The Bio Report

People with attention-deficit/hyperactivity disorder who use stimulants to treat the condition often face afternoon crashes and require the use of booster doses. Cingulate Therapeutics is using its proprietary drug delivery technology to target this $14 billion market with timed released formulations of commonly used ADHD drugs it is developing. We spoke to Shane Schaffer, CEO of Cingulate, about ADHD, the company drug delivery technology, and its clinical path forward.

Problems of women’s reproductive health represent a growing worldwide concern, but it remains an area of unmet medical needs. ObsEva is advancing a late-stage clinical pipeline with development programs focused on treating endometriosis, uterine fibroids, preterm labor and improving IVF outcomes. We spoke to Ernest Loumaye, CEO of ObsEva, about the issue of women’s reproductive health, the company’s lead therapies in development, and the plan for commercializing its products.

CytoDyn’s lead candidate for HIV is part of a new class of therapies that work by protecting healthy cells by blocking viral infection. But the receptor that the drug targets also plays a role in cancer metastasis and may provide a new approach to treating a wide range of cancers. We spoke to Richard Pestell, chief medical officer of CytoDyn, about the drug, how it works, and why it may have value in a range of serious medical conditions.

The rise of the Internet, social media, and communications technology has transformed consumer behavior. Consumers today are better informed, expect a high-level of engagement, and a modern service experience. Shay Brill, vice president of corporate development for Atlantic Research Group, in a white paper released at the recent Global Genes Rare Patient Advocacy Summit, argues that similar changes are underway in the behavior of patient-consumers. We spoke to Brill about these trends, how they’re is changing drug developers’ relationships with patients, and what these changes mean for sponsors of clinical trials.

DxTerity is a genomics company that’s providing real-world monitoring of patients with autoimmune disease with from-home RNA testing to improving the management of these conditions. The company’s technology not only has the potential to change the way diseases such as multiple sclerosis, lupus, and rheumatoid arthritis are managed, but change the way clinical trials in these conditions are conducted. We spoke to Bob Terbrueggen, CEO of DxTerity, about the company’s from-home RNA tests, how they work, and the potential application of the technology.

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.

Stem cell therapies represent an area of great promise for treating intractable eye diseases, but there’s growing concern about clinics that promote costly stem cell treatments that have not been approved by the U.S. Food and Drug Administration and have not been proven to be safe or effective. This is already a multi-billion business and researchers have found some disturbing practices that have resulted into harm to patients. We spoke to Ajay Kuriyan, assistant professor at the Flaum Eye Institute and University of Rochester Medical Center, about his research into these clinics, how they sometimes disguise their work as legitimate clinical trials, and why this represents a threat not only patients, but this emerging therapeutic area.

Say “Oklahoma” and someone might think of wheat fields, natural gas, or the wind sweepin' down the plain. Cutting edge biomedical research, though, is probably not the first thing that comes to mind. Nevertheless, the Oklahoma Medical Research Foundation has for more than 70 years been conducting innovative scientific work that has forged new understandings of disease and made discoveries that have led to new drugs and diagnostics. We spoke to Manu Nair, vice president of Technology Ventures for the Oklahoma Medical Research Foundation, about the work it does, its efforts to commercialize its discoveries, and the challenges of getting on industry’s radar when you are outsides of a major biotech center.

Recursion Pharmaceuticals is reinventing the drug discovery process by turning biology into a data science problem. The company has set an audacious goal for itself of developing 100 drugs in 10 years. Though Recursion initially focused on repurposing existing drugs to treat rare diseases, it has expanded its work into new disease areas and is looking at new chemical entities as well. We spoke to Chris Gibson, founder and CEO of Recursion, about the approach the company is taking, the challenges of mixing biologists and data scientists together, and why he’s holding fast to his goal of attaining an unparalleled level of drug development efficiency through the use of artificial intelligence.

The blood-brain barrier provides essential protection against pathogens while allowing needed oxygen and nutrients to pass. However, one challenge it presents is getting therapeutics delivered to the brain and central nervous system. Bioasis Technologies has developed a way to attach fusion proteins to drugs to allow them to pass the blood-brain barrier. We spoke to Mark Day, CEO of Bioasis, about its platform technology, what is known about it from testing to date, and the potential therapeutic implications of being able to deliver drugs systemically that can reach the central nervous system and brain.

There’s been much made of the potential of the microbiome to address disease and promote wellness. While much of the therapeutic efforts in this area have focused on the microbiome of the gut, Azitra has developed a platform for selecting bacteria native to the skin and engineering it to produce therapeutic proteins. We spoke to Travis Whitfill, chief science officer of Azitra, about the company’s platform, why it may be preferable to apply bacteria to the skin that can produce therapeutic proteins where they are needed, and why some of the biggest opportunities for the technology may be in the health and beauty markets.