The Bio Report

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 discovery to care.

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.