FDA Approves Anike / Penpulimab-kcqx for Non-Keratinizing Nasopharyngeal Carcinoma: Key Insights for Oncologists

FDA approves Penpulimab-kcqx / Anike

The FDA approved Anike (Penpulimab-kcqx) on April 23, 2025, for non-keratinizing nasopharyngeal carcinoma (NPC, WHO type 2/3). This anti-PD-1 IgG1 monoclonal antibody offers a new treatment option for oncologists managing NPC, a cancer prevalent in East and Southeast Asia with incidence rates of 15-50 cases per 100,000. This post explores Anike (Penpulimab-kcqx)’s approval, clinical trial data from Studies AK105-304 and AK105-202, safety profile, dosing, and its role in NPC treatment, as covered in Oncology Tube’s recent video.

Anike (Penpulimab-kcqx) Approval and Indications for NPC

Anike (Penpulimab-kcqx) is approved for two indications in non-keratinizing NPC:

• First-line treatment of recurrent or metastatic NPC, combined with cisplatin (AUC 80-100 mg/m²) or carboplatin (AUC 5) and gemcitabine (1 g/m²).
• Single-agent therapy for metastatic NPC post-platinum chemotherapy and one prior therapy.

This approval builds on Anike (Penpulimab-kcqx)’s prior FDA breakthrough and fast-track designations for third-line metastatic NPC, highlighting its clinical potential.

How Anike (Penpulimab-kcqx) Works: Mechanism of Action

Anike (Penpulimab-kcqx), an engineered IgG1 antibody, blocks PD-1 to enhance T-cell anti-tumor activity. It lacks Fcγ receptor binding to reduce antibody-dependent cellular cytotoxicity (ADCC) and eliminates complement-mediated cytotoxicity, minimizing off-target immune effects. This design positions Anike (Penpulimab-kcqx) as a targeted immunotherapy for NPC.

Clinical Trial Results: AK105-304 and AK105-202

Anike (Penpulimab-kcqx)’s approval is backed by two trials:

1. Study AK105-304 (NCT04974398)
   This phase 3, randomized, double-blind trial enrolled 291 patients with recurrent or metastatic NPC, previously untreated with systemic chemotherapy for metastatic disease. Patients received either Anike (Penpulimab-kcqx) (200 mg IV every 3 weeks) with cisplatin or carboplatin plus gemcitabine, followed by Anike (Penpulimab-kcqx) maintenance, or placebo with the same chemotherapy regimen.
   • Progression-Free Survival (PFS): Median PFS was 9.6 months (Anike (Penpulimab-kcqx) arm) vs. 7.0 months (placebo arm), with a hazard ratio (HR) of 0.45 (95% CI: 0.33, 0.62; p<0.0001).
   • 12-Month PFS Rates: 31% (Anike (Penpulimab-kcqx)) vs. 11% (placebo).
   • Objective Response Rate (ORR): 78% (Anike (Penpulimab-kcqx)) vs. 62% (placebo; p=0.002).
   • Duration of Response (DOR): Median DOR in the Anike (Penpulimab-kcqx) arm was 8.4 months.
   • Overall Survival (OS): OS data were immature, with 70% of pre-specified deaths reported, showing no detrimental trend for Anike (Penpulimab-kcqx).
2. Study AK105-202 (NCT03866967)
   This phase 2, open-label trial in China enrolled 125 patients with unresectable or metastatic NPC post-platinum therapy and at least one prior treatment. Patients received Anike (Penpulimab-kcqx) (200 mg IV every 2 weeks) for up to 24 months.
   • ORR: 28% (95% CI: 20, 37) per RECIST v1.1.
   • DOR: Median DOR was not reached (95% CI: 9.2 months to not estimable).

Safety Profile of Anike (Penpulimab-kcqx) in NPC Treatment

Adverse events (AEs) in ≥20% of patients on Anike (Penpulimab-kcqx) with chemotherapy included:

• Nausea (62%)
• Vomiting (45%)
• Hypothyroidism (38%)
• Constipation (29%)
• Decreased appetite (27%)
• Weight decrease (24%)
• Cough (22%)
• COVID-19 infection (21%)
• Fatigue, rash, and pyrexia (each 20%)

Immune-mediated AEs were pneumonitis (5%), colitis (3%), hepatitis (2%), endocrinopathies (4%), nephritis with renal dysfunction (1%), and skin reactions (6%), requiring vigilant monitoring. Fatal AEs occurred in 1% of patients, with one case each of pneumonitis, septic shock, colitis, and hepatitis.

Dosing Guidelines for Anike (Penpulimab-kcqx)

• Combination Therapy: 200 mg IV every 3 weeks with chemotherapy, followed by Anike (Penpulimab-kcqx) maintenance.
• Single-agent Therapy: 200 mg IV every 2 weeks.
  Treatment continues until disease progression, unacceptable toxicity, or a maximum of 24 months.

Anike (Penpulimab-kcqx)’s Role in NPC Management and Future Research

Anike (Penpulimab-kcqx) offers a PFS benefit (HR 0.45) and a manageable safety profile for NPC, addressing a critical need in high-prevalence regions like East and Southeast Asia. Ongoing trials are exploring its use in hepatocellular carcinoma (HCC) and non-small cell lung cancer (NSCLC), potentially broadening its oncology applications.

Oncology Tube Video Highlights

Oncology Tube’s video provides a concise overview of Anike (Penpulimab-kcqx)’s approval:

• Approval and Mechanism (00:01-00:31): Details on Anike (Penpulimab-kcqx)’s FDA approval and anti-PD-1 mechanism.
• Clinical Trials (00:45-02:21): Results from AK105-304 and AK105-202, including PFS, ORR, and DOR.
• Safety and Dosing (02:37-03:52): AEs, immune-mediated reactions, and dosing schedules.
• Epidemiology and Impact (03:54-04:24): NPC prevalence, regulatory milestones, and clinical significance.
• Future Trials (04:40-04:48): Research in HCC and NSCLC, plus resources.

The video includes visuals like Kaplan-Meier curves, trial schematics, and a clinician holding an IV bag, illustrating Anike (Penpulimab-kcqx)’s application in clinical settings.

Additional Resources

• FDA Approval Notice: FDA Announcement
• Clinical Trial Data: NCT04974398 (AK105-304), NCT03866967 (AK105-202)
• Subscribe to #OncologyTube: Subscribe

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SOURCE MATERIAL: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-penpulimab-kcqx-non-keratinizing-nasopharyngeal-carcinoma

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