August 18, 2015
By Mark Terry, BioSpace.com Breaking News Staff
Ann Arbor, Mich.-based Esperion Therapeutics, Inc. , announced yesterday that the U.S. Food and Drug Administration (FDA) confirmed LDL-C as an acceptable clinical surrogate endpoint for its trial therapeutic, ETC-1002.
ETC-1002 is designed to lower cholesterol for patients who have not had success using statins, such as Pfizer Inc. ’s Lipitor. The compound is a first-in-class, oral, once-a-day drug that lowers cholesterol by inhibiting ATP citrate lyase (ACL), a key enzyme that is involved in the creation of cholesterol.
After meeting with the FDA last week, it was decided that the drug will not require a cardiovascular outcomes trial (CVOT). Esperion plans to conduct a CVOT before applying for a new drug application (NDA) anyway for broader label indications. The company is on schedule to start a Phase III development trial program by the end of this year.
“After an informative and collegial meeting with the FDA, we are pleased that LDL-C remains an accepted clinical surrogate endpoint for the approval of an LDL-C lowering therapy such as ETC-1002 in patients with HeFH and/or patients with ASCVD,” said Tim Mayleben, president and chief executive officer of Esperion in a statement. “We have a clear regulatory path forward for development and approval of ETC-1002, an oral, once-daily treatment option for these patients that require additional LDL-C lowering.”
The company will face stiff competition. The FDA recently approved Regeneron Pharmaceuticals, Inc. and Sanofi ’s Praluent for cholesterol. Amgen’s Repatha is expected to be approved this month to treat cholesterol. Aegerion Pharmaceuticals, Inc. is also marketing Juxtapid, used to treat homozygous familial hypercholesterolemia (HoFH), but that drug has a price tag of $295,000 per year, compared to Praluent’s $14,600 annual price.
Investors seemed to like the news. had been on an overall rise much of the year, although it showed some volatility. Shares traded for $20.69 on Oct. 16, 2014, rose to $70.06 on Feb. 18, 2015, and to $112.70 on Mar. 23 and $115.30 on May 19. Shares dropped on June 12 to $74.24, rose to $98.91 on July 17, then dropped to $44.61 on Aug. 6. Shares are up today, trading for $78.75.
Analysts at Needham & Company LLC gave Esperion stock a “buy” rating on June 10 with a price target of $130. RBC Capital analysts on April 22 issued an “outperform” and a $150 price target. Zacks gave it a “hold” rating on May 1, which it recently shifted to a “buy.” Chardan Capital analysts on May 22 set a “sell” rating and a $55 price target.
Joel Beatty, an analyst with CitiVelocity, reiterated a “buy” rating, and wrote today, “With conservative market size assumptions of 5.8 million secondary prevention patients on a statin, it would take less than 6 percent market penetration to achieve $1 billion in annual sales, assuming a $3,000 per year price. … We still see a meaningfully large commercial opportunity with the newly planned label.”
In an investor call with analysts yesterday, Mayleben said, “We now have a clear regulatory path forward” and expects to talk to “a number of potential partners” regarding marketing the drug nationally and internationally.
The company is also studying ETC-1002 in a fixed-dose combination with ezetimibe, and has two other compounds in its preclinical pipeline.
