BiQ: Some Thoughts on Today's Biotech Selloff (11/03/25)

At the time of this writing, biotech investors appear to remain in a sour mood, with the XBI currently down by over 3%. I believe the selloff is at least partially attributable to the PR issued by QURE this morning stating that the FDA may no longer agree that the P1/2 trials for AMT-130 are adequate to support a BLA, making the timing of a potential BLA filing uncertain. This seems to have soured investors' mood towards biotech in general. I have also been fielding many questions from BiQ Premium subscribers this morning, both in BiQ Premium Chat and via email, about how this might affect the chances of approval for other specific biotech stocks approaching regulatory milestones, such as REPL, KURA, CAPR, LXEO, and others.

I think it's important to keep today's news in perspective. In total, only 39 patients were enrolled in the AMT-130 P1/2 trials across the US and Europe. Only 10 patients were randomized to a sham control group in Cohort 3, which was initiated later. So this means only 39 patients, with only 10 enrolled in a sham control arm, for an indication that has an estimated prevalence of around 75K patients across the US and EU.

Personally, this was always a concern for me, and is also why I exited my QURE position in my personal portfolio months ago (to be fair, I also missed out on much of the recent upside). Dr. Vinay Prasad has stated on several occasions that the FDA is open to regulatory flexibility for rare indications with relatively small patient populations; however, he has also stated that the agency would require a higher standard of evidence for larger indications. To be clear, I did not know and could not predict that the FDA would change its stance on AMT-130, but I did consider it to be a risk factor.

It's also important to keep in mind that the regulatory flexibility for AMT-130 was granted prior to Dr. Prasad's tenure at the FDA, and in the past, he has demonstrated a clear willingness to overturn prior FDA regulatory guidance that was issued under the previous FDA leadership, especially if such guidance conflicts with his priorities.

So, while I have my own concerns with some of the policy decisions taken by new management at HHS and the FDA, and while I think it is understandable that today's news creates anxiety for biotech investors, I don't think it changes the new regulatory landscape. It's important to stay focused on why the FDA acted as it did, and keeping that in mind, I don't think today's news regarding AMT-130 should come as a major shock, nor do I think it contradicts the direction the FDA has taken under its new leadership.

Please refer to the BiQAP Live spreadsheet on the Active Portfolio page or the iQCS for additional information.

QURE share price at time of publication: $ 33.25

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