Today UroGen announced an outcome for a meeting held by the Oncologic Drugs Advisory Committee (ODAC) of the U.S. Food and Drug Administration (FDA). The goal of this Adcom, or advisory panel was to establish a vote on whether or not the risk/benefit profile of UGN-102 is adequate for the treatment of patients with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). It was revealed that the ODAC voted 4 to 5 that the risk/benefit profile is favorable for UGN-102 to treat these patients with this type of bladder cancer. This doesn’t spell the end of the company at all, for now at least. That’s because while the FDA can take the ODAC’s recommendation into consideration, it doesn’t have to follow it.
Ultimately, it is going to be up to the FDA whether or not UGN-102 should be approved to treat these patients with LG-IR-NMIBC. Having said that, investors won’t have to wait a long time for this because the FDA has set up a Prescription Drug User Fee Act (PDUFA) target action date of June 13th of 2025. This U.S. agency can decide to approve this drug for the treatment of these patients either on or at any time before this set date. The vote is honestly mixed, and hopefully the FDA will approve the drug for these patients. The reason why is because it is an unmet medical need, as for the time being there are no-FDA approved drugs for this indication. As a matter of fact, these bladder cancer patients are distraught.
When speaking specifically about LG-IR-NMIBC, it is a highly recurrent disease whereby these patients must continuously undergo repeat surgeries. Of course, having elderly people do this often is not highly ideal. If a therapy like UGN-102 can be approved to help these patients undergo this, then it should be highly welcomed. Again, this is going to be up to the FDA to make a final decision on whether or not UGN-102 should be approved for these patients. The efficacy is definitely there, whereby positive data has been continuously released by this company. Long-term data from the phase 3 ENVISION study, released at the Society of Urologic Oncology (SUO) revealed that patients at 3 months had obtained a 79.6% complete response (CR) rate. Even better, those patients who responded at 3 months had an 82.3% duration of response (DOR) at 12 months.
What’s shocking to me though, is the risk/benefit analysis that was done by ODAC. The reason is because, of course, there were treatment-emergent adverse events (TEAEs) but they were only mild-to-moderate in terms of severity. Plus, they resolved or were resolving on their own. Such issues noted were fatigue, urinary retention, dysuria, urinary tract infection, and a few others. Despite these TEAEs, if patients can receive a treatment like UGN-102 (mitomycin) and forego the need for repeat surgeries, it should be highly welcomed. The way that this drug from UroGen works is by using its hydrogel-based RTGel technology to extend exposure of bladder tissue to the chemotherapy drug mitomycin. The outcome is a prolonged effect of this chemotherapy drug given via intravesical (within the bladder) instillation. The whole point of this is to offer a non-surgical approach, which LG-IR-NMIBC are accustomed to needing because of disease recurrence.
Whether or not FDA approval is given for UGN-102 for these specific bladder cancer patients remains to be seen. However, the company is at least attempting to diversify its risk with respect to its pipeline. Consider that it is expected to initiate a phase 3 study in mid-2025, which is going to use next-generation UGN-104 for the treatment of patients with low-grade upper-tract urothelial cancer (LG-UTUC). What’s so special about this candidate is that it is going to take UroGen’s proprietary RTGel technology and combine it with medac GmbH licensed mitomycin formulation. Even another candidate being developed with RTGel and medac’s mitomycin to treat patients with LG-IR-NMIBC is being explored in the ongoing phase 3 UTOPIA study. Enrollment for this late-stage trial is expected to be completed by mid-2025. The point here is that this provides another shot on goal in targeting these LG-IR-NMIBC patients, in case the company fails to obtain U.S. marketing approval of UGN-102 with the upcoming PDUFA target action date.