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RNA:
Was curious to see the stock up then saw rumors on twitter Sasha Damouni (@SashaDamouni) followed up with the company and tweeted this
CNCE:
Concert Pharmaceuticals Presents Positive 48-Week Results from Phase 2 Clinical Trial of CTP-499 in Diabetic Kidney Disease
http://ir.concertpharma.com/releasedetail.cfm?ReleaseID=842797
The stock was down to 7.69 after being halted according to flyonthewall CHTP-499 failed Phase 2... or did it? Missed on primary but...
http://www.theflyonthewall.com/permalinks/entry.php/CNCEid1999409/CNCE-Concert-Pharmaceuticals-reports-positive-results-from-Phase--trial-of-CTP-?utm_source=twitterfeed&utm_medium=twitter&utm_campaign=theflyonthewall.com
http://www.xconomy.com/boston/2014/04/25/how-a-kidney-drug-almost-torpedoed-concert-pharmas-ipo/?single_page=true
This is interesting. Since when did Jeter start batting left handed
http://a.espncdn.com/photo/2014/0423/mlb_spray_cr_300x300.jpg
(from http://espn.go.com/blog/sweetspot/post/_/id/46442/defensive-notes-rockies-heyward-hunter)
CELG:
Missed the part about it being Oral! I was mesmerized by the upfront for a program entering Phase 3 that I never heard of before. Catching up on twitter seems not much known about the program. Is 58% durability in Crohns good?
CELG:
Celgene Acquires Late-Stage Product for Crohn’s Disease and Other Gastrointestinal Disorders
http://finance.yahoo.com/news/celgene-acquires-stage-product-crohn-113000957.html
I guess Ariad investors are looking for a wild pitch while others balk
SRPT / RNA:
I am actually in the camp that drisapersen may be approvable but I think safety appears to be so vastly in favor of eteplirsen that it would make it difficult for drisapersen to compete without greater efficacy. I found it curious that RNA did not elect to continue extension studies one consequence is now SRPT may likely enroll more of those patients.
I thought that SRPT still had avenues to challenge the patents. If it were me I would seek to settle sooner to access patients for both trials and (hopefully) reimbursed patients. I would go higher on the royalty to get a deal (high single digit) but I just don't get the impression Chris would.
SRPT:
I am probably reading too much into it and I haven't been a shareholder long enough to know how they generally word fund raising PR's but I found this part interesting (for speculating):
ITMN:
It wouldn't be the first time Dan Welch sold a company to GILD .
A couple years back I think they were one of the rumored suitors for the company and given they've made a couple of acquisitions (think both were private companies) to get IPF programs and they already target pulmonologists it would seem to make some sense. Given that things that seem to make sense rarely pan out I doubt it ends up happening
One (long shot) suitor that to me makes a lot of sense is actually UTHR. The Remodulin franchise will start getting generic competition soon they have/are generating a ton of cash and they have US operations but have done a poor job at getting approvals in Europe and I don't have reason to think they can do a better job selling there. InterMune would seem to be the exact compliment. A big problem though is InterMune's price tag and I don't get the impression Dr. Rothblatt is ready to give up that much of the company (to me Welch would be the preferred CEO of a combined company...by far).
Are the laws different in Europe or is it the nature of the patent? Wasn't there a similar situation with Amphastar and Momementa in that the use of the patents in research was permitted?
Just to add to your point, I doubt that Vimizim would have ever been approved by a BMRN of 10 years ago (2 arms and much larger N) not to mention being better understanding of patients to enroll and managing AE's.
Placebo controlled trials are not a panacea especially when there is still a lot to learn in the treatment of the disease. Since I don't have as good of knowledge/history as others here I like the PAH example as its one I am familiar with. Remodulin was actually approved on pretty bad data. Part of the problem was they enrolled some very mild patients which we now know its difficult to show benefit for. They actually ended up with a broad approval that included Class II patients but in reality very few class II patients used Sub-q (and later IV) Remodulin. UTHR has since done longer term observational studies comparing to the natural history and I doubt you find anyone who disagrees that the treatment has a meaningful benefit. Also IMO what the Remodulin approval did do was help to attract others to the field (The generous pricing/reimbursement helped of course).
I don't disagree that the company should have been better equipped to proceed to Phase 3 (I went through old calls and Years ago they were thinking approval by this time) and thus have sufficient quanties to be able to offer compassionate use.
IMO SRPT is really trying to have their cake and eat it too. Namely to have a trial where one arm are amenable to their drug and another arm of DMD patients who are not so you can then later get a class approval with limited additional trials. From an investment standpoint it would be great if they could have gotten it but perhaps a more realistic approach would have expedited things. Perhaps I am mistaken but I thought they could still enroll patients in Europe even though RNA has patents (and not submit there perhaps just give the drug for free for those patients till the IP is settled) to get more patients. I am one who certainly believes they should have tried to make some agreement with RNA.
I was (perhaps still am) naive to the process when AZY was approved but certainly don't doubt you. To me Astrue was more speaking to the way FDA changed to be more accommodating even when the data by todays standards was marginal and how it lead to more/better therapies.
SRPT / FDA:
I wouldn't go as far as you in my blame of the company and defense of the FDA. I thinks its fair to put some blame on the company for not being ready to scale mfg and openly discussing/raising hopes of families for accelerated approval. However the FDA did appear to reverse their willingness to accept the current data for accelerated approval. Also I fail to see how not approving the drug in this case is whats best for patients. Its not a benign condition the patients will clearly die and have a difficult end of life experience too I couldn't fathom being a parent and going through that especially knowing there is a remote chance a drug could prolong my sons life.
I did a quick look at NVIV and they seemed too early stage and thin for my comfort so I didn't pursue it. I thought he left there and was more there on an interim basis but just going by memory. He was also briefly CEO at EPIX but where he really did his best work was TKT
I am probably biased in my beliefs (not just in the SRPT case where I am long) as I believe FDA should be much more accommodating especially where there are limited options.
While the Pfizer/PLX Gaucher case may be a bit different because of need (at TKT he was hoping to get their Gaucher product approved on 1 trial and was critical of Genzyme basically being permitted a monopoly for quite an extended period) I prefer his HIV analog and how the mindset shifted because of need (and a large public outcry).
Even though I am long SRPT I think SRPT management has not done the best job and am surprised some of the patient advocates haven't been critical of them. I think Astrue has a good compromise proposal to allow for a Phase 3 without hurting enrollment and yet get drug to patients (though I think Accelerated approval beforehand would remove more obstacles and be more compassionate). I don't know if they can or would want to be so broadly open but it certainly would help quiet some of the criticism if the agency came out public and said they would approve compassionate use thus putting more pressure on SRPT to get the manufacturing capacity ready (I believe they've completed a couple runs at larger scale already).
FDA / SRPT:
The FDA Returns to Its Dark Ages
http://www.weeklystandard.com/blogs/fda-returns-its-dark-ages_786828.html?page=1
A very well written article (no surprise since he is a lawyer) by one of my favorite x-CEO's.
h/t @TheRacetoYes
I suffered through a bad internet connection last night and couldn't even follow play by play... maybe I was lucky though
Oh and how dare you jinx Freedie
Thanks for the details.
Does anyone have a handy reference for whats reviewable? I am curious if the in-the-neighborhood play is or not. TIA
RTRX:
Nice call on Martin being a bit too optimistic on things. I still have mixed feelings on the company and its programs. I was however impressed with the recent deals despite potential IP and other issues. I suspect there may be more "growing pains" along the way and am still on the sidelines with no position.
JAZZ / GENTY:
$JAZZ launched Defitelio in Germany & Austria in Mar: $590/vial -> Avg price of ~$99K/course(21-day) in an adult pt of avg body wt of 70kg.
Thanks to Andy Biotech (@AndyBiotech)
Treatment of HCV with ABT-450/r–Ombitasvir and Dasabuvir with Ribavirin
From NEJM
http://www.nejm.org/doi/full/10.1056/NEJMoa1315722
the link for the PDF is
http://www.nejm.org/doi/full/10.1056/NEJMoa1315722
Vital Therapies:
I spent a little more time looking at the company and decided its unlikely I would take a position (regardless of the IPO price). I could live with it regulated as both a device and biologic since the device side seems like it would not be an approval obstacle. What I didn't like was they have been running trials for quite some time and the results are not clearly convincing. I also figured adoption would be a challenge though I think they could command a very high price and likely will need just a tiny sales force.
ADMS:
Taking a look at the company and noticed the terms of their FRX (ACT) royalty are ones I don't recall seeing before. I can see the logic from the FRX (ACT) end but not sure I see it from the ADMS side perhaps it explains the descent upfront given its basically a reformulation?
http://ir.adamaspharma.com/secfiling.cfm?filingID=1047469-14-3461&CIK=1328143
Ohad Hammer has a nice write up on the company and why he thinks its undervalued even without the prostate cancer indication
http://www.orf-blog.com/exelixis-down-but-not-out-plenty-of-catalysts-in-2014/
Vital Therapies:
Here is a link to their Retail Roadshow presentation. Seems like an interesting company though if people are still excited about liver disease may be like other IPO's and too expensive for my taste. I didn't look at their filings yet just a quick listen of their presentation.
http://www.retailroadshow.com/sys/launch.asp?k=31378214148
Link to Website:
http://vitaltherapies.com/
Link to prospectus:
http://www.retailroadshow.com/sys/pdf.asp?d=/shows/VTL120u_rr/DATDXGC/prosp.pdf
HALO:
That is a good observation on the possibility that the addition of Abraxane may have led to the thromboembolic events. Not having a science background it was just speculation on my part but I felt that using a much lower dose of the other agents perhaps even in a different (more frequent) schedule along with PEG-PH20 may be a better approach (both in terms of efficacy and safety). I liked (I'm no longer long HALO) the PEG-PH20 program and don't see this as the end of the compound. After the phase 1 I speculated the data weren't as clear and perhaps the path to approval would be a lot longer... Not being a fan of their insulin programs perhaps once the market (finally) gives up on it I'd consider HALO again at a (hopefully much lower) valuation.
Ay OK thank you forgot about the different concentration so that makes its less straightforward.
TEVA / MYL / MNTA:
(Serious question) What is to stop insurers from reimbursing a different dose for a generic product (if approved) off label? Then patients can keep their 3x week dosing and presumably they would get a discounted price.
SRPT/RNA:
Parents of boys with Duchenne urge drug OK
http://www.post-gazette.com/news/health/2014/03/30/the-finding-strength/stories/201403300187
For an article in a newspaper it seems to give a pretty good assessment of the current situation and has a couple of good quotes from scientists/regulators.
I wouldn't ordinarily post about a patient but when they speak to the press I figure its OK to post as they want their message out.
Glaxo heart drug that failed trial shows potential benefit
Seems there is quite a bit of mixed opinion on the drug from my twitter feed.
GSK PR
http://us.gsk.com/html/media-news/pressreleases/2014/GSK-presents-data-from-Phase-III-STABILITY-study-of-darapladib.html
http://in.reuters.com/article/2014/03/30/us-heart-glaxosmithkline-idINBREA2T09620140330
and the NEJM article
http://www.nejm.org/doi/full/10.1056/NEJMoa1315878
full PDF
http://www.nejm.org/doi/pdf/10.1056/NEJMoa1315878
BoA comments thanks to @lomu_j
https://twitter.com/lomu_j/status/450256620031713280/photo/1
John Carroll article
http://www.fiercebiotech.com/story/glaxosmithklines-darapladib-team-finds-hope-phiii-bomb/2014-03-30
Glaxo heart drug that failed trial shows potential benefit
Seems there is quite a bit of mixed opinion on the drug from my twitter feed.
GSK PR
http://us.gsk.com/html/media-news/pressreleases/2014/GSK-presents-data-from-Phase-III-STABILITY-study-of-darapladib.html
http://in.reuters.com/article/2014/03/30/us-heart-glaxosmithkline-idINBREA2T09620140330
and the NEJM article
http://www.nejm.org/doi/full/10.1056/NEJMoa1315878
full PDF
http://www.nejm.org/doi/pdf/10.1056/NEJMoa1315878
BoA comments thanks to @lomu_j
https://twitter.com/lomu_j/status/450256620031713280/photo/1
John Carroll article
http://www.fiercebiotech.com/story/glaxosmithklines-darapladib-team-finds-hope-phiii-bomb/2014-03-30
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 May-Dec events list
26th Annual ROTH Conference
3/9-12
http://www.wsw.com/webcast/roth28/
Barclays Capital Healthcare Conference
3/11-13
http://cc.talkpoint.com/barc002/031114a_bc/
BioCentury 21st Annual Future Leaders in the Biotech Industry
3/28/14
https://www.biocentury.com/conferences/futureleaders/dates
Needham 13th Annual Healthcare Conference
4/8-9
http://wsw.com/webcast/needham65/#table1
Username: needham
Password: healthcare13
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://www.jefferies.com/OurFirm/Conferences/325/
Goldman Sachs Healthcare Conference
6/10-11
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
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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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Here is a direct link to the BioCentury Conference Webcasts today
https://www.biocentury.com/conferences/futureleaders/dates/schedule
I believe they are a potential competitor
http://blogs.wsj.com/japanrealtime/2014/03/26/robotics-firm-with-evil-name-wins-investors/
EXEL:
It seems to me a pretty big drop for not stopping early! I've looked at EXEL in the past and am tempted my issue with the company has been dilution and now there are some other therapies but still seems to have a place in market.
I haven't looked at them closely enough to form a definitive opinion. I realize IPF is not an easy indication so am willing to give them a pass (I believe the J&J collaboration was when I first heard of them). It seems to me they've been touting their library since at least then whether it is just taking time to develop/partner or its not as valuable as it would seem is something I would want to get comfortable with first. Their former CMO went to (and is now out of) Nektar too (I didn't find her especially impressive).
I did find BIND appealing at a low valuation to take a position but the real value may be early stage/with partners we don't know much about.