23/03/2021

Augustine Therapeutics, a VIB spin off that focuses on the development of novel innovative therapies for rare peripheral neuropathies and neurogenerative disorders, has received EUR 1.2 million in funding from the Flanders Agency for Innovation & Entrepreneurship (VLAIO). The grant will be used to support the development of new medicines for peripheral neuropathies through innovative research by elucidating the mode of action of second-generation selective HDAC6 inhibitors developed by Augustine Therapeutics.

The project awarded by this grant will be further characterizing the biology of Charcot-Marie-Toothdisease (CMT), a severe debilitating disease affecting nearly 2.6 million people worldwide (Source: National Institute of Neurological Disorders and Stroke, NIH). Moreover, Augustine Therapeutics aims at identifying the potential neuroprotective actions of an innovative therapeutic strategy: HDAC6 inhibitors. Gaining knowledge on the mode of action of these compounds will be the foundation for the development of new cures for peripheral neuropathies disorders including CMT.

Sylvain Celanire, CEO, comments: “This funding is not only an excellent boost for our innovative research towards first-in-class medicines for CMT-patients; it is also an external validation of the quality and importance of our pioneering work. We are grateful for the financial support from VLAIO and pleased to support its mission of advancing world-class innovation in the Flanders region.”

Charcot-Marie-Tooth Disease, a severe debilitating neurodegenerative disease
CMT is the most common form of inherited peripheral neuropathy that affects 1 in 2500 people (Source: European Charcot-Marie-Tooth Federation (ECMTF)) and is one of the most prevalent of the so-called rare diseases (defined by the EC Regulation 141/2000). CMT and all its variants represent an important unmet medical need as there are no approved therapies available for these patients. CMT is clinically characterized by the progressive loss of force and sensation due to gradual denervation. Patients with CMT experience difficulty walking and frequently fall because of weakness of the lower legs and feet.

As the disease progresses, patients show hand and foot deformities. CMT disease presents differently in each person, even between affected members of the same family. In several cases, middle-aged patients require the use of a wheelchair and need assistance in everyday life. As such, CMT imposes a devastating impact on the patients’ quality of life.

A program holding great promise for potential breakthrough in the treatment of CMT and other neuropathies
The HDAC6 protein plays a central role in various processes that are key for neuronal survival. HDAC6 was identified by Prof. Ludo Van Den Bosch and his research group one decade ago as a relevant therapeutic target for the development of new drugs for the treatment of CMT (Source: Nature Medicine, 2011). 

Ludo Van Den Bosch, founding scientist and member of the 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 two-and-a-half-year R&D project will enable further understanding how to use HDAC6 inhibitors in various disease model systems. This information is needed for the development of novel and safer drugs to treat CMT. It is 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, aiming to gain in-depth knowledge about the biology of CMT and characterizing the potential protective action of HDAC6 inhibitors at the neuronal and axonal level.

Jérôme Van Biervliet, Managing Director of VIB, comments: “We are very happy with the financial support and recognition from VLAIO for the ongoing strategic collaboration between VIB and our spinoff Augustine Therapeutics. The VIB Discovery Sciences team and the VIB lab of Ludo Van Den Bosch have been continuously supporting Augustine in advancing this innovative project. We look forward to the new treatment options our spin-off will develop to benefit patients suffering from neuromuscular diseases.”

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