Pioneering Development of Life-Changing Treatments by Leveraging the Untapped Mechanisms of the Lysosome

Ray Takigiku, PhD

Problem Tech Solves


Tech Brief

Cancer continues to inflict devastating consequences worldwide. Initially Bexion was founded to develop innovative treatments for cancer. A Phase I, all-comers solid tumor trial evidenced an extremely compelling toxicity and tolerability profile. The Phase 1 program also demonstrated preliminary single agent evidence of activity across multiple tumors. As BXQ-350 targets ubiquitous solid tumor features, antitumor effects have been documented across a range of solid tumors, including brain tumors. BXQ-350’s broad anti-tumor activity reflects its involvement in multiple pathways potentially representing universal tumor physiologies. Further, it shows activity in tumor cell lysosomes, and targets externalized phosphatidylserine (PS), another tumor agonistic onco-marker. In vitro studies show BXQ-350 catalyzes various cellular processes, including apoptosis, necrosis and other cellular death pathways consistent with activity at the lysosome. Preclinical and clinical data are supporting identification of a mechanism-based biomarker for BXQ-350 activity. Clinical and non-clinical data provide evidence of activity in multiple CNS diseases, including benefit against peripheral neuropathy, another large and important unmet therapeutic need. Bexion is currently in a Phase 1/2 clinical trial for a rare pediatric brain tumor (Diffuse Intrinsic Pontine Glioma-DIPG) among newly diagnosed patients and plans to initiate two additional adult Phase 1/2 programs in 2021-2. Newly diagnosed stage 4 colorectal cancer patients will participate in a double blinded Phase 2 study with one cohort receiving SOC + BXQ-350 and the other cohort receiving SOC + placebo. Both oncology and peripheral neuropathy endpoints will be included. A 3rd phase 1/2 study will be conducted among newly diagnosed GBM patients along with SOC.

Tech Differentiators

The challenge in finding a cure for cancer is due to the complexity of the disease and multiple oncogenic changes leading to dysfunctional cancer cells. Current therapies include combining therapeutics to address this, but often lead to resistance. BXQ-350 is unique as it renormalizes the function of the lysosome, key to properly disposing of abnormally reproducing cancer cells. BXQ-350 also renormalizes sphingolipid metabolism, that is critical for cellular function and inter- and intra-cellular signaling mechanisms, leading to cancer cell deaths. In addition to impacting cancer cells’ proliferation and viability, BXQ-350 stimulates the immune system favoring an antitumoral tumor micro-environment. BXQ-350 has been shown to be very well-tolerated in heavily treated adult and pediatric cancer patients: no MTD observed in the 98 patients enrolled in the two phase 1 studies. This excellent safety profile supports exploring BXQ-350 in combination studies with existing therapies without concerns of side effects. There were promising signs of clinical activity in the single agent phase 1 safety studies in terminally-ill patients: 2 patients had a partial response and stayed on study for 2 years, 8 patients had a progression free survival longer than 6 months, and 2 patients are still on study after 5 years. Preclinical studies have demonstrated that BXQ-350 was synergistic with therapeutic agents including cytotoxic agents, immune checkpoint inhibitors and radiation. Bexion will be initiating clinical studies in colorectal and glioblastoma cancer patients, in combination with standard of care, to demonstrate the safety and clinical benefit of combining BXQ-350 with current cancer therapies.


Bexion will be collaborating with physicians at the Mayo Clinic for the Phase 1/2 colorectal trial and MD Anderson for the Phase 1/2 GBM trial.

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