uniQure (QURE) Falls on Mixed Huntington's Disease Study Results
uniQure Inc. QURE announced interim results from its ongoing phase I/II study of AMT-130 for the treatment of Huntington’s disease after 24 months of follow-up. The phase I/II study of AMT-130, a one-time administered investigational gene therapy, is being carried out in the United States, in two cohorts, on 26 patients with early-manifest Huntington’s disease.
The first cohort comprised 10 patients receiving a low dose strength of AMT-130 (6 treated, 4 control). The second cohort encompassed the remaining 16 patients receiving a high dose strength of AMT-130 (10 treated, 6 control). All patients received either of the two AMT-130 dose strengths or an imitation (sham) surgery.
uniQure reported that both doses of AMT-130 demonstrated preliminary evidence of clinical and functional benefits, including favorable trends in Total Motor Score, Total Functional Capacity and the composite Unified Huntington’s Disease Rating Scale compared with natural history. The candidate was overall well-tolerated with a manageable safety profile at both doses.
However, mixed biomarker results were observed in the two dose groups of the study, with respect to the Neurofilament light chain (NfL) protein and Mutant Huntingtin protein (mHTT) levels in cerebrospinal fluid (CSF). A high NfL count is an indicator of worsening neurodegenerative disease, while high levels of mHTT protein, which is a driver of Huntington's disease, signifies a deteriorating condition of the patient.
As a result, uniQure’s stock fell 40% on Wednesday, marking investors’ concerns regarding the significantly high CSF NfL and mHTT proteins in the high-dose group of the study, despite encouraging results from the low-dose group. Year to date, shares of QURE have plunged 48.7% compared with the industry’s decline of 7.2%.
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Upon examination of the CFS NfL levels in the two cohorts of the study, it was observed that the low-dose group experienced a 12.9% decrease in NfL after two years, while the high-dose group experienced a 51.5% increase after a year. Only two high-dose patients were reported to have demonstrated a continued decline in CSF NfL to 27.4% above baseline at the end of 18 months. Hence, patients in the high-dose group showed alarmingly high NfL levels while both the low-dose group and the control group observed a decline in NfL levels at 12 months.
Similarly, upon examination of CSF mHTT, it was observed that the low-dose group experienced a reduction of 8.1% at 24 months, while the high-dose group witnessed an average increase of 39.7% above baseline in one year. CSF mHTT level increased by 4.7% in the control group.
uniQure is currently planning its next steps regarding the development of AMT-130. The company anticipates completing enrollment in the high-dose cohort of the AMT-130 study in Europe in early third-quarter 2023. In the second half of 2023, the company expects to initiate a third cohort in the ongoing phase I/II study of AMT-130 to further investigate both doses in combination with perioperative immunosuppression to evaluate near-term safety. Up to 10 patients are anticipated to be enrolled in the third cohort.
New data from both the U.S. and EU studies are expected in the fourth quarter of 2023. QURE also plans on meeting the respective regulatory authorities to discuss data from the U.S. and EU studies to determine the pathway for further advancing the clinical development of AMT-130.
uniQure N.V. Price and Consensus
uniQure N.V. price-consensus-chart | uniQure N.V. Quote
Zacks Rank and Stocks to Consider
uniQure currently has a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Adaptimmune Therapeutics ADAP, Akero Therapeutics AKRO and ADMA Biologics, Inc. ADMA, each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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ADMA beat estimates in each of the trailing four quarters, delivering an average earnings surprise of 19.13%.
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