uniQure Reports Q2 2024 Financials and Company Update

uniQure N.V., a prominent gene therapy company based in Lexington, Mass., and Amsterdam, announced significant advancements and financial results for the second quarter of 2024. CEO Matt Kapusta reported notable progress in clinical trials and the company's efforts to reduce capital requirements.

A highlight in their updates is the U.S. FDA's Regenerative Medicine Advanced Therapy (RMAT) designation for AMT-130, an investigational gene therapy for Huntington’s disease. This designation allows for early and frequent interactions with the FDA to expedite clinical development. Positive interim data from Phase I/II studies in the U.S. and Europe showed a dose-dependent slowing of disease progression in patients treated with AMT-130 and reductions in neurodegeneration biomarkers. The company plans to meet with the FDA in the latter half of 2024 to discuss further development pathways for AMT-130. Patient enrollment for a third cohort is ongoing, aiming to evaluate both doses of AMT-130 with a focus on safety and tolerability. An additional interim update from the ongoing trials is expected in mid-2025.

In addition to Huntington’s disease, uniQure has initiated patient screenings for three new Phase I/II clinical studies:
- AMT-260 for refractory mesial temporal lobe epilepsy (mTLE)
- AMT-191 for Fabry disease
- AMT-162 for SOD1 amyotrophic lateral sclerosis (ALS)

Enrollment for these studies is expected to begin in the third quarter of 2024. Each study will involve up to 12 patients in different dose cohorts across multiple centers in the U.S.

On the financial front, uniQure has undertaken several capital preservation initiatives. The recent sale of its Lexington manufacturing facility is projected to reduce the workforce by 65%, saving approximately $75 million annually, including the retirement of $50 million in debt. As of June 30, 2024, the company reported a strong cash position of about $524 million, expected to fund operations through the end of 2027.

The reorganization aims to streamline operations and ensure sufficient funding for key milestones. Pierre Caloz, the Chief Operating Officer, will leave the company following the sale of the Lexington facility. Amin Abujoub, Ph.D., will take on the new role of Chief Technical Operations Officer, overseeing global contract manufacturers and internal operations.

Financial results for the second quarter showed an increase in revenue to $11.1 million compared to $2.4 million in the same period of 2023, primarily due to higher collaboration and license revenues. Research and development expenses decreased to $33.7 million from $46.0 million, and selling, general, and administrative expenses were reduced to $15.8 million from $21.2 million. The net loss for the quarter was $56.3 million, a reduction from $68.5 million in the previous year.

uniQure continues to focus on delivering transformative gene therapies for severe diseases, with ongoing efforts in Huntington's disease, temporal lobe epilepsy, ALS, and Fabry disease. These advancements highlight the company's commitment to innovative treatments and strategic financial management to support its long-term goals.