17 October, 2019.
Mataró, Barcelona, Spain and Charleroi, Belgium, October 17, 2019 – Minoryx Therapeutics, a company that specializes in the development of innovative treatments for orphan Central Nervous System (CNS) diseases, today announces that its lead drug candidate, leriglitazone (MIN-102), has been granted Orphan Drug Designation in Friedreich’s Ataxia by the US Food and Drug Administration (FDA).
Thursday, October 17, 2019
Wednesday, October 16, 2019
Orphan Drugs In Development For The Treatment Of Friedreich’s Ataxia: Focus On Omaveloxolone
Shaila D Ghanekar, Wai Wai Miller, Colin J Meyer, Kevin J Fenelon, Alvin Lacdao, Theresa A Zesiewicz; Degenerative Neurological and Neuromuscular Disease 15 October 2019 Volume 2019:9 Pages 103—107 doi:10.2147/DNND.S180027
Investigations into the pathological mechanisms of FRDA have revealed the central role of frataxin deficiency and the subsequent lack of transcription factor Nrf2. Consequently, the Nrf2 activators discussed here as well as the several treatments in development focus on reversing the oxidative stress environment caused by frataxin deficiency. While the discussed orphan drugs show promise in the treatment of FRDA, further investigations must be conducted in order to ensure the efficacy and safety of such drugs, as well as to determine even more therapeutic options.
Investigations into the pathological mechanisms of FRDA have revealed the central role of frataxin deficiency and the subsequent lack of transcription factor Nrf2. Consequently, the Nrf2 activators discussed here as well as the several treatments in development focus on reversing the oxidative stress environment caused by frataxin deficiency. While the discussed orphan drugs show promise in the treatment of FRDA, further investigations must be conducted in order to ensure the efficacy and safety of such drugs, as well as to determine even more therapeutic options.
Monday, October 14, 2019
Reata Announces Positive Topline Results from the MOXIe Registrational Trial of Omaveloxolone in Patients with Friedreich’s Ataxia
ACHIEVED PRIMARY ENDPOINT OF STATISTICALLY SIGNIFICANT IMPROVEMENT IN MFARS COMPARED TO PLACEBO AFTER 48 WEEKS OF TREATMENT
CONFERENCE CALL WITH MANAGEMENT SCHEDULED FOR OCTOBER 15, 2019, AT 8:00 AM ET
IRVING, Texas, Oct. 14, 2019 (GLOBE NEWSWIRE) -- Reata Pharmaceuticals, Inc. (Nasdaq: RETA), a clinical-stage biopharmaceutical company, announced today that the registrational Part 2 portion of the MOXIe Phase 2 trial of omaveloxolone in patients with Friedreich’s ataxia (FA) met its primary endpoint of change in the modified Friedreich’s Ataxia Rating Scale (mFARS) relative to placebo after 48 weeks of treatment. Patients treated with omaveloxolone (150 mg/day) demonstrated a statistically significant, placebo-corrected 2.40 point improvement in mFARS after 48 weeks of treatment (p=0.014). Omaveloxolone treatment was generally reported to be well-tolerated. Based on these positive results, and subject to discussions with regulatory authorities, the company plans to proceed with the submission of regulatory filings for marketing approval in the United States and internationally.
CONFERENCE CALL WITH MANAGEMENT SCHEDULED FOR OCTOBER 15, 2019, AT 8:00 AM ET
IRVING, Texas, Oct. 14, 2019 (GLOBE NEWSWIRE) -- Reata Pharmaceuticals, Inc. (Nasdaq: RETA), a clinical-stage biopharmaceutical company, announced today that the registrational Part 2 portion of the MOXIe Phase 2 trial of omaveloxolone in patients with Friedreich’s ataxia (FA) met its primary endpoint of change in the modified Friedreich’s Ataxia Rating Scale (mFARS) relative to placebo after 48 weeks of treatment. Patients treated with omaveloxolone (150 mg/day) demonstrated a statistically significant, placebo-corrected 2.40 point improvement in mFARS after 48 weeks of treatment (p=0.014). Omaveloxolone treatment was generally reported to be well-tolerated. Based on these positive results, and subject to discussions with regulatory authorities, the company plans to proceed with the submission of regulatory filings for marketing approval in the United States and internationally.
Sunday, October 13, 2019
Iron–Sulfur Cluster Metabolism Impacts Iron Homeostasis, Ferroptosis Sensitivity, and Human Disease
Vladislav O. SviderskiyErdem M. TerziRichard Possemato, Ferroptosis in Health and Disease. Springer, Cham. doi:10.1007/978-3-030-26780-3_12
We ask whether dysregulation of ISC synthesis, observed in multiple pathological settings including neurodegenerative diseases such as Friedreich’s Ataxia, sideroblastic anemia, and cancer, leads to pathology as a result of ferroptosis.
We ask whether dysregulation of ISC synthesis, observed in multiple pathological settings including neurodegenerative diseases such as Friedreich’s Ataxia, sideroblastic anemia, and cancer, leads to pathology as a result of ferroptosis.
Saturday, October 12, 2019
Reata Pharmaceuticals Reacquires Rights From AbbVie to Develop and Commercialize Bardoxolone Methyl, Omaveloxolone, and All Next-Generation Nrf2 Activators
IRVING, Texas, Oct. 10, 2019 (GLOBE NEWSWIRE) — Reata Pharmaceuticals, Inc. (Nasdaq: RETA), a clinical-stage biopharmaceutical company, today announced the reacquisition of development, manufacturing and commercialization rights concerning its proprietary Nrf2 activator product platform originally licensed to AbbVie, Inc. (AbbVie) for territories outside of the United States with respect to bardoxolone methyl (bardoxolone) and worldwide with respect to omaveloxolone and other next-generation Nrf2 activators. As a result, Reata now possesses exclusive, worldwide rights to develop, manufacture and commercialize bardoxolone methyl (bardoxolone), omaveloxolone, and all other next-generation Nrf2 activators, excluding certain Asian markets for bardoxolone which are licensed to Kyowa Kirin Co., Ltd.
Friday, October 11, 2019
Induced pluripotent stem cells-derived neurons from patients with Friedreich ataxia exhibit differential sensitivity to resveratrol and nicotinamide
Pauline Georges, Maria-Gabriela Boza-Moran, Jacqueline Gide, Georges Arielle Pêche, Benjamin Forêt, Aurélien Bayot, Pierre Rustin, Marc Peschanski, Cécile Martinat & Laetitia Aubry ; Scientific Reports volume 9, Article number: 14568 (2019) doi:10.1038/s41598-019-49870-y
By comparing the effects of both molecules on different cell types that may be considered to be non-relevant for the disease, such as fibroblasts, or more relevant to the disease, such as neurons differentiated from iPSCs, a differential response was observed; this response suggests the importance of developing more predictive in vitro systems for drug discovery. Our results demonstrate the value of utilizing human iPSCs early in drug discovery to improve translational predictability.
By comparing the effects of both molecules on different cell types that may be considered to be non-relevant for the disease, such as fibroblasts, or more relevant to the disease, such as neurons differentiated from iPSCs, a differential response was observed; this response suggests the importance of developing more predictive in vitro systems for drug discovery. Our results demonstrate the value of utilizing human iPSCs early in drug discovery to improve translational predictability.
Tuesday, October 8, 2019
Minoryx Therapeutics termine le recrutement de FRAMES, son essai clinique de phase 2 du leriglitazone dans l’ataxie de Friedreich
Mataró, Barcelone, Espagne et Charleroi, Belgique, le 8 octobre 2019 - MinoryxTherapeutics, une société spécialisée dans le développement de traitements innovants contredes maladies orphelines du système nerveux central (SNC), annonce aujourd’hui avoirterminé le recrutement des patients dans son essai clinique de phase 2 FRAMES sur l’ataxiede Friedreich avec son candidat médicament leriglitazone (MIN-102), un nouvel agoniste du PPARγ.
L’essai FRAMES est une étude multicentrique et randomisée, en double-aveugle contreplacebo, qui vise à évaluer l’efficacité et l’innocuité du leriglitazone chez les patients souffrantd’ataxie de Friedreich. Le recrutement de 39 patients dans quatre pays européens a été réalisé au cours des derniers quatre mois et demi en avance sur les prévisions. Les patients, âgés de 12 à 60 ans, vont recevoir durant un an une dose quotidienne de leriglitazone en suspension orale. L’objectif principal de l’essai consiste à évaluer l’effet du candidat médicament sur la progression de la maladie. Cet effet sera mesuré par imagerie de pointe de la moëlle épinière. Les objectifs secondaires incluent l’innocuité et la tolérance, l’effet sur d’autres observations cliniques, telles que les retours des patients, l’échelle d’incapacité fonctionnelle et les biomarqueurs exploratoires.
L’essai FRAMES est une étude multicentrique et randomisée, en double-aveugle contreplacebo, qui vise à évaluer l’efficacité et l’innocuité du leriglitazone chez les patients souffrantd’ataxie de Friedreich. Le recrutement de 39 patients dans quatre pays européens a été réalisé au cours des derniers quatre mois et demi en avance sur les prévisions. Les patients, âgés de 12 à 60 ans, vont recevoir durant un an une dose quotidienne de leriglitazone en suspension orale. L’objectif principal de l’essai consiste à évaluer l’effet du candidat médicament sur la progression de la maladie. Cet effet sera mesuré par imagerie de pointe de la moëlle épinière. Les objectifs secondaires incluent l’innocuité et la tolérance, l’effet sur d’autres observations cliniques, telles que les retours des patients, l’échelle d’incapacité fonctionnelle et les biomarqueurs exploratoires.
Minoryx completa el reclutamiento del ensayo clínico de Fase II de leriglitazona para la ataxia de Friedreich
Mataró (Barcelona)-Charleroi (Bélgica), 8 de octubre de 2019. Minoryx Therapeutics, compañía especializada en el desarrollo de tratamientos innovadores para las enfermedades huérfanas del sistema nervioso central (SNC), anuncia hoy que ha completado el reclutamiento de pacientes en el ensayo clínico FRAMES de Fase II de su fármaco leriglitazona (MIN-102) para la ataxia de Friedreich (FRDA).
FRAMES es un ensayo multicéntrico, aleatorio, doble ciego y controlado con placebo que evaluará la eficacia y la seguridad de leriglitazona en pacientes con FRDA. En total se han reclutado a 39 pacientes en cuatro países europeos en solo cuatro meses y medio, mucho antes de lo previsto. Los pacientes, que tienen entre 12 y 60 años, recibirán leriglitazona administrada una vez al día por vía oral durante un año. El objetivo principal del ensayo es controlar el efecto sobre la progresión de la enfermedad, que se medirá mediante imágenes de resonancia magnética de última generación de la médula espinal. Los objetivos secundarios incluyen la seguridad y tolerabilidad.
FRAMES es un ensayo multicéntrico, aleatorio, doble ciego y controlado con placebo que evaluará la eficacia y la seguridad de leriglitazona en pacientes con FRDA. En total se han reclutado a 39 pacientes en cuatro países europeos en solo cuatro meses y medio, mucho antes de lo previsto. Los pacientes, que tienen entre 12 y 60 años, recibirán leriglitazona administrada una vez al día por vía oral durante un año. El objetivo principal del ensayo es controlar el efecto sobre la progresión de la enfermedad, que se medirá mediante imágenes de resonancia magnética de última generación de la médula espinal. Los objetivos secundarios incluyen la seguridad y tolerabilidad.
MINORYX THERAPEUTICS COMPLETES ENROLLMENT IN FRAMES PHASE 2 TRIAL WITH LERIGLITAZONE IN FRIEDREICH’S ATAXIA
Mataró, Barcelona, Spain and Charleroi, Belgium, October 8, 2019.
Minoryx Therapeutics, a company specializing in the development of innovative treatments for orphan central nervous system (CNS) diseases, today announces that it has completed recruitment in the FRAMES phase 2 clinical trial of its novel PPARγ agonist, leriglitazone (MIN-102), in patients with Friedreich’s Ataxia.
FRAMES is a multicenter, randomized, double-blind, placebo-controlled trial that will assess the efficacy and safety of leriglitazone in patients with Friedreich’s Ataxia. Recruitment of 39 patients in four European countries was completed in just four and a half months, well ahead of schedule. The patients, aged 12-60 years, will receive leriglitazone, administered once a day as an oral suspension, for a total of one year. The primary objective of the trial is to monitor the effect on disease progression. This will be measured through state-of-the art imaging of the spinal cord. Secondary objectives include safety and tolerability, effect on additional clinical measures, such as patient reported outcomes, functional disability scores and exploratory biomarkers.
Minoryx Therapeutics, a company specializing in the development of innovative treatments for orphan central nervous system (CNS) diseases, today announces that it has completed recruitment in the FRAMES phase 2 clinical trial of its novel PPARγ agonist, leriglitazone (MIN-102), in patients with Friedreich’s Ataxia.
FRAMES is a multicenter, randomized, double-blind, placebo-controlled trial that will assess the efficacy and safety of leriglitazone in patients with Friedreich’s Ataxia. Recruitment of 39 patients in four European countries was completed in just four and a half months, well ahead of schedule. The patients, aged 12-60 years, will receive leriglitazone, administered once a day as an oral suspension, for a total of one year. The primary objective of the trial is to monitor the effect on disease progression. This will be measured through state-of-the art imaging of the spinal cord. Secondary objectives include safety and tolerability, effect on additional clinical measures, such as patient reported outcomes, functional disability scores and exploratory biomarkers.
Subscribe to:
Posts (Atom)
