Right Brain Bio: an update

I shared the story of Right Brain Bio and how we expect to conquer Parkinson's on George Ackerman's Q&A podcast, available on on youtube.

In this week's post I will share some of the operational details so you see how the project is advancing.

Manufacturing

Manufacturing has been the main focus over the past several weeks. Even though the drug has been sold in the US market by two companies and in Japan by another, my efforts to form a partnership or even just buy the drug were unsuccessful. So we shifted our attention to the manufacture of the drug ourselves, which allows us to do so with a final form more appropriate for use in Parkinson's than the form currently being sold.

I contacted several contract manufacturers, with a goal of identifying one based in the US to make oversight a bit easier than if using a company based in India or China. And there are many choices. A major factor to consider is whether the manufacturer has already figure out how to manufacture the drug. and when the target is the US market, such a company would file their process with the FDA. Anyone can look up which companies have done so by searching the FDA website for Drug Master File (DMF) submissions.

Having a DMF does not mean it is as simple as signing a contract and waiting for the delivery of the drug. For example, manufacturing using a 15-step process means higher risk of problems (including contaminants) than in a 4-step process. There is just more that can go wrong.

On a high level, I know what we need from a manufacturer. But this is too important to accept any uncertainty. So we hired an expert chemistry and manufacturing group (with which I worked on a project together a few years ago). Our engagement with potential manufacturers will be more sophisticated than I could manage alone. We expect to begin drug manufacturing by the end of this year, keeping us on schedule to launch the Phase 2A clinical trial in 2025.

Logistics

In parallel with getting manufacturing going, there are three other tasks to start in parallel.

(1) In the event we choose a manufacturer outside the USA, we need a partner to transport, import (through Customs) and securely store.

(2) By the time we are getting close to receiving the drug, we need to have developed and proven reliable/accurate a testing platform - an assay. We'll use that assay to prove that the drug we receive is what it needs to be, including in its potency, purity, sterility etc. The manufacturer will have their tests to prove what they are shipping us, and we will have ours to prove that we are receiving the right material. Yes this is redundant, but that's necessary when manufacturing a drug to be administered to people.

(3) Once we have the drug substance - basically in powder form - we need to make the drug product to give to people. Think of that as making a capsule or a pill that allows for the drug to be absorbed into the body in a reliable, predictable way.

Our chemistry and manufacturing partner is helping us move all three of these tasks forward.

These tasks are not the sexy part of developing a drug, but they are amongst the most important. Without completing these tasks perfectly, no clinical trial should be allowed to start - and the FDA feels similarly.

The next steps

We are fortunate that our first investment is sufficient to allow for these tasks to move forward. In parallel, we are engaging with clinicians and clinical trialists to make final the clinical trial protocol, including through meetings at the International Parkinson and Movement Disorder Society meeting in September and the Michael J. Fox Foundation Therapeutics Conference earlier this month.

As we land additional investments, we are set to move quickly with submission of the IND (Investigational New Drug) application to the FDA, which is the request to conduct a clinical trial in the United States. And then the next stage begins, the clinical trial that we expect to prove that our approach to treating Parkinson's is worth continuing.