Founder, Inventor, CSO and Chairman of PILA PHARMA, Dr. Dorte X. Gram presents the short PILA PHARMA story and rationale behind treatment of metabolic conditions with a #TRPV1 / #Capsaicin receptor antagonist, to the DNSG 2024 Symposium, the 41st International Symposium on Diabetes and Nutrition, which took place in Uppsala, Sweden on June 27th – 30th, 2024.
This short presentation is done in English.
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Pila Pharma is a clinical stage, TRPV1-receptor specialised company developing novel treatments for #Obesity, Type-2 diabetes and also obtained an FDA orphan drug designation for the rare-disease #Erythromelalgia.
Pila Pharma aim to develop XEN-D0501, a TRPV1 antagonist, as a novel treatment of #diabetes and other #cardiometabolic related disorders.
Furthermore the company is evaluating the molecule for treatment of pain in the rare disease Erythromelalgia for which the company was awarded Orphan Drug Designation by the US FDA in 2021.
Other potential diseases with a neuro-inflammatory background are being evaluated as additional indications.
The molecule, XEN-D0501, appears to be safe in healthy volunteers as well as in patients (previously been in trials for overactive bladder disease, chronic cough and type 2 diabetes) and has been shown to significantly improve insulin secretion in people with type-2 diabetes following 1 month oral treatment with XEN-D0501, 4 mg BI-Daily.
In order to determine effects over more than 1 month, a 13 week toxicology programme has recently been conducted in rodents and non-rodents, without clinical adverse events.
Furthermore, good data has been shown on ANP, a cardiovascular biomarker for heart failure. A highly statistically significant reduction which indicates a strong cardio protective benefit of XEN-D0501.
Thus CVD is also of great interest in the coming clinical trial.
Current plan for the diabetes/obesity project is to move on with a small dose-finding study in overweight type-2 diabetic patients to determine the best dose level for a phase 2b. It’s aimed to also observe various key endpoints including weight loss, energy burn, and cardiovascular markers.
This will be followed by a larger phase 2b trial in approx. 300 patients.
Upon successful trial results PILA PHARMA have already seen and expect significant Pharma interest in both partnership/acquisition.
For the rare-disease project, first step is to conduct a proof of concept study in fewer patients, subsequently seek european orphan drug status and then move on to a combined phase 2/3 adaptive design confirmatory registration trial.
We aspire to be a clinical late-stage company making strides into the ever-increasing obesity/diabetes space with real potential to improve the #healthspan for a large proportion of the global population.