Soin Neuroscience Seeks Orphan Drug Status for Scleroderma Therapy TV1001SR

Soin Neuroscience recently submitted an orphan drug application to the U.S. Food and Drug Administration (FDA) for the company’s investigational drug TV1001SR (sodium nitrite), being developed for the treatment of scleroderma.

Several completed clinical and preclinical trials have demonstrated TV1001SR enhances the growth of small arteries and promote blood flow. This mechanism of action potentially could be helpful for people living with scleroderma, the company believes.

According to the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), estimates for the number of people in the U.S. with the systemic form of scleroderma range from 40,000 to 165,000. The number of people with all scleroderma-related disorders ranges between 250,000 and 992,500.

“Our modeling suggests that patients suffering from systemic sclerosis will see a marked improvement in blood flow and help reverse or prevent damage to the small arteries that are damaged by the disease,” Amol Soin, MD, founder and CEO of Soin Neuroscience, said in a press release.

The company noted that patients with scleroderma often develop damage to their internal organs due to poor blood flow, and also may develop painful and debilitating ulcers due to the damage to small blood vessels.

“Another major advantage is that TV1001SR has been shown to be a good painkiller in patients who suffer from damage to small arteries. Given the non-addicting and non-sedating nature of the medication, it can truly help people who are suffering, without the side effects of other painkillers,” Soin added.

“The mechanism of action of TV1001SR appears to treat the actual cause of the pain and should also improve the symptoms a patient experiences, while at the same time helping to improve the blood flow that was lost due to damage to the small blood vessels. I really think we can help a lot of people,” Soin concluded.

Soin Neuroscience is expecting a response from the FDA on the orphan drug application in four to six months. Soin’s research team is planning a Phase 3 clinical trial to evaluate TV1001SR in scleroderma patients; the trial is expected to initiate dosing patients in the next year.