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news.US-based Isis Pharmaceuticals has started a Phase I/IIa clinical trial of ISIS-HTT, a Gen. 2.0+ antisense drug, for the treatment of patients with Huntington's disease (HD), a rare genetic neurological disease.
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The company said that ISIS-HTT 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.
Currently, there are no disease-modifying treatments for HD, with current therapies focused only on treating disease symptoms.
The European Medicines Agency (EMA) has also granted orphan drug designation for ISIS-HTT to treat patients with HD.
Isis Pharmaceuticals Research senior vice-president Frank Bennett 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-HTT, the first therapeutic approach designed to treat the genetic cause of HD.
"Together with Roche, we are committed to investigating this approach to treat patients with HD, a devastating disease that typically affects generations of families."
In this randomized, placebo-controlled, dose escalation Phase I/IIa trial, ISIS-HTT will be administered intrathecally as an injection directly into the cerebral spinal fluid.
The company said that intrathecal administration of antisense drugs has been shown to be well tolerated in multiple clinical studies in patients.
Isis Pharmaceuticals chief operating officer Lynne Parshall said: "Initiating the clinical study of ISIS-HTT 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."
The drug is being developed as part of a collaboration which combines Isis’ antisense expertise with Roche’s scientific knowledge in developing neurodegenerative therapeutics.