Treating devastating genetic forms of childhood epilepsy
Path Therapeutics measures improvements in mitochondrial health to identify drugs with novel mechanisms of action in the CNS
The Big Idea
By identifying drugs that restore mitochondrial function to a non-diseased state, Path Therapeutics is able to identify drugs with unexpected mechanisms of action.
Path Therapeutics' drug screening platform is based on the knowledge that restoration of neurometabolic outputs to baseline levels can improve the symptoms of many brain disorders, including seizures. Path is currently optimizing its lead compound, PT-320, which restores cellular bioenergetics and decreases seizure frequency in in vivo zebrafish and mouse models of Dravet Syndrome.
About Path Therapeutics
Path Therapeutics is a private, Canadian-based biology platform company in the central nervous system (CNS) space spun out from the University of Calgary and formed by collaborators Dr. Jong Rho, a pediatric epileptologist and a foremost expert on the role of metabolism in the epileptic brain and Dr. Deborah Kurrasch, a developmental neuroscientist and an expert in pharmacology and animal models of CNS disorders. In 2017, Path Therapeutics launched its commercialization efforts, with Dr. Aiping Young, a serial biotech entrepreneur and former President/CEO of Lorus Therapeutics, joining the Team in 2018. Path Therapeutics has raised more than $4.5M to date, largely in non-dilutive grants and is actively pursuing a $5M Series A round. Path's initial focus is pediatric epilepsy, with a lead program in Dravet Syndrome.
Accelerated drug discovery
Path Therapeutics has developed a novel neurometabolic, phenotypic drug discovery platform 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 stubborn 30-40% of epileptic patients are refractory to current medications (with pediatric patients especially resistant) 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 to reach 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, aging and beyond.
Initial focus on pediatric epilepsies
Epileptic encephalopathies (EEs)
Diagnosed in neonatal, infancy, or early childhood
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
Considerable expertise in EEs, including access to patient populations for clinical trials
Large unmet clinical need for patients for whom daily medications for life are the mainstay treatment
EEs are rare disorders and qualify for Orphan Drug designation with the FDA and Rare Pediatric Disease priority review
Path Therapeutics' strategy is to screen repurposed libraries for which the molecular target is well-characterized to enable reverse identification of druggable proteins. The target is then validated using a variety of genetic and pharmacological techniques, followed by traditional target-based drug discovery. Using this approach, Path Therapeutics has developed the PT-300 series as its lead program, with others in the pipeline.
Blinded test of the platform
Approved antiseizure drugs restore bioenergetics and failed drugs do not
We blinded 21 commercially available antiseizure drugs, plus 3 drugs that failed Phase III clinical trials for missed endpoints in epilepsy. The platform uncovered 20 of the 21 available antiseizure drugs (yellow squares) and none of the failed drugs (grey triangles).
PT-300 series drugs restore mitochondrial function in vivo in epileptic 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-320 blocks seizures
Neuroprotection in Dravet mice
PT-320 can block the onset of epileptic seizures in vivo in Dravet Syndrome mouse models.
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"
Get in Touch
3330 Hospital Drive NW
Calgary, AB T2N 1N4