Biotechnology is no stranger to the creation and analysis of large amounts of data, whether it is “-omics” in nature or from patients in clinical trials. However, having access to masses of datasets is only a good starting point; sooner or later actionable information will need to be drawn from those resources.

That is why so much buzz has been created by the term “big data,” which encompasses a range of initiatives being created to develop new methods, tools and analytics, to take advantage of the very large, complex and diverse datasets being created from biomedical research.

In this second of a continuing series, we look at two companies that have developed advanced analytics platforms and tools designed to interpret big data that will ultimately help accelerate the drug discovery process.

There is no doubt that being able to harness and mine the utility of those big data will be critical for innovative drug development going forward. Yet reducing massive databases into byte-sized pieces that are both meaningful and actionable represents an enormous leap, and we are only just beginning to figure out how to take the first steps in that process.

“Certainly we don’t have an issue with the quantity of data,” Niven Narain, president and chief technology officer of Berg Pharma LLC, told BioWorld Insight. “Genomics, however, does not tell the entire story of the patient; its utility can only be expressed when these data are integrated with functional data and other clinical parameters.”

The Boston-based company, which was co-founded by billionaire businessman, venture capitalist and real estate mogul Carl Berg, is positioning itself at the cutting edge of supercomputing, artificial intelligence and drug discovery.

In simple terms, its Interrogative Biology platform takes trillions of datasets to understand the biology of normal cellular processes, overlayed by the disease “state” in those tissues, in order to shine a light on the proteins that are causing the disease. (See BioWorld Today, Feb. 18, 2014.)

From higher order mathematics and predictive algorithms, the company is flipping the drug discovery model and using those tools to allow the body to “tell us what has gone wrong and design drugs from that starting point,” Narain noted.

The platform has allowed the company to build a portfolio of early stage drug candidates and diagnostics across multiple indications, with the largest concentration in cancer.

The early expression of that activity is BPM 31510, currently in phase Ib trials for solid tumors and is among the first cancer drugs ever to be identified through artificial intelligence, the company said. Since cancer cells like to grow in a low oxygen and high lactic acid environment, the molecule, which resides in mitochondria, aims to restore oxidative phosphorylation and confer re-capitulation of the BCL-2 protein family potential to induce cell death, a process cancer evades.

The trial for BPM 31510 in 300 to 400 patients with solid tumors, including pancreatic, triple-negative breast cancer, liver cancer and brain cancer, is ongoing. The program will make use of the company’s drug discovery platform to characterize a molecular and metabolic footprint for each patient based on multiple tissue samples. The data will allow investigators to assess response of the patients’ cells to treatment. That information can then be used in subsequent patient selection in later clinical trials.

A ‘ROAD MAP’ FOR DRUG DISCOVERY

Branford, Conn.,-based Bioxcel Corp. is working with industry partners to help improve upon its decision-making in drug discovery. The firm has developed a cloud-based big-data analytics platform, known as PharmGPS a disease area-focused, analytics engine encompassing all major and specialty therapeutic areas.

The platform draws together data and knowledge from the clinical and patient outcomes arena as well as the molecular research environment. Using a big data approach to those multiple, and often diverse datasets, key insights can be derived for the most appropriate drug discovery strategy.

Recently, the company introduced an orphan disease suite to aid in the discovery, development, licensing and commercialization of drugs for the approximately 7,000 rare and ultra-rare diseases that have been identified.

“We continue to innovate and expand the potential of PharmGPS to address the unmet medical needs of those with rare diseases,” Vimal Mehta, company chairman told BioWorld Insight. “We are providing access to this suite through a partnership model to design and develop breakthrough treatments.”

The company noted that less than 5 percent of rare diseases currently have effective treatments. Adding to the complexity is the fact that those diseases span multiple therapeutic areas. The firm’s underlying metadata and analytics for the gamut of orphan diseases, its 3,000 associated genes and hundreds of disease pathways related to therapeutic modality, is designed to address that complexity in a highly systematic manner and help partners explore innovative ideas and uncover valuable potential therapeutic approaches.

By Peter Winter

BioWorld Insight Editor