FDA Gives ISA101b Fast Track Designation for HPV 16+ Oropharyngeal Cancer


The first patients with human papillomavirus 16–positive oropharyngeal cancer have been dosed with ISA101b, which was given a fast track designation by the FDA, as part of a phase 2 study.

ISA101b has received a fast track designation from the FDA for the treatment of patients with recurrent and metastatic human papillomavirus (HPV) 16–positive oropharyngeal cancer, according to a press release from drug developer ISA Pharmaceuticals.1

The fast track designation is intended to facilitate the development of investigational therapies that may help to address unmet medical needs for serious or life threatening diseases. An ongoing randomized, double-blind phase 2 trial is examining cemiplimab (Libtayo) with or without ISA101b and has an estimated enrollment of 194 patients (NCT03669718).

“Recurrent and metastatic HPV16-positive OPC is a form of head and neck cancer with a high unmet medical need. The Fast Track designation for ISA101b underlines the potential benefit of this immunotherapy for patients suffering from this disease,” Leon Hooftman, chief medical officer at ISA Pharmaceuticals, said in a press release.

ISA101b elicits strong and specific immune responses to HPV16 virus proteins and creates a robust T-cell immune response against cancerous cells or tissues. In the ongoing trial, patients in the experimental arm will receive ISA101b 3 times over 3 weeks plus cemiplimab every 3 weeks for up to 24 months, and the control arm will receive a matched placebo plus cemiplimab at the same dose.

The primary outcome measures are overall response rate and safety, with a key secondary end point of duration of response.

To be eligible for the trial, patients need to be 18 years or older, have a diagnosis of histologically confirmed recurrent or metastatic HPV16-positive oropharyngeal cancer with tumors that express PD-L1, and be candidates for anti–PD-L1 therapy. Patients who have experienced disease progression on or after a platinum-based chemotherapy regimen are also eligible. Additionally, an ECOG performance status of 0 or 1 is required, as well as measurable disease by CT or MRI imaging.

Patients with untreated metastatic or unresectable tumors that do not express PD-L1 and aren’t candidates for an anti–PD-L1 therapy are not eligible for enrollment. Additionally, those with known brain metastases or leptomeningeal metastases or a serious or uncontrolled medical disorder are not allowed. Patients with a history of other malignancies 3 years or fewer prior to entry are also excluded, with the exception of basal cell or squamous cell carcinoma of the skin that was treated with local resection only, carcinoma in situ of the cervix, prostate or breast cancer, or low grade non-muscle invasive superficial bladder cancer in situ.

The compound is currently being studied in HPV16-positive cancers in combination with cemiplimab in several phase 2 clinical trials. Similarly, ISA101b is also being examined as a single agent in HPV16-positive cervical cancer, an indication for which the compound has received an orphan drug designation.2


  1. ISA Pharmaceuticals receives fast track designation for lead product ISA101b. News release. ISA Pharmaceuticals. September 14, 2021. Accessed September 14, 2021. https://bit.ly/3Aac1Je
  2. ISA Pharmaceuticals receives US orphan-drug designation for ISA101b in HPV16-positive cervical cancer. News release. ISA Pharmaceuticals. July 1, 2020. Accessed September 14, 2021. https://prn.to/3zlfun7
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