Rare Daily Staff

Prodrug developer KemPharm said that it has entered into a technology licensing and assignment agreement with Genco Sciences to use the company’s early research-stage nano-particulate amphetamine technology for a potential prodrug to treat pediatric Tourette’s syndrome when accompanied by attention deficit hyperactivity disorder.

Tourette’s syndrome is an inherited neuropsychiatric disorder with onset in childhood, characterized by the presence of motor and phonic tics. The treatment goal is managing symptoms to achieve optimum functioning, rather than eliminating symptoms. In 90 percent of cases, Tourette’s syndrome exists in conjunction with another disorder. The most frequent co-occurring condition in people with Tourette (63 percent) is ADHD, though the cause of this association is uncertain.

KemPharm has a proprietary platform technology to generate prodrug versions of FDA-approved drugs in the areas of pain, ADHD, and other central nervous system disorders. Prodrugs are medications that are converted to their active ingredient within the body. Using such an approach is a way to potentially avoid abuse of substances, such as amphetamines, or better control how and where the active drug is released within the body.

Genco previously received Orphan Drug designation from the U.S. Food and Drug Administration for its product candidate for pediatric Tourette’s syndrome.

“Today’s announcement of the license and assignment agreement with Genco provides KemPharm’s discovery and development team the opportunity to apply our prodrug expertise to a potential orphan drug area of the ADHD market,” said Travis Mickle, president and CEO of KemPharm. “In a subset of pediatric Tourette’s syndrome patients, there is a recognized problem of ADHD that requires additional treatment concurrent to treating the symptoms of Tourette’s.”

Under terms of the agreement, KemPharm will be responsible for financing and managing all product development. In exchange, KemPharm will retain full intellectual property and commercial ownership of any product developed as a result of this agreement. Genco will be eligible to receive certain milestone and royalty-based or value share payments. Additional details about the agreement were not disclosed.

“KemPharm’s success at creating unique prodrug product candidates in multiple treatment areas allows us to see real potential for a prodrug to be successful as the basis for treating this rare pediatric disease,” said Subhash Desai, CEO of Genco. “By treating the underlying ADHD, we believe we can unlock the door to also treating tics and/or Tourette’s syndrome to provide an effective therapy for the unmet needs of those affected by this complex neurologic disorder.”

October 4, 2017