A phase 1, open-label, single-center study of absorption, metabolism and excretion and absolute bioavailability in humans of leriglitazone (MIN-102) in 2 parallel cohorts of male healthy subjects - hAME and ABA combined study with leriglitazone

Start Date08 Mar 2022

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100 Clinical Results associated with Leriglitazone

100 Translational Medicine associated with Leriglitazone

100 Patents (Medical) associated with Leriglitazone

Literatures (Medical) associated with Leriglitazone

03 Oct 2024·Brain

Leriglitazone halts disease progression in adult patients with early cerebral adrenoleukodystrophy

Article

Author: Berger, Johannes ; Barbier, Magali ; Pichon, Bertrand ; Yazbeck, Elise ; Mochel, Fanny ; Blanco, Bernardo ; Galanaud, Damien ; Perlbarg, Vincent ; Weinhofer, Isabelle ; Martinell, Marc ; Pascual, Silvia ; Chaumette, Boris ; Huiban, Camille ; Golse, Marianne ; Fatemi, Ali

AbstractCerebral adrenoleukodystrophy (CALD) is an X-linked rapidly progressive demyelinating disease leading to death usually within a few years. The standard of care is haematopoietic stem cell transplantation (HSCT), but many men are not eligible due to age, absence of a matched donor or lesions of the corticospinal tracts (CST). Based on the ADVANCE study showing that leriglitazone decreases the occurrence of CALD, we treated 13 adult CALD patients (19–67 years of age) either not eligible for HSCT (n = 8) or awaiting HSCT (n = 5).Patients were monitored every 3 months with standardized neurological scores, plasma biomarkers and brain MRI comprising lesion volumetrics and diffusion tensor imaging.The disease stabilized clinically and radiologically in 10 patients with up to 2 years of follow-up. Five patients presented with gadolinium enhancing CST lesions that all turned gadolinium negative and, remarkably, regressed in four patients. Plasma neurofilament light chain levels stabilized in all 10 patients and correlated with lesion load. The two patients who continued to deteriorate were over 60 years of age with prominent cognitive impairment. One patient died rapidly from coronavirus disease 2019.These results suggest that leriglitazone can arrest disease progression in adults with early-stage CALD and may be an alternative treatment to HSCT.

01 Jun 2024·CPT: Pharmacometrics & Systems Pharmacology

Clinical pharmacokinetics of leriglitazone and a translational approach using PBPK modeling to guide the selection of the starting dose in children

Article

Author: Rodríguez‐Pascau, Laura ; Eckland, David ; Pascual, Silvia ; Martinell, Marc ; Pizcueta, Pilar ; Pina, Guillem ; Ke, Alice ; Meya, Uwe ; Poli, Sonia ; Lindauer, Andreas ; van de Wetering, Jeroen ; Traver, Estefania

AbstractLeriglitazone is a unique peroxisome proliferator‐activated receptor‐gamma (PPARγ) agonist that crosses the blood–brain barrier in humans and clinical trials have shown evidence of efficacy in neurodegenerative diseases. At clinical doses which are well‐tolerated, leriglitazone reaches the target central nervous system (CNS) concentrations that are needed for PPARγ engagement and efficacy; PPARγ engagement is also supported by clinical and anti‐inflammatory biomarker changes in the Cerebrospinal fluid in the CNS. Plasma pharmacokinetics (PK) of leriglitazone were determined in a phase 1 study in male healthy volunteers comprising a single ascending dose (SAD) and a multiple ascending dose (MAD) at oral doses of 30, 90, and 270 mg and 135 and 270 mg, respectively. Leriglitazone was rapidly absorbed with no food effect on overall exposure and showed a linear PK profile with dose‐exposure correlation. A physiologically based pharmacokinetic (PBPK) model was developed for leriglitazone based on phase 1 data (SAD part) and incorporated CYP3A4 (fmCYP3A4 = 24%) and CYP2C8‐mediated (fmCYP2C8 = 45%) metabolism, as well as biliary clearance (feBIL = 19.5%) derived from in vitro data, and was verified by comparing the observed versus predicted concentration‐time profiles from the MAD part. The PBPK model was prospectively applied to predict the starting pediatric doses and was preliminarily verified with data from five pediatric patients.

26 Oct 2023·Journal of translational medicine

Mitochondrial modulation with leriglitazone as a potential treatment for Rett syndrome.

Article

Author: Vergara, Cristina ; Oyarzábal, Alfonso ; Castells, Alba A ; Alcántara, Soledad ; Pizcueta, Pilar ; García-Cazorla, Angels ; Rodríguez-Pombo, Pilar ; Martinell, Marc ; Rodríguez-Pascau, Laura ; Musokhranova, Uliana ; Grau, Cristina ; Xiol, Clara

BACKGROUNDRett syndrome is a neuropediatric disease occurring due to mutations in MECP2 and characterized by a regression in the neuronal development following a normal postnatal growth, which results in the loss of acquired capabilities such as speech or purposeful usage of hands. While altered neurotransmission and brain development are the center of its pathophysiology, alterations in mitochondrial performance have been previously outlined, shaping it as an attractive target for the disease treatment.METHODSWe have thoroughly described mitochondrial performance in two Rett models, patients' primary fibroblasts and female Mecp2^tm1.1Bird-/+ mice brain, discriminating between different brain areas. The characterization was made according to their bioenergetics function, oxidative stress, network dynamics or ultrastructure. Building on that, we have studied the effect of leriglitazone, a PPARγ agonist, in the modulation of mitochondrial performance. For that, we treated Rett female mice with 75 mg/kg/day leriglitazone from weaning until sacrifice at 7 months, studying both the mitochondrial performance changes and their consequences on the mice phenotype. Finally, we studied its effect on neuroinflammation based on the presence of reactive glia by immunohistochemistry and through a cytokine panel.RESULTSWe have described mitochondrial alterations in Rett fibroblasts regarding both shape and bioenergetic functions, as they displayed less interconnected and shorter mitochondria and reduced ATP production along with increased oxidative stress. The bioenergetic alterations were recalled in Rett mice models, being especially significant in cerebellum, already detectable in pre-symptomatic stages. Treatment with leriglitazone recovered the bioenergetic alterations both in Rett fibroblasts and female mice and exerted an anti-inflammatory effect in the latest, resulting in the amelioration of the mice phenotype both in general condition and exploratory activity.CONCLUSIONSOur studies confirm the mitochondrial dysfunction in Rett syndrome, setting the differences through brain areas and disease stages. Its modulation through leriglitazone is a potential treatment for this disorder, along with other diseases with mitochondrial involvement. This work constitutes the preclinical necessary evidence to lead to a clinical trial.

News (Medical) associated with Leriglitazone

11 Dec 2024

·pipelinereview.com

Leriglitazone has met the primary endpoint in NEXUS, the pivotal trial for pediatric patients with cerebral Adrenoleukodystrophy

• Statistically significant difference in arrested disease in leriglitazone treated patients when compared to the natural history of the disease • Submission of European marketing authorization application (MAA) for leriglitazone expected mid-2025. BARCELONA, Spain and DÜSSELDORF, Germany I December 11, 2024 I Minoryx Therapeutics, a late-stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders and Neuraxpharm Group (Neuraxpharm), a leading European specialty pharmaceutical company focused on the treatment of CNS disorders, today announced that the NEXUS trial has been completed and that the primary endpoint has been met. The parties intend to file for European Marketing Authorization of leriglitazone in pediatric and adult cerebral Adrenoleukodystrophy (cALD) patients by mid-2025. The NEXUS trial was a 96-week, pivotal, open-label study designed to evaluate the efficacy and safety of once-daily oral dosing of leriglitazone in pediatric patients with cALD. The primary endpoint assessed the proportion of patients that had clinical and radiological arrested disease at week 96 or at a visit prior to hematopoietic stem cell transplant (HSCT). The evaluable population in the study consisted of 20 patients treated for a minimum of 24 weeks. All 20 patients remained clinically stable whilst on treatment and 7 out of 20 patients (35%) met the arrested disease criteria, which is significantly greater than the 10% self-arrested patients that would be expected from natural history (p<0.05). Leriglitazone was well tolerated in all children, there were no treatment-related serious adverse events nor discontinuations due to treatment related adverse events. The full results of the NEXUS trial will be presented at upcoming neurology conferences. “Cerebral ALD in boys is a devastating disease both for the patients and their families. Treatment options are limited, physicians and families of patients have constantly been searching for better tools and therapies to manage cALD in boys.” said Patricia Musolino, Global Principal Investigator of Nexus. “The NEXUS results attest that leriglitazone addresses a critical unmet need for non-invasive treatments that can be administered at the moment brain lesions are identified to arrest or slow down lesion growth.” “We are very pleased with the positive results from NEXUS demonstrating that leriglitazone not only arrests the demyelinating brain lesions in children with cALD but also arrests clinical progression. These results are further supported by evidence from the cALD related endpoints of ADVANCE1 in adult patients and the findings reported in cALD patients treated under compassionate use1” said Marc Martinell, CEO, Minoryx. ”Together with our partner, Neuraxpharm, we intent to submit the MAA to the EMA for this life-threatening condition as soon as possible.” Dr. Jörg-Thomas Dierks, CEO, Neuraxpharm said; “cALD is a very serious neurological disorder with devastating outcomes for patients and their families. The results from this trial are extremely encouraging and we are fully committed to working with Minoryx to swiftly provide patients with an effective new treatment for this otherwise fatal and cruel disease.” Based on the successful results from NEXUS, Minoryx and Neuraxpharm have begun to compile the regulatory file for MAA submission in the European Union. About leriglitazone Leriglitazone is Minoryx Therapeutics’ novel orally bioavailable, selective PPAR gamma agonist with a potential first-in-class and best-in-class profile for CNS diseases. It has demonstrated 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, mitochondrial dysfunction, oxidative stress, and axonal degeneration. In clinical trials, leriglitazone showed clinical benefit in both pediatric X-ALD patients in NEXUS and adult X-ALD patients in ADVANCE. Results from NEXUS demonstrate that pediatric cALD patients are clinically and radiologically stable after 96 weeks of treatment or at a visit prior to HSCT. Data from ADVANCE showed that leriglitazone reduces the progression of lesions and the development of progressive cALD in adult patients. Another clinical trial in adult male patients with progressive cALD (CALYX2) is currently recruiting in the US, Argentina, Brazil and Europe. Leriglitazone has been granted orphan drug status for X-ALD from the FDA and the EMA and Fast Track and Rare Pediatric Disease designation from the FDA for the treatment of X-ALD. About NEXUS NEXUS is a 96-week, pivotal, open-label, multicenter study (NEXUS; NCT04528706) of once-daily oral leriglitazone in pediatric patients with cALD. NEXUS has enrolled 23 patients and the evaluable population consisted of those patients treated for a minimum of 24 weeks (n=20). The primary endpoint assesses the proportion of evaluable patients that have clinically and radiologically arrested disease at week 96 or at a visit prior to HSCT (success criteria: one-sided 95% [CI] > 10%). Secondary endpoints include change from baseline in NFS and Loes score (LS). Change from baseline in lesion volume and plasma biomarker concentrations are exploratory endpoints. About X-ALD and cALD X-linked adrenoleukodystrophy (X-ALD) is an orphan neurodegenerative disease. The global incidence of X-ALD is approximately 6-8/100,000 live births. Boys and adult men with X-ALD can, at any point in their lifetime, develop cALD, which is characterized by demyelinating brain lesions that may become rapidly progressive, leading to acute neurological decline and death. These lesions can produce severe symptoms such as loss of voluntary movements, inability to swallow, loss of communication, cortical blindness and total incontinence and death with a mean survival of 3 to 4 years. Progressive cALD occurs in 31-35% of ALD patients in childhood with typical onset between the age of 2-12 and up to 60% of adult patients with X-ALD will develop progressive cALD over time. There is currently no pharmacological treatment available for cALD. In childhood, Hematopoietic Stem Cell Transplantation (HSCT) can arrest the disease. However, it is an aggressive procedure and is only available for a portion of patients. Gene therapy based HSCT is not globally available, and it requires myeloablative chemotherapy with associated comorbidities. In adults, experience in HSCT is very limited and this intervention is often not recommended. In addition, all X-ALD patients reaching adulthood develop adrenomyeloneuropathy (AMN). This form progresses chronically and cALD patients with advanced AMN are largely ineligible to HSCT due to the poor prognosis of the treatment. About Minoryx Minoryx Therapeutics is a registration stage biotech company focusing on the development of novel therapies for orphan central nervous system (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 to treat 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, Criteria BioVentures, 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 https://www.minoryx.com/ . About the Neuraxpharm Group Neuraxpharm is a leading European specialty pharmaceutical company focused on the treatment of the central nervous system (CNS), including both psychiatric and neurological disorders. It has a unique understanding of the CNS market built over 35 years. Neuraxpharm is constantly innovating, with new products and solutions to address unmet patient needs and is expanding its portfolio through its pipeline, partnerships and acquisitions. The company has more than 1,000 employees and develops and commercializes CNS products through a direct presence in more than 20 countries in Europe, two in Latin America, one in the Middle East and globally via partners in more than 50 countries. Neuraxpharm is backed by funds advised by Permira. Neuraxpharm manufactures many of its pharmaceutical products at Neuraxpharm Pharmaceuticals (formerly Laboratorios Lesvi) in Spain. For more information, please visit www.neuraxpharm.com . 1 ADVANCE, a pivotal phase 2/3 randomized, double-blind, placebo-controlled, clinical study with an open-label extension, was designed to assess the efficacy and safety of leriglitazone in male patients with X-ALD. 2 CALYX, a phase 3, multicenter, randomized (1:1), double-blind, placebo-controlled, clinical study, has been designed to compare the efficacy and safety of leriglitazone in male adult patients with progressive cALD ( https://clinicaltrials.gov/study/NCT05819866 ). SOURCE: Neuraxpharm

Phase 3Clinical ResultOrphan DrugFast Track

11 Jul 2024

·www.businesswire.com

BrainTale Biomarker Platform Supports Drug Development in Demyelinating Disease

STRASBOURG, France--( BUSINESS WIRE )--BrainTale, a medtech deciphering white matter to enable better brain care, spin-off of the Paris Region Greater Hospitals announces the publication of the article by Marianne Golse in the peer-reviewed journal Brain entitled “ Leriglitazone halts disease progression in adult patients with early cerebral adrenoleukodystrophy “ supporting the benefits of its biomarker platform as non-invasive reliable and objective lesion-assessment tool in the drug development targeting white matter diseases. It enables the non-invasive monitoring of disease evolution and demonstration of therapeutic efficacy in adrenoleukodystrophy patients. Long underestimated in neuroscience, white matter, which represents 60% to 80% of the human brain, plays a key role in its proper functioning, development, and aging, whether normal or pathological. Accordingly, since its creation in 2018, BrainTale has been developing non-invasive, accessible, actionable in multicentric frameworks, and clinically validated measurement and predictive tools for patients suffering from brain diseases, such as CALD. With a strong collaborative approach, the company’s ambition is to provide investigators with relevant measure of the brain to improve patient care and clinical development of new disease-modifying drugs. CALD is an X-linked demyelinating disease that progresses rapidly and usually leads to death within a few years. As a rare condition, underdiagnosed and insufficiently understood, CALD is an example of crucial medical need patient population awaiting therapeutic options and reliable diagnostic and monitoring tools. The article “ Leriglitazone halts disease progression in adult patients with early cerebral adrenoleukodystrophy ” published in Brain and accessible here shows notably that DTI biomarkers as provided by Braintale are relevant markers of myelin integrity in humans, and that they are sensitive to disease progression in myeloneuropathy and CALD patients population. Connecting white matter quantification with clinical symptoms, the data provided by the BrainTale platform demonstrated strong correlation with total lesion load and neurofilament light chains (NfL). Of note, BrainTale reports allow easy interpretation of calibrated DTI metrics, offering to imaging centers and physicians the white matter measures and biomarker perspective in their daily practice. Professor Fanny Mochel, Coordinator of the Paris Rare disease center for leukodystrophies and final author of the publication, comments: "B rainTale supports patient care through optimization of new disease-modifying drug development alongside its routine use in clinical settings. With its “DTI 2.0” technology approach, the BrainTale platform can monitor disease evolution and contribute to assessing therapeutic efficacy – a needed game-changer for leukodystrophy patients ". About BrainTale BrainTale is an innovative European medtech company measuring the brain through white matter quantification and standardization based on a commercially available software medical device solution. This regulatory-cleared AI software offers quality controlled, non-invasive, reliable and clinically validated reports after diffusion tensor MRI data processing. BrainTale enables drug developers, leading academic researchers and physicians to improve patient care by understanding neuroinflammatory and neurodegenerative conditions, assess brain lesions evolutions and take appropriate decisions in the clinic and during drug development. BrainTale empowers the scientific and medical community with objective measure to transform brain care. Because brain diseases have become the medical issue of our time, we can no longer wait. At BrainTale, we strongly believe that by exploring better, we can treat faster, together. For more information, please visit www.braintale.eu

29 Feb 2024

·www.prnewswire.com

Rare Disease Day 2024 - Call for greater awareness of X-linked adrenoleukodystrophy and cerebral adrenoleukodystrophy - special film launched

BARCELONA, Spain and DÜSSELDORF, Germany, Feb. 29, 2024 /PRNewswire/ -- Neuraxpharm Group (Neuraxpharm), a leading European specialty pharmaceutical company focused on the treatment of central nervous system (CNS) disorders, and Minoryx Therapeutics, a late stage biotech company focused on the development of therapies for orphan central nervous system disorders, reaffirm their commitment on Rare Disease Day 2024 to finding better treatments for patients suffering from a range of rare diseases, such as leukodystrophy, including X-linked adrenoleukodystrophy (X-ALD) and more specifically cerebral adrenoleukodystrophy (cALD). This Rare Disease Day, to raise awareness of this disease, Neuraxpharm and Minoryx announce the publication of a special information film with the participation of world renowned expert Professor Fanny Mochel, the Director of the Reference Center for Leukodystrophies - La Pitié-Salpêtrière Hospital, Paris, and Karen Harrison, Support Services Manager of Alex, The Leukodystrophy Charity (Alex-TLC). In the film, Professor Mochel describes the disease and the need for regular screening to identify patients as early as possible, while Karen Harrison speaks powerfully about the patient experience and need for better treatments. The film can be found on Neuraxpharm's and Minoryx's websites, YouTube and LinkedIn channels. Watch the film here. Dr. Jörg-Thomas Dierks, Chief Executive Officer of Neuraxpharm, said: "The impact this disease has on patients and their families cannot be overstated. At Neuraxpharm, we are committed to bringing better treatment to patients so that we can improve the lives of those affected by this devastating disease. We hope that this film will raise awareness and understanding of the condition." Marc Martinell, Chief Executive Officer of Minoryx said: "The X-ALD community deserve all our support to help them face the extremely challenging consequences of this disease, which in the case of cALD are fatal. We want to raise awareness of the diseases and the importance of regular monitoring and early detection as these are critical factors for timely management of these patients." On 26 January 2024, Neuraxpharm and its partner Minoryx Therapeutics stated that they are seeking a re-examination of leriglitazone, Minoryx's novel brain-penetrant and selective PPAR gamma agonist, by the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) for conditional approval for patients with cALD. Minoryx and Neuraxpharm strongly believe that leriglitazone has a positive benefit / risk balance in patients with cALD. About X-ALD and cALD X-linked adrenoleukodystrophy (X-ALD) is an orphan neurodegenerative disease. The global incidence of X-ALD is approximately 6-8/100,000 live births. Boys and adult men with X-ALD can, at any point in their lifetime, develop cALD, which is characterized by demyelinating brain lesions that may become rapidly progressive, leading to acute neurological decline and death. These lesions can produce severe symptoms such as loss of voluntary movements, inability to swallow, loss of communication, cortical blindness and total incontinence and death with a mean survival of 3 to 4 years. Progressive cALD occurs in 31-35% of ALD patients in childhood with typical onset between the age of 2-12 and up to 60% of adult patients, with X-ALD will develop progressive cALD over time. There is currently no pharmacological treatment available for cALD. In childhood, Hematopoietic Stem Cell Transplantation (HSCT) can arrest the disease. However, it is an aggressive procedure and only available for a portion of patients; autologous HSCT is not globally available; this still requires hemo-ablation therapy with associated comorbidities and long-term safety data is being gathered. In adults, experience in HSCT is very limited and the intervention is often not recommended. In addition, all X-ALD patients reaching adulthood develop adrenomyeloneuropathy (AMN), characterized by progressive spastic paraparesis, as well as progressive deterioration of balance and sensory function, and development of incontinence. This form progresses chronically with onset of symptoms typically in adulthood, affecting both men and women, and has poor prognosis. About leriglitazone Leriglitazone is Minoryx Therapeutics's novel orally bioavailable and selective PPAR gamma agonist with a potential first-in-class and best-in-class profile for CNS diseases. It has demonstrated 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 mitochondrial dysfunction, oxidative stress, neuroinflammation, demyelination and axonal degeneration. In clinical trials, leriglitazone showed clinical benefit in both adult X-ALD patients in ADVANCE and pediatric X-ALD patients in NEXUS. Data from ADVANCE showed that leriglitazone reduces the progression of lesions and the development of progressive cALD. Results on radiological stabilization seen in NEXUS after 24 weeks of treatment were similar to those attained with Hematopoietic Stem Cell Transplant (HSCT) or ex-vivo gene therapy, hence it is expected that leriglitazone could provide a comparable clinical benefit to cALD patients. Leriglitazone has been granted orphan drug status for X-ALD from the FDA and the EMA and Fast Track and Rare Pediatric Disease designation from the FDA for the treatment of X-ALD. About the Neuraxpharm Group Neuraxpharm is a leading European specialty pharmaceutical company focused on the treatment of the central nervous system (CNS), including both psychiatric and neurological disorders. It has a unique understanding of the CNS market built over 35 years. Neuraxpharm is constantly innovating, with new products and solutions to address unmet patient needs and is expanding its portfolio through its pipeline, partnerships and acquisitions. The company has c.1,000 employees and develops and commercializes CNS products through a direct presence in more than 20 countries in Europe, two in Latin America, and globally via partners in more than 40 countries. Neuraxpharm is backed by funds advised by Permira. Neuraxpharm manufactures many of its pharmaceutical products at Neuraxpharm Pharmaceuticals (formerly Laboratorios Lesvi) in Spain. For more information, please visit About Minoryx Minoryx Therapeutics is a registration stage biotech company focusing on the development of novel therapies for orphan central nervous system (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 to treat 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

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Indication	Highest Phase	Country/Location	Organization	Date
Adrenoleukodystrophy	Preclinical	EU	Minoryx Therapeutics SL	25 Jan 2023
Friedreich Ataxia	Discovery	DE	Minoryx Therapeutics SL	26 Mar 2019
Friedreich Ataxia	Discovery	BE	Minoryx Therapeutics SL	26 Mar 2019
Friedreich Ataxia	Discovery	FR	Minoryx Therapeutics SL	26 Mar 2019
Friedreich Ataxia	Discovery	ES	Minoryx Therapeutics SL	26 Mar 2019

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04528706 (NEXUS, PipelineReview) Manual	Not Applicable	Adrenoleukodystrophy	20	leriglitazone	(jlufatpmzl) = All 20 patients remained clinically stable whilst on treatment and 7 out of 20 patients (35%) met the arrested disease criteria, which is significantly greater than the 10% self-arrested patients that would be expected from natural history (p<0.05). wighjupevx (vgfcfvzcts )	Positive	11 Dec 2024
NCT03231878 (ADVANCE, Pubmed) Manual	Phase 2/3	Adrenoleukodystrophy	116	Leriglitazone	tmpuevyoxr(nxlnnmbeon) = uutvvlrczo hetshvbpff (gctitglyvo, 41·4) View more	Negative	01 Feb 2023
				Placebo	tmpuevyoxr(nxlnnmbeon) = qbfapcaatw hetshvbpff (gctitglyvo, 60·5) View more
NCT03917225 (FRAMES, Pubmed)	Phase 2	Friedreich Ataxia	39	Leriglitazone	azjzeauszy(ygdowfpksg) = iklxbdemgr vzzgcllcul (iywzwsqqbg )	-	01 Dec 2022
				Placebo	azjzeauszy(ygdowfpksg) = sdkvzeenvt vzzgcllcul (iywzwsqqbg )

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