Neuropeutics Inc, a start-up company launched by University of Toronto researchers to tackle neurodegenerative diseases, has partnered with medical research charity LifeArc to develop a new drug for amyotrophic lateral sclerosis (ALS) and other neurodegenerative conditions.
“Partnering with LifeArc marks a pivotal milestone in our mission to create novel effective treatments for ALS,” says Marc Shenouda, CEO and co-founder of Neuropeutics Inc, who is a postdoctoral fellow at U of T’s Tanz Centre for Research in Neurodegenerative Diseases.
“By combining our collective expertise in disease pathology and translational capabilities, we aim to accelerate the path toward a breakthrough therapy that could transform the lives of those courageously living with ALS and other neurodegenerative diseases."
In ALS and some other diseases, a protein called TDP-43 aggregates in neurons of the brain and spinal cord, eventually causing cell death and leading to neurodegeneration. Shenouda, who had planned to become a physician while a life sciences undergraduate at U of T, shifted gears as graduate student and began looking for molecules that could reduce these protein aggregates.
Working with Janice Robertson, a Tanz Centre researcher, professor and James Hunter Family Chair in ALS Research in the department of laboratory medicine and pathobiology at U of T’s Temerty Faculty of Medicine, Shenouda co-discovered a molecule that helped break up TDP-43 aggregates and prevented their formation.
The molecule, which they named JRMS-22, reduced the clumping of TDP-43 in mouse models of ALS and frontotemporal dementia by approximately 30 per cent after only two weeks of treatment.
In more recent research, they discovered the same molecule also reduces aggregations of other disease-relevant proteins, including mutant superoxide dismutase-1 and alpha synuclein — broadening the potential applications of the molecule to other diseases such as Alzheimer’s, Parkinson’s, dementia with Lewy bodies and multiple system atrophy.
After their initial discovery, Shenouda wanted to ensure the molecule did not remain in academic journals but would advance toward patient use. Robertson suggested they commercialize the molecule, and they founded Neuropeutics in spring 2023.
“The idea of a start-up was initially very foreign to me and required a lot of learning, but I knew it was exactly what I had to do,” says Shenouda. “It’s a risky journey to create a company, but it will be extremely rewarding when this potential drug reaches the patients who need it the most.”
Shenouda continues to optimize the molecule in collaboration with LifeArc and the Robertson’s lab, for oral administration and clinical trials. At the same time, he builds the business through pitch competitions and partnerships to secure investments for the company.
“A key mission of the Tanz Centre is to develop therapies for patients suffering from neurodegenerative disease,” says Graham Collingridge, director of the Tanz Centre. “A start-up company such as this one is precisely what is needed. Without commercialization, there will be no new therapies.”
Shenouda and Robertson have participated in innovation and accelerator programs, such as Lab2Market Validate and Launch, and Creative Destruction Lab-Toronto Neuro Stream. They have also received support through the Health Innovation Hub and U of T’s broader innovation ecosystem, and were recipients of the Connaught Innovation Award.
In the last year, Shenouda’s team garnered a top-three national and top-24 global finish at the Entrepreneurship World Cup last summer, placed third in the Falling Walls Lab Toronto competition, and won three awards at Sweet Biopharma Day in Montreal this fall.
Shenouda says pitching has provided him with new skills to bring his discovery to the business world — including ways to speak about his work to attract people without deep scientific knowledge, and how to explain the business potential of the new company.
“As researchers, we want to develop an innovative drug to offer hope to people living with neurodegenerative diseases, and we think that's what everyone is going to be excited about. But we also need to demonstrate how the company can make money,” says Shenouda. “The science is not enough. You need a sharp business plan to attract investor money, and that’s how to push your drug development forward.”