ISA Pharmaceuticals’ ISA101 Studied in Phase II Combination Trial with Checkpoint Inhibitor Nivolumab

December 07, 2015

• Trial in patients with HPV16-positive, incurable solid tumors initiated by MD Anderson Cancer Center in collaboration with ISA and Bristol-Myers Squibb

Leiden, The Netherlands, December 7, 2015 – ISA Pharmaceuticals B.V., a clinical-stage immunotherapy company focusing on rationally designed immunotherapeutics against cancer and persistent viral infections, today announced the start of a Phase II combination trial, which will be conducted at The University of Texas MD Anderson Cancer Center (Houston, TX).

The goal of the open-label Phase II study is to investigate whether a combination of ISA101 with Nivolumab can help to control cancer that has spread in the body. The safety of the study drugs will also be evaluated. Nivolumab, a PD-1 immune checkpoint inhibitor, is marketed by Bristol-Myers Squibb as Opdivo for the treatment of various cancers.
The study will enroll approximately 28 patients with HPV16-positive, incurable solid tumors. Patients will receive three subcutaneous administrations of ISA101 at three week intervals, and Nivolumab intravenously every 2 weeks, beginning on day 8 after receiving the first ISA101 dose.

The primary endpoint of the study is overall response rate (ORR) at 11 weeks, defined as the sum of patients with complete response (CR) and partial response (PR) divided by the number of evaluable subjects at 11 weeks from start of treatment. RECIST 1.1 criteria will be used for assessment. Further details of the study can be found at https://clinicaltrials.gov/ct2/show/NCT02426892. The study is expected to be completed in late 2018.

Funding is being provided in part by MD Anderson’s Moon Shots Program, which was launched in 2012 to dramatically improve survival for patients diagnosed with several types of cancer over the next decade. Additional funding for the trial comes from the Stiefel Oropharyngeal Research Fund in support of MD Anderson’s Oropharynx Program, which treats and conducts research on cancers that occur in the portion of the throat behind the mouth.

“We look forward to exploring the combination of these two promising immunotherapy approaches, which simultaneously elicit the induction of tumor specific immune responses and block immune suppressive mechanisms,” said Erminia Massarelli, MD, PhD, assistant professor of Thoracic/Head and Neck Medical Oncology at MD Anderson and principle investigator of the study.

The trial is open to all HPV16-related cancers regardless of organ of origin.

“Our HPV-16 specific immunotherapeutic has been tested in six clinical trials as a standalone treatment against various cancers and pre-cancerous lesions,” said Ronald Loggers, CEO of ISA. “The trial at MD Anderson is the second combination trial of ISA101. We are already including HPV16-positive cervical cancer patients in Belgium and the Netherlands in a trial in combination with standard chemotherapy. We are very committed to developing the optimum treatment schedule for patients with HPV-positive (or driven) cancers at different stages of the disease.”

About ISA Pharmaceuticals

ISA Pharmaceuticals B.V. is an immunotherapy company developing rationally designed, fully synthetic immunotherapeutics against cancer and persistent viral infections. The company has built a proprietary immunotherapy platform based on the Synthetic Long Peptide (SLP®) concept and AMPLIVANT® technology, which have the potential to generate safe and effective immunologic responses with a known mechanism of action. Synthetic long peptides are broadly applicable to multiple targets and ideally suited for monotherapy, as essential components in combination with conventional cancer treatments, and with novel immunomodulators such as Nivolumab. SLP® immunotherapies are designed to fully harness and direct the body‘s own defenses towards fighting the disease. In addition, ISA has begun to develop personalized SLP® immunotherapies targeting tumor specific, mutation derived neo-antigens. These SLP®’s are based on the analysis of the specific DNA mutations in a tumor sample and designed and synthesized for each individual patient.

ISA´s most advanced clinical-stage immunotherapeutic is ISA101, an SLP® immunotherapy targeting human papillomavirus (HPV)-induced diseases. It is currently in clinical development in advanced and recurrent cervical cancer, incurable HPV16-postive solid tumors and anal intraepithelial neoplasia (AIN). Clinical proof-of-concept has been established in vulvar intraepithelial neoplasia (VIN), a pre-cancerous disease caused by HPV.

The company was founded in 2004 by Aglaia Oncology Fund and is based in Leiden, The Netherlands. For more information, please visit www.isa-pharma.com

SLP® and AMPLIVANT® are registered trademarks in Europe.