South Side-Based Knopp Lands $345M Deal for Lou Gehrig's Disease Drug

Article excerpt

A Pittsburgh research company said Wednesday it signed a deal potentially worth $345 million for final-stage development and commercialization of its treatment for Lou Gehrig's disease and possibly other degenerative nerve disorders.

Knopp Neurosciences Inc., on the South Side, announced the deal with Biogen Idec Inc., of Weston, Mass., on its new drug dexpramipexole, also known as KNS-760704, for treating amyotrophic lateral sclerosis, or ALS.

Some longtime investors in the 6-year-old Knopp compared its breakthrough research to another discovery made locally 55 years ago concerning another debilitating disease: Jonas Salk's discovery of a polio vaccine.

"This is the first drug ever to show the ability to slow the progression of ALS, to slow the rate of functional loss, potentially allowing the patient to walk, talk, eat and breathe better and longer," said CEO Michael Bozik at a news conference Downtown.

The deal is the latest in a series of significant investment deals won by Pittsburgh technology companies.

ALS causes progressive weakness in a patient due to the degeneration of nerve cells that control a body's voluntary muscles. "It's a progressive paralysis of the body while a person's mind stays intact," Bozik said.

ALS is always fatal, and patients generally die within two years of definitive diagnosis, but the Knopp drug slows the course of the disease by as much as 50 percent, Bozik said.

The drug penetrates a patient's nervous system and promotes energy production in nerve cells which, when stressed by ALS, require a great deal of energy, said Tom Petzinger, Knopp's executive vice president of business development and public affairs.

Petzinger is a founder of Pittsburgh life sciences company incubator Launchcyte LLC, which helped create and invested in Knopp.

"In the 31 counties of Western Pennsylvania, we generally work with 300 ALS patients and their families," said Michael Bernarding, executive director of the ALS Association of Western Pennsylvania, who spoke at the news conference.

Bernarding called the Knopp drug "exciting," saying that while ALS has been diagnosed in patients for 200 years, there is just one drug approved by the Food and Drug Administration for treating ALS, a drug Bernarding called "not very effective."

Nationwide, 20,000 to 30,000 people suffer from ALS, with about 5,000 diagnosed annually, Bernarding said.

Under terms of the agreement, Biogen will lead development of the drug and its potential commercialization worldwide, while Knopp will provide development support and conduct commercialization activities under Biogen's direction. …