Augustine Therapeutics developing new medicines for neuromuscular diseases

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Artistic representation of nerves

Augustine Therapeutics had a lot of announcements this month: the VIB spin-off just appointed Dr. Sylvain Celanire as CEO, strengthened its Board of Directors, set up a Scientific Advisory Board and received a €1.2 million VLAIO grant. All of the above is going to help the company to develop therapies for rare peripheral neuropathies and neurodegenerative disorders, starting with Charcot-Marie-Tooth disease. Backed by some major players in the Belgian life sciences ecosystem, this start-up is accelerating fast!

By Amy LeBlanc

Augustine Therapeutics was founded in 2019 as a spin-off of VIB and KU Leuven. Based on the groundbreaking neurological research of Prof. Ludo Van Den Bosch, Prof. Joris De Wit and Prof. Bart De Strooper, the drug discovery company is building a portfolio of first-in-class therapies for rare neurodegenerative disorders. Augustine Therapeutics’ approach is based on new insights into the neuroprotective potential of small-molecule HDAC6 inhibitors for disorders like Charcot-Marie-Tooth disease.

A debilitating disorder

Charcot-Marie-Tooth disease (CMT) is a genetic condition that slowly results in a damaged peripheral nervous system. Although rare, it is nevertheless the most common inherited neuropathy in Europe, affecting about 1 in 2500 people. Patients with CMT experience a progressive loss of sensation and weakness in the hands and feet, which can lead to difficulty in walking and frequent falls. As the disease progresses, patients develop hand and foot deformities; many end up requiring mobility aids such as wheelchairs.

Inhibitors of HDAC6 hold great promise for the treatment of CMT and other neuropathies. – Ludo Van Den Bosch, VIB – KU Leuven

Although CMT is rarely fatal, it has an enormous impact on the lives of patients. Currently, treatment options are limited to symptomatic care like physiotherapy and painkillers, meaning there is a strong unmet need for effective therapies able to halt or reverse the progression of this disorder.

First potential therapy

This is where Augustine Therapeutics’ research comes in. The company’s €1.2 million VLAIO grant will be used by for further research into HDAC6, a protein which plays a central role in processes that are key for neuronal survival. HDAC6 was identified by Prof. Ludo Van Den Bosch and his research group a decade ago as a potential therapeutic target for CMT drug development. Prof. Van Den Bosch, scientific founder of Augustine Therapeutics and now member of the company’s Scientific Advisory Board, explains:

“Inhibitors of HDAC6 hold great promise for the treatment of CMT and other neuropathies. We already made great progress in understanding its mode of action. The new ambitions described in the project funded by VLAIO are the next steps for us to ultimately provide safe and effective solutions for patients.”

This funding is not only an excellent boost for our innovative research towards first-in-class medicines for patients; it is also an external validation of the quality and importance of our pioneering work in CMT and beyond. – Sylvain Celanire, Augustine Therapeutics

The two-and-a-half-year R&D project is aiming to expand our general knowledge of the biology of CMT and characterize the potential protective action of HDAC6 inhibitors at the neuronal and axonal level. Understanding how best to use HDAC6 inhibitors may lead to therapies in not only CMT, but also in other neuropathies like ALS.

Sylvain Celanire, Augustine Therapeutics’ newly appointed CEO, comments: “This funding is not only an excellent boost for our innovative research towards first-in-class medicines for patients; it is also an external validation of the quality and importance of our pioneering work in CMT and beyond.”

It takes a village

Many years of research went into this area before Augustine Therapeutics was actually founded. The studies currently underway are a collaboration between Augustine Therapeutics, VIB Discovery Sciences and the team of Ludo Van Den Bosch at the VIB/KU Leuven Center for Brain & Disease Research. Prof. Van Den Bosch isn’t the only scientist involved either: Prof. Joris de Wit has also joined the company’s Scientific Advisory Board. Work from his and Prof. Bart De Strooper’s labs formed the basis of another project being pursued by Augustine Therapeutics. Asked about his involvement with Augustine Therapeutics, Prof. de Wit shares:

“I am excited to join the Scientific Advisory Board of Augustine Therapeutics. The projects that are in the pipeline are highly innovative and can truly bring solutions to patients.”

Read this BioVox article to find out how Augustine Therapeutics bridged the valley of death back in 2019.

With Dr. Erik Tambuyzer, permanent representative of PMV, appointed as a member of the Board of Directors, and Dr. Sylvain Celanire taking on the CEO position, it’s safe to say the company is well supported by a group of experienced individuals. The VIB spin-off is also being backed by funds with serious clout in the local ecosystem, including V-Bio Ventures, PMV, Advent France Biotechnology and the Gemma Frisius Fund, which all contributed to its €4.2 million seed funding in 2019. The company has recently set up its drug discovery laboratories at the Leuven Bio-Incubator, as Dr. Sylvain Celanire explains:

“I am very much looking forward to guiding Augustine Therapeutics towards approved therapies for patients in need. The new facilities at the Leuven Bio-Incubator are suited to accomplishing this mission, ideally situated adjacent to the VIB-KU Leuven Centre for Brain & Disease Research and the Leuven University Hospitals at Gasthuisberg. With the many talented people involved, and such great support from the local ecosystem, I’m looking forward to our progress over the next few years.”