The Bio Report

United Biomedical has long been in the business of producing animal vaccines, but in recent years it has turned its attention to developing vaccines for chronic human diseases including neurological diseases, diabetes, and other targets. More recently it has used its vaccine platform to develop a multitope peptide COVID-19 vaccine and spun it out into a division dubbed COVAXX. We spoke to COVAXX co-founder and CEO Mei Mei Hu and COVAXX co-founder and Chairman Peter Diamandis, about the company’s efforts to develop a COVID-19 vaccine, how its peptide-based vaccine works, and the case for this approach. Since recording this interview, COVAXX began dosing participants in its phase 1 trial September 28, 2020.

CAR-T therapies are an area of great promise for improving outcomes for cancer patients, but the process of preparing cells taken from a patient and genetically modifying them is time consuming and costly. Exuma Biotech believes it can address the cost and time involved in the preparation of these products with its rapid, point-of-care platform technology. We spoke to Greg Frost, chairman and CEO Exuma Biotech, about CAR-T therapies, how its technology works, and how it is working to cut the cost, and speed the delivery of these immunotherapies to patients.

The administration of biologics can be challenging because of the sheer volume of product needed to deliver a dose to a patient. It is for this reason that many of these therapies must be infused into a patient, a process that is disruptive, costly, and can take several hours. Halozyme’s Enhanze drug delivery technology has been used by a number of biologics producers to take a therapy that would otherwise need to be infused to allow it to be administered with a subcutaneous injection. We spoke to Renee Tannenbaum, vice president of global partnering for Halozyme , about the company’s Enhanze drug delivery technology, how it works, and the company’s reorganization to focus on partnerships around it.

NASH, a form of fatty liver disease, causes progressive damage and can lead to inflammation, fibrosis, and the development of cancer. It has become an increasing health concern as the Western diet has spread throughout the globe. Today it is estimated that as much as 5 percent of the world’s population has NASH with a greater percent of people with the condition in the United States. There is no simple blood test to diagnose NASH, which means it often progresses to a fibrotic stage before it is detected. There is also no approved therapy to treat it. We spoke to Robert Foster, CEO of Hepion, about the company’s experimental therapy for NASH, how it works, and why it may also have utility as a treatment for COVID-19.

The complexity of neurodegenerative diseases has made it a challenging area for drug developers. Gene Kinney, CEO of Prothena, has long been involved in the pursuit of therapies for diseases such as Alzheimer’s and believes there has been great progress in understanding and targeting these conditions. In fact, he says we are entering a golden age of neuroscience. We spoke to Kinney about the state of drug development for neurodegenerative conditions, Prothena’s pipeline, and its effort to target protein dysregulation in these diseases.

The U.S. Food and Drug Administration delivered a blow to Pharnext in 2019 when it told the company it should conduct an additional late-stage study of its experimental therapy for the rare neurological condition Charcot-Marie Tooth disease. A manufacturing problem that affected the dose delivered to some patients invalidated one the arms of its study. The company uses artificial intelligence and network pharmacology to identify molecules that may be involved in modulating a disease and searches for synergistic combinations of both existing and novel therapies to treat conditions. Since it’s clinical setback, the company named David Horn Solomon as its new CEO, sharpened its focus, and moved forward with an additional study. We spoke to Solomon about the company’s approach to therapeutic development, its focus on neurological conditions, and the case for using combination therapies as the best way to tackle the diseases it targets.

MindMotion GO is a first-of-its-kind mobile neurorehabilitation therapy system that helps people regain motor and task functions through a gamified approach. While the game playing aspects of the technology may help engage patients in activities designed to help them recover from brain injury, the technology is validated by clinical studies and has won regulatory clearance. It should not be confused with conventional video games. We spoke to John Krakauer, chief medical advisor for MindMaze, about neurorehabilitation, how MindMotion Go works, and the benefits of being able to move neurorehabilitation out of the medical centers to treat patients in their homes, particularly during a pandemic.

People with the rare and fatal genetic disease spinal muscular atrophy in recent years have seen the approval of an antisense therapy as well as a gene therapy. Genentech has now won U.S. Food and Drug Administration approval for Evrysdi, the first oral, at-home treatment for the condition. We spoke to Levi Garraway, chief medical officer and head of global product development at Genentech, about Evrysdi, how it works, and how it fits into the choices physicians and patients have when it comes to treating spinal muscular atrophy.

While the revolution in genomics has led to rapid improvements in the cost and speed of sequencing and created new insights into the genetic drivers of health and wellness, proteomics has lagged behind. Being able to capture a comprehensive view of the changing level of proteins in an individual could play a significant role in bringing about an era of precision medicine. SomaLogic is providing a push in that direction with its SomaScan Discovery platform, which can read 5,000 protein measurements in the blood through a single assay. We spoke to Roy Smythe, CEO of SomaLogic, about the role proteins play in health and wellness, the way the company’s technology works, and the opportunity for it to help advance the area of precision medicine.

The problem of drug resistance isn’t limited to bacteria. It is also a growing concern with fungal species that is causing an increasing need for new agents to combat these microbes. Scynexis is developing the experimental therapy ibrexafungerp, the first of a new class of therapies for serious fungal infections. We spoke to Marco Taglietti, president and CEO of Scynexis, about the problem of drug resistant fungal infections, the company’s experimental therapy ibrexafungerp, and why it may provide a new way of treating a range of serious fungal infections.