Bold Therapeutics, a clinical-stage biopharmaceutical company, announced that the U.S. Food & Drug Administration (FDA) has granted BOLD-100 an Orphan Drug Designation (ODD) in the treatment of gastric (stomach) cancer. BOLD-100 is a first-in-class ruthenium-based small molecule therapeutic that (1) alters the unfolded protein response (UPR) through selective GRP78 inhibition; and (2) induces reactive oxygen species (ROS) which causes DNA damage and cell cycle arrest. Collectively, these effects result in cell death in both sensitive and resistant cancers, giving BOLD-100 the potential to significantly improve outcomes in a wide range of both solid and liquid tumors in combination with other anti-cancer therapies ranging from traditional chemotherapies to targeted therapies.

The Orphan Drug Act provides a wide range of special development incentives for sponsors developing drugs that address rare or 'orphan' diseases, including: protocol assistance; the opportunity for accelerated approval; discounts on registration fees; and eligibility for seven years of post-approval market exclusivity. While gastric cancer is highly prevalent in Asia, it is considered an orphan disease in North America and Europe, with approximately 27,000 new cases diagnosed in the U.S. annually.

"We are pleased that the FDA granted BOLD-100 an Orphan Drug Designation in the treatment of gastric cancer, adding to our existing Orphan Drug Designation in pancreatic cancer. Data submitted in the orphan drug application demonstrated that BOLD-100 is potentially effective against this deadly cancer," stated Jim Pankovich, EVP, Clinical Development at Bold Therapeutics. "Importantly, we continue to gather additional data on BOLD-100 from our ongoing Phase 1b trial in the treatment of advanced gastric, pancreatic, colorectal and bile duct cancers and anticipate applying for one or more Breakthrough Therapy Designations for BOLD-100 as clinical efficacy data becomes available. Breakthrough Therapy Designation is reserved for drugs that treat a serious or life-threatening disease or condition and that demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. To incentive development for these significant unmet medical needs, a Breakthrough Therapy Designation further reduces FDA approval timelines."

"We look forward to continuing to work with the team at Bold Therapeutics to ensure an optimal global regulatory strategy is developed and rolled out across its pipeline," added Paul Cronin, Director, SCENDEA (a leading product development and regulatory consulting practice), who supported Bold Therapeutics in obtaining its ODD.

Bold Therapeutics continues to actively enroll patients in its Phase 1b trial of BOLD-100 in combination with FOLFOX (5-fluorouracil, leucovorin, oxaliplatin) for the treatment of patients with advanced gastrointestinal cancers at six sites in Canada: Cross Cancer Institute (Edmonton, Alberta); Princess Margaret Cancer Centre (Toronto, Ontario); Ottawa General Hospital (Ottawa, Ontario); Juravinski Cancer Centre (Hamilton, Ontario); Jewish General Hospital (Montreal, Quebec); and Royal Victoria Hospital (Montreal, Quebec). This adaptive design trial is expected to transition into a Phase 2 trial later this year, with additional clinical sites to be added in both the U.S. and South Korea.