First-in-human clinical trial of proprietary oral gel to treat radiation-induced oral mucositis in cancer patients

Summary Oral mucositis, a severe oral ulceration, is a common toxic effect of radio- or chemoradio-therapy (RT or CRT) and a limiting factor to using the optimal dose of radiation for effective cancer treatment. Intensity modulated RT (IMRT) and stereotactic Body RT (SBRT) spare more normal tissues and lessen chronic side effects, but not oral mucosa in head and neck cancer patients. Targeted therapies and immunotherapies make oral mucositis more prevalent than before. To date, Palifermin, a protein derived from keratinocyte growth factor, is the only targeted therapy approved by the Food and Drug Administration (FDA) for preventing oral mucositis in patients with hematopoietic malignancy followed by bone marrow transplant (4% of the at-risk population), but it has no effect on existing mucositis. Allander Biotechnologies has developed a proprietary biologic that shows prophylactic and therapeutic effects on radiation-induced oral mucositis in mice and dogs upon topical application to oral mucosa. We have 1) locked the process of the cGMP (certified Good Manufacturing Practice) platform for manufacturing and release criteria; 2) established efficacy dose range and defined pharmacodynamics (PD) based on mechanisms of actions; 3) completed GLP toxicology studies in rodents and canines that show no drug-associated toxicity; 4) developed pharmacokinetics (PK) PK/Toxicokinetic (TK) and immunogenic assays needed for clinical trial. Under this SBIR Direct Phase II application, we will: file our Investigational New Drug (IND) application to conduct the first-in-human clinical trial in oral mucositis cancer patients. The primary goal of this trial is to assess the safety and feasibility of our drug being used as an oral gel rinse to treat oral mucositis. The secondary objective is to define the efficacy dose range and recommend Phase II clinical trial dose and schedule (RP2DS). Our ultimate goal is to initiate Phase II/III trials afterwards and file a New Drug Application (NDA) after completing clinical trials.