In a Phase 3 study, patients who saw a complete response at three months after receiving UroGen Pharma’s therapy had an 82% chance of having no cancer progression another year later.
UroGen’s treatment, known as UGN-102, delivers a decades-old chemotherapy known as mitomycin as a gel and could provide a non-surgical treatment option for certain bladder cancer patients. Historically, treating bladder cancer with drugs has been challenging because the bladder empties frequently. But UroGen’s “reverse thermal” gel, which is liquid when cold but thickens with body heat, can keep the treatment there for hours.
The biotech, which already markets a similar gel for a form of urothelial cancer, hopes the longer-term data will pave the way for an approval in a bigger market.
UroGen’s shares $URGN rose 40% on the data released Thursday.
Last year, UroGen announced that two Phase 3 studies of the treatment in low-grade intermediate-risk non-muscle invasive bladder cancer achieved their primary goals. However, the duration of response is a key aspect of the data package for the FDA, UroGen CEO Liz Barrett said. In low-grade bladder cancer, which spreads less aggressively but often recurs, the goal is to stop the cancer from coming back after treatment for as long as possible.
The one-year data from the ENVISION study included 108 patients. UroGen also reported that at 15 and 18 months, an estimated 80.9% were still responding, out of 43 and nine patients respectively.
“They really treat everything locally, which is why they do these repetitive surgeries,” Barrett said. “But it would be great if you could figure out a way for medicines, i.e. chemotherapies, to stay in there long enough to actually have an impact because it only stays in as long as the person is able to hold their urine.”
UroGen in January started a rolling submission to the FDA in hopes of winning commercial approval for the bladder cancer treatment. Since mitomycin has long been FDA-approved, the company is seeking approval for the gel via an FDA pathway that allows it to use information from past studies on the drug.
UroGen plans to seek priority review, which Barrett said would set it up to launch its therapy commercially in the first months of 2025 if approved.
Johnson & Johnson, via its 2019 acquisition of TARIS Biomedical, is also developing a treatment with the goal of keeping drugs from being flushed from the bladder too quickly. Its drug-device, which is known as the “pretzel” for its close resemblance, slowly releases a treatment such as chemotherapy over time.
Editor’s note: This story was corrected to reflect that the 82% 12-month duration of response was a Kaplan-Meier estimate and to add UroGen’s stock move.