IND-enabling toxicology studies to reach IND approval for a proprietary biologic to treat oral mucositis in cancer patients

NIH RePORTER · NIH · SB1 · $1,230,285 · view on reporter.nih.gov ↗

Abstract

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. 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. Our biologic possesses multiple functions needed for oral mucositis healing. Under the previous SBIR Phase II funding supporting Allander’s drug development, we have 1) identified the lead drug and formulated it as oral gel; established the manufacture platform and quality control (QC) panels ready to be implemented for quality assurance (QA) for certified Good Manufacture Practice (cGMP); 2) established efficacy dose range in dogs that be scaled to the treatment dose in human oral mucositis patients; 3) defined pharmacodynamics (PD) based on mechanisms of actions; 4) de-risked toxicity and oncogenic potential; 5) developed pharmacokinetics (PK) PK/Toxicokinetic (TK) and immunogenic assays needed for toxicology studies and clinical trial. Under this Commercialization Readiness Program (CRP) application, we will: a) manufacture our drug under Good Engineering Practice (GEP) conditions to supply the Good Lab Practice (GLP) toxicology studies; b) perform in-use dose formulation tests of our drug in toxicology studies and finalize dose-formulation analyses to be used in clinical trial; c) perform systemic GLP toxicology studies in small and large animal species with the safety data to support future maximum clinical dose. The milestones under this funding are to apply for Investigational New Drug (IND) of our drug and receive approval from the FDA to initiate our Phase I clinical trial. Our ultimate goal is to complete the first-in-human Phase I trial and initiate Phase II/III trials afterwards and file a New Drug Application (NDA) after completing clinical trials.

Key facts

NIH application ID
10933015
Project number
5SB1DE024659-05
Recipient
ALLANDER BIOTECHNOLOGIES, INC.
Principal Investigator
Xiao-Jing Wang
Activity code
SB1
Funding institute
NIH
Fiscal year
2024
Award amount
$1,230,285
Award type
5
Project period
2023-09-21 → 2025-08-31