RT001

BioJiva Reports Results of Pilot Phase 2 Clinical Trial of RT001 in Patients with Amyotrophic Lateral Sclerosis (ALS)

Retrieved on: 
Thursday, September 15, 2022
ALS Functional Rating Scale - Revised, ALS, Week, GLOBE, Degenerative disease, RT001, Patient, Amyotrophic lateral sclerosis, Pharmaceutical industry

LOS ALTOS, Calif., Sept. 15, 2022 (GLOBE NEWSWIRE) -- BioJiva, a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class therapies for degenerative diseases, today reported data from its completed multicenter, randomized, double-blind, placebo-controlled pilot Phase 2 clinical trial evaluating RT001, the company’s lead development candidate, in patients with amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease). The trial’s prespecified primary endpoint was change from baseline in the revised ALS Functional Rating Scale (ALSFRS-R) following 24 weeks of treatment. At 24 weeks, data demonstrated that patients treated with RT001 experienced less worsening (a 3.3-point reduction from baseline) in ALSFRS-R score as compared to greater worsening (4.6-point reduction from baseline) for the placebo group. Similarly, patients treated with RT001 experienced less worsening (a 13.3-point increase from baseline) in their score on the 40-item ALS assessment questionnaire (ALSAQ40) as compared to the placebo group (a 17.2-point increase from baseline). While suggesting a signal of directional improvement with RT001 treatment, these findings did not achieve statistical significance due to the small size of the study.

Key Points: 
  • While suggesting a signal of directional improvement with RT001 treatment, these findings did not achieve statistical significance due to the small size of the study.
  • This six-month randomized Phase 2 pilot study ( NCT04762589 ), which was followed by a six-month open-label extension during which all patients received RT001, was conducted at four ALS Centers of Excellence in Europe.
  • Investigators enrolled 43 patients with ALS who had symptom duration of less than three years.
  • BioJiva is not a progression of Retrotope and BioJiva is not the former Retrotope.

Positive Results from Studies of Retrotope’s RT011 in Animal Models of Retinal Degeneration to be Featured in Oral Presentations at 2nd Annual Dry AMD Therapeutic Development Summit

Retrieved on: 
Tuesday, October 19, 2021
ALS, IND, Visual impairment, Tissue, Death, PUFA, Cell damage, Investigational New Drug, Western world, FA, Reading, Oxidative stress, GLOBE, Patient, University, INAD, Animal, RT001, LA, Retinal pigment epithelium, Demonstration, GA, AMD, Infantile neuroaxonal dystrophy, Collection, Amyotrophic lateral sclerosis, Time, DHA, University of Pennsylvania, Vicious Circle, RATS, Disease, Drusen, LPO, Time-invariant system, Retina, Dihydroxy-E,Z,E-PUFA, Geographic atrophy, Inflammation, Central retinal artery occlusion, Degenerative disease, Dry, Injection, Â, Severe cognitive impairment, RPE, Lipid peroxidation, LOS, Program, Research, Stress (linguistics), PSP, Therapy, Vaccine, Pharmaceutical industry, Dietary supplement

LOS ALTOS, Calif., Oct. 19, 2021 (GLOBE NEWSWIRE) -- Retrotope, a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class therapies for degenerative diseases, today announced that positive results from preclinical studies of RT011, the second compound to emerge from the company’s novel technology platform, are being reported in multiple oral presentations at the 2nd Annual Dry AMD Therapeutic Development Summit. Findings to be presented demonstrate that RT011, which is being developed as an oral therapy for the treatment of dry age-related macular degeneration (AMD), provided dose-dependent protection in animal models of oxidative damage implicated in dry AMD. Importantly, study results showed that animals treated with RT011 experienced preservation of retinal cells, photoreceptors, and visual function relative to control animals. The conference, which is being conducted in a virtual format, is being held October 19-21, 2021.

Key Points: 
  • Findings to be presented demonstrate that RT011, which is being developed as an oral therapy for the treatment of dry age-related macular degeneration (AMD), provided dose-dependent protection in animal models of oxidative damage implicated in dry AMD.
  • Importantly, study results showed that animals treated with RT011 experienced preservation of retinal cells, photoreceptors, and visual function relative to control animals.
  • The company expects to file an Investigational New Drug (IND) application for RT011 in the first half of 2022 and initiate first-in-human clinical studies in dry AMD soon thereafter.
  • Study results demonstrated that animals treated with RT011 experienced decreasing levels of retinal damage with increasing tissue concentrations of RT011.

Retrotope Reports Data from Phase 2/3 Clinical Trial of RT001 and Concurrent Natural History Study in Patients with Infantile Neuroaxonal Dystrophy (INAD)

Retrieved on: 
Wednesday, October 6, 2021
Infantile neuroaxonal dystrophy, Food, PLA2G6, Infant, Degenerative disease, Failure, FA, University of California, Patient, NDA, Lipid peroxidation, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Therapy, RT001, Hypotonia, Amyotrophic lateral sclerosis, FDA, Disorder, Death, Spasticity, AMD, PSP, Dihydroxy-E,Z,E-PUFA, INAD, University, Food and Drug Administration, LPO, Neurology, Comparative effectiveness research, LA, Disease, Autonomic nervous system, New Drug Application, Pneumonia, HIV disease progression rates, Mitochondrial membrane transport protein, Technology, DHA, ALS, Research, Oxidative stress, Tissue, Brain, Genetics, Radical (chemistry), LOS, Survival, SAP, Membrane, Neurodegeneration, Totality, Seizure, GLOBE, Metabolism, Pharmaceutical industry

LOS ALTOS, Calif., Oct. 06, 2021 (GLOBE NEWSWIRE) -- Retrotope, a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class therapies for degenerative diseases, today reported data from its Phase 2/3 clinical trial of RT001 in patients with infantile neuroaxonal dystrophy (INAD) and its concurrent natural history study of disease onset and progression in INAD patients. Results demonstrated statistically significant improvements in overall survival and progression free survival for patients treated with RT001 as compared to control. These survival endpoints included a combination of efficacy and safety measures. Additionally, clinical improvements were observed in patients receiving RT001 as measured by the Modified Ashworth Spasticity Scale, the study’s primary efficacy endpoint, and other measurements of efficacy as compared to patients in the natural history study. These single efficacy outcomes did not reach statistical significance likely due to the small study size.

Key Points: 
  • Results demonstrated statistically significant improvements in overall survival and progression free survival for patients treated with RT001 as compared to control.
  • The treatment study of RT001 included 19 INAD patients and the concurrent natural history study included 36 INAD patients as the control arm.
  • Patients in the treatment study received RT001 for a minimum period of one year with a 30-day treatment free follow up period.
  • Infantile Neuroaxonal Dystrophy is an ultra-rare, infantile genetic neurological disorder and part of a spectrum of diseases called PLA2G6-associated neurodegeneration.

Retrotope Announces Completion of Enrollment in Phase 2 Study of RT001 in Patients with Progressive Supranuclear Palsy (PSP)

Retrieved on: 
Wednesday, August 11, 2021
ALS, Hospital, Radical (chemistry), Amyotrophic lateral sclerosis, Dihydroxy-E,Z,E-PUFA, Speech, Health, Medulla oblongata, Walking, Safety, FA, Caregiver, Patient, Retrotope, Physician, Neurodegeneration, PSP, Lipid peroxidation, Technology, Hannover Medical School, Membrane, Massachusetts General Hospital, Death, Cognition, INAD, Tissue, HIV disease progression rates, LOS, Clinical trial, Brain, Behavior, Government budget balance, RT001, Massachusetts, LA, LPO, Research, Oxidative stress, Cell, Degenerative disease, AMD, Department of Neurology, Xuanwu hospital, History, FDA, Disease, GLOBE, Infantile neuroaxonal dystrophy, Mitochondrial membrane transport protein, Neurology, DHA, Pharmaceutical industry

PSP is a progressive, neurodegenerative disorder that dramatically impacts the health and lives of not only patients, but also their families and other caregivers.

Key Points: 
  • PSP is a progressive, neurodegenerative disorder that dramatically impacts the health and lives of not only patients, but also their families and other caregivers.
  • Study participants have been randomized to receive either RT001 or placebo daily for 48 weeks.
  • The primary endpoint of the trial is change from baseline in the PSP Rating Scale (PSPRS) at 48 weeks.
  • The study also includes several secondary and exploratory endpoints intended to further elucidate the efficacy and safety profile of RT001 as compared to placebo.

DGAP-News: US FDA Allows Trial to Proceed for Retrotope's RT001 in the Treatment of Progressive SupraNuclear Palsy (PSP)

Retrieved on: 
Tuesday, April 28, 2020
Branches of biology, Chemistry, Physical sciences, Biochemistry, Di-deuterated linoleic acid ethyl ester, Lipids, Membrane biology, Lipid peroxidation, Cell membrane, Retrotope, Deuterated drug, RT001, Retrotope

In addition, the FDA provided useful guidance and suggestions for detailed design elements of the trial.

Key Points: 
  • In addition, the FDA provided useful guidance and suggestions for detailed design elements of the trial.
  • PSP is a serious neurodegenerative disease that profoundly affects the quality and length of life in adults1.
  • A regionally specific increase in lipid peroxidation damage, the target of RT001, has been observed in PSP.
  • RT001 is a chemically stabilized fatty acid that confers resistance to lipid peroxidation in mitochondrial and cellular membranes via a novel mechanism.

DGAP-News: Retrotope Initiates Project to Test RT001 in an Animal Model of Lung Damage to Protect Against COVID-19 Impact

Retrieved on: 
Thursday, April 16, 2020
Branches of biology, Biochemistry, Di-deuterated linoleic acid ethyl ester, Trinucleotide repeat disorders, Chemistry, RTT, Huntington's disease, Medicine, RT001, Retrotope

Hence, even when treated with the current standard of care, the recovery rate for patients progressing onto ventilator support is low.

Key Points: 
  • Hence, even when treated with the current standard of care, the recovery rate for patients progressing onto ventilator support is low.
  • Several theories abound, one of which points to an overly-stimulated immune system leading to long-term lung damage or death.
  • In the first test of the effect of RT001 on lung damage, rodents will be orally dosed with either RT001 or a non-active control.
  • Expanded Access trials calibrating endpoint effects of RT001 in ALS, PSP, Huntington's disease, and others are also underway.