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Appreciate your and iwfal's points of Adaptive Trials. I can't add much to the discuss that hasn't been pointed out. I was curious on how the statistical analysis would be handled which you gave a couple possibilities for. If I recall correctly Emil had said about the possibility to enrich the trial by enrolling different types of patients (more likely to show benefit in a shorter period of time). Perhaps iwfal's skepticism is well founded and only those companies already good at running trials would navigate Adaptive Trials efficiently. The WSJ was more argue to the point of just how blinded are Placebo controlled trials along with patients gaming the entry criteria and some quitting when know not on study drug.
The FDA side is up now
http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/BloodVaccinesandOtherBiologics/BloodProductsAdvisoryCommittee/UCM407012.pdf
Link to all documents:
http://www.fda.gov/AdvisoryCommittees/CommitteesMeetingMaterials/BloodVaccinesandOtherBiologics/BloodProductsAdvisoryCommittee/ucm386681.htm
I believe there will also be a free webcast at this link:
https://collaboration.fda.gov/bpac714/
Webcast Calendar
[Please see updating procedure at
the end of this post. All times are
U.S. ET unless indicated otherwise.]
NOTE: ANYONE MAY UPDATE THIS FILE
Edits: Removed entries > ~1 month old. Added Wedbush and Canaccord Conferences
Wedbush 2014 Life Sciences Conference
http://wsw.com/webcast/wedbush28/
8/12-13
34th Annual Canaccord Genuity Growth Conference
http://wsw.com/webcast/canaccord14/
8/13-14
Morgan Stanley Healthcare Conference
9/9
Bank of America Merrill Lynch Healthcare Conference
9/17-19
UBS European Conference
11/12
Stifel Healthcare Conference 2014
11/18
Jefferies Healthcare Conference
11/19-20
Oppenheimer 25th Annual Healthcare Conference
12/10
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Researchers Fret as Social Media Lift Veil on Drug Trials
Maybe "Placebo" controlled trials should no longer be consider the gold standard they once were...
http://online.wsj.com/articles/researchers-fret-as-social-media-lift-veil-on-drug-trials-1406687404
Emil Kakkis has been a big advocate for an adaptive trial design (in particular for rare diseases where populations are small and efficacy could be small/take long to show significance). Perhaps its time to explore these/other changes to speed development?
So how has a higher percentage of cash per share KBIO or TRGT?
Do you see any value in KBIO? I had looked at them a long time ago but when the CFO left (former BMRN CFO) I lost interest.
Morphosys / Roche / gantenerumab:
Morphosys today announced the royalty rate 5.5% - 7%
http://www.morphosys.com/pressrelease/morphosys-ag-reports-results-first-six-months-2014
CC Transcripts:
I use Seeking alpha and sometimes morning star as well. I have others bookmarked (below) but haven't used the others much to vouch for how good/bad they are:
http://www.morningstar.com/earnings/
http://media.corporate-ir.net/media_files/Event/2002/dec/ccbn/vagenda.htm
http://www.conferencecalltranscripts.org/
http://seekingalpha.com/search/transcripts (Seeking Alpha)
OT:
An End To Our Relationship With Yahoo, A New Era For SA Contributors
Thanks to @PropThinker for pointing this out.
http://seekingalpha.com/forum/showthread.php?tid=23495
I am not active on seeking alpha but probably over 90% of the articles I read were a result of a link from yahoo with the stock quote. I imagine a lot of other readers did the same. Wonder if this will significantly reduce the speculative moves articles have caused in the past.
Edit: Apparently you need to be logged in to Seeking Alpha to read the posting.
Changes:
AL East Winner: O's (from Yankees)
2nd in AL West: A's (from Rangers)
AL Wildcard #1: A's (from Rangers)
TB #1: Most HR's: Jose Abreu (White Sox) (from Davis O's)
Jazz Pharmaceuticals Announces Agreement To Acquire Rights To Defibrotide In The Americas From Sigma-Tau Pharmaceuticals, Inc.
http://investor.jazzpharma.com/phoenix.zhtml?c=210227&p=irol-newsArticle&ID=1944485
IMO JAZZ did a nice job buying up the NA rights for Defibrotide. I don't know that FDA approval will be easy with the current data but they didn't pay very much for the rest of NA rights. I guess another buyer (of GENT) could have done the same thing. Good job by Jazz I probably should have bought Jazz with my GENT proceeds but I couldn't get over passing on Jazz years ago (after they bought Orphan Medical).
There was also a conference call on the deal.
http://investor.jazzpharma.com/phoenix.zhtml?c=210227&p=irol-EventDetails&EventId=5163585
ARIA:
The 8-k is out. Seems some of the votes were relatively close no wonder they were busy calling shareholders.
http://www.sec.gov/Archives/edgar/data/884731/000119312514249677/d746940d8k.htm
They had convertible debt from a prior offering that didn't mature until 2017. They came to terms with some of the debt holders to convert early since the stock was well in the money (they converted at 12.77). Not unusual in fact I am pretty sure they even did something similar many years ago my first time as a shareholder.
ARIA:
I believe everything passed but no vote count was given. An 8-k will be released with the official vote (didn't see it yet).
ARIAD Annual meeting earlier today was webcast. Slides and webcast available here:
http://investor.ariad.com/phoenix.zhtml?p=irol-eventDetails&c=118422&eventID=5147033
it is worth a lot more than $7B longer term because it validated VRTX's corrector approach to combo
Well they could always go back for seconds
Thanks!
I noticed it seems to be a smooth transition based on the advance notice. Just from conference calls I didn't put much value on the CEO so I'm not that shaken by it but you would know better than me
I actually thought people would get more excited about the ability to deliver enzymes orally. I don't know if the market doesn't buy it yet or I underestimated the value.
I wasn't sure I understood the PR is it 5 days of dosing +2 day washout so 4 total doses (28 days)?
Do they not have tox data to dose for a longer period? Any idea on when we might get a longer duration 2B?
Thanks in advance as always!
BTW, I've been long for a little while now
If that is a quiz I would guess Replagal and Fabrazyme but TKT didn't get approved first so maybe they were a little after?
Thanks jq. I though blue too risky (for me) but certainly would like to see gene-therapy move forward. I had asked a while ago (I believe on SI) how much of blue's valuation was attributed to this program vs. the Celgene alliance to try to better understand the risk. In the end I feel more comfortable with something like BMRN where it could be substantial upside but a fraction of their current valuation (I doubt anyone is putting any value on one indication much less multiple):
http://www.bizjournals.com/sanfrancisco/blog/biotech/2014/04/hemophilia-biomarin-bmrn-factor-ix-factor-viii.html?page=all
Your reply makes sense about focusing on a subset of patients most at risk. I'll have to start following gene therapy programs more closely.
GLPG:
The R&D Webcast begining in a few minutes is available here:
http://www.media-server.com/m/p/ynnq8y9p
blue:
Thats about 100 million market cap per patient. Thats a pretty good market cap increase per patient .
I don't follow them closely so can't make a comment on whether its justified or not. I am curious though what type of long term safety data the FDA may want to see (BMRN has a gene therapy program with big market potential). Does anyone know if there is even guidance out on that?
Actelion:
Selexipag meets primary endpoint in pivotal Phase III GRIPHON outcome study in patients with pulmonary arterial hypertension
http://www1.actelion.com/en/journalists/latest-news/griphon-results.page?
Selexipag decreased the risk of a morbidity/mortality event versus placebo by 39% (p<0.0001). Efficacy observed was consistent across the key subgroups; age, gender, WHO Functional Class, PAH etiology and background PAH therapy. Patients were treated for up to 4.3 years. The overall tolerability profile of selexipag in GRIPHON was consistent with prostacyclin therapies.
tolerability profile of selexipag in GRIPHON was consistent with prostacyclin therapies
Royalties:
Here is an interesting PDF on royalties. I was actually searching for something completely different when it appeared in my search results and caught my attention. I skimmed through it and while I am not sure about some of their assumptions (e.g. costs for clinical trials) I did find the (long) section of various deals, begining on page 59, very useful and worth holding on to for reference/comparison.
http://www.techtransfer.csir.co.za/wp-content/uploads/2012/06/Royalty-Rates-for-Pharmaceuticals-and-Biotechnology-7th-Edition.pdf
Thanks I signed up for it last night and sure enough Robby goes 0-4. I didn't get credit for Tulowitzki being 1-3 though. I guess if I get to 57 with this streak I'll owe you a cut but don't count on me winning .
Thanks I see they reached the number of events in february
http://www1.actelion.com/sites/en/scientists/development-pipeline/phase-3/selexipag.page
I was/am not too familiar with the program but it looks like they are enrolling pretty advanced patients (but not on parenteral therapy) based on the low 6MWK entry criteria. I didn't see it mentioned so I assume they can be on another oral agent (in Phase 2 they were). I find it interesting that 6MWK is a secondary not primary. Also it looks like the Phase 2 missed on 6MWK.
I think UTHR did a poor job getting their oral partially approved. IMO there was a strong evidence of a dose response (in mono and more importantly combo) though perhaps a better formulation would have helped increase the delivered dose. I'll give Actelion credit they are doing a better job of changing the endpoint and running much larger studies to try to show a perceived benefit over existing agents.
UTHR
Was down about 10% today. I haven't been following as closely lately anyone know why? I know Remodulin had a patent challenge don't know exact timeline for when the markman hearing is.
Wow thanks I've been picking a week or two at a time and just peeking in now and then. This kinda snuck up on me I guess I'll have to put more thought into the picks now or maybe I shouldn't mess with what works .
Webcast Calendar
[Please see updating procedure at
the end of this post. All times are
U.S. ET unless indicated otherwise.]
NOTE: ANYONE MAY UPDATE THIS FILE
Edits: Removed entry > ~1 month old. Added JMP & Wells Fargo
UBS 2014 Global Healthcare Conference
5/19-21
http://www.ubs.com/global/en/investment-bank/key-investor-conferences/global.html
Jefferies 2014 Global Healthcare Conference
6/2-5
http://wsw.com/webcast/jeff82/
Goldman Sachs Healthcare Conference
6/10-11
http://cc.talkpoint.com/gold006/061014a_an/
William Blair 2014 Growth Stock Conference
6/10-12
http://wsw.com/webcast/blair48/???? Where ????=presenting company symbol (sorry no link for webcast directory)
https://www.williamblair.com/News-and-Events/Events/2014/June/10/34th-Annual-Growth-Stock-Conference.aspx
Wells Fargo 2014 Healthcare Conference
6/17-18
http://cc.talkpoint.com/well001/061714a_ac/
Galapagos R&D Day Webcast from NY
6/17 8:00am
http://www.glpg.com/index.php/newspress-releasespress-releases/archive/webcast/
Citi European Healthcare One on One Conf.
6/18
https://www.seeuthere.com/rsvp/invitation/invitation.asp?id=m989a8e-1XQEUSUHGKT1Q&sutEventRoleID=m989a8e-4KU32HRLY2255&sutGuid=41803
JMP Securities Healthcare Conference
6/24-25
http://www.wsw.com/webcast/jmp24/
JP Morgan European Conference
6/26
https://www.jpmorgan.com/pages/jpmorgan/investbk/global/na/usconferences
Credit Suisse Healthcare Conference
7/1
Morgan Stanley Healthcare Conference
9/9
Bank of America Merrill Lynch Healthcare Conference
9/17-19
UBS European Conference
11/12
Stifel Healthcare Conference 2014
11/18
Jefferies Healthcare Conference
11/19-20
Oppenheimer 25th Annual Healthcare Conference
12/10
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Procedure for Updating Calendar
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2. Make your additions or modifications, inserting new items in alphabetical or chronological order as the case may be.
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The final on the official voting question #3
http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/AnestheticAndAnalgesicDrugProductsAdvisoryCommittee/UCM400200.pdf
VOTE: Should FDA require cardiovascular outcomes trials for peripherally active mu opioid receptor antagonists being developed for the treatment of opioid-induced constipation in patients with chronic, non-cancer pain? Discuss your answer.
a. Yes, for all peripherally active mu opioid receptor antagonists
b. Yes, but only for specific peripherally active mu opioid antagonists.
c. No.
Discuss your answer. If you choose option “b”, please specify which specific mu opioid
antagonists should be required to conduct a cardiovascular outcome trial and what
concerns form the basis for such a requirement.
Its a two day panel so I imagine the vote is just tomorrow. You can watch (free) here if your interested:
https://collaboration.fda.gov/aadpac614/
(use Guest login)
other info available here:
http://www.fda.gov/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/AnestheticAndAnalgesicDrugProductsAdvisoryCommittee/ucm390304.htm
Webcast Calendar
[Please see updating procedure at
the end of this post. All times are
U.S. ET unless indicated otherwise.]
NOTE: ANYONE MAY UPDATE THIS FILE
Edits: Removed entries > ~1 month old. Added GS link, William Blair Conference, GLPG R&D Day
Bank of America Merrill Lynch Health Care Conference
5/13-15
UBS 2014 Global Healthcare Conference
5/19-21
http://www.ubs.com/global/en/investment-bank/key-investor-conferences/global.html
Jefferies 2014 Global Healthcare Conference
6/2-5
http://wsw.com/webcast/jeff82/
Goldman Sachs Healthcare Conference
6/10-11
http://cc.talkpoint.com/gold006/061014a_an/
William Blair 2014 Growth Stock Conference
6/10-12
http://wsw.com/webcast/blair48/???? Where ????=presenting company symbol (sorry no link for webcast directory)
https://www.williamblair.com/News-and-Events/Events/2014/June/10/34th-Annual-Growth-Stock-Conference.aspx
Galapagos R&D Day Webcast from NY
6/17 8:00am
http://www.glpg.com/index.php/newspress-releasespress-releases/archive/webcast/
Citi European Healthcare One on One Conf.
6/18
JP Morgan European Conference
6/26
Credit Suisse Healthcare Conference
7/1
Morgan Stanley Healthcare Conference
9/9
Bank of America Merrill Lynch Healthcare Conference
9/17-19
UBS European Conference
11/12
Stifel Healthcare Conference 2014
11/18
Jefferies Healthcare Conference
11/19-20
Oppenheimer 25th Annual Healthcare Conference
12/10
--
Procedure for Updating Calendar
When adding or modifying entries, please follow these steps:
1. Copy the complete text from the old calendar.
2. Make your additions or modifications, inserting new items in alphabetical or chronological order as the case may be.
3. Near the top of the message, give a very brief description of your changes (e.g. “Edits: Added JPMorgan conference”).
4. Post the updated calendar in a new message as a reply to the message with the old calendar.
NKTR / AZ / PGNX / SLXP: June 11-12, 2014, Meeting of the Anesthetic and Analgesic Drug Products Advisory Committee (AADPAC)
The briefing documents are available here:
http://www.fda.gov/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/AnestheticAndAnalgesicDrugProductsAdvisoryCommittee/ucm390304.htm
A free webcast will be available to here:
https://collaboration.fda.gov/aadpac614/
Note: At the access page, please sign in as a guest. No password is required.
Thanks to @BioRunUp for the link
I was curious about their Dry-AMD drug and found this PR on their website. It seems they are using the same drug for Stargardt disease and Dry-AMD. I haven't looked at Pub-Med yet to see if there is anything interesting about modified Vitamin A to help in Dry AMD
http://www.alkeus.com/20110104.html
Is there a reason (Regulatory, mechanistic, etc.) why Halozyme was able to get prophylaxis heparin added to the protocol (as appossed to dose reduction) so quickly and yet for Iclusig after (relatively speaking) more analysis dose reductions are being explored (as opposed to adding aspirin, heparin)?
Naloxegol for Opioid-Induced Constipation in Patients with Noncancer Pain
http://www.nejm.org/doi/full/10.1056/NEJMoa1310246?query=TOC
Full PDF
http://www.nejm.org/doi/pdf/10.1056/NEJMoa1310246
Only one major cardiovascular event was considered by the investigator to be related to the study drug, and it occurred in the placebo group.
RTRX:
Was wondering why it was down early (thanks to @BioRunUp for the link)
http://www.secform4.com/insider-trading/1525354.htm
He is a lot smarter than me would figure he plans better for Est. Taxes... maybe he thought the stock would be a lot higher by now ?