About ALESTA Therapeutics
Pioneering Small Molecule Therapeutics
Pioneering Small Molecule Therapeutics at Leiden
Based in Leiden, Netherlands, our company is leading the development of innovative small molecules for rare diseases. With advanced chemistry, our clinical candidates are backed by extensive pre-GLP toxicology and are gearing up for GLP toxicology studies.
We are supported by a syndicate of renowned investors, including Frazier Life Sciences, Droia Ventures, Novartis Venture Fund, RTW Investments, RV Invest, Thuja Capital and SSI Strategy.
ALE1
A new approach for treating hypophosphatasia
Hypophosphatasia (HPP) is a rare genetic disorder that severely affects the development of bones and teeth due to defective mineralization. HPP can result in a spectrum of symptoms, including fragile bones, early tooth loss and, in adult patients, clinically significant muscle weakness and fatigue. The US prevalence is estimated to be around 50,000 patients and approximately 75,000 in the EU (including UK).
Current treatments only target bone-related symptoms and have low tolerability. We are developing an oral small molecule therapy, ALE1, that has the potential to address the entire spectrum of HPP and fundamentally transform patient care. ALE1 is currently progressing through GLP toxicology studies, and Alesta expects to initiate clinical studies in 2025.
ALE2
Targeting GCN2 to combat Charcot-Marie-Tooth disease
Charcot-Marie-Tooth (CMT) is a debilitating inherited neurological disorder that affects motor and sensory functions in the hands and feet, impacting approximately 126,000 people in the US and 2.6 million worldwide. Research has linked CMT to genetic alterations in tRNA synthetases, such as GARS, which are crucial for tRNA loading with amino acids. These alterations can lead to significant motor neuropathy, characterized by disabling muscle weakness and potential immobility.
We are developing ALE2 as an oral small molecule therapy for specific forms of CMT. ALE2 targets chronic neurotoxicity by inhibiting GCN2, a key regulator of the Integrated Stress Response (ISR) that becomes activated by ribosomal stalling caused by tRNA synthase mutations associated with CMT.
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