CymaBay Therapeutics Reports Positive Results from its Pilot Phase 2 Clinical Study of MBX-8025

CymaBay Therapeutics, Inc. (NASDAQ:CBAY) today announced top line results from its pilot Phase 2 clinical study of MBX-8025 in patients with homozygous familial hypercholesterolemia (HoFH).  The study demonstrated that the range of responses to MBX-8025 was broad, but that MBX-8025 provided a clinically meaningful reduction in low-density lipoprotein cholesterol (LDL-C) for a subset of patients. This is the first study to demonstrate the potential utility of a PPARδ agonist in HoFH.
According to the company news:

This was an open label, dose escalation study of 12 weeks duration conducted at five centers in Europe and Canada. Thirteen patients were enrolled, all of whom had genetically confirmed HoFH, including two subjects who had functionally negative mutations in their LDL receptor (LDL-R) genes. All of the subjects were taking ezetimibe and were on maximum statin therapy. None of the study participants received lomitapide, mipomersen or a PCSK9 inhibitor. Eight patients were undergoing concomitant apheresis on a weekly or biweekly schedule. Despite being on maximal conventional therapy, the average baseline LDL-C was 368 mg/dL. Subjects received once daily treatment with 50 mg of MBX-8025 for 4 weeks, after which the dose was escalated to 100 and 200 mg in successive 4-week periods. The goals of the study were to evaluate the effect on LDL-C as well as a spectrum of other lipid-related parameters, including PCSK9 levels, and to collect safety information.

Harold Van Wart, Chief Executive Officer of CymaBay said:

We are encouraged by the meaningful response in LDL-C reductions observed in a number of patients in the study and plan to evaluate the feasibility of conducting a pilot study of MBX-8025 in combination with a PCSK9 inhibitor.

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