Clinical trials testing Minoryx Therapeutics' leriglitazone (MIN-102), an investigational oral therapy for all forms of X-linked adrenoleukodystrophy (ALD) — including childhood cerebral ALD (CALD) and adrenomyeloneuropathy (AMN) — will receive up to €25 million ($29.4 million) in funding from the European Investment Bank (EIB). This funding aims to boost research and development in orphan neurodegenerative diseases for which no approved treatments are available. In the EU, an orphan disease is one that affects one in 2,000 people in the European population. “We are very happy to sign an agreement that reaffirms the EIB's commitment to fostering innovation in the healthcare sector by driving the development of new treatments that will have a positive impact on the quality of life of people affected by rare diseases of the central nervous system,” Ricardo Mourinho Félix, EIB’s vice-president, said in a press release. Leriglitazone is a selective PPAR gamma agonist that is able to cross the body's protective blood-brain barrier and enter the central nervous system, comprised of the brain and spinal cord. Once there, it prevents inflammation and oxidative stress — both processes related to ALD — and therefore protects nerve cells from damage. This therapy has been granted orphan drug status for the treatment of ALD, also known as X-ALD, in the European Union and the U.S. EIB now will fund two clinical studies to test leriglitazone as a treatment for CALD and AMN. CALD is the most aggressive form of X-ALD and it usually affects children between ages 2 and 12. AMN, meanwhile, is known as adult-onset ALD and is usually less severe and progresses more slowly. “The EIB brings Minoryx valuable financial resources, alongside our strong and supportive shareholders base, which, we believe, is a clear endorsement of the potential of leriglitazone to improve the lives of patients living with debilitating neurodegenerative disorders,” said Didier Le Normand, Minoryx's chief financial officer. The study in CALD (2019-000654-59), called NEXUS , is an open-label Phase 2 trial designed to assess the efficacy and safety of leriglitazone in 13 boys with early stage lesions related to CALD. The trial seeks to determine if leriglitazone can stop disease progression when administered before a blood stem cell transplant — the standard treatment for CALD. Secondary goals include changes in neurological function and in brain inflammation and overall survival. The ADVANCE study (NCT03231878), in AMN, is a double-blind, placebo-controlled, Phase 2/3, multi-center trial that has enrolled 116 men, ages 18 to 65, at centers across Europe and the U.S. Its objective is to evaluate leriglitazone's efficacy in preventing AMN progression. This parameter will be measured by the participants' ability to stand and walk as an indicator of motor function. “The current clinical studies with leriglitazone continue to advance as planned and we remain on track to report topline data from the pivotal ADVANCE study before the end of the year,” said Marc Martinell, PhD, co-founder and CEO of Minoryx. Leriglitazone also is being analyzed as a potential treatment for Friedreich's ataxia, another neurodegenerative disease characterized by a lack of coordination and muscle strength, and by heart disease. The just-completed study was a Phase 2 trial (NCT03917225) called FRAMES. “This EU support will help Minoryx to develop breakthrough therapies for genetic diseases and treatments for diseases of the central nervous system," said Paolo Gentiloni, the European commissioner for the economy. "The coronavirus pandemic has shown how important it is to continue to push scientific boundaries and deliver medication for rare diseases," Gentiloni said. "The European Commission will continue to support companies’ efforts in this sense at every opportunity.” The EIB will fund this project through a venture debt operation, a type of debt financing given to early stage companies and startups. This initiative targets European companies with up to 3,000 employees in fields such as biotechnology and health sciences. It has already granted over €2 billion ($2.35 billion) in financing for projects in sectors such as robotics, artificial intelligence, and biomedicine.