- Esperion Therapeutics Inc. on Tuesday reported another positive trial result to add to the data set for its cholesterol-lowering drug candidate bempedoic acid.
- Topline results from a Phase 2 study showed adding bempedoic acid to background therapy of PCSK9 inhibitor reduced LDL cholesterol by 27% compared to baseline, versus an increase of 3% for patients who received placebo plus treatment with a PCSK9 inhibitor.
- Shares in Esperion initially surged on the news, rising more than 8% during the day before settling back to close up just 1% Tuesday.
While PCSK9 inhibitors drugs are highly effective, uptake has been much slower than expected as payers have thrown up hurdles to the expensive medicines. Low-cost statins and other generic cholesterol medicines appear to remain more cost-effective for most patients.
Esperion, however, believes it can find a market niche between the pricey PCSK9 blockers and generic treatments with a more moderately priced drug that still delivers decent LDL lowering.
Bempedoic acid is an oral, once-daily ATP citrate lyase (ACL) inhibitor which reduces cholesterol biosynthesis, thereby lowering so-called 'bad' cholesterol or LDL-C. A recent study showed the drug reduced LDL-C by 23% from baseline, whereas those on placebo had a 5% increase in LDL-C levels.
In this more recent study, Esperion added bempedoic acid to PCSK9 inhibitors, aiming to showcase the drug's complementary ability.
"The results of this Phase 2 study show that bempedoic acid can also be safely and effectively used in patients who require additional LDL-C lowering despite taking an injectable PCSK9 inhibitor," said Esperion CEO Tim Mayleben in a March 27 statement.
Esperion expects to file bempedoic acid for U.S. approval both as monotherapy and in combination with ezetimibe by the first quarter of 2019, with a submission to the European Medicines Agency to follow shortly thereafter.
Regulatory submission remains pending chronic safety data, due in mid 2018, which Jefferies analyst Michael Yee describes as "a significant derisking event for the company and key catalyst for stock appreciation" if it proves positive.
"We believe the upcoming Phase III long-term safety trial (Study 1) will be positive in May and could serve as a key catalyst to push the stock towards our upside scenario of $100+," Yee added.