Great article Kayaker. Sylvie Gregoire has demonstrated that she is not afraid to swing the bat and has been very successful at it. She was first out of the gate to recognize the value of Transcend and seized the opportunity to do a collaborative with Bioasis. Growing the Shire Human Genetic Therapies division from $330 million to $1.4 billion in 5 years is no small feat (beating even her own expectations). With their commitment to growing the CNS Lysosomal Storage Disease business (evidenced by the CNS programs for MPS II, MPS III & MLD programs), it seems that we have technology that is required to achieve those goals. Their current intrathecal programs for the CNS, are in my estimation, inferior to Transcend. There are a number of competitors that are knocking at Shire's door (Genzyme, Green Cross & Pfizer just to name a few), who in my estimation would be well served with having Transcend in their arsenal. Options for Shire's competitors to have BBB transport technology are growing slim since the deal earlier this year with GSK & Angiochem.
Admittedly, the Shire work program has taken longer than most of us had anticipated (maybe not everyone, but certainly longer than I thought), but it is the nature of how the "pharma beast" operates. Can't imagine that we have much remaining on the scientific front before we enter business development talks. Seems that there are only two options: Either the science didn't work and Shire is not interested (don't think that is a real possibility given our prior success with transporting Aldurazyme for MPS I into the CNS) or we are entering a timeframe that so many of us have speculated on, where one day in the not to distant future wake up to an NR stating Shire has exercised an option. In my estimation, Shire was the right company for Bioasis to collaborate with and is the most likely to exercise an option with a pre-clinical biotech.
If Bioasis can execute a licensing agreement with Shire, they will demonstrate that many other licensing deals can realistically be achieved (oncology, MS, ALS, pain management, huntington's, Alzheimer's, Parkinson's etc.). From an annual market perspective, Lysosomal Storage Disease is dwarfed by most all the mentioned CNS diseases. This is where the collaborative pipeline becomes important. We've done a collaborative with Abbott and I hear rumours that there are many others at the table as well. Being able to have precedence of a licensing deal with Shire, will cause the market to apply those licensing metrics to the other collaboratives. It is at this stage, that the institutional crowd is interested. They are not involved in our marketplace at the moment (at least not to any significant way), but a licensing deal will bring them and cause them to look seriously at the other ongoing collaboratives.
With respect to the TTU BT2111 program, results from the Phase III program should be arriving in the not to distant future. Delivering 6.6% across the BBB, success with the ADCC assay, success with the xenograft model & success in the pathology study all point to a single result in Phase III: Success in being able to treat HER+ brain metasteses with a $5.2 billion dollar a year drug.
PS: Rob's your travelling east coast uncle.