CEO: Dr. Ido Nevo
XoNovo is a preclinical-stage biopharmaceutical company engaged in the development of a first-in-class small-molecule therapeutic for preventing the progressive loss of neuronal function across multiple neurodegenerative diseases. Our short-term focus is studies of lead drug XN001 in support of filing an Investigational New Drug (IND) application for Batten Disease [BD] (also known as Neuronal Ceroid Lipofuscinoses; NCL), a fatal neurodegenerative orphan disorder, by leveraging recent FDA regulations for an accelerated IND approval process for orphan diseases. XoNovo utilizes a revolutionary approach of selective modulation of autophagy and axonal transport machinery in neurons. XN001 has been extensively tested in both in vitro and in vivo models, showing promising results in neurodegenerative diseases including BD, Alzheimer’s, Glioblastoma, Muscular Dystrophy and Tuberos Sclerosis.
XoNovo was established in FutuRx Incubator in October 2014.
The unique scientific approach of XoNovo is based on the elucidation of molecular and biochemical signaling pathways required for neural function and regeneration. This novel mechanism supports autophagy, a natural cellular system of improving protein trafficking and turnover. Autophagy has been shown to be severely compromised in BD as well as in other neurodegenerative diseases.
XN001, a first-in-class drug candidate, is a synthetic small molecule derivative of a natural brain metabolite Lanthionine Ketimine (LK). XN001, a selective inhibitor for CDK5/p25, augments the autophagy mechanism with therapeutic effects on the structure and function of neurons. XN001 is an orally-available brain-penetrating molecule that showed initial efficacy in BD cellular and animal model systems with a solid safety profile and potential to treat BD.
Batten Disease (BD) is an inevitably fatal inherited disorder of the CNS that is caused by recessive mutations in the CLN genes. It is considered an orphan disease, with a prevalence of 1/25-50,000 births in developed countries (www.orphan.net). There are 8 known CLN mutations, with CLN3 being the most frequent cause of Juvenile NCL (JNCL). JNCL patients are characterized by disease onset in early childhood (5-10 years). The classic form of JNCLs typically is manifest with deteriorating vision; blindness occurs within a few years, cognitive abilities decline, and epilepsy starts. Dementia and motor disturbances worsen progressively, and psychiatric problems also have been reported. The disease is inevitably fatal, usually by ~20 years of age.
Current treatment for NCL patients is only supportive, and consists of palliative care with administration of anticonvulsive drugs as well as educational, psychological, and psychiatric management .
The addressable market for NCLs disorders is estimated at $0.5-1.0B per year based on the market size of comparable orphan indications with similar incidence (e.g., Fabry and Gaucher diseases). The NCL disorder market has three known potential competitors: BioMarin Pharmaceuticals, developing BMN190 (cerliponase alfa) for the treatment of children with CLN2 disease for which a Biologics License Application was recently accepted for review by FDA, academic clinical trial at University of Rochester for CellSept efficacy in Juvenile BD, and Abeona Therapeutics developing ABO-201 as an AAV-based gene therapy for CLN3.
The Company’s management team is comprised of individuals having proven track records of founding and managing successful companies, as well as converting discovery research into products:
Dr. Ido Nevo: Acting CEO
PhD in Cellular and Molecular Biology from Tel-Aviv University, Israel; post-doctoral research at the Neuro-Oncology Branch, National Cancer Institute, National Institutes of Health, MD, USA. With over 10 years of experience in cancer research, Dr. Nevo has significant leadership and management experience.
Dr. Ben Shai: Senior Researcher
PhD in Cellular and Molecular Biology from Tel Aviv University; studied immunology, virology and cancer research; worked as a scientific consultant at Medidactic as head of a novel drug application project for the Ministry of Health of Israel on behalf of Boehringer Ingelheim.
Mrs. Rinat Sack: Head of CMC
BSc in Chemical Engineering from Technion; MSc in Organic Chemistry from Weizmann Institute. 14 years experience in drug product formulation development, project management of early development, scale-up manufacturing and submission as well as drug substance and CMC project management in leading pharmaceutical companies in Israel.
Prof. Kenneth Hensley: CTO, Co-Founder and Inventor
Associate Professor of Pathology and Research Director for Pathology, University of Toledo, OH, USA; PhD Chemist with more than two decades of experience in applied neuroscience research and development.
Dr. Rafi Gidron: Chairman, Co-Founder
Founder and Chairman of Israel Brain Technologies. Serial entrepreneur, responsible for founding and managing technology companies in various disciplines, including Chromatis as the largest exit of an Israeli company. Served as Associate Director of the National Science Foundation Center at Columbia University.
 Mole S. E. Williams R. E. Goebel H. H. (Eds.) (2011). The neuronal ceroid lipofuscinosis (Batten disease), Oxford University Press Second edition.