LSDs and Other Speculations In response to some comments made here today, I thought I'd bang off some quick comments about LSDs, Hunter Syndrome, Chiesi, Bioasis and such.
I don't know whether Chiesi is still going to develop four LSDs under its contract with Bioasis. My guess is that the agreement is in force in a real manner, not in the "old soldiers just fade away" manner. I believe there is substantial opportunity there and that the work will be advanced to either success or failure.
If those LSDs are being advanced with Protalix, and if one of the LSDs is Fabry disease, as we suspect it is, then their could have been, or may continue to be, delays in the program until Protalix's ProCellEx manufacturing facilities are inspected and approved by the FDA for PRX-102. The Bioasis/Chiesi agreement was signed after Covid became an issue but before it was realized (or announced) that the FDA would not be able to inspect the facilities. In turn, that caused the Chiesi/Protalix/FDA fast-tracking agreement to expire in April 2021, requiring Chiesi/Protalix to resubmit a PRX-102 FDA new drug application after the ProCellEx facilities are approved.
So, for me, the question has been whether it would be a good business decision to start new programs if PRX-102 and the manufacturing facilities have not yet been approved by the FDA. My guess is that the four LSDs will be advanced and they may already be advanced to a degree, but until we hear something, we really don't know anything.
I also have speculated that because Protalix may be involved with Chiesi and the LSDs, that xB3-004 and xB3-progranulin may also be advanced through some sort of partnership among Chiesi, Protalix and Bioasis. That, as I have speculated, could lead to much larger agreements and partnerships among these players.
I also think it a possibility that xB3-008 for Hunter Syndrome (MPS II) may be one of the four LSDs in the Chiesi/Bioasis agreement, or will be done with those partners as an extension of that agreement. xB3-008 was listed in the Bioasis development pipeline before Chiesi came along. As I have previously speculated, because Chiesi wanted to keep the four LSDs secret, and if Hunter Syndrome was one of the four, then Bioasis had to leave Hunter Syndrome in its pipeline or it would have been implicitly revealed as one of the secret LSDs. My hope is that Hunter Syndrome remains completely owned by Bioasis, but it bothers me a bit that Bioasis has announced Phase 1 plans for the drug. Can Bioasis do that trial without a partner? That guidance could be given by Bioasis if there is revenue expected from further business deals, something I think is a very strong possibility.
Finally, pof made the following comment, "BTI is positioned to be a potential strategic monster in the Lysosomal Storage Disorder space."
I'm not sure I agree with that. Bioasis sold four LSDs to Chiesi for $3 million and $138 million in milestones, which is only about $45 million each. Bioasis low royalty rates (1%, so far) have not inspired confidence. These are not monster-making numbers. I expect Bioasis to gain recognition for its contribution but Bioasis will not gain much financially from it all. Like I say, I hope xB3-008 remains wholly-owned by Bioasis but I'm sceptical of that.
There's lots of good stuff that can Bioasis can accomplish but they have to take more steps to prove that xB3 works and is safe, and the company has to prove that it can negotiate monster deals if it's going to be a monster in any space.
Ok, gone again, although I might have something to say about the financials. I forgot they were due this week.
jd