Prostate cancer is the most commonly diagnosed non-skin cancer in men in the U.S. It is estimated that more than 190,000 new U.S. cases will be diagnosed in 2020 alone. Approximately 80% of newly diagnosed prostate cancer is localized. When detected at an early stage, it grows slowly and progression to metastases within 10 years is uncommon. Historically and according to current clinical guidelines, the two treatment options for men with this type of prostate cancer are at opposite ends of the spectrum: active surveillance or radical therapy, which includes radical prostatectomy and radiation therapy. Active surveillance reduces overtreatment, avoids treatment-related side effects, preserves quality of life, and allows men to maintain normal activities and work schedules. However, only about 45% of men with early stage prostate cancer choose active surveillance and 55% are treated immediately with radical therapy and are exposed to the side effects of those treatments. Additionally, approximately half the men who do start with active surveillance undergo radical therapy eventually. Hence, despite the progress in the adoption of active surveillance over the past decade, three-quarters of early stage prostate cancer patients eventually receive radical treatment and are exposed to unnecessary high risk of morbidity, including urinary incontinence, erectile dysfunction and disruption of bowel function. TOOKAD is a light-activated drug that enables partial gland ablation where only the portion of the prostate known to have cancer is treated. It has the potential to slow the progression of the disease and delay radical treatment and the morbidity associated with it.
Clinical data supporting the safety and efficacy of TOOKAD have been gathered in five studies with 652 participants, including the pivotal study PCM301. This study was a Phase III, prospective, multicenter, open-label, 1:1 randomized study that compared TOOKAD with active surveillance in 413 patients. Results from this pivotal study were the focus of the presentation to ODAC.. The study met its co-primary and secondary endpoints, demonstrating that TOOKAD delivers clinically meaningful and statistically significant reductions in local disease progression and conversion to radical therapy compared to active surveillance. The safety profile of TOOKAD was primarily composed of mild, temporary events and did not preclude future treatment options. Follow-up data five years after randomization have not identified any new safety signals and indicate durability of the clinical benefit.