EXCLUSIVE: Esperion Therapeutics, Inc. CEO Says FDA Lift Means ETC-1002 Will Be in Phase III Trials By End of 2015

February 3, 2015
By Riley McDermid, BioSpace.com Breaking News Sr. Editor

The chief executive officer of cholesterol lowering pharmaceutical firm Esperion Therapeutics, Inc. told BioSpace Tuesday that the company is “enormously pleased” by yesterday’s decision by the U.S. Food and Drug Administration (FDA) to lift its nearly five-year PPAR partial clinical hold on Esperion’s drug ETC-1002—a move that could see the company conducting Phase III trials as soon as the fourth quarter of 2015, he said.

News of the FDA’s action pushed shares of Esperion up more than 10 percent in after-hours trading Monday, as investors and Wall Street watchers who have been sitting on the sidelines readied themselves for the company’s new pipeline to hit a vast and lucrative market as early as next year.

As of midday Tuesday, Esperion’s stock had hit an all-time high for the year. That support has Esperion readying itself to conduct longer clinical trials on its pipeline of oral, low-density lipoprotein cholesterol (LDL-cholesterol) lowering therapies for the treatment of hypercholesterolemia and other cardio-metabolic risk markers.

“We are enormously pleased with the FDA’s positive response because we are now permitted to conduct clinical studies with 1002 of longer than six months in duration, including the initiation of the Phase III long-term safety study when we’re ready to do so,” CEO Tim Mayleben told BioSpace in an interview. “We are planning to hold our end of Phase II meeting with FDA in mid-2015 and initiate our Phase III studies by the fourth quarter of 2015.”

The FDA originally placed a peroxisome proliferator-activated receptor (PPAR) partial clinical hold on ETC-1002 in 2009, but its action yesterday has now given Esperion a fast-tracked timeline, said Mayleben.

“There is an immediate impact,” he told BioSpace. “The removal of the partial clinical hold allows us to proceed with planning for our long term safety study as part of our Phase III program.”

Competition in the crowded cholesterol drug space is always on the radar of companies attempting new therapies, though Mayleben seemed sanguine Tuesday about the prospect of a bruising fight for market share in upcoming quarters.

“We don’t anticipate an immediate effect on competition within our market niche, however, note that the LDL-cholesterol lowering therapeutic area is highly competitive and is very large--revenue estimates are as high as $50 billion by 2020” he told BioSpace.

“We are targeting ETC-1002 for hypercholesterolemic patients with the highest unmet medical need, statin intolerant patients, which we believe represents a $6 to $8 billion target market opportunity,” he said. “ETC-1002 is an oral, once-daily small molecule drug that can provide the LDL-cholesterol lowering of a high dose statin or PCSK9i mono therapy without the potential for muscle-related side effects and with the convenience of a once-daily oral pill.”

Mayleben also lauded the FDA’s professionalism and said Esperion had an open line of communication with the agency throughout the long process of meeting approval and regulatory hurdles.

“The process with FDA was quite good,” said Mayleben. “ETC-1002 was labeled a potential PPAR in late 2009 and placed on a partial clinical hold. We initiated two-year carcinogenicity studies in mid-2012 and completed those studies in mid-2014. We reviewed reports from those studies in December 2014 and submitted our complete response to FDA for the PPAR partial clinical hold in early January.”

That response included two-year carcinogenicity study reports and a summary of almost 600 patients of clinical experience with ETC-1002 emphasizing the absence of any classic PPAR activation signals in humans, as well as results from non-clinical investigations demonstrating that the primary mechanism of action is inhibition of ATP citrate lyase, a “pivotal” enzyme involved in cholesterol biosynthesis.

“Our complete response was received by FDA in early January before the end of January the PPAR partial clinical hold was removed by FDA allowing us to conduct long term clinical trials,” said Mayleben.

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