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Expert Interview

Slingshot members are talking to an expert! The topic is:

Examining uniQure's AMT-130 and PTC Therapeutics' PTC518: Data on Slowing Huntington's Disease Progression

Ticker(s): QURE, PTCT

Who's the expert?

Institution: Novant Health Matthews Medical Center

  • Outpatient neurologist and Director of Memory Care 
  • Manages 300-400 patients with Alzheimer's Disease
  • Manages 10 patients with Huntington's Disease

Interview Questions
Q1.

 The interim data from the AMT-130 trial demonstrated an 80% slowing of disease progression in the composite Unified Huntington's Disease Rating Scale (cUHDRS) at 24 months for high-dose patients. Can you elaborate on the significance of these findings for the HD community?

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Q2.

The study showed a statistically significant reduction of cerebrospinal fluid (CSF) neurofilament light protein (NfL) levels in patients treated with AMT-130. How does the reduction of NfL correlate with the slowing of neurodegeneration in HD?

Added By: catalin_admin
Q3.

Given that AMT-130 showed near-baseline stability in motor and cognitive function over 24 months, what potential long-term benefits can patients expect from this treatment?

Added By: catalin_admin
Q4.

AMT-130 has been generally well-tolerated with a manageable safety profile. Can you discuss any specific adverse events observed and how they were managed?

Added By: catalin_admin
Q5.

The PIVOT-HD study indicated dose-dependent lowering of mutant huntingtin (mHTT) protein in both blood and CSF. What implications does this have for dose optimization in future treatments?

Added By: catalin_admin
Q6.

PTC518 showed favorable trends on HD clinical assessments, including the Total Motor Score (TMS). How do these trends translate into real-world improvements in daily functioning for HD patients?

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Q7.

With uniQure's AMT-130 receiving the first-ever Regenerative Medicine Advanced Therapy (RMAT) designation for HD and the FDA lifting the partial clinical hold on PTC518, what are the next regulatory steps for these therapies?

Added By: catalin_admin
Q8.

How do the clinical and biomarker outcomes of AMT-130 compare to those observed with PTC518, and what does this mean for future treatment strategies in HD?

Added By: catalin_admin

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