This up-and-coming star on the global biotech scene aims to crack the hard code of finding better treatments for neurodegenerative diseases.

The name of the company, Muna, means “to remember” in Old Norse and reflects the focus of discovering and developing medicines for neurodegenerative diseases, including Alzheimer’s, Parkinson’s, Multiple Sclerosis and Frontotemporal Dementia. These are diseases that impact memory, movement, language, behavior and personality, resulting in disability and death of millions of patients around the world.

“I’m proud of working with a great team of leaders and scientists at Muna, collaborating to do great science, discover impactful new medicines, and build a global company that thrives.”

Rita Balice-Gordon, PhD, CEO of Muna Therapeutics, has previously led academic teams while being a professor of neuroscience at the University of Pennsylvania School of Medicine, as well as global R&D-teams focused on neurology and rare disease at Pfizer and Sanofi. These experiences taught her how to ask and answer scientific questions that could impact patient lives in meaningful ways, she says. “I’m proud of working with a great team of leaders and scientists at Muna, collaborating to do great science, discover impactful new medicines, and build a global company that thrives.”

 

The name of the company, Muna, means “to remember” in Old Norse and reflects the focus of discovering and developing medicines for neurodegenerative diseases.

 

Integrated key pieces into a cohesive whole

Created in early 2020, Muna is an early stage biotech company, incorporated in Denmark and based in Copenhagen, Leuven, Belgium, and in the U.S. The startup’s projects received early funding from Novo Holdings, through its NovoSeeds group in Copenhagen.

“Anders Hinsby, one of our co-founders, Jakob Busch Petersen, our Chief Medical Chemistry Development Officer, and I worked together with our scientific founders and investors to expand our scientific and drug discovery efforts rapidly by in-licensing two programs from Axxam, a strategic partner research organization based in Milan with whom we continue to work closely,” describes Rita.

The company also merged with K5 Therapeutics, a new startup company founded by Dr. Bart De Stropper from VIB and KU Leuven, Belgium, based on his and his lab team’s work.

“The funding we raised will allow us to bring our two drug discovery programs to early clinical studies, continue to grow our R&D group, and build and maintain our two innovative platforms.”

“We integrated these key pieces into a cohesive whole, presented our corporate and scientific strategy to investors, and raised a first round of funding of 60 million EUR in mid-2021 from several investors in Europe and the US, including Sofinnova Partners, Novo Holdings, Droia Ventures, LSP Dementia Fund, Polaris Partners, Polaris Innovation Fund, Sanofi Ventures, V-Bio Ventures and VIB,” says Rita. Niels Plath joined Muna shortly thereafter as its Chief Scientific Officer, and the company rapidly established an R&D team in Copenhagen.

“The funding we raised will allow us to bring our two drug discovery programs to early clinical studies, continue to grow our R&D group, and build and maintain our two innovative platforms,” says Rita.

She projects that they will file their first investigational new drug application (IND) in the next 15-18 months. “We plan to begin one or two new small molecule drug discovery programs by the end of this year, based on the new insights generated from our platforms, and we plan to add an average of one new program per year thereafter.”

Denmark offers a vibrant and dynamic ecosystem for scientific research, biotech and biopharma, believes Rita.

“An entrepreneurial spirit is alive and well in Copenhagen, Aarhus and in other regions of the Nordics.”

“There are excellent scientists across various disciplines, leaders and entrepreneurs, highly qualified to fill many kinds of positions in life sciences companies,” she says. “An entrepreneurial spirit is alive and well in Copenhagen, Aarhus and in other regions of the Nordics, creating opportunities for investment from Danish and other sources, new jobs, advancing knowledge and improving human health and quality of life.”

Aligning various business practices and financial matters across different countries, while respecting the laws of each in which they do business, can be challenging from time to time, she adds.

 

Muna Therapeutics’ management team

 

The four R’s and two platforms

The company’s scientific focus is divided into four areas, which they call the 4 R’s; repairing the function of brain cells, resolving chronic inflammation in the brain that exacerbates neurodegeneration, restoring neuroprotection to slow or stop disease progression and preserve key brain functions like cognition, and resilience to disease, by understanding how brain cells can stay functional despite disease pathology.

“Dr. Glerup and his lab have used cutting-edge structural biology and cell and animal models to understand how brain cells internalize and traffic key proteins, and how these processes are disrupted in disease.”

“We are fortunate to be working with two world-class neuroscientists, Dr. Bart De Strooper of VIB and KU Leuven, and Dr. Simon Glerup of Aarhus University. Dr. De Strooper and his lab have done groundbreaking work that has advanced our understanding of the pathophysiology of Alzheimer’s Disease. His and his colleagues’ work has suggested several potential new ways to treat this disorder. Dr. Glerup and his lab have used cutting-edge structural biology and cell and animal models to understand how brain cells internalize and traffic key proteins, and how these processes are disrupted in disease,” says Rita.

Dr. De Strooper and Dr. Glerup’s work forms the foundation for two platforms that Muna uses to discover and validate new targets for disease-modifying medicines. The first, based on work from the De Strooper lab, is an all-in-human target discovery and validation platform, involving spatial transcriptomic and bioinformatic analyses of the genes expressed by different brain cell types in postmortem samples from patients compared to healthy individuals. Genes and pathways that are differentially expressed are further evaluated in human cells and innovative humanized mouse models for their potential as points of intervention for new potential medicines.

“This platform is the focus of Muna’s work in Leuven and our colleagues there not only use these approaches to find and evaluate new drug targets, they provide important work in collaboration with colleagues based in Copenhagen that accelerates Muna’s drug discovery and development work on two known targets that we are advancing to the clinic as rapidly as we can,” says Rita.

“These capabilities allow us to very quickly identify potential new compounds that could be developed into medicines.”

The second platform, based on work from the Glerup lab, is a structural biology platform, involving protein chemistry, crystallography and computational chemistry capabilities, that allows the company to understand and analyze disease relevant protein targets, and determine where and how new compounds they discover might bind to these targets and influence their function. “These capabilities allow us to very quickly identify potential new compounds that could be developed into medicines,” explains Rita.

 

Bart De Strooper and Simon Glerup, Scientific founders, Muna Therapeutics

 

A unique approach

In the longer term, Rita Balice-Gordon and the rest of the team are growing Muna to be an end-to-end neurocentric life sciences company, “Which means that we are able to discover new drugs, test them in patients in clinical trials, and commercialize them so that patients and physicians have our medicines as impactful treatment options,” she says.

The company are in the early stages of this effort and there is much work ahead, but Rita says that its capabilities in medicinal chemistry and human biology make it unique among other neurodegeneration-focused biotechs.

“Our approach to both of these programs is unique – the way we affect the drug targets with our molecules, how they get into the brain, their safety and other properties, and the effect they have on brain cells resulting in slowing of disease progression.”

“We have used these capabilities to discover new small molecules that can modulate the function of two drug targets, one for Alzheimer’s and other neurodegenerative diseases, and the other for Multiple Sclerosis and Parkinson’s. Our approach to both of these programs is unique – the way we affect the drug targets with our molecules, how they get into the brain, their safety and other properties, and the effect they have on brain cells resulting in slowing of disease progression,” she emphasizes.

“While like many early stage biotech companies, we don’t currently have medicines on the market, there is high unmet need for medicines for neurodegenerative diseases, and the global markets for new medicines greatly exceed supply,” she adds.

Featured photo of Rita Balice Gordon, CEO, Muna Therapeutics