Introduction

Reata is committed to developing novel therapeutics for patients with serious and life-threatening diseases. AS, FA, and PAH are diverse, chronic diseases in which mitochondrial dysfunction, inflammation, and oxidative stress are implicated. Mitochondrial dysfunction occurs when cellular energy production is impaired in favor of production of proinflammatory mediators including cytokines and ROS. Chronic inflammation can lead to tissue fibrosis, organ remodeling, or other organ damage.1

Alport Syndrome (AS)

  • AS is the second most common inherited cause of kidney failure and affects as many as 60,000 people in the US.9 90% of men with X-linked AS develop ESRD before age 40.10 Approximately 12% of women with X-linked AS develop ESRD by age 40, and this increases to 40% by age 8011,12
  • It is caused by a genetic disorder in which type IV collagen defects alter the structure and function of the glomerular basement membrane, the kidney’s main filtering apparatus.10 Treatment approaches include annual monitoring, supportive care, diet, lifestyle management, and off-label use of blood pressure-lowering therapies approved for other forms of CKD. There is a significant unmet need for additional therapies13-16

Friedreich’s Ataxia (FA)

  • FA is an inherited, progressively debilitating degenerative neuromuscular disorder for which there are no approved therapies.17 FA affects approximately 6000 individuals in the US and 22,000 globally.18,19  Many individuals with FA succumb to their disorder at an early age.20 Currently, FA management is aimed at alleviating symptoms and maintaining optimum functioning17

Connective Tissue Disease-Associated Pulmonary Arterial Hypertension (CTD-PAH)

  • CTD-PAH is often a late and fatal occurrence in individuals with CTD.21  Between 10% and 15% of patients with scleroderma or lupus erythematosus, for example, have CTD-PAH.22-25  In the US, the 5-year survival for CTD-PAH is approximately 44%, while the survival of PAH from other causes is substantially greater.26

Autosomal Dominant Polycystic Kidney Disease (ADPKD) (coming soon)

References
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  15. Alport Syndrome Foundation. The renal diet: Potassium. http://alportsyndrome.org/blog/the-renal-diet-potassium/. Accessed November 8, 2018.
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  18. Vankan P. Prevalence gradients of Friedreich’s ataxia and R1b haplotype in Europe co-localize, suggesting a common Palaeolithic origin in the Franco-Cantabrian ice age refuge. J Neurochem. 2013;126(suppl 1):11-20.
  19. Polek B, Roach MJ, Andrews WT, Ehling M, Salek S. Burden of Friedreich’s ataxia to the patients and healthcare systems in the United States and Canada. Front Pharmacol. 2013;4:66.
  20. Parkinson MH, Boesch S, Nachbauer W, Mariotti C, Giunti P. Clinical features of Friedreich’s ataxia: Classical and atypical phenotypes. J Neurochem. 2013;126(suppl 1):103-117.
  21. Pulmonary Hypertension Association. Types of pulmonary hypertension. http://phassociation.org/patients/aboutph/types-of-ph/. Accessed November 8, 2018.
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  24. Hsu VM, Chung L, Hummers LK, et al. Development of pulmonary hypertension in a high-risk population with systemic sclerosis in the Pulmonary Hypertension Assessment and Recognition of Outcomes in scleroderma (PHAROS) cohort study. Semin Arthritis Rheum. 2014;44(1):55-62.
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