No Exosome Therapy Yet Approved in the U.S. — Korean Developers Aim to Enter Clinical Trials This Year

KBioHealth Regulatory Science Support Group Helps Overcome GMP and CMC Barriers
Director Kim Jong-won: “Rapid Clinical Trial Approval and Marketing Authorization Are Essential for Successful Commercialization”

[Dailypharm, Reporter Lee Hye-kyung] Attention is turning to whether exosome therapeutics supported by the Regulatory Science Support Group at the Osong Medical Innovation Foundation (KBioHealth) will advance into Phase 1 clinical trials this year.

Exosomes are nanoscale vesicles enclosed by a lipid bilayer and are attracting growing attention as a promising next-generation therapeutic modality.

On the 23rd, Kim Jong-won, Director of the Regulatory Science Support Group, held a briefing with reporters specializing in Korea’s Ministry of Food and Drug Safety at the Chungcheongbuk-do C&V Center in Osong. He presented the organization’s support activities for exosome development to date and discussed future development strategies.

Kim said, “KBioHealth’s goal is to help researchers move their development programs through clinical trials, regulatory approval, commercialization, and ultimately to patients.”

He added, “There are currently no exosome therapeutic products approved by the U.S. FDA, and we are supporting domestic development programs aimed at creating therapies for indications including acute kidney injury and atopic dermatitis.”

Kim also called for a more forward-looking approach from Korea’s regulatory authorities to support the domestic pharmaceutical and biotechnology industry.

He emphasized the need to prevent situations in which Korean developers, unable to obtain timely clinical trial approval domestically, are forced to pursue studies in countries such as the United States or Australia. At the same time, he stressed that regulatory innovation should create room for proactive administration without placing all administrative or ethical responsibility solely on the Ministry of Food and Drug Safety.

Established in 2021, the Regulatory Science Support Group provides companies with CDRMO services designed to support new drug development, including development strategy planning, regulatory support, characterization analysis, manufacturing process optimization, efficacy evaluation, and candidate optimization.

Kim Jong-won, Director of the Regulatory Science Support Group at KBioHealth.

KBioHealth is also working with the Ministry of Food and Drug Safety (MFDS) on workshops covering regulatory approval for advanced biopharmaceuticals and recombinant DNA products, while carrying out eight regulatory support projects newly secured in 2023.

Exosomes are nanoscale vesicles enclosed by a lipid bilayer. They contain a variety of ligands, cell-derived proteins, growth factors, and nucleic acids, and are known to play an important role in intercellular communication.

The biological information carried by exosomes can potentially be used not only for disease diagnosis and biomarker discovery, but also for the development of therapeutics that modulate cellular signaling. Because exosome-based approaches may preserve some of the therapeutic functions of conventional cell therapies while reducing risks such as immune rejection and tumor formation, they have attracted considerable attention. According to PubMed, approximately 46,000 papers related to exosomes were published between 2010 and 2022.

However, significant challenges remain in the development of exosome therapeutics. These include variations in exosome size, heterogeneity among exosome populations, the need to isolate specific exosome subtypes for targeted therapeutic effects, and limitations in analytical characterization and quality control caused by that heterogeneity.

One representative KBioHealth support case involved ILIAS Biologics under a Ministry of Health and Welfare one-stop drug development program.

The Regulatory Science Support Group developed and optimized a stable lyophilized formulation, conducted proteomic and lipidomic analyses, established manufacturing processes, and supported bioanalytical testing and immunogenicity assessment. It also supported GMP manufacturing, quality control, and stability testing of the drug substance.

ILIAS, meanwhile, established an exosome Master Cell Bank (MCB) and Working Cell Bank (WCB), produced nonclinical study materials, developed quality analytical methods, and prepared applications for nonclinical development and a Phase 1 clinical trial.

As a result, the company received Phase 1 clinical trial approval from Australia’s Therapeutic Goods Administration (TGA) in February 2022 and subsequently completed the trial.

Kim said, “There are likely areas of overlap among the various research projects currently underway. By leveraging these common elements, we will work to help one or two companies enter exosome-related clinical trials within this year.”

Despite such progress, however, no exosome therapeutic candidate has yet received clinical trial approval in Korea.

ILIAS moved its development program to Australia and began its Phase 1 trial there in April 2022, completing the study in October last year. Brexogen is also currently conducting a Phase 1 trial in the United States. Meanwhile, ExoStemTech, which submitted a Phase 1 clinical trial application to the MFDS in April 2022, ultimately did not receive approval.

Kim noted that while the FDA and TGA apply somewhat different regulatory standards, “in principle, the FDA can be said to review essentially the same types of data as the MFDS,” calling for a more forward-looking regulatory approach toward clinical development in Korea.

At the same time, he emphasized that regulatory flexibility must be matched by greater effort from industry.

A representative from the Regulatory Science Support Group said, “We sometimes find that companies do not fully understand the regulatory requirements needed during technology development or fail to make sufficient efforts to address them.”

The representative added, “Companies themselves need to understand and internalize the various regulatory requirements involved in progressing a product toward clinical development.”