Release Date: May 09, 2025
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- uniQure NV (QURE, Financial) reported significant progress in its Huntington's disease program, with AMT-130 receiving breakthrough therapy designation from the FDA.
- The company has a strong financial position with over $400 million in cash, providing flexibility to advance its pipeline.
- uniQure NV (QURE) has expanded its clinical pipeline with new studies in refractory temporal lobe epilepsy, Fabry disease, and SOD1 ALS.
- The company successfully completed an $80 million follow-on offering, further strengthening its balance sheet.
- uniQure NV (QURE) is on track for a planned BLA submission for AMT-130, with productive interactions with the FDA supporting this timeline.
Negative Points
- Revenue for the first quarter decreased significantly to $1.6 million from $8.5 million in the same period in 2024, primarily due to reduced collaboration and contract manufacturing revenue.
- There were three serious adverse events related to immunosuppression in Cohort 3 of the AMT-130 trial, although they were resolved with standard care.
- The company faces challenges in maintaining momentum with its pipeline, as evidenced by the need for ongoing regulatory interactions and data analysis.
- uniQure NV (QURE) is still in the early stages of commercial planning for AMT-130, with details on market strategy and partnerships yet to be finalized.
- The company has not yet finalized its approach for a confirmatory study for AMT-130, which could impact future regulatory approvals.
Q & A Highlights
Q: How confident are you on the three-year follow-up data on the cUHDRS, and what would you consider a good outcome?
A: Walid Abi-Saab, Chief Medical Officer, stated that they have been monitoring patients for a long time and have no indication of losing efficacy over time. They believe the dose-dependent reduction in cUHDRS observed at the two-year data will be maintained at three years, and they feel very confident about their results going forward.
Q: Will the third quarter update include the propensity match scoring based on the agreed-upon SAP, or will that analysis be withheld for the BLA submission only?
A: Walid Abi-Saab explained that they plan to agree with the FDA on the statistical analysis plan before locking the database and analyzing the data. They will share topline results but must be careful about the degree of data shared before FDA review.
Q: Can you elaborate on the three immunosuppression-related SAEs reported in Cohort 3 and any changes made to the protocols or perioperative management?
A: Walid Abi-Saab noted that AMT-130 is generally safe and well-tolerated. In Cohort 3, they observed three adverse events related to immunosuppression: mania, infection, and fever, all of which resolved with standard interventions. They believe a short two-week course of corticosteroids is optimal for future use.
Q: How are you thinking about potentially evaluating AMT-130 in patients with earlier or later-stage disease?
A: Walid Abi-Saab mentioned that the initial trial focused on early manifest patients to maximize function preservation. They are considering earlier or later interventions but need to discuss with regulators to determine the best path forward. The decision will depend on generating data to show benefit across different stages.
Q: What is the latest data set the FDA has seen, and have they seen any Cohort 3 data with the updated immunosuppression regimen?
A: Walid Abi-Saab clarified that the FDA has seen data from 24 patients at two years, based on a June 2024 cut. No additional data, including Cohort 3, has been shared since then. The two-week steroid regimen will be new, as previous cohorts did not include systematic perioperative immunosuppression.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
