CymaBay Therapeutics Inc (NASDAQ:CBAY) reported top line data from its pilot Phase II clinical trial of MBX-8025 in enrollments suffering with homozygous familial hypercholesterolemia. The trial showed that MBX-8025 offered a clinically substantial reduction in LDL-C for a subset of patients. It marks the first trial to show the potential efficacy of a PPARδ agonist in homozygous familial hypercholesterolemia. Harold Van Wart, the CEO of CymaBay, said that this trial offers the first validation that MBX-8025 possess potential efficacy for the cure of homozygous familial hypercholesterolemia.

The trial details                                                                                                 

CymaBay reported that the study was a dose escalation trial of 12 weeks performed at five centers in Canada and Europe. They enrolled 13 patients, and all of them suffered from genetically confirmed HoFH. Moreover, two enrollments showed functionally negative mutations in LDL-R genes. These patients were taking ezetimibe and also were receiving maximum statin treatment. None of the trial subjects received lomitapide, PCSK9 or mipomersen inhibitor.

Out of 13, eight subjects were taking concomitant apheresis on a biweekly or weekly schedule. In spite of being on highest conventional treatment, the mean baseline LDL-C came in at 368 mg/dL. Enrollments were given daily treatment of MBX-8025, 50 mg once in a day for four weeks, after which the regimen was changed to 100 and 200 mg in following 4-week periods. The objectives of the trial were to assess the impact on LDL-C and other lipid-related parameters, and to gather safety information.

The expert speaks

Expressing his views on the trial, Dr. Evan Stein stated that despite the availability of new treatments, most patients with homozygous familial hypercholesterolemia remain away from their LDL-C marks and there exists a dire requirement for new treatment approaches. The trial result that MBX-8025 reduces LDL-C, despite the unanticipated increase in PCSK9, proposes that more trials may be needed to further evaluate the potential of MBX-8025 therapy in patients with homozygous familial hypercholesterolemia. The CEO of CymaBay said that they are encouraged by the substantial response in LDL-C decline noted in a number of enrollments in the trial and intend to assess the feasibility of performing a pilot trial of MBX-8025 drug in combination with a ‘PCSK9’ inhibitor.

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