RT001 / BioJiva - LARVOL DELTA

Double blind trial of a deuterated form of linoleic acid (RT001) in Friedreich ataxia. (PubMed, J Neurol) - "The results of this study provide no evidence for a significant benefit of RT001 at the dosages tested in this Friedreich ataxia patient population."

Journal • Ataxia • CNS Disorders • Friedreich ataxia • Movement Disorders

BioJiva Reports Results of Pilot Phase 2 Clinical Trial of RT001 in Patients with Amyotrophic Lateral Sclerosis (ALS) (GlobeNewswire) - P2 | N=40 | NCT04762589 | Sponsor: Retrotope, Inc. | “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.”

P2 data • Amyotrophic Lateral Sclerosis • CNS Disorders

An Expanded Access Protocol of RT001 in Amyotrophic Lateral Sclerosis-Initial Experience with a Lipid Peroxidation Inhibitor. (PubMed, Muscle Nerve) - "RT001 was administered safely to a small group of people living with ALS in the context of an EAP. There is an ongoing randomized, double-blind controlled study of RT001 in ALS."

Clinical • Journal • Amyotrophic Lateral Sclerosis • CNS Disorders • Gastrointestinal Disorder

A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia (clinicaltrials.gov) - P3 | N=65 | Completed | Sponsor: Retrotope, Inc. | Active, not recruiting ➔ Completed | Trial completion date: Dec 2021 ➔ Aug 2021

Trial completion • Trial completion date • Ataxia • Friedreich ataxia • Movement Disorders

RT001 in Amyotrophic Lateral Sclerosis (clinicaltrials.gov) - P2 | N=40 | Recruiting | Sponsor: Retrotope, Inc. | Trial completion date: Sep 2021 ➔ Sep 2022 | Trial primary completion date: Sep 2021 ➔ Aug 2022

Trial completion date • Trial primary completion date • Amyotrophic Lateral Sclerosis • CNS Disorders

Phase 2/3 Clinical Trial Results: Safety & Efficacy Results of RT001, a site-specific (C11) di-deutero synthetic homologue of linoleic acid, in Infantile Neuroaxonal Dystrophy (AAN 2022) - "There was statistically significant improvement in survival, and trends toward improvement in all functional assessments, in children treated with RT001 compared to the natural history cohort. We posit that these results are clinically meaningful, and suggest that RT001 can slow disease progression in children with INAD."

Clinical • P2/3 data • CNS Disorders • Genetic Disorders • Infectious Disease • Movement Disorders • Pneumonia • Respiratory Diseases

Retrotope Drug Improves Survival in Ultra-Rare Genetic Childhood Disease (BioSpace) - “On April 21, Retrotope completed enrollment of its Phase II trial of RT001 in ALS (Lou Gehrig’s disease). They exceeded the enrollment target of 40 patients in less than six weeks. They expect data from the trial by the end of the year.”

P2 data • Amyotrophic Lateral Sclerosis • CNS Disorders

A Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy (clinicaltrials.gov) - P2/3; N=19; Active, not recruiting; Sponsor: Retrotope, Inc.; Trial completion date: Jun 2021 ➔ Jun 2022

Clinical • Trial completion date • CNS Disorders

Retrotope Announces Completion of Enrollment in Phase 2 Study of RT001 in Patients with Progressive Supranuclear Palsy (PSP) (GlobeNewswire) - “Retrotope…announced that enrollment has been completed for its multicenter Phase 2 clinical trial evaluating RT001, the company’s lead development candidate, in patients with progressive supranuclear palsy (PSP). The study’s enrollment target of 40 patients was exceeded in less than six weeks, highlighting the PSP patient community’s significant interest in participating in a clinical trial of a novel, oral treatment approach to this devastating neurodegenerative disease.”

Enrollment closed • CNS Disorders • Progressive Supranuclear Palsy

RT001 in Progressive Supranuclear Palsy-Clinical and In-Vitro Observations. (PubMed, Antioxidants (Basel)) - "MSCs derived from patients with PSP have elevated basal levels of LPO, ROS, and mitochondrial dysfunction. These findings are reversed after incubation with RT001. In PSP patients, the progression of disease may be reduced by treatment with RT001."

Journal • Preclinical • CNS Disorders • Metabolic Disorders • Movement Disorders • Progressive Supranuclear Palsy

Retrotope Announces Initiation of Phase 2 Study of RT001 in Patients with Progressive Supranuclear Palsy (PSP) (GlobeNewswire) - “Retrotope…announced that the first patient has been dosed in a multicenter Phase 2 clinical trial evaluating RT001, the company’s lead development candidate, in patients with progressive supranuclear palsy (PSP).”

Trial status • CNS Disorders • Progressive Supranuclear Palsy

RT001 in Patients With Progressive Supranuclear Palsy (PSP) PROGRESSIVE SUPRANUCLEAR PALSY (clinicaltrials.gov) - P2; N=40; Recruiting; Sponsor: Retrotope, Inc.

Clinical • New P2 trial • CNS Disorders • Movement Disorders • Progressive Supranuclear Palsy

A RANDOMIZED, DOUBLE-BLIND, CONTROLLED, PHASE 2 STUDY TO ASSESS EFFICACY, LONG TERM SAFETY AND TOLERABILITY OF RT001 IN SUBJECTS WITH PROGRESSIVE SUPRANUCLEAR PALSY. (clinicaltrialsregister.eu) - P2; N=40; Ongoing; Sponsor: Retrotope, Inc

Clinical • New P2 trial • CNS Disorders • Movement Disorders • Progressive Supranuclear Palsy

Retrotope Announces Completion of Enrollment in Phase 2 Study of RT001 in Patients with Amyotrophic Lateral Sclerosis (ALS) (GlobeNewswire) - “Retrotope…announced that enrollment has been completed for its multicenter 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 study’s enrollment target of 40 patients was exceeded in less than six weeks...Based on this rapid patient enrollment, Retrotope expects data from the trial to be available by the end of 2021.”

Enrollment status • P2 data • Amyotrophic Lateral Sclerosis • CNS Disorders

[VIRTUAL] Retrotope, Inc. (BIO 2021) - "The company’s lead candidate, RT001, is in clinical development for a range of orphan neurodegenerative diseases including infantile neuroaxonal dystrophy, Friedreich’s ataxia, amyotrophic lateral sclerosis (ALS) and progressive supranuclear palsy. In addition, the company is advancing a second candidate, RT011, toward the clinic for the treatment of dry age-related macular degeneration (AMD)."

Age-related Macular Degeneration • Amyotrophic Lateral Sclerosis • Ataxia • CNS Disorders • Complement-mediated Rare Disorders • Dry Age-related Macular Degeneration • Friedreich ataxia • Macular Degeneration • Movement Disorders • Ophthalmology • Progressive Supranuclear Palsy • Retinal Disorders

Retrotope Announces Initiation of Phase 2 Study of RT001 in Patients with Amyotrophic Lateral Sclerosis (ALS) (GlobeNewswire) - “Retrotope…announced that the first patients have been dosed in a multicenter Phase 2 clinical trial evaluating RT001, the company’s lead development candidate, in patients with amyotrophic lateral sclerosis…expects the study to enroll rapidly, and data to be available by the end of 2021.”

P2 data • Trial status • Amyotrophic Lateral Sclerosis • CNS Disorders

A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia (clinicaltrials.gov) - P3; N=65; Active, not recruiting; Sponsor: Retrotope, Inc.; Trial completion date: Apr 2021 ➔ Dec 2021; Trial primary completion date: Feb 2021 ➔ Nov 2021

Clinical • Trial completion date • Trial primary completion date • Ataxia • Friedreich ataxia • Movement Disorders

Retrotope Granted Rare Pediatric Disease Designation from FDA for Lead Development Candidate, RT001, in Two Life-Threatening Neurodegenerative Indications (GlobeNewswire) - "Retrotope...today announced that the U.S. Food and Drug Administration (FDA) has granted two rare pediatric disease designations to RT001...The first rare pediatric disease designation is for the treatment of...Friedreich’s ataxia (FA). In addition, RT001 has been granted Fast Track designation by the FDA for the treatment of FA....Retrotope is eligible to receive a priority review voucher should the company secure approval for RT001 in either one of these indications....Additionally, the company is currently conducting a pivotal Phase 2/3 trial of RT001 in patients with FA. Enrollment in this study was completed in late 2020 and data is expected to read out by the end of 2021."

Enrollment closed • Fast track designation • FDA event • P2/3 data • Friedreich ataxia

RT001 in Amyotrophic Lateral Sclerosis (clinicaltrials.gov) - P2; N=40; Recruiting; Sponsor: Retrotope, Inc.

Clinical • New P2 trial • Amyotrophic Lateral Sclerosis • CNS Disorders • Complement-mediated Rare Disorders

A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PHASE 2 STUDY TO ASSESS EFFICACY, LONG TERM SAFETY AND TOLERABILITY OF RT001 IN SUBJECTS WITH AMYOTROPHIC LATERAL SCLEROSIS (clinicaltrialsregister.eu) - P2; N=40; Ongoing; Sponsor: Retrotope, Inc.

Clinical • New P2 trial • Amyotrophic Lateral Sclerosis • CNS Disorders • Complement-mediated Rare Disorders

Retrotope Appoints Rick E Winningham as Chairman of Board of Directors in Advance of Key Clinical Trial Data Readouts (GlobeNewswire) - “A Phase 2 clinical trial of RT001 is planned in patients with ALS, with first patient dosing expected in the first quarter of 2021 and data read out anticipated by the end of 2021. This trial is being informed by the company’s single-arm expanded access program study for RT001 in ALS, which treated 18 patients for up to 24 months and demonstrated a slowing of disease progression when compared to historical placebo responses from a separate ALS clinical trial.”

New P2 trial • P2 data • Amyotrophic Lateral Sclerosis • CNS Disorders

A Natural History Study of Infantile Neuroaxonal Dystrophy (clinicaltrials.gov) - P=N/A; N=45; Active, not recruiting; Sponsor: Retrotope, Inc.; Recruiting ➔ Active, not recruiting; Trial primary completion date: Dec 2020 ➔ Dec 2021

Clinical • Enrollment closed • Trial primary completion date • CNS Disorders

A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia (clinicaltrials.gov) - P3; N=65; Active, not recruiting; Sponsor: Retrotope, Inc.; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed • Ataxia • Friedreich ataxia • Movement Disorders

US FDA allows trial to proceed for Retrotope’s RT001 in the treatment of Progressive Supranuclear Palsy (PSP) (Issuer Direct) - "Retrotope announced today that it received a 'Study May Proceed' letter from US Food and Drug Administration (FDA) related to its recently filed Investigational New Drug (IND) application for the use of RT001 in the treatment of PSP....Retrotope filed this IND for a PSP Phase 2/3 trial after 3 patients treated by physicians collaborating with Retrotope in PSP Expanded Access protocols showed encouraging results after more than a year on drug."

FDA event • IND • New P2/3 trial • CNS Disorders • Progressive Supranuclear Palsy

A Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy (clinicaltrials.gov) - P2/3; N=19; Active, not recruiting; Sponsor: Retrotope, Inc.; Trial completion date: Jul 2020 ➔ Jun 2021

Clinical • Trial completion date • CNS Disorders