Apabetalone Fares Well in Early Clinical Trial
The trial met its primary goal of adding apabetalone to standard care to improve pulmonary vascular resistance (PVR), a measure that reflects the internal resistance to blood flow within the pulmonary arteries.
“The study successfully achieved its primary objective of confirming the feasibility of the clinical study design and demonstrated encouraging results of apabetalone treatment in patients with PAH,” Steeve Provencher, MD, said in a press release. Provencher is director of the pulmonary hypertension program at Laval University in Quebec, Canada, lead investigator for the trial, and also its sponsor.
The Phase 1 APPROACH-p trial (NCT03655704) enrolled seven people with PAH, all of whom were given apabetalone (taken by mouth twice per day), in addition to their existing treatments, for 16 weeks. The pilot study is open-label, meaning that both investigators and participants know what is being administered.
Resverlogix reports that the treatment was well-tolerated, with all study participants completing the 16-week trial. The treatment also improved PVR and other heart-related parameters.
Other measures (quality of life, physical function, etc.) also are being measured in APPROACH-p, but those data were not released yet.
Resverlogix is planning a larger double-blind Phase 2 trial called APPROACH-2 that will compare apabetalone against a placebo in order to further test the investigational medicine’s safety and efficacy. Enrollment in APPROACH-2 is expected to start this year, according to Resverlogix.
The company had received funding in 2019 for the Phase 2 trial from the Canadian Institutes for Health Research.
The result of the Phase 1 trial “sets the ground for the planned larger double-blind study,” Provencher said.
Apabetalone is an oral medication that works by blocking the activity of bromodomain and extra-terminal domain (BET) proteins. Specifically, apabetalone acts via an epigenetic mechanism, which means it doesn’t change the sequence of the genetic code itself, but alters how DNA is “read” to affect gene and cellular activity.
According to Resverlogix, the selective effect of apabetalone on BET proteins also may have benefts for patients with COVID-19, diabetes mellitus, chronic kidney disease, end-stage renal disease, and other conditions.