|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 2018|
CARLSBAD, Calif., June 22, 2017 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) today announced the completion of enrollment in the Phase 1/2a randomized, placebo-controlled, dose escalation study of IONIS-HTTRx in patients with Huntington’s disease (HD). Dosing in the final patient cohort continues, and Ionis plans to report top-line results from this study around year-end 2017. The safety and tolerability profile of IONIS-HTTRx in the completed cohorts of the Phase 1/2a study supports its continued development. Patients who complete the Phase 1/2a study will be eligible to participate in an open-label extension (OLE) study that Ionis plans to initiate in the next several months. Roche, Ionis’ partner for this drug, continues to advance and support the program. IONIS-HTTRx is the first therapy in clinical development targeting the cause of HD by reducing the production of the toxic mutant huntingtin protein (mHTT) from the mutated huntingtin gene.
“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 C. Frank Bennett, Ph.D., senior vice president of research at Ionis Pharmaceuticals. “We believe that IONIS-HTTRx, which is designed to reduce the production of all forms of the huntingtin (HTT) protein – the known cause of HD, represents the most promising opportunity to address this significant unmet medical need. Together with our partners at Roche, we are committed to developing IONIS-HTTRx, which has the potential to transform the treatment of HD.”
ABOUT IONIS-HTTRx and HUNTINGTON’S DISEASE (HD)
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
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.
ABOUT IONIS PHARMACEUTICALS, INC.
IONIS’ FORWARD-LOOKING STATEMENT
In this press release, unless the context requires otherwise, “Ionis,” “Company,” “we,” “our,” and “us” refers to Ionis Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals, Inc. SPINRAZA® is a registered trademark of Biogen.
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SOURCE Ionis Pharmaceuticals, Inc.
Ionis Pharmaceuticals Investor and Media Contact: D. Wade Walke, Ph.D., Vice President, Corporate Communications and Investor Relations, 760-603-2741