MEDIPOST Secures FDA Agreement for Single Pivotal Phase 3 Study of CARTISTEM® in the U.S.

A Major Step Forward for CARTISTEM® in the United States

MEDIPOST has announced an important milestone in the development of its stem cell therapy product, CARTISTEM®, for the treatment of knee osteoarthritis. The company has secured agreement from the U.S. Food and Drug Administration (FDA) to proceed with a single pivotal Phase 3 clinical study in the United States.

This development is a significant achievement for MEDIPOST and could help bring CARTISTEM® to patients in both the United States and Canada faster than previously expected.

The FDA's agreement also provides the company with an opportunity to streamline its clinical development strategy while reducing both development timelines and overall costs.

What This Means for CARTISTEM® Development

Clinical development in the United States can often be lengthy and expensive, especially for advanced therapies such as cell-based treatments.

By securing FDA agreement for a single pivotal Phase 3 study, MEDIPOST expects to simplify the development process and move more efficiently toward regulatory approval.

The company believes this approach could lead to:

• Faster completion of clinical development

• Reduced overall development costs

• Earlier Biologics License Application (BLA) submission

• Quicker potential entry into the U.S. market

This milestone strengthens MEDIPOST's position as it works to expand the availability of CARTISTEM® beyond Asia and into major international markets.

Leveraging Existing Clinical Success

One of the key reasons MEDIPOST was able to secure this agreement is its strong body of existing clinical evidence.

The company utilized successful Phase 3 clinical study results from both South Korea and Japan to support discussions with the FDA.

These studies provided important evidence regarding the efficacy and safety of CARTISTEM® in patients with knee osteoarthritis.

In addition to clinical trial data, MEDIPOST will also include real-world evidence collected in South Korea as part of its proposed evidence package.

Real-World Data Strengthens the Case

Real-world evidence is becoming increasingly important in regulatory decision-making because it reflects how therapies perform outside of controlled clinical trial settings.

MEDIPOST has gathered data from approximately 550 patients who received CARTISTEM® treatment at least three years ago in South Korea.

This long-term data will be used as supportive evidence for the anticipated outcomes of the upcoming U.S. Phase 3 study.

The inclusion of both clinical trial results and real-world patient outcomes provides a stronger foundation for future regulatory submissions.

Company Leadership Highlights the Importance of the Milestone

According to Antonio Lee, Global President of MEDIPOST, the agreement provides important advantages that will help the company move forward efficiently.

The company plans to use the time and cost savings generated through this streamlined approach while drawing upon its previous experience conducting successful Phase 3 trials in South Korea and Japan.

MEDIPOST believes this experience will help ensure smooth execution of the U.S. clinical program.

The company also sees this achievement as an important catalyst for its broader commercialization plans and ongoing global partnership discussions.

Supporting U.S. Commercialization Goals

The FDA agreement comes at an important time for MEDIPOST as it continues to expand its global presence.

Following recent clinical success in Japan, the company is actively pursuing strategies to introduce CARTISTEM® into new international markets.

The United States represents one of the largest opportunities for innovative treatments targeting knee osteoarthritis, a condition that affects millions of people worldwide.

An accelerated development pathway could help MEDIPOST position itself more competitively within this market while expanding treatment options for patients.

The Growing Need for New Osteoarthritis Treatments

Knee osteoarthritis is one of the most common joint disorders globally and is a leading cause of pain and disability.

As people age, cartilage within the knee joint gradually breaks down, causing pain, stiffness, reduced mobility, and a lower quality of life.

Current treatment options often focus on symptom management through medications, physical therapy, injections, or surgery.

Regenerative therapies such as CARTISTEM® are attracting increasing attention because they aim to address underlying tissue damage rather than simply managing symptoms.

This has created growing interest in advanced cell therapies that may offer long-term benefits for patients with knee osteoarthritis.

What Is CARTISTEM®?

CARTISTEM® is an allogeneic stem cell therapy developed from human umbilical cord blood-derived mesenchymal stem/stromal cells (hUCB-MSCs).

The therapy is designed to treat knee osteoarthritis by supporting cartilage repair and regeneration within the damaged joint.

Unlike traditional treatments that primarily focus on pain relief, CARTISTEM® aims to improve the structural condition of the knee.

The therapy became the world's first regulatory-approved allogeneic human umbilical cord blood-derived mesenchymal stem cell product when it received approval in South Korea in 2012.

Since then, it has been used extensively in clinical practice.

Strong Commercial Experience in South Korea

CARTISTEM® already has a long commercial track record in South Korea.

Since its launch in 2012, more than 36,000 patients have received treatment with CARTISTEM®.

This extensive real-world experience provides valuable information regarding the therapy's long-term use and performance in routine clinical settings.

The success achieved in South Korea has laid the foundation for MEDIPOST's international expansion efforts.

About MEDIPOST

Founded in 2000, MEDIPOST is a global biotechnology company specializing in stem cell therapies and regenerative medicine.

The company operates across multiple areas of the cell therapy industry, including:

• Discovery research

• Clinical development

• Cord blood banking

• Cell and gene therapy contract development and manufacturing (CDMO)

MEDIPOST has built a strong reputation for innovation in regenerative medicine and remains focused on developing advanced therapies that address significant unmet medical needs.

Looking Ahead

The FDA's agreement to allow a single pivotal Phase 3 study for CARTISTEM® marks an important milestone in MEDIPOST's global growth strategy.

By leveraging successful clinical data from South Korea and Japan, along with long-term real-world evidence, the company has positioned itself to potentially accelerate the development and commercialization of CARTISTEM® in the United States.

As the demand for innovative osteoarthritis treatments continues to grow, this milestone brings MEDIPOST one step closer to expanding access to CARTISTEM® for patients worldwide and strengthening its position as a leader in regenerative medicine.