The Bio Report

When David Fajgenbaum was in medical school, he became stricken with a rare autoimmune disease that nearly killed him. Though he recovered, he would suffer recurring, life-threatening flares only to discover the poor state of research into condition. Fajgenbaum chronicles his rare disease journey and his efforts to drive research and find treatments in his new book “Chasing My Cure: A Doctor’s Race to Turn Hope into Action.” Fajgenbaum, who is executive director of the Castleman Disease Collaborative Network, will be featured at this year’s Global Genes Rare Patient Advocacy Summit, which begins in San Diego September 18. Ahead of the summit, we spoke to him about his experience as a rare disease patient, his efforts to find treatments for his conditions, and how his innovative approach to developing a patient-driven research agenda has provided a roadmap for other rare disease organization to follow suit.

Among the many challenges to developing stem cell therapies is the need to match donors to recipients and the risk of graft vs. host disease that requires the use of immunosuppressants. Athersys has developed a stem cell therapy platform known as MultiStem that makes use of a type of stem cell that doesn’t carry the risk of causing an immune response. What’s more, cells from a single donor can be expanded to provide potentially millions of doses to treat a wide range of conditions making it a scalable, off-the-shelf therapy. The company’s is in late-stage clinical testing of its cell therapy for the treatment of ischemic stroke, a leading cause of serious disability. We spoke to Gil Van Bokkelen, CEO of Athersys, about the company’s MultiStem platform, how it works, and the range of indications it is pursuing.

Immunotherapies have mostly sought to harness the adaptive immune system, but Silverback Therapeutics believes its technology platform will allow it to enlist the innate immune system in the fight against cancer, fibrosis, and infectious diseases. The company is developing antibody conjugates that activate myeloid cells to target tumors. It’s lead indication is in HER-2 positive tumors. We spoke to Peter Thompson, co-founder and CEO of Silverback, about the company’s platform technology, its potential applications, and the challenges of activating the innate immune system in a targeted fashion.

Artificial intelligence is all the rage in drug discovery today, but there are efforts to apply to technology to other aspects of the drug development continuum to improve decision making and increase efficiency. One such example is Phesi, which has developed an artificial intelligence platform to improve clinical site selection, evaluate protocol designs and patient inclusion criteria, and manage the implementation of trials. We spoke to Gen Li, founder and president of Phesi, about his company’s AI platform, how it works, and the case for the use of artificial intelligence as a tool to improve drug development.

Antibody-drug conjugates marry the precise targeting of an antibody to a cytotoxic payload. That has the potential to provide a powerful treatment approach to a variety of cancers with less toxicity than systemically delivered chemotherapy. The problem has been producing large quantities of antibody-drug conjugates in a consistent manner where all the pieces are where they belong. Sutro Biopharma has developed a technology platform that allows it to precisely design and consistently manufacture these molecules. We spoke to Bill Newell, CEO of Sutro, about the promise of antibody-drug conjugates, the company’s platform technology, and its lead therapeutic candidates.

While much of the focus of drug developers looking to act on the genetic mechanisms underlying diseases has focused on the genes that code for proteins, Syros Pharmaceuticals is looking to the regulatory regions of the genome. This is the noncoding part of the genome that controls the expression of genes, turning them on or off, or modulating their level of activity. We spoke to Eric Olson, chief scientific officer of Syros Pharmaceuticals, about the company’s discovery platform, its pursuit of cancer and monogenic diseases, and the lead programs in its pipeline.

Anixa Biosciences is harnessing the immune system to both diagnose and treat cancer. It recently announced a licensing agreement with the Cleveland clinic for a novel breast cancer vaccine that is being developed for both therapeutic and prophylactic use. It’s lead candidate is a CAR-T cell therapy in development for ovarian and other solid tumors. We spoke to Amit Kumar, president and CEO of Anixa, about the company’s strategy to pursue both therapeutic and diagnostic products, its partnerships with leading research institutes, and its development strategy going forward.

There’s growing interest in cannabinoids for their therapeutic potential in a range of conditions, but one challenge therapeutic developers face is a reliable and consistent supply. Teewinot Life Sciences is using synthetic biology to produce a range of cannabinoids. We spoke to Michael Luther, president and CEO of Teewinot Life Sciences, about the companies process for producing cannabinoids, its business model, and the therapeutic and consumer areas it plans to pursue.

Much has been made about a cancer moonshot, but in the world of oncology, rocket fuel is rarely a topic of conversation except maybe as a metaphor. EpicentRx, though, is looking to rocket fuel as a source for its lead experimental therapy RRx-001. It believes this high-energy molecule, derived from rocket fuel, has the potential to improve immunotherapies and radiotherapies, as well as having anticancer activity on its own. We spoke to Corey Carter, president and CEO of EpicentRx about how RRx-001 came about, how it works, and the company’s oncolytic viral pipeline also in development.

The complexity of individual tumors creates a challenge for physicians to determine the optimal treatment for any given patient. This complexity may also require a combination of therapies to target the multiple pathways a given cancer may exploit to grow and spread. My Personalized Therapeutics is taking a model systems approach to pair patients to customized drug regimens by using fruit fly models of a patient’s cancer to screen hundreds of FDA approved therapies and combinations. We spoke to Laura Towart, co-founder and CEO of My Personalized Therapeutics, about the company’s Personalized Discovery Process, how it works, and why it may lead to better outcomes for cancer patients.

As biomedical data proliferates and the life sciences becomes more closely integrated with data science, the ability for researchers to access, use, and learn from the growing universe of information requires new tools. InveniAI is seeking to address this need with its artificial intelligence platform for biopharmaceutical companies to both monitor innovation and to enhance their own drug development capabilities. We spoke to Krishnan Nandabalan, president and CEO of InveniAI, about the company, how its artificial intelligence platform is being used, and what he expects the ultimate payoff from this technology will be.

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 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.

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.

Josiah Zayner calls himself a biohacker. He’s got a Ph.D. in molecular biophysics from the University of Chicago and worked as a fellow in NASAs synthetic biology program. But it is Zayner’s evangelism for democratizing the tools of biotechnology, his flair for attention-grabbing self-experimentation, and efforts to share the knowledge and equipment necessary to perform procedures like gene editing that have given him some notoriety. As founder and CEO of The ODIN, Zayner is helping move biotechnology from the labs of universities and biopharmaceutical companies to high schools and garages. We spoke to Zayner about his efforts, how biotechnology is becoming accessible to laypeople, and how he sees this fueling innovation.

One-third of adults suffer from high blood pressure and 9.4 million people a year will die from complications relating to the condition. In fact, cardiovascular disease remains the leading cause of death worldwide. Quantum Genomics is developing a new class of drugs that target an enzyme in the brain for the treatment of high blood pressure and the prevention of related cardio-vascular risks. We spoke to J.P. Milon, CEO of Quantum Genomics, about its platform technology, how it works, and why it may have promise as an approach to treating both hypertension and heart failure.

Atrial fibrillation, a condition characterized by an irregular and rapid heartbeat, affects up to 6.1 million Americans and can cause strokes. It is not well addressed by current medical approaches. Chris Larson, an adjunct associate professor at Sanford Burnham Prebys Medical Discovery Institute is part of a team working to find new drugs to treat the condition by identifying every gene in the human genome that can affect the rhythm of the heart. We spoke to Larson about the condition, SBP’s drug discovery efforts, and why he left industry to work on drug discovery at a non-profit institute.

Current therapies for autoimmune diseases represent a $100 billion market, but available drugs often provide limited benefits to patients or cause problematic side effects. Landos Biopharma is targeting the area of autoimmune diseases with an initial focus on inflammatory bowel disease by pursuing therapies that target a novel pathway. We spoke to Landos founder and CEO Josep Bassaganya-Riera, about IBD, why new therapies are needed, and the approach Landos is taking.

The ability to print human tissue is changing the way drug research is conducted today and opens the possibility of one-day printing replacement organs with the technology. CellInk saw an opportunity in making bioinks and started to produce a line of lower-priced printers as well in the hopes of expanding the marketing by lowering the cost of the technology. We spoke to Erik Gatenholm, CEO and co-founder of CellInk, about the technology, how its being used today, and how he expects applications for it to evolve.

Bing Wang trained as an electrical engineer, but his experience as a cancer survivor led him into the world of healthcare investment banking and then to his current role as CEO of the cancer therapeutics company Refuge Biotechnologies. Refuge is leveraging CRISPR technology, but rather than editing genes, the company is using it as a way to activate or inhibit specific genes by harnessing it for its targeting mechanism. We spoke to Wang about his own journey from patient to CEO, the company’s technology, and why he believes this will lead to safer and more effective cancer therapies.

As the year draws to a close, we continue what’s become an annual tradition and look back on the year in biotech and ahead to the J.P. Morgan Healthcare Conference and beyond with Adam Feuerstein, senior biotech writer for STAT News. Adam tries to make sense of a puzzling year for biotech that seemed full of contradictions, offers some thoughts on the highs and lows of the past 12 months, and discusses what he’ll be watching for during the J.P. Morgan Healthcare Conference.

The ability to manipulate and understand biology at the molecular level coupled with powerful computing capabilities is opening up new possibilities to harness technology to improve human health. The OS Fund is focused on investing in entrepreneurs developing platform technologies that have the ability to address global problems. We spoke to Bryan Johnson, co-founder of the OS Fund, about his investment vision, what it takes to attract his eye, and how he sees these platform technologies reshaping the world healthcare and beyond.

Paul Keckley is a long-time healthcare policy analyst whose both worked within hospital systems and advised major healthcare organizations, but when he recently collapsed on a golf course due to severe dehydration, he was given an up-close and personal view of the murky world of hospital billing. We spoke to Keckley about his experience, the challenges of deciphering hospital bills, and why he thinks it will be market forces, rather than price transparency, that rein in healthcare costs.

Matt State is known for his work trying to understand the biology underlying psychiatric illnesses, but as the chairman of the University of California, San Francisco’s Department of Psychiatry, part of his job has been to help address the city’s homelessness crisis. Homelessness is a complex issue, but the role mental health plays in the problem is often overlooked. With about 10,000 people who are homeless in San Francisco, more than a third of these people have a psychiatric or substance abuse problem. We spoke to State about the connection between homelessness and mental health issues, UCSF’s efforts in this area, and how it fits with the mission of a leading public institution.

Gecko Biomedical’s first product Setalum is a sealant that prevents bleeding after the use of sutures to repair blood vessels, but the polymers underlying the product represent a platform technology that can be used to develop a broad array of tissue reconstruction offerings addressing bone, nerve, ophthalmic, and urological applications. We spoke to Christophe Bancel, CEO of Gecko, about the company’s platform technology, its versatility, and how the company is looking to partnering to expand its reach.

In 1988, researchers showed that they could extend the lifespan of worms by manipulating the genetics of these model organisms. The implications that the normal aging process could be altered ignited scientific research into the emerging area of geroscience. As the Buck Institute for Research on Aging readies to mark the anniversary of this landmark research with a day-long celebration, we spoke to Gordon Lithgow, chief academic officer of the Buck. Lithgow discussed how the field has advanced from that key discovery, our understanding of aging today, and what progress has been made to identify compounds that could extend healthy years of life.

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.