Ryan, There is quite a bit of info on the LGND thread, so I'll answer your specific question on the FDA reforms here. LGND like most Biotechs focusing on life threatening diseases will benefit from the accelerated review. In the case of cancer, the FDA has already undergone some revisions which allow for easily and quickly measured parameters like tumor shrinkage to be used as an acceptable end-point (in contrast to a much more difficult end-point such as decreased mortality or increase life span).
In addition to the above, LGND will benefit greatly from the new regulations regarding off-label use. LGND has compounds (Panretin and Targretin) in Phase III clinicals for small cancer indications (Kaposi Sarcoma and Cutaneous T-Cell Lymphoma). Both compounds have shown promise for other tumor types and in fact are in phase II trials for many indications. The off-label provision will allow LGND to promote approved products for additional uses. LGND's timing is excellent, because the compounds could be on pharmacy shelves sometime next year and off label use will allow LGND to take some of the Phase II or initial Phase III data which should be complete next year and promote for additional indications.
The biggest market will be type II diabetes. LGND has started a small Targretin Phase II study in Europe. This study is to determine optimal dosing of Targretin and to generate some clinical data for a mega diabetes deal. The deal may be announced as early as next week. After signing, the partner will aggresively initiate large Phase II or III trials. Next year when Targretin is approved for CTCL or lung cancer, it could be promoted for diabetes, base on Phase II or initial Phase III data.
Targretin's diabetes applications are particulaly exciting, because Targretin, a rexinoid, is a natural parttner for TZDs such as WLA's blockbuster, Rezulin. Rezulin ands Targretin each activate a different receptor (PPAR gamma and RXR alpha, respectively) and the two receptors form a complex and then turn on genes to overcome insulin resistance, the underlying cause of type II diabetes. Although each compound works well alone, they produce additive effects when taken together (at least in mouse models of diabetes and obesity). Thus the new FDA legislation will allow LGND and its partner (JNJ?) to promote Targretin for type II diabetes while it is still in Phase II or Phase III trials because it will have already been approved for cancer. |
