Leuven, Belgium, 27 February 2024 – Augustine Therapeutics, a biotech company based in Leuven focused on developing innovative medicines for rare neurodegenerative disorders, received a 2023 Lawrence and Isabel Barnett Drug Development Program grant from the US ALS Association. The USD 300K grant will be used to support the preclinical development of a new therapeutic class of orally active and brain-penetrant small molecule HDAC6 inhibitor candidates for the potential treatment of amyotrophic lateral sclerosis (ALS), in collaboration with the VIB-KU Leuven Center for Brain & Disease Research.

A devastating disease

Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's Disease, is a devastating neurodegenerative disorder that affects the motor nerve cells in the central and peripheral nervous system. It is a progressive disease that gradually weakens the muscles, leading to complete paralysis and respiratory failure. With an average life expectancy of two to five years after diagnosis, ALS is incurable, and there are few treatment options available to address the functional deficits and disease progression. Currently, around 330,000 people worldwide are living with this disease. In this context, it's crucial to continue researching new therapies for ALS and accelerate the fight against this debilitating condition.

Advancing novel drug candidates

The aim of Augustine Tx is to test a new therapeutic strategy that prevents the loss of contact between nerve and muscle by inhibiting a specific enzyme, called HDAC6. This enzyme affects the system that transports proteins from one part of the nerve cell to another, which is essential for maintaining the health of the nerves of the motor system.

Thanks to its unique drug discovery platform, Augustine Tx has identified an entirely new class of non-hydroxamic acid and non-fluoromethyl oxadiazole small molecules HDAC6 inhibitors with peripheral-restricted or brain-penetrant properties. A development candidate is currently advancing into regulatory studies and first-in-human clinical trials, thanks to the support of the US CMT Research Foundation. Now, they have the backing of another patient advocacy group, the ALS Association, to bring a novel candidate into advanced preclinical studies.

"This grant will help in advancing multiple promising lead candidates through an extended set of biology, pharmacology, safety, and toxicity studies," says Dr. Frederik Rombouts, Head of Medicinal Chemistry at Augustine Tx, and Principal Investigator of the funded project. "With the help of the US ALS Association, we aim to help select the best candidate of this new class of HDAC6 inhibitors for advanced preclinical proof-of-concept studies."

Indeed, a second aim will be to evaluate this candidate through in vitro and in vivo models for ALS. To achieve this, Augustine Tx will collaborate with the Laboratory of Neurobiology at the VIB-KU Leuven Center for Brain & Disease Research.

"We will use our established platform with human induced pluripotent stem cells (hiPSCs) as well as mouse models," says Prof. Ludo Van Den Bosch (VIB-KU Leuven), founding scientist of Augustine Tx and Co-Principal Investigator. "This will be done in close collaboration with Professor Philip Van Damme from the University Hospital in Leuven and the Leuven Center for ALS (LEUCALS)."

U.S. AS ASSOCIATION BACKING

"We urgently need more and better treatments that can improve and extend the lives of people with ALS," said Kuldip Dave, Ph.D., Senior Vice President of Research at the ALS Association. "HDAC6 is a promising target, and we are proud to support continued preclinical investigations into potential new ALS therapies that leverage this pathway. Getting promising treatments out of the laboratory and into clinical testing as quickly as possible is key to making ALS a livable disease until we can cure it."

Sylvain Celanire, Augustine Tx' CEO, says: "It is an honor to receive the support of the ALS Association, a patient group at the forefront of public policy, working to improve the lives of people living with ALS and their caregivers. It is an additional confirmation of the quality of our research and expertise in accelerating a novel potential therapeutic option for ALS patients."

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About Augustine Therapeutics

Augustine Therapeutics is a Belgian biotech company, 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. For more information, visit https://augustinetx.com/.

About VIB

VIB's core mission is to generate disruptive insights in the molecular underpinning of life and to translate these actively into impactful innovations for patients and society. VIB is an independent research institute where some 1,800 top scientists from Belgium and abroad conduct pioneering basic research. As such, they are pushing the boundaries of what we know about molecular mechanisms and how they rule living organisms such as human beings, animals, plants, and microorganisms. Based on a close partnership with five Flemish universities – Ghent University, KU Leuven, University of Antwerp, Vrije Universiteit Brussel, and Hasselt University – and supported by a solid funding program, VIB unites the expertise of all its collaborators and research groups in a single institute. VIB's technology transfer activities translate research results into concrete benefits for society such as new diagnostics and therapies and agricultural innovations. These applications are often developed by young start-ups from VIB or through collaborations with other companies. This also leads to additional employment and bridges the gap between scientific research and entrepreneurship. VIB also engages actively in the public debate on biotechnology by developing and disseminating a wide range of science-based information. More info can be found on www.vib.be.

About the ALS Association

The ALS Association is the largest ALS organization in the world. The Association funds global research collaborations, assists people with ALS and their families through its nationwide network of certified clinical care centers, and advocates for better public policies for people with ALS. The Association is working to make ALS a livable disease while urgently searching for new treatments and a cure. Through its Lawrence and Isabel Barnett Drug Development Program, the Association supports the preclinical assessment of new and repurposed ALS therapies that have a high probability of entering the clinic within 3 years.  For more information about the ALS Association, visit www.als.org. For more information about the Lawrence and Isabel Barnett Drug Development Program, visit here

Media Contact

Sylvain Celanire
Chief Executive Officer
Email: info@augustinetx.com