Pila Pharma AB has announced the selection of Cambridge University Hospital as the primary clinical trial site for its PP-CT03 study, which will investigate XEN-D0501, a TRPV1 receptor antagonist, in patients with type 2 diabetes and obesity. Professor Mark Evans, a scientific advisor to Pila Pharma, will lead the study as the principal investigator. The company also plans to seek scientific advice from the UK Medicines and Healthcare products Regulatory Agency (MHRA) to refine the trial design.
Optimizing PP-CT03 Study Design
Pila Pharma aims to strengthen the PP-CT03 study design to enhance its potential for providing clear input into the development and regulatory plan for XEN-D0501 in treating diabetes and obesity. Seeking scientific advice from the UK MHRA is a proactive step to ensure the study meets regulatory standards and increases the likelihood of a smooth regulatory approval process.
Professor Evans stated, "I'm excited to be working with PILA PHARMA and colleagues on planning a study to help determine the optimal dosing strategy for XEN-D0501. As part of this planning, we will take advantage of the opportunity to take scientific advice from the UK MHRA about the best scientific design for our planned study."
Strategic Trial Enhancements
The decision to conduct the trial at a hospital site addresses previous internal safety concerns related to non-hospital settings. Dorte X. Gram, CSO of Pila Pharma, commented, "I'm very glad that we could quickly adapt our plans to conduct PP-CT03 in a prime hospital setting with our long-term scientific advisor Professor Evans. Following discussions with him and his team, we agree on the benefit of first asking for regulatory scientific advice, despite the subsequent delay of study timelines. The benefit, as we see it, will be that we can 'hit the nail the first time', meaning that we will increase our chances of straight regulatory approval of the trial application following submission."
The company anticipates receiving scientific feedback from the MHRA within 30 workdays from the scientific advice meeting in Q1 2025, after which clinical trial application submission plans will resume.
Focus on Functional Weight Loss
CEO of Pila Pharma, Gustav H. Gram, highlighted the importance of exploring potential extra tests and measurements available in the hospital setting. These measurements could provide more detailed and higher quality data regarding the mechanism and its potentially "functional weight loss properties," specifically whether body weight loss is primarily fat-mass or muscle-mass. This focus aligns with the increasing interest in understanding the composition of weight loss, as confirmed by recent discussions at Obesity Week® in the United States.
About XEN-D0501
XEN-D0501 is a selective, synthetic, potent small molecule TRPV1 antagonist. Previous phase 2a clinical trials (PP-CT01 and PP-CT02) have demonstrated that XEN-D0501 is well-tolerated in individuals with obesity and type 2 diabetes. The PP-CT02 trial also showed that XEN-D0501 (administered as 4 mg twice daily for 28 days) significantly enhanced the endogenous insulin response to oral glucose compared to placebo. Furthermore, ANP, a cardiovascular biomarker for heart failure, was statistically significantly reduced.
About Diabetes and Obesity
Diabetes affects over 537 million people globally, with type 2 diabetes accounting for approximately 90% of cases. Obesity is an even larger pandemic, expected to affect over 1 billion people by 2025. Both conditions present significant unmet medical needs, driving the demand for new and effective treatments.
The objective of the PP-CT03 study is to identify the maximum tolerable dose of XEN-D0501 in people living with obesity and type 2 diabetes and to evaluate the safety profile following 3 months of chronic treatment. In addition to the safety assessment, PP-CT03 will include sufficient participants to allow for efficacy readouts on the reduction of body weight.
