The Bio Report

In 2022, investment in biopharmaceutical companies, pipeline activity, and the launch of novel medicines all dropped from the previous year. A new report from IQVIA’s Institute for Human Data Sciences argues the downturn, after two record-setting years, is a post pandemic return to longer-term trends. We spoke to Murray Aitken, executive director of the IQVIA Institute for Human Data Science, about what happened to the biopharmaceutical sector in 2022, how to make sense of the data, and the shifting landscape in therapeutic and geographic investments.

The aging process is associated with the development of diseases. BioAge Labs is seeking to expand healthy lifespans by developing therapies for aging-related diseases that target key pathways involved in the aging process. The company has built a platform that combines systems biology and AI to leverage extensive data sets to uncover molecular drivers of aging-related diseases. We spoke to Kristen Fortney, CEO of BioAge, about the company's approach to identifying targets for aging-related diseases, its therapeutic pipeline, and whether it's pursuing any novel targets yet.

Cell therapies are revolutionizing the way cancers are treated, but existing cell therapies have their limits. They have been more successful at treating hematologic tumors than solid tumors, and they can sometimes cause serious side effects, such as the destruction of antibodies or what’s known a cytokine storms in which the immune system gets over-revved and attacks healthy cells. Triumvira Immunologics is developing autologous and allogenic T cell therapies that it believes can address the limitations of existing cell therapies and be used to treat both liquid and solid tumors. We spoke to Paul Lammers, CEO of Triumvira, about the company’s platform technology, why it’s robust and versatile, and why its lead indication is for a cancer where effective treatments already exist.

A complex of proteins that play an essential role in regulating the innate immune system known as the NLRP3 inflammasome is becoming a growing target of interest among drug developers to disrupt immune cell signaling. Halia Therapeutics is developing a pipeline of therapies that target the NLRP3 inflammasome to address not only inflammatory disorders like psoriasis and colitis, but neurologic conditions such as Alzheimer’s disease. We spoke to David Bearss, president and CEO of Halia, about the NLRP3 inflammasome, the role it plays in Alzheimer’s and other neurologic conditions, and the case for this therapeutic approach.

Though the transplantation of insulin-producing islet cells has been used to treat people with type 1 diabetes, such procedures require the use of immunosuppressive drugs to prevent the immune system from rejecting the cells. iTolerance is developing platform technology that can be used with tissue, organoid, and cell therapies without the need for life-long immunosuppression. We spoke to Anthony Japour, president and CEO of iTolerance, about the company’s platform technology that eliminates the need for immunosuppressive drugs with tissue and cell transplantation, the company’s lead experimental cell therapy for type 1 diabetes, and the broad range of regenerative therapies that could benefit from the technology.

Dyadic is working to bridge the gap between high-yield, low-cost, and large-scale industrial biotechnology, and low-yield, high-cost, small-scale biopharmaceuticals. It’s C1 technology, which is a fungal expression system, can efficiently produce enzymes and other proteins. Earlier this year, the company achieved a milestone when it began dosing patients in a phase 1 clinical trial in South Africa of its COVID-19 booster vaccine. The company expects the first-in-human trial to accelerate the adoption of the C-1 production platform for vaccine and therapeutic candidates. We spoke to Mark Emalfarb, president and CEO of Dyadic, about the company’s fungal-based manufacturing platform, how it can product large volumes of enzymes and other proteins in a fast and cost-effective manner, and the potential this has to change the way biologics are manufactured.    

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.