The Science Behind AUS_001
AUS_001 has the potential to offer life-changing cancer treatment that targets cancer cells with limited impact on healthy cells. With strong pre-clinical results across 24 cancer types and the ability to reach the brain, AUS_001 has the potential to transform cancer care.
How it works
AUS_001 is a powerful microtubule-destabilizing agent, meaning it disrupts the formation of the structural support system that cells rely on to divide and grow. This mechanism causes cancer cells to stop multiplying and triggers programmed cell death (apoptosis), helping to eliminate the cancer.
What sets AUS_001 apart is its ability to preferentially target cancer cells while leaving healthy cells largely unharmed in pre-clinical models. This is possible due to the reversible nature of its engagement with its target, allowing cancer cells to be exploited without causing permanent damage to normal cells.
In fact, it takes on average 20 times more AUS_001 dosage to harm healthy cells compared to cancer cells!
Beyond its selective action, AUS_001 has demonstrated the ability to cross the blood-brain barrier. This makes it a potential treatment for difficult-to-treat brain cancers like glioblastoma. Its bioavailable formulation also ensures it can effectively reach tumors, organs, and brain tissues.
Results
3D-spheroid cultures
Inhibits growth of primary glioblastoma multiforme patient-derived 3D spheroid models, even if Temozolomide resistant.
In vivo data
Efficacy in seven different in vivo cancer models.
Crossing of the blood-brain barrier.
Accumulation in tumors, organs, and brain tissues, i.e., desirable pharmacokinetics.
Tumor vasculature disruption.
Lack of myelosuppression or other overt toxicities in immuno-competent mouse study (21 days, P.O.).
Non-emetic response in ferrets.
In vitro data
High potency against 24 types of cancer.
Encouraging safety margin.
No carcinogenic effect observed.
HERG analysis established cardiac safety profile.
No apparent effect on fibrin clot or thrombin formation over time as assessed by human pooled plasma spike-in assays.
Reduced concern for peripheral neuropathy.
Pre-clinical data
In vitro data
AUS_001 has demonstrated high potency against 24 different types of cancer, showing efficacy in 273 of 280 cancer cell lines. This includes difficult-to-treat tumors such as glioblastoma and pancreatic cancer, which have been sensitive to AUS_001.
The drug showed a 20x higher threshold for inhibiting the growth of healthy non-neoplastic cells compared to cancer cells, indicating a promising safety profile.
While AUS_001 caused a reversible neurotoxic effect in midbrain and cortical neurons, it showed a significantly lower risk of sustained neurotoxicity than paclitaxel-treated neurons, which experienced lasting damage even after treatment cessation.
AUS_001 demonstrated resistance to mechanisms commonly responsible for drug resistance, providing further confidence in its potential as an effective cancer therapy.
In vivo data
AUS_001 has demonstrated efficacy across seven different in vivo cancer models, including breast cancer, pancreatic cancer, bladder cancer, kidney cancer, lung cancer, melanoma, and glioblastoma.
AUS_001 successfully crosses the blood-brain barrier, effectively targeting hard-to-treat brain tumors.
The drug accumulates in tumors, organs, and brain tissues, showing desirable pharmacokinetics and supporting its use in targeting a wide range of tumor types.
AUS_001 has shown potential in disrupting tumor blood vessels, which is critical for limiting tumor growth and metastasis.
In an immunocompetent mouse study (21 days, oral administration), AUS_001 exhibited no signs of myelosuppression or other severe toxicities.
In a ferret study, no vomiting was observed after treatment, indicating a significant reduction in common chemotherapy-related side effects.
Pre-clinical data
In vivo data
AUS_001 has demonstrated high potency against 24 different types of cancer, showing efficacy in 273 of 280 cancer cell lines. This includes difficult-to-treat tumors such as glioblastoma and pancreatic cancer, which have been sensitive to AUS_001.
The drug showed a 20x higher threshold for inhibiting the growth of healthy non-neoplastic cells compared to cancer cells, indicating a promising safety profile.
While AUS_001 caused a reversible neurotoxic effect in midbrain and cortical neurons, it showed a significantly lower risk of sustained neurotoxicity than paclitaxel-treated neurons, which experienced lasting damage even after treatment cessation.
AUS_001 demonstrated resistance to mechanisms commonly responsible for drug resistance, providing further confidence in its potential as an effective cancer therapy.
In vitro data
AUS_001 has demonstrated efficacy across seven different in vivo cancer models, including breast cancer, pancreatic cancer, bladder cancer, kidney cancer, lung cancer, melanoma, and glioblastoma.
AUS_001 successfully crosses the blood-brain barrier, effectively targeting hard-to-treat brain tumors.
The drug accumulates in tumors, organs, and brain tissues, showing desirable pharmacokinetics and supporting its use in targeting a wide range of tumor types.
AUS_001 has shown potential in disrupting tumor blood vessels, which is critical for limiting tumor growth and metastasis.
In an immunocompetent mouse study (21 days, oral administration), AUS_001 exhibited no signs of myelosuppression or other severe toxicities.
In a ferret study, no vomiting was observed after treatment, indicating a significant reduction in common chemotherapy-related side effects.
Dose Range Findings (DRF)
Dose Range Finding (DRF) studies conducted in mice to determine safe dosing levels for GLP toxicology studies revealed encouraging results. The doses tested were 500 mg/kg, 1000 mg/kg, and 1500 mg/kg.
Results
No unscheduled deaths related to the test article.
No alterations in hematology or serum chemistry outside of the normal biological range.
No statistically significant changes in organ weights.
No significant macroscopic findings in necropsied animals.
Histopathology showed minor or no toxicity, except for mild inflammation in the cecum in the 1000 mg/kg and 1500 mg/kg groups.
Data presented at peer-reviewed meetings
Corporate presentation
Help us offer hope for cancer patients worldwide
The science behind AUS_001 shows incredible potential in both in vitro and in vivo models, and we are one step closer to bringing this life-saving treatment to patients. Yet we can’t do it alone. Your support is invaluable as we continue to develop AUS_001 and move towards human trials.