Reata Pharmaceuticals sold some of its shares, generating sales of approximately $115.9 million to be used to support the clinical development of the company’s pipeline, including omaveloxolone, its lead investigative product for Friedreich’s ataxia, according to a press release.
Friedreich’s ataxia is caused by a genetic defect that leads to reduced production of the mitochondrial protein frataxin. Due to a lack of frataxin, cells are unable to correctly balance iron levels, have low levels of energy because mitochondria — which supply power for cells — are dysfunctional, and cells are more sensitive to the harmful effects of oxidative stress.
These biological effects of frataxin deficiency are responsible for the muscular and neurological symptoms of this disease. Given this, finding ways to prevent oxidative stress and restore mitochondrial function is an attractive therapeutic avenue for these patients for which there are no approved therapies.
Omaveloxolone, formerly known as RTA 408, was designed to specifically target the biological features of Friedreich’s ataxia. This investigative compound will activate a protein called Nrf2, which has been shown to be less active in Friedreich’s ataxia patients. This is an important metabolic protein since it promotes normal mitochondrial function, while it can also improve the levels of antioxidant agents.
Reata is currently evaluating the safety and effectiveness of omaveloxolone in the two-part MOXIe Phase 2 trial (NCT02255435), which is still recruiting participants. The company expects to enroll a total of 172 patients between the ages of 16 and 40 with genetically confirmed Friedreich’s ataxia, and no comorbidities, such as diabetes, heart disease, or infections.
The study is being conducted at several clinical sites in the U.S. — California, Florida, Georgia, Iowa, Ohio, and Pennsylvania — but also in Australia, Austria, and the United Kingdom.
Results recently released from the first part of the trial demonstrated that omaveloxolone seems to be safe and it can efficiently increase the levels of Nrf2 in Friedreich’s ataxia patients. In addition, 12 weeks of treatment with the optimal dose of omaveloxolone led to neurological function improvements in patients without foot deformity.
The second part of the trial will evaluate the optimal dose effectiveness and safety of omaveloxolone compared to a placebo. Long-term open label evaluation of this potential therapy is planned following part two of the MOXIe trial, which will include patients who completed the two parts of the study.
Recently, the U.S. Food and Drug Administration (FDA) granted orphan drug status to omaveloxolone for the treatment of Friedreich’s ataxia.
“Orphan drug designation serves as an important milestone for our company as it recognizes the promise of omaveloxolone as a potential new treatment for FA,” Warren Huff, CEO of Reata, said in a press release. “In light of the recent, encouraging clinical data, we are hopeful that omaveloxolone will be the first therapy approved for patients with FA.”
Part of the funds Reata received from the share offering will also go into the company’s working capital and other general corporate purposes besides its clinical pipeline development.