ANKTIVA® Approved in Macau for BCG-Unresponsive Bladder Cancer – First Asia Authorization

ImmunityBio, Inc. (NASDAQ: IBRX) secured regulatory approval from the Pharmaceutical Administration Bureau (ISAF) of Macau SAR, China, for ANKTIVA® (nogapendekin alfa inbakicept-pmln). The approval is for apply in combination with Bacillus Calmette-Guérin (BCG) in adult patients battling non-muscle invasive bladder cancer (NMIBC) that has grow unresponsive to BCG treatment, specifically those with carcinoma in situ (CIS) with or without papillary tumors. This marks the first regulatory green light for ANKTIVA® in Asia, representing a key step in the company’s global expansion strategy.

Expanding Treatment Options for a Challenging Condition

NMIBC represents the vast majority of newly diagnosed bladder cancer cases. However, when initial treatment with BCG fails – a scenario known as BCG-unresponsive disease – therapeutic options become significantly limited. CIS, a high-grade form of NMIBC, carries a substantial risk of progressing to muscle-invasive bladder cancer, a more aggressive and potentially fatal form of the disease. Currently, radical cystectomy, the surgical removal of the bladder, is often recommended in these cases. But What we have is a major operation with significant implications for a patient’s quality of life, and many individuals are either not medically fit for surgery or prefer to avoid it.

ANKTIVA® offers a potential alternative, aiming to provide durable disease control while preserving bladder function. The approval addresses a critical unmet demand for patients who have exhausted standard treatment options. The therapy’s mechanism of action differs from traditional approaches, focusing on bolstering the body’s own immune response to fight cancer.

A Reliance-Based Pathway to Market

The Macau approval was granted through a “reliance-based review” process. This increasingly common regulatory pathway allows authorities to leverage prior assessments conducted by established agencies like the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The FDA previously approved ANKTIVA® for this indication, and the EMA has also reviewed the therapy. This approach expedites access to innovative treatments by reducing redundant evaluations, while still allowing local regulators to apply their own jurisdiction-specific requirements. It’s a strategy designed to balance speed with thorough oversight.

How ANKTIVA® Works: Activating the Immune System

ANKTIVA® is a first-in-class interleukin-15 (IL-15) receptor agonist. Unlike immune checkpoint inhibitors, which operate by releasing brakes on the immune system, ANKTIVA® actively stimulates the expansion and activation of cytotoxic immune cells. Specifically, it promotes the proliferation of natural killer (NK) cells and CD8-positive cytotoxic T lymphocytes, crucial components of the immune system’s ability to target and destroy cancer cells. It also encourages the development of long-lived memory immune cells, potentially providing sustained anti-tumor activity. When administered alongside BCG, ANKTIVA® is intended to strengthen the immune response within the bladder microenvironment.

Clinical Trial Data Supports the Approval

The approval in Macau is based, in part, on data from the QUILT-3.032 clinical trial. Results published in peer-reviewed journals, including NEJM Evidence and The Journal of Urology, showed a complete response rate of 71 percent among patients with BCG-unresponsive NMIBC with CIS with or without papillary tumors. The median duration of response was 26.6 months. These findings suggest that ANKTIVA® can induce meaningful and lasting remissions in a patient population with limited treatment options.

Broader Implications for ImmunityBio and the Asia-Pacific Region

While Macau has a relatively compact population, the approval is strategically significant for ImmunityBio. It represents the company’s first foothold in the Asia-Pacific region and could pave the way for approvals in larger markets. According to ImmunityBio, the company is pursuing regulatory expansion across 34 countries and territories. The Macau approval demonstrates the viability of leveraging reliance-based regulatory pathways to accelerate access to ANKTIVA® globally.

Dr. Soon-Shiong Highlights a “Paradigm Change”

Dr. Patrick Soon-Shiong, founder and Executive Chairman of ImmunityBio, expressed enthusiasm about the approval, calling it a “paradigm change” in the treatment of the immune system. In a post on X (formerly Twitter), he described the approval as the start of “unstoppable” global expansion for ANKTIVA®.

Looking Ahead: Real-World Impact and Future Regulatory Steps

The authorization in Macau expands the geographic availability of ANKTIVA®, but its ultimate impact will depend on several factors. These include the speed of clinical adoption by physicians, the establishment of favorable reimbursement policies, the company’s ability to scale up manufacturing to meet demand, and the long-term outcomes observed in real-world clinical practice. Ongoing regulatory reviews in other countries will also be crucial. For patients with BCG-unresponsive NMIBC, this approval provides a new therapeutic avenue, offering the potential for bladder preservation and improved quality of life. The coming months and years will reveal the extent to which ANKTIVA® becomes a standard of care in bladder cancer treatment, both in Asia and worldwide.