FDA Grants Orphan Drug Designation to OBI Pharma’s Investigational Gastric Cancer Treatment

TAIPEI, Taiwan, Jan. 21, 2020 /PRNewswire/ — OBI Pharma, Inc., a Taiwan biopharma company (TPEx: 4174), today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for OBI-999 for the treatment of Gastric Cancer. OBI-999 is a first-in-class antibody drug conjugate targeting Globo H, a glycolipid antigen. On December 26, 2019 OBI-999 was granted Orphan Drug Designation for Pancreatic Cancer.

A Phase 1/2 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 Gastric, Pancreatic, Colorectal and Esophageal Cancers (ClinicalTrials.gov Identifier: NCT04084366). The objective of the trial is to verify the safety and preliminary efficacy profile of OBI-999 in these patient populations.

Tillman Pearce, MD, CMO, OBI Pharma noted, “Advanced gastric cancer is an orphan disease where targeted therapies are lacking for the majority of patients. OBI-999 is an antibody-drug conjugate therapeutic targeting the cancer-associated glycolipid antigen, Globo H. OBI-999 incorporates a validated payload, MMAE, with a proprietary linker technology. It has demonstrated high effectiveness in xenograft models of metastatic gastric cancer that overexpress Globo H. Clinical development of this agent will be guided by evaluation of patients based on a validated Globo H immunohistochemistry assay, which will allow selection of patients whose tumors overexpress this tumor antigen for clinical investigation.”

About Gastric Cancer

Gastric cancer (GC), or stomach cancer, is a disease in which malignant cancer cells form in the lining of the stomach. The majority of GC (90-95%) presents as adenocarcinomas arising from the mucosa layer. Some of the potential risk factors associated with GC include Helicobacter pylori infection, high intake of salt, low consumption of fruits and vegetables, obesity, heavy alcohol consumption, chronic atrophic gastritis and cigarette smoking.

GC is a deadly disease and an estimated prevalence in 2019 at 100,189 individuals in the US, with a low 5-year survivability of 31.0%. Current standard treatment options for patients with advanced or recurrent GC are associated with limited efficacy and unfavorable toxicity profile, especially for extended use or maintenance treatment in a patient population which is often already frail and cachectic.

About Orphan Drug Designation (ODD)

The orphan drug designation provides OBI Pharma with potential benefits, including market exclusivity upon regulatory approval is received, exemption of FDA application fees, and tax credits for qualified clinical trials. The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for rare diseases or conditions that affect fewer than 200,000 people in the U.S.

About OBI-999

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. 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 (gastric, pancreatic, lung 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. OBI Pharma owns global rights to OBI-999.

About OBI Pharma

OBI Pharma, Inc., is a Taiwan biopharmaceutical company that was established in 2002. Its mission is to develop and license novel therapeutic agents for unmet medical needs against cancer targets such as Globo Series (including Globo H and SSEA-4), AKR1C3, and other promising targets.

The company’s novel first-in-class immuno-oncology therapeutic portfolio is targeted against Globo H: Adagloxad Simolenin (formerly OBI-822) and OBI 833 (vaccines), OBI-888 (mAb) and OBI-999 (ADC) and SSEA-4: OBI 866 (vaccine); OBI-898 (mAb) and OBI-998 (ADC). The company’s novel first-in-class AKR1C3 targeted therapy is OBI-3424 (small-molecule prodrug) that selectively releases a potent DNA alkylating agent in the presence of the aldo-keto reductase 1c3 (AKR1C3) enzyme. Additional information can be found at www.obipharma.com.

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