AI-powered drug discovery: update (IX)
AI-powered drug discovery: update (IX)
*️⃣ Collaboration Pharmaceuticals, ♦️ Auransa, 💠 Healx, 👾 DIOSynVax and ⚛️ GreenStone
“What makes the difference between stars and the others is not their academic IQ, but their emotional IQ. They are better able to motivate themselves.”
By Daniel Goleman, Emotional Intelligence: Why It Can Matter More Than IQ
For the previous newsletters of this series of updates specifically on AI drug discovery companies with assets in preclinical and clinical trials 👉
Index for “AI-powered drug discovery: update part IX” 🧵
Collaboration Pharmaceuticals
Auransa
Healx
DIOSynVax
GreenStone Biosciences
*️⃣ Collaboration Pharmaceuticals Inc
Collaborations Pharmaceuticals (2015, US) is a privately owned company that performs research and development on innovative therapeutics for multiple rare and neglected infectious diseases. Their AI platform to aid in drug discovery and toxicology assessment as well as to identify and translate early preclinical to clinical stage assets consists of: Assay Central, a ML platform used to create and build ML models such as MegaTox, MegaTrans and MegaPredict that can be used to predict molecular properties and bioactivities. MegaSyn is their tool for de novo design of molecules. UV-adVISor is a novel ML approach to predict UV-VIS spectra for molecules. Other applications include: Self-Driving Labs as a key part of the design-make-test-analyze cycle and Rare Disease Registry Development a patient registry to store detailed information about patients with a specific disease or syndrome potentially allowing the collection of ‘real world data’.
So far, their work using ML for bacteria, viruses, parasites and rare diseases has led to identification of molecules with potential in vivo activity that can lead to NIH funding to build their pipeline of drug candidates:
Viruses: Ebola Virus Entry / Glycoprotein, HIV-1 NNRTI, CCR5, Enteroviruses Capsid, COVID-19 PIpro, Lysosome, Kinases (CAMK1, MELK), Hepatitis B Disease Undisclosed, Nipah Virus Disease Undisclosed, Yellow Fever Undisclosed, Chikungunya Undisclosed, Marburgvirus Disease Undisclosed
and H1N1 Neuraminidase. All in Preclinical development (in vivo).
Bacteria/Parasites: Malaria Hemozoin Formation, Chagas Disease Undisclosed, S. aureus Undisclosed, C. neoformans / gattii Undisclosed, Tuberculosis Various, M. abscessus Undisclosed and Neisseria gonorrhoeae Undisclosed. All in Preclinical development (in vitro and in vivo).
Rare Diseases: Batten CLN1 (chaperone) PPT1, Batten CLN1 (ERT) PPT1
and Pitt Hopkins Syndrome Nav1.8.
Batten Disease is a genetic disease caused by an autosomal recessive mutation characterized by progressive intellectual and motor deterioration, seizures, loss of vision, and early death. There is currently no treatment for (CLN1). Accordingly, at Collaboration Pharmaceuticals they are developing a recombinant human enzyme replacement therapy for CLN1. They had their preIND meeting with the FDA in May 2023.
On March 7, 2024, they were awarded a Phase II SBIR Commercialization Readiness Pilot (CRP) Program Technical Assistance and Late-Stage Development grant by the National Institutes of Health / National Institute of Neurological Disorders and Stroke for “Manufacture of an intracerebroventricular Enzyme Replacement Therapy for CLN1 Batten Disease”.
On April 11, 2024, they announced the release of a registry for “Batten Disease CLN1 Registry” at https://cln1registry.collaborationspharma.com,
to help them identify CLN1 families currently living with this ultra-rare disease.
Cancer: Multiple Cancers FLT3, KIT, LRRK2, CLK2, Neuroblastoma Various
and Chordoma Various. All in Preclinical development (in vitro and in vivo).
Various: Alzheimer's Disease AChE, BChE, GSK3B, Psychoplastogens 5HT2A
Countermeasures Various, Stroke CCR4, CCR3 and Obesity 5HT2C. All in Preclinical development (in vitro and in vivo).
Since 2017, CPI has been located on the North Carolina State University Centennial Campus in the incubator space. On May 17, 2024, Collaborations Pharmaceuticals announced the opening of a new office in Raleigh, NC, to house their software development, business and operations teams. As part of the recent expansion, the company’s original three-office space has been revamped for larger-scale BSL-2 biology and chemistry laboratory operations. To date, the company has been funded by over $21M in grants from the NIH, the DOD, and the One North Carolina Small Business Program.
♦️ Auransa Inc
Auransa (2014, Palo Alto, California, United States) has developed an AI-driven platform capable of ingesting complex human disease data to understand the biology diseases. Auransa's proprietary predictive computational platform, SMarTR Engine (ML and advanced analytics), utilizes computational approaches to tackle disease heterogeneity to predict targets and compounds, generating insights from molecular data.
So far, Auransa has successfully generated a broad pipeline of drug candidates addressing cancer, auto-immune, infectious and metabolic diseases:
Lead Program: AU-409 for Liver Cancer, Phase 1
Novel, first-in-class compound for liver cancer, which is the fastest growing cancer in the US and EU, and has a high prevalence in Asia.
On March 29, 2023, Auransa and the University of Southern California (USC) announced a collaboration on a Phase I clinical trial to evaluate AU409, a new kind of treatment for cancers of the liver and solid tumors with liver dominant disease. In preclinical studies, the compound showed anticancer activity against hepatocellular carcinoma (HCC). AU409 has a unique mechanism of action that impacts gene expression
Lead Program: AU-018 for Heart-Safe Chemotherapy, IND enabling
Heart-safe chemotherapy for the $1B anthracycline chemotherapy market, to help improve tolerability and raise the standard of cancer care.
Auransa has partnerships with:
Roche Genetech for discovering novel compounds that can protect patients from GI toxicity while undergoing cancer treatment,
POLARISqb to discover novel treatments for diseases that disproportionately affect women, such as ovarian cancer and triple negative breast cancer,
On August 12, 2022, Auransa and Polaris Quantum Biotech (Polarisqb), a quantum computing-based drug design company, announced exciting progress in a joint project to tackle triple-negative breast cancer. By using Auransa’s SMarTR Engine and human disease data, they have identified a biological pathway, likely to be important for patient outcomes. Within this pathway, the companies predicted two promising protein targets. Using Polarisqb’s Tachyon platform, the companies have designed a virtual chemical space containing billions of molecules for each protein target, and searched it for novel molecular structures.
Polarisqb is the first company in the world to develop a drug discovery engine that utilizes the optimisation power of quantum computing to search unstructured, exponentially large datasets to explore novel chemical space, and create new drugs.
Lee's Pharm, a major pharmaceutical company headquartered in China, to advance the clinical development of AU-018 for heart-safe chemotherapy and AU-409 for advanced liver cancers, as part of clinical studies being conducted in China,
In August 2024, Lee’s Pharmaceutical Holdings Limited (HK:0950) announced the initiation of a Phase I clinical trial for AU409. The trial, which marks the first human study of AU409 in Asia, is focused on patients who have not responded to standard treatments and aims to establish the drug’s safety, dosage and preliminary efficacy. Results from the trial, conducted at Queen Mary Hospital in Hong Kong, are anticipated to influence new drug registration in China. And
the USC Norris Cancer Center running the U.S. Phase 1 trial of AU-409 for advanced liver cancer and other solid tumors that metastasize to the liver.
Auransa has raised $26.5M.