Cyclo Therapeutics Inc (CYTH)

[NPC1Parent]

I am the parent of a child that has Niemann Pick type C1. I have watched this board over the last year or so. I try to keep up with companies that are developing therapies for NPC1.
People seem to focus on the competition between MNK and CTD. The truth is, they both have a lot competition and may both fail.
CTD’s claim that cyclodextrin crosses the blood brain barrier is a half-truth. It does, in very small amounts.
https://www.apnews.com/466c904683d946dd99b188664d730acd
“Levels of drug are 30 ug/ml to 450 ug/ml. This means that the drug crosses the blood-brain-barrier. It may explain why Trappsol® Cyclo™ in compassionate use programs has been linked to neurologic benefits. “
This is well below the therapeutic levels achieved via lumbar puncture.
Regardless, there is at least one company that claims to have a better formulations of cyclodextrin that does cross the blood brain barrier. Aten Porcus (Oraxion). A year ago, they signed an option agreement with an unnamed pharma company. I don’t know what became of that.
https://www.prnewswire.com/in/news-releases/oraxion-executes-exclusive-option-to-license-orx-301-672665083.html

In fairness, there may still be a need to treat systemic manifestations of NPC1. For example, treatment of the liver. Currently, IV Cyclodextrin is the only available therapy. However, there is competition there as well. Orphazyme has recently reported very positive data from their Phase II/III trial.
https://www.globenewswire.com/news-release/2019/01/30/1707336/0/en/Orphazyme-reports-positive-results-from-full-data-set-of-Phase-II-III-arimoclomol-trial-in-Niemann-Pick-disease-Type-C-NPC.html

While Orphanzyme did not address systemic manifestations, there are some that believe their approach may be superior, at least for some patients, than cyclodextrin.

Likewise, Intrabio is going to trial with UDCA for the treatment of NPC1 liver manifestations.
https://www.accesswire.com/532110/IntraBio-Receives-Niemann-Pick-Disease-European-Orphan-Drug-Designation-for-IB4000
Thus far, their data has been good. Still too early to say for sure.
"The clinical experience of IB4000 in patients with NPC, different in age and clinical features, suggest UDCA may be an attractive therapy for the disease. All patients tolerated the drug well, and in the patients with abnormal liver transaminases, IB4000 improved, if not normalised these parameters,"

More interestingly, there is well-funded company out of Oxford that is developing a method of delivering functioning NPC1 protein. If there are successful, Cyclodextrin will quickly become irrelevant.
https://www.evoxtherapeutics.com/
https://www.evoxtherapeutics.com/pipeline-programs
https://www.prnewswire.com/news-releases/evox-therapeutics-completes-35-5-million-series-b-financing-885256481.html

If you are betting on cyclodextrin, beware that there is competition from much better funded companies with different and possibly better approaches.