Imagine being diagnosed with a rare genetic disease and finding out there’s no treatment available. This is the case for hundreds of millions of people worldwide. The truth is 7,000 rare diseases affect more than 700 million people globally, but only 5% have an approved cure or therapy.

The high upfront costs of traditional drug discovery combined with small patient sizes, have resulted in rare diseases being neglected by the pharmaceutical industry. Using a classical drug discovery approach to find rare-disease cures just isn’t practical or feasible for many different reasons, and this is where Modelis comes in.

Founded in July 2018, Modelis is building a drug discovery platform that uses data, artificial intelligence (AI), machine learning (ML), and small animal models to identify new drug candidates and repurpose proven treatments for rare and complex disorders with unmet needs.

“We like to say we do drug discovery differently. Drug discovery requires deep knowledge of a disease’s pathology before being able to start any therapeutic discovery journey. This process can then cost millions of dollars and take up to 10 years before a drug can even begin being tested in humans. In the market for rare-disease drugs, there’s no time or money for that, so we decided to turn this drug discovery model on its head.”

James Doyle

Chief executive officer and co-founder, Modelis

Speeding up the process

Modelis’ approach accelerates the early stages in the drug discovery and development process. First, using data and AI, they rapidly evaluate the relevance of potential targets and pathways to the pathology of a disease, a necessary but traditionally rate-limiting step in the process. They then use simple animal models (such as worms and fish) to confirm their viability as therapeutic targets, and test the efficacy of potential therapies, developing more data and insights as they go.

“We use simple animal models because eighty percent of the genes causing human diseases are evolutionarily conserved in small animals like worms and fish,” Doyle says. “This means we can leverage these similarities to study complex disorders in simple vessels and develop new therapies—or repurpose existing treatments—in fast, cost-friendly ways.”


Therapeutic breakthroughs

Early on, the idea of using small animals to test small molecules seemed far-fetched, but Alex Parke, co-founder of Modelis, and his colleagues decided to try anyway. They tested more than 4,000 approved compounds on sick worms, resulting in the first therapeutic breakthrough for Amyotrophic Lateral Sclerosis (ALS)—a neuromuscular disease that attacks the neurons and spinal cord—in 25 years.

Through this research, his colleagues identified a short list of fifteen of molecules and eventually confirmed the efficacy of Pimozide (an FDA-approved therapy for schizophrenia) to treat ALS in fish and mice. This was the first example of worm-to-human drug discovery and proof of concept of Modelis’ approach. This discovery resulted in a large-scale, nationwide Phase 2b trial which is now underway, examining the effect of Pimozide therapy in patients with ALS after six-month periods.


Bridging the gap between bench and bedside

Modelis is currently advancing rapid-development programs to treat six rare diseases. While the company works with industry partners, their family-centric model also empowers patients and advocacy organizations to drive and champion medical research for specific diseases. They also currently have three projects

backed by venture philanthropists, with a current research focus on neurological disorders. Modelis’ approach, however, has broad applicability across a range of indications and diseases including developmental diseases, neurological disorders, metabolic disorders, cancer, cardiovascular disorders and aging.

“We are driven by a desire to bridge the gaps between bench and bedside in our search for faster cures,” says Doyle. “Our platform will only get smarter over time, leading us to develop deeper understandings of disease pathology to discover new therapies and cures for those that need them most.”

Modelis is part of the first cohort of the adMare BioInnovations acceleration programme and is based at Technoparc Montréal. This programme, supported by the Government of Quebec and the Ville de Montréal, aims to develop the province’s most promising life-science start-ups.

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