The first of a raft of experimental PSP drugs in the pipeline has begun enrollment in a large treatment trial.
The ORION trial (don’t ask me what the acronym stands for) is sponsored by Amylyx Pharmaceuticals. Their press release is here. A bit more information is at the company’s website. Their email address is clinicaltrials@amylyx.com.
Clinicaltrials.gov lists more info, including a phone number (in the UK): +44 (808) 1642604. Only a few of the planned 33 sites in the US are open so far and no sites outside of the US has yet opened, to my knowledge. Those will be in Europe, Canada and Japan.
The product is actually two drugs called taurursodiol and sodium phenylbutyrate. They have a variety of beneficial actions in brain cells affected by PSP, but it’s not known which would be most important. Both drugs are already on the market, the first over-the-counter and the second with prescription. Neither alone is intended for brain diseases and each alone has only a minimal effect on the brain. But the two together have a synergistic effect on the brain and spinal cord that has proven modestly beneficial in ALS and has already received FDA approval for that disorder under the brand name Relyvrio.
There’s a lot more you’d want to know about the study’s design and the participants’ obligations, so please read the material at the links I’ve provided above and contact the company directly.
Amylyx has hired a contract research organization called Cronos to actually run the trial. They’re a subsidiary of a company called IQVIA. So if you encounter those names, no worries.
One more little thing: full disclosure. I’m a paid consultant for Amylyx, but that’s only for advice in the design of the trial and instructing the examining doctors how to use the PSP Rating Scale. They don’t pay me to recruit patients and my fee does not depend on the trial’s enrollment. Nor do I own stock in the company or have any other financial incentive to see the drug succeed. (Of course, I do have an emotional incentive for that — no secret there.)
PSP Blog 
Dr. Golbe:
Thank you for posting the update on the Relyvrio trials. If this is the first time it is being given to patients with PSP, why is it phase 3 (as opposed to 2)? Are the results from the ALS trials deemed applicable? I read that a phase 3 trial is also taking place for ALS, to give the FDA more information than provided by the phase 2 trials, even though they approved the drug.
I think you’re right that the safety data from the ALS trials were deemed adequate to justify approving a large, Phase 3 PSP trial. Of course, that trial is gathering safety data as well as efficacy data, and if patients with PSP have some odd, important adverse effect that didn’t happen to people with ALS, the safety monitoring committee can halt the trial.
Thank you very much for the information.. Yet, since we live in Europe, we are not yet started. Even though , whenever we tried to apply for participation, they never accepted patients outside their own country. Is this the idea? Are all countries excluded when a trial runs outside the chosen one?? Then, the news should not even include other countries but only the… selected ones! We would be grateful for your guidance and infromation. We are all equal to participate I think, so shouldn t it be somehow safeguarded the right to be included? Thank you
This is not something that relates to my consulting for drug companies. I agree that from an ethical standpoint, everyone should have access to clinical trials. But laws in specific countries may limit access to non-citizens, and language differences may also be an issue. I’ll ask someone at IQVIA or Amylyx next chance I have and report back.
Following my previous comment, would you be kind enough to let us know how to contact CRONOS?? Can t find any contact info… thank you
Cronos is a subsidiary of IQVIA. Try https://coas.iqvia.com/
Thank you for the update Dr. Golbe. Would you also share your thoughts about the results of the Transposon phase 2 trial for TPN-101 as it relates to PSP?
Dear Azadeh:
That drug is very promising. I posted about it in November 2023.
I have no further information on it.
Thanks. LG