Lenire Biosciences Inc., a biopharmaceutical company developing novel treatments for Fragile X Syndrome (FXS) and related disorders, today announced that it has agreed to a licence from UCL (University College London) for VSN16R, a small molecule positive modulator of large-conductance, calcium-activated potassium (“BK”) channels, as a novel therapy for FXS. The development of this technology was supported by UCL Business (UCLB), the commercialisation company of UCL.
FXS is a rare, inherited condition that can cause issues including developmental delays, learning and behavioural difficulties, physical abnormalities, anxiety, attention or hyperactivity disorders and autism spectrum disorder. In the UK around 1 in 4,000 men and 1 in 6,000 women are affected by FXS.
VSN16R is an orally bioavailable compound based on anandamide, a natural signalling molecule in the endocannabinoid system in humans. It was developed in the laboratory of Professor David Selwood (Wolfson Institute for Biomedical Research, UCL), as part of a study that has completed phase II human trials for muscle stiffness and involuntary muscle spasms in multiple sclerosis, a common symptom known as spasticity. A large and diverse set of preclinical studies have implicated deficient BK channel activity in the etiology of FXS, and recent studies in a mouse model of the syndrome demonstrated efficacy of VSN16R in rescuing FXS-related phenotypes. Lenire plans to develop VSN16R to treat patients with FXS.
“Extensive research into the role of altered BK channel expression, activity and regulation in FXS models provides a strong rationale for pharmacologically augmenting its activity as a disease-modifying treatment for FXS that may improve many of its core symptoms,” shared Dr. Peter Vanderklish, Co-Founder of Lenire.
FXS is the most prevalent inherited form of intellectual disability and a leading cause of autism spectrum disorder. There are currently no disease-modifying therapies for FXS, and some of the most problematic symptoms, such as anxiety, are largely refractory to medications that would be used to treat them in the neurotypical population.
Dr. Craig Erickson, Professor of Psychiatry at Cincinnati Children’s Hospital Medical Center and Director of the Cincinnati Fragile X Research and Treatment Center commented, “We are excited to collaborate with Lenire to test VSN16R in patients with FXS.”
Professor David Selwood commented, “The effect of our drug in the disease models is transformative; we are very excited to see this drug go forward to the clinic in people with Fragile X.”
Barny Cox, Senior Business Manager at UCLB commented, “With convincing preclinical efficacy in FXS, as well as an excellent clinical safety profile, we are optimistic that VSN16R will prove effective in providing meaningful relief to patients with this debilitating disorder.”
About Lenire Biosciences
Lenire Biosciences is privately held, clinical stage biopharmaceutical company that is developing novel, patent protected therapeutics for the treatment of Fragile X Syndrome (FXS), a leading cause of intellectual disability world-wide that also presents with anxiety, attention deficit, hyperactivity, autism spectrum behaviours and other disabling symptoms. More information on the Company can be viewed at https://lenirebiosciences.com.
David H. Crean
Chief Executive Officer