Bold Therapeutics Receives FDA Clearance to Add U.S. Sites for Phase 1b / 2a Trial of BOLD-100 in the Treatment of Advanced GI Cancers

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Jul 07, 2021, 08:38 ET

VANCOUVER, BC, July 7, 2021 /PRNewswire/ -- Bold Therapeutics, a clinical-stage biopharmaceutical company, is pleased to announce that the U.S. Food and Drug Administration (FDA) has cleared an Investigational New Drug Application (IND) amendment for BOLD-100-001, allowing for the addition of clinical trial sites in the United States, starting with H. Lee Moffitt Cancer Center & Research Institute in Tampa, Florida and the University of California, Los Angeles in Los Angeles, California.

BOLD-100-001 (clinicaltrials.gov/ct2/show/NCT04421820) is an open-label, multicenter two-stage study designed to evaluate the safety and efficacy of BOLD-100 in combination with FOLFOX (5-fluorouracil, leucovorin, oxaliplatin) chemotherapy in patients with advanced gastric, pancreatic, colorectal and bile duct cancers. BOLD-100-001 is currently enrolling patients at six clinical trial sites in Canada: Cross Cancer Institute in Edmonton, Alberta (PI Jennifer Spratlin); Princess Margaret Cancer Centre in Toronto, Ontario (PI Grainne O'Kane); Ottawa General Hospital in Ottawa, Ontario (PI Rachel Goodwin); Juravinski Cancer Centre in Hamilton, Ontario (PI Elaine McWhirter); and Jewish General Hospital and Royal Victoria Hospital in Montreal, Quebec (PIs Petr Kavan and Jamil Asselah, respectively).

"This FDA clearance is a major milestone for Bold Therapeutics as it allows us to more rapidly enroll patients and further diversify our patient population" stated Jim Pankovich, EVP, Clinical Development. "Our corporate mission is to address significant unmet needs in oncology, and we are optimistic that BOLD-100 can meaningfully improve outcomes in patients with these difficult-to-treat 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 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 to immuno-oncology agents. Bold Therapeutics is a synthetic drug that can be manufactured efficiently at commercial scale, and Bold Therapeutics previously completed a 46-patient Phase 1 monotherapy study of BOLD-100 in patients with advanced solid tumors in which BOLD-100 was well-tolerated with minimal hematologic and neurologic side effects. This strong safety profile and remarkable preclinical synergy in combination with a wide range of existing anti-cancer therapies supported continued clinical development. The FDA previously granted Orphan Drug Designations (ODDs) to BOLD-100 in both pancreatic and gastric cancer – and Bold Therapeutics expects to apply for Breakthrough Therapy Designations (BTDs) in a range of indications as preliminary clinical efficacy data becomes available late this year.

"Despite significant advances in oncology in the past decade, such as the introduction of immuno-oncology agents, cancer drug resistance remains a persistent unmet challenge in the $187B global oncology market. Cancer is increasingly treated with combinations of therapies with complementary mechanisms-of-action rather than individual targeted therapies, against which cancer cells can readily adapt, quickly rendering them ineffective," said E. Russell McAllister, Founder and CEO. "With a unique multimodal pro-apoptotic mechanism-of-action and a clean Phase 1 safety profile, BOLD-100 is positioned not to replace existing effective therapies, but to make them better – and, in some cases, perhaps much better. We chose the name Bold Therapeutics because it is unquestionably bold to go after the most difficult-to-treat indications with the shortest mean survival times first. Yet this is exactly where there is the greatest unmet medical need and the greatest potential to improve outcomes. Preclinically, BOLD-100 has repeatedly generated unprecedented responses in these indications in vivo, such as extending mean survival times in an AsPC-1 gemcitabine-resistant pancreatic cancer model by a staggering 300% -- and we are now in the process of testing to see if BOLD-100 can generate similar outcomes in man."

In recognition of its disruptive potential, Bold Therapeutics was selected as a 'Top Innovator' in the Life Sciences / Healthcare category at the Venture Summit / Virtual Connect Global where Bold Therapeutics will be presenting on July 7.

For more information, please visit the Company's website at www.bold-therapeutics.com

Contact:
E. Russell McAllister, CEO
(604) 262-9899
[email protected]

SOURCE Bold Therapeutics Inc.