A Follow-up on UroGen’s PDUFA

Friday’s Trading

Right after the last note was published, UroGen’s stock price soared 43% to $7.37, close to the pre-ADCOM levels. The best explanation of Friday’s price activity is a gamma squeeze, followed by some shorts getting margin-called and covering out of necessity. A leak is possible but unlikely a week ahead of the PDUFA decision. It is also possible that there was a dialogue between the FDA and UroGen, and the funds were disclosed. However, it is mostly all speculation.

Thoughts and Further Research

We spent the weekend exploring the PDUFA further and would like to raise a few new points. First, we again reviewed UroGen’s response to the ADCOM decision at the TD Cowen Oncology Summit. A few points stand out. The CEO, Liz Barrett, claimed that the company was in discussions with the FDA regarding labeling before the ADCOM decision. Labeling is typically one of the last aspects of the application, which some may interpret to mean that the efficacy and safety discussion has already concluded. It is unclear if this is the case, as in this instance, labeling discussions may have occurred sooner than in other applications, as the ADCOM would initiate the majority of the efficacy discussions. Given the ADCOM was convened less than a month before the PDUFA date, it is unlikely that the date could have been met if labeling discussions had not taken place prior. UroGen also claimed the RCT discussion in the ADCOM biased the committee as it took place before the vote. However, the FDA themselves wanted the discussion to occur, so I do not take that as a positive for the PDUFA.

Second, we re-watched the ADCOM. It is clear the two FDA presenters, Sandeep Agrawal and Brain Weiss, were not positive about UGN-102. They questioned the relevance and interpretability of the primary study, ENVISION, while raising safety concerns in comparison to the SOC, TURBT + intravesical chemotherapy. The vote was 5-4 against approval. All five votes against approval came from medical oncologists. The four votes for approval came from the two urologists, the patient advocate, and one medical oncologist. I discount the patient advocate vote, as I doubt their opinion significantly sways the FDA. Some would say the fact the two urologists on the panel voted for the treatment is promising. However, there may be a financial conflict of interest for them. Danie Spatt raised an interesting point: UroGen’s treatment costs $ 140,000. There may be a strong economic incentive for them to have additional treatment options here.

The only point that is concerning for the bear case is Pazdur’s comments at the end. He said, “I consider this a split vote; let’s talk with the sponsor.” This is open to interpretation, but the translation is certainly not a strong CRL.

Lastly, we reviewed previous instances where the FDA disagreed with the ADCOM on the oncology front. An article published in 2020 (Vinay Prasad, a recent notable FDA appointee, was one of the authors) detailed how the FDA never goes against a positive ADCOM recommendation but disagreed with a negative recommendation 7 out of 30 times. However, those seven times involved life-threatening cancers with few other treatment options. The contexts are not at all similar. Given this, it would be unprecedented for the FDA to approve this treatment. It is still possible, and Pazdur’s comments are vague, but we believe there is a significantly higher likelihood of a CRL compared to approval.