The Bio Report

The convergence of information technology with the life sciences is opening up new ways of approaching the challenges of drug discovery and development. While much has been made about approaches that seek to leverage artificial intelligence, Nanome is bringing virtural reality into the process. With the company’s technology, scientists can take a fantastic journey to view and manipulate proteins, compounds, and other molecules at a nanoscale. We spoke to Steve McCloskey, founder and CEO of Nanome, about the company’s virtual reality platform, how scientists can use it to collaborate in real time, and its potential to change the way researchers discover and develop drugs.

The high cost of developing therapies impedes the pharmaceutical industry’s ability to bring innovative products to market, but also leaves potentially valuable discoveries languishing on university shelves. The Swiss non-profit foundation Molecule is trying to alter the landscape by establishing an online marketplace to create shared ownership of intellectual property and provide a new funding mechanism for early-stage experimental therapies that could also lead to distributed research and development of promising drugs. We spoke to Paul Kohlhaas, founder of Molecule, about the market it is developing, how it would work, and the challenges it will need to overcome to make it a reality.

Mesenchymal stem cells may be a powerful way to treat a range of medical conditions but realizing the therapeutic potential of these cells has been hampered in part by the challenges of manufacturing a consistent product at scale. Cynata Therapeutics believes its platform technology addresses these challenges. The company lead cell therapy candidate is being developed to treat graft vs. host disease, a potentially deadly response to the transplantation of cells from a donor. We spoke to Ross MacDonald, CEO of Cynata, about the company’s platform technology, the potential for these therapies, and the indications the company is pursuing.

The placebo response in clinical trials can derail a promising experimental therapy that might benefit patients. In cases where trials rely on subjective endpoints or patient-reported outcomes, the placebo response can be more pronounced. Tools4patient has developed a means of identifying patients who are likely to be placebo responders and allow trial sponsors to take steps to account for that in clinical studies. We spoke to Erica Smith, vice president of business development at Tools4Patient, about the placebo response, the consequence this phenomenon has on drug development, and how the company is seeking to address this problem.

Artificial intelligence is being embraced the pharmaceutical industry as a way to reduce the time and cost of drug development. BioXcel Therapeutics is using AI to leverage as a means to identify new targets for approved drugs and clinically-validated drug candidates with a focus on oncology and neurology. We spoke to Vimal Mehta, CEO and founder of BioXcel Therapeutics, about the company’s AI platform, what makes it unique, and its pipeline of repurposed therapies.

The ability to capture and parse data in real-time and at a vast scale is transforming our approach to biomedical research and has the potential to change the way we think about the causes of disease, public health, and medical interventions. Paul Glimcher, president and CEO of Datacubed Health and professor of Neural Science, Economics and Psychology, at New York University, where he directs the Institute for the Study of Decision Making, is conducting an ambitious study known as The Human Project. The study will gather data on the everyday habits and health of 10,000 New Yorkers over 20 years to find critical connections between biology, behavior, and the environment to drive evidence-based public policies to improve lives. Through his company Datacubed Health, he’s using the same technologies to drive patient-centered research. We spoke to Glimcher about his work, how technology is changing the nature of health research, and how he sees digital technology transforming the healthcare continuum from discover

Cidara Therapeutics is developing new anti-infectives using its Cloudbreak platform to develop antiviral conjugates that both directly kill pathogens and also enlist a patient’s immune cells to attack and eliminate them. The company is advancing its first therapy developed through the platform to the clinic, CB-012, an experimental therapy that is being developed to both treat and prevent flu infections. We spoke to Jeff Stein, CEO of Cidara, about the therapy, how it works, and the need the company is trying to address.

Roivant Sciences doesn’t fit easily into conceptions of drug companies, venture capital firms, or accelerators. It is building highly-focused drug development companies around promising undervalued assets it licenses. In five years’ time, it’s raised more than $3 billion, amassed a broad pipeline of more than 35 therapies, and has more than 800 employees. We spoke to Eric Venker, chief operating officer of Roivant, about the company’s business model, how it leverages its resources, and how it may be changing the industry’s approach to drug development.

Advances in precision medicine have helped to reshape the cancer landscape with the development of diagnostics to determine whether a patient may benefit from a specific drug. Little progress, though, has been made outside of the area of oncology to avoid the waste of time and money on therapies that won’t work for an individual patient. Scipher Medicine is working to change that for the treatment of autoimmune diseases by identifying which patients would benefit from which drugs. We spoke to Alif Saleh, CEO of Scipher, about the company’s approach, the opportunity it is addressing, and how he sees the area of precision medicine evolving.

Concerns about the threat posed by the growth of drug-resistant pathogens has often focused on the need to reinvigorate our arsenal of antibiotics, but health authorities are paying new attention to the rise of invasive fungal infections. Globally, about 1.5 million people each year die from these infections. There’s been a lack of investment and innovation in new types of antifungals and the worry is growing about the emergence of fungal strains that are resistant to existing treatments. We spoke to Ciara Kennedy, president and CEO of Amplyx, about the need for new antifungals, why this problem is emerging, and her company’s first-in-class experimental therapy to fight fungal infections.

The effort to cost-effectively accelerate the development of new therapies has led to the rise of new business models that seek to provide expertise, capital, and access to professional networks that can help early-stage companies navigate pitfalls and overcome challenges. Xontogeny is a bit of a hybrid in its approach, preferring the term aggregator to accelerator or incubator. We spoke to Chris Garabedian, chairman and CEO of Xontogeny, about the company’s approach, what it looks for in the biotechs it backs, and what he’s learned from his own experience as a biotech executive that he thinks will benefit others.

Agenus is an immuno-oncology company with a long history and a broad portfolio. To help fund its pipeline, it entered into a significant alliance with Gilead Sciences in December 2018 and has used an unusual funding mechanism by raising money through what it described as the first asset-backed digital-security offering. We spoke to John Castle, head of translational medicine and bioinformatics at Agenus, about the company’s approach to managing and prioritizing its pipeline, its use of artificial intelligence, and the recent deals it’’s done to fund development.

The recent late-stage failure of another high-profile Alzheimer’s disease drug candidate is a reminder of the challenges of developing drugs for neurological diseases. These challenges are driven by the fact that there are hundreds of different brain cell types and complex circuits and pathways in the brain that make it difficult to identify the right protein to target to treat a given disease. Cerevance says its proprietary platform overcomes the limitations of must approaches used today to study human brain cells. We spoke to Brad Margus, CEO of Cerevance, about the company’s platform technology, how it addresses the challenges drug developer face, and the programs it has advanced to date.

Intensity Therapeutics argues that cancer is both a micro and macro disease. Therapies need to act at the site of tumors, but they also need to treat the systemic effects of the disease that results from micro-metastases. Intensity is developing cancer therapies that are delivered directly into tumors, but also stimulate the innate immune system to address the more systemic effects of cancer. We spoke to Lewis Bender, CEO of Intensity, about the company’s platform technology, how it enlists the innate immune system in the battle against cancer, and the thinking behind the company’s approach.

SQZ Biotech believes its platform technology can enable the engineering of virtually any function into any cell type allow for a new world of cell therapies to revolutionize the treatment of diseases. As its name implies, it does this by squeezing a cell to momentarily disrupt its membrane to allow it to insert a range of substances. We spoke to Armon Sharei, CEO of SQZ Biotech, about the company’s platform technology, its initial focus on oncology and auto-immune diseases, and the broad potential for this approach.

As concerns grow about the rise of antibiotic resistant bugs, the ability to combat them is being undermined by the economics of antibiotic drug development. Many large pharmaceutical companies stepped away from the space and despite the passage of the GAIN Act in 2012 and government investment in antibiotic research and development, drug companies argue more action is needed. We spoke to Michael Dunne, chief scientific officer at Iterum Therapeutics, about the state of the antibiotic arsenal today, the challenges drug developers face, and whether Congress will be willing to provide the industry incentives to address societal needs at a time when there is growing animosity towards the industry over issues such as pricing.

Neurotrope is developing its experimental therapy bryostatin as a potential treatment for Alzheimer’s disease and other neurodegenerative conditions. The company believes the drug has multiple mechanisms of action and can stimulate synaptic growth factors to repair damaged synapses, form new ones, and prevent neuronal death. The company, though, found itself in a controversy over the way it characterized results from a phase 2 study of the drug in 2017. It is now conducting a second phase 2 study of the drug in the hopes of demonstrating meaningful benefits for patients. We spoke to Daniel Alkon, chief scientific officer of Neurotrope, about bryostatin, the controversy over the way it reported its previous results, and the path forward for the drug.

As people age, their bodies lose the ability to regenerate as well as they were able to when they were younger. Alkahest believes this is tied to changes in the mix of proteins that are present in the blood as we age. It is developing therapies for diseases of aging that seek to target specific proteins that are upregulated in diseases of aging, as well as providing a wide set of proteins drawn from purified human plasma as a therapeutic approach to counter the depletion of regenerative proteins. We spoke to Elizabeth Jeffords, chief commercial and strategy officer for Alkahest, about the science underlying the company’s approach, the controversy around others who have offered so-called “young-blood” transfusions, and the pipeline the company is pursuing.

There was a time when scientists could spend their careers talking only to other scientists about their work, but that’s changed. Whether it’s the current funding climate, the need to understand complex scientific issues underlying public policy debates, or opportunities to fund translational work and commercialize important discoveries, scientists are increasingly being tasked with addressing lay audiences. In “Championing Science: Communicating your Ideas to Decision Makers” the husband and wife team of Roger and Amy Aines offer a detailed guide for scientists on how to communicate effectively with non-scientists. Roger Aines is chief scientist of the energy program at Lawrence Livermore National Laboratory and Amy Aines is CEO of Damianakes Communications. Each bring their professional experience to the book. We spoke to the duo about their book, why it's needed, and why they believe scientists not only need to learn how to communicate their ideas, but to compel action and change the world for the better.

Kleo Pharmaceuticals is a developing next-generation immuno-oncology drugs that are small-molecule compounds designed to act like biologics. Kleo’s compounds activate patients’ immune systems to target and destroy cancer cells but are faster and less costly to design and produce than biologics. We spoke to Doug Manion, CEO of Kleo, about the company’s platform technology, why he thinks it will produce safer and more effective immunotherapies, and the potential to use these compounds in conjunction with existing biologics to enhance their activity.