Camurus announces positive results from POSITANO phase 2b study of octreotide SC depot

Overview

Camurus, an international, science-led biopharmaceutical company, 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).  

About the condition: polycystic liver disease

• 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.

Comments from the coordinating investigator

• The POSITANO study shows that CAM2029 significantly reduces liver and liver cyst volume compared to placebo while showing improvements in symptoms as assessed by relevant patient reported outcomes”, says Dr Joost P.H. Drenth, Professor of Hepatology, Amsterdam University Medical Center and coordinating investigator for the POSITANO study. 
• There is a high unmet medical need for treatment of patients with polycystic liver disease and the POSITANO study brings hope for our patients.”

The POSITANO trial

• POSITANO enrolled 71 participants with symptomatic PLD randomized to treatment with one out of two dosing regimens of CAM2029 or placebo in a 1:1:1 ratio. 
• The study met the primary endpoint showing a statistically significant relative reduction of the height-adjusted liver volume (htTLV) from baseline to week 53 of 4.3% (p=0.044) for the combined CAM2029 groups compared to placebo. 
• 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.

Words from the CEO: Camurus

• The POSITANO study met the primary endpoint showing that CAM2029 reduces liver and cyst growth and can improve disease symptoms and other outcomes in patients with polycystic liver disease”, says Fredrik Tiberg, Camurus’ president & CEO, CSO. 
• Based on the results, Camurus intends to discuss the design of a confirmatory Phase 3 study with regulatory authorities in the US and Europe.”

CAM2029: safety profile

• 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 diarrhoea, 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 US Food and Drug Administration (FDA) and in the EU by the European Commission.

About Polycystic liver disease

• Polycystic liver disease (PLD) 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 (dyspnoea), indigestion (dyspepsia), gastro-esophageal reflux, and limited mobility. 
• Rare complications are hepatic cyst haemorrhage, 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. 
• There is currently no approved pharmacological treatment for PLD. Clinical studies indicate that somatostatin receptor ligands, e.g., octreotide, can slow down cyst growth, decrease fluid secretion, and reduce the liver volume.

The molecule of focus: 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 tumours (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 GEP-NET (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.

About the company: Camurus

Camurus is an international, science-led biopharmaceutical company committed to developing and commercializing innovative, long-acting medicines for improving the lives of patients with severe and chronic diseases.

Optimize Your trial insights with Clival Database.

Are you exhausted from the uncertainty of trial insights pricing? Clival Database ensures the clarity in the midst of the global scenario for clinical trials to you.

Clival Database is one of the best databases that offers an outstanding number of clinical trial data in terms of 50,000+ molecules and from primary regulatory markets as well as new entrants like Indian and Chinese markets.

With Clival, you get accurate positioning of historical sales data, patent database, company profiling, safety & efficacy, and prediction of launch of new innovative molecules helping you to align your research and driving down the cost.

To add value, we further break down our analytics for you so that improving your operational effectiveness; optimizing your clinical trials; and offering you accurate and high-quality data at lowest possible prices becomes possible.

Elevate your trial success rate with the cutting-edge insights from Clival database.

Check it out today and make more informed sourcing decisions! Learn More!