In the Phase 1/2a study, dose-dependent reductions of mHTT were observed in participants treated with IONIS-HTTRx. In addition, the safety and tolerability profile of IONIS-HTTRx observed in the Phase 1/2a study supports continued development. Ionis and
"We are encouraged by the performance of IONIS-HTTRx in the Phase 1/2a clinical study. The dose-dependent reductions of mHTT we observed in the study substantially exceeded our expectations and we were equally encouraged by the safety profile of the drug," said Dr.
"We are extremely pleased that to have reached this important milestone in our collaboration with
"The results of this trial are of ground-breaking importance for Huntington's disease patients and families. For the first time, a drug has lowered the level of the toxic disease-causing protein in the nervous system, and the drug was safe and well tolerated. The key now is to move quickly to a larger trial to test whether IONIS-HTTRx slows disease progression," said Dr.
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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 U.S. Food and Drug Administration (
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 individuals with symptomatic HD and more than 200,000 people at risk of having inherited 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 trinucleotide sequence in the gene that encodes for the HTT protein is repeated 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, and current products focus only on managing 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.
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) has been approved in global markets for the treatment of spinal muscular atrophy (SMA). Biogen is responsible for commercializing SPINRAZA. Drugs that have successfully completed Phase 3 studies include inotersen, an antisense drug Ionis is developing to treat patients with hereditary TTR amyloidosis (hATTR), and volanesorsen, an antisense drug discovered by Ionis and co-developed by Ionis and Akcea Therapeutics to treat patients with either familial chylomicronemia syndrome or familial partial lipodystrophy. Akcea, an affiliate of Ionis, is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious cardiometabolic diseases caused by lipid disorders. If approved, volanesorsen will be commercialized through Ionis' affiliate, Akcea. Inotersen filings for marketing approval have been submitted in the U.S. and EU. Volanesorsen filings for marketing approval have been submitted in the U.S., EU, and
IONIS' FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding Ionis' alliance with
In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our," and "us" refers to
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D. Wade Walke, Ph.D.; Vice President, Corporate Communications and Investor Relations; 760-603-2741 OR Alissa Santa Maria; Assistant Director, Corporate Development; 760-603-2643 OR Jennifer Capuzelo; Assistant Director, Corporate Communications and Investor Relations; 760-603-2606