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At 24 weeks, data demonstrated that patients treated with RT001 (linoleic Acid) 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.


Lead Product(s): Di-deuterated Linoleic Acid Ester

Therapeutic Area: Neurology Product Name: RT001

Highest Development Status: Phase IIProduct Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable September 15, 2022

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RT001 to impact the progression of INAD based on the compound’s demonstrated ability to down-regulate lipid peroxidation used in patients with infantile neuroaxonal dystrophy.


Lead Product(s): Linoleic Acid

Therapeutic Area: Rare Diseases and Disorders Product Name: RT001

Highest Development Status: Phase II/ Phase IIIProduct Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable October 06, 2021

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Comapny's first D-PUFA drug candidate in development, RT001, has shown robust safety in over 50 patient years of oral dosing, and we expect RT011 to have a similar safety profile.


Lead Product(s): DHA

Therapeutic Area: Ophthalmology Product Name: RT011

Highest Development Status: PreclinicalProduct Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable July 21, 2020

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Retrotope filed this IND for a PSP Phase 2/3 trial after 3 patients treated with RT001 in PSP Expanded Access protocols showed encouraging results after more than a year on drug.


Lead Product(s): Linoleic Acid

Therapeutic Area: Neurology Product Name: RT001

Highest Development Status: IND EnablingProduct Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable April 25, 2020

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A post-treatment tissue evaluation will determine if RT001 is capable of ameliorating inflammatory lung injury.


Lead Product(s): RT001

Therapeutic Area: Infections and Infectious Diseases Product Name: Undisclosed

Highest Development Status: PreclinicalProduct Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable April 16, 2020

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The company stated that, RT001 has a synergistic downstream benefit in the pathophysiology of PSP. This orphan designation encourages clinical trials and rapid review of data from trials to treat PSP.


Lead Product(s): Linoleic Acid

Therapeutic Area: Neurology Product Name: RT001

Highest Development Status: IND EnablingProduct Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable February 18, 2020

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With this trial, Retrotope expands its pipeline of indications for RT001 which is currently being studied in a pivotal trial in Infantile Neuroaxonal Dystrophy, which is fully enrolled.


Lead Product(s): Linoleic Acid

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Phase II/ Phase IIIProduct Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable January 09, 2020

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