OBI-902 received orphan drug designation from the FDA for the treatment of patients with cholangiocarcinoma.
The FDA has granted orphan drug designation to OBI-902 for the treatment of patients with cholangiocarcinoma, according to an announcement from OBI Pharma.1
Cholangiocarcinoma is a rare and aggressive type of cancer found in bile ducts between the liver and small intestine. As a TROP2-targeted antibody-drug conjugate (ADC), OBI-902 utilizes a strong topoisomerase 1 inhibitor payload that effectively kills tumor cells. OBI-902 more specifically, is a novel site-specific glycan-conjugated ADC that uses OBI’s GlycOBI® technology which boosts hydrophilicity and stability. Throughout preclinical studies, OBI-902 has shown promising data with antitumor efficacy, a favorable safety profile, and improved pharmacokinetic characteristics. Notably, there are currently no FDA-approved ADCs for patients with cholangiocarcinoma.
"Based on our preclinical data, OBI-902 has several important advantages over other TROP2 ADCs either approved or in development; including high stability in blood circulation, excellent bystander effect that extends the killing to neighboring cancer cells lacking TROP2 expression, potential ability to overcome drug resistance, and outstanding activity in animal and organoid models of cancer,” Heidi Wang, PhD, chief executive officer of OBI Pharma, stated in a news release.
OBI-902 is being evaluated for the treatment of patients with advanced solid tumors in a phase 1/2 trial (NCT07124117).2 The open-label, dose-escalation, cohort-expansion study is enrolling patients who are at least 18 years old, have histologically or cytologically confirmed metastatic or advanced solid tumors that are uncurable through local therapies, and an ECOG performance status of 0 or 1.
Patients who are less than 3 weeks from prior cytotoxic chemotherapy or radiation therapy, have received less than 5 half-lives or 3 weeks from prior biologic therapies, underwent major surgical procedure (as defined by the investigator) or significant traumatic injury within 28 days prior to first dose, had sensory or motor neuropathy of grade 2 or greater, or had history of solid organ transplant are being excluded from the trial.
During the dose-escalation portion of the study, patients will receive OBI-902 at dose levels ranging from 1.6 mg/kg to 10 mg/kg every 3 weeks. The cohort-expansion phase of the trial will divide patients into arms based on tumor type; patients in the cholangiocarcinoma arm will receive OBI-902 at the putative recommended phase 2 dose (RP2D).
The trial’s primary objectives are safety and tolerability, determining the maximum tolerated dose and RP2D, objective response rate, clinical benefit rate, and disease control rate of OBI-902 in patients with cholangiocarcinoma and other advanced solid tumors.
Secondary objectives include progression-free survival, duration of response, and overall survival in patients with cholangiocarcinoma and other advanced solid tumors.
“Importantly, this marks the first time an ADC that incorporates OBI's proprietary GlycOBI® ADC technology is being evaluated in patients, including those diagnosed with cholangiocarcinoma. We look forward to investigating this potential best-in-class TROP2 ADC in the clinic." Wang added in the news release.1
