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PBYI:
I didn't see anyone post this one of the better SEC filings I've seen in a while (for entrainment at least) .
http://www.sec.gov/Archives/edgar/data/1033409/000119312515399745/d103340ddfan14a.htm
Got it from someone on twitter sorry can't recall who first posted it.
Setting The Pace In The Race To Treat NASH
Posted by Rosana Kapeller in From The Trenches, Translational research
http://lifescivc.com/2015/12/setting-the-pace-in-the-race-to-treat-nash/
h/t Bruce Booth @LifeSciVC
I agree very interesting letter.
bluebird ASH Investor Event
Webcast 8:30pm ET
http://edge.media-server.com/m/p/gzjrqmdb/lan/en
I still don't own a (Pure) GT play I am actually starting to like the field more as a few things are starting to happen (probably in this order):
1-Safety is proven out
2-Valuations are more realistic
3-Expectations are coming down to earth
Is it a particular iteration that cannot be dosed more than once or is the problem bigger than that? The idea of yearly (or some other longer interval) treatment is actually a better business model and has less hurdles than once and done .
I still prefer indications that have little/poor other treatment options and ones that the potential to greatly increase/improve life expectancy/quality so the risk tolerance may be higher.
AZN / FGEN:
I posted this on SI. I only skimmed through the call for pertinent discussion on Roxadustat so can't comment on anything else (its 1:50 in total length).
Event URL: http://engage.vevent.com/rt/azemea/index.jsp?seid=1549
The presentation was straight forward not too much depth (starts around 32 min mark +/-)
There were a couple questions in the Q&A
1. (Around 1:20 mark +/-) About the CV safety. For dialysis they are looking at superiority over EPO (meta analysis of all the studies), powered for 20% safety improvement but that is not required for approval. For the non-dialysis saying they should be equal safe as placebo.
2. (Around 1:28 mark +/-) Shortening time to US filing (from 2018)? Said very unlikely only said event driven trial & need sufficient number to do CV analysis (no talk of interim).
3. (Around 1:30 mark +/-) Given less aggressive hemoglobin targets today how it effects potential to show CV risk reduction. Think doesn't matter much if anything works to advantage.
Does anyone know the (proper) procedure for obtaining a drug to compare it to an inhouse drug or for further research? For example when a company is developing a generic do they just have a doc write a perscription despite its use not being for a patient? What if the drug has a REMS? I recall tracleer tried (is trying) to keep generics out via this route.
I became curious following this twitter thread. Is this legal?
https://twitter.com/markbaum4/status/672835449480605696
KBIO / Turing:
Why acquire the drug via KBIO and not Turing?
I heard the (short) call and in the remarks Martin (also slide 25) said about exploring possible deal with Turing though no assurance of consummating one. I imagine a (reverse) merger is still on the table.
While they opened the call for Q&A there were no questions on the call. Its only about 10 minutes (less the opening disclaimer) but seems to be script from the slide deck so you can look at that or listen .
http://ir.kalobios.com/eventdetail.cfm?EventID=167900
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: Clarified 12/7 ASH presentation, Consolidated ALNY R&D Day
Credit Suisse Annual Healthcare Conference
11/9-12
https://cc.talkpoint.com/cred001/110915a_ae/
Incyte RA-BEGIN and RA-BEAM presented/call
11/11 9:00am
http://investor.incyte.com/phoenix.zhtml?c=69764&p=irol-calendar
Barclays SMID Cap Conference
11/16
Stifel Healthcare Conference
11/17-18
http://www.tweisel.com/Stifel/ConferencesAndEvents/Webcasts/
Canaccord Genuity Medical Technology and Diagnostics Forum
11/19
http://www.canaccordgenuity.com/en/cm/News-Events/Events/
Jefferies Global Healthcare Conference
11/18-19
http://wsw.com/webcast/jeff92/
BioFEST
11/19-20
http://www.biofest.in/2015/main/
LD Micro
12/1-3
http://wsw.com/webcast/ldmicro9/
Piper Jaffray Healthcare Conference
12/1-2
http://www.piperjaffray.com/2col.aspx?id=365
Ophthotech 2015 Investor Day
12/3 8:30am ET
http://edge.media-server.com/m/p/rkrqbsvw
SGEN Investor and Analyst Event at American Society of Hematology Annual Meeting
12/7 8:00pm ET
http://edge.media-server.com/m/p/zn3w9se8
Lilly Animal Health and Alzheimer's Disease Review
12/8
https://investor.lilly.com/eventdetail.cfm?eventid=161845
Oppenheimer 26th Annual Healthcare Conference
12/8-9
http://www.opco.com/conferences/healthcare15/index.aspx
ALNY R&D Day webcast
12/10/15, 8:00-11:30am ET
http://edge.media-server.com/m/p/fy9m5tbr
Valeant Investor Day
12/16 8:00am ET
http://ir.valeant.com/investor-relations/events-and-presentations/Event-Details/2015/Valeant-Investor-Day/default.aspx
Biotech Showcase 2016
1/11-15
http://www.ebdgroup.com/bts/index.php
J.P. Morgan 34th Annual Healthcare Conference
1/11-14
http://jpmorgan.metameetings.com/confbook/healthcare16/login.php
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Procedure for Updating Calendar
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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, Consolidated Updates, Updated Conferences and links for December/January
Credit Suisse Annual Healthcare Conference
11/9-12
https://cc.talkpoint.com/cred001/110915a_ae/
Incyte RA-BEGIN and RA-BEAM presented/call
11/11 9:00am
http://investor.incyte.com/phoenix.zhtml?c=69764&p=irol-calendar
Barclays SMID Cap Conference
11/16
Stifel Healthcare Conference
11/17-18
http://www.tweisel.com/Stifel/ConferencesAndEvents/Webcasts/
Canaccord Genuity Medical Technology and Diagnostics Forum
11/19
http://www.canaccordgenuity.com/en/cm/News-Events/Events/
Jefferies Global Healthcare Conference
11/18-19
http://wsw.com/webcast/jeff92/
BioFEST
11/19-20
http://www.biofest.in/2015/main/
LD Micro
12/1-3
http://wsw.com/webcast/ldmicro9/
Piper Jaffray Healthcare Conference
12/1-2
http://www.piperjaffray.com/2col.aspx?id=365
Ophthotech 2015 Investor Day
12/3 8:30am ET
http://edge.media-server.com/m/p/rkrqbsvw
Investor and Analyst Event at American Society of Hematology Annual Meeting
12/7 8:00pm ET
http://edge.media-server.com/m/p/zn3w9se8
Lilly Animal Health and Alzheimer's Disease Review
12/8
https://investor.lilly.com/eventdetail.cfm?eventid=161845
Oppenheimer 26th Annual Healthcare Conference
12/8-9
http://www.opco.com/conferences/healthcare15/index.aspx
Valeant Investor Day
12/16 8:00am ET
http://ir.valeant.com/investor-relations/events-and-presentations/Event-Details/2015/Valeant-Investor-Day/default.aspx
Biotech Showcase 2016
1/11-15
http://www.ebdgroup.com/bts/index.php
J.P. Morgan 34th Annual Healthcare Conference
1/11-14
http://jpmorgan.metameetings.com/confbook/healthcare16/login.php
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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.
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OCRX: I don't recall exact time line though I recall them saying about opening more sites in Europe and with the summer slow-down there is a reason to be optimistic about an uptick in enrollment going forward.
The clinicaltrials.gov entry hasn't been updated in some time as of March they didn't have any x-US sites listed.
https://clinicaltrials.gov/ct2/show/study/NCT01966419
Are you serious? I think you need to check some facts/math. You asked a question I answered it with a back-of-the-hand calculation that is reasonable no exaggeration!
SRPT:
They are still enrolling so say they finish enrolling 1H '16 add 48 weeks for treatment, 3-6 months to gather data/file and then 8 month priority review it could be a 2.5 +/- year delay.
SRPT / BMRN:
Appreciate your comments. I would say the gap in our views is diminishing .
I would say the risk in a SRPT approval is increased post briefing docs/panel but without seeing the docs and in particular the questions its hard for me to quantify. I think baring strong pressure from the DMD community the FDA's preferred solution will be to nicely say lets wait and see the results of the confirmatory study (I'd even venture that is one of the questions put to the panel).
Even if I were not long SRPT I am curious to see the briefing docs to see their points of concern (aside from the obvious ones investors expect, small n, dystrophin quantity/measurement, single site, etc.).
While it could be the company (SRPT) is misleading investors I get a much better tone with Ed Kaye and would expect just around the edges differences and not major ones so that leaves me puzzled with:
1-Why did the FDA ask for a historical control if they don't put much value on it (based on briefing docs/panel)
2-Why did FDA even accept package if data were insufficient shouldn't they have not accepted file to begin with? Is it only to satisfy outside pressure and have public review or does pressure go beyond that?
3-To your point about FDA wanting 2-3yr RCT if you check the confirmatory study while the randomized period is 48weeks they do have 96 week measurements so I am guessing it was similar to RNA's agreement with perhaps a bigger compromise and saying we will collect 2 year data. It may actually turn out to help SRPT as I agree that 48 weeks seems hard to show a treatment benefit though we may end up in the same place with comparison to historical control for the second year.
BMRM Adcomm:
I am long BMRN (& SRPT) and I would approve it. That being said I don't think it gets approved with current data and I don't think BMRN does another trial (unless approved in EU and need to). I think the documents and panel (the questions in particular) were almost setup to get a no sentiment.
I actually came away from the presentation thinking the data is not as bad as what I read from the documents. Maybe FDA was trying to be less confrontational but the questions from panel on the smaller studies especially made me think they weren't as negative as the docs made it out to be.
For those that say in any study some will get better some will get worse regardless of treatment the one thing I find a bit harder to sign off on is several families claiming their son would deteriorate during the period drug was suspended and then stabilize after drug was started (sometime afterward). From what I've learned in DMD that is unusual. I wonder if there is some subset of patients that benefit for some (as yet) unknown reason?
Drugs on demand
Controversy in Brazil over access to a purported cancer cure could set a harmful precedent.
h/t steve usdin @steveusdin1
http://www.nature.com/news/drugs-on-demand-1.18873?WT.mc_id=TWT_NatureNews
I've heard this is a very big story in Brazil. The university was ordered to make the drug available to anyone who wanted it. Aside from obvious concerns about its stage of development the university said they don't have the ability to manufacture the drug especially to the scale needed with the huge publicity it has gotten.
When I first heard about it I did a tiny bit of research and while I don't think its a good idea to have a university make an unproven drug available its at least a viable drug candidate which perhaps may show efficacy in some indication (if its every given the opportunity).
People on Adam's blog reported the same with the first link. During the intermission I got a message saying to switch to the new link. Perhaps they reached their quote on that one too?
BMRN FDA Panel Webcast
The Free link was changed to the following (presumably to handle demand with additional capacity)
https://collaboration.fda.gov/pcnsac112415/
BMRN / SRPT / PTCT:
Some links for those interested:
Free Webcast: https://collaboration.fda.gov/pcns1115/
Briefing Material: http://www.fda.gov/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/PeripheralandCentralNervousSystemDrugsAdvisoryCommittee/ucm467181.htm
Adam Feuerstein Live Blog: http://www.thestreet.com/story/13374430/1/biomarin-fda-panel-live-blog.html
Yeah what a conservative tone! I was hoping to go back to the mid 3's and exit the remainder of my position in the mid 2's no thanks I think I will hold on.
MOC:
I don't know if the company will issue a PR of this when the news was first announced 1/6/15 the volume was over 5 million and over 500k when confirmed on 6/18/15 so I would imagine a PR would get a lot more attention then is happening today.
MOC filed an 8k award confirmed for 250M contract
http://www.sec.gov/Archives/edgar/data/864509/000114420415067374/v425415_8k.htm
BMRN Briefing Doc:
Granted its one thing to approve a drug vs. accept/review but considering FDA reconsidered and accepted basically an open label trial in leu of a RCT trial seems to go against their standard so wouldn't that alone imply some sort of pressure?
I also think Janet Woodcock didn't do the agency any favors by being so chummy with the families.
BMRN:
BMRN / SPRT:
Appreciate your observations.
I am still going through the documents though from what I've read the tone sounds as if the data is insufficient
1-It seems FDA is not even accepting the smaller studies as positive
2-The lack of supportive secondary endpoints
3-It would seem even if FDA becomes accepting of some efficacy the review may still be delayed as you noted they still want to see other natural history data and it appears some other analysis (risk/benefit) are needed.
While FDA may want 2-3yr RCT and given the limited benefit and patient variability it may certainly be so, however, I highly doubt that either company would be willing to undertake that given the time to enroll and large number of dropouts one would expect and then there is the ethical aspect of basically asking a fairly young age person to take 10% of their life in a trial possibly on placebo!
BMRN / SRPT:
Yeah I guess you can't be 100% wrong if you take opposite views . Basically there are arguments for and against and I would venture FDA isn't 100% decided either. I don't get these analysis that say such and such percent chance of approval.
I am long both companies and don't see how the read thru of this document can be seen as having a positive or negative read thru to the SRPT. Drisapersen's data package has different issues than eteplirsen's. For example the dystrophin concern of FDA is not a 100% positive for SRPT as FDA may still questions the amount and accuracy of quantifying.
Being long BMRN I still see three positive scenario's (US, EU, or royalty on eteplirsen). With a negative vote I get the sense some may seek an opportunity as their pipeline has several near term catalysts (Pompe data, PEG-PAL data, Batten data/filing, etc.)
I don't believe FDA picked separate dates as a conspiracy for/against either company but rather as a matter of being a bureaucracy and being able to timely review packages submitted 2 months apart could not be sped up without (unfairly) delaying one. That being said there are a couple of conceivable consequences:
1-BMRN can argue there is no other (US) approved product. Panel can't put the positive's of one against the negatives of another.
2-FDA's document has some clear concerns and if they want to publicly make the case against it being efficacious and reasonably safe they can accomplish that without a complete public outcry as some will (mistakenly) think that eteplirsen is will be made available. However the FDA can fall back on the insufficient data and say lets wait for the confirmatory study to complete before approving (I believe that is what happened with DNDN). I still think BMRN did not start confirmatory studies because of the added pressure to approve now vs. waiting for data.
Being long SRPT I think the pressure to approve a drug outweighs the limited data my main concern is around FDA kicking the bucket down the road to wait for the confirmatory study. I thinking the fact that they accepted the file and told the company they would accept safety data from the ongoing studies and SRPT is complying puts some added public pressure to decide now baring some major uncertainty in the data that we don't know (particularly on the safety side).
KBIO:
For what its worth on his Youtube stream now he is doing an interview talking about KBIO and liking 003 in CML looking to start a trial soon with data in Q1/Q2 and go from there. Didn't catch it all but also noted other ideas/plans
Here is the link if anyone is interested:
https://t.co/Ez8X7Bon7v
My guess was he bought the position to do a reverse merger with Turing. As I recall rtrx was the same way (and I thought it cost him considerably more when factoring the cost of the equity he gave up). Given the sharp rise I wonder if scraps that idea and takes a short-term gain instead.
To echo what Peter said I've seen a couple (preclinical) papers that say the same thing about drugs not being able to reach the tumor in sufficient quantities. That is the same theory behind how Halozyme's PEG-PH20 is supposed to work.
FGEN DMD trial
The clinicaltrials.gov record is up for the non-ambulatory trial.
https://clinicaltrials.gov/ct2/show/NCT02606136
Management really is conservative its a 2 year treatment period! At least its open label if they see anything. Thought they may try to enroll more patients though.
The primary endpoint is FVC but they have a number of secondary endpoints.
Interestingly the dosing regimen is different then some of their other 3019 studies (35 mg/kg, every 2 weeks).
Ocera 11/17/15 Stifel Healthcare Conference Notes
IV program
- Length of stay of HE patients important more than 60% in for > 5 days, hospitals make money by day 4.
- Hyperion Phase 2 ammonia scavenger (Ravicti - glycerol phenylbutyrate) stat sig at reducing future episodes, hospitalizations and severity. Good harbinger since contains same active ingredient. (Horizon decided to terminate program because of use in urea cycle disorder - much higher price)
- 80% power to see difference at study end that was seen at planned interim. Primary is time to meaningful clinical improvement in encephalopathy symptoms (Present as K-M curve with log rank analysis).
- If slide 18 accurately represents the powering then it looks like benefit is close to 40 hours!
- stratifying enrolled patients for West Haven Scale at entry, most are 2 or 3 expect very few 4's
- Standard of care may vary a little bit but not restricted in what is used.
Oral program
- Now deciding if moving forward with 1 or 2 (that look most promising of the 3) of the oral candidates to optimize and move forward with.
- Feel confidant have a BID formulation and possibly QD going forward
- Optimize formulation and repeat (phase 1) middle of next year.
- See IV as $500-600M opportunity, Oral as $800-900M opportunity (US only)
- COM patent 2030
OCRX: Presents at 4:30pm ET today at the Stifel Conference. Don't know the format if Q&A and not just presentation perhaps they get asked about the PK study and the need to optimize. The direct link for those interested is
http://www.veracast.com/webcasts/stifel/healthcare2015/33112542233.cfm
Is the layman's translation reading between the lines that there was a spike in the concentration and they want a more consistent dose? I was hoping they could get this into Phase 2 (patients) relatively soon. I guess given the mechanism showing they can get the desired concentration in healthy subjects is at least encouraging for ammonia lowering activity in patients.
As I recall they previously did a short thesis on Questcor. I have not seen the *new* one on Mallinckrodt but if the thesis centers around acthar I wonder if there really is anything new or not. Though they get a lot of notarity and seem to move stocks in the short term I haven't been overly impressed with Criton reports when I've read them. They seem to find a couple of points and then over sensationalize them.
Very sad. I remember when he was coming up through the minors and then right before going to the majors had some dominant outings. He had lost a bit on his fast ball by the time the Braves traded him to the angels there was talk of arm problems but I didn't keep up. Prayers for his family and friends. He and Kris Medlen were good friends and I believe Medlen actually started Hanson's twitter account I checked it this morning and nothing on it for a few years.
It doesn't specifically says so but my reading is that it works for delaying onset of dry AMD and slowing the progression of dry to wet. Is that your reading too?
Since the FoB's are being named uniquely once a patient starts on Brand/generic do you envision less switching (then traditional generics) because of potential safety issues (immuniginicity)?