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uniQure Provides Update on Low-Dose Cohort in P-I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease

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uniQure Provides Update on Low-Dose Cohort in P-I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease

uniQure Provides Update on Low-Dose Cohort in P-I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease

Shots:

  • The company reported the safety & biomarker data from an ongoing P-I/II trial to evaluate AMT-130 in 10 patients (6 received AMT-130 & 4 with imitation surgical procedure) with HD for 12mos.
  • The results showed that therapy was found to be well tolerated at the lower dose of 6x1012 vector genomes with no serious AEs @1yr. of follow-up, reduction in mean CSF NfL & 8% increase over baseline in 6 treated patients while remained stable or slightly declined in 4 patients
  • 53.8% mean reduction of mHTT in CSF @12mos. in 4 patients while increased over baseline @ 1/3/6/9mos. & reduced by 16.8% in 3 patients. The additional data includes 24 & 12 mos. follow-ups in the low & high dose US cohort are expected in H1’23

Ref: GlobalNewswire | Image: uniQure 

Click here to­ read the full press release 

Neha

Neha is a Senior Editor at PharmaShots. She is passionate and very enthusiastic about recent updates and developments in the life sciences and pharma industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots. She can be contacted at connect@pharmashots.com.

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