Hemorrhagic Cystitis (HC) is a common side-effect of cancer treatment, affecting some 10 to 40 percent of patients who receive high dose chemotherapy or radiation therapy. Most often its symptoms include hematuria, bladder pain, and irritative voiding, though it can also lead to scarring of the bladder wall, bladder atrophy, systemic infection, urosepsis, or urinary obstruction. Onset can occur during treatment or immediately after treatment, or it may be delayed for months. While rarely fatal, HC compounds the suffering of cancer patients.
There are no FDA-approved drugs currently available for the treatment of HC. Today's most effective option is hyperbaric oxygen (HBO) therapy. This process requires patients to sit in a hyperbaric chamber for long periods of time. For many, HBO therapy is widely unavailable and prohibitively expensive, ranging from $15k and $50k per treatment.
LP-10 is a proprietary liposomal formulation of tacrolimus, a potent immunosuppressant and anti-inflammatory agent. To date, LP-10 has shown promising efficacy in animal models of both chemotherapy-induced cystitis and radiation cystitis. As a result, Lipella has received orphan designation from the FDA for the use of tacrolimus in HC treatment. The company also received NIH funding to complete the remaining toxicology studies needed for FDA approval for human studies. These toxicology studies have been completed with no indication of adverse events due to LP-10.
We plan to initiate recruitment in an NIH-funded, Phase-2 LP-10 clinical trial in 3Q2018.