~ Reduction of 28% of workforce not related to HEMGENIX® manufacturing obligations; Total cost savings of
~ Discontinuing investments in more than half of research and technology projects, centralizing operations, and streamlining organization ~
~ Prioritizing continued development of AMT-130 in Huntington’s disease and near-term initiation of clinical trials for AMT-260 in refractory mesial temporal lobe epilepsy, AMT-162 in SOD1-ALS, and AMT-191 in Fabry disease; Multiple potential value drivers expected over next two years ~
~ As a result of reprioritization,
“At uniQure, our highest priority is to deliver innovative, life-changing therapies to patients with significant unmet needs. To accomplish our mission and generate near-term value for our stakeholders, we will implement a strategic restructuring of our business,” stated
“These were difficult but necessary decisions,” he added. “I’m truly grateful for the commitment and contributions of our many colleagues, and we remain fully committed to delivering on our promise to patients in need. We also look forward to sharing longer-term, interim data on our Huntington’s disease program in late fourth quarter of this year and initiating clinical trials for our other programs as rapidly as possible.”
Restructuring Operations and Reducing Expenses
Following an extensive review of the pipeline, the Company will discontinue more than half of its research and technology projects, including AMT-210 for the treatment of Parkinson’s disease and multiple undisclosed programs. The company will focus its research efforts on a limited number of projects believed to have optimal risk, value and speed attributes, including AMT-161 for c9orf72 amyotrophic lateral sclerosis (ALS), AMT-240 for autosomal dominant Alzheimer's disease, and next-generation AAV capsid development. As a result of the reprioritization, the Company will be closing a research lab in
The Company will also consolidate all GMP manufacturing into its
As a result of the restructuring plan, the Company expects:
- Elimination of 114 positions, which represents 28% of the workforce not committed to HEMGENIX manufacturing obligations, and approximately 20% of the total workforce
- Total cost savings of approximately
$180 million over the next three years
- Current balance of cash, cash equivalents and investment securities of
$628.6 million as ofJune 30, 2023 (excludes$100 million milestone payment subsequently received fromCSL Behring ) to fund operations into the second quarter of 2027
- One-time restructuring costs of approximately
$2.3 million , primarily incurred in the fourth quarter of 2023
Prioritizing Clinical-stage Programs
The Company plans to focus resources on driving execution across four clinical-stage programs and expediting near-term proof-of-concept data readouts.
- AMT-130 for the treatment of Huntington’s disease: In the fourth quarter of 2023, the Company plans to provide a clinical update from both the
U.S. Phase I/II study of AMT-130, including 18- and 30-month follow-up data from the treated patients in the high- and low-doseU.S. cohorts, respectively, and for the first time, the European Phase I/II study. In the first quarter of 2024, the Company expects to meet with the FDA to review the data and discuss future development of AMT-130. Also in 2024, the Company will present up to 3-year follow-up data on all patients in theU.S. and European Phase I/II trials, including at least 2-year follow-up data on more than half of patients treated with AMT-130.
- AMT-260 for the treatment of refractory mesial temporal lobe epilepsy: In the third quarter of 2023, the Company announced the clearance of an investigational new drug (IND) for the Phase I/IIa clinical study of AMT-260. Screening and patient enrollment is expected to begin in the fourth quarter of 2023 with first patient dosing occurring in the first quarter of 2024.
- AMT-162 for the treatment of SOD1-ALS: The Company expects to initiate patient screening in the fourth quarter of 2023 with first patient dosing occurring in the first quarter of 2024.
- AMT-191 for the treatment of Fabry disease: The Company continues to expect to submit an IND in the fourth quarter of 2023 and to begin patient dosing in 2024.
Aligning Leadership Team
Changes on the Company’s leadership team are aligned with this strategic reorganization and pipeline prioritization.
“I want to express my sincere gratitude to Ricardo for his leadership in advancing our research and development programs over the last several years and for bringing innovative ideas and energy to the Company,” added
About uniQure
uniQure’s mission is to reimagine the future of medicine by delivering innovative cures that transform lives. The recent approvals of our gene therapy for hemophilia B – a historic achievement based on more than a decade of research and clinical development – represents a major milestone in the field of genomic medicine and ushers in a new treatment approach for patients living with hemophilia. We are now leveraging our modular and validated technology and manufacturing platform to advance a pipeline of proprietary gene therapies for the treatment of patients with
uniQure Forward-Looking Statements
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," “establish,” "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," “seek,” "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, statements that the Company’s restructuring will have anticipated cost savings of approximately
uniQure Contacts:
FOR INVESTORS: | FOR MEDIA: | |
Direct: 339-970-7536 Mobile: 617-680-9452 m.cantor@uniQure.com | Direct: 617-306-9137 Mobile: 617-306-9137 c.russo@uniQure.com | Direct: 339-970-7558 Mobile:339-223-8541 t.malone@uniQure.com |
Source: uniQure Inc.
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