The Bio Report

Type 2 diabetes affects nearly 500 million people worldwide and more than 34 million people in the United States. The disease carries an elevated risks of heart attack, stroke, and other serious complications. Fractyl Health believes the approach to treating diabetes by controlling blood sugar levels and other symptoms has been faulty. Research has implicated a critical role the first section of intestine, known as the duodenum, plays in the condition and the company has developed a minimally invasive endoscopic procedure that it believes can correct the problem. We spoke to Harith Rajagopalan, CEO of Fractyl Health, about the company’s experimental procedural therapy to target the root cause of diabetes, how it works, and the path forward to commercialization.

The efforts to develop drugs for neurologic and psychiatric conditions has been plagued with failure. Herophilus is combining organoids derived from patient stem cells along with machine learning to gain new insight into the biology underlying these diseases and discover and develop more effective drugs. We spoke to Saul Kato, co-founder and CEO of Herophilus, about the drug development challenges for these diseases, how his company is using organoids and machine learning to better understand how to target them, and its growing pipeline therapeutic candidates.

While the class of immunotherapies known as checkpoint inhibitors has brought a promising new approach to treating cancer, the development of resistance to these therapies limits the number of patients they benefit. Portage Biotech is building a set of platform technologies and a pipeline of immuno-oncology therapeutic candidates to address this problem. We spoke to Ian Walters, CEO of Portage about the problem of resistance, the company’s pipeline of candidates, and its portfolio-based business model.

Earth will not live forever. Whether its destruction comes as the result of cosmic inevitability or human-caused cataclysm, the planet will eventually meet its demise. Though that may be billions of years from now, Chris Mason in his book “The Next 500 Years,” argues humans have a more obligation to do what they can ensure life from Earth can extend beyond the planet. Mason, principal investigator on NASA’s twins study and a professor at Weill Cornell Medicine, discussed the effects of space on the human body, how it may be possible to genetically engineer human and other life forms to thrive in environments less hospitable than Earth through the use of new genetic tools, and the ethical considerations around these issues.

While great strides have been made the fight against cancer with new therapies, dose-limiting toxicities of these agents can have a big impact on the quality of life for patients and lead doctors to alter dosing at the price of optimal outcomes. Rather than treating the symptoms of the side effects of cancer drugs, OnQuality Pharmaceuticals is developing a pipeline of therapies that target the biologic pathways that at their source. It is developing targeted supportive therapies to treat such things as dermatologic conditions and diarrhea caused by cancer medicines. We spoke to Michael McCullar, CEO of OnQuality, about the need his company is addressing, its pipeline, and how he expects others to think about the value of the therapies the company is developing.

While great progress has been made in understanding the human genome, its functional counterpart, the human proteome, remains relatively unexplored. In part, that’s because advances in the tools to identify the far bigger universe of protein variants has been lacking. Seer Bio has developed a platform for large-scale proteomics studies that it says is both unbiased and scalable. We spoke to Omid Farokhzad, chair and CEO of Seer Bio, about the state of proteomics, How Seer’s technology works, and why it can help advance our understanding of health and disease.

The development of mRNA therapies is allowing drug developer to pursue targets once considered undruggable. Anima Biotech has developed a platform that allows it to use small molecule that selectively control mRNA and can decrease or increase the translation of proteins. The approach has broad applicability as evidenced by the company’s pipeline, which includes experimental therapies for oncology, neurology, infectious disease, and fibrosis. We spoke to Yochi Slonim, CEO of Anima, about the company platform technology, how it can modulate the expression of proteins, and how it is leveraging its technology through partnerships with some of the world’s largest pharmaceutical companies.

The biopharmaceutical industry has long struggled with R&D productivity. Long time industry strategic consultant Mike Rea, founder and CEO IDEA Pharma, thinks he’s hit on a possible solution. Taking a cue from the tech industry, Rea in May announced the launch of Protodigm, which he describes as a contract skunkworks company. The approach is intended to allow a multi-disciplinary team work autonomously to take its clients early-stage assets and explore multiple development options at once with the intent of de-risking innovation while saving time and money. We spoke to Rea about the industry’s challenges with R&D productivity, the benefits Protodigm’s skunkworks approach could provide, and how it will work with industry.

While cities across the country have lifted COVID-19 restrictions and public gatherings have returned, the lack of effective treatments for people who develop severe reactions to the virus continues to pose a public health problem. The situation is acute in less developed nations where vaccination levels are low, and hospitals continue to be overrun with patients. Researchers at Mount Sinai earlier this year reported in the journal Cell that a widely available and inexpensive drug targeting inflammatory genes has reduced morbidity and mortality in mice infected with the virus that causes COVID-19. The drug, topotecan, is now in clinical trials in India as a potential treatment for patients who suffer from a hyper-inflammatory response to the virus. We spoke to Ivan Marazzi, senior author of the study and an associate professor of microbiology at the Icahn School of Medicine at Mount Sinai, about topotecan, what makes it compelling as a potential treatment for COVID-19, and why it may be well suited for treati

Osteoarthritis is a painful, progressive disease of the joints for which there is no cure. The condition affects more than 32.5 million adults in the United State and the incidence is growing as a result of aging, obesity, and sports injuries. Flexion Therapeutics is developing an experimental gene therapy to treat the condition. Rather than correcting an underlying genetic mutation, the experimental therapy delivered into the joint codes for the production of the anti-inflammatory protein interleukin-1 receptor antagonist. It is expected to deliver as-needed anti-inflammatory activity to joint tissues over the long-term. We spoke to Mike Clayman, co-founder and CEO of Flexion Therapeutics, about osteoarthritis, the problem with existing therapeutic approaches, and the company’s efforts to develop a gene therapy to get the body to produce an anti-inflammatory protein as needed.

Sumitomo Dainippon Pharma’s $3 billion strategic transaction with Roivant Sciences that gave rise to Sumitovant, represents an effort by the Japanese drug company to make the drug development process faster and more efficient. A critical piece of this effort is steps the company has taken to harness information technology to capitalize on existing knowledge to inform decision making. We spoke to Bill McMahon, chief algorithmic analytics officer of Sumitovant, about the approach Sumitovant is taking, the way uses information technology to improve the drug development process, and how the subsidiary may be influencing the way its parent company operates.

The ability to identify diseases at their earliest stages through the detection of minute levels of biomarkers offers the potential to make interventions before the outward physical manifestations of illnesses can bloom and long-term damage can occur. Quanterix is developing a platform of ultra-sensitive biomarker tests to give researchers greater insight into the transition from health to disease and the ability to detect diseases at an earlier point in their development. We spoke to Kevin Hrusovsky, chairman, president, and CEO of Quanterix, about the company’s ability to detect minute amounts of biomarkers, the implications this has for drug development and diagnostics, and how this can help drive a future of precision health.

The marriage of synthetic biology to mRNA is creating the potential for a range of programmable therapeutics that can provide new ways of treating deadly and chronic diseases. Strand Therapeutics is creating a platform for developing these long-acting mRNA drugs that it says can be precise, multi-functional, and deliver potentially curative treatments with a single dose. We spoke to Jake Becraft, co-founder and CEO of Strand, about the company’s programmable mRNA therapeutic platform, how it works, and the indications it is pursuing.

Cardiometabolic conditions, such as type 2 diabetes and heart disease, have long been treated with pharmacologic interventions. Better Therapeutics believes cognitive behavioral therapy delivered through its prescription digital therapeutics platform, will provide better outcomes at a lower cost. We spoke to David Perry, co-founder and executive chairman of Better Therapeutics, about the company’s prescription digital therapeutics platform, the case for personalized cognitive behavior therapy delivered this way, and what it will take to get doctor to prescribe it and payers to reimburse for it.

The side effects of opioids and the potential for addiction underlies the need for new approaches to the treatment of pain. SiteOne therapeutics is developing a pipeline of therapies that target sodium ion channels to block the electric signals sent from the site of pain. Unlike other similar drugs, SiteOne is able to target these sodium channels with great specificity to avoid the types of side effect that have limited the value of drugs that target these proteins. We spoke to John Mulcahy, president and CEO of SiteOne, about the company’s approach to pain, its pipeline, and how its therapies are able to hit their targets with precision. Special offer to listeners of The Bio Report: With the Digital Library from DeepDyve, you can search 100 million scientific papers with full access to 20 million articles, annotate them, and share with colleagues. It’s one-stop, affordable research. Try one month of DeepDyve’s enterprise service for free. Go to deepdyve.com/podcast and use the code BIOREPORT.

Earlier this year NovelluxDx rebranded itself as Fore Biotherapeutics signaling a shift from being a diagnostics company to its new life as a precision cancer therapeutics drug developer. Building on its functional genomics platform, the company is in-licensing small molecule drug candidates it sees having potential to treat hyper-targeted patient populations. We spoke to Usama Malik, CEO of Fore Biotherapeutics, about the move from diagnostics to drug development, the approach Fore is taking, and why it thinks it can see value in molecules that others may miss.

When variants of the COVID-19 virus emerged in late 2020, it raised concerns about whether people already infected with the original virus, or vaccinated against it, would be able to generate a protective immune response that would confer protection against these new strains. Researchers at the National Institutes of Health’s National Institute of Allergy and Infectious Diseases and Johns Hopkins University recently reported encouraging findings from a study that took a detailed look at the antibodies in the blood from COVID-19 patients. To do this, they used a deep immunomics technology platform developed by ImmunoScape. We spoke to Brian Abel, senior director of business development at ImmunoScape and lead of the company’s COVID-19 Vaccine Task Force, about the COVID-19 study, the company’s technology, and how it is being used to develop the next generation of immunotherapies.

Hypoxia, a potentially fatal shortage of oxygen in tissues, is a complicating factor in a variety of serious illnesses including cancer, cardiovascular disease, and respiratory conditions. Diffusion Pharmaceuticals lead experimental candidate, TSC, enhances the ability of the body to deliver oxygen to where it is needed most. We spoke to Bob Cobuzzi, CEO of Diffusion Pharmaceuticals, about hypoxia, the company’s lead therapeutic candidate to address it in a broad range of conditions, and its clinical development strategy for the drug.

Wet age-related macular degeneration is a progressive disease and a leading cause of vision loss in patients over the age of 60. Vision loss is caused by the leakage of blood and other fluid from abnormal blood vessel growth underneath and into the retina. Though the condition is treated by a class of therapies known as VEGF inhibitors, these biologics need to be repeatedly injected into patients’ eyes and for a variety of reasons patients’ real-world experience with the drugs don’t match clinical trial outcomes. Adverum Biotechnologies is developing a gene therapy to treat the condition. Rather than addressing an underlying genetic cause though, the gene therapy carries a sequence that causes the eye to produce aflibercept, the VEGF inhibitor marketed as Eyelea and Zaltrap. We spoke to Laurent Fischer, CEO of Adverum, about the company’s gene therapy, how it works, and why the one-and-done approach could translate into better results for patients.

TNF inhibitors, such as Humira and Remicade, have been a great biotech success story. These multi-billion franchises treat a range of autoimmune conditions including rheumatoid arthritis, psoriasis, and Crohn’s disease. Jim Woody, who led Centocor’s development team for Remicade, the first of the TNF-inhibitors, is today in pursuit of new uses for these therapies. Now CEO of 180 Life Sciences, Woody and his team are pushing TNF inhibitors into new indications for inflammatory and fibrotic conditions. We spoke to Woody about the role of TNF in the inflammatory process, the indications his company is pursuing, and why these well-established drugs have gone untapped for these purposes. The Bio Report welcomes DeepDyve as a sponsor. Search 100 million scientific papers with full access to 20 million articles. It’s one-stop, affordable research. Try the enterprise version of the service free for one month. Go to deepdyve.com/podcast and use the code BIOREPORT