Reata Pharmaceuticals, Inc. RETA announced that the FDA has granted an orphan drug designation to its pipeline candidate, bardoxolone methyl (bardoxolone), for the treatment of autosomal dominant polycystic kidney disease (ADPKD), an inherited form of kidney disease.
Notably, the orphan drug designation is granted to drugs capable of treating rare diseases that affect less than 200,000 people in the United States. The designation also makes the company entitled to certain other benefits including tax credits related to clinical trial expenses and exemption from the FDA user fee. The status also makes bardoxolone eligible for seven years of marketing exclusivity in the United States.
Shares of Reata Pharmaceuticals have soared 56.6% so far this year, outperforming the industry’s increase of 1%.
Last year, the company announced encouraging data from the phase II PHOENIX study on bardoxolone in the ADPKD cohort. Notably, the phase II study is evaluating bardoxolone for four rare forms of chronic kidney disease (CKD). Patients treated with bardoxolone for 12 weeks, experienced a mean increase from baseline in estimated glomerular filtration rate (eGFR) of 9.3 mL/min/1.73 m2, a measure of the kidney function. Moreover, 96% of the subjects showed an improvement in eGFR after a 12-week regimen.
Following positive results from the PHOENIX program, Reata Pharmaceuticals started the phase III FALCON study for treating patients with ADPKD. Last week, the company enrolled the first patient for the evaluation. The primary efficacy endpoint of this study is to see the change from baseline in eGFR as compared to placebo after 48 weeks of treatment followed by a 4-week drug withdrawal period.
ADPKD is a common form of inherited CKD, caused by mutations in PKD1 and PKD2 genes. Despite the standard of care treatment, it often causes end-stage kidney disease and almost 50% patients require dialysis or kidney transplant by the age of 60. Notably, this is the third orphan drug designation for bardoxolone to address kidney ailment distinguished by mitochondrial dysfunction and inflammation and the second designation for the treatment of patients with rare forms of CKD.
On approval, bardoxolone may provide an important new treatment option for ADPKD patients.
Zacks Rank & Stocks to Consider
Reata Pharmaceuticals currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the healthcare sector include Acorda Therapeutics, Inc. ACOR, Repligen Corporation RGEN and Merus N.V. MRUS, all sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Acorda’s loss per share estimates have been narrowed 6.5% for 2019 and 6.9% for 2020 over the past 60 days.
Repligen’s earnings estimates have been revised 12% upward for 2019 and 12% for 2020 over the past 60 days. The stock has surged 43.6% year to date.
Merus’ loss per share estimates have been narrowed 24.3% for 2019 and 29.1% for 2020 over the past 60 days.
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