Safety update sets up filings for Esperion cholesterol drug

New analysis shows no clinically relevant differences between the bempedoic acid and placebo groups. (Bryan Brandenburg/CC by-SA 3.0)

Esperion has the 52-week safety data it needed for its cholesterol therapy bempedoic acid, clearing the way for regulatory filings next year.

The new data “is very clean and should put to rest some of the primary concerns previously on [the drug’s] safety,” according to analysts at Jefferies, who think the company’s $1 billion market cap undervalues the company.

A few months ago, a preliminary read-out from the placebo-controlled study revealed 13 fatalities in its treatment arm—around three times the number in the control group—spooking investors and leading to a big sell-off in shares. The fall came despite assertions by Esperion’s management that the deaths were unrelated to the drug and came amongst older, sicker patients, with five attributed to lung cancer.

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Esperion now says the latest readout “showed no clinically relevant differences between the bempedoic acid and placebo groups” when it came to adverse events, serious adverse events, discontinuations and fatalities, with the latter seen in 1.1% and 0.8% of patients, respectively.

Jefferies said that numerous experts have “said repeatedly that the imbalances in the first phase 3 study were noise/random and don't think they were a problem.”

This is the final phase 3 trial in Esperion’s registration program for bempedoic acid, which could provide a cheaper, oral alternative to PCSK9 inhibitors from Amgen and Regeneron/Sanofi, that must be delivered by injection, for patients who can’t keep their cholesterol levels under control using statins alone.

Tim Mayleben, Esperion’s CEO, said the biotech is now “one step closer to being able to provide convenient, cost-effective and complementary LDL-C lowering therapies for use by patients who require additional LDL-C lowering.”

In the trial, bempedoic acid reduced LDL-cholesterol by an additional 18% when added to patients on the maximum-tolerated dose of statins, and there were also improvement trends on major cardiovascular events (MACE) that were consistent with the results of Esperion’s first phase 3 trial. A definitive verdict on cardiovascular outcomes will have to wait for the results of the CLEAR Outcomes trial due in 2022.

Jefferies said Esperion is looking cheap for a company that also has wholly owned rights to a drug that offers up to 50% cholesterol reduction when combined with Merck & Co’s Zetia (ezetimibe) in a single-tablet regimen—which Esperion has said is also due for filing in the first quarter of 2019.