ANN ARBOR—Esperion Therapeutics Inc. (NASDAQ:ESPR) has provided a development update for its lead drug candidate, bempedoic acid, and financial results for the first quarter ended March 31.
“The potential of bempedoic acid is being broadly realized as evidenced by publication and presentation of Phase 3 clinical study results in the New England Journal of Medicine, the American College of Cardiology 2019 Scientific Sessions and other industry leading platforms,” Tim Mayleben, Esperion president and CEO, said in a press release. “This high-profile external validation of the potential of bempedoic acid to meet the LDL-C lowering needs of the millions of patients with high LDL-C despite the use of currently accessible therapies confirms the beliefs of our lipid management team. We will continue to work closely with regulatory authorities to bring our LDL-C lowering therapies to physicians, payers and their patients.”
Company officials noted Esperion’s new license and collaboration agreement with Daiichi Sankyo Europe (DSE) to commercialize bempedoic acid and the bempedoic acid / ezetimibe combination tablet in the European Economic Area and Switzerland. Payments to Esperion under the agreement include $150 million upfront upon execution of the LCA, $150 million upon first commercial sale in the territory (as defined by the LCA), up to $600 million in additional regulatory and commercial milestones payments as well as 15 to 25 percent tiered royalties on net territory sales.
In February, Esperion submitted two New Drug Applications for bempedoic acid and the bempedoic acid / ezetimibe combination tablet to the U.S. Food and Drug Administration, and completed the formal validation process with the European Medicines Agency (EMA) of Esperion’s two Marketing Authorization Applications (MAAs), and officially started the review procedure for both bempedoic acid and the bempedoic acid / ezetimibe combination tablet.
In March, the company published new studies in the New England Journal of Medicine and the Journal of the American Heart Association, and presented a study at the American College of Cardiology Scientific Sessions.
In May, the company announced FDA acceptance for review of the NDAs for bempedoic acid and the bempedoic acid / ezetimibe combination tablet, with a target action Prescription Drug User Fee Act (PDUFA) date of Feb. 21, 2020 and Feb, 26, 2020, respectively. The company also released results from the initial clinical study of the 100 mg sustained release formulation of bempedoic acid that demonstrated consistent (29 percent) LDL-C lowering as well as favorable safety parameters. These results provide initial proof-of-concept for the sustained release formulation of bempedoic acid to increase efficacy, extend the patent life of the bempedoic acid franchise into 2038 while utilizing a 505(b)(2) regulatory pathway to approval, and reduce manufacturing costs.
Esperion expects full-year 2019 net cash used in operations to be $25 to $35 million. Company officials said current cash resources, coupled with expected milestone payments under the European commercial collaboration agreement, as well as bempedoic acid and the bempedoic acid / ezetimibe combination tablet commercial sales, are sufficient to fund operations until operating cash flow is positive.
As of March 31, cash, cash equivalents, restricted cash and investment securities available-for-sale totaled $229.7 million compared with $136.3 million at December 31, 2018.
Revenue was $145.4 million for the first quarter of 2019, compared to zero for the comparable period in 2018. Revenue was attributable to the initial recognition of the upfront payment from the DSE collaboration agreement.
Research and development expenses were $46.3 million for the first quarter of 2019, compared to $40.9 million for the comparable period in 2018. The increase in research and development expenses was primarily related to the clinical development costs for bempedoic acid and the bempedoic acid / ezetimibe combination tablet.
General and administrative expenses were $12.2 million for the first quarter of 2019, compared to $6 million for the comparable period in 2018. The increase in general and administrative expenses was primarily attributable to costs to support public company operations, including costs to support pre-commercialization activities, further increases in our headcount and stock-based compensation expense, and other costs to support our growth.
Esperion had net income of $87.4 million or $3.07 a share for the first quarter of 2019, compared to a loss of $46.1 million or $1.73 a share for the comparable period in 2018.
Bempedoic acid is a new class of non-statin cholesterol-reducing drug that avoids some of the side effects of statin treatment, including muscle pain and weakness. It has been tested on more than 2,600 patients in scientific studies.
The effect of bempedoic acid on cardiovascular morbidity and mortality has not yet been determined. Esperion initiated a global cardiovascular outcomes trial to assess the effects of bempedoic acid on the occurrence of major cardiovascular events in patients with, or at high risk for, cardiovascular disease, who are only able to tolerate less than the lowest approved daily starting dose of a statin and considered “statin averse.” The CVOT — known as CLEAR Outcomes — is an event-driven, global, randomized, double-blind, placebo-controlled study expected to enroll approximately 12,600 patients with hypercholesterolemia and high CVD risk at over 1,000 sites in approximately 30 countries.
More at www.esperion.com.