http://www.raredr.com/news/rns60-orphan-drug-designation
FDA Pushes ALS Drug Through the Pipeline with Orphan Drug Designation

Mathew Shanley

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Revalesio’s investigational drug RNS60 to treat amyotrophic lateral sclerosis (ALS).

A phase I study is ongoing to evaluate the safety and tolerability of the therapy in patients with ALS.

Commonly referred to as “Lou Gehrig’s Disease,” ALS is a fatal, neurodegenerative condition in which patients’ muscle functionality is progressively lost. Patients with the condition gradually lose the ability to move, speak, swallow, and breathe. Death is usually expected within 3-5 years of diagnosis.
The only currently-approved treatment for the disease is Radicava (edaravone). Per the NIH, ALS affects approximately 12-15,000 patients in the United States and an estimated 6,000 new patients are diagnosed annually.

A clinical trial of RNS60 in ALS (NCT02525471) was initiated at Massachusetts General Hospital in October 2015 and has an estimated completion date in March 2018. 18 patients are currently enrolled to assess the following outcome measures: safety, tolerability, blood biomarkers of inflammation, neuroimaging biomarkers, pulmonary function, strength, and functional status.

RNS60 does not contain an active pharmacological agent. Rather, it is an oxygenated saline solution that is believed to create charge-stabilized nanostructures that improve cellular function in a number of neurological and musculosketal diseases.

“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,” said Greg Archambeau, President of Revalesio in a press release. “Our treatment impacts the underlying energetic deficits in cells, rather than a single molecular target. 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.”

RNS60 is first administered via either intravenous infusion (375ml) once a week and then by inhalation (4ml) for the remaining 6 days of the week. The administration will continue for 24 weeks. Subjects in the study have the option to continue to receive the drug for an additional 24 weeks, making the duration of the study 48 total weeks. The same dosages apply to the extension phase.

RNS60 has been comprehensively tried in preclinical toxicological studies and has displayed very few side effects. Additionally, RNS60 was well tolerated in 3 phase I human safety studies; 1 following intravenous administration and 2 after inhalation.

A Phase 2 study evaluating nebulized RNS60 for the treatment of ALS is expected to begin early in the first quarter of 2019, and will evaluate the ALS functional rating scale-revised (ALSFRS-R) score as a primary outcome.

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