Injecting a virus into the body and hoping for the best seemed like a real long shot to begin with. I believe AAVL will fail also. Extracting cells, modifying them outside the body, then reinserting has much more promise.
"Cured" is one of those amorphous terms. Cancer patients are typically called cured if there is no sign of the disease five years after treatment, but that doesn't mean it won't reappear in the future. "Not in need of treatment" is a better way to think about it. BLUE is batting 1.000 so far. All patients treated to date have reached a level of good globin, after which no blood transfusions have been necessary. There have been no serious treatment related adverse events. The longer this outstanding progress continues, the greater the confidence in BLUE's approach by the medical community, hopefully culminating in FDA approval.
The endpoints for SCD success are absence of symptoms and treatment related adverse events. What I would like to see at EHA for SCD is a table of patients like BLUE has provided for BT, with information like age/sex, date of treatment, globin levels, etc. It's important that BLUE report serious AE's also. It could take time for SCD symptoms to dissipate as good globin levels rise, so BLUE may want to have six months to a year of data in hand before presenting efficacy results.
The FBT ETF, which tracks the biotech index, does own stock and will do a ton of buying and selling at the close.
Presented by Dr. Doberstein. Topics are in the order presented with notes. No mention of NKTR-171.
2. BAX-855 - PDUFA November, 2015, launch by the end of 2015.
3. NKTR-061 - Phase 3 complete 1Q16.
4. NKTR-023 - Phase 3 complete 2H16.
5. NKTR-181 - Two efficacy studies anticipated to last 18-24 months each. First with opioid-naïve patients started in February, second with opioid-experienced patients being designed. Interim analysis may result in increasing the number of patients in the study.
6. NKTR-102 - No additional NKTR-102 studies will be started until reviews with US and EU regulatory agencies are complete. Data will be presented at future scientific events. No timeline given.
7. NKTR-214 - IND in 4Q15. Combo treatment with CTLA-4 inhibitor resulted in durable response without further dosing in mice re-infected with tumor.
-- "Dr. Ivan Gergel was brought to the company for one reason only - to ensure that NKTR-181 meets its primary endpoint in Phase III and is commercially approved"
I believe that the same words were written about the late Dr. Robert Medve. It isn't easy. NKTR is going with another withdrawal test for Phase 3. This time, it's back pain instead of OA of the knee, NSAID use is not permitted and acetaminophen is the rescue drug. Under Dr. Medve's design, it would be clear that NKTR-181 was superior to NSAID's. That isn't clear from the new study design. Recent retrospective study reviews appear to show that acetaminophen is not effective against back pain. We will see if that affects study results. NKTR also could run a parallel arm efficacy study, NKTR-181 vs. NSAID's. That would provide clear evidence of the superiority of NKTR-181 to NSAID's.
This isn't a slam dunk by any means.
Retrospective review shows that acetaminophen is no better than placebo for relieving lower back pain. They want to avoid the use of NSAID's. Hopefully not many patients will need the rescue drug.
Search for "Common painkiller may not ease lower back pain or osteoarthritis".
As I commented to hoyas before, more important than initial weekly script numbers are the numbers of unsubsidized refills. ARNA Belviq had very good initial prescription numbers, but the first scripts were subsidized by the company. The number of refills dropped off quite sharply as customers had to start paying for it themselves. I don't think we can get a real feel for Movantik's potential until the end of the year draws near.
A related number is Movantik sales. There is typically an initial sales pop as pharmacies stock their shelves, so expect that in Q2. We'll see what happens after that.