Dive Brief:
- Shares in Reata Pharmaceuticals nearly doubled by value in trading this week, surging higher on positive mid-stage data for the Texas-based biopharma's experimental drug bardoxolone methyl in patients with chronic kidney disease (CKD).
- One-year results from the Phase 2 CARDINAL study of patients with CKD due to Alport syndrome showed a statistically significant improvement in kidney function as measured by estimated glomerular filtration rate. Positive results from the autosomal dominant polycystic kidney disease cohort in another study, called PHOENIX, were also reported.
- Analysts from Jefferies, an investment firm, believe Phase 3 data sufficient for accelerated approval of bardoxolone in Alport syndrome could be available by the second half of 2019.
Dive Insight:
Alport syndrome is a rare, genetic form of chronic kidney disease.
"Today's CARDINAL data demonstrate that one year of bardoxolone treatment can improve kidney function in Alport syndrome patients that have had progressive loss of kidney function while on standard of care," said Warren Huff, Reata's CEO.
"Further, the magnitude of the observed retained eGFR benefit after withdrawal of drug versus the historical rate of eGFR loss suggests that the Phase 3 portion of CARDINAL is conservatively powered with respect to the key secondary endpoint of retained eGFR benefit."
In the CARDINAL study, Reata's drug significantly increased from baseline the estimated glomerular filtration rate at week 52 by an average of 4.1 mL/min/1.73 m2, four weeks after bardoxolone treatment was withdrawn. This indicates a retained benefit to treatment, Reata said.
According to Reata, the Food and Drug Administration has said placebo-corrected retained estimated glomerular filtration rate (eGFR) after one year of treatment with bardoxolone can serve as a route to an accelerated approval. While "on treatment" results show the efficacy of the drug, the retained results indicated whether it has harmed or preserved the kidney following withdrawal of treatment.
"We believe the new data derisked the [Phase 3's] potential for success as well as the ADPKD program," wrote Jefferies Maury Raycroft in a note to investors.
"For safety, the retained benefit demonstrated that bardoxolone methyl is not damaging the kidney [and this] should help win over [key opinion leader] support for the program. We believe the benefit may have long-term implications including potential prevention of [end-stage renal disease]," Maury added.