The $698K SBIR Award from the NIH is awarded to Virtus Therapeutics, a Yale Spinout founded by Dr. Choukri Ben Mamoun, for its groundbreaking PKAN therapy development.

Virtus Therapeutics, a biotech spinout originating from Yale University research led by Dr. Choukri Ben Mamoun, has announced it was awarded a $698K Small Business Innovation Research (SBIR) Award from the National Institute of Health (NIH) for its focus on advancing a novel treatment for pantothenate kinase-associated neurodegeneration (PKAN), a rare and often fatal genetic disorder. Virtus Therapeutics, leveraging Dr. Ben Mamoun's groundbreaking research, was initially funded by the Yale faculty accelerator fund, The Blavatnik Fund for Innovation at Yale.

Virtus Therapeutics is dedicated to advancing a novel treatment for pantothenate kinase-associated neurodegeneration (PKAN), a rare and often fatal genetic disorder characterized by the body's inability to properly utilize vitamin B5, also known as pantothenic acid. Individuals with PKAN typically experience progressive neurological deterioration, leading to a range of debilitating symptoms such as muscle stiffness (dystonia), difficulty with coordination, speech impairment, and cognitive decline. PKAN primarily affects children and young adults, and its symptoms tend to worsen over time, often leading to a loss of ambulatory capacity. PKAN is caused by mutations in the pantothenate kinase gene (PANK2), which encodes the first enzyme involved in the production of coenzyme A (CoA) from vitamin B5. CoA is essential for various cellular processes, including energy production and the synthesis of essential molecules.

Virtus Therapeutics is pioneering a transformative therapy based on an innovative class of small molecule activators. These activators stimulate the utilization of vitamin B5 by activating a distinct enzyme, compensating for the altered or lost activity of PanK2 in PKAN patients. 

This NIH award represents more than just a scientific investment; it's a lifeline of hope for those affected by PKAN and other neurodegenerative diseases. With this support, Virtus Therapeutics’ quest to develop new drugs gains significant momentum, propelling us forward on our mission to transform the course of neurodegenerative disease treatment and to provide relief to countless individuals and families.” said Dr. Choukri Ben Mamoun.

The NIH funding will facilitate research and development efforts aimed at evaluating the efficacy of lead compounds through cell-based assays and animal models, with the ultimate goal of identifying clinical candidates capable of altering the disease's progression and potentially reversing its clinical outcomes.

This groundbreaking research journey traces its origins to an initial support from the Program for Therapeutics in Connecticut Health (PITCH). Additional support for research on PKAN in the Ben Mamoun Lab is provided by The NBIA Disorders Association. Dr. Ben Mamoun is also the recipient of the 2020 and 2021 Blavatnik Awards and the founder of Yale spinout, Curatix.

About Virtus Therapeutics

Virtus Therapeutics is dedicated to advancing a novel treatment for pantothenate kinase-associated neurodegeneration (PKAN), a rare and often fatal genetic disorder. The company is pioneering a transformative therapy based on an innovative class of small molecule activators. Founded by Dr. Choukri Ben Mamoun, Virtus Therapeutics research and development is led by Dr. Anasuya Pal, a former Associate Research Scientist in the Ben Mamoun Lab at Yale.  Virtus Therapeutics is headquartered at BioCT Innovation Commons (The Commons) in Groton, CT. For more information on Virtus Therapeutics, contact: virtusadm@virtustherapeutics.com