And another post by JD. This explains why BTIs business model is the key to success.
I'm not too interested in studying the pharmacokinetics (PK) and relative efficacy of the many versions of an enzyme that treat a given LSD. In fact, I'm not too interested in those details for any therapeutics. If a pharma is interested in transporting a given therapeutic into the brain then that's all I'm interested in. I would think that biOasis has the same attitude. We're not interested in taking on the risks associated with all the therapeutics out there.
The biOasis business model pretty much demands an agnostic approach to therapeutics. If Shire wants to license Elaprase then I guess that MTfp-Elaprase would be their fusion protein of choice for MPS II. If somebody else wants the LSD license, I would think that they have an enzyme structure in mind for MPS II. Why should we care? Whatever the pharmas might have, including Shire with Elaprase, they have nothing compared to whoever can get it into the brain. biOasis will license the Transcend LSD rights to the highest bidder and the winner will choose the enzyme structures.
Remember that the different versions of any protein are created to satisfy many criteria including efficacy, safety, PK, cost of manufacture, IP protection, competition, etc. When any therapeutic is bolted onto MTfp, it instantly becomes a patentable new chemical entity.
With respect to the high costs of the rare disease therapies such as Elaprase, probably only a small part of that cost has anything to do with manufacturing costs. Whether there are 2000 patients worldwide or 2,000,000, it costs about the same to develop a therapeutic and to put it through trials, although many of the orphan drug and other fast track policies of the FDA do mitigate some of those costs. Nevertheless, the high cost of these drugs has everything to do with the cost of developing the drugs and the cost of doing business rather than the cost of manufacture.
But make no mistake, these drugs can be costly to manufacture and the pharmas will do what they can to cut costs. Again, this is the beauty of the biOasis business model. If the exclusivity of a therapeutic is a criteria for the commercial success of the therapeutic, then Elaprase seems to be a perfect example of that. But Shire had to put a lot of effort and money into achieving that exclusivity. Some other company can achieve the same exclusivity by simply bolting MTfp onto I2S, just as biOasis and Scarpa have done, and suddenly Elaprase, however unique it might be, is relegated to history.
As a business model, you couldn't ask for a better situation, and this is something that too many investors don't understand. We're a carrier. We're FedEx. We don't care what's in the package. We just promise that we'll get it to New York by noon tomorrow. That's it. And we're better at it than anybody else. It actually looks like we're the only one that can do it.
The bottom line is this, however great Elaprase might be, Shire can't get it into the brain without using a pump and a hose connected directly into the spinal column. However great the engineering is, it's still brings to mind something essentially medieval. Time moves on, discoveries are made, old methods are dropped. If MTfp-I2S effectively treats MPS II in both the brain and periphery, as has been shown by biOasis in mice, then that's where the business will go.
FedEx: When it absolutely, positively has to be there overnight.
biOasis: When it absolutely, positively has to be there right now
