The Bio Report

It can take up to six years to accumulate enough evidence to support advancing an experimental drug into clinical development. twoXAR is working to rapidly accelerate that process through the use of Big Data and its proprietary algorithms to find screen large public and proprietary data sets to indentify new drug candidates and determine their efficacy. We spoke to Andrew Radin, co-founder and CEO of twoXAR, about the company’s technology, how it’s being applied today, and the evolution of the company’s strategy.

Robert Goldman had been a successful Internet age inventor having developed some of the critical technology underlying the way digital music is stored, searched, and purchased online. But when his sister developed cancer, he found himself turning his skills as an inventor into an unchartered area for him. Eventually he developed a new type of catherter to deliver cancer medicines directly to where they are needed in the body. But despite his company’s success at winning marketing clearance from the U.S. Food and Drug Administration, his company Vascular Designs has had difficulty raising money. On October 17 Vascular Designs launched a campaign to raise $500,000 through the crowdfunding site Indigogo. We spoke to Goldman about his IsoFlow catheter, the funding environment for innovative medical device companies, and his decision to turn to crowdfunding.

One of the issues researchers face is getting access to needed biospecimens with detailed clinical data that can expand the understanding of a specific disease or treatment. The difficulty in obtaining biospecimens can hamper R&D productivity, extend the time of project, and increase costs. Novaseek has developed a platform that provides access to real world data and supports the ability to define patient cohorts based on detailed clinical criteria, such as demographics, diagnoses, medications, and lab test results, and supports the collection of biospecimens at various time points. The platform also offers healthcare organizations an opportunity to develop a new source of revenue. We spoke to Kate Torchilin, CEO of Novaseek, about the challenges researchers face in getting biospecimens, how Novaseek seeks to address that, and how the company’s platform can help to accelerate biomedical research.

Evaluate Pharma, in its latest World Preview report, is projecting solid growth for prescription drug sales through 2022. Driving that growth is both the new products expected to come to market during that time and the industry’s embrace of orphan drugs, the source of half of that growth. We spoke to Antonio Iervolino, head of forecasting for Evaluate, about the new report, the growing number of drug approvals, and the challenges drugmakers face that could alter the outlook for the industry.

The growing promise to harness biology to address environmental, agricultural, health, and energy needs is fueling a new bioeconomy. The state of engineering biology will be on display at the SynBioBeta SF 2016 conference in South San Francisco October 4 through October 6. We spoke to John Cumbers, founder of SynBioBeta, about the state of the industry, the challenges it faces, and the long-term potential it holds for transforming our manufacturing sector.

About 380,000 infants in the United States—about one in 10— are born prematurely each year. Preterm births, defined as any birth before 37 weeks of pregnancy, is the leading cause of death for infants and puts these babies at greater risk for cerebral palsy, respiratory and cardiovascular complications, and delays in development. Sera Prognostics has developed a predictive diagnostic that can identify pregnant woman at risk of delivering prematurely, allowing for interventions. We spoke to Greg Critchfield, CEO of Sera Prognostics, about the test, how it works, and what it take to get doctors to adopt the test and payers to reimburse for it.

The Johns Hopkins University School of Medicine and the Salk Institute for Biological Studies are leading a $15.4 million effort to develop new systems for quickly screening libraries of drugs for potential effectiveness against schizophrenia and bipolar disorder. The National Institute of Mental Health is funding the work. We spoke to Hongjun Song, professor of neurology and neurosciences at the Johns Hopkins University School of Medicine, about the effort, the challenges of understanding the underlying biological mechanism of these disorders, and how the consortium expects to change the development of drugs to treat mental illnesses.

The recent controversy over Mylan Pharmaceuticals sharp increase to the price of its EpiPen, an emergency injection of epinephrine to treat someone suffering a severe and potentially fatal allergic reaction, has once again ignited public debates over drug pricing. While drugmakers have been in the sights of the public, other players in the supply chain have escaped scrutiny. David Balto, a Washington, D.C.-based antitrust attorney, in a recent piece in The Hill, calls attention to the role pharmacy benefits managers play in the price equation. We spoke to Balto about PBMs, their growing power, and the perverse incentives they have to work against the best interest of consumers.

Werner’s and Hutchinson-Gilford Progeria Syndromes are rare, genetic diseases that causes children to appear to suffer from advanced aging at young ages. The syndromes are of particular interest to researchers because of the potential they may have to help understand the biology of normal again and lead to ways to counter aging-related diseases. We spoke to Brian Kennedy, president and CEO of the Buck Institute for Research on Aging, about his progeria research, the potential to repurpose existing drugs to combat these syndromes, and what insights into normal aging are being gained from the study of progeria.

Concern about the spread of the Zika virus has sparked efforts to develop therapies and vaccines to counter it. But the outbreak reflects a growing threat from zoonetic diseases, once thought of as being contained to tropical regions, that are making incursions into the developed world. We spoke to Jim Pannucci, director for infectious disease research at Southern Research, about Zika, the work his organization is doing around the virus, and whether we need to rethink investment in prevention and treatment of such diseases.

Lee Hood was a visionary who helped enable the genomics age. Journalist Luke Timmerman, in his newly published biography of Hood, chronicles the scientist career and achievements, as well as the controversies that surrounded him. We spoke to Timmerman about Hood, the conflicts that shaped his career, and the significance of his contributions to the field of biology.

Despite continued scientific and medical progress, the American Society of Clinical Oncology offered a mixed picture in its annual report on The State of Cancer Care in America earlier this year. While advances have been made in combating some cancers, mortality rates for others remain unchanged or have increased. There’s disparity in care, concerns about the rising cost of therapy, and uncertainty about how new payment models will affect care. We spoke to Julie Vose, immediate past president of ASCO, about the report, where additional work is needed, and how to balance the need to make cancer care affordable while encouraging innovation.

People with multiple chronic conditions represent about 5 percent of the patient population in the United States, but account for half of the nation’s healthcare spending. In order to improve care and reduce costs overal there’s an urgent need to do a better job of treating these patients, according to a recent perspective in the New England Journal of Medicine from the heads of five leading healthcare foundations. We spoke to David Blumenthal, president of the Commonwealth Fund and one of the authors of the piece, about why improving care for this population is critical, why there’s not a single solution to treating what is actually a diverse population of patients, and what these foundations are doing to address the problem.

Neglected diseases and drugs to counter addiction represent unmet medical needs, but they have been areas that pharmaceutical companies have been reluctant to pursue. Savant HWP is in clinical development on an experimental drug that targets both of these indications. We spoke to Stephen Hurst, CEO of Savant HWP about the company’s strategy, the unique compound that can address two seemingly unrealated indications, and the role priority review vouchers can play in providing drug developers with incentives to pursue indications that might otherwise be ignored.

The use of biomarkers has long held the promise accelerating drug development and producing safer and more targeted drugs to meet the needs of patients. The explosion of genetic, proteomic, and metabolomic data, as well as the emergence of the human microbiome, is rapidly growing the world of biomarkers, but with that comes challenges for researchers and drug developers about knowing what’s available and how well validated these biomarkers may be. We spoke to John Audette, president and co-founder of Amplion, about the company’s database, which seeks to make biomarker information accessible to researchers, the role biomarkers can play in improving the drug development process, and how their increased use can bring about a long anticipated era of precision medicine.

Incubators and accelerators have been proliferating in California, but little has been done to track their activities and analyze their performance. The California Business Incubation Alliance recently released a study of the performance of incubators and accelerators in California, the contribution they make to the state’s economy, and whether they deliver value to the entrepreneurs that use them. We spoke to Matt Gardner, CEO of the California Technology Council and lead author of the report, about its findings, why gathering data about incubators and accelerators is so challenging, and why the findings include a word of caution to entrepreneurs. As a matter of full disclosure, Matt is a friend, client, and partner and I served as an editor on this report.

The growing problem of antibiotic-resistant bacteria and the evolution of next-generation sequencing technology is giving rise to new approaches to combat what’s become a serious global health threat. Epibiome, which describes itself as a precision microbiome engineering company, is working to develop cocktails of bacteriophages to use in animal and human health. We spoke to Nick Conley, CEO of Epibiome, about the urgent need for new therapeutics, the renewed interest in phages, and Epibiome’s unique approach to address the problem.

The ability to find and tumor cells and DNA fragments shed by tumors circulating in the blood has given rise to liquid biopsies. This emerging area of diagnostics promises to improve cancer care, better monitor patient response to a therapy, personalize care, and provide earlier evidence of recurrence. We spoke to Andre de Fusco, CEO of Cynvenio, about the company’s liquid biopsy rare cell isolation platform, and its implications for changing how cancer patients

KaloBios, a biotech best known for a series of disastrous events including failed clinical trials, a bankruptcy filing, lawsuits, and the arrest of its CEO, is being reborn. In the process, it may provide a valuable contribution to the ongoing discussion over drug pricing. In April, the development-stage company unveiled a pricing plan based on transparency, affordability, and reasonable profit. We spoke to Cameron Durrant, CEO of KaloBios, about his efforts to clean up the mess he inherited, turnaround the fortunes of the company, and introduce an innovate pricing model that could reshape the way industry thinks about pricing its products.

Despite a rapid advance of technology there’s been little change in the preclinical drug development process. Vium is hoping to change that by using sensors, automation, and bioinformatics to change the way data is gathered in the preclinical process to fuel better and faster decisions about the potential value of an experimental drug. We spoke to Tim Robertson, co-founder and CEO of Vium and Joe Betts-Lacroix, co-founder and CTO of Vium, about the company, how its hoping to change the preclinical drug development process, and why that’s essential.

The biotechnology industry continued to post record numbers, attract significant investment, and won approval for a solid number of new drugs in 2015. Nevertheless, EY in its new Beyond Borders report warns that there’s a deceleration within the industry that points to threats that could undermine the robust growth it has enjoyed. We spoke to Glen Giovannetti, EY Global Biotechnology Leader, about the new report, the need for the industry to innovate pricing models for new drugs, and why as companies seeks to demonstrate the value of a product they will need to consider the unique perspective of each payer.

Wall Street’s attention will turn to Chicago as the annual meeting of the American Society of Clinical Oncology gets underway June 3 to June 7. Though this is a scientific conference, it is one closely watched by investors, who have been pouring through the abstracts that have been available and keeping an eye open for late-breakers that could move stocks. We spoke to Jon Gardner, deputy news editor for EP Vantage, about the ASCO meeting, what the early abstracts say, and who will likely be making headlines at this year’s meeting.

Lupus is an often misunderstood and misdiagnosed autoimmune disease. While some 1.5 million people—mostly women—have lupus, it can take years for someone with the disease to get a correct diagnosis. We spoke to Susan Manzi, medical director of the Lupus Foundation of America and Co-founder and director of the Lupus Center of Excellence at Allegheny Health Network, about the disease, why there are so few treatments available today, and why she holds out hope for the pipeline of new therapies moving through the clinic.

Global innovation activity experienced broad gains across industry sectors last year, according to Thompson Reuters newly released 2016 State of Innovation report. We spoke to Anthony Trippe, senior patent analyst for Thomson Reuters about the report, its upbeat outlook, and what it says about the direction of innovation in the biotechnology, pharmaceutical, and medical device sectors.

The Campaign for Sustainable Rx Pricing, a coalition of healthcare stakeholders, thinks that the rising cost of prescription drugs can be addressed through a series of market-based solutions that will strike a balance between preserving innovation and ensuring affordability. Its recently released plan outlines steps to increase transparency, competition, and value. We spoke to John Rother, executive director of the Campaign for Sustainable Rx Pricing, about the organization, its focus on market-based solutions, and what it will take to make them a reality.

The emergence of smartphones at the center of a growing universe of digital health devices is giving rise to new ways to monitor and measure our bodies in real time. MyDx, one of the latest entrants into this area, is working to enable consumers to test the food they eat, the water they drink, and the air they breathe to ensure they’re safe. Its first offering allows cannabis users to analyze samples for THC content, the presence of pesticides, and their potential effects on users. We spoke to Daniel Yazbeck, chairman and CEO of MyDx, about his handheld analyzer, how it works, and how he expects it to both enable and be fueled by changing consumer behavior in the emerging world of the quantified self.

The Obama Adminstration’s Cancer Moonshot initiative is a boost to cancer researchers, but Bruce Zetter says in order for it be as effective as possible, it must not repeat the same funding priorities of the past and instead should support innovative projects and research against the deadliest cancers. In a recent commentary in Politico, Zetter, a professor of cancer biology at Harvard Medical School and Boston Children’s Hospital, notes that researchers follow the money and that the cancers with which we have made the least progress still seem to get the least amount of funding. We spoke to Zetter about his commentary, the opportunities being created by the Cancer Moonshot, and how he thinks cancer funding needs to change.

For people with type I diabetes, the day-to-day management of the disease can be a full-time job involving the monitoring blood glucose levels and injection of insulin. The medical device giant Medtronic has been working toward the development of what it’s called an artificial pancreas, a steady improvement of pump and sensor technology to free type I diabetes patients and allow them to think less about the management of their disease. We spoke to Fran Kaufman, chief medical officer of Medtronic Diabetes, about the progress the company has made, its newest technology that is moving towards the market, and how close it is to realizing its vision of an artificial pancreas.

By using data from routine testing about the genetic evolution of the HIV /AIDS virus in the Canadian province of British Columbia, researchers have shown they are able to identify emerging outbreaks of the disease in near real-time and intervene to control its spread. The first-of-its-kind system is the focus of a study in an April edition of The Lancet HIV. We spoke to Art Poon, Associate Research Scientist for the British Columbia Centre for Excellence in HIV/AIDS and lead author of the study, about the monitoring system, the impact it has had, and whether such an approach is transferable to other regions and other disease outbreaks.

As pricing pressure grow on the pharmaceutical industry, a recent article in Invivo argues that drugmakers must look to a variety of new pricing models built around collaborations with payers and providers. Such an approach, the authors argue, would minimize conflicts between stakeholders, close the information gap about the real-world value of new drugs, and allow for a more evidence-based approach to pricing. We spoke to Susan Garfield, a principal in EY's Life Sciences Advisory Services practice and co-author of the Invivo article, about the need for new approaches to pricing, why the existing unit price approach is too one-dimensional, and what’s at stake for the industry if it fails to innovate new models.

The France-based biotech Gensight Biologics is developing gene therapy treatments for rare, mitochondrial, and neurodegenerative diseases of the eye. Its lead therapeutic candidate is in late-stage trials for Leber’s Hereditary optic neurophathy, a sudden condition that afflicts young adults and leaves almost all of them blind. We spoke to Gensight’s CEO Bernard Gilly about the company, the challenges and promises of gene therapy, and why eye disease is a particularly attractive target for this emerging area of treatment.

Nearly $3 billion a year is wasted on cancer drugs because of the way they are packaged, according to a study in BMJ. These expensive drugs that are injected or infused come in fixed size vials and patients often require less drug than what is sold as a single dose. Because of restrictions on the ability to use leftover drug, the result is that it is usually thrown out. Nevertheless, drugmakers have little incentive to do things differently because the waste enhances their profitability. We spoke to Leonard Saltz, an oncologist with Sloan Kettering Memorial Cancer Center and coauthor of the study, about the findings, why Europe doesn’t have the same problem, and what can be done to stop the waste.

The walking dead live, but it's value, not brains, they are eating. So-called zombie funds, life sciences venture capital funds that are fully invested and unable to raise new money, still maintain their board seats. But Outcome Capital’s Managing Directors Oded Ben-Joseph and Arnie Freeman say their divergent interest from their fellow board members often lead to disputes that can end the life of promising technologies and lead to acquisitions that leave much value on the table. We spoke to Ben-Joseph and Freeman about the boardroom dynamics were zombies lurk, the consequence of this board misalignment, and how acquirers may seek to leverage the situation

Last month, the Senate confirmed Robert Califf, a cardiologist and clinical researcher from Duke University, as commissioner of the U.S. Food and Drug Administration. Though he was overwhelming confirmed, some expressed concerns about his ties to the pharmaceutical industry. Others used the confirmation process to protest the FDA’s handling of opioid painkillers as the problem of addiction to these drugs have become a growing concern. We spoke to Peter Pitts, a former FDA associate commissioner and president of the Center for Medicine in the Public Interest, about Califf, why he’s well suited for the job, and the challenges he will face in his new role.

Bird Rock Bio, a small, San Diego-based biotech, is planning to take aim at some of the biggest biologics on the market with an antibody in development to treat rheumatoid arthritis that it says it expects to market at an annual cost of just $2,000 a year. That compares to around $30,000 a year for drugs such as Humira, Remicade, and Enbrel that are TNF inhibitors. The experimental therapeutic known as Gerilimzumab targets IL-6. We spoke to Paul Grayson, president and CEO of Bird Rock Bio about the company, the benefits of targeting IL-6, and how the company expects to be able to offer the drug at such a disruptive price point.

The embattled pharma executive Martin Shkreli, whether intentional or not, has ignited a long smoldering public policy discussion about the pricing of pharmaceuticals. As there are growing calls for steps to restrain drug prices, there are also industry concerns about the effect such actions would have on the ability to attract investment and pursue the development of high-risk, innovative therapies. We spoke to Denis Corin, CEO of Q BioMed, an acceleration and development company, for a perspective on the fallout from Shkreli, how to balance innovation with affordability, and whether he expects any lasting impacts on small drug developers.

The California Institute for Regenerative Medicine, California’s voter-established stem cell agency, is thinking about its future. The institute recently issued a strategic plan for the next five years that it says represents a “radical overhaul of the way the institute conducts business” and emphasizes “coordination, speed, partnerships, and patients.” We spoke to Randy Mills, president of the California Institute for Regenerative Medicine, about the strategic plan, the ambitious goal it has set for the next five years, and whether CIRM will have a life beyond the $3 billion voters approved for its funding.

About 1.3 million people in the United States have type 1 diabetes, which requires constant monitoring and regular injections of insulin. The autoimmune disease attacks the insulin producing beta cells in the pancreas and replacement of those cells has long been viewed as an ideal approach to treating the disease. The problem, though, has been finding a reliable supply or replacement cells and protecting them from the body’s immune system while allowing them to recieve needed nutrients, as well as deliver insulin to the body. San Diego-based ViaCyte is now in human clinical trials with a promising approach to treating the disease. We spoke to Paul Laikind, president and CEO of ViaCyte, about the company’s cell therapy, its critical financial support from the California Institute for Regenerative Medicine and JDRF, and why its investigational product may represent a functional cure.

Non-steroidal anti-inflammatory drugs are the most widely used medications in the world. They are used to treat pain resulting from diseases such as arthritis, but these drugs can raise blood pressure and cause serious problems including heart attacks, strokes, and death. We spoke to Paul Waymack, founder, chairman, and chief medical officer of Kitov Pharmaceuticals about its efforts to bring to market a combination therapy that addresses these safety concerns. Waymack discussed the issues surrounding pain medications, his company’s approach, and how the company’s smart FDA strategy is significantly cutting its cost and time to market.

The American Association for Cancer Research, with seven leading cancer research centers, has launched Project GENIE to determine how to better tailor treatments to patients’ individual cancers. The effort will take tumor genomic profiling data from the various institutions and aggregate it into a single database for researchers to harness. We spoke to Barrett Rollins, chief scientific officer at the Dana-Farber Cancer Institute, about the project, how far down the path of precision medicine we have travelled, and how he expects it to reshape cancer care in the years to come.

As medical device makers are building network connectivity and intelligence into their products, they are adding new vulnerabilities and risks as well. The U.S. Food and Drug Administration has just issued draft guidance for postmarket management of cybersecurity of medical devices. It follows previous guidance on premarket submissions for management of cybersecurity in medical devices. We spoke to Russell Jones, partner with Deloitte & Touche, cyber risk leader for the firm’s State of California Practice and co-leader of the firm’s medical device safety and security practice, about the FDA actions, how these connected devices can be exploited by hackers, and what medical device makers can learn from industries that have already faced these issues.

The pharmaceutical industry appears to have lost ground in 2015 on the ongoing debate around drug pricing, a problem that Christopher Bowe sees in part as a failure of leadership within the industry. In a recent guest column in Forbes, Bowe, who advises industry CEOs on communicating their strategies and ideas, argued that the industry needs CEOs capable of reframing the discussion with fresh ideas, forging new alliances, and disrupting existing business models. We spoke to Bowe about this leadership vacuum, why it is critical for the industry to move beyond its tired arguments, and what it needs to do to rebuild trust and credibility with the public.

As 2015 came to a close, Congress passed a package of tax extenders that among other things expanded the Research and Development Tax Credit and made it permanent. The move represents a big win for innovation-based industries, but the life sciences in particular, which will benefit from a new provision that allows companies to capitalize on the credit ahead of producing revenue. We spoke to Dan Mennel, California Market Leader of Strategic Federal Tax Services for Grant Thorton and Matt Gardner, CEO of the California Technology Council, about the R&D tax credit, what it does, and what it means for the life sciences.

The year 2015 was another big one for biotech with record dealmaking, big drug approvals, and solid performance, but it somehow didn’t feel as good as it looks on paper. Pricing concerns that garnered big attention are expected to grow in intensity in the new year with the presidential election and negotiations for the renewal of the Prescription Drug User Fee Act. We spoke to Adam Feuerstein, senior columnist for TheStreet.com, about the year in biotech, the highs and lows, and what’s ahead in the new year.

Growing costs pressures, the integration of technology, and the transformation of the patient into a healthcare consumer is giving rise to a new health economy. In its report on the top health industry issue for 2016, PwC highlights the forces expected to have the most impact on the industry in the coming year. We spoke to Karla Anderson, principal of U.S. pharmaceuticals and life sciences for PwC, about the report, how an increasing emphasis on value is reshaping the sector, and what’s in store for 2016.

Despite a record number of new drug approvals this year, the return on R&D investment for the largest pharmaceutical companies continues to fall, according to a new report from the Deloitte Centre for Health Solutions and Deloitte’s R&D services group. In fact, the report finds R&D returns for this group of companies have fallen to their lowest point since Deloitte began tracking them in 2010. We spoke to Neil Lesser, principal with Deloitte Consulting in the Life Sciences strategy practice and a leader in the Research & Development strategy practice, about the report, the pressures on the industry that are hurting returns, and what R&D strategies companies can pursue that might reverse the trend.

Retail prices for more than 100 widely used specialty drugs rose nearly 11 percent in 2013, according to a new report from AARP Public Policy Institute. The report found that the average annual cost of a specialty drugs used to treat chronic diseases rose to more than $53,000 — greater than the U.S. median income and more than twice the $23,500 median income of people on Medicare. We spoke to Leigh Purvis, director of health services research in AARP’s Public Policy Institute about the report, trends that are fueling demand for these drugs, and whether there are public policy solutions to rein in rising drug prices that can be implemented without harm to innovation.

A team of biohackers is developing the first open source protocol to produce insulin simply and economically. The hope is that their work will serve as the basis for generic production of insulin and provide a foundation for continued research into improved versions of the life saving biologic. We spoke to Anthony Di Franco, co-founder of the Open Insulin project and a board member of Counter Culture Labs, about the work, the challenges they are encountering, and whether the DIY movement can teach the corporate world anything about cost-effective innovation.

A large number of clinical trials underlying the approval of drugs never come into public view. This not only has legal and ethical ramifications, but implications for the healthcare system as a whole. We spoke to Jennifer Miller, founding president of Bioethics International and assistant professor at the NYU School of Medicine about her recent study in BMJ Open, part of an effort to improve transparency through the creation of a Good Pharma Scorecard. Miller discussed the study, the scorecard, and the state of transparency in the pharmaceutical industry.

The power of genetic engineering will soon be available for the home thanks to the Amino One, a piece of hardware about the size of a laptop computer that would allow users to manipulate the DNA of microorganisms for productive purposes or just plain fun. We spoke to Julie Legault, founder and CEO of Amino One’s creator Amino Labs, about the device, its target market, and the implications of putting the tools of synthetic biology into the hands of kids.