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

Data Readout from Potentially Pivotal Phase 2/3 INAD Trial Expected 1H ‘01; Phase 2 ALS and Pivotal Phase 2/3 Friedreich’s Ataxia Trial Readouts Expected by Year-End

Los Altos, California, UNITED STATES

LOS ALTOS, Calif., Feb. 11, 2021 (GLOBE NEWSWIRE) -- Retrotope, a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class therapies for degenerative diseases, today 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 decades of drug discovery, development, regulatory and commercialization experience in the global biopharmaceutical industry to his role as chairman. A member of the board of directors of the Biotechnology Industry Organization (BIO), his addition provides Retrotope with access to valuable leadership and expertise as it continues its transformation into a late clinical-stage drug development company with multiple clinical trial data readouts anticipated in 2021.

Mr. Winningham has served as chairman of the board of Theravance Biopharma (NASD: TBPH), a biopharmaceutical company focused on organ-selective medicines, since 2013, and the company’s CEO since its spin-off from Theravance, Inc. (renamed Innoviva, Inc.) in 2014. Starting in 2001, and up until the Theravance Biopharma spin-off, he served as CEO of Theravance, Inc., where he was also chairman of the board from 2010 to 2014. From 1997 to 2001, Mr. Winningham was president of Bristol-Myers Squibb’s Oncology/Immunology/Oncology Therapeutics Network (OTN), while also serving as the company’s president of global marketing from 2000 to 2001. Over a fifteen-year period beginning in 1986 with BMS and its predecessor, Bristol-Myers, Mr. Winningham held various U.S. and global management positions. Mr. Winningham has led the global development and commercialization of medicines across multiple therapeutic areas including oncology, cardiovascular, anti-infective, antiviral, respiratory, gastrointestinal and neuroscience.

“I am excited to join the Retrotope board as it transitions into a late clinical-stage biopharmaceutical company. By leveraging one of the most unique research platforms in the industry in pursuit of reducing cellular degeneration, Retrotope has created a broad pipeline of promising first-in-class programs aimed at some of the most serious and underserved degenerative diseases of our time, including orphan neurodegenerative conditions with no treatment options at all,” said Mr. Winningham. “2021 promises to be an important year with significant clinical progress as the company reads out data from three studies of its lead development candidate RT001. Additionally, Retrotope continues to make excellent progress advancing its second development candidate, RT011, toward the clinic as a potential treatment for dry age-related macular degeneration.”

Retrotope’s novel drug discovery and development platform is focused on combatting the oxidative stress and cellular degeneration that arises from lipid peroxidation (LPO). While all healthy human tissues undergo this physiological process of cell degeneration and repair, it is well-established that a wide range of serious degenerative diseases are precipitated when the LPO process becomes out of balance. Polysaturated fatty acids (PUFAs), which make up cell and mitochondrial membranes throughout the body and are vital to healthy cellular function, are the target of the LPO process due to their inherent instability. Free radicals in the body exploit the instability of PUFAs to trigger chain reactions that drive LPO hyperactivity. Retrotope’s novel platform technology is designed to stabilize PUFAs and down-regulate LPO in order to protect membranes from degeneration.

Retrotope has created a pipeline of first-in-class new chemical entity (NCE) drug candidates that are being developed for a number of diseases ranging from orphan neurodegenerative indications to large market degenerative diseases, all of which are triggered or accelerated by LPO.   RT001, the company’s lead development candidate, is a clinical-stage isotopically stabilized, synthetic linoleic acid (LA) that has been safely administered to more than 100 patients, spanning more than 1,000 patient months. The company’s second development candidate, RT011, is an isotopically stabilized, synthetic docosahexaenoic acid (DHA) that is advancing toward clinical development.

RT001 Clinical Program Highlights:

Infantile Neuroaxonal Dystrophy (INAD) – The most advanced clinical program in the company’s pipeline, Retrotope has completed dosing and expects data to read out from its potentially pivotal Phase 2/3 trial of RT001 in the first half of 2021.

INAD is an ultra-rare, fatal, infantile genetic neurological disorder that is eligible for Rare Pediatric Disease (RPD) designation and a RPD voucher from the U.S. Food and Drug Adminstration (FDA).

Friedreich’s Ataxia (FA) – Enrollment was completed in the ongoing pivotal Phase 2/3 trial of RT001 in FA in late 2020. Data from this trial is expected to read out by the end of 2021. Proof-of-concept for RT001 in FA was demonstrated in the company’s previously completed Phase 1/2 placebo-controlled trial.

FA is a rare, debilitating, life-shortening pediatric neurodegenerative disease. Like INAD, FA qualifies for RPD designation and a RPD voucher from FDA.

Amyotrophic Laterial Sclerosis (ALS) – 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.

Progressive Supranuclear Palsy (PSP) – A Phase 2 clinical trial of RT001 is planned in patients with PSP, with first patient dosing expected in the first half of 2021 and data read out anticipated in 2022. To date, RT011 has been administered to three PSP patients through an expanded access program, with all having received treatment for more than 27 months. For all three of these patients, disease progression significantly slowed or reversed at 12 months and 24 months according to standard severity scales used in the measurement of the disease.

RT011 Preclinical Program Highlights:

Dry Age-Related Macular Degeneration (AMD) – Retrotope expects to file an Investigational New Drug (IND) application for RT011 in mid-2021 and initiate first-in-human clinical studies in dry AMD soon thereafter. Unlike most other dry AMD treatments in development, RT011 is an oral drug, offering key administration advantages. Promising preclinical results have suggested the ability of RT011 to protect retinal cells and preserve photoreceptors in various AMD-related animal models.

Dry AMD is an early stage of AMD prior to a patient’s advancement to wet AMD and the ultimate onset of vision loss. It affects more than 7.5 million Americans and more than 20 million adults in the western world. Treatments for this condition are limited and there are no approved oral therapeutic options, providing a significant market opportunity for RT011.

About Retrotope

Retrotope is a clinical-stage biopharmaceutical company focused on the development of first-in-class therapies for degenerative diseases ranging from orphan neurodegenerative indications to large market degenerative conditions. The company leverages its proprietary drug discovery platform to create novel, disease-modifying drugs designed to combat the oxidative stress and cellular degeneration that arises from lipid peroxidation (LPO). It does so through the creation of isotopically stabilized synthetic versions of polysaturated fatty acids (PUFAs) that trigger the downregulation of the LPO process. The company’s lead development candidate, RT001, is in clinical development for a range of orphan neurodegenerative diseases including infantile neuroaxonal dystrophy (INAD), Friedreich’s ataxia (FA), amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) and progressive supranuclear palsy (PSP). In addition, the company is advancing its second development candidate, RT011, toward the clinic for the treatment of dry age-related macular degeneration (AMD).

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