The primary endpoint for the study was change in the mFARS score relative to placebo after 48 weeks of treatment. Omaveloxolone met this primary endpoint with a statistical significance. Patients treated with omaveloxolone experienced a mean improvement of -1.55 points from baseline; while patients treated with placebo experienced a mean worsening of +0.85 points from baseline.  The observed placebo-corrected improvements were increasing over the course of treatment with the largest improvement observed after 48 weeks of treatment.   

Omaveloxolone treatment also improved several secondary endpoints included in the study. 

Safety and Tolerability of Omaveloxolone

The study demonstrated that omaveloxolone was well tolerated.  Four (8%) omaveloxolone patients and two (4%) placebo patients discontinued study drug due to an adverse event (AE).  The reported AEs were mild to moderate with the most common including: headache, nausea, increased aminotransferases, fatigue and abdominal pain. The increases in the aminotransferases are a pharmacological effect of omaveloxolone, which increases production of aminotransferases in vitro, and the firm believes that they are related to the restoration of mitochondrial function. 

Indeed, in the trial, the aminotransferase increases were associated with improvements (reductions) in total bilirubin and were not associated with liver injury. The overall rate of serious adverse events (SAEs) was low, with three patients in each group reporting SAEs while receiving study drug. Two additional omaveloxolone-treated patients reported SAEs approximately two weeks after receiving their final dose.

Comments from the Experts

Ronald Bartek, President of the Friedreich’s Ataxia Research Alliance (FARA), said, “The results of MOXIe represent a truly historic moment for the patients, families, and caregivers that comprise the Friedreich’s ataxia community. Based on the results reported about omaveloxolone, we are hopeful that our community will finally have its first approved therapy that can slow this relentlessly progressive disease…”   

Warren Huff, President and Chief Executive Officer of Reata, said, “Patients living with Friedreich’s ataxia experience a devastating and progressive loss of neurological function. The MOXIe trial with omaveloxolone is the first study to demonstrate a significant improvement in neurological function in patients with this disease. We believe that the MOXIe findings announced today bring us closer to our goal of providing an urgently needed therapy to patients with Friedreich’s Ataxia …”

Full MOXIe study results will be presented at a future medical meeting.

Read more details about Part 2 of MOXIe Phase 2 trial in the press releases at the Reata Therapeutics  website at: http://www.reatapharma.com/

Observation

Improving the symptoms and slowing the progression of FA represent historical news. Omaveloxolone will become the first approved treatment for FA to reach the clinic, probably worldwide. FA affects around one in 50,000 people worldwide. Its major neurological symptoms include muscle weakness and ataxia – loss of balance and coordination. The condition mostly affects the spinal cord and the peripheral nerves that connect the spinal cord to the body’s muscles and sensory organs. Also affected is the cerebellum function whose task is coordinating body movements. On the heart, FA effects a range from mild abnormalities to life-threatening problems in the cardiac muscle.  

Omaveloxolone possesses antioxidative and anti-inflammatory actions. It improves mitochondrial bioenergetics. It is currently in clinical trials for multiple neurological conditions, including  FA, mitochondrial myopathies, immune-oncology and the prevention of corneal endothelial cell loss following cataract surgery.

Omaveloxolone’s mechanism of action is a combination of activation of the antioxidative transcription factor Nrf2 and inhibition of the pro-inflammatory transcription factor NF-kB. Nrf2 regulates multiple genes that produce antioxidative potential and the production of cellular energy (i.e., adenosine triphosphate (ATP)) within the mitochondria. Unlike other known and used antioxidants, such as vitamin E, or Coenzyme Q10 which provide a specific and finite antioxidative potential, omaveloxolone, through Nrf2, broadly activates intracellular and mitochondrial antioxidative pathways, in addition to pathways known to directly increase mitochondrial biogenesis.    

Reata Pharmaceuticals applies its deep scientists understanding of its cellular response to injury toward discovering and developing novel therapeutics. The firm’s Nrf2 activators, bardoxolone methyl and omaveloxolone, take advantage of new insights into how genes involved in cellular metabolism and mitochondrial function play a primary role in switching the cellular inflammatory response on and off. 

Reata focuses on molecular pathways that regulate cellular metabolism, inflammation and the response to cellular stress in serious and life-threatening diseases that have limited or no approved therapies.

Prohost Observations

We believe that Reata Pharmaceuticals has the technologies and tools that promise to bring treatments to many diseases that have yet to find them. The stock has rallied several times and is currently trading at $165.23 with a market cap of $4.7 billion. The firms’ abstracts have been appreciated by investors but the emerging positive results make this firm’s pipeline interesting to investors.  We are assessing this firm’s capabilities and the timing of its future accomplishments and will post the outcome of our studies and evaluations on the Prohost website as soon as we accomplish the task.

Reata is worth following up on and buying into its stock at the right time.

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