July 09, 2018 Retrotope Announces Publication of Preclinical Research Showing Cognitive Improvements in a Huntington’s Disease Model Using Deuterated Polyunsaturated Fatty Acid (D-PUFA) Drug Candidate

Retrotope and collaborators led by Dr. Marie-Francoise Chesselet, the Charles H. Markham Professor
of Neurology, and Interim Chair of the Department of Neurology at UCLA, have published a paper demonstrating that the Company’s D-PUFA drug candidate, RT001, reduces cognition deficits in a preclinical in vivo model of Huntington’s disease, while simultaneously downregulating brain lipid peroxidation biomarkers. The paper, entitled “Deuterium reinforced linoleic acid lowers lipid peroxidation and mitigates cognitive impairment in the Q140 knock in mouse model of Huntington's disease,” was published in The FEBS Journal.

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June 28, 2018 Retrotope Receives FDA Clearance to Commence RT001 Phase 2/3 Clinical Trial in Patients with Infantile Neuroaxonal Dystrophy (INAD)

Retrotope announced today that the U.S. Food and Drug Administration (FDA) has granted the company approval to conduct an open-­label Phase 2/3 clinical trial of its investigational drug RT001 to evaluate
efficacy and safety in patients with infantile neuroaxonal dystrophy (INAD). RT001 is the
first-­in-­class of a new category of drugs called D-­PUFAs (deuterated polyunsaturated fatty
acids), which are designed to protect against free radical damage resulting in cell death that
is a hallmark of numerous neurodegenerative diseases including INAD. To date, Retrotope
has enrolled two INAD patients in two separate Expanded Access trials, the first patient
having begun treatment in March 2017 and the second patient having enrolled in November
2017. Promising results from the first study were presented at the recent annual meeting of
the American Academy of Neurology, and treatment of both children remains ongoing.
RT001 has been granted U.S. FDA orphan drug designation for the treatment of PLA2G6
associated neurodegeneration (PLAN), which includes INAD.

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June 11, 2018 Retrotope Announces the Initiation of Expanded Access (Compassionate Use) Trials of RT001 in Two Neurodegenerative Disorders

Late Onset Tay Sachs and Neuroserpinosis single patient trials mark the fourth and fifth trials of Retrotope’s drug candidate in an untreated neurological disease. 

Retrotope announced today the start of two single patient, Compassionate Use trials of RT001 at major university medical centers in Late Onset Tay Sachs (LOTS) disease and Familial Encephalopathy with Neuroserpin Inclusion Bodies (FEIN or neuroserpinosis).

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April 24, 2018: Retrotope Announces RT001 Clinical Data Presentation at the 2018 American Academy of Neurology (AAN) Annual Meeting.

Retrotope announced today positive findings from a compassionate use study of the company’s lead candidate, RT001, in a patient with Infantile Neuroaxonal Dystrophy (INAD), as presented at the 70th AAN Annual Meeting, being held in Los Angeles, CA, April 21-27, 2018. The study showed that administration of RT001 to a ~2.5-year-old patient appears to have arrested disease progression and led to re-acquisition of lost developmental milestones.

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April 18, 2018: Retrotope Announces Peer-Reviewed Publication of Positive Phase 1b/2a Findings for RT001 in Friedreich’s Ataxia

Retrotope announced today peer-reviewed publication of positive Phase 1b/2a trial results for the company’s lead candidate, RT001, in patients with Friedreich’s ataxia (FA).

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November 16, 2017: Retrotope initiates second patient for Compassionate Use of RT001 for an ultra-rare genetic neurological disease, Infantile Neuroaxonal Dystrophy.

Retrotope announced today that it has enrolled a second subject in a compassionate use trial of the ultra-rare, neurological disease, Infantile Neuroaxonal Dystrophy (INAD). 

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November 2, 2017: US FDA Grants Orphan Drug Designation for Retrotope’s RT001 in the Treatment of  Phospholipase 2G6 (PLA2G6)-Associated Neurodegeneration.

Retrotope announced today that the U.S. Food and Drug Administration’s (FDA’s) Office of Orphan Products Development (OOPD) granted orphan drug designation for its chemically-modified polyunsaturated fatty acid drug (RT001) for the treatment of PLA2G6 associated neurodegeneration (PLAN).  Physicians collaborating with Retrotope have previously received approval from the FDA’s Division of Neurology Products to test RT001 in Expanded Access trials of two patients having Infantile Neuroaxonal Dystrophy (INAD), a severe childhood PLA2G6-induced neurodegeneration.   These trials are underway.

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October 27, 2017: Study of Retrotope’s patented deuterated polyunsaturated fatty acid drugs is published in The FEBS Journal, and shows drugs fully rescue Alzheimer’s loss of cognition in a mouse model.

The study’s results demonstrate that combinations of Retrotope’s deuterated polyunsaturated fatty acid (D-PUFA) drugs rescue multiple cognition deficits in an aldehyde dehydrogenase 2 (ALDH2-/-) sporadic Alzheimer’s knockout mouse to unaffected wild type performance. Collaborators from Vanderbilt University and the University of Arkansas Stable Isotope Laboratory assisted in analyzing tissues for oxidation markers and tissue drug incorporation.

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April 26, 2017: Retrotope and collaborators to present poster at the 2017 American Academy of Neurosciences Meeting in Boston on stabilized PUFA drug efficacy in multiple Alzheimer’s models.

Retrotope and its collaborators, Dr. Brian Bennett of Queens University, Kingston, ON, Canada, and Dr. Mark Mattson, the National Institute of Aging, will present a joint poster at AAN, Boston this week entitled “Isotope-reinforced Polyunsaturated Lipids (D-PUFAs) Mitigate Symptoms in Different Alzheimer’s Disease Mouse Models”.   The results demonstrate that combinations of Retrotope’s deuterated polyunsaturated fatty acid (D-PUFA) drugs are active vs. inactive fat controls at both lowering amyloid beta fragments in brain in the APP/PS1 NIH Alzheimer’s mouse and reversing multiple cognition deficits in an aldehyde dehydrogenase 2 (ALDH2-/-) early onset knockout mouse.  Further collaborators from Vanderbilt University and the University of Arkansas Stable Isotope Laboratory are additional authors who assisted in analyzing tissues for oxidation markers and tissue drug incorporation.

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April 2, 2017: Retrotope to present first human data on the safety and early efficacy of its novel stabilized lipid drug platform against neurodegeneration.

On April 24, 2017 Dr. Theresa Zesiewicz from the University of South Florida Movement Disorders Clinic, will give a podium presentation at the American Academy of Neurology, Boston meeting, of clinical trial results of RT001 in the neuromuscular disease, Friedreich’s ataxia (FA).  The study showed excellent safety and tolerability of the novel drug class, including early signals of efficacy (and even reversal) of disease progression.  The trial, a randomized, double-blind, comparator controlled, study of RT001 in 18 FA patients for 28 days met all of its primary safety, tolerability and pharmacodynamics (PK) goals. While biological activity was not a primary goal of the study, a number of clinically important disease progression measures showed signals of drug effect unexpected in such a short, small study.

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September15, 2016: Retrotope announces Phase I/II Clinical Trial Results of RT001 in Treatment of Friedreich’s ataxia.

Dr. Theresa Zesiewicz, principal investigator in Retrotope’s first-in-human clinical trial of RT001 in Friedreich’s ataxia (FA), today presented early results from Retrotope’s Phase I/II trial conducted at the University of South Florida Ataxia Research Center and the Collaborative NeuroSciences Network in Long Beach, CA. The trial, a randomized, double-blind, comparator controlled, two-dose study of RT001 in 18 FA patients for 28 days, met all of its primary safety, tolerability and pharmacodynamics (PK) goals. While biological activity was not a primary goal of the study, a number of clinically important activity measures were tested, found to be highly correlated to well-studied disease severity scales and showed multiple, unexpected, robust signals of drug effect at one or more doses.

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June 1, 2016: US FDA Grants Orphan Drug Designation for Retrotope’s RT001 in the treatment of Friedreich’s ataxia. First-in-human clinical trial for RT001 is now fully enrolled.

Retrotope announced today that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development granted orphan drug designation for its stabilized fatty acid drug (RT001) for the treatment of Friedreich’s ataxia (FA). This follows the recent announcement that RT001 was well tolerated with no serious adverse events or dose limiting toxicities in the first cohort of its Phase 1/2 clinical trial in FA patients.

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February 19, 2016: Retrotope advances RT001 in clinical trials to treat Friedreich’s ataxia.
Announces open enrollment of highest dose cohort after safety review

Retrotope announced today the successful completion of the first dose cohort and the opening of patient enrollment for the highest dose cohort in its ongoing 28-day study of orally dosed RT001 in Friedreich’s ataxia (FA) patients. RT001 was well tolerated and no serious adverse events or dose limiting toxicities were observed.

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November 20, 2015: Retrotope announces opening of second clinical trial site for enrollment in Friedreich’s ataxia clinical trial

Retrotope announces the opening of second clinical trial site, the Collaborative Neuroscience Network, LLC. (“CNS”) in Long Beach, California, for the ongoing 28-day, first-in-human randomized, double-blind, controlled, ascending dose study of orally dosed RT001 to evaluate the safety, tolerability, pharmacokinetics (PK), disease state, and exploratory endpoints in patients with Friedreich’s ataxia (FA).

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August 17, 2015: Retrotope announces open enrollment for Friedreich’s ataxia clinical trial

Retrotope announces the opening of enrollment for a 28-day, first-in-human, randomized, double-blind, controlled, ascending dose study of orally dosed RT001 to evaluate the safety, tolerability, pharmacokinetics (PK), disease state, and exploratory endpoints in patients with Friedreich’s ataxia (FA).

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April 19, 2015: Retrotope appoints Dr. Ernst-Guenter Afting to its Board of Directors as it enters clinical stage.

Retrotope announced today that it has added Dr. Ernst-Guenter Afting to its Board of Directors in anticipation of its first-in-human clinical trial in the neurodegenerative disease, Friedreich’s ataxia.

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March 31, 2015: Retrotope appoints Dr. Peter Milner to its Board of Directors as it enters clinical stage.

Retrotope announced today that it has added Dr. Peter Milner to its Board of Directors in anticipation of its first-in-human clinical trial in the neurodegenerative disease, Friedreich’s ataxia.

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March 30, 2015: Retrotope presents at the International Ataxia Research Conference in Windsor, UK.

Retrotope gave a presentation today at the International Ataxia Research Conference. The talk entitled: “An upcoming clinical trial testing the safety and efficacy of a stabilized polyunsaturated fatty acid in Friedreich’s ataxia” elucidated the company’s progress towards an Investigational New Drug (IND) application for the fatal, orphan disease, Friedreich’s ataxia (FRDA).

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January 15, 2015: Retrotope closes $10M Series B financing.

The round was co-led by one of Retrotope’s original investors, Dr. Harry J. Saal, and a number of new outside angel investors. Groups participating in the round included past investors Life Science Angels, and new investors North Texas Angels, Green Park and Golf Ventures, SDL Ventures, and other insider and new individuals and family offices.

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January 15, 2015: Retrotope announces publication of results for a Parkinson’s Disease Model.

Results show that lipid peroxidation is required for alpha-synuclein induced cell death, a Parkinson’s Disease Model. The research demonstrates both the fact that lipid peroxidation is essential for alpha-inflicted cytotoxicity, and a novel therapeutic approach to mitigating this problem.

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December 9, 2014: Retrotope announces the issuance of new patents.

Retrotope, Inc., an emerging, clinical stage degenerative disease therapeutic company, announced today the issuance of a broad patent on its core technology of isotopically stabilized nutrient drugs.

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November 13, 2014: Retrotope appoints Dr. Harry J. Saal as Chairman of the Board of Directors.

Retrotope, Inc., an emerging, clinical stage degenerative disease therapeutic company, announced today that its long time investor, Dr. Harry J. Saal, will join the Board of Directors as its Chairman.

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