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Here is the link to the full episode
http://www.nbc.com/dateline/video/dateline-june-14-2015/2871922?onid=209511#vc209511%3D1
NBC Dateline has an episode this Sunday on EPP http://t.co/U7VX9nyXVp
PCSK9 Panels:
If anyone is interested in the panel voting comments here are links courtesy of Jessica Adams @RxRegA
http://www.sac-tracker.com/emdac-20150609-rw
http://www.sac-tracker.com/emdac-20150610-rw
The Pentagon’s gamble on brain implants, bionic limbs and combat exoskeletons
Doesn't talk about specific companies but a nice article on the DARPA push into biological research. There is a nice audio file in the article too.
http://www.nature.com/news/the-pentagon-s-gamble-on-brain-implants-bionic-limbs-and-combat-exoskeletons-1.17726
PRTA:
Webcast Clinical Results from Phase 1 Study of PRX002
June 17 2:30pm PT
http://ir.prothena.com/events.cfm
OPKO and Bio-Reference Laboratories Joint Conference Call to Discuss Merger
6/11 8:30am ET
http://phx.corporate-ir.net/phoenix.zhtml?p=irol-eventDetails&c=84759&eventID=5195654
What 2 months is that? Do you take issue with their report of making 98% of the padufa dates in 2014 (see below link)? (I don't track it, and it wouldn't surprise me if the success rate of hitting padufa bounced around some - but I know of now sustained trend)
BTW - do you have some suggested process change that doesn't substantively increase the chance of a bad drug getting on the market?
I take the start of submission and not the "starts its review" but even by that standard the authors aren't quite accurate according to this chart
http://www.fda.gov/downloads/AboutFDA/CentersOffices/CDER/ManualofPoliciesProcedures/UCM218757.htm
Never-the-less the authors were implying the FDA process is quite good when they in-fact extended their review by 2 months which is moving in the opposite direction. Sure the stats can look better if you get more time to review to be considered within your guidelines.
I don't claim to be familiar with 21st Century Cures Act and I don't doubt it likely has points that I wouldn't support. As far as the review process is concerned I just get the impression the authors see only the risky aspects without weighing in on the potential positives aspect to get medicine to patients who may have no other options.
I reread it and could see how someone would interpret what the authors said as correct. But I contend its incorrect and at the very least misleading .
21st Century Cures Act:
I guess there are two ways to look at everything its unfortunate the authors chose the more restrictive is better. The authors lost a lot of creditability in my book by the time I got to the end of page 1 with their incorrect fact that contradicts (a little bit) their general thesis FDA is moving in the right direction. I will leave it as a quiz to see who can find it first
Immuno-Oncology:
I don't have a list and not sure if this qualifies as fringe but I have been long Nektar for a while now and their immunotherapy program is pretty under the radar (of course their 1.5B market cap doesn't make it a pure play here). They have used their PEG technology in pretty creative ways to develop some pipeline candidates. Their Immuno-oncology is very early though.
NKTR-214 which targets IL-2 is finally going into patients.
http://finance.yahoo.com/news/nektar-md-anderson-cancer-center-120000043.html
Here is a poster from last years ASCO (preclinical) combining with Checkpoint inhibitors or anti-PD-1 agents.
http://www.nektar.com/pdf/pipeline/NKTR-214/NKTR-214_ASCO_Poster_2014.pdf
They mentioned 2 other pre-clinical programs that they said they would talk about at their R&D Day (10/8)
NKTR-255 (IL-15 Cancer Immunotherapy)
NKTR-218 (IDO Inhibitor)
I have been long the stock for a couple years and recently bought more thinking their royalty revenue potential represents good value even at current prices and then you have a potential big opportunity if NKTR-181 hits (it failed Phase 2 but they went ahead into P3 with a modified trial) and the under the radar Oncology assets especially now that 102 isn't priced in. I think NKTR-214 is really interesting if they really got the toxicity issue around IL-2 reduced.
This is the slide deck from the jeffereies conference:
http://files.shareholder.com/downloads/NKTR/0x0x833274/536E44DF-E108-4C26-B9FC-39D57C2EDA9E/FINAL_JEFFERIES_NKTR_Presentation-06022015.pdf
And here are the links to the webcast:
http://wsw.com/webcast/jeff88/nktr/
http://wsw.com/webcast/jeff88/nktr.bo/ (break out session)
I would be very interested in hearing others opinion.
The company posted an update on Scenesse Commercialization
http://www.asx.com.au/asxpdf/20150605/pdf/42z0rjjm90p2rm.pdf
The name sounded familiar for some reason so I googled him.
Here is his LinkedIn profile he matches the background of the litigation.
https://www.linkedin.com/pub/michael-flannigan/12/324/b04
This is interesting
https://www.sec.gov/litigation/opinions/34-47142.htm
Sounds like the same person from the firm with the "analyst report" (its basically one page talking about Gottbetter Capital and 4 std pages of filler and they try to get your email address to get it).
http://www.brokerbanksecurities.com/contact
I'd venture its a good bet the SA person/group and this are related or at least broker bank is trying to ride the coattails of the SA person/group.
OCRX:
Do you know if the enriching by excluding the quick responders is a modification to the enrollment criteria after the interim or if it was existing from the start of the trial? The clinical trials record doesn't say one way or the other.
https://clinicaltrials.gov/ct2/show/NCT01966419
For those interested there were no slides on the webcast but on the website there is a June slide deck (Though the PDF has a May name) that I think Linda was referring to. Here is the direct link:
http://files.shareholder.com/downloads/ABEA-5YQ58G/0x0x825938/4D2E5766-F904-49C8-9864-F09130444F72/Ocera_Investor_Presentation_-_May_2015.pdf
Here is a link to EKSO's response posted on their IR page
http://ir.eksobionics.com/seeking-alpha-faq
Is there a specific item you are concerned with?
I did a skimming of the article and found it to be generally taking small bits of truth and either twisting something or making inference not necessarily there. Aside from that it was pretty obvious what the intent was and the author tried to be as overly dramatic as possible to "scare" (IMO). SO after I skimmed it I added 25% to my position.
The stock certainly has risks but that article was not *news* IMO and its not surprising that the effect is short-lived.
Axovant:
I saw the presentation (not paying full attention) and didn't get a high confidence in the presentation to bother to pursue DD. The slides had the lower dose (combined with Aricept I believe) matching the comparator alone at 24 or 48 weeks (don't recall). They did mention Lundbeck is in phase 3 with 5-HT6 antagonist.
Here is the lastest S-1A if anyone is interested (I'm not at this point).
http://www.sec.gov/Archives/edgar/data/1636050/000104746915005125/0001047469-15-005125-index.htm
Axovant Sciences Ltd.:
Alzheimers Company is on retail roadshow. Claim positive 600+ patient study.
http://t.co/RSLIgM4J5v or
http://www.retailroadshow.com/sys/launch.asp?k=44370190867
(no opinion on company haven't looked at them yet)
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, Updated June Events. Added July/August Events
Bank of America Merrill Lynch Healthcare Conference
5/11-14
UBS Global Healthcare Conference
5/18-20
http://www.ubs.com/global/en/investment-bank/key-investor-conferences/global.html
http://ubsiconf.dealogic.com/iConference2/Conferences/Custom/List/180?menuItemId=100
BioEquity Europe
5/19-20
http://www.biocentury.com/conferences/bioequityeurope/dates
Jefferies 2015 Global Healthcare Conference
6/1-4
http://wsw.com/webcast/jeff88/
William Blair Annual Growth Stock Conference
6/9
http://wsw.com/webcast/blair49/???? where ????=Symbol of presenting company (sorry couldn't find way to see all presenters)
Goldman Sachs Healthcare Conference
6/10-15
http://cc.talkpoint.com/gold006/060915a_ae/
Citi European Healthcare Conference
6/15-16
BIO International Convention
6/15-18
http://convention.bio.org/schedule/
JMP Life Sciences Conference
6/23-24
http://wsw.com/webcast/jmp27/
2nd Annual ROTH Healthcare Day
6/24
http://www.roth.com/main/Page.aspx?PageID=7005
J.P. Morgan European Health Care Conference
6/25
https://www.jpmorgan.com/pages/jpmorgan/investbk/conferences/european_healthcare
Cantor Fitzgerald's Healthcare Conference
7/8
Wedbush Life Sciences Management Access Conference
8/12-13
Rodman & Renshaw 17th Annual Global Investment Conference
9/8-10
http://www.meetmax.com/sched/event_30869/~public/conference_home.html?event_id=30869
--
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.
Sorry about that here is the corrected full link
http://onlinelibrary.wiley.com/enhanced/doi/10.1111/bjd.13598/
I often wonder why companies go to these conferences and not webcast which would potentially reach a much greater audience then those who attend. I guess some may not have the cash for the webcast fee .
2015 Marcum Microcap Conference
There are actually a couple of (fringe?) Bio's with webcasts today if anyone is interested the link for today is below (registration required):
http://wsw.com/webcast/marcum3/#table2
I wonder if at some point a company may proactively purchase one (since so few available) to keep a competitor from getting first mover advantage.
PPV's:
Its interesting how much these have gone for. When BMRN got theirs I had no idea what type of value to place on them and when they sold it (I think 60-70m) I thought it was a good price obviously not by the later deals.
I think these tend to be of greatest value to two groups the sellers if they are small biotech to raise cash without diluting and to the buyers (though there is a large market then I thought). For BMRN it really didn't appear to be of much value since most of their pipeline will get priority review anyway. Similarly UTHR said they did not see 100-200M of much value for them what they hope to do is be able to trade it to get rights to a compound (they mentioned specifically perhaps getting an approved compound but pediatric use).
U.S. House committee approves bill to speed new drugs to market
http://www.reuters.com/article/2015/05/21/usa-medicine-house-idUSL1N0YC1OV20150521
The bill, known as the 21st Century Cures Act, requires the Food and Drug Administration to incorporate patient experience into its decision-making, streamline its review of drugs for additional uses, and consider more flexible forms of clinical trials.
Neurocrine Biosciences Analyst and Investor Day today at 12:00pm ET
http://phoenix.corporate-ir.net/phoenix.zhtml?p=irol-eventDetails&c=68817&eventID=5194200
http://lifesci.rampard.com/20150521/reg.jsp (Webcast Link)
IPF:
Esbriet and Ofev are not without their side effects. I'd imagine discontinuations will continue to be significant for both especially since efficacy is modest and likely patients’ perception is even less benefit.
Some long term data on Scenesse was published in the British Journal of Dermatology
Long-term observational study of afamelanotide in 115 patients with erythropoietic protoporphyria
http://onlinelibrary...1111/bjd.13598/
IPF - FVC as an acceptable endpoint:
Intermune didn't directly say so but I think it was pretty much implied when they were delaying in starting the CAPACITY studies. They had talked about getting an SPA and then proceeded to start the Phase 3 despite not getting it. I remember Dan Welch saying something to the effect of disagrement between the understanding of the disease. He worded it as the company's understanding of IPF was more advanced then the agency. I didn't buy it at the time but give them credit in the end the FDA did accept it. Also at the ADCOM one of the things the agency asked the panel to vote on was meaningfulness of 10% FVC decline.
FGEN:
You have some valid points about what the company says about their results and their corresponding progress in advancing 3019. I am tending to believe the data but don't have an answer as to why its so slow.
With the strict reimbursement in Europe if they want to accept patients with lower FVC they may still have a good pool of patients given the size of this "Orphan" indication. Plus Esbriet and Ofev are not without their side effects. I'd imagine discontinuations will continue to be significant for both especially since efficacy is modest and likely patients perception is even less benefit.
FGEN:
I am actually long more for 3019 while realizing that today the majority of the value is in the anemia program. This is one stock (I hope) to hold on for many years when 3019's value in multiple fibrotic diseases aside from IPF could be realized.
Peter posted some things about them being unable to find the right patient population. Intermune did delay a while with Pirfenidone (and the molecule was around for decades) but part of that was they had Actimmune that they thought would work.
Intermune claimed in both CAPACITY and ASCEND that a small percentage of the patients actually drove the outcome. In ASCEND 46 (out of 278) Pirfenidone patients had > 10% decline vs. 88 (out of 277). So basically 70% of the placebo group had no impact on the primary end point. So I think FGEN doesn't have to be perfect in selecting the right patients. Additionally Pirfenidone has never been shown able to reverse fibrosis like FGEN claims was done in some patients. What I wonder if the company is trying to find just these patients which may be an impossible task with the current understanding of the disease? Just pure speculation on my part.
ICPT:
I don't follow ICPT as closely as others here. In general I don't like co-primary endpoints in reading Adam's writeup it seems they missed on NASH resolution in Phase 2 granted the larger trial size should help here. That being said and not being knowledgeable here it would be a guess but I would guess higher than 20% odds of success
SRPT:
Sarepta Therapeutics Announces Plans to Submit Rolling NDA for Eteplirsen following Today’s Pre-NDA Meeting with the FDA
I know its a busy day in bio land but surprised no one posted this yet.
http://investorrelations.sarepta.com/phoenix.zhtml?c=64231&p=irol-newsArticle&ID=2050184
- Held pre-NDA meeting and agreed on rolling submission with the Agency -
- Will submit first two components of the NDA this week -
- Plans to complete the submission by mid-year -
CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 19, 2015-- Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the Company held a pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) regarding its lead product candidate, eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD). Sarepta has agreed with the Agency to initiate a rolling NDA submission and will submit the non-clinical and CMC components of the NDA by the end of this week. As previously announced, Sarepta plans to submit the final component of the NDA by mid-year 2015.
"We will initiate a rolling NDA submission to facilitate the regulatory review of the NDA,” said Edward Kaye, M.D., Sarepta’s interim chief executive officer and chief medical officer. “The initiation of our NDA submission for eteplirsen marks a significant milestone for the Duchenne community and we look forward to completing the submission by the middle of the year and to continuing to work with the Agency towards the goal of providing treatments to patients as quickly as possible.”
IPF - Roche / FGEN:
Here is some interesting data on patients who have FVC decline of > 10% on Esbriet after 6 months and stay on.
http://www.medpagetoday.com/MeetingCoverage/ATS/51613
Among patients in the pirfenidone groups, 58.8% had no further decline in FVC, compared with 38.2% of those in the placebo groups (P=0.059).
Results in an on-treatment analysis were similar, finding one patient in the pirfenidone group and 15 in the placebo group having a further 10% or more decline or death, for a relative difference of 83.3% (P=0.032), Nathan reported. In this analysis, there were no deaths in the pirfenidone group and 10 in the placebo group during the 6 months after the initial decline, with a relative difference of 100% (P=0.056).
...
"FVC may not be the whole story. This is a very complex disease," he said.
GLPYY / GLPG:
I can vouch for the change it did in one of my accounts but in another it seems to be in the process of changing (i.e. in one view it appears as GLPG but doesn't have updated prices and can't trade it at least not on line).
I took a little off the table today. Not that I don't like the company just buying some other things. The one thing in the F-1 that was a bit of a (negative) surprise were the royalty rates for 634 and the CF program. I actually expected a little higher rate especially for 634.
PLI - IPF:
I don't know much about them. In general I've become cautious of IPF programs till they have some patient data. I never looked at their Anemia drug, I think their market cap and need for cash deterred me especially once I saw their Fibrosis compound was still early.
FGEN - IPF:
I tend to agree with Peter and the disease being quite heterogeneous. I think disease severity is not necessarily a good predictor of being able to show a treatment benefit. One thing InterMune attempted to do was find patients who were declining in the theory that Pirfenidone would be more likely to show a treatment effect in those patients. I suspect you are aware of this already but for those that aren't its a pretty strange disease in that someone could be stable for some time and then have a sudden drop in lung function and become (relatively) stable again for some time.
I am very curious to see the Phase 2 data as well and I am curious to see if change in fibrosis correlates to FVC change. If so I still like the idea of somehow trying to incorporate that as a primary end point especially if they go head-to-head with Esbriet or Ofev (as opposed to add-on).
GLPG / CNCE:
I looked at CNCE and thought their earlier stage products were more interesting (from a commercial standpoint at least). With the failure of deuterated xyrem it certainly creates more doubt (in my mind at least) so I'd want to see some data.
GLPG has their own potentiator that they claim is better (I believe about 25% by one measure) which is already in humans. I believe they also had several backup compounds so even if they have a set back I don't think they would try to get CNCE's compound.
Getting to jq's point about trying to GLPG1837 in F508del homozygous does anyone know if that is a GLPG call or an ABBV call? The partnership structure is that GLPG does the Phase 1's but I wonder if that is more a strategic decision where ABBV would need to make the call.
Assuming ABBV opts in 634 I wonder what direction GLPG will go with all the cash they will have between what they have now, the IPO and the 200+possible 50 they will get from ABBV.
PIRS:
If your interested Jason Napodano tweeted a link to his zacks report on the company. His reports tend to be pretty thorough (though tends to have a lot of speculatives names can't say I always agree with his price targets either)
http://scr.zacks.com/files/PIRS-Initiation-April-2015_v004_b335tq.pdf