Alesta Therapeutics, a biotechnology company focused on developing transformative small molecule therapies for rare diseases, announced the closing of an oversubscribed €65 million Series A funding round. This funding round was co-led by Frazier Life Sciences and Droia Ventures, with participation from Novartis Venture Fund, RTW Investments, RV Invest, Thuja Capital, and SSI Strategy.
Headed by a team of seasoned industry veterans with a strong chemistry background and expertise in rare disease drug development, the company is developing two small molecule therapeutics designed for oral administration. It expects to initiate clinical studies this year.
The company’s lead asset ALE1 is a unique, orally active therapeutic candidate for hypophosphatasia (HPP), which is a rare genetic disorder caused by mutations in the ALPL gene. This condition leads to defective bone and tooth mineralization, causing fragile bones, early tooth loss, and clinically significant muscle weakness and fatigue in adult patients.
ALE1 inhibits a novel target for reducing inorganic pyrophosphate (PPi) levels, a key metabolite central to the disease’s pathology. Preclinical data supports ALE1 as a clinical candidate, demonstrating the desired reduction of PPi levels across multiple animal models following oral administration.
ALE1 is currently progressing through GLP toxicology studies, and Alesta expects to initiate clinical studies in 2025. And the company believes that ALE1 has the potential to address the entire spectrum of HPP and fundamentally transform patient care across this underserved disease. This asset was in-licensed from 1cBio, and the 1cBio team continues to support Alesta.
Alesta is also developing ALE2 for addressing 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. And this condition affects over 10,000 patients across the US, EU and Japan.
ALE2 targets chronic neurotoxicity by inhibiting GCN2, which is a key regulator of the Integrated Stress Response (ISR) that becomes activated by ribosomal stalling caused by tRNA synthase mutations associated with CMT. The preclinical data supports ALE2 as a promising candidate for clinical development.
The Alesta team combines in-house chemistry and rare disease drug development expertise with insights from global academic and industry experts. Alesta is led by:
— Ilan Ganot, Chief Executive Officer
— Matthias Van Woensel, Ph.D., Chief Scientific Officer
— Benit Maru, MBChB, Ph.D., Chief Medical Officer
Along with the CEO, Alesta’s Board of Directors includes:
— Laura Brass, Ph.D., Novartis Venture Fund
— Luc Dochez, Droia Ventures
— Dan Estes, Ph.D., Frazier Life Sciences
— Janwillem Naesens, Droia Ventures
KEY QUOTES:
“Closing this financing marks an important moment for Alesta. 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.”
– Ilan Ganot, Chief Executive Officer of Alesta Therapeutics
“We are proud to support Alesta Therapeutics in its mission to address critical unmet needs in rare diseases. The preclinical progress of ALE1 and ALE2 underscores the company’s potential to deliver much-needed treatment options, and we are excited to support the continued advancement of these programs in 2025 and beyond.”
– Luc Dochez, Chairman of the Board
