The Bio Report

Winning regulatory approval for a drug to treat obesity would seem like a great accomplishment, but for Orexigen Therapeutics, that’s when the hard work began. After its marketing partner Takeda ended their agreement because of disappointing sales, the company found itself in the position of having to market a drug to doctors who often don’t consider obesity an illness and believe willpower and discipline, not a pill, is what’s needed. Orexigen’s solution centered on a novel approach. The company decided to make a pitch directly to patients and connect them to telehealth-based doctors, who could ensure use of the drug is appropriate and help them avoid the embarrassment they may feel when speaking to their own doctors. We spoke to Thomas Cannell, Chief Operating Officer and President of Global Commercial Products for Orexigen, about its obesity drug Contrave, its marketing strategy, and whether it represents a marketing model that others may follow.

Though breakthroughs in the ability to read, write, and edit DNA have broad implications for healthcare, they are also fueling a far-reaching transformation of industries and laying the foundation for a new bioeconomy. The SynBioBeta conference, which has developed into a critical annual event for innovators and investors within the synthetic biology sector, will be held in San Francisco October 3 through October 5. We spoke to John Cumbers, founder of SynBioBeta, about trends within synthetic biology, key drivers and challenges for the sector, and why companies in all industries today need to begin crafting their own bio strategies.

The pharmaceutical industry has long argued that high drug prices are necessary to incentivize investment in and fund high-risk research and development of innovative new therapies. In a working paper published by the Institute for New Economic Thinking, William Lazonick, professor of Economics at the University of Massachusetts Lowell, and his colleagues challenge the industry’s premise. They argue that top pharmaceutical companies, spend more of their profits on buying back their shares to boost their stock prices than they do on R&D, a move that enriches senior executives. We spoke to Lazonick about the paper, why he believes this so-called financialized business model is counterproductive to innovation, and what steps he thinks are necessary to change the landscape.

Gonorrhea is a common sexually-transmitted infection, but the growth of an antibiotic-resistant strain of the disease is creating what the U.S. Centers for Disease Control and Prevention has called an urgent public health threat that requires aggressive action. Entasis Therapeutics is launching a pivotal study of Zoliflodacin, a new class of oral antibiotic that has demonstrated potent activity against resistant gonorrhea. The company last month entered into a novel partnership with the non-profit Global Antibiotic Research & Development Partnership to fund the pivotal trial and assure access to the drug in low- and middle-income countries if successful. We spoke to Manos Perros, CEO of Entasis, about Zoliflodacin, the partnership with GARDP, and whether it serves as a model for the development of a broader arsenal of new antibiotics.

A number of clinical successes and the U.S. Food and Drug Administration’s granting of designations that provide accelerated pathways to experimental therapies speaks to the progress of research funded by the California Institute for Regenerative Medicine. We spoke to Kevin McCormack, ‎senior director of public communications and patient advocate outreach for CIRM, about the growing pipeline of therapies, the changing regulatory environment, and whether the institute will have a future beyond its existing funding.

The problem of counterfeit and falsified medicines is not just an economic problem for the pharmaceutical industry, but also a threat to public health. TruTag Technologies is seeking to address the problem with its nano-scale technology that allows drugmakers to encode information on individual doses of medication to authenticate a drug and provide details on its origin and intended market. We spoke to Barry McDonough, senior vice president of business development for TruTag, about the technology, how it works, and how it can address a global health concern.

Mosquitoes are more than just pesky. Certain types of insect serve as efficient vectors for infectious diseases that pose great harm to humans. Oxitec, a subsidiary of Intrexon, has developed a genetically altered male of the Aedes aegypti mosquito designed to mate with wild females to produce offspring that die before becoming adults. The effort is meant to provide a highly targeted alternative to insecticides that are broad acting, can cause harm to humans and other animals, and may be unable to effectively reach their intended targets in urban environments. We spoke to Hadyn Parry, CEO of Oxitec, about how the company breeds billions of mosquitos that can’t reproduce, how it delivers them to where they are needed, and how the company is addressing the regulatory barriers to demonstrate its technology is safe and effective.

Scrutiny of drug prices around the globe is expected to exert growing pressure on the biopharmaceutical sector. EvaluatePharma, in its recently issued World Preview 2017, Outlook to 2022 says that despite consensus forecasts for worldwide drug sales hitting more than $1 trillion in 2022, it does reflect a drop from the same period last year. We spoke to Antonio Iervolino, head of forecasting for evaluate pharma, about the new report, the outlook for the sector and the potential for a new patent cliff with the advent of biosimilars.

Celiac disease, an autoimmune disorder that has helped to drive the gluten-free food craze, can carry serious complications. There’s no treatment for the disease, which afflicts an estimated 2.4 million people in the United States. PvP Biologics is taking a unique approach to treating celiac disease with its Kumamax, a synthetic enzyme that degrades the parts of gluten that trigger an immune response. We spoke to Adam Simpson, president and CEO of PvP Therapeutics, about the interesting history behind Kumamax, how it works, and why it will be the only drug PvP ever develops.

The lack of clarity over healthcare reform, changes to tax policy, and concerns about new pricing pressures are creating uncertainty over the future health of the biotech industry. EY, in its just released annual report on the industry, looks at the effect the growing uncertainty has had on the performance of the biotech companies and strategies for contending with what’s ahead. We spoke to Glen Giovannetti, Global Biotechnology Leader for EY, about the new Beyond Borders report, what the numbers tell us, and ongoing efforts for the industry to adapt to a healthcare world moving from volume to value.

The high cost of drug development, the challenge of translational research, and continuing concerns with R&D efficiency has had entrepreneurs, investors, and drugmakers open to experimenting with new models of innovation. COI Pharmaceuticals, born out of a collaboration between the pharmaceutical giant GlaxoSmithKline and Avalon Ventures, is one such model that is showing traction. With management expertise, R&D infrastructure, and a collaborative environment, COI is providing promising startups with a way to accelerate their development in a capital efficient way. We spoke to Jay Lichter, president and CEO of COI Pharmaceuticals, about the COI model, the challenges of cost-effective innovation, and what can be learned from COI’s experience.

The production of biologics is costly and takes time. Dyadic International has developed a proprietary manufacturing process that replaces the use of Chinese hamster ovary cells, long used to produce protein therapeutics, with a fungus that has a long history in industrial biotechnology applications. The company believes its process can produce drugs faster, in greater volumes, and at significantly lower costs than biotech processes in use today. We spoke to Mark Emalfarb, CEO of Dyadic, about its manufacturing process, the benefits it could bring, and why it may have big implications for drugmakers.

Alzheimer’s disease is the sixth leading cause of death and it continues to rise. Today it is costing the U.S. healthcare system $200 billion a year and there are no therapies that prevent, halt, or reverse the disease’s progression. United Neuroscience is taking a new approach to the disease with its endobody vaccines, a class of synthetic biologics it is developing to treat Alzheimer’s and other CNS conditions. We spoke to Ajay Verma, chief medical officer of United Neurosciences, about Alzheimer’s, the company’s platform technology, and the promise of enlisting the immune system in the fight against CNS diseases.

Gene therapy represents an expanding area of potential to correct and modulate the activities of genes driving diseases. One of the major challenges these groundbreaking technologies face, though, is delivering them to where they need to go within the body to be effective. Nanogenic Solutions believes its solved the problem with its LipTide technology that marries a payload carrying-lipid with synthetic peptides that target cell suface receptors and allow DNA or RNA into the cell. We spoke to Simon Newman, director of preclinical development for Nanogenic Solutions, about the challenges of gene therapy, the LipTide technology, and what it could mean for advancing a range of RNA and DNA therapies.

Brexit, the United Kingdom’s plan to withdraw from the European Union, carries a long list of unintended consequences, one of which is the relocation of the European Medicines Agency. As many as 20 cities are competing to become the new home of the drug regulator. We spoke to Rory Mullen, International Investment Executive for IDA Ireland, about Dublin’s efforts to woo the EMA, the process going forward, and why its viewed as an economic prize.

Nearly one in three drugs has a postmarket safety event, according to a study published earlier this month in JAMA. The study looked at 222 novel therapies approved between 2001 and 2010. We spoke to Nicholas Downing, clinical fellow in the Department of Medicine at Brigham and Women's Hospital and lead author of the study, about the findings, the limits of clinical trials, and whether the U.S. Food and Drug Administration needs to do more to improve surveillance of drugs once they are approved.

Climate change is not often viewed as a public health threat, but it can have unexpected consequences on the spread of infectious diseases. As changing temperatures make new areas more hospitable to mosquitos, its can raise the risk of mosquito-borne diseases, such as dengue, chikungunya, and zika in areas not usual thought as prone to outbreaks of tropical disease. We spoke to Erin Mordecai, assistant professor of Biology at Stanford University and lead author of a recent study in PLoS Neglected Tropical Diseases that looks at how climate change may affect the spread of mosquito-borne disease. We spoke to Mordecai about the study, what it means for potential infectious disease outbreaks, and how public health officials and drugmakers in the developed world may need to think differently about neglected tropical diseases.

Ten years ago, a group of San Francisco State University alumni working at Genentech got together with the Michael Goldman, the university’s chairman of the biology department, to establish the school’s annual Personalized Medicine Conference. This year, the conference will revisit topics from its past ten years as well as exploring the future of personalized medicine ask speakers to reflect on how personalized medicine has changed their lives. We spoke to SF State’s Goldman and Dan Maher, one of the alumni who drove the creation of the conference, about the state of personalized medicine, the pace of advances, and what’s surprised them most about where we are today.

The promise of precision medicine to provide more effective and safer cancer therapies that target the genetics driving each patient’s disease has been hindered by the lack of understanding of the specific genetic alterations underlying many cancers. Elana Fertig thinks math can solve that problem. Fertig, with a nod to the algorithm Netflix uses to help recommend movies users might like, is working to identify the genes that drive an individual’s cancer. Fertig, assistant professor of Oncology Biostatistics and Bioinformatics at Johns Hopkins Kimmel Cancer Center, discussed the proliferation of genetic data relating to cancer, how researchers may be able to capitalize on that, and how such an approach may also bring a new understanding of why patients suffer relapses and develop resistance to cancer therapies.

For anyone who has ever sought to find the price of healthcare services before obtaining them it will come as little surprise that information is not readily available. A recent study from Public Agenda, with support from the Robert Wood Johnson Foundation and the New York State Health Foundation, set out to find how Americans seek and use healthcare price information. We spoke to David Schleifer Director of Research for Public Agenda about what extent healthcare consumers seek price information, the implications of having a system with little price transparency, and what can be done to fix the problem.