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The company is developing a pill that causes stem cells to regenerate muscle to battle debilitating Duchenne muscular dystrophy

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By Richard Dal Monte

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Raising children is full of surprises like the time your toddler takes their first step, or the day your preschooler starts speaking in rhyme. As pleasant as these moments might be, there are tragic and frightening surprises that parents never see coming.

Duchenne muscular dystrophy (DMD), is one such surprise.

A rare genetic disease afflicting boys its found in about six per 100,000 individuals in North America and Europe, and symptoms usually present between two and three years of age DMD causes progressive muscle degeneration and weakness due to a non-functional protein called dystrophin, which helps keep muscle cells intact.

While existing treatments have helped extend life expectancy for people with DMD from their teens into their 20s and 30s, they are only temporary and dont address a critical issue: the inability of the muscles to regenerate.

Satellos Bioscience Inc. (TSXV: MSCL), a Canadian biotechnology company, is advancing game-changing science in skeletal muscle regeneration to improve the quality and duration of patients lives.

Our notion is that if we can restore muscle repair, well significantly reduce the severity and the progression of the disease

Michael Rudnicki, OC, PhD, co-founder and chief scientific officer, Satellos Bioscience Inc.

The key to slowing the progression of DMD is muscle regeneration

Founded in 2018 by biotech entrepreneurs Frank Gleeson and Michael Rudnicki, Satellos work is based on the research of Rudnicki, a senior scientist and the director of the Regenerative Medicine Program and the Sprott Centre for Stem Cell Research at the Ottawa Hospital Research Institute as well as a professor in the Department of Medicine at the University of Ottawa and CEO and scientific director of the Canadian Stem Cell Network.

The companys vision is to invent and develop medicines that reset the bodys innate ability for self-repair, aiming to create a treatment to solve muscle disorders, starting with Duchenne muscular dystrophy.

Our notion is that if we can restore muscle repair, well significantly reduce the severity and the progression of the disease, Rudnicki says.

He explains that Satellos discovered that the loss of function in those who have DMD is the result of problems with how muscle stem cells divide, wherein a process known as cell polarity is defective.

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Satellos main program, then, is focused on developing a drug that would correct muscle stem cell polarity and restore the bodys innate muscle repair and regeneration process by sending a signal to the muscle stem cell.

Gleeson says, In our bodies, we have biochemical pathways of different kinds and these are essentially like communication towers or satellites that enable mobile phone users to talk to one another. Similarly, signals are relayed through proteins interacting with each other, sometimes through biochemical stimulation, sometimes through physical interaction and sometimes through a combination of the two. Weve identified communication pathways that signal to muscle stem cells to act in certain ways, and were identified errors in these signals that inhibit proper muscle stem cell function.

Were intervening in those biochemical pathways to correct the signalling and restore stem cell function. It is space-age stuff and at the same time, its grounded in elegant science and a deep understanding of the mechanisms in play.

End of 2023 could see human safety trials

Satellos identified these pathways using MyoReGenXTM, a proprietary discovery platform grounded in decades of pioneering muscle stem cell knowledge established by Rudnickis research lab. That platform allows Satellos to conduct research by removing muscle fibre from the body, preserved intact, and studying it for biochemical cues.

So far, researchers have done preclinical work on proof of concept by studying genetically engineered mice and have found no serious side effects from drug candidates. While they continue on that path, Satellos aims to proceed with human safety trials by the end of 2023 and, once its through the safety phase, Gleeson says its possible the company could approach the U.S. Food and Drug Administration for accelerated approval in 2025.

While this is early science, we dont believe were that far away from being able to offer something helpful to patients, he says. To the patients, of course, it seems another lifetime of waiting and were very sensitive to that. But, from where we are in the context of typical drug development of 10 years and a billion dollars, were not that far away. And were working every day to find safe ways to go there sooner.

We strongly believe that we offer to the community of DMD patients and their families the chance for a disease-modifying treatment, something that could meaningfully alter an individuals life.

Broad palette of potential treatments

Frank and I had a meeting of minds very early about the aim to develop small molecules that mobilize stem cells to stimulate regeneration in tissue as a way forward to treat diseases, as a way forward for regenerative medicine, Rudnicki says. Weve incubated that idea through many, many conversations over the years.

Thus, DMD isnt the only target for Satellos leading-edge science. Considering the possibilities for its treatments to address muscle regeneration opens up a spectrum of conditions that could be addressed.

Not only do we see this as a relevant to multiple wasting disorders, he says, we think that other stem cells in other parts of the body can be targeted in the retina, in the brain, in the gut, the blood systems.

Gleeson notes that there are about 30 different types of dystrophies alone, in addition to sports injuries, surgical recovery and aging concerns.

The palette is very, very broad, he says.

A smart investment while doing good

Because that palette is broad, so is the potential good Satellos research can do, and thats a key part of the companys attraction to investors.

As well, in addition to executing a reverse takeover with iCo Therapeutics Inc. as one step in a strategy to raise capital and build value for shareholders, Satellos was able to do a concurrent financing of $7.25 million, which allowed the company to ramp up its team and amplify its drug discovery and development efforts.

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David Bautz, a senior biotechnology analyst with Zacks Small Cap Research, says Satellos is way undervalued compared to other companies doing DMD research.

The gene therapy approach [of some other companies] is not working, so Satellos is trying something thats completely different, says Bautz, who has a PhD in biochemistry. The theory behind it is very exciting, in my opinion. Theyre seeing exciting things in preclinical models, which is a good start.

From that perspective, while still risky, I think a lot of that risk is taken away by the fact Satellos valuation currently is so low, he says. And clearly the market values companies that are doing DMD treatment.

Indeed, Gleeson says the companys strong leadership team has established relationships with advocacy groups such as Parent Project Muscular Dystrophy, which made a US$1 million investment in its research.

Adds Rudnicki, These patients really have a significant demand for help. If they can use their hands or limbs to be more self-sufficient, even for a couple more years, thats a huge improvement in quality of life And we are very hopeful we can do more.

This is about changing the lives of these children who are unable to walk and are going to die at a young age.

And the potential for Satellos and its MyoReGenX platform doesnt stop with DMD because there are dozens of other degenerative conditions in muscle tissues as well as other parts of the body that could prove good targets for Satellos technology, among them complications associated with aging.

Says Rudnicki: Not only do we see this as relevant to multiple wasting disorders, we think that other stem cells in other parts of the body can be targeted in the retina, for example, possibly even in the brain.

For more information on Satellos Bioscience, visit its website.

Make sure to follow Satellos on social media for the latest updates:

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There can be no guarantee that the Company will obtain the required regulatory authorization/s to commence clinical trials in humans on a timely basis, or at all. The Company may not be able to translate its novel discoveries into viable therapeutic treatments suitable for clinical development.

This story was provided by Market One Media Group for commercial purposes.

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Satellos Bioscience pursues a revolutionary approach to treat muscle degeneration, give new hope to patients and parents - Financial Post