Tag Archives: MOTOR

Reata Enrolls First Patient in the MOTOR Study, a Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Mitochondrial Myopathies

IRVING, Texas, July 8, 2015 – Reata announces the enrollment of the first patient in MOTOR, a Phase 2 dose-ranging study examining the safety, tolerability, and efficacy of RTA 408 Oral Capsules for the treatment of patients with mitochondrial myopathies (MM).

MOTOR is a multi-center study planned for approximately 52 patients with MM. MM is a collective term for a group of individual rare diseases associated with mitochondrial DNA mutations. These defects cause respiratory chain deficits and impaired energy production. Most patients with MM share a similar phenotype with skeletal muscle weakness and fatigue. They also may have additional symptoms due to impaired energy production in other organ systems and often have reduced lifespans. Approximately 20,000 people in the United States are believed to have a form of MM. There are currently no approved therapies for MM.

The primary efficacy endpoint is the change in peak workload (Watts/kg) during exercise testing. The secondary endpoint includes a patient’s 6-minute walk distance. The study is also exploring the change in peak oxygen utilization during maximal exercise testing and changes in the Fatigue Severity Scale.

“Emerging translational research demonstrates that activation of Nrf2 (the target of RTA 408) can improve mitochondrial function and cellular energy production. These observations underlie our hypothesis that RTA 408 may improve exercise capacity and quality of life in patients with mitochondrial myopathies,” noted Dr. Colin Meyer, Reata’s Chief Medical Officer. “We are hopeful that RTA 408 will benefit mitochondrial myopathy patients, and we appreciate the guidance and support that Reata has received from the UMDF and the MM patient community.”

“We are very excited this trial will be underway with its first patient,” said Charles A. Mohan, Jr., Executive Director and CEO of the United Mitochondrial Disease Foundation. “Coordination, communication and collaboration between our industry partners and our patients to promote and support clinical trials is the only way we will accelerate the development of diagnostic tools, therapies, and potential cures for mitochondrial disease. We are pleased with the role Reata has taken in this endeavor and honored to call them a partner. We must all remember; no patients no trials, no trials no treatments nor cures.”

For more information on this study, visit: https://clinicaltrial.gov/ct2/show/NCT02255422.

About RTA 408 and Bioenergetics Effect

RTA 408 activates the body’s anti-oxidative pathways through transcription factor Nrf2 and is able to improve mitochondrial function. In mouse models of bioenergetic disease, RTA 408 demonstrated the ability to increase glucose uptake, fatty acid oxidation and oxygen consumption, direct signs of healthier cellular metabolism (http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3003357). RTA 408 analogs have also demonstrated in mouse models of oxidative stress the ability to induce genes related to mitochondrial biogenesis through the activation of Nrf2, which showed signs of potentially improving muscle function (http://www.ncbi.nlm.nih.gov/pubmed/21457778).

About Reata Pharmaceuticals, Inc.

Reata Pharmaceuticals, Inc. is a privately held company aiming to translate innovative research into breakthrough medicines for difficult diseases that have significant unmet needs. Reata is the leader in developing a novel class of drugs with potent transcription-regulating activity, called antioxidant inflammation modulators (AIMs). AIMs activate Nrf2, promoting the production of numerous antioxidant, detoxification, and anti-inflammatory genes, and inhibit NF-κB, a gene that regulates many pro-inflammatory proteins. The pharmacology of the AIMs mimics that of endogenous prostaglandin metabolites that are responsible for the orchestrated resolution of inflammation. The anti-inflammatory, cytoprotective and energy metabolism effects of AIM pharmacology have been documented in more than 250 scientific papers and are potentially relevant to a wide range of diseases.

CONTACT:
Reata Pharmaceuticals, Inc.
(972) 865-2219
info@reatapharma.com

Investor Relations:
The Trout Group
Lee M. Stern, CFA
lstern@troutgroup.com
(646) 378-2992

Reata Announces the Initiation of Phase 2 Studies Examining RTA 408 for the Treatment of Friedreich’s Ataxia and Mitochondrial Myopathies

IRVING, Texas, September 30, 2014 – Reata has received clearance from the Division of Neurology Products of the FDA to begin two new Phase 2 clinical programs in patients with Friedreich’s Ataxia and Mitochondrial Myopathies. Both of these orphan diseases are associated with reduced energy production, fatigue, and impaired exercise capacity. There are no existing therapies specifically approved to treat patients with these diseases.

Friedreich’s ataxia (FA) is an inherited disorder caused by defects in the gene for frataxin, a protein that regulates iron levels in the mitochondria. Defects in frataxin result in mitochondrial iron overload, causing impaired metabolism, oxidative stress, and damage to mitochondrial DNA. Patients with FA suffer progressive degeneration of the central and peripheral nervous systems, impaired coordination and gait, and fatigue from energy deprivation and muscle loss.

Mitochondrial Myopathies are a collection of individual orphan diseases that are associated with mitochondrial DNA mutations. These defects cause respiratory chain deficits and impaired energy production. Most of these patients share a similar phenotype characterized by skeletal muscle weakness and fatigue. These patients also may have other symptoms due to impaired energy production in other organ systems.

RTA 408 works by inducing Nrf2, which regulates multiple genes that play both direct and indirect roles in the production of cellular energy (i.e., adenosine triphosphate or ATP) within the mitochondria. Directly, activation of the Nrf2 pathway increases the efficient use of fuel (fatty acids and glucose) by mitochondria and increases mitochondrial biogenesis and basal oxygen consumption. Indirectly, activation of Nrf2, through its antioxidative effects, balances reducing equivalents and maintains mitochondrial homeostasis and efficiency. In addition to its positive effects on metabolic efficiency, Nrf2 activation has been shown in preclinical studies to promote muscle repair and recovery and reduce markers of oxidative stress and muscle injury.

“Our collaborators and we have shown in preclinical studies that genetic or pharmacologic Nrf2 activation positively regulates mitochondrial function and energy production. We hope to translate this effect into improved physical functioning and reduced fatigue in patients with Friedreich’s ataxia and mitochondrial myopathies. These rare, debilitating diseases currently have no approved therapies,” noted Dr. Colin Meyer, Reata’s Chief Medical Officer.

The two initial Phase 2 trials will both be multi-center, double-blind, randomized, dose-ranging, placebo-controlled studies. The primary efficacy endpoint in both studies will be peak work as assessed during exercise testing. The studies will also explore changes in other measures of physical activity, fatigue, and biomarkers associated with mitochondrial functioning.

About Reata Pharmaceuticals, Inc.

Reata Pharmaceuticals, Inc. is a privately held company aiming to translate innovative research into breakthrough medicines for difficult diseases that have significant unmet needs. Reata is the leader in developing a novel class of drugs with potent transcription-regulating activity called antioxidant inflammation modulators (AIMs). AIMs activate Nrf2, promoting the production of numerous antioxidant, detoxification, and anti-inflammatory genes, and inhibit NF-κB, a transcription factor that regulates many pro-inflammatory proteins. The pharmacology of the AIMs mimics that of endogenous prostaglandin metabolites that are responsible for the orchestrated resolution of inflammation. The anti-inflammatory, cytoprotective and energy metabolism effects of AIM pharmacology have been documented in more than 250 scientific papers and are potentially relevant to a wide range of diseases.

Due to the broad applicability of AIM biology, Reata is actively conducting or initiating phase 2 programs with AIMs in multiple therapeutic areas, including pulmonary arterial hypertension, oncology, Friedreich’s ataxia, mitochondrial myopathies, dermatology, and ophthalmology.

CONTACT:
Reata Pharmaceuticals, Inc.
(972) 865-2219
info@reatapharma.com

Investor Relations:
The Trout Group
Lee M. Stern, CFA
lstern@troutgroup.com
(646) 378-2992