Lipella Pharmaceuticals to Advance Lead Candidate to Phase 2b After FDA Type-C Meeting Guidance

Lipella Pharmaceuticals Inc., a clinical-stage biotechnology company, has announced that it has received guidance from the U.S. Food & Drug Administration (FDA) concerning its LP-10 (liposomal tacrolimus) clinical program. The company plans to begin a Phase 2b clinical trial of LP-10 as a treatment for hemorrhagic cystitis in the latter half of 2024, targeting approximately 36 patients.

The FDA has preliminarily agreed with Lipella's trial design, which includes the study’s goals, safety measures, control group monitoring, dosing protocol, primary efficacy endpoint, and sample size considerations. Dr. Michael Chancellor, Chief Medical Officer of Lipella, expressed confidence in the study's design, stating that this clarity will help the company meet its regulatory objectives and significantly enhance its value proposition.

LP-10 is a liposomal formulation of tacrolimus administered intravesically. It aims to treat moderate to severe hemorrhagic cystitis, a condition characterized by significant urinary blood loss often associated with cancer treatments. In a Phase 2a study, LP-10 showed promising results in 13 patients, leading to improvements in urinary symptoms, including reduced hematuria, less cystoscopic bleeding, and fewer ulceration sites. The pharmacokinetic analysis demonstrated that LP-10 has a very short duration of systemic uptake.

Hemorrhagic cystitis presents a severe challenge, especially for those undergoing certain cancer therapies. Currently, there are no FDA-approved treatments for moderate to severe cases of this condition, which is both highly morbid and potentially fatal. Lipella is focusing on this patient group, which numbers around 60,000 annually.

Lipella Pharmaceuticals is dedicated to developing new drugs by reformulating active ingredients found in existing generic drugs, optimizing these reformulations for new applications. The company focuses on diseases with significant, unaddressed morbidity and mortality where no approved drug therapy currently exists. Notably, Lipella completed its initial public offering in December 2022.

This FDA guidance is a significant milestone for Lipella, allowing the company to advance its clinical trial program for LP-10 with greater confidence. By addressing the unmet needs in hemorrhagic cystitis treatment, Lipella aims to provide a new therapeutic option for patients who currently have limited choices.