LEXINGTON, Mass. and AMSTERDAM – uniQure N.V. (NASDAQ: NASDAQ:QURE), a gene therapy company, has initiated a Phase I/II clinical trial for its investigational treatment AMT-162, targeting ALS caused by SOD1 mutations. The first patient received a dose in the EPISOD1 trial, which is being conducted across multiple centers in the United States.
The open-label trial will assess the safety, tolerability, and preliminary efficacy of AMT-162 in patients with SOD1-ALS, a rare and progressive motor neuron disease. Up to 12 patients will be enrolled in three dose-escalating cohorts, receiving the therapy alongside a short course of immunosuppression.
AMT-162 is an AAVrh10-based gene therapy designed to knock down the expression of the mutated SOD1 protein, which is toxic to motor neurons and leads to muscle weakness and eventually death. The therapy, administered intrathecally, has been granted Orphan Drug status and Fast Track designation by the FDA.
The trial will measure neurofilament light chain and SOD1 protein levels to evaluate signs of efficacy. Currently, four U.S. sites are active, with seven more expected to be operational by the first quarter of 2025.
ALS, specifically the form caused by SOD1 mutations, results in the loss of motor neurons, leading to muscle atrophy and respiratory failure. It is estimated that around 3,400 individuals globally have SOD1-ALS. The average life expectancy after diagnosis is three to five years.
uniQure is recognized for its gene therapy for hemophilia B and is advancing a pipeline of gene therapies for various severe diseases. This trial marks the company's third gene therapy program entering clinical stages.
This news is based on a press release statement, and the trial's details can be found on the clinicaltrials.gov website under identifier NCT06100276.
In other recent news, gene therapy company uniQure has seen significant developments in its clinical portfolio. The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to the company's investigational gene therapy, AMT-191, for the treatment of Fabry disease. Meanwhile, uniQure's Huntington's Disease gene therapy, AMT-130, has been producing compelling data, leading to discussions with the FDA about potential accelerated approval.
Further, Raymond James resumed coverage on uniQure, assigning an Outperform rating, highlighting the company's robust clinical portfolio and financial runway extending to the end of 2027. H.C. Wainwright and Stifel have also maintained a Buy rating on uniQure, following these positive developments.
In addition, uniQure has undergone significant organizational restructuring, including a 65% reduction in its workforce and the sale of its manufacturing facility in Lexington, Massachusetts. This move is part of the company's strategy to prioritize the development of its gene therapy programs. Genezen has acquired uniQure's commercial gene therapy operations in Lexington, enhancing its global gene therapy development capabilities.
Lastly, on the governance side, uniQure's shareholders have approved an expansion of the company's share incentive plan, reappointment of board members, and the adoption of the 2023 statutory annual accounts. However, Paula Soteropoulos, a non-executive director, has chosen not to stand for re-election at the next annual meeting. These are the recent developments in the company's operations.
InvestingPro Insights
As uniQure N.V. (NASDAQ: QURE) embarks on this pivotal Phase I/II clinical trial for AMT-162, investors should consider some key financial metrics and insights from InvestingPro.
The company's market capitalization stands at $274.65 million, reflecting its position in the specialized gene therapy sector. Despite the promising clinical developments, uniQure faces some financial challenges. According to InvestingPro Tips, the company is "quickly burning through cash" and "suffers from weak gross profit margins." This is evident in the reported gross profit margin of -606.8% for the last twelve months as of Q2 2024.
However, it's not all gloomy for uniQure. InvestingPro Tips also indicate that "analysts anticipate sales growth in the current year," which could be partly attributed to the advancement of their clinical pipeline, including the AMT-162 trial for SOD1-ALS. Additionally, "6 analysts have revised their earnings upwards for the upcoming period," suggesting some optimism about the company's near-term prospects.
The company's revenue for the last twelve months as of Q2 2024 was $27.71 million, with a notable quarterly revenue growth of 359.37% in Q2 2024. This substantial growth aligns with the company's progress in bringing gene therapies to market.
It's worth noting that uniQure's stock price has fallen significantly over the last three months, with a -38.09% total return. This could present an opportunity for investors who believe in the long-term potential of uniQure's gene therapy pipeline.
For those interested in a deeper dive into uniQure's financials and prospects, InvestingPro offers 10 additional tips, providing a more comprehensive analysis of the company's investment potential.
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