Camurus' POSITANO Study Reveals Promising Effects of CAM2029 for Patients with Polycystic Liver Disease
CAM2029 Shines in Latest POSITANO Study
Camurus AB has recently shared groundbreaking results from its Phase 2b POSITANO study, focusing on CAM2029, a subcutaneous depot formulation of octreotide. This clinical trial aims to assess the efficacy and safety of CAM2029 in individuals diagnosed with symptomatic Polycystic Liver Disease (PLD), a rare genetic disorder characterized by the progressive development of cysts within the liver. This condition significantly impacts patients' quality of life, often resulting in debilitating symptoms.
The POSITANO study was a well-structured, 12-month, randomized, double-blind, placebo-controlled trial. It involved 71 participants who were experiencing symptomatic PLD. In the trial, participants were assigned to receive one of two dosing regimens of CAM2029 or a placebo in a 1:1 ratio. It is particularly exciting to note that the study demonstrated a statistically significant reduction in both the height-adjusted liver volume (htTLV) and total liver cyst volume when compared to the placebo group. Specifically, the results showcased a 4.3% reduction in htTLV (p=0.044) and an 8.7% reduction in total liver cyst volume (p=0.016) at the end of the treatment period.
Dr. Joost P.H. Drenth, Professor of Hepatology at the Amsterdam University Medical Center and the lead investigator of the POSITANO study, stated, "The results indicate that CAM2029 significantly reduces liver and cyst volume, with concurrent improvements in patient-reported outcomes. This is promising news for the large population affected by PLD, where treatment options are critically limited."
Understanding Polycystic Liver Disease
PLD is a rare genetic condition that can lead to significant challenges for patients. Approximately 37,000 individuals are affected in the US and EU, with a notable majority being women. Without any currently approved pharmacological treatments, patients often endure severe symptoms including abdominal pain, discomfort, and even limitations in mobility.
In terms of demographic factors, age and gender have been found to play critical roles in the severity of PLD, with women being disproportionately affected. Many patients report symptoms and are diagnosed in their 30s, following an observable increase in waist size along with other distressing symptoms. The POSITANO study's positive outcomes, especially considering the unmet medical needs of this patient population, are significant.
Safety and Tolerability
CAM2029 has displayed a well-tolerated safety profile, consistent with the known safety data for established injectable somatostatin receptor ligands such as octreotide and lanreotide. The most frequent side effects reported during the study included mild to moderate gastrointestinal disturbances and injection site reactions. Additionally, no new or unexpected safety findings were observed, reinforcing the potential of CAM2029 as a viable treatment option.
Fredrik Tiberg, President and CEO of Camurus, noted, "The results from the POSITANO trial motivate us to engage with regulatory authorities regarding a Phase 3 study, aimed at solidifying our findings and bringing CAM2029 to market for those suffering from polycystic liver disease."
Next Steps
Following the randomized segment of the POSITANO study, an open extension phase is in place to allow ongoing treatment for patients with CAM2029. This extension will help gather further long-term safety and efficacy data. Meanwhile, Camurus is actively seeking to discuss the design of a confirmatory Phase 3 study with regulatory bodies in both the US and Europe.
In conclusion, the promising findings from the POSITANO study could herald a new era for patients with polycystic liver disease, providing hope and potential relief in what has been a challenging medical landscape without effective treatment options for many years.