Q: What's the most overhyped pharma story? A: PCSK9
It's quite well understood that although $AMGN is looking at HoFH patients (probably in the hopes of pulling off orphan drug status only), right out of the gate AMGN hopes its new PCSK9 drug will be the next statin. The problem is though, for orphan drug status, the drug candidate should treat less than 200K in the US (and there are millions more high LDL patients than that). Also, there needs to be a lack of current therapies, and the US has two - $AEGR's Juxtapid & $ISIS's Kynamro. Last but not least, the potential orphan drug needs to be "clinically superior" to the current therapies and PCSK9 hasn't proven its anything close to the current therapies for HoFH patients. Furthermore, for the 1/6 of HoFH patients in the last clinical trial who did benefit somewhat from taking PCSK9, due to their very high levels of LDL, these patients will most likely take BOTH the $AEGR (easy pill form) drug and $AMGN drug. Also, PCSK9 is supposed to be revolutionary for the cardiovascular pharma industry. Yet the revolution is here - it's over the counter statins, which work just as well (with a proven safety history). PCSK9 might sell to the approximate 3% (or less) who can't take statins, yet otherwise I don't really see even 3% of the "$10B/yr market" for AMGN, especially with all of the coming PCSK9 competition. Do you?
1. Evolocumab works by a different mechanism than either Juxtapid or Kynamro both of which have black box warnings.
2. Evolocumab may work in patients where statins don't work and that population is different than those who can't take statins.
3. Evolocumab can be taken with statins.
4. Not likely to be prescribed both Juxtapid and evolocumab.
Just my view.