8-K: ImmunityBio Faces FDA Roadblock: ANKTIVA sBLA for Bladder Cancer Papillary Indication Receives Refusal to File Letter

ImmunityBio's supplemental biologics license application (sBLA) for ANKTIVA in BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) for papillary disease has received a Refusal to File (RTF) letter from the FDA, despite prior guidance.

Summary

• ImmunityBio received a Refusal to File (RTF) letter from the FDA regarding its sBLA for ANKTIVA plus BCG in BCG-unresponsive NMIBC for the papillary indication.
• The company had previously received unanimous guidance and encouragement from the FDA in January 2025 to submit the sBLA based on data from a single-arm trial.
• The sBLA was submitted in March 2025, but the FDA issued the RTF letter on May 2, 2025.
• ImmunityBio has requested an urgent meeting with the FDA to address the inconsistencies and determine the best path forward.
• The RTF letter does not affect the prior FDA approval of ANKTIVA with BCG for BCG-unresponsive NMIBC with papillary tumors with CIS (Cohort A).
• Data presented at the 2025 American Urological Association (AUA) meeting suggested ANKTIVA+BCG was considered best in class and best in disease by thought leaders.
• The company highlights data showing a 99% disease-specific overall survival rate at 12 months and 96% at 36 months in BCG-unresponsive patients with papillary disease without CIS.
• The company emphasizes the importance of providing this treatment option to patients facing potential bladder removal and disease progression.

Sentiment

Score: 4

Explanation: The sentiment is slightly negative due to the FDA's refusal to file the sBLA, despite previous positive guidance. However, the company is actively working to resolve the issue and has a previously approved indication for the drug.

Positives

• ANKTIVA is already approved by the FDA for BCG-unresponsive NMIBC with CIS with or without papillary tumors.
• Data suggests ANKTIVA+BCG offers a high disease-specific survival rate and bladder preservation in patients with papillary disease.
• The company is actively seeking to resolve the RTF issue with the FDA and explore all available options.

Negatives

• The FDA issued a Refusal to File (RTF) letter for the sBLA, creating uncertainty about the future availability of ANKTIVA for the papillary indication.
• The RTF letter contradicts prior guidance from the FDA, raising concerns about the regulatory process.
• The delay in approval could impact patients who may benefit from this treatment option.

Risks

• There is a risk that the FDA may not reverse its decision on the RTF letter.
• The regulatory review process could be lengthy and uncertain.
• The company may face challenges in obtaining approval for ANKTIVA for the papillary indication.
• Limited resources at the FDA could cause potential delays.

Future Outlook

ImmunityBio plans to engage with the FDA to address the RTF letter and determine the best path forward for the sBLA submission. The company is seeking a Type A meeting and is open to an FDA Advisory Committee meeting.

Management Comments

• Dr. Patrick Soon-Shiong stated that the company is fully determined to work with the FDA as quickly as possible to explore the best path forward.
• Dr. Soon-Shiong questioned the inconsistency of approving ANKTIVA+BCG for patients with Papillary with CIS disease, while refusing to file the sBLA for patients with Papillary without CIS disease.
• Dr. Rachel Sherman recommended that the company seek a rapid meeting with the Agency to resolve this issue and minimize the delay and threat to well-being of patients who could benefit significantly from avoiding a cystectomy.

Industry Context

The treatment of BCG-unresponsive NMIBC is an area of unmet medical need, with limited treatment options available. ANKTIVA represents a potential new treatment option for these patients, but the RTF letter creates uncertainty about its availability.

Comparison to Industry Standards

• The press release mentions that ANKTIVA+BCG was considered best in class and best in disease by thought leaders at the 2025 American Urological Association (AUA) meeting when compared to all the therapies currently approved or in development.
• The data presented of 99% disease specific survival at 12 months and over 82% patients not requiring bladder removal is presented as the most durable data to date in the Papillary setting for bladder preservation.

Stakeholder Impact

• The RTF letter could negatively impact patients with BCG-unresponsive NMIBC who may benefit from ANKTIVA.
• The company's shareholders may be concerned about the potential delay in approval and the impact on the company's financial performance.

Next Steps

• ImmunityBio has requested an urgent meeting with the FDA to resolve the inconsistencies and determine the best path forward.
• The company is open to an FDA Advisory Committee meeting as part of the regulatory process.

Key Dates

Keywords

ANKTIVA, ImmunityBio, NMIBC, Bladder Cancer, FDA, sBLA, Refusal to File, BCG, Papillary Disease, Immunotherapy