Lipella Pharmaceuticals Inc. (Nasdaq: "LIPO") ("Lipella," "our, "us" or the "Company") today announced the successful completion of a Phase 2A clinical trial evaluating the safety and efficacy of its drug candidate LP-10 for hemorrhagic cystitis.

The study was led by Dr. Jason Hafron of Michigan Institute of Urology, and the results will be presented as a late-breaking presentation at the American Urological Association (AUA) Annual Meeting in Chicago on April 30, 2023.

LP-10 has received Orphan Disease Designation from the US Food and Drug Administration (FDA) for the treatment of hemorrhagic cystitis, a potentially fatal disease with great unmet need and no currently approved drug treatment.

The company's multi-center, dose-escalation Phase 2A clinical trial recruited 13 subjects with moderate to severe refractory hemorrhagic cystitis. These subjects were treated with up to two courses of LP-10 intravesical bladder instillations. All subjects tolerated LP-10 instillations and completed the study without report of product related serious adverse events. LP-10 pharmacokinetic analysis demonstrated very short duration of systemic uptake. A dose response was noted, and there were decreased hematuria, decreased cystoscopic bleeding and ulceration sites, and improved patients' urinary symptoms. Responder analysis noted complete response in three subjects, partial response in seven subjects, and no response in three subjects.

"The successful completion of the clinical trial of LP-10 is a critical milestone in Lipella's development," stated Jonathan Kaufman, Ph.D., Chief Executive Officer of Lipella. "This milestone takes us one step closer to bringing a first-in-class treatment for the cancer survivor community with hemorrhagic cystitis."

Dr. Jason Hafron, Principal Investigator of the national trial, noted, "Patients were able to tolerate LP-10, and the treatment response is fast. Having a safe and effective drug to treat hemorrhagic cystitis would be a major service to the field of urology."

"Lipella's next step will be to communicate with the FDA on the study results and pathway toward seeking regulatory approval for LP-10," stated Dr. Michael Chancellor, M.D., Chief Medical Officer of Lipella.