The Bio Report

Targeting the Right Proteins to Treat Neurological Diseases

The recent late-stage failure of another high-profile Alzheimer’s disease drug candidate is a reminder of the challenges of developing drugs for neurological diseases. These challenges are driven by the fact that there are hundreds of different brain cell types and complex circuits and pathways in the brain that make it difficult to identify the right protein to target to treat a given disease. Cerevance says its proprietary platform overcomes the limitations of must approaches used today to study human brain cells. We spoke to Brad Margus, CEO of Cerevance, about the company’s platform technology, how it addresses the challenges drug developer face, and the programs it has advanced to date.

A Treat Locally, Act Globally Strategy for Treating Cancer

Intensity Therapeutics argues that cancer is both a micro and macro disease. Therapies need to act at the site of tumors, but they also need to treat the systemic effects of the disease that results from micro-metastases. Intensity is developing cancer therapies that are delivered directly into tumors, but also stimulate the innate immune system to address the more systemic effects of cancer. We spoke to Lewis Bender, CEO of Intensity, about the company’s platform technology, how it enlists the innate immune system in the battle against cancer, and the thinking behind the company’s approach.

Squeezing New Possibilities into Cell Therapies

SQZ Biotech believes its platform technology can enable the engineering of virtually any function into any cell type allow for a new world of cell therapies to revolutionize the treatment of diseases. As its name implies, it does this by squeezing a cell to momentarily disrupt its membrane to allow it to insert a range of substances. We spoke to Armon Sharei, CEO of SQZ Biotech, about the company’s platform technology, its initial focus on oncology and auto-immune diseases, and the broad potential for this approach.

Industry Calls for New Incentives to Spur Development of New Antibiotics

As concerns grow about the rise of antibiotic resistant bugs, the ability to combat them is being undermined by the economics of antibiotic drug development. Many large pharmaceutical companies stepped away from the space and despite the passage of the GAIN Act in 2012 and government investment in antibiotic research and development, drug companies argue more action is needed. We spoke to Michael Dunne, chief scientific officer at Iterum Therapeutics, about the state of the antibiotic arsenal today, the challenges drug developers face, and whether Congress will be willing to provide the industry incentives to address societal needs at a time when there is growing animosity towards the industry over issues such as pricing.

Company Seeks to Prove Marine-Derived Alzheimer’s Drug Is No Fish Story

Neurotrope is developing its experimental therapy bryostatin as a potential treatment for Alzheimer’s disease and other neurodegenerative conditions. The company believes the drug has multiple mechanisms of action and can stimulate synaptic growth factors to repair damaged synapses, form new ones, and prevent neuronal death. The company, though, found itself in a controversy over the way it characterized results from a phase 2 study of the drug in 2017. It is now conducting a second phase 2 study of the drug in the hopes of demonstrating meaningful benefits for patients. We spoke to Daniel Alkon, chief scientific officer of Neurotrope, about bryostatin, the controversy over the way it reported its previous results, and the path forward for the drug.

Targeting the Plasma Proteome to Address Diseases of Aging

As people age, their bodies lose the ability to regenerate as well as they were able to when they were younger. Alkahest believes this is tied to changes in the mix of proteins that are present in the blood as we age. It is developing therapies for diseases of aging that seek to target specific proteins that are upregulated in diseases of aging, as well as providing a wide set of proteins drawn from purified human plasma as a therapeutic approach to counter the depletion of regenerative proteins. We spoke to Elizabeth Jeffords, chief commercial and strategy officer for Alkahest, about the science underlying the company’s approach, the controversy around others who have offered so-called “young-blood” transfusions, and the pipeline the company is pursuing.

Teaching Scientists to Be Effective Communicators

There was a time when scientists could spend their careers talking only to other scientists about their work, but that’s changed. Whether it’s the current funding climate, the need to understand complex scientific issues underlying public policy debates, or opportunities to fund translational work and commercialize important discoveries, scientists are increasingly being tasked with addressing lay audiences. In “Championing Science: Communicating your Ideas to Decision Makers” the husband and wife team of Roger and Amy Aines offer a detailed guide for scientists on how to communicate effectively with non-scientists. Roger Aines is chief scientist of the energy program at Lawrence Livermore National Laboratory and Amy Aines is CEO of Damianakes Communications. Each bring their professional experience to the book. We spoke to the duo about their book, why it's needed, and why they believe scientists not only need to learn how to communicate their ideas, but to compel action and change the world for the better.

Designing Small-Molecule, Immuno-Oncology Drugs That Act Like Biologics

Kleo Pharmaceuticals is a developing next-generation immuno-oncology drugs that are small-molecule compounds designed to act like biologics. Kleo’s compounds activate patients’ immune systems to target and destroy cancer cells but are faster and less costly to design and produce than biologics. We spoke to Doug Manion, CEO of Kleo, about the company’s platform technology, why he thinks it will produce safer and more effective immunotherapies, and the potential to use these compounds in conjunction with existing biologics to enhance their activity.