Tuesday, March 12, 2019 5:26:51 PM
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.
https://www.prnewswire.com/in/news-releases/oraxion-executes-exclusive-option-to-license-orx-301-672665083.html
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.
Recent CYTH News
- Cyclo Therapeutics Presents Encouraging Preliminary Safety Data from Ongoing Pivotal Phase 3 Study and Substudy for the Treatment of Niemann-Pick Disease Type C1 (NPC1) • Business Wire • 09/05/2024 01:05:00 PM
- Kuehn Law Encourages CYTH, ARCH, CEIX, and VGR Investors to Contact Law Firm • PR Newswire (US) • 08/26/2024 01:37:00 PM
- Form 425 - Prospectuses and communications, business combinations • Edgar (US Regulatory) • 08/22/2024 01:37:04 PM
- Form 8-K - Current report • Edgar (US Regulatory) • 08/22/2024 01:36:40 PM
- Rafael Holdings and Cyclo Therapeutics Enter Into a Definitive Merger Agreement • Business Wire • 08/22/2024 10:50:00 AM
- Rafael Holdings and Cyclo Therapeutics Enter into a Definitive Merger Agreement • GlobeNewswire Inc. • 08/22/2024 10:50:00 AM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 08/20/2024 05:50:50 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 08/20/2024 05:49:54 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 08/20/2024 05:49:29 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 08/20/2024 05:49:08 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 08/20/2024 05:48:45 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 08/20/2024 05:48:24 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 08/20/2024 05:47:47 PM
- Cyclo Therapeutics Reports Second Quarter 2024 Financial Results and Provides Business Update • Business Wire • 08/15/2024 01:00:00 PM
- Form 10-Q - Quarterly report [Sections 13 or 15(d)] • Edgar (US Regulatory) • 08/14/2024 09:28:23 PM
- Cyclo Therapeutics Announces Notice of Decision to Grant European Patent Application for Treatment of Alzheimer’s Disease from European Patent Office • Business Wire • 07/29/2024 01:05:00 PM
- Form 8-K - Current report • Edgar (US Regulatory) • 07/17/2024 06:35:02 PM
- Cyclo Therapeutics to Present at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2024 • Business Wire • 06/25/2024 01:25:00 PM
- Rafael Holdings Reports Third Quarter Fiscal 2024 Financial Results • GlobeNewswire Inc. • 06/14/2024 08:01:00 PM
- Cyclo Therapeutics Achieves Landmark Milestone with Completion of Enrollment of Last Patient in Phase 3 Pivotal TransportNPC™ Trial of Niemann-Pick Type C1 • Business Wire • 05/30/2024 01:00:00 PM
- Cyclo Therapeutics Reports First Quarter 2024 Financial Results and Provides Business Update • Business Wire • 05/16/2024 12:05:00 PM
- Cyclo Therapeutics Reports Full Year 2023 Financial Results and Provides Business Update • Business Wire • 03/18/2024 01:05:00 PM
- Cyclo Therapeutics Announces Issuance of U.S. Patent Covering the Use of Trappsol® Cyclo™ for the Treatment of Alzheimer’s Disease • Business Wire • 03/14/2024 01:34:00 PM
- Rafael Holdings Reports Second Quarter Fiscal 2024 Financial Results • GlobeNewswire Inc. • 03/13/2024 09:04:24 PM
- Form 8-K/A - Current report: [Amend] • Edgar (US Regulatory) • 03/04/2024 09:33:34 PM
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