Pancreatic Cancer Rx Granted Orphan Drug Designation
A Taiwan biopharma company announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for OBI-999 for the treatment of Pancreatic Cancer.
The FDA's Office of Orphan Drug Products grants orphan status to support the development of medicines for rare diseases or conditions that affect fewer than 200,000 people in the USA.
OBI-999 is a novel first-in-class Antibody Drug Conjugate (ADC) with a proprietary linker technology that provides a consistent Drug-to-Antibody ratio (DAR) for cancer treatment that is based on Globo H, an antigen expressed in up to 15 epithelial cancers. OBI-999 uses a Globo H antibody to target cancer cells of high Globo H expression.
By releasing a small molecule chemotherapeutic drug through the specificity of the antibody, it directly deploys cytotoxic therapy at the targeted cancer cells.
A clinical trial of OBI-999 has commenced enrollment at the University of Texas M.D. Anderson Cancer Center, with Dr. Apostolia M. Tsimberidou as the Principal Investigator, in patients with locally advanced or metastatic solid tumors, including Pancreatic, Gastric, Colorectal, and Esophageal Cancers.
Tillman Pearce, M.D., CMO, OBI Pharma noted in a press release, "We are very excited about the potential value that OBI-999 may provide to patients with pancreatic cancer given both the high potency we have observed using OBI-999 in pancreatic cancer xenograft models and because many pancreas cancers highly overexpress Globo H, the glycolipid target of OBI-999, using the validated IHC assay that will be available for selecting patients for the Phase 2 portion of this first-in-human clinical trial."
Pancreatic Cancer is a deadly disease that currently affects 69,839 people in the US and has a survival rate of only 8.5 percent at 5-years.
In addition, treatment options are limited to surgical resection for patients with early stages of the disease and these patients may only have a 5-year survival rate of up to 34.3 percent.
As Pancreatic Cancer is asymptomatic in early stages, a majority of patients are undiagnosed or misdiagnosed until advanced stages of the disease. Surgery is no longer effective at this stage of the disease, leaving a large population with limited treatment options.
Pancreatic Cancer originates in the exocrine or endocrine pancreatic cells and is thought to be caused by poor diet, smoking, and genetic factors.
OBI-999 is currently in a Phase 1/2 clinical trial (ClinicalTrials.gov Identifier: NCT04084366) to test its safety and efficacy as an oncology ADC therapy.
In pre-clinical xenograft animal models in multiple tumor types (pancreatic, lung, gastric, and breast), OBI-999 has demonstrated profound tumor shrinkage at various doses. In pre-clinical single and repeated dose toxicology studies, OBI-999 was well-tolerated and achieved a favorable safety margin which warrants further clinical development.
The FDA orphan drug designation provides OBI Pharma with potential benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees, and tax credits for qualified clinical trials.
OBI Pharma, Inc., is a Taiwan biopharmaceutical company that was established in 2002. Additional information can be found at OBI Pharma.
Pancreatic Cancer news published by Vax Before Cancer.
- OBI Pharma Granted FDA Orphan Drug Designation for the Treatment of Pancreatic Cancer for Its Antibody-Drug Conjugate (ADC)
- OBI Pharma Announces Initiation of a Phase 1/2 Study of Its Antibody-Drug Conjugate (ADC) Targeted Cancer Therapy, OBI-999
- Phase 1/2 Study of OBI-999 in Patients With Advanced Solid Tumors