Isis Pharmaceuticals Initiates Clinical Study of ISIS-HTT Rx in Patients With Huntington’s Disease

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First therapy designed to directly target the cause of disease Isis Earns $22 Million Milestone Payment from Roche

Isis Pharmaceuticals, Inc.(NASDAQ: ISIS) today announced that it has initiated a Phase 1/2a clinical study of ISIS-HTTRx in patients with Huntington’s disease (HD). ISIS-HTTRx is the first therapy to enter clinical development that is designed to directly target the cause of the disease by reducing the production of the protein responsible for HD. HD is a rare genetic neurological disease in which patients experience deterioration of both mental abilities and physical control. Presently, there are no disease-modifying treatments for HD, with current therapies focused only on treating disease symptoms.   ISIS-HTTRx has been granted orphan drug designation by theEuropean Medicines Agency for the treatment of patients with HD.  Orphan drug designation is granted to products designed to diagnose, prevent or treat life-threatening or very serious conditions that affect not more than five in 10,000 persons in the European Union.

Isis Pharmaceuticals, Inc.

“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. Together withRoche, we are committed to investigating this approach to treat patients with HD, a devastating disease that typically affects generations of families,” said C. Frank Bennett, Ph.D., senior vice president of research at Isis Pharmaceuticals.

“Initiating the clinical study of ISIS-HTTRx in patients with HD is the first step in developing a treatment that could significantly impact a patient’s disease. It is also an important milestone in our collaboration with Roche. As we advance this program, we will continue to benefit from Roche’s scientific expertise in developing therapeutics for neurodegenerative conditions,” said B. Lynne Parshall, chief operating officer of Isis Pharmaceuticals.

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.  In this study, ISIS-HTTRx  will be administered intrathecally as an injection directly into the cerebral spinal fluid. Intrathecal administration of antisense drugs has been shown to be well tolerated in multiple clinical studies in patients.

“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,” said Robi Blumenstein, president of CHDI Management, which oversees the activities of CHDI Foundation, a nonprofit research organization exclusively dedicated to the development of therapies that will slow the progression of HD. “It’s exciting that therapeutic candidates grounded in the biology of Huntington’s disease are finally making their way to clinical trial.”

ABOUT ISIS and ROCHE
Roche and Isis are collaborating to develop antisense drugs to treat HD. The alliance combines Isis’ antisense expertise with Roche’s scientific knowledge in developing neurodegenerative therapeutics. With the initiation of the Phase 1/2a study for ISIS-HTTRx, Isis earned a $22 million milestone payment from Roche. To date, Isis has earned $52 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 ISIS-HTTRx if it is commercialized. Roche has the option to license ISIS-HTTRx from Isis through the completion of the Phase 1/2a study. Prior to option exercise, Isis is responsible for the discovery and development of ISIS-HTTRx. If Roche exercises its option, it will assume responsibility for global development, regulatory and commercialization activities for the drug.

CHDI Foundation, Inc. provided financial and scientific support to Isis’ HD drug discovery program through a development collaboration with Isis. Over time, CHDI will be reimbursed for its support of Isis’ program out of milestone payments received by Isis.

ABOUT ISIS-HTTRx and Huntington’s Disease
ISIS-HTTRx is a Gen. 2.0+ antisense drug in development for the treatment of Huntington’s disease. ISIS-HTTRx  is designed to reduce the production of all forms of the huntingtin protein, which is the protein responsible for HD. As such, ISIS-HTTRx offers a unique approach to treat all patients with HD. HD is a rare genetic, progressive neurological disease resulting in deterioration in mental abilities and physical control. 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 HTT 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, and current approaches only focus on managing the severity of some disease symptoms.

 

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Source: Isis Pharmaceuticals

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