Alesta Therapeutics Raises $67 Million as It Emerges from Stealth

Rare Daily Staff
Alesta Therapeutics, a Leiden, Netherlands-based biotechnology company focused on developing small molecule therapies for rare diseases, emerged from stealth as it announced it raised $67 million (€65 million) in a series A financing round.

Frazier Life Sciences and Droia Ventures co-lead the round, which included participation from Novartis Venture Fund, RTW Investments, RV Invest, Thuja Capital, and SSI Strategy.

Alesta is developing two small molecule drugs designed for oral administration and expects to initiate clinical studies in 2025. Ilan Ganot, an investment banker who began a biotech founder and CEO after his son was diagnosed with Duchenne muscular dystrophy, is CEO of Alesta.

The company’s lead experimental therapy, ALE1, is an orally active candidate for the treatment of hypophosphatasia (HPP), a rare genetic disorder caused by mutations in the ALPL gene. The condition leads to defective bone and tooth mineralization, resulting in fragile bones, early tooth loss and, in adult patients, clinically significant muscle weakness and fatigue.

ALE1 inhibits a novel target to reduce levels of inorganic pyrophosphate (PPi), a key metabolite central to the disease’s pathology. Preclinical data supports ALE1 as a clinical candidate, demonstrating a reduction of PPi levels across multiple animal models following oral administration.
Alesta is currently conducting toxicology studies on ALE1 and expects to initiate clinical studies in 2025. The company believes that ALE1 can potentially address the entire spectrum of HPP. The asset was in-licensed from 1cBio, and the 1cBio team continues to support Alesta.

Alesta is also developing ALE2 to address tRNA mutations that cause specific forms of Charcot-Marie-Tooth (CMT), a group of inherited peripheral neuropathies that lead to progressive muscle weakness, sensory loss, and limb deformities.

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.

“Closing this financing marks an important moment for Alesta,” said Alesta CEO Ganot, CEO of Alesta Therapeutics. “With the support of our investors, the expertise and commitment of our team and collaborations with leading academics and disease advocacy groups, we are poised to make significant strides in addressing the unmet needs of patients with rare diseases. This funding enables us to advance ALE1 toward clinical proof of concept and accelerate the development of ALE2, bringing us closer to delivering transformative therapies to those who need them most.”