An opinion based on research, with his/her thought process not dictated by his/her share portfolio (alone). Thank you!
It's a shame though that there's a (felt) need to mention if shares held in another company operating in this space is required.
A statement I can agree with. Just a few things though I'd like to add:
Orphan Drug Market Exclusivity:
"The FDA’s implementing regulations have narrowly interpreted the ODA’s exclusivity provision in Section 360cc. For example, the regulations state that exclusivity protects only the approved indication or use of a designated drug, and thus the FDA allows two different manufacturers to have orphan-drug exclusivity for the same drug for the same disease, if the drug is indicated for use in different patient populations. In other words, the FDA treats orphandrug exclusivity as specific to the designated use or indication of the drug, rather than extending exclusivity to cover multiple indications for use. At least one federal circuit court has expressed disagreement with this interpretation of the ODA, which the FDA still uses (see, e.g., Catalyst Pharmaceuticals Inc. v. Becerra, 14 F.4th 1299 (11th Cir. 2021)). For more information on the Catalyst case, see CRS Report R47653, The Orphan Drug Act and Catalyst Pharmaceuticals, Inc., v. Becerra, by Hannah-Alise Rogers."
https://crsreports.congress.gov/product/pdf/IF/IF12605Ryoncil has been approved in pediatric patients with SR-aGvHD and label extension/expansion is aimed at adults with SR-aGvHD.
Cynata's trial is aiming at adults in HR-aGvHD, which is essentially that population Osiris was initially targeting with Prochymal. SR-aGvHD in the pediatric sub-group was a result of post-hoc analysis of two failed P3 trials undertaken by Osiris (Protocol 265 and 280):
* There was no statistical difference between Prochymal and placebo on the
primary endpoints for either the steroid-refractory (35% vs. 30%, n=260) or the
first-line (45% vs. 46%, n=192) GvHD trials.
* The primary endpoint for the steroid-refractory GvHD trial (durable complete
response) for the per-protocol population approached statistical significance
(40% vs. 28%, p=0.087, n=179).
* In patients with steroid-refractory liver GvHD, treatment with Prochymal
significantly improved response (76% vs. 47%, p=0.026, n=61) and durable
complete response (29% vs. 5%, p=0.046).
* Prochymal significantly improved response rates in patients with
steroid-refractory gastrointestinal GvHD (88% vs. 64%, p=0.018, n=71).
* In pediatric patients, Prochymal showed a strong trend of improvement in
response rates (86% vs. 57%, p=0.094, n=28).
https://www.fiercebiotech.com/biotech/osiris-therapeutics-announces-preliminary-results-for-prochymal-phase-iii-gvhd-trialsThat brings us to your next point, "superiority."
Due to lack of Ryoncil data in clients with (HR)-aGvHD, which data would CYP's data being compared against? 45%? Or MSB-GVHD001 trial data in children, despite data suggesting that pediatric patients showed a strong trend of improvement not seen in adults?
Or would it fall back to Rux data including toxic side effects?
Originally is there another product approved in aGvHD as first line treatment other than steroids/combo therapy with steroids?
Then again, so far we have seen NIL data from CYP-001 in adults with HR-aGvHD. Maybe it works just as good as a glass of water afterall.
Ryoncil becoming the SoC by the time the trial starts?
The P2 has already started and is currently sitting at over 40% of the 60 adult patients to be recruited.
Or was that someone referring to a subsequent P3 trial? If so, SoC for SR-aGvHD or aGvHD - difference is about double the patient population, a bit less actually since the P2 trial is recruiting high risk patients, and potentially 72h:
72h can make a big difference. So does potentially off-label use vs free clinical trial participation.
If Cynata is successful/superior, I can't see it beong squeezed out and if it falls short in comparison to Ryoncil, it serves no purpose, neither in the US, here in AU, or on the Virgin Islands in my opinion. It would have merely served as an expensive indication to show that iPSC-MSCs are safe in humans.
I don't get the feeling that Cynata holders are overly concerned at this stage to be honest. Even if aGvHD is off the table due to sub-par clinical results, there are many other indications, including DFU etc. and based on a MC of A$60 million, it won't need much to see the SP move. The recent Nature article highlighted the advantages of CYP's iPSC-MSC, which could either be utilised as a stand-alone therapy in various indications, or in combination with existing MSC technology as interest in such has already been expressed according to MK.
The fact that you have certain MSB posters popping in constantly to try and stir the pot, is more so a sign of insecurity on their behalf, especially when in the end the non-argument is being closed off with "miles away from approval."
(20min delay)