This project, entitied "Preclinical Development of Tracrolimus for Radiation Cystitis" will fund key experiments to advance the commercial development of LP-10. LP-10 is a liposomal tacrolimus formulation for local (topical) intravesical administration to the urinary bladder. The formulation provides active drug levels in the bladder with significantly reduced systemic levels. Radiation cystitis is a rare disease defined by lower urinary tract symptoms that include dysuria, hematuria, and hemorrhage. There are currently no approved therapies to treat RC, which can severely degrade a patient's quality of life, require long-term follow-up treatment, and, in some patients, lead to death. This project is public-private collaboration between Lipella Pharmaceuticals Inc., the University of Pittsburgh School of Medicine, and William Beaumont Hospital. Lipella owns intellectual property rights to the LP-10 formulation as well as rights associated with its recent receipt of orphan designation from the FDA. The FDA has also provided Lipella with pre-clinical requirements for IND consideration of LP-10. These requirements constitute the studies described in this proposal. The scope of work includes further pharmaceutical and pharmacological characterization of LP-10 (Phase I) and IND-enabling studies (Phase II). This research is translational, and bridges basic research to clinical development. Progress in this direction will directly support our ultimate goal of developing a safe and effective therapy ready for commercialization.
This research proposal for the National Institutes of Health Small Business Innovation and Research (SBIR) program entitiled Preclinical Development of Tacrolimus for Radiation Cystitis will further the development of a novel bladder tacrolimus formulation targeting a rare disease with an unmet medical need. Radiation cystitis is defined by lower urinary tract symptoms that include dysuria, hematuria, and hemorrhage with an annual U.S. incidence of approximately 60,000. There are currently no approved therapies for radiation-induced bladder damage. Given the lack of good treatment options available for patients, the potential impact of this proposal could be dramatic.