Causal inference
modeling at scale
Drug discovery, repurposing
& validation
Causal modeling is a proven method to ensure success in clinical trials. Over 40,000 citations demonstrate the validity of and demand for this methodology.
Clinical Validation
Real time analysis predicts COVID-19 drug trial outcomes with perfect accuracy
- Realtime prospective analysis: During the pandemic, we predicted the approval of each new Drug for severe illness due to COVID19 with perfect accuracy.
- The pandemic allowed for a short turnaround between start of trial and clinical approval or failure by the FDA.
- For each new trial that was started, we build causal models and predicted approval.
- Our predictions were flawless and even allowed for the discovery of a new treatment, CDK6 inhibition, which we successfully tested in-vitro.
| Disease | Drug Target Gene | Mechanism | biotx.ai Prediction | Clinical Trial Result |
|---|---|---|---|---|
| Severe COVID-19 | Interleukin 1 (IL1) | Repurposing potential of Anakinra in severe COVID-19 | success | Phase III trial; approved |
| Severe COVID-19 | Interleukin 6 receptor (IL6R) | Repurposing potential of Tocilizumab in severe COVID-19 | success | Phase III trial; approved |
| Severe COVID-19 | Glucocorticoid Receptor (NR3C1) | Repurposing potential of Dexamethasone in severe COVID-19 | success | Phase III trial; approved |
| Severe COVID-19 | Januskinase 1/2 (JAK1/2) | Repurposing potential of Baricitinib in severe COVID-19 | success | Phase III trial; approved |
| Severe COVID-19 | Cycline dependent kinase 6 (CDK6) | Repurposing potential of Palbociclib in severe COVID-19 | success | Clinical case report; in vitro confirmation |
| Severe COVID-19 | Tubulin (TBB4B) | Repurposing potential of Colchicine and Sabizabulin in severe COVID-19 | failure | Failed Phase II/III |
| Severe COVID-19 | Interferon beta 1 (IFN1B) | Repurposing potential of Interferon beta in severe COVID-19 | failure | Failed Phase II/III |
| Severe COVID-19 | Bruton’s tyrosine kinase (BTK) | Repurposing potential of Acalabrutinib in severe COVID-19 | failure | Failed Phase II/III |
Successful backtesting in a wide range of diseases
Our other predictions are doing just as well. In 16 instances we identified the correct drug target which ended up passing clinical trials.
Further backtesting highlights the validity of causal modeling across disease areas.
| Disease | Drug Target Gene | Mechanism | biotx.ai Prediction | Clinical Outcome |
|---|---|---|---|---|
| Cardiovascular and thrombotic disease | Coagulation factor X | Repurposing potential of factor Xa inhibitors in other cardiovascular disease subtypes | success | success, Phase III clinical trial |
| Cardioembolic stroke | Coagulation factor XI | Protective effects of factor XI inhibition in cardioembolic stroke | success | success, Phase III trial evidence in venous thromboembolism |
| Non-alcoholic steatohepatitis | Fibroblast growth factor 21 (FGF21) | Favourable effects of circulating FGF21 on cardiometabolic biomarkers | success | success, Phase II clinical trial |
| Heart failure | Glucagon-like peptide 1 (GLP1) | Protective effects of GLP1 agonism in heart failure | success | success, Phase III trial underway (NCT01800968) |
| Cardiometabolic disease | Glucose-dependent insulinotropic polypeptide (GIP) | Favourable effects of GIP agonism on bodyweight, lipid traits, coronary artery disease and inflammation | success | success, phase III clinical trials |
| Cardiovascular disease | Interleukin 6 receptor (IL6R) | Beneficial effects of IL6R inhibition in severe COVID-19, increased risk of infectious, allergic and autoimmune disease, identify biomarkers to measure efficacy, repurposing potential in atherosclerotic disease | success | success, Phase II and III clinical trial evidence |
| Cardiovascular disease | Proproteinconvertase subtilisin/kexin type 9 (PCSK9) | Protective effects of PCSK9 inhibition | success | success, phase III clinical evidence |
| Coronary heart disease | Niemann-Pick C1-like 1 (NPC1L1) | Protective effects of NPC1L1 inhibition | success | success, phase III clinical evidence |
| Cardiovascular disease | Cholesteryl ester transfer protein (CETP) | Protective effects of CETP inhibition on cardiovascular disease | success | success, phase III clinical evidence |
biotx.ai enables causal modeling at scale
We enable drug discovery and repurposing via large-scale screening
- Discover all diseases that can be treated by a given drug
- Discover all drugs that can be used to treat any given disease
- Explore all disease mechanisms and pathways
Our platform and biobank built over 5 years enables scaling
Customer Validation
Successful use cases by our biotech customers
Immungentics, a German biotech focused on Alzheimerʼs disease, were able to not only find causal biomarkers crucial for their study design, but in addition, multiple used multiple causal models from biotx.aiʼs analyses to better understand the exact mode of action of their drug thiethylperazine via the ABCC1 transporter.
Alfa Intes, a family-owned Italian pharma, through biotx.ai discovered new indications for their drug Indomethacin, and is in the process of picking indication for a further deal with biotx.ai.
Eternygen, a German biotech, found via our platform that one of the potential indications for their compound will not work and has shifted focus onto another indication instead; next deal has been signed.
Clinical Validation
Real time analysis predicts COVID-19 drug trial outcomes with perfect accuracy
- Realtime prospective analysis: During the pandemic, we predicted the approval of each new Drug for severe illness due to COVID19 with perfect accuracy.
- The pandemic allowed for a short turnaround between start of trial and clinical approval or failure by the FDA.
- For each new trial that was started, we build causal models and predicted approval.
- Our predictions were flawless and even allowed for the discovery of a new treatment, CDK6 inhibition, which we successfully tested in-vitro.
| Disease | Drug Target Gene | Mechanism | biotx.ai Prediction | Clinical Trial Result |
|---|---|---|---|---|
| Severe COVID-19 | Interleukin 1 (IL1) | Repurposing potential of Anakinra in severe COVID-19 | success | Phase III trial; approved |
| Severe COVID-19 | Interleukin 6 receptor (IL6R) | Repurposing potential of Tocilizumab in severe COVID-19 | success | Phase III trial; approved |
| Severe COVID-19 | Glucocorticoid Receptor (NR3C1) | Repurposing potential of Dexamethasone in severe COVID-19 | success | Phase III trial; approved |
| Severe COVID-19 | Januskinase 1/2 (JAK1/2) | Repurposing potential of Baricitinib in severe COVID-19 | success | Phase III trial; approved |
| Severe COVID-19 | Cycline dependent kinase 6 (CDK6) | Repurposing potential of Palbociclib in severe COVID-19 | success | Clinical case report; in vitro confirmation |
| Severe COVID-19 | Tubulin (TBB4B) | Repurposing potential of Colchicine and Sabizabulin in severe COVID-19 | failure | Failed Phase II/III |
| Severe COVID-19 | Interferon beta 1 (IFN1B) | Repurposing potential of Interferon beta in severe COVID-19 | failure | Failed Phase II/III |
| Severe COVID-19 | Bruton’s tyrosine kinase (BTK) | Repurposing potential of Acalabrutinib in severe COVID-19 | failure | Failed Phase II/III |
Successful backtesting in a wide range of diseases
Our other predictions are doing just as well. In 16 instances we identified the correct drug target which ended up passing clinical trials.
Further backtesting highlights the validity of causal modeling across disease areas.
| Disease | Drug Target Gene | Mechanism | biotx.ai Prediction | Clinical Outcome |
|---|---|---|---|---|
| Cardiovascular and thrombotic disease | Coagulation factor X | Repurposing potential of factor Xa inhibitors in other cardiovascular disease subtypes | success | success, Phase III clinical trial |
| Cardioembolic stroke | Coagulation factor XI | Protective effects of factor XI inhibition in cardioembolic stroke | success | success, Phase III trial evidence in venous thromboembolism |
| Non-alcoholic steatohepatitis | Fibroblast growth factor 21 (FGF21) | Favourable effects of circulating FGF21 on cardiometabolic biomarkers | success | success, Phase II clinical trial |
| Heart failure | Glucagon-like peptide 1 (GLP1) | Protective effects of GLP1 agonism in heart failure | success | success, Phase III trial underway (NCT01800968) |
| Cardiometabolic disease | Glucose-dependent insulinotropic polypeptide (GIP) | Favourable effects of GIP agonism on bodyweight, lipid traits, coronary artery disease and inflammation | success | success, phase III clinical trials |
| Cardiovascular disease | Interleukin 6 receptor (IL6R) | Beneficial effects of IL6R inhibition in severe COVID-19, increased risk of infectious, allergic and autoimmune disease, identify biomarkers to measure efficacy, repurposing potential in atherosclerotic disease | success | success, Phase II and III clinical trial evidence |
| Cardiovascular disease | Proproteinconvertase subtilisin/kexin type 9 (PCSK9) | Protective effects of PCSK9 inhibition | success | success, phase III clinical evidence |
| Coronary heart disease | Niemann-Pick C1-like 1 (NPC1L1) | Protective effects of NPC1L1 inhibition | success | success, phase III clinical evidence |
| Cardiovascular disease | Cholesteryl ester transfer protein (CETP) | Protective effects of CETP inhibition on cardiovascular disease | success | success, phase III clinical evidence |
Pipeline
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