A new drug, apabetalone in combination with the top standard of care, including high-intensity statins, has been launched by Resverlogix.
The treatment was intended for the secondary prevention of Major Adverse Cardiac Events (MACE) in patients with type 2 diabetes mellitus and recent acute coronary syndrome.
Apabetalone is a first-in-class, small molecule, Bromodomain and Extra Terminal Domain (BET) inhibitor that selectively targets Bromodomain 2 (BD2) at the transcription level.
This investigational drug is believed to regulate dysregulated epigenetics of chronic diseases through selective BD2 inhibition.
The BET proteins contribute to a variety of diseases by increasing the production of dysregulated proteins.
The breakthrough designation given by the US FDA is supported by the data from the phase 3 BET on MACE trial that evaluated the safety and efficacy of apabetalone in the prevention of MACE in high-risk type 2 diabetes patients with coronary heart disease.
The patients were randomised to receive either apabetalone 100mg twice daily or placebo, with a standard of care therapy for 120 weeks.
The primary endpoint was the reduction in narrowly defined MACE, defined as a single composite endpoint of cardiovascular death, nonfatal myocardial infarction or stroke.
The results showed that apabetalone did not meet the study's primary endpoint. However, it did achieve main secondary endpoints showing significant reductions in hospitalizations due to congestive heart failure vs placebo, along with cardiovascular outcomes in two pre-specified subpopulations, including patients with chronic kidney disease.
Significant improvements in cognition were reported cognition when treated with apabetalone in patients with moderate to severe cognitive decline.
Resverlogix is also assessing the effects of apabetalone in patients with pulmonary arterial hypertension (early phase 1), end-stage renal disease treated with hemodialysis (phase 1/2), and Fabry disease (phase 1/2).