uniQure Reports Q1 2024 Financial Results and Recent Progress

uniQure N.V., a prominent gene therapy company focused on delivering transformative treatments for patients with severe medical conditions, announced its financial results for the first quarter of 2024 from its locations in Lexington, Mass., and Amsterdam. The company highlighted significant advancements in its ongoing projects and future plans.

CEO Matt Kapusta noted substantial progress in three primary areas: defining the future for AMT-130 in Huntington’s disease, launching three new Phase I/II clinical trials, and managing financial resources judiciously. uniQure aims to start discussions with the FDA regarding AMT-130 in the second quarter of 2024. They plan to present extended follow-up data from their Phase I/II trials by mid-year, encompassing up to three years of patient observation across 29 treated individuals.

uniQure is also set to begin new Phase I/II studies. They have obtained IRB approvals for initial sites and identified potential participants. Enrollment for the Fabry disease study is slated to start in the second quarter of 2024, with the SOD1-ALS and MTLE studies following in the third quarter.

Kapusta emphasized the company's ongoing review of operations to cut costs and boost shareholder value, aiming to complete this evaluation by mid-2024. He expressed confidence in the value of uniQure’s assets and the necessity for a focused investment strategy.

Recent Developments

AMT-130 for Huntington’s Disease
uniQure is preparing to engage with the FDA in the second quarter of 2024 to outline the clinical and regulatory path for AMT-130. The discussions will likely include interim data from the ongoing Phase I/II trials and the potential use of natural history comparators. The company intends to update its regulatory plans and provide more clarity on a potential approval pathway for AMT-130 by the end of the year. Mid-2024 will see a clinical update from the ongoing Phase I/II trials, including up to three years of follow-up on 29 treated patients. Additionally, a third cohort of up to 12 patients is being dosed to evaluate safety and tolerability, with enrollment expected to finish in the second half of 2024.

New Phase I/II Clinical Studies
- AMT-191 for Fabry Disease: Patient enrollment is expected to start in the second quarter of 2024. The study involves two cohorts of six adult male patients each and will focus on evaluating safety, tolerability, and early efficacy signs.
- AMT-162 for SOD1-ALS: Enrollment is set for the third quarter of 2024. This multi-center, open-label trial will include three cohorts of up to four patients each, receiving a one-time intrathecal infusion with immunosuppression.
- AMT-260 for Refractory Mesial Temporal Lobe Epilepsy (MTLE): Patient enrollment is also expected in the third quarter of 2024. The trial will consist of two parts: an open-label phase with two dosing cohorts of six patients each and a subsequent randomized, controlled trial to generate proof-of-concept data.

Financial Highlights

- Cash Position: As of March 31, 2024, uniQure held $555.7 million in cash and investment securities, down from $617.9 million at the end of 2023. This is expected to fund operations into the second quarter of 2027.
- Revenues: The company reported revenues of $8.5 million for the first quarter of 2024, compared to $5.3 million in the same period of 2023. The increase was primarily due to higher collaboration and license revenue.
- R&D Expenses: Research and development costs were $40.7 million, down from $60.8 million in the first quarter of 2023. The decrease was largely due to lower expenses in the ALS SOD1 program and other preclinical areas.
- SG&A Expenses: Selling, general, and administrative expenses decreased to $13.9 million from $17.8 million in the same period last year, mainly due to reduced professional and intellectual property fees.
- Net Loss: The net loss for the first quarter of 2024 was $65.6 million, or $1.36 per share, compared to $77.2 million, or $1.63 per share, in the first quarter of 2023. 

uniQure remains committed to advancing its gene therapy pipeline and achieving significant milestones in its ongoing clinical trials and financial management.

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