Belgium-based biotech Augustine Therapeutics has raised €78m ($85m) in funding to support the development of its lead candidate as it enters a Phase I/II trial in Charcot-Marie-Tooth (CMT) disease.
Novo Holdings and Jeito Capital co-led the $85m round, with participation from Asabys Partners, Eli Lilly, AdBio partners, V-Bio Ventures, PMV, VIB, Gemma Frisius Fund, the US-based CMT Research Foundation and Newton Biocapital. This follows an initial €17.5m round led by Asabys Partners in July 2024.
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The new funding will primarily support the clinical advancement of Augustine’s lead candidate, AGT-100216.
Augustine’s approach focuses on inhibiting the cytosolic histone deacetylase 6 (HDAC6) enzyme, which plays a key role in neurodegeneration and aging-related cellular processes. The company’s scientific foundation is based on the research of Professor Ludo Van Den Bosch from the VIB-KU Leuven Center for Brain and Disease Research, who identified HDAC6 inhibition as a promising therapeutic strategy for CMT and other neuropathies.
AGT-100216 will now enter a Phase I/II proof-of-concept clinical trial for CMT disease, with patient enrolment set to begin later this year. Beyond its lead programme, Augustine will also use the capital to further develop two discovery-stage HDAC6 inhibitors targeting “peripherally restricted and blood-brain barrier-penetrant HDAC6i” for undisclosed neurodegenerative and cardiometabolic conditions.
In the announcement accompanying the funding, Augustine Therapeutics’ CEO Gerhard Koenig said: “The therapeutic potential of HDAC6 is widely recognised in our industry, but previous drug approaches have been sub-optimal, particularly for chronic diseases.”
Augustine aims to overcome these limitations by using a novel non-hydroxamate, non-hydrazide producing chemotype, designed to enhance efficacy and safety, explained Koenig.
Interest in HDAC6 has been growing in the biopharma industry. In November 2023, Novartis expanded into the space with an $80m upfront acquisition of a Phase I asset from Handing Chong Kun Dang Pharmaceutical. Potentially worth up to $1.3bn, the deal granted Novartis exclusive rights to develop and commercialise CKD-510, an investigational HDAC6-inhibiting CMT treatment with orphan drug designation from the US Food and Drug Administration (FDA).
CMT is a hereditary neurological disorder caused by genetic mutations that affect the peripheral nerves, leading to muscle weakness and sensory impairment in the hands, arms, legs, and feet. Some mutations damage the nerves directly while others disrupt the protective myelin sheath, both resulting in impaired nerve signal transmission. Beyond CMT, Tenaya Therapeutics’ HDAC6 inhibitor is being evaluated in a Phase I trial in heart failure with preserved ejection fraction.
