Rare Disease Report

ISIS Starting Phase 1/2a Huntington's Disease Trial

JULY 21, 2015
James Radke, PhD
Drug targets the underlying cause of the genetic condition.

New hope for patients with Huntington’s disease. Isis Pharmaceuticals has announced the initiation of a phase 1/2a clinical study of ISIS-HTTRx in patients with Huntington's disease. ISIS-HTTRx uses antisense technology to block the synthesis huntingtin protein—the protein responsible for Huntington's disease.

Currently, there are no treatments available that target the pathophysiology of Huntington’s disease and if successful, this treatment could represent a major breakthrough for this devastating neurological condition.

C. Frank Bennett, PhD, senior vice president of research at Isis Pharmaceuticals said, "Although the toxic protein produced from the huntingtin (HTT) gene in HD patients has been a target of interest for many years, no therapies have advanced to clinical trials to treat the underlying cause of the disease. Our antisense technology has enabled us to discover and develop ISIS-HTTRx, the first therapeutic approach designed to treat the genetic cause of HD.”

Isis’ development of ISIS-HTTRx included collaborative efforts from both big pharma and advocacy.

Isis is partnering with Roche to develop ISIS-HTTRx. With the initiation of the phase 1/2a study for ISIS-HTTRx, Isis earned a $22 million milestone payment from Roche.

In addition, CHDI Foundation, a advocacy group focused on finding a cure for Huntington’s disease, provided financial and scientific support to Isis. CHDI will be reimbursed for its support of Isis' program out of milestone payments received by Isis.

Robi Blumenstein, of the CHDI Foundation said "The initial development of this antisense drug for Huntington's disease came out of a longstanding productive partnership between Isis and CHDI, and its advancement now to clinical trial is testament to Isis' perseverance and scientific expertise.”

The randomized, placebo-controlled, dose escalation Phase 1/2a clinical study will evaluate the safety and activity of ISIS-HTTRx  in patients with early stage HD.  

Huntington’s Disease and ISIS-HTTRx

Huntington's Disease is an inherited brain disorder that results in the progressive loss of both mental and motor control. Symptoms usually appear between the ages of 30 to 50, and worsen over a 10 to 25 year period until the patient eventually dies. In Huntington’s disease, the gene that encodes for the huntingtin protein contains a trinucleotide sequence that is repeated in the gene multiple times resulting in the huntingtin protein being toxic to neurons. ISIS-HTTRx is a Gen. 2.0+ antisense drug designed to reduce the production of all forms of the huntingtin protein.


Isis Pharmaceuticals Initiates Clinical Study of ISIS-HTT Rx in Patients With Huntington's Disease [press release]. Carlsbad, CA: Isis Pharmaceuticals; July 21, 2015. http://www.prnewswire.com/news-releases/isis-pharmaceuticals-initiates-clinical-study-of-isis-htt-rx-in-patients-with-huntingtons-disease-300115250.html

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