The Bio Report

Sam Gambhir understood the ravages of cancer. His wife developed breast cancer and survived. His son later developed brain cancer at the age 15 and died. And in 2020, Gambhir himself succumbed to cancer. Before he died, though, Gambhir, who served as division chief of the Canary Center for Early Cancer Detection and Molecular Imaging at Stanford University, hit upon an idea. Rather than hunt for cancers in the hopes of making an early diagnosis, he devised a way for them to produce synthetic biomarkers to cause them to reveal themselves. He co-founded Earli, which seeks to enable the diagnosis cancers when they are most treatable. We spoke to David Suhy, co-founder and chief scientific officer of Earli, about the company’s synthetic biomarker technology that makes cancers visible with a PET scan, how it works, and how this has the potential to alter outcomes for patients by enabling treatments of patients before their disease progresses.

Finding diverse groups of qualified participants for clinical studies can slow the development of needed medicines. The difficulty in attracting racial and ethnic minorities, women, and the elderly also creates concerns that trial results will fail to reflect what would happen in the real world. Some 80 percent of clinical trials fail to meet enrollment deadlines and an average of 30 percent of participants drop out in part because of the location and duration of these studies. CVS Health in 2021 launched CVS Clinical Trial Services, which capitalizes on CVS’ rich database of patients and its large footprint that puts a CVS location within 10 miles of 85 percent of the U.S. population. We spoke to Owen Garrick, chief medical officer of Clinical Trial Services at CVS Health, about the company’s push into clinical trials, the need to expand the diversity of participants in these studies, and how CVS is leveraging its resources to increase access to and participation in clinical trials.

The microbial world has been a rich source of medicines, but our ability to explore the full potential of the microbes both in us and around us has been limited by technology and the difficulty of culturing most microbes in a lab. Biosortia Microbiomics has developed a platform for finding, amplifying, and screening microbes as a potential source of novel, small molecule drugs. We spoke to Ross Youngs, CEO and founder of Biosortia, about the case for exploring various microbiomes to discover new small molecule drugs, how the company’s platform technology enables it to investigate a much broader range of microbes, and its business model for capitalizing on the discoveries it makes.

One of the underlying limitations of most cancer therapies is the fact that only about 1 to 2 percent of the drug that’s administered ever reaches tumors. That limits the dose because of the potential for unwanted side effects, and limits the efficacy by delivering less than ideal amounts of drug to tumors. Shasqi is developing cancer therapies that use click chemistry to activate agents at the site of tumors to reduce systemic toxicity and increase the anti-tumor activity of its medicines. We spoke to José Mejía Oneto, founder and CEO of Shasqi, about the company platform technology, how it works, and why this can lead to safer and more powerful cancer therapies.

The gut is the body’s largest sensory organ with a surface area 100 times that of the skin. It contains more nerve cells than the spinal cord, most of the immune system, and 95 percent of the body’s serotonin. Kallyope has assembled a discovery and translation platform to developed therapies to treat metabolic, gastrointestinal, and neurologic conditions by targeting the gut-brain connection. We spoke to Nancy Thornberry, chair of R&D for Kallyope, about the gut-brain connection, the company’s platform technology, and the case for targeting these interactions to treat a wide range of conditions.

Providers hold vast amounts of health data that can be harnessed to gain better insights into diseases, improve outcomes for patients, and help bring about an era of precision medicine. Despite advances in artificial intelligence to capitalize on these stores of data, much of it has remained siloed and out of the reach of researchers and drug developers who could put it to use. Last year, a group of 14 health providers caring for tens of millions of patients through thousands of care facilities across the country formed Truveta, a company that has built an AI platform to make structured and de-identified patient data available for research. We spoke to Terry Myerson, CEO of Truveta, about its platform technology, the work it is enabling today, and how AI and the availability of real-world data is changing how biomedical research is conducted.

There’s been growing interest in exploring the potential of psychedelics to treat neuropsychiatric conditions. Onsero Therapeutics is part of a growing list of companies that are developing therapies that target the 5-HT2A serotonin receptor, the receptor to which several of psychedelics bind, and one that has been clinically validated in a number of psychiatric conditions. The company believes the therapies it’s developing have the potential to provide the treatment benefits of these substances without their better-known effects. We spoke to Tim Piser, chief scientific officer of Onsero, about the case for these drugs to treat conditions like mood disorder, how it’s developing its experimental therapies so they don’t induce effects, and why he believes benefits of these drugs can exist independently of them packing a psychedelic punch.

When people think of the potential for artificial intelligence to impact the biopharmaceutical industry, the focus is usually on drug discovery or in the clinic to help guide decision making with regards to pairing the right drug to the right patient. Yseop is focused on a more mundane use for the technology—the writing of clinical data reports. The company had been working in the financial services industry when it was approached by Sanofi to see if it could apply its natural language generation to make the creation of these reports faster and less expensive. We spoke to Emmanuel Walckenaer, CEO of Yseop, about artificial intelligence, its potential to change the way biopharmaceutical companies work in non-obvious ways, and why a large number of biopharmaceutical companies are turning AI to do more with less.

ProFound Therapeutics has developed platform technology to catalogue and understand previously undetected human proteins and the role they play in health and disease. By focusing on proteins in the process of translation, the company said it has identified tens of thousands of undiscovered proteins that can provide untapped therapeutic targets. We spoke to Avak Kahvejian, co-founder and CEO of ProFound Therapeutics, about the company’s platform technology, the vastness of the unexplored proteome it seeks to discover, and how this can lead to the development of breakthrough medicines.

It was a difficult year for biotechs with drops in the major biotech indices, fewer new drug approvals, and more than 125 companies announcing layoffs. But there were also triumphs to celebrate. We continue our annual tradition of ending the old year and ushering in the new one with Adam Feuerstein, Polk Award Winning journalist and senior biotech writer for STAT. We discuss the good, the bad, and the ugly of biotech in 2022; Feuerstein’s annual best and worst CEO list; and what’s ahead at JPMorgan and beyond in 2023.

Checkpoint inhibitors are not new, but Immutep is developing therapies that target LAG-3, a checkpoint discovered by its chief scientific officer. The LAG-3 gene codes for a protein that plays a role in the regulation of the immune system and is expressed on T cells. Immutep is seeking to target LAG-3 to both stimulate the immune system to treat cancers and suppress the immune system to treat autoimmune disease. We spoke to Marc Voigt, CEO of Immutep, about the LAG-3 checkpoint, the company’s lead experimental candidate, and its partnerships with GSK and Novartis to develop therapies for cancer and autoimmune diseases.

The ability to engineer biology is not only revolutionizing how we treat illness, but the way grow food, and produce goods as well. With this ability, though, comes an awareness of how the same technology can be used for nefarious purposes. We spoke to Matt McKnight, general manager of Biosecurity of Ginkgo Bioworks, about our increasing ability to engineer biology, the potential risks that come with that, and the steps we are taking to safeguard ourselves against those risks.

Cytokines control nearly all aspects of the immune system. As drugs, though, they have limited potential because of inherent drawbacks. Simcha Therapeutics is engineering versions of cytokines to activate various immune cells in the fight against cancer. Simcha’s most advanced program, ST-067, is the first in a new class of interleukin-18 based immunotherapies designed to overcome a key defense mechanism that the tumor microenvironment produces to prevent Interleukin-18 from doing its work. We spoke to Sanuj Ravindran, CEO of Simcha Therapeutics, about the company’s engineered cytokines, how they work, and why they may represent a powerful addition to the immunotherapy arsenal.

Arcellx is developing controllable cell therapies for the treatment of cancer and autoimmune diseases. It’s platform technology allows it to mark diseased cells within the body for destruction. Its cell therapies then destroy the flagged cells. We spoke to Rami Elghandour, chairman and CEO of Arcellx, about the company’s platform technology, how it works, and how it may lead to safer and more effective cell therapies.

Despite the large human and societal costs of neuropsychiatric disorders, many of these conditions have poor or no treatment options. And there has been a dearth of new mechanisms to treat major neuropsychiatric disorders for decades. PsychoGenics is seeking to change that with its unique approach to drug discovery for these conditions. Rather than rely on a target-based approach, the company has created high-throughput phenotypic screens using validated mouse models and artificial intelligence to detect behavioral changes. We spoke to Mark Varney, chief scientific officer of PsychoGenics, about the company’s use of phenotypic screens to discover new drugs for neuropsychiatric disorders, how it works, and the case for this approach.

Chronic, cancer-related pain affects between 30 and 50 percent of patients undergoing cancer treatment and more than 70 percent of patients with advanced disease. Linda Carlson, Enbridge research chair in psychosocial oncology at the University of Calgary Cumming School of Medicine, is working with digital therapeutics developer Rocket VR Health to study the potential of a virtual reality-guided mindfulness intervention program in adult cancer patients with chronic, cancer-related pain. The thinking is that mindfulness could mitigate chronic cancer-related pain by regulating both physical and emotional resistance to it and that virtual reality could create an immersive environment that could focus patients’ attention on present moment experiences making it easier for them to achieve mindfulness achieve more effective results. We spoke to Carlson about the problem of chronic pain in cancer, the case for mindfulness, and why the use of virtual reality might provide better outcomes for patients.

One reason immunotherapies can fail in cancer is that the tumor microenvironment can suppress activation of the immune system. Surface Oncology is using multiple platform technologies to discover new antibody therapies that activate both the innate and adaptive immune systems to improve how people with cancer respond to these medicines. We spoke to Rob Ross, CEO of Surface Oncology, about the tumor microenvironment, the approach Surface is taking to activate both the adaptive and innate immune system, and its lead therapeutic candidates in development.

The deepening understanding of the biology of aging is giving rise to a growing list of companies that seek not only to address diseases of aging, but find ways to extend the healthy years of life. LongeVC is a venture capital firm focused on seed and early-stage investment in longevity statups. It’s also built a community of like-minded companies to support startups, as well as an acceleration program for them. We spoke to Sergey Jakimov, founding partner of LongeVC, about the firm’s approach to investing, what constitutes a longevity play for it, and the network of companies it has assembled to help advance the startups it backs.

A subset of patients treated with checkpoint inhibitors respond particularly well to these immunotherapies. These so-called “elite responders” produce antibodies that modulate immune cells in the tumor microenvironment. OncoResponse is working to discover the antibodies these elite responders produce to develop a pipeline of cancer therapies. We spoke to Bob Lechleider, chief medical officer of OncoRepsonse, about the tumor microenvironment, how his company is studying elite responders to immunotherapies to discover new antibodies to modulate the immune system, and his company’s lead program in clinical development.

Obesity rates in the United States have doubled in the past 20 years and without changes, nearly half of the adult population will be obese by 2030. Though there are many approaches to weight loss—diet and exercise, drugs, surgery—their safety and efficacy varies widely from patient to patient. Phenomix Sciences is using phenotyping to understand the type of obesity each individual has to tailor treatments that address it specifically. Based on work at the Mayo Clinic, Phenomix breaks obesity into four different subtypes. We spoke to Mark Bagnall, CEO of Phenomix, about the company’s efforts to bring precision medicine to the treatment of obesity, the testing it performs, and the science underlying its approach.