An exclusion criterion in the Eribulin Phase 3 was "Patients with brain metastasis presenting clinical symptoms". There is no such exclusion in the BEACON trial. The NKTR-102 Phase 2 explicitly excluded patients with known or suspected CNS metastases.
I suspect that the brain mets would be treated independently with surgery and/or radiotherapy whenever possible. That should be allowable under the study protocol. It kind of muddies up the drug vs. drug comparison, but what's most important is the outcome of the total treatment, as measured by OS.
Two large stockholders are no longer passive.
1. Mayflower Capital Partners BV, Willem A. M. de Vocht, 196,600 shares, 9.2% stake.
2. Eriksen Capital Management LLC, Tim Eriksen, 137,447 shares, 6.29% stake.
It was very informative. BLUE is in continuous interaction with the FDA and EMA. Related to registration, they are discussing how many patients need to be treated and for how long they need to be monitored to confirm durability of response. They expect the current BT and SCD studies to be fully enrolled in 2015. There is a new, improved vector for CCALD.
Here is how I interpret the entry. Suppose a patient receives chemo treatment monthly for one year and the cancer remains in remission for that time. After the twelfth dosing, it is discovered the cancer has progressed and the patient started on NKTR-102 one month following the final dosing. I believe the "Time from Last Chemo to Entry" would count as one month in that case. It seems like you would count that as one year. Correct me if I misinterpret what you think. To be considered highly refractory means that previous treatments are ineffective. If my interpretation of that entry is correct, there really isn't anything in the poster that directly conveys the refractory nature of the patients
You appear to refer to the table entry labeled "Time from Last Chemo to Entry". We have differing interpretations of that. Your seem to interpret it as measuring the time from when they started their most recent course of treatment. I believe it measures from the time they received their most recent chemo dosing. There is a world of difference in those meanings.
There are three biomarkers that are typically evaluated in breast cancer, estrogen receptor (ER), progesterone receptor (PR) and Her2. Triple negative means that all three evaluate as negative. A triple negative patient is Her2 negative by definition. And "Great Find"???? Come on, this poster has been on the NKTR website for years and is the basis for the BEACON study. Aren't you familiar with it? It has been discussed many times on this board and there are numerous threads about it.
Here's something else I had forgotten but needs mentioning again. The term "desperately ill" has been used to describe the patients in the Phase 2 study. As evidence, posters state that many patients entered the study about one month after progression. I do not think that the two are related, it says nothing about how long prior to the progression that the cancer was under control. But here's the key bit of information that slipped through the cracks. Patients entering Phase 2 were limited to a *maximum* of two prior courses of chemo in a metastatic or locally advanced setting. Patients entering the BEACON study were required to have a *minimum* of two prior courses, and it could range up to five courses. I'd say that patients entering BEACON were farther advanced than those in the Phase 2 on average. The BEACON entry criteria are virtually identical to those of the eribulin Phase 3.
Out of the 70 patients in the Phase 2, 64 of them were HER2 negative. Five were in the 14 day group, one in the 21 day group. So 34 out of 35 of the 21 day Phase 2 group were HER negative. It's pretty safe to say that the one HER2+ patient didn't radically alter the results.
Lest anyone forget the NKTR-102 Phase 2 results, I'll mention them again. The data is from a poster presentation on the NKTR website. NKTR-102 OS needs to beat TPC by 2+ months to achieve superiority.
The median OS of 13.1 months for the 21 day cycle was based on 35 patients. The OS of the 14 day cycle (also 35 patients) was 8.8 months. That's a monster difference. If you look closely at the Kaplan-Meier graph, you will see a large survival difference between the 16th and 17th patients in the 21 day cohort. Shift the curve one patient to the left and median OS becomes about 10.3 months. A single patient survival is probably the determining factor in running this $100M+ Phase 3!
Scaling the manufacturing is critical. 100 successful patients for approval will take about eight years at the rate BLUE is running now. 1000 patients per year after approval (BT and SCD) works out to about three per day. You have to get there from here by the end of the decade, if things go well in the trials.
One thing I hope they will discuss on the tenth is progress on increasing capacity. In his video, Leschly said that was what worried him most. BLUE is operating at about one transplant per month, that rate needs to be increased to one or more per week. I would like to know if they have had discussion with the FDA on the proper way to advance the program towards approval. I will be disappointed if they try to cowboy the program the way SRPT has done with eteplirsen.
Time for release of initial data from the NORTHSTAR study. Here are my expectations. Anything less, and I believe the stock will get whacked.
1. Patient 1102 - No additional transfusions required, patient deemed cured.
2. Patient 1104 - Path similar to 1102, may have required transfusions, BA-T87Q increasing monthly.
3. Patient 1106 - Cured from the start.
4. At least two additional patients being treated, no serious adverse events.
5. Three previous patients deemed cured, no serious adverse events.
Talk about slime of the highest order. Search for "Ex-Retrophin CEO Sold Stock While Publicly Urging Investors to Buy".
In September and October, Shkreli tweeted that he was continuing to buy RTRX stock and added dribs and drabs according for Form 4's filed at the time. He stated he hadn't sold a share. What wasn't revealed until today is that at the same time he sold millions of dollars of forward sales contracts for his RTRX shares at 68 cents on the dollar. So technically he didn't sell and still owns the stock and has voting rights, but he has to turn it over soon to the buyer.
Also, raising the price of the one day course of treatment with Biltricide from $100 to $100,000 wasn't a sarcastic comment by AF. It is part and parcel of Shkreli's business plan.
Tweets from AF today. Marty is trying to form another company.
@MartinShkreli now seeking approx $12M from outside investors for Turing, down from $25M.
@MartinShkreli intends to use money raised to buy Biltricide, an anti-parasitic drug sold by Bayer, per sources familiar with the deal.
Biltricide is 1-day treatment for parasitic worms, costs approx. $100. @MartinShkreli plans to raise price to $100,000, sources say.
The five years of marketing exclusivity with QIDP probably means less to NKTR/Bayer than it does to others. It is hard to imagine a generic drug manufacturing company wanting to replicate the inhaler even as it comes off-patent. And the Bayer formulation goes hand in hand with the inhaler design.
SRPT up 13% because competitor RNA bought out for 55%+ premium.
SRPT and RNA a textbook example of why management counts. SRPT had first and probably superior drug for Duchenne MD. But SRPT spent its time hounding the FDA for AA based on very small patient study and did little else to advance the drug. RNA took the slow road to advance to Phase 3. Now RNA is months ahead of SRPT, and has a much better shot at AA today. That's why BMRN bought them instead of SRPT.
With over six feet of snow in total, had driveway cleared and car on the street by Friday afternoon. My street was always passable even during the thickest of the snow. Living one block from the fire department has its advantages. There were nearby places with close to eight feet of snow, and people were complaining that their street hadn't been plowed by Friday night. There are very high expectations for the highway department.
If you own a payloader, you can write your own ticket. There are lots of people making Christmas money. You can work 24 hours a day if you are able.
There have been numerous catastrophic roof collapses with flat roofed buildings. A couple are close to me, including an auto body shop. "Mr. Smith, it will take some extra time to fix your car. We moved it inside to protect it from the snow, then...". Lots of people shoveling off roofs. My children's school is closed until after Thanksgiving, in part so they can get structural engineers to verify roof safety. There is also a lot of residential damage. It's a good time to be in the construction business. My damage was limited to a couple of outdoor lighting fixtures and some trees. A mature smoke tree was pretty much wasted. But nothing worth filing an insurance claim over.