Developing a Zauberkugel in 2025

Paul Ehrlich (1854-1915) is recognized for much of the foundation of modern therapeutics. He imagined that there were drugs that could be identified which treated specific disorders without causing toxicity to the normal cells. He applied that vision to his development of one of the first targeted therapies, an antibiotic. At the time, the drug commonly used to treat syphilis was also toxic to normal cells. Ehrlich’s team identified a synthetic molecule that would kill syphilis only – without the toxicity. Using this approach he developed what he called “Zauberkugel” meaning a magic bullet. His contributions are impressive in number and in their breadth.

Ehrlich claimed that success in medical research was based on the “four Gs” of geduld (patience); glück (luck); geschick (skill); and geld (money). Right Brain Bio’s development of RB-190 is strong on patience, luck and skill, but still a bit short on the money. We start 2025 with an optimistic plan for fundraising that will enable us to hit major milestones.

It’s dangerous to speak too optimistically in the tightly regulated worlds of drug development and financial investments. We're not supposed to claim we can do something until it is proven. So I try hard to refer to the possibilities rather than discussing in absolute terms. And conventionally there would be a very detailed disclosure statement pointing out that all that follows is a vision rather than a promise. But I’ll skip that since this newsletter is not asking for you to invest, just to be aware of the possibilities for progress in 2025.

Activities in progress for early 2025 include:

1. Manufacturing. While we previously started the process of identifying and qualifying drug manufacturers, we hired an expert in drug chemistry and manufacturing to review that information and lay out plans for manufacturing the drug for the clinical trials. I expect we’ll have our plan in place soon which will allow us to formalize a relationship with a manufacturer and get that process going.

2. Regulatory. Our development of RB-190 is only within the United States, as it and Japan are the only countries where the drug was approved for its prior use. Because RB-190 is a repurposed drug, which means it was already approved in the US for another use (in this case to treat a complication of a rare tumor), the FDA allows for faster and cheaper path to market compared to the requirements of a brand new drug. This makes sense because we know that such a drug already underwent a ton of laboratory and clinical testing.

To start the first clinical trial requires approval of an Investigational New Drug (IND) application. We will be starting to work on this application soon, though some of the components are already near final form. This is a ton of work and generally is thousands of pages long. And a significant part of the IND is the data showing that our manufacturing process works and the drug is ready to be administered to people. These data can’t be complete before the middle of the year as it will take that long to manufacture the drug and generate these data to show that we manufactured the drug properly.

3. Operations. A big part of preparing for, conducting and completing a trial is work typically performed a contract research organization (CRO). CROs are standardly used by large and small companies developing drugs. They can perform a broad range of roles, from contracting with the sites conducting the trial to managing how they enroll people. They oversee data collection and verify that all the information is recorded accurately. They manage how study drug is shipped to sites. They gather information if a trial participant experiences something adverse, and if serious, report it to the FDA. The list is lengthy! We recently launched the process of identifying CROs that are best suited to work with Right Brain Bio to conduct this trial safely and with the rigor to assure the results can be trusted.

What comes next?

When we manage Ehrlich’s 4 Gs the right way, we believe it makes sense to plan for the best-case scenario that our IND is submitted midyear, the FDA approves it within 2-3 months and then we can launch the trial by year’s end. It will be a busy year, and with the 4Gs, a productive one as we move this therapy into the clinic and hopefully show that it will be a transformative one, becoming Ehrlich’s Zauberkugel for Parkinson’s disease.