SHARE

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.

DISCLOSURE: The views and opinions expressed in this article are those of the authors, and do not represent the views of journaltranscript.com. Readers should not consider statements made by the author as formal recommendations and should consult their financial advisor before making any investment decisions. To read our full disclosure, please go to:

https://www.journaltranscript.com/disclaimer

SHARE
Previous articleIn Pursuit Of Revenue, Gold Mining USA Inc (OTCMKTS:GMUI) Turns To Acquisition
Next articleWhat’s Wrong With Insys Therapeutics Inc (NASDAQ:INSY)?
Steve Kanaval: Portfolio Manager/Writer/ Market Analyst Steve began his career in the Trading Pits in Chicago making markets at the Chicago Mercantile Exchange (NYSE:CME) the Chicago Board of Trade and the CBOE in the early 80’s. He ran the Morgan Stanley Derivative Prop Trading for the firm specializing in Index Arbitrage. He continued his career as a Trader/Portfolio Manager for multiple Hedge Funds during the Internet Boom of the 90’s managing large portfolios. Steve is known as an expert in MicroCap Technology Stocks and the emerging Digital Currency markets as a Portfolio Manager for his Family Office. Steve has managed portfolio’s in volatile asset classes for 3 decades as a commodity trader, hedge fund manager and digital currency trader and miner. Steve publishes his views on the asset classes in a public forum and has published more than 10,000 articles simplifying these complex and volatile assets for readers. His work is published on multiple sites including Bloomberg, Equities.com, Hacked.com, CryptoCurrencyNews as a paid contributor. His work includes research, journalism and archived video on important market volatility related to stocks, digital currency and other volatile misunderstood asset classes. He offers a humorous, unique insight and the related back stories and drivers for readers interested in volatility and emerging market assets. Full disclosure Steve is long 25 digital currencies and sits on the board of multiple public companies involved in digital currencies, and owns shares in these companies from time to time.

LEAVE A REPLY