Gene therapy company uniQure N.V. (QURE:NASDAQ), which is focused on developing single gene therapy treatments that might potentially offer curative results for severe genetic diseases of the central nervous system and liver including Huntington's disease, ALS, Alzheimer's, Fabry disease, hemophilia B, Parkinson's and refractory temporal lobe epilepsy, today announced "safety and biomarker data from 10 patients enrolled in the low-dose cohort of the ongoing Phase I/II clinical trial of AMT-130 for the treatment of Huntington's disease."
The firm advised that of the 10 randomized patients participating in the U.S. based study, 6 were administered AMT-130 and the remaining 4 patient subjects underwent an imitation (placebo-like) surgical procedure.
uniQure's President of R&D Ricardo Dolmetsch, Ph.D. commented, "We are encouraged by this 12-month update on the patients enrolled in the low-dose cohort…Thus far in the clinical trial, AMT-130 has been well-tolerated, with no serious adverse events related to the gene therapy and NfL levels approaching baseline."
"We are pleased to observe trends suggesting target engagement that are supported by the lowering of mHTT protein in evaluable patients receiving AMT-130 and we look forward to presenting additional clinical data, including functional outcomes, on all patients from this important study next year," Dolmetsch added.
The company explained that all 10 adult patients (4 men, 6 women) between the ages of 34 and 58 years of age in the trial's low-dose group were identified as having early-stage Huntington's disease having been clinically diagnosed with CAG repeats between 40-44, baseline Total Functional Capacity scores of 10-13, and Total Motor Scores of 7-23. The firm stated that all 10 participants were evaluable for NfL measurements. Of the 10 individuals, 7 patients were evaluable for mHTT protein in the CSF measurements, 4 of whom received treatment with AMT-130 and 3 who were not dosed in the control group. uniQure explained that the three other patients did not have a measurable baseline CSF mHTT value.
The company reported that after 12 months of follow-up monitoring of the six treated patients in the low-dose cohort, AMT-130 was shown to be well-tolerated at the lower dose of 6x1012 vector genomes and no serious AMT-130 specific adverse events or drug reactions were recorded. Additionally, uniQure mentioned that after one year following treatment structural MRIs did not reveal any demonstrable clinical safety findings.
The firm highlighted that the results from the low-dose cohort showed "a mean reduction of 53.8% of mutant HTT (mHTT) observed in cerebral spinal fluid (CSF) at 12 months in evaluable patients treated with AMT-130."
The company's CEO Matt Kapusta remarked, "We have made excellent progress in our clinical investigation of AMT-130 and now have a total of 36 patients enrolled across our two Phase I/II clinical studies in the U.S. and Europe…Enrollment in the high-dose cohort of our open-label European study is well underway and is expected to be completed by the end of this year. We look forward to continued collaboration with the Huntington's disease community to advance AMT-130 as a potential treatment for those living with Huntington's disease."
The firm advised that the Phase 1/2 study was designed to evaluate the safety, tolerability and efficacy of AMT-130 in treating Huntington's disease. The randomized trial enrolled a total of 26 patients who were split into a 10-person low-dose cohort and a 16-person high-dose cohort.
uniQure noted that it intends to add a third cohort of 18 participants prior to the end of next year in order to explore using alternative stereotactic navigation systems to simplify placement of catheters for AMT-130 infusions. It is expected that the treated portion of this third cohort will each be given the higher dose of 6x1013 vg.
The company explained that "Huntington's disease is a rare, inherited neurodegenerative disorder that leads to motor symptoms including chorea, and behavioral abnormalities and cognitive decline resulting in progressive physical and mental deterioration." The firm noted that at present there on no approved medicines to prevent or mitigate progression of the disease.
uniQure is a gene therapy firm headquartered in Amsterdam that is working to create and develop single-application treatments with potential curative results to treat genetic and other devastating diseases. The firm uses its technically validated modular platform to rapidly advance its pipeline of adeno-associated virus (AAV)-based gene therapies that are being developed to treat numerous central nervous system (CNS) and liver related diseases such as hemophilia B, Huntington's disease, refractory temporal lobe epilepsy, Fabry disease and several others. In addition, the company is now working on four undisclosed programs in the area of cardiovascular and muscles diseases.
uniQure started the day with a market cap of around $717.9 million with approximately 46.6 million shares outstanding and a short interest of about 8.2%. QURE shares opened 11% higher today at $17.07 (+$1.68, +10.92%) over yesterday's $15.39 closing price. The stock has traded today between $17.01 and $19.59 per share and is currently trading at $19.45 (+$4.06, +26.38%).
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