Shares in Esperion Therapeutics rose more than 40 percent after the company reported that a Phase IIb study of ETC-1002 in patients with hypercholesterolaemia met its primary endpoint, showing significantly greater reductions in LDL-cholesterol than Merck & Co.'s Zetia (ezetimibe). CEO Tim M. Mayleben said the data "are transformational for both ETC-1002 and Esperion," adding that the ACL inhibitor could be "a new oral therapeutic option for patients with hypercholesterolaemia, especially those with statin intolerance."
The ETC-1002-008 study randomised 348 patients with hypercholesterolaemia, who were washed out of any lipid-regulating therapies prior to a run-in period of five weeks, to receive ETC-1002 at one of two doses, Zetia alone or a combination of the two drugs for 12 weeks. According to Esperion, a total of 177 patients in the trial had a history of statin intolerance.
Top-line results showed that patients who received ETC-1002 monotherapy had up to 27 percent and 30 percent reductions in LDL-C at doses of 120 milligrams and 180 milligrams, respectively, which were significant compared to Zetia alone. In addition, patients who received ETC-1002 dosed at 120 milligrams in combination with Zetia had LDL-C reductions of up to 43 percent, which Esperion noted were significantly different from those given Merck's drug alone. Further, patients taking ETC-1002 dosed at 180 milligrams in combination with Zetia had reductions in LDL-C of up to 48 percent, which were also significantly different from Zetia monotherapy. The company said that the reductions occurred within the first two weeks of dosing and continued throughout the treatment period.
Esperion added that patients given ETC-1002, either alone or in combination with Zetia, also demonstrated significantly greater reductions than Merck's therapy in high-sensitivity C-reactive protein, a marker of inflammation in coronary disease. Study results showed that ETC-1002 was safe and well tolerated, with discontinuation rates due to adverse events comparable to those seen with Zetia.
Study investigator Christie Ballantyne remarked "these are very encouraging results. It basically shows efficacy, and it shows that [ETC-1002] can be added to [Zetia] to get more efficacy." Ballantyne suggested that while "statins are going to stay first-line drugs. There's lots of people who have a hard time taking a high dose of statin or taking a statin at all."
However, Ballantyne said that if approved, ETC-1002 is likely going to be less expensive than PCSK9 inhibitors, such as Amgen's evolocumab and Sanofi and Regeneron Pharmaceuticals' alirocumab, which are likely to be approved next year. Ballantyne noted that while PCSK9 inhibitors are more effective, the lower cost of ETC-1002 could make it a potential option that physicians could prescribe before putting patients on the former injectable therapies. JMP Securities analyst Jason Butler predicts that ETC-1002 could generate peak annual sales of more than $1 billion.
For related analysis, see ViewPoints: Esperion's cardiovascular bet de-risked; is Big Pharma watching?
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