ATLANTA (March 2, 2023) The CMT Research Foundation (CMTRF), a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease (CMT)*, is pleased to announce a collaboration with Augustine Therapeutics, a Belgian pharmaceutical company focused on the development of treatments for neuromuscular and neurodegenerative disease, and in particular CMT.

The Augustine therapy is based on small molecule inhibitors of HDAC6, a class II histone deacetylase and well-known molecular target in neurodegenerative disorders and neuropathies. Their third generation HDAC6 inhibitors do not demonstrate the toxicities associated with earlier generations and therefore represent a very promising novel class of therapeutic molecules. Augustine’s lead candidate has demonstrated significant potential in preclinical studies. In mice with CMT1A, the candidate has demonstrated HDAC6 target engagement, reversal of disease phenotypes, and axonal damage biomarkers, resulting in cessation of disease progression during the 4-week study. 

With funding from CMTRF, Augustine will continue this development to gain an in-depth understanding of the compound by conducting pharmacological dose range finding, non-GLP toxicology and safety studies, and long-term efficacy tests in rodents. Conducting these studies will be essential to advance the development of the potential therapy enabling the company to progress towards clinical studies in humans. Apart from their lead candidate, Augustine will also continue assessments on other candidates identified using their drug discovery platform.

“Augustine aims to deliver the first truly curative option for CMT patients. Following promising preclinical studies, we are on the right track to advance our lead candidate toward future clinical tests,” says Prof. Ludo Van Den Bosch, VIB/KU Leuven, founding scientist at Augustine and chairman of its Scientific Advisory Board.

“This development progress will be greatly expedited thanks to the support provided by the CMT Research Foundation along with our historical investors as we move towards IND enabling studies,” adds Sylvain Celanire, CEO of Augustine.

“HDAC6 inhibitors are promising as potential therapeutics for several forms of CMT. With funding from the CMT Research Foundation, Augustine will be able to continue research on their promising lead candidate tackling CMT1A disease,” says Cleary Simpson, CEO of CMTRF “We are hopeful that this collaboration will accelerate the progress of finding a cure for CMT1A patients who currently have no available treatment options.”


CMT Research Foundation (CMTRF) is a patient-led, non-profit focused on delivering treatments and cures for CMT. The foundation identifies significant obstacles or deficiencies impeding progress towards a cure and seeks out collaborators to address these issues. It’s their mission to raise funds to invest in promising science with high potential of leading to treatments and cures. Founded by two patients who are driven to expedite drug delivery to people who live with CMT, the 501(c)(3) federal tax-exempt organization is supported by personal and corporate financial gifts.

Augustine Therapeutics is a spin-off company of VIB/KU Leuven backed by seed investors V-Bio Ventures, PMV, AdBio Partners, VIB and Gemma Frisius Fund. The company focuses on the development of novel, innovative medicines for rare neurodegenerative disorders, tackling severe debilitating diseases with high unmet medical needs such as Charcot-Marie-Tooth disease, a serious and rare disease, as well as other peripheral neuropathies. The company is building a portfolio of first-in-class therapeutic modalities from the ground-breaking research of Prof. Ludo Van Den Bosch from the VIB/KU Leuven, Center for Brain and Disease Research.

*Charcot-Marie-Tooth encompasses a group of inherited, chronic peripheral neuropathies that result in nerve degradation. CMT patients suffer from progressive muscle atrophy of legs and arms causing walking, running and balance problems and abnormal hand and foot functioning. CMT affects one in 2,500 people, including 150,000 Americans and nearly 3 million people worldwide. At the moment, there is no treatment or cure for CMT.