The Bio Report

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.

Colorectal cancer is the third most common form of the cancer. While screening is an effective means of preventing it, many people fail to get a colonoscopy because of the invasive nature of the procedure and the preparation and sedation that goes with it. Check-Cap is a clinical-stage company developing C-Scan, the first capsule-based system for preparation-free, colorectal cancer screening. The capsule uses ultra-low dose X-ray and wireless communication technologies to generate information on the contours of the inside of the colon as it passes through it. This creates a 3D map that allows physicians to look for polyps and other abnormalities. We spoke to Alex Ovadia, CEO of Check-Cap, about colorectal cancer, why people avoid being screened as they should, and how the company’s C-scan capsule-based system works.

To see the affects of aging all you have to do is look in a mirror and watch the changes over time. Fountain Therapeutics is training its artificial intelligence platform to look at individual cells to detect changes that occur as cells get older and discover therapeutics that target underlying mechanisms of aging. The company believes this will provide new ways to target therapies to treat a range of diseases associated with aging. We spoke to John Dimos, CEO of Fountain, about the company’s AI platform, it’s approach to understanding aging at a cellular level, and how it provides new ways of discovering and developing therapeutics for diseases of aging.

The emergence of immunotherapies has represented a powerful addition to the cancer arsenal, but frequently they fail to deliver benefits to patients. Understanding what therapies will benefit which patients remains a challenge because of the complexity of the immune system. Immunai is applying artificial intelligence to map the immune system and understand its complexities at a granular level to better understand its role in health and disease. It is applying what it learns to avoid clinical trial failures, improve combinations of immunotherapies, and guiding future therapeutic development in cancer and a broad range of other conditions. We spoke to Danny Wells, scientific founder of Immunai, about the company’s efforts to map the immune system, the challenges in doing so, and how this has the potential to improve drug development.

Artificial intelligence is working its way into all aspects of pharmaceutical companies’ operations. While much attention has been given to the role these systems can play in drug discovery, IQVIA sees a significant opportunity to use them to transform the area of regulatory compliance. We spoke to Ronan Brown, senior vice president and head of IQVIA Integrated Global Compliance, about the role AI system can play in improving flagging returns on investment in R&D by allowing regulatory departments to operate more efficiently, breakdown data silos within pharmaceutical companies that hamper performance, and allow companies to focus less on rote work and more on regulatory strategies.

Delivering biologics orally rather than through injection has been an intriguing goal but has proven difficult. Most efforts have focused on finding ways to turn these large protein molecules into formulations where they would not breakdown in the along the digestive tract before they can be absorbed and provide a therapeutic benefit. Rani Therapeutics has taken an unusual tact. Rather than reconceiving the biologic, Rani has reconceived the pill itself. The company has developed what it calls a “robotic” pill that carries the therapeutic to the gut where it injects the drug into the wall of the intestines. We spoke to Mir Imran, chairman and CEO Rani Therapeutics, about how the Rani Pill delivers biologics orally, the technology underlying it, and how the company thinks about the opportunities it will pursue.