uniQure (QURE) Q2 FY26 earnings call: guidance and Q&A

How quickly can the team operationalize the confirmatory study, given the FDA's requirement for it to be nearly fully enrolled at accelerated approval, and what is the likely patient enrollment number?

Management believes the FDA's intent is for timely completion post-approval, not full enrollment at approval. They are confident in demonstrating this and have started preparatory activities for a global study. Recruitment will prioritize countries where the drug is not yet available. Sample size is still under discussion with the FDA, so it's premature to disclose.

“So the idea is that most of the recruitment post approval will occur in countries before the drug becomes available in those countries such that we can complete the study on time.”

asked by Debjit Chattopadhyay · answered by Walid Abi-Saab

What have you learned about operational bottlenecks at trial sites regarding AMT-130 delivery, and how should investors think about realistic year 1 throughput for activated centers?

The company has been deeply engaged with treatment centers, recognizing that each hospital is unique. They are mapping processes across neurology, neurosurgery, and other specialties to address potential bottlenecks proactively. While capacity is challenging to quantify due to variables like neurosurgeon availability and OR suites, they are working with an initial number of centers and will build from there. ZOLGENSMA and ELEVIDYS are considered reasonable analogs for hospital-based procedures, though no analog is perfect.

“I will say that we don't see it as a bottleneck because we're doing everything that we can to work with these institutions ahead of a potential BLA approval to make sure that at the point of that BLA approval, we're able to move forward as quickly as possible.”

asked by Joseph Schwartz · answered by Kylie O'Keefe

Are you expecting an AdCom meeting for the BLA submission? What is the progress on studies for patients with low striatal volume and shorter neurosurgical administration, and when might data be seen?

Management expects an AdCom is likely and welcomes it, preparing for it. The low striatal volume cohort has been fully recruited, but it's too early for efficacy data, though some safety data has been shared. There is no ongoing cohort specifically focusing on shorter surgical administration at this point.

“Regarding the low striatal volume cohort, that cohort has been fully recruited, but it's a bit early right now to share any of the efficacy data.”

asked by Unknown Analyst · answered by Walid Abi-Saab

Can you provide more color on the standard of care control arm in the confirmatory study and how it might impact recruitment and retention, given its open-label nature?

Patients randomized to the standard of care arm will be eligible to receive AMT-130 after 3 years, without needing to meet original inclusion criteria, provided it's safe. The strategy involves prioritizing US recruitment initially, then focusing on countries where AMT-130 is not yet commercially available to minimize dropout. Statistical techniques will be used to manage dropouts, and patients with Huntington's disease are dedicated to participating in studies.

“We believe that in our case, there's going to be a couple of items that are going to be paying attention to. One is that people who would be randomized to standard of care will be eligible to receive AMT-130 at the -- after 3 years.”

asked by Lydia Erdman · answered by Walid Abi-Saab

Is accelerated approval dependent on the completion of enrollment for the confirmatory study, or will the application be held until it's completed?

Accelerated approval does not require completion of the confirmatory study, but the FDA's intent is to ensure the study can be completed in a timely manner post-approval. Management is confident they can operationalize the study quickly, focus on US pre-approval, and conduct it globally in countries where the product is not commercially available, thus addressing FDA priorities.

“Accelerated approval is -- you don't have to complete the study to get accelerated approval. But the FDA wants to ensure that the study can be completed in a timely manner.”

asked by Uy Ear · answered by Matthew Kapusta

Why is the FDA considering TFC as the primary endpoint over cUHDRS for the confirmatory trial, and how might this influence their review of the upcoming package?

The FDA views cUHDRS as an intermediate clinical endpoint reasonably likely to predict efficacy for accelerated approval. However, philosophically, they tend to lean towards functional endpoints like Total Functional Capacity (TFC) for confirmatory studies, as TFC measures independence and has quality of life aspects, which is considered a more 'pure' functional endpoint.

“Total functional capacity is a measure of independence. It has a lot of aspects that are quality of life associated. And our sense is that in discussions that the FDA had with us as well as other sponsors that they tend to lean more towards total functional capacity as a primary endpoint for a confirmatory study.”

asked by Kristen Kluska · answered by Matthew Kapusta