UniQure and PTC Therapeutics both share promising results from current drug trials.

uniQure Announces Update on U.S. Phase I/II Clinical Trial of AMT-130 Gene Therapy for the Treatment of Huntington’s Disease

Today UniQure announced promising interim data from the Phase I/II trial of their #GeneTherapy AMT-130. The encouraging update shows early signs of potential clinical benefit and supportive trends in NfL, a key marker of neuronal damage. Patients treated with either dose of AMT-130 appear to have largely preserved function and are trending favourably to natural disease course at up to 24 months. They believe these interim results provide early hope for patients with Huntington's disease.

UniQure is re-encouraged by the results and plans to continue and extend the program.

Read UniQure press release

PTC Therapeutics Shares Positive Interim Data from PIVOT-HD Clinical Trial in Huntington's Disease Patients

Interim data showed:

  • Dose-dependent lowering of blood Huntingtin (HTT) protein levels at 12 weeks 
  • Favorable tolerability profile with no treatment-related serious adverse events or NfL spikes (Neurofilament light chain (NfL) holds promise as a biomarker for disease progression and prognosis in Huntington's disease)

Dr. Matthew Klein, Chief Executive Officer, PTC Therapeutics, Inc. said:

"We are very pleased with the encouraging data from the PIVOT-HD interim analysis demonstrating dose-dependent HTT lowering, desired CSF exposure and a favourable tolerability profile without evidence of treatment-related serious adverse events or CSF NfL spikes"

Read PTC Therapeutics press release

We will share more details when we have them, this includes details of a webinar organised by UniQure and the Huntington’s Disease Society of America.

You can read more in the HD Buzz article.