IONIS Press Release Causes More Questions and Concerns

March 9, 2018

 

I've stayed silent...very silent, since the press release on March 1, 2018, from Ionis Pharmaceuticals, Inc.:  

 

"IONIS-HTT Rx (RG6042) Top-Line Data Demonstrate Significant Reductions of Disease-Causing Mutant Huntingtin Protein in People with Huntington's Disease"

 

The Huntington's community waited patiently for this update since the previous press release on December 11, 2017: Ionis Pharmaceuticals Licenses IONIS-HTT Rx to Partner Following Successful Phase 1/2a Study in Patients with Huntington's Disease

 

Since March 1, 2018, WeHaveAFace has received many emails and Facebook messages regarding this news/update. Inasmuch, we [as patients and caregivers] have our own questions.  On December 13, 2017, I went LIVE on WeHaveAVoice Radio because the BUZZ was utterly overwhelming across all social media platforms.  Not only was it my opinion, but it was my responsibility to ask our community to be extremely cautious about this news.  

Since then, we waited again for additional information regarding the Ionis/Roche press release.  To be quite frank, I sincerley hoped that it would broaden the conversation and bring forward many answers. The March 1st press release offered promise.  Sadly, only a portion of the questions and concerns we received were going to be answered. 

 

Actual questions from Huntington's patients and caregivers:

- Did any of the patients who participated in this trial show signs that the disease was slowing?

- What was the CAG of the patients who participated?

- Did they only accept patients with early onset of the HD?

- Did is slow or stop chorea? Depression? Anxiety?

- Will this be available for Juvenile Huntington's disease?

 

So what do we know?

The initial goal of this study was to examine safety and tolerability of the drug. It was then discovered that the IONIS-HTTRx (RG6042), could lower the mutant huntingtin protein. "Top-line IONIS-HTTRx (RG6042) Phase 1/2 Study Results: Conference Call and Webcast"(Slide 55).  Good news, right? 

As per EHDN (European Huntington's Disease Network):