Augustine raises $85m for Charcot-Marie-Tooth treatment

Augustine Therapeutics, a biotechnology firm based in Belgium, has successfully secured €78 million ($85 million) in funding to advance its lead drug candidate as it moves towards a Phase I/II clinical trial for Charcot-Marie-Tooth (CMT) disease. This recent financial boost was co-led by Novo Holdings and Jeito Capital, and saw the involvement of several investors including Asabys Partners, Eli Lilly, AdBio Partners, V-Bio Ventures, PMV, VIB, Gemma Frisius Fund, the US-based CMT Research Foundation, and Newton Biocapital. This funding round follows an earlier €17.5 million investment led by Asabys Partners in July 2024.

The primary focus of this new funding will be to support the clinical progression of Augustine's leading candidate, AGT-100216. The company’s innovative approach centers on inhibiting the enzyme histone deacetylase 6 (HDAC6), recognized for its pivotal role in neurodegeneration and aging-related cellular processes. The scientific foundation of Augustine Therapeutics is built upon the research conducted by Professor Ludo Van Den Bosch from the VIB-KU Leuven Center for Brain and Disease Research. Professor Van Den Bosch's work identified the inhibition of HDAC6 as a promising therapeutic strategy for treating CMT and other neuropathies.

AGT-100216 is set to enter a Phase I/II proof-of-concept clinical trial for CMT disease, with patient enrolment expected to commence later this year. In addition to advancing its lead program, Augustine plans to utilize the investment to develop two discovery-stage HDAC6 inhibitors. These inhibitors target "peripherally restricted and blood-brain barrier-penetrant HDAC6i" for unspecified neurodegenerative and cardiometabolic conditions.

Gerhard Koenig, Augustine Therapeutics’ CEO, highlighted the industry-wide recognition of HDAC6's therapeutic potential, noting that past drug approaches have been less than ideal, especially for chronic diseases. Augustine is aiming to address these limitations by employing a novel non-hydroxamate, non-hydrazide producing chemotype, engineered to improve both efficacy and safety.

Interest in HDAC6 inhibitors is mounting within the biopharmaceutical sector. In November 2023, Novartis expanded its presence in the field with an $80 million upfront investment for a Phase I asset from Handing Chong Kun Dang Pharmaceutical. This agreement, potentially valued at up to $1.3 billion, granted Novartis exclusive rights to develop and market CKD-510, an experimental HDAC6-inhibiting treatment for CMT with orphan drug status from the US Food and Drug Administration (FDA).

Charcot-Marie-Tooth disease is a hereditary neurological disorder caused by genetic mutations that impact the peripheral nerves, resulting in muscle weakness and sensory impairments in the hands, arms, legs, and feet. Certain mutations directly damage the nerves, while others disrupt the protective myelin sheath, leading to impaired nerve signal transmission. Beyond CMT, the HDAC6 inhibitor from Tenaya Therapeutics is undergoing evaluation in a Phase I trial for heart failure with preserved ejection fraction.