The approval came from the U.S. Food and Drug Administration’s Cardiovascular and Renal Products Division. It follows an FDA decision to grant apabetalone Investigational New Drug (IND) status, which will help accelerate its approval process.
“We are thrilled to have received approval to investigate a second major indication along with FDA regulatory advancement for apabetalone at the same time,” Donald McCaffrey, president and CEO of Resverlogix, said in a press release. “This is a very exciting prospect for Resverlogix’s overall advancement.
“This progress is very encouraging and further validates our previously released data that confirmed apabetalone’s ability to differentially affect genes and proteins between advanced chronic kidney disease (CKD) patients and normal subjects,” he added.
Apabetalone, also known as RVX-208, is a selective inhibitor of the BET family of proteins that regulate mechanisms responsible for turning genes on and off. Because of the proteins’ important regulatory role, inhibiting them can help people with CKD, diabetes, heart disease, Alzheimer’s and other conditions.
Resverlogix is assessing apabetalone’s ability to treat high-risk CVD patients with type 2 diabetes in the Phase 3 BETonMACE clinical trial (NCT02586155).
A Phase 1 trial in New Zealand showed that a single 100-mg dose of apabetalone reduced the levels of several inflammation-associated proteins in the blood of people whose kidneys were severely impaired — those with stage 4 CKD. The proteins included the well-known kidney disease markers interleukin-6 and osteopontin.
The Phase 2a study will look at apabetalone’s ability to treat CKD in combination with standard of care therapies.
The trial will be divided in two parts. Part A will examine apabetalone’s ability to treat eight CKD patients who are on dialysis, and its pharmacokinetics, or the way it moves through the body. The Part A results will help researchers select a range of doses to test in Part B, which will involve 30 patients. That stage will compare the effectiveness of a combo therapy that includes apabetalone with the effectiveness of a placebo.
Researchers want to see if apabetalone, in combination with standard of care, can lower biomarkers of kidney disease better than standard of care alone. The trial will also look at the safety of apabetalone.
“Apabetalone and BET inhibition represent a truly novel approach to potentially impacting the extreme high risk of cardiovascular disease (CVD) events in this important patient population,” said Kamyar Kalantar-Zadeh, chairman of Resverlogix’s Renal Clinical Advisory Board.
“We are excited to test apabetalone and the new science of epigenetics and BET inhibition on advanced CKD patients,” he added. “These patients are in great need of novel approaches to reduce CVD risk.”