The Bio Report

The availability of a growing body of real-world evidence has regulators considering how clinical trials using disparate sources of data might work. Many see harnessing such information as a way to provide better insight into the safety and efficacy of drugs while reducing the cost of clinical trials. A number of issues, though, will need to be addresses as the U.S. Food and Drug Administration takes a first pass at a framework for using real-world evidence. We spoke Nancy Dreyer, chief scientific officer & senior vice president of real-world & analytic solutions for IQVIA, about real-world data, its potential to change the way clinical trials are conducted, and the challenges to applying it.

Immunotherapies are changing the way physicians treat various cancers, enabling and enlisting a patient’s immune system in the fight against a deadly disease. But this approach has largely been limited to the field of oncology. Cidara Therapeutics, which is developing new anti-infectives, is using its Cloudbreak platform to develop antibody-drug conjugates that directly kill pathogens and also direct a patient’s immune cells to attack and eliminate bacterial, fungal, or viral pathogens. We spoke to Jeff Stein, CEO of Cidara Therapeutics, about the company’s platform technology, the pipeline its advancing, and why innovative approaches are needed to address infectious disease.

Nucleoside analogs are a widely used class of chemotherapeutic agents, but the ability of cancer cells to evade them and develop resistance limits their benefits and increases their side effects. NuCana, which is developing a new class of cancer agents dubbed ProTides believes it can address this problem with nucleoside analogs by delivering them efficiently into cancer cell and preventing their degradation before they can act. We spoke to Hugh Griffith, founder and CEO of Nucana, about the pr oblems of resistance, how the company’s ProTide technology works, and the pipeline the company is advancing in the clinic.

Synlogic is using synthetic biology to engineer probiotic bacteria to deliver therapeutic benefit. By targeting the microbiome of the gut, the company believes it can produce systemic effects to treat metabolic and other diseases with its new class of therapies it dubs Synthetic Biotics. We spoke to Aoife Brennan, interim CEO and chief medical officer of Synlogic, about the company’s platform technology, how its seeking to exploit the microbiome, and how its engineered bacteria may offer a safer and more effective way to treat certain conditions.

One way to improve drug discovery and development is to work with more accurate models of human biology and human disease. Organ-on-a-chip technology that provides three-dimensional cell models in conventional microfluidic plates is helping to do that. Mimetas is one company that’s delivering this technology to the hands of researchers today. We spoke to Jos Joore, co-founder and CEO of Mimetas about the technology, how its changing drug development, and whether organ-on-a-chip technology may one day find its way into the clinic.

Insilico Medicine is working to harness artificial intelligence to address diseases of aging and in the process reinvent the way new drugs are discovered and developed. Its AI platform is integrated into the continuum of the discovery and development process and seeks to improve target identification, the selection of drug candidates, and predict clinical trial outcomes. In addition to working in collaboration with the large pharmaceutical companies, Insilico is pursuing internal drug discovery programs in range of diseases of aging. We spoke to Alex Zhavoronkov, CEO of Inisilco Medicine, about the company’s platform technology, the potential for AI to transform the discovery and development of drugs, and why Insilico focuses its efforts on diseases of aging.

Digital health technologies are providing new ways of monitoring patients and delivering care. In the realm of clinical trials, they provide a way to remove geographic barriers to patient participation, improve compliance, and reduce costs while also creating a way to capture real-world data. We spoke Bryan Silverman, CEO of ObvioHealth, about the company’s ClamiIt platform, the workings of siteless clinical trials, and the potential for digital technologies to address a variety of challenges trial sponsors face.

The convergence of artificial intelligence and the life sciences is promising to revolutionize the entire healthcare continuum including the way drugs are discovered, how clinical trials are conducted, the methods physicians use to diagnose patients, and how patients are monitored and treated. The life sciences investment firm Foresite Capital, armed with a new $668 million fund, is helping to bring about this change by backing innovative companies working at this nexus information technology and biotechnology. We spoke to Brett Zbar and Vik Bajaj, managing directors of Foresite, about their new fund, what constitutes a Foresite investment, and why they believe this is not just the latest fad, but a meaningful change in the way life sciences companies seek to address the challenges in all aspects of healthcare.

In this encore edition of The Bio Report, we revisit an interview that original ran in December 2017. Climate change and the increasing frequency and intensity of extreme weather events carry a toll on human health. Not only do floods, hurricanes, and other similar phenomenon cause death and injury, they also create long-term health effects. Jesse Bell, a research scientist at the North Carolina Institute for Climate Studies at North Carolina State University recently examined the issues in an article in the Journal of the Air & Waste Management Association. We spoke to Bell about the health consequences of these weather events, the challenges they create for public health systems to plan and prepare, and why new research is needed to better understand the relationship between these events and human health.

One of the reasons for the high cost of drug development is that most drugs fail in clinical development. Even though preclinical testing can provide a good insight into whether a drug hits an intended target, once in the body, drugs can interact with a large number of proteins and have unintended consequences. A-Alpha Bio, a spinout from the University of Washington's Institute for Protein Design and Center for Synthetic Biology, is developing a platform that can measure thousands of protein interactions simultaneously and how drugs affect them. We spoke to David Younger, co-founder and CEO of A-Alpha, about the company’s platform, the bottleneck in drug development it is addressing, and the business model it is pursuing.