- Leriglitazone’s clinical proof of concept data in Friedreich’s Ataxia published in Neurology Genetics2022-11-29
Published data highlights therapeutic potential to treat patients with this condition
Mataró, Barcelona (Spain) November 29, 2022 - Minoryx Therapeutics, a late-stage biotech company focused on the development of treatments for orphan central nervous system (CNS) disorders today announces the publication in Neurology Genetics of efficacy and safety results from a Phase 2, randomized, double-blind, placebo-controlled study on the effects of leriglitazone on patients with Friedreich’s Ataxia (FRAMES study).
Although the primary endpoint was not met in this study, results from secondary endpoints provide evidence for clinical proof of concept for use of leriglitazone in patients with Friedreich’s Ataxia and support assessment in larger studies.
Iron accumulation in the dentate nucleus, a biomarker related with disease pathogenesis, was significantly reduced on the active arm. Additionally, numerical differences ware also observed in the spinal cord on metabolic markers assessed by magnetic resonance spectroscopy, as well as on CCFS, a functional assessment of upper limb ataxia. Treatment with leriglitazone was generally well tolerated.
Full text: https://ng.neurology.org/content/8/6/e200034
Leriglitazone (MIN-102) is Minoryx’s novel orally bioavailable and selective PPARγ agonist with a potential first-in-class and best-in-class profile indicated for CNS diseases. It has demonstrated sufficient brain penetration and a favorable safety profile. It showed robust preclinical proof-of-concept in animal models of multiple diseases by modulating pathways leading to neuroinflammation, demyelination and mitochondrial dysfunction. In clinical trials, leriglitazone showed clinical benefit for X-ALD patients and clinical proof of concept in Friedreich’s Ataxia patients supporting further larger studies in the future.
About Friedreich’s Ataxia
Friedreich’s Ataxia (FRDA) is a devastating, orphan genetic disease characterized by loss of coordination and muscle strength, resulting from the degeneration of nerves caused by a genetic defect. The disease is characterized by frataxin deficiency leading to mitochondrial dysfunction; symptoms range from the inability to coordinate movements to imbalance, muscle weakness and tremors. Within 10-15 years after disease onset, patients lose their ability to stand, sit and walk. Friedreich’s Ataxia is fatal, mainly due to cardiac failure. It affects approximately one in 40,000 people worldwide. There is currently no curative therapy available; existing treatments solely address symptoms.
Minoryx is a registration stage biotech company focusing on the development of novel therapies for orphan CNS diseases with high unmet medical needs. The company’s lead program, leriglitazone (MIN-102), a novel, brain penetrant and selective PPAR gamma agonist, is being developed in X-linked Adrenoleukodystrophy (X-ALD) and other orphan CNS diseases. The company is backed by a syndicate of experienced investors, which includes Columbus Venture Partners, CDTI Innvierte, Caixa Capital Risc, Fund+, Ysios Capital, Roche Venture Fund, Kurma Partners, Chiesi Ventures, S.R.I.W, Idinvest Partners / Eurazeo, SFPI-FPIM, HealthEquity, Sambrinvest and Herrecha, and has support from a network of other organizations. Minoryx was founded in 2011, is headquartered in Spain with Belgian facilities and has so far raised more than €120 million.
For more information, please visit www.minoryx.com