#Sweden #Lund #Camurus #CAM2029 #Polycystic Liver Disease

Camurus’ POSITANO Study: A Breakthrough in Treating Polycystic Liver Disease

Camurus, the Swedish biopharmaceutical company, has unveiled exciting topline findings from its Phase 2b study known as POSITANO, assessing the efficacy of CAM2029 in treating patients diagnosed with symptomatic Polycystic Liver Disease (PLD). This year's results underscore the potential for CAM2029 to revolutionize care for this rare condition that affects approximately 37,000 people in the US and Europe, particularly spotlighting the plight of women who are disproportionately impacted.

Polycystic liver disease is characterized by progressive cyst formation within the liver, leading to numerous severe symptoms that significantly impair quality of life. As highlighted by Dr. Joost P.H. Drenth, a hepatology professor involved in the study, the outcomes are both a scientific triumph and bring renewed hope for patients currently without approved treatment options.

Study Design and Findings

The POSITANO trial was a 12-month, randomized, double-blind, placebo-controlled study that included 71 participants. Each subject was assigned to receive either CAM2029 or a placebo in a 1:1 ratio, evaluating the drug's effectiveness in reducing liver and cyst volume. The results were statistically significant; CAM2029 led to an average reduction of 4.3% in height-adjusted total liver volume over the trial duration compared to the placebo group. Additionally, liver cyst volume shrank by 8.7%, proving the treatment's potential to alleviate some of the most distressing symptoms associated with PLD.

The enhancements in patients’ quality of life were evident through improved patient-reported outcomes, suggesting that CAM2029 initiates positive changes not only in physical health parameters but also enhances overall well-being. As Fredrik Tiberg, the President and CEO of Camurus expressed, this study is pivotal as it meets urgent healthcare needs and aligns with Camurus' dedication to innovation in healthcare.

Safety Profile and Future Research

In terms of safety, CAM2029 demonstrated a profile consistent with previous injectable somatostatin receptor ligands. Most noted side effects included mild to moderate gastrointestinal disturbances, with no serious new safety concerns raised during the trial period. Long-term efficacy and safety data collection will continue through an ongoing 2.5-year open extension phase for participants.

Camurus plans to initiate discussions with regulatory authorities in both the US and Europe as they work toward the design of a confirmatory Phase 3 study. The emphasis is not only on scientific advancement but also fulfilling the urgent unmet need for effective PLD treatments.

The Future of PLD Treatment

The landscape of incapacitating diseases like PLD is often marred by the absence of potent treatment options. CAM2029 offers a promising glimmer of hope for patients suffering from this debilitating illness. With support from the FDA and European Commission, which have both designated CAM2029 as an Orphan Drug, the path ahead looks encouraging.

As more results from the POSITANO study move into public forums and detailed publications, the community’s anticipation grows. For now, patients and healthcare providers alike can focus on the strides made through studies like POSITANO, which not only deepen our understanding of polymorphous conditions like PLD but also pave the route for the future of treatment strategies.

About Camurus

Camurus is committed to advancing care that transforms the lives of patients facing chronic conditions by developing long-lasting therapeutic interventions. Utilizing proprietary FluidCrystal® technology, the company has set a course to tackle significant health challenges across various areas, including cancer and endocrine disorders, fostering a vision of innovative healthcare prominent in today’s biopharmaceutical landscape. For further information, please visit Camurus' website.