Application for PD-1 Inhibitor Penpulimab in Nasopharyngeal Cancer Submitted to FDA


Penpulimab may be a future treatment option for patients with nasopharyngeal cancer in the third line of therapy dependent on success of a recent biologics license application submitted to the FDA.

third-lineA biologics license application (BLA) for the PD-1 inhibitor penpulimab (AK105) for the treatment of patients with metastatic nasopharyngeal cancer in the third line setting has been submitted to the FDA, according to Akeso, Inc. who is co-developing the agent with Sino Biopharmaceutical Limited.1

The BLA is being reviewed under Real-Time Oncology Review (RTOR), which is meant to accelerate the drug approval process. This is the first time a PD-1 inhibitor from China is being considered by this process.

Data from a phase 2 AK105-202 trial (NCT03866967) of penpulimab in patients with metastatic nasopharyngeal cancer who progressed after 2 or more prior lines of therapy were presented at the 36th Society for Immunotherapy of Cancer (SITC) Annual Meeting.2 The results indicated that the agent maintained a favorable safety profile while inducing promising antitumor activity for this patient population.

The multicenter, single-arm, open-label study enrolled patients to receive 200 mg of penpulimab every 2 weeks until disease progression or unacceptable toxicity. The primary end point was objective response rate (ORR) by RECIST 1.1 assessed by independent review committee. Key secondary end points included disease control rate, progression-free survival, and duration of response (DOR). Tumor tissue was archived for assessment of PD-L1 expression, with a tumor proportion score of 50% or greater being considered PD-L1 positive.

With a median follow-up time of 7.9 months (range, 0.9-16.9), the confirmed ORR in 111 patients who progressed after 2 or more lines of therapy, including platinum-containing chemotherapy, was 27.0% (95% CI, 19.0%-36.3%). Patients positive for PD-L1 expression had a higher rate of objective response at 39.5% (95% CI, 25.0%-55.6%) versus 19.7% (95% CI, 10.9%-31.3%) in those whose tumors were negative.

The disease control rate was 49.5% (95% CI, 39.9%-59.2%), which included all patients who had at least 1 ongoing response or stable disease awaiting confirmation. The median DOR was not reached (95% CI, 0.95+ to 11.43+) and the rates of patients with a DOR of at least 6 months was 85.6% (95% CI, 52.5%-96.3%).

Treatment-related adverse effects (TRAEs) of therapy occurred in 79.2% of patients overall, with 14.6% of the population having events of grade 3 or greater in severity. The most common TRAEs were fever (24.5%), hypothyroidism (24.6%), anemia (23.1%), increased alanine aminotransferase (17.0%), and decreased white blood cell count (10.8%). Grade 3 or greater TRAEs were abnormal hepatic function (2.3%) and anemia (2.3%). Discontinuations from TRAEs occurred in 3.1% of patients. Serious AEs occurred in 10.0% of patients.

Penpulimab previously received breakthrough therapy and fast track designations from the FDA for the treatment of metastatic nasopharyngeal cancer in the third line setting. In China, the agent is expected to receive approval for the treatment of relapsed or refractory Hodgkin lymphoma.


1. Akeso’s Penpulimab Monoclonal Antibody Submitted BLA in the United States. News release. Akeso, Inc. May 24, 2021. Accessed May 24, 2021.

2. Chen X, Wang W, Zou Q, et al. A phase II study of the anti-programmed cell death-1 (PD-1) antibody penpulimab in patients with metastatic nasopharyngeal carcinoma (NPC) who had progressed after two or more lines of chemotherapy. J Immunother Cancer. 2020;8(suppl 3):804.

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