Roche & Isis Join Forces on Antisense Drug for Huntington’s Disease

Brain, Huntington's disease, Roche, Isis Pharmaceuticals

San Diego’s Isis Pharmaceuticals (NASDAQ: [[ticker:ISIS]]) and Roche, the Swiss Pharmaceuticals giant) have formed an alliance to develop new treatments for Huntington’s disease, according to a statement released today. Roche agreed to pay Isis $30 million upfront, with potential revenue from licensing and milestone payments totaling $362 million. In addition, Isis would get tiered royalties on potential drug sales.

Isis, which is actually based north of San Diego in Carlsbad, CA, specializes in antisense therapies, developing drugs that target harmful proteins produced by mutated genes that are responsible in certain types of disease. By synthesizing a strand of nucleic acid that binds to the mutated gene’s messenger RNA (mRNA), antisense drugs block protein production.

There is currently no effective treatment or cure for Huntington’s disease, an inherited genetic brain disorder that results in the progressive loss of both mental faculties and physical control. The goal of current therapies is to slow symptoms, which include behavioral changes, abnormal movements, and dementia, and to help patients function for as long and as comfortably as possible.

Huntington’s occurs in about five to 10 out of every 100,000 people from Western Europe. It is estimated that about 30,000 people in the United States are affected. Symptoms usually appear between the ages of 30 to 50, and progressively worsen over a 10- to 25-year period.

Initially, research will focus on Isis’ lead drug candidate that blocks production of all forms of the huntingtin (HTT) protein, the protein responsible for Huntington’s disease.

In a statement, Isis COO B. Lynne Parshall says, “We believe that Roche’s expertise in developing CNS drugs, along with their clinical development experience, will greatly enhance our development efforts for this program and allow us to move forward more rapidly. In addition, by utilizing Roche’s brain shuttle technology with our antisense drug discovery capabilities, we have the potential to significantly improve the therapeutic potential for this program.”

Author: Bruce V. Bigelow

In Memoriam: Our dear friend Bruce V. Bigelow passed away on June 29, 2018. He was the editor of Xconomy San Diego from 2008 to 2018. Read more about his life and work here. Bruce Bigelow joined Xconomy from the business desk of the San Diego Union-Tribune. He was a member of the team of reporters who were awarded the 2006 Pulitzer Prize in National Reporting for uncovering bribes paid to San Diego Republican Rep. Randy “Duke” Cunningham in exchange for special legislation earmarks. He also shared a 2006 award for enterprise reporting from the Society of Business Editors and Writers for “In Harm’s Way,” an article about the extraordinary casualty rate among employees working in Iraq for San Diego’s Titan Corp. He has written extensively about the 2002 corporate accounting scandal at software goliath Peregrine Systems. He also was a Gerald Loeb Award finalist and National Headline Award winner for “The Toymaker,” a 14-part chronicle of a San Diego start-up company. He takes special satisfaction, though, that the series was included in the library for nonfiction narrative journalism at the Nieman Foundation for Journalism at Harvard University. Bigelow graduated from U.C. Berkeley in 1977 with a degree in English Literature and from the Columbia University Graduate School of Journalism in 1979. Before joining the Union-Tribune in 1990, he worked for the Associated Press in Los Angeles and The Kansas City Times.