Enrollment in Phase 1/2a Study of IONIS-HTT Rx in Patients with Huntington's Disease Completed and Open-Label Extension Study to Open in 2H 2017
"We are encouraged by the safety profile of IONIS-HTTRx we have observed to date in the completed dosing cohorts in the Phase 1/2a study. Upon completion and full analysis of this study, the next step for this program will be to conduct a study to investigate whether decreasing mutant huntingtin protein with IONIS-HTTRx can slow the progression of this terrible disease," said
ABOUT IONIS-HTTRx and HUNTINGTON'S DISEASE (HD)
IONIS-HTTRx is an antisense drug in development for the treatment of HD. IONIS-HTTRx is designed to reduce the production of all forms of the huntingtin (HTT) protein, which in its mutated variant (mHTT) is responsible for HD. As such, IONIS-HTTRx offers a unique approach to treat all patients with HD, irrespective of their individual HTT mutation. IONIS-HTTRx has been granted orphan drug designation by the
HD is a rare genetic, progressive, neurodegenerative disease resulting in deterioration in mental abilities and physical control. In the U.S., there are approximately 30,000 symptomatic patients and more than 200,000 people at-risk of inheriting HD. HD is referred to as a triplet repeat disorder and is one of a large family of genetic diseases in which certain gene sequences are mistakenly repeated. In HD, the gene that encodes for the HTT protein contains a trinucleotide sequence that is repeated in the gene more than 36 times. The resulting mHTT protein is toxic and gradually damages neurons in the brain. Symptoms of HD usually appear between the ages of 30 to 50 years, and continually worsen over a 10 to 25-year period. Ultimately, the weakened individual succumbs to pneumonia, heart failure or other complications. Presently, there is no effective disease-modifying treatment for HD available, and current approaches only focus on managing some of the disease symptoms.
ABOUT IONIS/ROCHE COLLABORATION
Roche and Ionis are collaborating to develop antisense drugs to treat HD. The alliance combines Ionis' antisense expertise with Roche's knowledge in clinical development of anti-neurodegenerative therapeutics. To date, Ionis has earned $55 million in upfront and milestone payments from its relationship with Roche and is eligible to earn additional milestone payments as the drug progresses in development, as well as royalties on sales of IONIS-HTTRx if it is commercialized. Roche has the option to license IONIS-HTTRx from Ionis through the completion of the Phase 1/2a study. Prior to option exercise, Ionis is responsible for the discovery and development of IONIS-HTTRx. If
CHDI Foundation, Inc. provided financial and scientific support to Ionis' HD drug discovery program through a development collaboration with Ionis. Over time, CHDI will be reimbursed for its support of Ionis' program out of milestone payments received by Ionis.
Ionis is the leading company in RNA-targeted drug discovery and development focused on developing drugs for patients who have the highest unmet medical needs, such as those patients with severe and rare diseases. Using its proprietary antisense technology, Ionis has created a large pipeline of first-in-class or best-in-class drugs, with over three dozen drugs in development. SPINRAZA® (nusinersen) is a drug that has been approved in the U.S. and
IONIS' FORWARD-LOOKING STATEMENT
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Ionis Pharmaceuticals Investor and Media Contact: D. Wade Walke, Ph.D., Vice President, Corporate Communications and Investor Relations, 760-603-2741