On March 1, 2022 Bold Therapeutics, a clinical-stage biopharmaceutical company, reported that they have successfully completed the Phase 1b (dose-escalation) portion of its seamless adaptive oncology trial of BOLD-100 in combination with FOLFOX in the treatment of advanced gastrointestinal cancers (colorectal, pancreatic, gastric and bile duct) (Press release, Bold Therapeutics, MAR 1, 2022, View Source [SID1234609309]).

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Bold Therapeutics Successfully Completes Phase 1b Trial and Advances into Global Phase 2 Trial of BOLD-100 in the Treatment of Advanced GI Cancers
Bold Therapeutics Successfully Completes Phase 1b Trial and Advances into Global Phase 2 Trial of BOLD-100 in the Treatment of Advanced GI Cancers
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 solid and liquid cancers and in combination with a wide range of existing anti-cancer therapies. The FDA previously granted BOLD-100 Orphan Drug Designations (ODDs) in both Gastric and Pancreatic cancers, and Bold Therapeutics anticipates leveraging clinical data from the Phase 1b portion of its trial for one or more Breakthrough Therapy Designations (BTDs) in 2022.

The Phase 1b data – which Bold Therapeutics anticipates presenting at an upcoming cancer conference – indicate that (1) BOLD-100 can be safely combined with FOLFOX chemotherapy at a dose of 625 mg / m2 (the highest dose level tested), with no new Grade 3 or 4 treatment-emergent adverse events; and (2) patients can safely remain on treatment for an extended number of treatment cycles. Based on this strong safety and tolerability profile, the Study Steering Committee unanimously endorsed proceeding immediately into the Phase 2 (dose-expansion) portion of the study.

"Completing our Phase 1b trial with a strong safety profile for BOLD-100 is a significant achievement for Bold Therapeutics – and while it is too early to say anything definitive, preliminary efficacy data is undoubtedly encouraging," said Jim Pankovich, EVP, Clinical Development. "Despite substantial headwinds from the ongoing COVID-19 pandemic, we were nevertheless able to successfully enroll and treat patients at our six clinical sites in Canada, and I wish to recognize the patients for their contribution to this study as well as the persistent and resilient efforts of our investigators."

The Phase 2 (dose-expansion) portion of the seamless adaptive trial of BOLD-100 will enroll 80 additional patients at 13 investigational sites worldwide: 6 sites in Canada; 2 sites in the U.S.; and 5 sites in South Korea. Interim and complete Phase 2 data is expected by year-end 2022 and late 2023, respectively.

"As BOLD-100 advances into Phase 2, Bold Therapeutics crosses another significant value inflection point," stated Glenn Walthall, Chairman of the Board of Bold Therapeutics. "As Bold Therapeutics’ largest institutional investor, we are encouraged with the results that we’ve seen thus far and optimistic that BOLD-100 may significantly improve outcomes in these difficult-to-treat cancers that are often refractory to conventional treatment options. Consistent with preclinical observations, a number of patients in the study who had previously failed on FOLFOX alone suddenly responded when BOLD-100 was added to the treatment regimen – a result that can only be described as remarkable."

Bold Therapeutics executed a regional option agreement with an undisclosed biopharmaceutical company in South Korea in 2020 and is actively seeking development partners in other territories. Bold Therapeutics is also seeking investors for a data-driven institutional Series B round to be closed later in 2022, likely concurrent with interim Phase 2 results.

"I am exceptionally proud of the agile and industrious Bold Therapeutics team without whom this success would not be possible," added E. Russell McAllister, CEO of Bold Therapeutics. "Through innovative programs like NRC-IRAP, the Canadian government has provided Bold Therapeutics with substantial support in advancing our scientific understanding of BOLD-100 that not only allowed us to advance in the treatment of gastrointestinal cancer indications, but also opened up promising areas for future development. As a result, they share this win with us. The strong results from this Phase 1b trial and the overall accumulation of data on BOLD-100 continue to excite support for the development of this innovative therapeutic for patients with a wide range of advanced cancers."