Akero Therapeutics, Inc., a cardio-metabolic non-alcoholic steatohepatitis (NASH) company developing pioneering medicines designed to restore metabolic balance and improve the overall health of NASH patients, 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.
About NASH
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 o...
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