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This biotech founder has a new plan to treat Tourette Syndrome, which affects 200,000 Americans each year

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Jeff Aronin is the CEO of Paragon Biosciences and founder of Emalex Biosciences.Jeff Aronin, CEO of Paragon Biosciences and founder of Emalex Biosciences.

Paragon Biosciences

  • Emalex Bioscience is developing a new treatment for Tourette Syndrome.
  • The disease affects around 1 in 160 children and has no cure.  
  • Emalex founder Jeff Aronin says clinical trial results for a new medication will be out by 2025. 

Tourette Syndrome, the nervous system disorder that causes tics, or rapid and repetitive twitches and sounds that are involuntary, can be a debilitating condition. 

While there are no reliable estimates of how common Tourette Syndrome is in adults, the Tourette Association of America estimates that the disease occurs in more than 1 in 160 children and that it is more common in boys than girls. The American Brain Foundation estimates that around 200,000 Americans have the most severe form of the condition.

Tourette Syndrome currently has no cure and few treatment options. According to  a survey of more than 1,100 people with Tourette Syndrome conducted by the Tourette Association of America, many patients report trying several medications to try and control their symptoms, but say they don’t feel current drugs do a good enough job at remedying their condition. 

Jeff Aronin, CEO of life sciences accelerator Paragon Biosciences, told Insider that Tourette Syndrome as a condition stood out to him because the disorder can be physically painful for patients but it is also socially challenging. According to the Tourette Association survey, half of adults with the condition said that tics prevented them from forming meaningful friendships or romantic relationships, and 27% reported attempting suicide in the past 12 months.

Aronin realized there was an unmet need where he could step in. He told Insider that while most drug developers find a molecule in a lab that could potentially treat a certain illness and then pursue that molecule through clinical trials, he’s taken the opposite approach. Instead, he and his team at Paragon identify an illness that needs more attention, and then work backwards from there to figure out what kind of drug might work best to treat that illness.

That’s how in 2018 he came to found Emalex Biosciences, a pharmaceutical startup, to test a new drug for Tourette Syndrome.

“The suffering, the misunderstanding of how serious and significant it is, I think really put it over the top,” he said. “People who aren’t patients don’t understand how impactful this can be and that really hit home.”

Emalex’s drug works in a different way than other Tourette treatments 

In spite of a tough market for biotech startups, Emalex has managed to raise $250 million from investors in November to develop its Tourette Syndrome drug candidate, called ecopipam. 

Many of the current approved therapies for Tourette Syndrome target D1 dopamine receptors in the brain. Dompamine is a neurotransmitter which is thought to affect compulsions and tics. While ecopipam also targets dopamine, it works on a different receptor than conventional treatments — the D2 receptor

The drug has received a Fast Track designation from the FDA, meaning that it could move through the agency’s review process more quickly than other medications. 

In April, Emalex published results from a mid stage trial with 74 participants that showed those who received its drug saw a 30% reduction in tics compared with those who received a placebo.

The company is currently testing ecopipam in a late stage trial with around 220 patients. Aronin told Insider that he expects data from the trial to be released in early 2025. 

A focus on unmet need has already been successful in narcolepsy

Rod Wong, a managing partner at RTW Investments, told Insider earlier this year that the companies that are most exciting in neuroscience are those that have a product focused on an unmet need. Emalex is a “perfect example of that,” he said.

Wong nominated Emalex for Insider’s July list of top biotech startups that are set to take off in the next 12 months.

Emalex isn’t the only company that Aronin has built to address illnesses with unmet need. Of the handful of companies in Paragon’s portfolio, Aronin said that the majority take a similar “backwards” approach to drug development. 

Another portfolio company, Harmony Biosciences, is focused on patients with narcolepsy, a chronic sleep disorder, and cataplexy, a condition that causes sudden muscle weakness or paralysis. The company has an FDA-approved medication, called Wakix, that has been approved to treat those two conditions, and Wakix is also being studied to treat other rare neurological conditions. 

Aronin said that like Emalex, Harmony was created by identifying an area of need first and then finding the best possible drug candidates.

Read the original article on Business Insider
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