SOUTH SAN FRANCISCO, Calif., Oct. 16, 2020 /PRNewswire/ -- Akero Therapeutics, Inc. (Nasdaq: AKRO), a cardio-metabolic non-alcoholic steatohepatitis (NASH) company developing pioneering medicines designed to restore metabolic balance and improve the overall health of NASH patients, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to efruxifermin (EFX), an investigational FGF21 analog for the treatment of NASH. The designation was granted based on the positive efficacy data recently reported from the company's Phase 2a BALANCED study. Based on available published data, EFX is believed to be the first drug candidate to receive a PRIME designation for treatment of NASH.
"We believe the EMA PRIME designation for efruxifermin provides important validation for the potential of EFX to become a foundational NASH monotherapy," said Andrew Cheng, M.D., Ph.D., president and chief executive officer of Akero. "This designation underscores not only the high level of unmet need for therapeutics to treat this life-threatening disease, but also EFX's potential to address multiple aspects of NASH and its associated comorbidities. We are grateful to the EMA for considering our application and for recognizing the potential benefits EFX could provide to patients, if approved."
In reaching its decision, the EMA's Committee on Human Medicinal Products (CHMP) indicated preliminary 16-week data from Akero's Phase 2a clinical trial, the BALANCED study of EFX in biopsy-confirmed NASH patients, show EFX's potential to meet unmet medical need for NASH therapies by addressing multiple aspects of NASH-related morbidity and mortality. The CHMP also expressed that the totality of data, including histology data as well as biomarker data, together with better glycemic control and amelioration of dyslipidemia, suggest potential benefit not only for treatment of NASH but also the broader context of metabolic syndrome common in NASH patients.
The BALANCED study, which evaluated EFX in treatment of biopsy-confirmed NASH patients for just 16 weeks, showed that 48% of all EFX-treated patients achieved at least a one-stage improvement in fibrosis without worsening of NASH, and 48% of all EFX patients achieved resolution of NASH without worsening of fibrosis. Patients treated with EFX also achieved relative reductions of liver fat ranging from 63-72% across three dose groups, compared to 0% for placebo. The study also showed that EFX improved multiple NASH comorbidities, including better glycemic control and improved lipoprotein profile as well as weight loss.
The PRIME program is designed to enhance regulatory support in the EU for the development of promising investigational medicines that, based on early clinical data, may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options. PRIME aims to provide multiple benefits so that these medicines can reach patients earlier: enhanced interaction and early dialogue with EMA, guidance on the overall development plan and regulatory strategy, and the potential for accelerated assessment of the marketing authorization application. For more information please visit the EMA website at www.ema.europa.eu.
The company plans to initiate a Phase 2b/3 adaptive trial of EFX in biopsy-confirmed NASH patients in the first half of 2021 based on guidance received from the U.S. Food and Drug Administration.
NASH (non-alcoholic steatohepatitis) is a serious form of NAFLD (non-alcoholic fatty liver disease) and is estimated to affect 17 million Americans and impacts more than a third of people with type 2 diabetes worldwide. NASH is closely linked to the obesity and diabetes epidemics seen around the world. NASH is characterized by an excessive accumulation of fat in the liver that causes stress and injury to liver cells, leading to inflammation and fibrosis, which can progress to cirrhosis, liver failure, cancer and eventually death. As a result, NASH has become a leading cause of liver transplants in the US and Europe.
Efruxifermin (EFX), formerly AKR-001, is Akero's lead product candidate for NASH. EFX has been shown to increase insulin sensitivity, improve lipoproteins, reduce liver fat and inflammation, and reverses fibrosis. The breadth of desirable metabolic effects offers potential to address the complex, multi-organ/tissue pathogenesis of NASH, including risk factors linked to cardiovascular disease – the leading cause of death in NASH patients. Engineered to mimic the biological activity profile of native human FGF21, EFX offers convenient once-weekly dosing.
About Akero Therapeutics Akero is a cardio-metabolic NASH company dedicated to reversing the escalating NASH epidemic by developing pioneering medicines designed to restore metabolic balance and improve overall health of NASH patients. The Company's lead product candidate, efruxifermin, has been evaluated in a 16-week Phase 2a clinical trial, the BALANCED study. Akero Therapeutics is headquartered in South San Francisco, CA. For more information, please visit www.akerotx.com.
Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements, including, but not limited to, statements regarding the Company's business plans and objectives, including future plans or expectations for EFX, upcoming milestones, and therapeutic effects of EFX as well as the dosing, safety and tolerability of EFX; expectations regarding the design, implementation, timing, and success of its current and planned clinical trials for EFX; the PRIME designation of EFX, potential benefits resulting from such designation and related implications; and the potential impact of COVID-19 on strategy, future operations, and clinical trials. Any forward-looking statements in this press release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include: risks related to the impact of COVID-19 on the Company's ongoing and future operations, including potential negative impacts on Akero's employees, third-parties, manufacturers, supply chain and production as well as on global economies and financial markets; the success, cost, and timing of the Company's product candidate development activities and planned clinical trials; the Company's ability to execute on its strategy; positive results from a clinical study may not necessarily be predictive of the results of future or ongoing clinical studies; regulatory developments in the United States and foreign countries; the Company's ability to fund operations; as well as those risks and uncertainties set forth more fully under the caption "Risk Factors" in Akero's Annual Report on Form 10-K for the year ended December 31, 2019 and most recently filed Quarterly Report on 10-Q, as filed with the Securities and Exchange Commission (SEC) as well as discussions of potential risks, uncertainties and other important factors in Akero's other filings and reports with the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Akero undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.