SBIR-STTR Award

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-privatecollaboration 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.

Thesaurus Terms:
Basic Science;Biological Availability;Bladder;Canis Familiaris;Caring;Cessation Of Life;Clinical;Clinical Research;Collaborations;Commercialization;Cyclophosphamide;Cystitis;Data;Development;Dinoprostone;Dose;Drug Formulations;Drug Kinetics;Dysuria;Effective Therapy;Follow-Up;Foundations;Funding;Goals;Hematuria;Hemorrhage;Histopathology;Hospitals;Human Wfdc2 Protein;Immunosuppressive Agents;Incidence;Inflammatory;Innovation;Intellectual Property;Interleukin-2;Intravenous;Intravesical;Intravesical Administration;Kidney;Kidney Function Tests;Lead;Letters;Life;Liposomes;Lower Urinary Tract Symptoms;Lymph Nodes;Marketing;Medical;Medical Schools;Men;Modeling;Novel;Oral;Orphan;Patients;Pelvic Cancer;Pharmaceutical Preparations;Pharmacologic Substance;Phase;Phase 1 Study;Pre-Clinical;Programs;Property Rights;Public Health Relevance;Quality Of Life;Radiation;Radiation Therapy;Rare Diseases;Rattus;Reaction;Research;Research Proposals;Research Study;Research Support;Rights;Route;Safety;Small Business Innovation Research Grant;Staining Method;Stains;Tacrolimus;Testing;Therapeutic;Therapeutics For Rare And Neglected Diseases;Tissues;Toxic Effect;Toxicology;United States National Institutes Of Health;Universities;Urinary Tract;Urine;Urologic;Urothelium;Woman;Work;

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.

Public Health Relevance Statement:


Public Health Relevance:
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.

NIH Spending Category:
Interstitial Cystitis; Orphan Drug; Rare Diseases; Urologic Diseases

Project Terms:
Basic Science; Biological Availability; Bladder; Canis familiaris; Caring; Cessation of life; Clinical; Clinical Research; Collaborations; commercialization; Cyclophosphamide; Cystitis; Data; Development; Dinoprostone; Dose; Drug Formulations; Drug Kinetics; Dysuria; effective therapy; follow-up; Foundations; Funding; Goals; Health; Hematuria; Hemorrhage; Histopathology; Hospitals; human WFDC2 protein; Immunosuppressive Agents; Incidence; Inflammatory; innovation; Intellectual Property; Interleukin-2; Intravenous; intravesical; Intravesical Administration; Kidney; Kidney Function Tests; Lead; Letters; Life; Liposomes; lower urinary tract symptoms; lymph nodes; Marketing; Medical; medical schools; men; Modeling; novel; Oral; Orphan; Patients; Pelvic Cancer; Pharmaceutical Preparations; Pharmacologic Substance; Phase; phase 1 study; pre-clinical; programs; Property Rights; Quality of life; Radiation; Radiation therapy; Rare Diseases; Rattus; Reaction; Research; Research Proposals; research study; Research Support; Rights; Route; Safety; Small Business Innovation Research Grant; Staining method; Stains; Tacrolimus; Testing; Therapeutic; Therapeutics for Rare and Neglected Diseases; Tissues; Toxic effect; Toxicology; United States National Institutes of Health; Universities; Urinary tract; Urine; urologic; Urothelium; Woman; Work