by Pooja Shete on  December 17, 2020 at 12:58 PM Drug News
New Drug Discovered to Treat Intellectual Disabilities
Fragile X Syndrome is a genetic disorder which cause mild to moderate intellectual disability. It is one of the leading causes of autism. A new drug has been discovered which is the first drug for the treatment for Fragile X Syndrome.

The research was conducted as collaboration between University at Buffalo and Tetra Therapeutics.

The drug BPN14770 showed positive results in phase 2 clinical study and improved cognitive function in adult male patients with Fragile X Syndrome.


According to the Centers for Disease Control and Prevention, Fragile X Syndrome is the most common cause of inherited intellectual disability and there is no cure for it.

Mark Gurney, PhD, founder and chief executive officer of Tetra Therapeutics said, "We are very excited about the results of this study. In addition to being safe and well tolerated, treatment with BPN14770 led to significant cognitive improvement, specifically in the language domains, and we also saw a clinically meaningful benefit in overall daily functioning. These findings validate our approach to treating this disease through a mechanism that addresses a core deficit in the disorder."

The research was conducted at Rush University Medical Center. The principal investigator of the study was pediatric neurologist Elizabeth Berry-Kravis, MD, PhD and the funding was provided by a nonprofit organization FRAXA Research Foundation which is dedicated to financing Fragile X Syndrome research.

The preclinical investigation of BPN14770 was conducted through collaboration between the biotechnology company Tetra Therapeutics and UB School of Pharmacy and Pharmaceutical Sciences faculty members James M. O'Donnell, PhD, dean and professor, and Ying Xu, MD, PhD, research associate professor.

BPN14770 drug blocks the activity of the enzyme phosphodiesterase-4D which affects learning and memory formation. It increases the levels of cyclic adenosine monophosphate (cAMP). According to the earlier studies, BPN14770 also has the potential to promote the maturation of connections among neurons, which is usually impaired in Fragile X Syndrome patients.

O'Donnell said, "The collaboration with Tetra Therapeutics has been interesting and productive, combining our lab's expertise in preclinical pharmacology and theirs in drug discovery and development. Seeing years of research lead to a successful trial for treatment of this serious genetic disorder is quite rewarding."

As BPN14770 can improve memory and cognitive function, it can also be used for the treatment of developmental disabilities, traumatic brain injury, Alzheimer's disease, and schizophrenia.

Source: Medindia

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