Who will benefit from RB-190?

I don't know who will benefit.

No one could know; not until several randomized clinical trials are completed. That's right, I won't know who will benefit from RB-190 even after the first trial. But this first trial will establish that people could benefit, which would be a remarkable step in shifting the focus of the entire field from adding dopamine to reducing dopamine (and its toxic side effects).

Who do I believe will benefit from RB-190?

Considering that no one with Parkinson's has been treated with RB-190, I am trying to communicate so that you don't interpret my statements to indicate that there is evidence that proves RB-190 is the solution to PD. I believe it will be. And here is what I believe, subject to the lack of clinical data. (Sorry if this seems confusing; I want to be sure the Securities and Exchange Commission understands that these are my predictions for what we will eventually learn, so I don't have to worry about any legal proceedings.)

Does type of Parkinson's matter?

I am swayed by the scientific studies that show that both the genetic and sporadic forms of Parkinson's have the common problems of excess cytosolic dopamine. In multiple models of genetic forms and models of sporadic disease, RB-190 reduces dopamine and reverses pathology. I do not believe this approach is only for one type or another.

Does severity of the disease matter?

I am biased by my work as a cardiologist with a situation similar to advocating dopamine reduction when the standard of care is to increase dopamine action - that of beta-blockers as a treatment for congestive heart failure (which is called heart failure with reduced ejection fraction by the medical community). That was a setting where people with limiting symptoms of shortness of breath and/or fatigue were given drugs to increase the effects of adrenaline and adrenaline-like chemicals (adrenergic drugs akin to dopaminergic drugs). Those drugs helped with symptoms for months but then were found to increase risks (in the case of adrenergic drugs, increased risk of death was seen when studies extended beyond a year). A couple of Swedish cardiologists understood that adrenaline and adrenergic drugs could be toxic and were the first to try beta-blockers. Eventually these were proven to make people feel better and live longer. They are now standard of care.

In the clinical trials of beta-blockers for heart failure, we learned that they helped whether people had early/mild disease or advanced disease. Even people on heart transplant waiting lists could benefit. The drugs became standard of care for people with mild, moderate or severe disease. Those with severe disease would frequently have more difficulty tolerating the initiation of the drug but still could benefit.

We learned that beta-blockers prevented toxicity of excess adrenaline. We also learned that once that toxicity was reduced, the individual cardiac muscle cells reverted from their pathologic, functionally impaired state back towards normal. The overall function of the heart could dramatically improve.

This experience is why I believe we will eventually learn that dopamine reduction therapy could be useful irrespective of the severity of Parkinson's.

I'm looking forward the launch and conduct of clinical trials with RB-190 because the data tell us there is great promise for this therapeutic approach. We need this trial and hopefully we'll have it funded and launched in 2026.