RE:RE:RE:RE:RE:RE:Gene Therapies I've just slapped this post together because I'm too busy to make it shorter and more concise. A book could be written about the science and commercial attributes of the stuff I've written here.
Whether you realize it or not, pof, you're getting closer to making my points that LSD enzyme replacement therapies (ERTs) have declining values.
Given this Shire/ArmaGen deal, and Shire's undisclosed but nevertheless "interesting" behaviour with Bioasis, what were Shire's motives? Were they really interested in advancing AGT-182, or were they interested in keeping it out of other companies' hands to protect their own Eleprase for Hunter syndrome, the world's most expensive drug at that time?
We'll never know Shire's motives for certain but we do know that the deal was specifically for AGT-182. We discussed the equity portions years ago. At the time, Bioasis was touting the deal and the AngioChem/Geron deal as reasons why the company considered LSDs to be "low hanging fruit," meaning easy entry with high value. The numbers discussed by the company at the time were low tens of millions up front and low hundreds of millions in milestone payments per LSD, at least for the top 4 or 5 LSDs.
The Shire/ArmaGen deal was a disappointment because it defined a lower value for a major LSD, Hunter syndrome. The problem, of course, was that we had no idea what the equity position was relative to the purchase price of AGT-182. One thing we can assume is that compared to Chiesi getting 4 LSDs for an average of $750,000 each up front and $34.5 million each in milestone payments, Armagen certainly got more than Bioasis did. And don't get me started about royalties. Bioasis has never revealed a royalty greater than 1% while the ArmaGen/Shire deal described "
future royalties up to double digits," which would be at least ten times the royalty rate that Bioasis has revealed.
But wait, it gets more interesting.
ArmaGen claimed that their BBB platform could be used with the insulin, transferrin and LRP-1 receptors, the latter being xB3's receptor. Ultimately, the insulin receptor was dropped for reasons we've discussed on here before (it can interfere with blood sugar). ArmaGen, after being acquired by JCR Parmaceuticals of Japan, now uses the transferrin (Tf) receptor. In fact, JCR's JR-141 has been approved for use in Japan for Hunter syndrome and is in late stage clinical trials elsewhere in the world.
And Takeda bought Shire.
And Takeda partners with Denali, who also uses the transferrin receptor with their F-Star TV transporter and has an entry in the Hunter syndrome business. Denali released data on it Hunter syndrome trial the other day.
Takeda is in bed with everybody but Bioasis! And everybody thinks they can make a big deal out of Hunter syndrome, including Bioasis.
Nonsense. You can't sell 4 LSDs for $750,000 up front and $34.5 million in milestones and then tell us that xB3-008 for Hunter syndrome is a big deal! As I've stated many times, xB3-008 was in the pipeline before the Chiesi deal. With the Chiesi deal the LSDs are secret so if Hunter syndrome is one of the LSDs then Bioasis had to keep xB3-008 in its pipeline to aboid revealing it as one of the secret LSDs. I could very well be wrong about this but the answer is not definitive in any Bioasis literature.
And even if Bioasis keeps Hunter syndrome, its value would seem to be quite small. But even if xB3-008 is worth a lot of money, Bioasis has not been making that case. It's doing the opposite.
I don't expect the LSDs with either Bioasis or Chiesi, even a Fabry ERT, to be really important to shareholders. The use of xB3-004 (IL1-Ra) for Fabry neuropathic pain, along with its many other uses like traumatic brain injury, MS and such, could be much more valuable. Add oncology and several other neurodegenerative diseases and Bioasis does get very interesting - IF xB3 WORKS AS HOPED, something Bioasis has not proven. I've written about the LRP-1 receptor and some off-target delivery problems it might have. Bioasis needs to publicly address this, either with a damn good deal or directly with a statement.
This is a difference of opinion and pof could be right about the LSDs, but so far, his case hasn't been made because Bioasis, by their actions, is arguing against him. I seem to be agreeing with Bioasis on the issue.
jd