Treating devastating genetic forms of childhood epilepsy
Path Therapeutics' MitoREAD platform measures improvements in mitochondrial function to identify novel drug targets for brain disorders
The Big Idea
By identifying pathways that restore mitochondrial function to a ‘healthy’ state, Path Therapeutics identifies new targets that can launch first-in-class drug discovery programs.
Path Therapeutics' MitoREAD platform is based on the knowledge that restoration of neurometabolic outputs to baseline levels is clinically beneficial across many brain disorders, thereby enabling the discovery of new druggable targets without any bias of underlying disease biology. Using MitoREAD, Path Therapeutics identified phosphodiesterase 4 (PDE4) as a new druggable target for the treatment of epilepsy and devised a novel allosteric modulator strategy to circumvent tolerability limitations observed in previous generations of PDE4 inhibitors. Path Therapeutics is currently conducting hit-to-lead optimization of its lead program and has proof-of-concept data demonstrating that PDE4 allosteric inhibitors decrease seizure frequency in mouse models of Dravet Syndrome, a hard-to-treat refractory epilepsy.
About Path Therapeutics
Path Therapeutics is a private, Canada-based biotech company focused on neuroscience therapeutics that was co-founded by collaborators Dr. Jong Rho, a pediatric epileptologist and a leading expert on the role of mitochondrial function in brain disorders, and Dr. Deborah Kurrasch, a pharmacologist with expertise in animal models of brain diseases. Path Therapeutics closed their pre-seed round in Dec 2019 with Viva Biotech and is currently fundraising their Seed round. Path Therapeutics’ initial focus is epilepsy, with a lead program in Dravet Syndrome.
Accelerated drug discovery
Path Therapeutics has developed a novel neurometabolic, phenotypic drug discovery platform (MitoREAD) to identify new protein targets against which de novo drug discovery can be conducted. To apply its technology, Path Therapeutics is focused on epilepsy whereby a persistent 30-40% of epileptic patients are refractory to current medications despite over eight decades of research and the advent of new drugs. Tellingly, over 90% of antiseizure drugs in the clinic target just a handful of channels and receptors localized to the synapse, underscoring that drugs with alternative mechanisms of action are needed for these pharmacoresistant patients.
Given that derangements in neuronal metabolism contribute to a wide variety of brain disorders, this technology has the potential to alter the future of drug discovery across CNS disorders including epilepsy, autism, and aging.
Initial focus on pediatric epilepsies
Epileptic encephalopathies (EEs)
Diagnosed in early life
Aggressive onset leading up to 1000+ seizures/day and sometimes premature death
Intractable and patients largely fail to respond to current drugs
Monogenic, making modeling in zebrafish straight-forward
Large unmet clinical need with Dravet individuals requiring daily medications for life as the mainstay treatment
A rare disorders and qualifies for Orphan Drug designation with the FDA and Rare Pediatric Disease priority review
Our solution: A PDE4 allosteric modulator as a first-in-class treatment for Dravet
Path Therapeutics conducted a proof-of-concept program for a lead candidate PT-301, which demonstrates efficacy in highly refractory models of Dravet syndrome
Restores MitoREAD bioenergetics in Dravet zebrafish
Bioenergetics in Dravet zebrafish model
Bioenergetics was decreased in Dravet mutant fish (grey squares) and restored to wildtype levels (black dots) by treatment with PT-300 series compounds (yellow symbols).
PT-301 blocks seizures
Achieves seizure freedom in Dravet mice
The gold-standard of anti-seizure medication discovery is the video-EEG assay, in which the number of spontaneous seizures are recorded 24 hrs/day for ten or more days. Treatment with PT-301 decreased the number of seizures per day to almost zero, achieving seizure freedom in this hard-to-treat epilepsy.
Our Founding Team
Aiping Young, MD/PhD
Dr Young is the co-founder and former President/CEO of Lorus Therapeutics. Dr Young raised over $100M in private equity for the company before Lorus merged with Aptose in 2016.
Deborah Kurrasch, PhD
Co-Founder and Chief
Dr Kurrasch is a basic science researcher and associate professor at the University of Calgary who has expertise in animal models of CNS disorders and pharmacology.
Jong Rho, MD
Co-Founder and Chief
Dr Rho is a clinician-scientist and professor of neurology at the University of California, San Diego, and one of the world’s foremost experts on metabolism in the epileptic brain.
For Investors and Partners
Path Therapeutics is turning CNS drug discovery on its head with its phenotypic approach to target identification.
Path Therapeutics is working to advance its biology platform technology through pharmaceutical partnerships and strategic investments from those who share its vision. Path Therapeutics is actively seeking discussions with parties interested in Dravet Syndrome and other epileptic encephalopathies, as well as in autism and aging.
"Big results require big ambitions"