Roche and AAN Huntington's Community Statement

April 20, 2018

I wish to thank Mai-Lise Nguyen (Patient Partnership and Advocacy - Roche, Switzerland), for reaching out to us at WeHaveAFace with the following letter regarding their RG6042 program update.


RG6042 (formerly known as IONIS-HTTRx ) development program update Presentation of Phase I/IIa study at world’s largest neurology conference, thank you to community



Dear global Huntington’s disease community,


Next week will be another milestone for the development program of the investigational medicine RG6042 (formerly known as IONIS-HTTRx ). On 24 April 2018 results from the RG6042 Phase I/IIa study in Huntington’s disease (HD) will be featured on the main stage of the American Academy of Neurology (AAN) annual meeting, the world’s largest forum for neurology research, which is attended by over 12,000 professionals from around the globe.  


We wanted you to be aware that news coverage about this study may occur over the next days. Out of more than 3,000 scientific submissions to AAN, the RG6042 Phase I/IIa study results is one of four presentations selected to be part of the meeting’s top-featured session. HD will receive a spotlight in the neurology community next week, and we would like to give special acknowledgements to (as Dr. Sarah Tabrizi said during her presentation at the CHDI conference) the “true research heroes”- the 46 participants of the Phase I/IIa study. These incredible individuals and their families had the bravery and commitment to join this first-in-human study and advance scientific progress.  


We are honored to work with trial investigators and Ionis Pharmaceuticals to share results of the world’s first drug study designed to reduce huntingtin protein at such prominent scientific forums as AAN and last month’s CHDI conference, where the data were first debuted. It is a testament to the broad interest in HD and the increasing hope for effective treatments. More importantly, it is a testament to the collaboration and support of the HD community.


What’s happened? 


• The Phase I/IIa study will be presented on 24 April at the AAN annual meeting. Overall results are similar to those previously announced at the CHDI conference.

- This was a 13-week, first-in-human study evaluating safety and tolerability, where 46 participants received four doses every 28 days.

- The study showed RG6042 was safe and well-tolerated at all doses.

- The study was not designed, or expected, to show an effect on clinical symptoms.

- Exploratory analyses showed that RG6042 lowered levels of mutant huntingtin protein, the protein that causes Huntington’s disease, in a dose-dependent manner.


• Based on the encouraging signals observed in this study, the development of RG6042 continues. Further research will focus on determining if RG6042 provides a meaningful clinical benefit and if lowering the mutant protein changes the course of HD.


This is an exciting time for HD, but there is still much work to be done. More research and larger studies are needed to determine if RG6042 can slow the relentless progression