LiPax™: Non-Muscle Invasive Bladder Cancer (NMIBC)
Common disease
Bladder cancer is a common disease affecting 3.4 million people globally with approximately 430,000 new cases every year. In the U.S., bladder cancer is the sixth most common type of cancer with more than 700,000 bladder cancer patients and approximately 120,000 new cases per year.
Bladder cancer is generally separated between non-muscle invasive bladder cancer (NMIBC) and muscle-invasive bladder cancer (MIBC). Approximately 74 percent of bladder cancer patients are diagnosed with NMIBC, prior to the tumor advancing from the urothelium (bladder lining) into the detrusor muscle of the bladder wall. Although NMIBC survival rates are high, it exhibits a high recurrence rate of 31-78 percent, severely impacting patients' quality of life and making it expensive to treat and placing a significant financial burden on the healthcare system.
Lack of effective treatments
Treatment of NMIBC is based on risk stratification. Most patients are initially treated with a transurethral resection of bladder tumor (TURBT) followed by a single, immediate instillation of intravesical chemotherapy to eliminate any residual tumor cells. Subsequent therapy is typically based on risk and consists of induction therapy weekly for six weeks under the current standard of care.
Common treatments include the live vaccine Bacillus Calmette-Guerin (BCG) and Mitomycin C (MMC). Unfortunately, MMC and BCG are inadequate for many patients with NMIBC. BCG is occasionally poorly tolerated and may introduce risk for systemic infections. MMC is hydrophilic and does not penetrate the urothelium on the bladder wall resulting in limited drug exposure to tumors and overall low efficacy. An acute shortage of BCG, and from time to time MMC, has resulted in rationing with the most serious cases given priority and the American Urological Association (AUA) advising that patients with high risk features are being advised on receiving radical cystectomy as a surgical alternative recommended by the AUA.
LiPax for NMIBC
LiPax is a neoliposomal intracavitary paclitaxel formulation in development for intravesical instillation in the treatment of NMIBC.
LIPAC announced the successful completion of a Phase 1-2a clinical trial that was designed to demonstrate safety, tolerability and a projected a two-year recurrence free survival (2-YR RFS) rates in patients with low-grade highly recurrent NMIBC treated with LiPax.
In the Phase 1 clinical studies in low and intermediate patients, LiPax demonstrated an 83% recurrence free survival (RFS) rate following TURBT and intravesical instillation of LiPax. The results represent a notable improvement over current SOC that achieve 49% RFS rates respectively in the same setting for low and intermediate risk NMIBC.
LIPAC demonstrated a 63% marker lesion response rate (MLRR) in the Phase 2a marker lesion clinical trial. The study was designed to predict long-term (two-year) recurrence free survival rates in patients with low-grade highly recurrent NMIBC treated with LiPax. LiPax demonstrated a 63 percent responder rate in highly recurrent patients treated with LiPax alone compared to 35% for existing therapies.
In the clinical studies, persistence of paclitaxel (the active product ingredient of LiPax) at above therapeutic levels was demonstrated for up to 48 hours following intravesical instillation, and urinary health related quality of life (HR QOL) responses (patient reported outcomes) indicated LiPax was well tolerated. In contrast to current SOC, no significant adverse events (Grade 3 or 4) were observed.