On June 6, 2023 Bold Therapeutics, a clinical-stage biopharmaceutical company developing first-in-class oncology therapeutics, reported positive interim results in advanced gastric and biliary tract cancer at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Bold Therapeutics, JUN 6, 2023, View Source [SID1234632533]).

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Bold Therapeutics’ 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 anticancer therapies ranging from traditional chemotherapies to targeted therapies and immuno-oncology agents.

Bold Therapeutics’ ongoing multinational Phase 1b/2 trial (NCT04421820) evaluates BOLD-100 in combination with FOLFOX (folinic acid / leucovorin, fluorouracil, and oxaliplatin) in patients with advanced gastrointestinal cancers. The trial has enrolled 110 patients with advanced gastrointestinal (biliary tract, colorectal, gastric, and pancreatic) cancers at sites in Canada, the United States, Ireland and South Korea, and the primary endpoints for the trial are progression-free survival (PFS), with overall survival (OS) and overall response rate (ORR) as secondary endpoints. Disease control rate (DCR) was also captured. Data from the full Phase 2 trial, which will include an additional 20 patients with advanced colorectal cancer, should be available in late 2023.

Data from 13 patients with advanced gastric cancer and 22 patients with biliary tract cancer was presented in the Gastrointestinal (Gastroesophageal, Pancreatic and Hepatobiliary) Cancer session as Poster 4098: "BOLD-100-001 (TRIO039): A Phase 1b/2a Study of BOLD-100 in Combination with FOLFOX Chemotherapy in Patients with Pre-Treated Advanced Gastric and Biliary Tract Cancer: Efficacy and Safety Analysis." 13 advanced gastric cancer patients (median 4th line) showed a median PFS of 5.5 months, OS of 15.0 months, ORR of 22%, and DCR of 89%, substantially higher than the most comparable benchmark values in a much earlier (median 2nd line) population of a median PFS of 4.6 months, OS of 7.1 months, ORR of 40%, and DCR of 69%. Mature data in an additional 8 advanced gastric cancer patients will be available in late 2023. 22 advanced biliary tract cancer patients (median 3rd line) showed a median PFS of 5.0 months, OS of 7.3 months, ORR of 6%, and DCR of 83%, substantially higher than the most comparable benchmark values in an earlier (true 2nd line) population from the ABC-06 FOLFOX study with a median PFS of 4.0 months, OS of 6.2 months, ORR of 5.0%, and DCR of 33%. Consistent with prior data, BOLD-100 in combination with FOLFOX proved to be exceptionally well-tolerated, with no new safety signals, and with patients remaining on therapy for up to 25 treatment cycles. Treatment-emergent adverse events (TEAEs) were observed in 33 (94%) of patients, with the most common TEAEs being decreased neutrophil count (43%), nausea (29%), and fatigue (20%) – rates comparable to FOLFOX.

Previously, interim data in metastatic colorectal cancer presented at AACR (Free AACR Whitepaper) 2023 indicated that patients treated with BOLD-100 and FOLFOX showed a median PFS of 4.7 months, OS of 9.8 months, ORR of 13%, and DCR of 87%, substantially higher than standard-of-care data for a similar patient population which showed a median PFS of up to 2.0 months, OS of up to 7.1 months, ORR of up to 1.6%, and DCR of up to 44%.

"Our presentation at ASCO (Free ASCO Whitepaper) marks a critical milestone for BOLD-100, with Bold Therapeutics successfully generating positive proof-of-concept data in three different difficult-to-treat gastrointestinal cancer indications where existing therapies are largely ineffective," stated Jim Pankovich, EVP of Clinical Development. "Importantly for patients, BOLD-100 achieves these improved outcomes safely, even in heavily pre-treated patients."

Bold Therapeutics is preparing to initiate a pivotal Phase 3 trial for BOLD-100 in the treatment of advanced colorectal cancer in 2024 and is currently evaluating potentially synergistic development and commercialization partnerships to support these efforts. Concurrently, Bold Therapeutics is exploring additional development indications for BOLD-100 while also advancing its pipeline of other novel metallotherapeutics.

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