Glycyx Awarded NIH SBIR Phase 2 Grant to Advance Axelopran and Pembrolizumab Study in Head and Neck Cancer

San Francisco, CA — September 2025 – Glycyx MOR, Inc. announced today that it has been awarded a National Institutes of Health (NIH) Small Business Innovation Research (SBIR) Phase II grant to advance a Phase 2 clinical trial evaluating axelopran in combination with pembrolizumab for patients with recurrent or metastatic head and neck squamous cell carcinoma (R/M HNSCC) who are taking opioids to manage cancer pain.

The open-label, single-arm study will be conducted at University of Pittsburgh Hillman Cancer Center and Emory University. The trial will assess the safety, tolerability, and potential efficacy of axelopran when co-administered with standard-of-care immunotherapy pembrolizumab. The primary endpoint is objective response rate (ORR), with secondary endpoints including overall survival (OS), progression-free survival (PFS), and changes in bowel motility and gut microbiome composition.

Recent findings in HNSCC, including those published in the Journal for ImmunoTherapy of Cancer (JITC), demonstrate that opioid signaling can profoundly suppress antitumor immunity by reducing CD4⁺ and CD8⁺ T-cell infiltration. Axelopran is designed to restore immune competence by reversing opioid-induced immunodeficiency, thereby enhancing the efficacy of immune checkpoint inhibitors such as pembrolizumab without increasing toxicity.

“Pain relief is unavoidable for nearly 70% of patients with advanced head and neck cancer, yet it can significantly reduce the effectiveness of immunotherapy,” said Lorin K. Johnson, Ph.D., Chief Scientific Officer of Glycyx. “This study will test whether axelopran can close that gap and improve outcomes for patients who rely on opioids for pain relief.”

“We are deeply grateful to the NIH and to our collaborators at UPMC and Emory for their partnership in advancing this important work,” said Justin Chickles, Chief Executive Officer of Glycyx. “Our goal is to bring forward a novel therapy that enhances the benefits of immune checkpoint inhibitors for patients who need them most.”

This research is supported by a National Cancer Institute (NCI), NIH SBIR Phase 2 grant.