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uniQure (QURE) Falls on Data From Huntington's Disease Study

·4 min read

uniQure N.V. QURE has announced data on AMT-130, a potential one-time gene-therapy for Huntington’s disease. Shares fell 29.6% in response as investors weren’t impressed.

The company released initial data on the first four patients enrolled in the lower-dose cohort of its ongoing phase I/II clinical trial of AMT-130 for the treatment of the disease.

The treatment was well tolerated with no significant safety issues related to AMT-130 in the first two treated patients through one year of follow-up.

Per the data, two of the four enrolled patients received AMT-130, and two patients experienced an imitation (sham) surgery in this randomized, blinded clinical trial being conducted in the United States.

Neurofilament Light Chain (NfL) rose as expected immediately following surgery and returned to baseline in treated patients.

These patients were a heterogeneous group representing a spectrum of Stage I-II early manifest disease with 41-44 CAG repeats, baseline Total Functional Capacity scores of 10-13 and Total Motor Scores of 7-23.

The gene therapy was generally well-tolerated in the treated patients at the lower dose. The safety profile of AMT-130 in the low-dose cohort to date supports continued enrollment of patients in the higher-dose cohort. Nineteen patients have been enrolled, including nine of 16 in the higher-dose cohort.

An update on the low-dose cohort of 10 patients, largely focused on safety, is expected in the second quarter of 2022 after patient unblinding. Full safety and efficacy data from the first two cohorts in the phase I/II study are expected in the first half of 2023 after all patients in the higher-dose cohort have achieved one year of follow-up.

uniQure plans to initiate a third cohort in the ongoing study in the second half of 2022 that will explore the use of alternative stereotactic navigation systems to simplify placement of catheters for infusions of AMT-130. The European, open-label phase Ib/II study of AMT-130 will enroll 15 patients with early manifest Huntington’s disease across two dose cohorts. The screening has also been initiated in the European open-label phase I/II study.

uniQure’s shares have surged 44% so far this year against the industry's decline of 20.8%.

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Last month, the company announced positive recommendation by the independent Data Safety Monitoring Board (DSMB) following a review of safety data from the four patient procedures in the higher-dose cohort of the phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease. With the positive recommendation, the final 12 patients in this second cohort are now cleared for enrollment.

Based on recommendations of the DSMB and steering committee, the company plans to disclose efficacy data, including volumetric MRI changes, once all patients in the first two cohorts are unblinded.

Gene therapies are promising but are complex to develop. Quite a few companies are developing gene therapies for various diseases.

uniQure currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the healthcare sector are Sarepta Therapeutics, Inc. SRPT, Editas Medicine EDIT and Viking Therapeutics VKTX, 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.

Estimates for Sarepta have narrowed from a loss per share of $6.95 to $4.99 for 2021 and from $4.78 to $3.61 for 2022 in the past 60 days. SRPT delivered an earnings surprise of 11.06%, on average, in the last four quarters.

Estimates for Viking Therapeutics have narrowed to a loss per share of $3.16 from $3.55 for 2021 and to $3.47 from $3.63 for 2022 in the past 60 days. VKTX delivered an earnings surprise of 2.06%, on average, in the last four quarters.

Estimates for Editas Medicine have narrowed to a loss per share of $3.08 from $3.59 for 2021 and to $3.40 from $3.64 for 2022 in the past 60 days. EDIT delivered an earnings surprise of 0.83%, on average, in the last four quarters.

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