BiQ: Will PRGN-2012 Share UGN-102's Fate? (URGN PGEN)

Following the events at Urogen's (URGN) ADCOM, I have been giving considerable thought to Precigen (PGEN), which has a PDUFA date of August 27, 2025, for PRGN-2012 for the treatment of RRP.

There are some key similarities between UGN-102 and PRGN-2012:

• Both therapies target conditions with high morbidity but low mortality.
• Both therapies are designed to replace surgical resections, which are often repeated multiple times over the patient's lifetime.
• Both companies are seeking approval based on single-arm pivotal trials.

However, there are also some differences:

• The pivotal trial for PRGN-2012 was conducted with FDA alignment on an accelerated approval pathway, with PGEN to conduct a confirmatory trial post approval (the confirmatory trial is already in progress). However, investors should note that URGN also had alignment with the FDA for a single-arm trial, provided the patient population was sufficiently large, although no confirmatory trial was requested at the time. The FDA never raised any objections to the size of the Envision trial during the ADCOM.
• PRGN-2012 treats a rare disease, whereas UGN-102 treats a more common condition with higher prevalence. There is some recent evidence to support the belief that the FDA has a more favorable disposition towards rare disease indications.

So, how should we interpret this? Do the FDA's objections regarding UGN-102 have a read-through to PRGN-2012?

Honestly, I'm not sure. The optimist in me would like to believe the FDA's approach with PRGN-2012 may, perhaps, even bolster the odds of a conditional approval for UGN-102 with a follow-up confirmatory trial, which would align with the approach the FDA took with PRGN-2012. However, the pessimist in me worries that there are enough similarities between UGN-102 and PRGN-2012 that the latter could meet the same fate as UGN-102. So far, the FDA has not requested an ADCOM for PRGN-2012, but this could change at any time between now and the PDUFA date.

In the case of PRGN-2012, I think it would be difficult for the FDA to make the case that Precigen acted contrary to the FDA's guidance; after all, as far as I know, no two-arm, placebo-controlled trial was requested. On the other hand, as far as single-arm trials go, I think the data from Urogen's Envision trial was just as strong, or arguably perhaps even stronger, than Precigen's pivotal trial for PRGN-2012. After all, UGN-102's 80% CR rate, with 80% of patients remaining in CR at 18 months, is pretty compelling data.

The one thing investors hate most is uncertainty, and I think the outcome of UGN-102's ADCOM creates additional uncertainty around PRGN-2012. I haven't made a decision yet on whether to make any changes to my portfolio positioning. PGEN currently carries a C+ risk rating in the BiQAP, primarily due to the regulatory risk, despite what I consider to be compelling data. My current plan is to see how Urogen plays out, and then possibly re-evaluate my Precigen position; however, that could change as new information becomes available.

However, investors interested in Precigen who want to avoid regulatory risk may want to consider waiting until after PRGN-2012 is approved before initiating a position.

Please refer to the BiQAP Live spreadsheet on the Active Portfolio page or the iQCS for additional information (PGEN, URGN).

URGN share price at time of publication: $4.05
PGEN share price at the time of publication: $1.37

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