Informatics helps virtual biotech bring compound to clinical trial

Laboratory informatics

This article is sponsored by Collaborative Drug Discovery.

Libra Therapeutics is a San Diego-based biotech focused on developing novel disease-modifying therapeutics, which restore the cellular balance that is disrupted in neurodegenerative diseases. The Libra portfolio includes three small molecule programmes, including their lead non-clinical programme targeting TRPML1 (based on an innovative assay generated by the contract research organization Axxam and hit compounds identified through multiple screening campaigns), which is on track to start Phase I clinical trials in 2025, states Martin Gill, Libra’s Vice-President and Head Of Biology.

Libra is a completely virtual company, based on a “plug and play concept and mode of operation”, Gill explains. With no in-house laboratory space, the company works closely with multiple international contract research organisations (CROs), including Axxam, which carry out all of the Libra R&D, from compound screening assays, to in vivo pharmacokinetic/pharmacodynamic and ADME work, as well as in vitro and in vivo pre-clinical safety assessments.

This virtual biotech set-up brings with it the need for a secure data management platform that can store diverse screening, chemistry and biology datasets from CRO partners, but also gives Libra users easy access, visualisation and comparative tools for data retrieval, review and analysis across disciplines.

Since its inception, Libra has used CDD Vault as its primary repository for all compound structural data and screening assay results, along with physicochemical and other in vitro biology data and associated metadata generated by its CRO partners.

“From my perspective, the Vault provides the high level of functionality and flexibility that might be expected from some of big pharma’s in house platforms, while remaining within the financial reach of smaller biotech companies and academic spinouts.” Key Vault functionality for Libra is the ability to link chemistry to biology. “Using CDD Vault we can maximise the value of the data that we have, to help identify and visually drive SAR, and make good hit-to-lead decisions,” Gill noted.

Libra’s pipeline targets mechanisms that can mitigate protein pathology associated with CNS degenerative disorders. The lead programme is focused on identifying small molecule activators of TRPML1 for the treatment of CNS degenerative disorders, as well as degenerative peripheral indications. IND-enabling studies for Libra’s lead TRPML1 programme are projected to start during the first half of 2024, with clinical Phase I single ascending dose and multiple ascending dose studies on track for 2025, Gill explains. Libra also has two additional discovery-phase programmes that are at the target validation stage.

The flexible Vault setup allows users to visualise and compare diverse datatypes in whichever way they need, Gill suggests. “User-friendly access is a significant plus point, and the Vault architecture lets us customise to suit different datatypes, so we don’t have to try to shoehorn our data into a rigid database or engineer complex, expensive workarounds. Using CDD Vault we can, for example, upload our biology results, extract the endpoints that we need, and have all the underlying data – for example, structural chemistry datasets – accessible alongside those biology results and endpoints.”

Security built in to CDD Vault is critical when working with multiple CROs. Access to the Vault can be restricted by authorisation for Libra users as well as for external users, which, Gill notes “protects the data from the potential for accidental internal harm,” as well as from any external security risk. Users can also export data into Excel, so outgoing files can be password protected.

Libra has initiated several confidential discussions with potential clinical development partners for its TRPML1 programme, and the flexibility of CDD Vault will prove invaluable, Gill suggests. “The Vault gives us the ability to select, pull up, and organise exactly the data that will be needed for these discussions, tailored to what each potential partner may want to see and understand. The user interface makes it easy to present the data in ways that are informative, and we can also generate customised, as well as standardised reports.”

About Collaborative Drug Discovery

CDD provides a modern approach to drug discovery informatics that is trusted globally by thousands of leading researchers. Its CDD Vault is a hosted informatics platform that securely manages both private and external biological and chemical data. It provides core functionality including chemical registration, structure activity relationship, inventory, visualisation and electronic lab notebook capabilities.

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