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Reinforcing the Molecules of Life to Combat Degenerative Diseases

Human cells are continuously damaged by oxidative stress that is driven by lipid peroxidation (LPO). The resulting cellular degeneration contributes to a wide array of devasting degenerative diseases.

Retrotope is developing a pipeline of proprietary therapeutics designed to treat degenerative disease by down-regulating LPO in order to protect against cellular degeneration.

Science
of LPO

Development
Pipeline

Patient
Resources

OUR
MISSION

At Retrotope, we are pioneering the development of first-in-class therapies for devastating degenerative diseases - ranging from orphan neurodegenerative indications to large market degenerative conditions - for which there are few to no treatment options.

We are leveraging our proprietary drug discovery platform technology, along with our dedicated team of scientific leaders and industry veterans, to create novel, disease-modifying therapies designed to combat the oxidative stress and cellular degeneration that arise from lipid peroxidation.

Pipeline

Compound	Indication	Discovery/Preclinical	Phase I	Phase II	Phase III
RT001 Stabilized Linoleic Acid (LA, ethyl ester)	Infantile neuroaxonal dystrophy (INAD)
	Friedreich's ataxia (FA)
	Amyotrophic lateral sclerosis (ALS)
	Progressive supranuclear palsy (PSP)
RT011 Stabilized Docosahexaenoic acid (DHA, ethyl ester)	Dry Age-Related Macular Degeneration (Dry AMD) and geographic atrophy (GA)
Other stabilized PUFAs	Huntington’s disease (HD), Spinocerebellar ataxia (SCA) and Multiple system atropy (MSA)

NEWS & EVENTS

Press releases

April 2021 - Retrotope Announces Completion of Enrollment in Phase 2 Study of RT001 in Patients with Amyotrophic Lateral Sclerosis (ALS)

Press releases

March 2021 - Retrotope Announces Initiation of Phase 2 Study of RT001 in Patients with Amyotrophic Lateral Sclerosis (ALS)

Events

May 2021 - Anil Kumar, Retrotope’s president and CBO, to deliver corporate presentation at H.C. Wainwright Global Life Sciences Conference

Press releases

February 2021 - Retrotope Granted Rare Pediatric Disease Designation from FDA for Lead Development Candidate, RT001, in Two Life-Threatening Neurodegenerative Indications

Press releases

February 2021 - Retrotope Appoints Rick E Winningham as Chairman of Board of Directors in Advance of Key Clinical Trial Data Readouts

Publications

August 2020 - INAD Rating Scale

The study’s enrollment target of 40 patients was exceeded in less than six weeks, highlighting the high demand for participation in the trial featuring a novel treatment approach for ALS. Based on this rapid patient enrollment, Retrotope expects...

The first patient was dosed on March 10, with an additional eight patients enrolled during the study’s first week. Clinical investigators have already reported additional patient interest beyond the 40 subjects planned for the trial. As such,...

Retrotope, a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class therapies for degenerative diseases, today announced that Anil Kumar, president and chief business officer, will deliver a corporate...

Company Currently Conducting Late-Stage Clinical Trials in Both Infantile Neuroaxonal Dystrophy (INAD) and Friedreich’s Ataxia (FA) RT001 also Granted Fast Track Designation by FDA in FA; Orphan Drug Designation by European Medicines Agency in...

Retrotope announced the appointment of Rick E Winningham as chairman of the company’s board of directors. Mr. Winningham, who currently serves as chairman and chief executive officer (CEO) of Theravance Biopharma, Inc., brings more than three...

The infantile neuroaxonal dystrophy rating scale (INAD-RS). INAD is an autosomal recessive neurogenetic disorder caused by biallelic pathogenic variants in PLA2G6. The downstream enzyme, iPLA2, plays a critical role in cell membrane homeostasis by...

Leadership