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Hanmi Developing New Biologics for Orphan Drug Market

2021.09.07

Global Phase II clinical trial for NASH treatment
Overseas clinical studies on new biologics developed using LAPscovery

A global Phase II clinical trial has been launched by Merck Sharp & Dohme (MSD) for the non-alcoholic steatohepatitis (NASH) drug candidate licensed-out by Hanmi Pharm. The Korean pharmaceutical powerhouse is also developing a new drug for NASH on its own, in addition to new drugs for congenital hyperinsulinemia and short bowel syndrome, by conducting a Phase II clinical trial. With the development of orphan drugs that are involved in glucagon secretion, the company plans to expand into the global biologics market based on its platform technology, LAPscovery.

◆MSD begins Phase II clinical trial for NASH drug candidate licensed-out by Hanmi Pharm



According to Clinical Trials, a clinical trial registration site operated by the US National Institutes of Health (NIH), MSD has provided an update on its Phase II clinical trial on efinopegdutide. The study is aimed at examining the safety and efficacy of the drug candidate in 130 adults diagnosed with nonalcoholic fatty liver disease (NAFLD) by administering the drug once a week for twenty-four weeks.

The multinational pharmaceutical company registered the clinical trial protocol in late June and began recruiting subjects at a clinical trial agency in Texas. It now has a total of nine participating institutions, including medical centers in California, Florida, North Carolina, and even Israel. The clinical trial protocol was approved by the Ministry of Food and Drug Safety (MFDS) of Korea in late July, and the study is set to begin at five institutions in Korea: Severance Hospital, Samsung Medical Center, Korea University Guro Hospital, Inha University Hospital, and Soonchunhyang University Bucheon Hospital.

Subjects will be administered either the investigational drug (efinopegdutide) or Ozempic (semaglutide), an antidiabetic drug from Novo Nordisk, and the efficacy will be assessed based on the liver fat content (LFC), weight, and cholesterol level, in addition to checking for adverse events.

Efinopegdutide is a GLP-1-based dual agonist developed by applying Hanmi Pharm’s LAPscovery platform technology. Its potential for application to a wide range of metabolic diseases has been recognized, as it simultaneously activates the GLP-1 receptor, which facilitates insulin secretion and suppresses appetite, and glucagon, which boosts metabolism.

Efinopegdutide was developed with the indications of Ozempic in mind. Janssen initially obtained the right to develop efinopegdutide for diabetes and obesity treatment for the global market back in November 2015, guaranteeing a contract price of up to USD 915 million. However, the rights to the drug candidate were returned by the multinational pharmaceutical company, which explained that a clinical study revealed its effectiveness in blood glucose regulation was inadequate. The development of efinopegdutide came to an abrupt halt until it was picked up by MSD, which discovered its potential as a therapeutic agent for NASH, in August 2020.

◆ Hanmi conducting clinical studies on two new drugs for NASH

NAFLD, an indication of efinopegdutide, refers to liver conditions where neutral fats build up in the liver with little or no alcohol or drug consumption.

While most NAFLD patients show signs of improvement through weight loss, it can progress into NASH and more serious liver conditions, such as liver cirrhosis and cancer. Although its prevalence has surged in recent years, there has yet to be an officially approved therapeutic agent.

Hanmi Pharm transferred the rights to develop efinopegdutide for NASH treatment for the global market (excl. Korea) in August last year under an USD 870 million (approx. KRW 1 trillion) contract. The upfront fee, which needs not be returned, was USD 10 million. Once the drug is commercialized, royalty will be additionally paid to Hanmi Pharm according to the sales revenue.

This is great news for Hanmi, which found a new partner in just a year after being returned the rights from Janssen, especially since the new partner immediately kicked off its clinical trials.

EvaluatePharma, a pharmaceutical market survey agency, forecasted that the global NASH treatment market will grow to KRW 30 trillion by 2030. While the development process will not be easy, once a therapeutic agent is successfully commercialized, the developer will be able to take a large chunk of the market.

Hanmi Pharm is also targeting the NASH treatment market with new pipelines developed based on LAPscovery. The GLP-1-based triple agonist, HM15211, is currently being evaluated through a Phase II clinical trial in the United States. The drug candidate activates three receptors (GLP-1, glucagon, and GIP), so it is anticipated to show effectiveness against inflammation and fibrosis.

◆ Four new biologics developed based on LAPscovery designated as orphan drugs

Hanmi Pharm is conducting research and development on new biologics centering on drug candidates that are known to be involved in glucagon secretion. The company is also endeavoring to expand its market by focusing not only on metabolic diseases and cancers but also rare diseases. It is targeting the global pharmaceutical market with four new drugs designated as orphan drugs.

The GLP-1-based triple agonist, HM15211, in particular, will be examined for its potential to be developed for various rare diseases, such as idiopathic pulmonary fibrosis, primary biliary cholangitis, and primary sclerosing cholangitis, in addition to NASH.



May, based on which it received an orphan drug designation from the US Food and Drug Administration (FDA) for treatment of idiopathic pulmonary fibrosis. In March last year, it was designated as an orphan drug for primary biliary cholangitis and primary sclerosing cholangitis, meaning that it has gained recognition for its potential as effectiveness treatment for the three orphan diseases.

The US FDA runs the Orphan Drug Designation program, as part of the efforts to encourage the development of rare and intractable diseases and life-threatening conditions. Orphan drug developers are offered tax deductions, application fee exemption, and, for products that are first to be approved for a specific orphan disease, a seven-year period of market exclusivity from the date of FDA marketing approval. Hanmi Pharm, for instance, was granted Fast Track by the FDA for NASH treatment approval in July 2020.

Hanmi Pharm has three other new drugs that were granted orphan drug designations from the FDA, aside from HM15211. They are efpegsomatropin, which is being developed for treatment of growth hormone deficiency, LAPS glucagon analog (HM15136) for congenital hyperinsulinemia, and LAPS GLP-2 analog (HM15912) for short bowel syndrome treatment. All three of these are new biologics developed based on Hanmi Pharm’s LAPscovery platform, and Phase II clinical trials are being conducted in the United States, Europe, and other regions to be released on the global market.

Source: Daily Pharm