-monthly administration with a prefilled autoinjector pen to facilitate easy self-administration by patients.