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Stephen Hawking
British physicist Stephen Hawking, shown here during a 2016 interview with Larry King, is almost completely paralyzed due to ALS but still leads an active professional life (Credit: Ora.TV)

Revalesio, a biomedical company headquartered in Tacoma, Wash., says it’s been cleared to benefit from federal incentives for the development of a drug that could help treat a neurodegenerative disease known as amyotrophic lateral sclerosis, or ALS.

ALS is known as Lou Gehrig’s disease, but today’s most famous patient is British physicist Stephen Hawking, who was diagnosed with the malady more than 50 years ago.

The disease affects 12,000 to 15,000 patients in the U.S., and an estimated 6,000 Americans are diagnosed with ALS every year.

Only two drugs have been approved for ALS since 1995. One was cleared for use in treatment this year. Revalesio’s investigational drug, RNS60, has been approved for clinical evaluation in the U.S. and Europe.

In preclinical trials, RNS60 has shown anti-inflammatory and protective effects in mouse models for ALS and multiple sclerosis. The clinical trials to date indicate that the drug is well-tolerated in human subjects.

This week Revalesio announced that the FDA has granted orphan drug designation for RNS60. Such designations are given to boost the development of drugs for rare diseases and disorders that affect fewer than 200,000 people in the U.S.

The designation opens the way for tax credits, reduced fees and other incentives as RN560 goes through what’s expected to be years of trials.

“With orphan status, we have increased access to FDA to help facilitate RNS60’s drug development plan and, hopefully, bring RNS60 to patients who are suffering from ALS,” Revalesio President Greg Archambeau said in a news release.

Revalesio’s latest study, involving 142 patients at Massachusetts General Hospital and at 20 clinical centers in Italy, is being funded in part by a $1 million grant from the ALS Association as an outgrowth of the Ice Bucket Challenge. Other partners in the study include the Northeast ALS Consortium and ALS Finding a Cure.

Amyotrophic lateral sclerosis is a progressive degenerative disorder affecting large motor neurons in the brain and spinal cord. As the disease progresses, patients suffer muscle wasting and progressive paralysis. Average life expectancy for an ALS patient is three to five years from time of diagnosis, the ALS Association says.

Lou Gehrig, the Yankee baseball slugger who brought the disease to attention in 1939, died two years after his diagnosis. In contrast, Hawking is still alive 54 years after being diagnosed with ALS at the age of 21. The early onset of Hawking’s symptoms and his survival to the age of 75 are highly unusual.

Archambeau said RNS60 takes advantage of a novel treatment strategy.

“Our treatment impacts the underlying energetic deficits in cells, rather than a single molecular target,” Archambeau said. “Having personally seen the devastating effects of ALS in a Revalesio colleague, we are motivated to find treatments that improve the health and quality of life for patients with ALS.”

Privately held Revalesio was founded in 2000 and has raised more than $38 million in three funding rounds, according to filings with the Securities and Exchange Commission.

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