Thinly traded nano cap CymaBay Therapeutics (NASDAQ:CBAY) is down 16% after hours on robust volume in response to its announcement of results in a Phase 2 pilot study assessing Orphan Drug-tagged MBX-8025 in patients with homozygous familial hypercholesterolemia (HoFH), an inherited disorder characterized by abnormally high levels of bad LDL cholesterol (LDL-C) leading to premature cardiovascular disease.
The 12-week, open-label, dose escalation trial enrolled 13 HoFH patients. All were receiving ezetimibe [Merck's (NYSE:MRK) Zetia] and were on maximum statin therapy. None were being treated with lomitapide [Aegion's (NASDAQ:AEGN) Juxtapid], mipomersen [Sanofi's (NYSE:SNY) Kynamro] or a PSCK9 inhibitor. The average baseline LDL-C level was 368 mg/dL (optimal level: less than 100 mg/dL). Subjects received 50 mg of MBX-8025 once daily for four weeks.
Two per-protocol analyses were performed on 12 subjects (one was excluded due to missed apheresis visits). A responder analysis showed three patients experienced a reduction in LDL-C greater than 30%, five patients at least 20% and seven patients at least 15%. The average maximum decrease was 19%. A second analysis, averaging values across all doses and dosing periods, showed an overall average decrease in LDL-C of 10%, with eight subjects showing a mean decrease of 16% and three greater than 20%. Four patients showed a mean increase in LDL-C of 4%.
Mean PCSK9 was elevated at baseline and increased significantly during treatment by an average of 43%, which was unexpected, and creates the need to assess the combination of MBX-8025 and a PCSK9 inhibitor [Amgen's (AMGN +0.1%) Repatha (evolocumab) or Regeneron (NASDAQ:REGN) and Sanofi's Praluent (alirocumab)] in a pilot study.
MBX-8025 is an agonist of peroxisome proliferator-activated receptor delta, a nuclear receptor that regulates genes involved in lipid transport, storage and metabolism in liver and muscle.
Aegion (AEGN -2.4%) has announced a new $20M share repurchase program good for the remainder of the year, using a "strong cash position" to buy back shares "at prices we continue to believe are attractive.”
The new authorization will pick up immediately after an existing $20M program is exhausted.
The company will execute the new program through one or more 10b5-1 plans, allowing for purchases even during times when the company has material nonpublic information, like undisclosed earnings.
Aegion will make purchase on the open market or otherwise.