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QURE:
The other (pessimistic) view is that they could resort to being a contract manufacturer. They hinted on the call about possible BD in that area. They will need no more than 500L capacity for the foreseeable future and can scale current facility to 2000L.
Of course I wonder why they won't succeed in Hemophilia B (I've discussed these before):
1) Their Vector is different and could potentially serve patients other GT cannot
2) A FIX expression > 10% would still be revolutionary compared to what exists today and would eliminate prophylactic factor replacement which is really the more meaningful clinical endpoint then an arbitrary expression percentage.
3) The company reiterated on the call their durability (6.5+ years now) with the same cassette and no loss expression as seen with other programs. So if ONCE is starting much higher but the expression diminishes over time one may prefer a more consistent albeit lower starting level (remains to be seen since both need much longer followup).
And Still their BMS collaboration remains one of the (if not the) most lucrative GT deal with a relatively novel (less competitive) area of focus.
Statin Use for the Primary Prevention of Cardiovascular Disease in Adults
US Preventive Services Task Force Recommendation Statement
Vinay Prasad @VinayPrasad82:
I first heard of him when he referred to advocates of Eteplirsen as Advocates of Snake Oil Salesmen (I took that as an attack on families). While I wouldn't disagree on evidence being weak for E I thought that statement personal. I actually then looked at his twitter feed saw he had a link to his (extremely promotional) website and a recent book. Seeing him tweet against everything from anti-statin, anti-immunotherapy, anti-tumor sequencing, anti-accelerated approval, anti-surrogate endpoints, etc. I certainly don't have his medical knowledge/background to refute point by point what he tweets but a few things are obvious:
1) He seems to cherry pick to support his cases
2) He is extremely self promotional/absorbed!
@VinayPrasad82 :
I don't get why he is so popular. I find him to be quite self promotional/absorb and his personal attacks on DMD families makes me wonder how he treats his patients especially if they have a rarer cancer with limited data.
Yes the Nektar dose is remarkably low though I was actually referring to the much lower dose of the other agents needed in the models.
I think people (myself at first too) were disappointed Nektar couldn't achieve better efficacy with higher doses. They didn't go in a lot of detail but mentioned in passing part of the reason with starting the combo dose at .003 was that not much greater activity seen at higher doses (though still early). I think the plan is to escalate to .006 Q2W in the combo studies. There is a chance they could go to .009 but want more data on monotherapy before deciding. For what its worth the presenters seemed very bullish on the biomarker activity they saw and think the drug is highly active at producing the desired effect. I've come around a bit too realizing the goal isn't monotherapy so it remains to be seen just how much synergy 214 adds by making the tumors hot (hotter).
Understood. I was surprised at how low of doses they were using. I also don't expect Nektar to go to a triple study anytime soon though its interesting their initial collaboration is with BMS that has both.
Nektar's presentation of 214 at SITC they talked about combining with BOTH anti PD-1 & CTLA4. If I heard correctly they said the doses in the models were < 1/10th of the dose used in monotherapy. Don't know much about TRIL's compound but I imagine at such low doses the safety may become less of an obstacle (Nektar 214 doesn't appear to have cross tox with Checkpoint in limited human data thus far). Here is the slide on the 3 agents
They will file rolling NDA in 1H '17. I read it as complete submission in 1H '17 which I would be happy with which likely pushes approval to early '18. I am actually a bit surprised they PR'd this as many companies don't say anything till they have meeting minutes.
http://www.asx.com.au/asxpdf/20161109/pdf/43crmn5rqqv8rs.pdf
OCRX ATM:
It looks like they sold 1.2M shares through the ATM during the quarter and perhaps some more in October too. I still think it may have been a prearranged sale since the stock is not that liquid to have supported some of those high volumes.
FGEN:
A couple of studies just posted by Astellas on clinicaltrials.gov
A Drug Interaction Study to Evaluate the Pharmacokinetics of ASP1517 and Lanthanum Carbonate Hydrate
https://clinicaltrials.gov/ct2/show/NCT02952040
A Study of Intermittent Oral Dosing of ASP1517 in Hemodialysis Chronic Kidney Disease Patients With Anemia
https://clinicaltrials.gov/ct2/show/NCT02952092
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/Updated November events; PRTK R&D Day; Added December-January Events
bluebird Bio Gene Therapy Day
10/13
http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-EventDetails&EventId=5236920
BIO Investor Forum
10/18-19
https://www.bio.org/events/bio-investor-forum
ARM’s Advanced Therapies Investor Day
11/3
http://eu.arminvestorday.com/agenda/
Credit Suisse Healthcare Conference
11/7-9
https://cc.talkpoint.com/cred001/110716a_as/
Eagle Pharmaceuticals Investor Day
11/11 8:00am ET
http://investor.eagleus.com/
Sanford C. Bernstein 4th Oncology Day
11/11
Stifel Healthcare Conference
11/15-16
http://wsw.com/webcast/stifel5/
Jefferies London Healthcare Conference
11/16-17
http://wsw.com/webcast/jeff100/
Canaccord Genuity MedTech & Diagnostics Forum
11/17
http://wsw.com/webcast/canaccord24/
Paratek R&D Day
11/17 1:00pm ET
http://investor.paratekpharma.com/phoenix.zhtml?c=253770&p=irol-calendar
Oppenheimer & Co. Inc. 2016 Life Sciences Summit
11/29
https://www.opco.com/conferences/life16/index.aspx
Piper Jaffray 28th Annual Healthcare Conference
11/29-30
http://www.piperjaffray.com/2col.aspx?id=1108
Deutsche Bank Pharmaceutical and Healthcare Corporate Day
12/1
Citi Global Healthcare Conference
12/7-8
Guggenheim Securities 4th Annual Boston Healthcare Conference
12/13
Goldman Sachs Healthcare CEOs Unscripted: A View from the Top
1/5/17
JP Morgan Annual Healthcare Conference
1/9-12/17
Biotech Showcase
1/9-11/17
https://ebdgroup.knect365.com/biotech-showcase/
NobleCON13
1/30-31/17
https://nobleconference.com/noblecon13
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Share price appreciation forgives a lot of sins
AVXS (RGNX) IONS BIIB:
Why couldn't they enroll patients x-US (perhaps some x-EU too) where I presume Nusinersen would be later to market? Given EU reimbursement timelines they could potentially get the majority in Europe and not have to resort to other territories.
Having detailed press releases or a conference call (where management is willing to answer questions) would be great now to get a better sense of the launch (which they should know). For example:
1-Are they receiving reorders from the same centers and what rate. This could help in gaging both compliance and number of patients (which I think could be higher than many of the estimates)
2-What is the revenue by territory. It would be great to get patient numbers by country but I doubt they'd give that at least a revenue break down would help to gage the launch.
I saw someone on sharescene post about production cost. I haven't (yet) looked at historical numbers and don't know if we have enough data (e.g. how much inventory did they have, what is the shelf life of Scenesse, etc.) to gage but with the limited info the company gives that may be one number to pay closer attention to.
As far a Vitiligo partnership people seem to have too high of expectations in terms of up-front. With the limited data I doubt they get a big up front payment if they even do a partnership deal.
As far as other uses I think the best bet are follow on Melanocyte products unless the indications have Orphan opportunity (and the 7/10 year protection that comes with it). The IP left on Scenesse and the time to get through P3 trials doesn't make much sense to purse with afamelanotide.
Bottom line I am happy with the numbers with the limited rollout thus far and think the revenue (for just Europe) could go higher than expected as patient numbers may be higher (as often happens in rare diseases). The US opportunity though smaller patient numbers than Europe should get a price premium and quicker launch (if/when approved) so in some ways it is more valuable than EU.
A terrific quarter (so perhaps a small leak since volume wasn't absurdly high)
http://www.asx.com.au/asxpdf/20161031/pdf/43ch19494svwrh.pdf
And they also got a letter from ASX inquiring about unusual price move (the company said no reason perhaps recent PR's about communications with FDA)
http://www.asx.com.au/asxpdf/20161031/pdf/43ch1dqsmy35nf.pdf
I don't disagree in general though there are exceptions of course (I'm hoping QURE is one and also long ADVM)
I was looking at rkrw's list from SI on 10/10/2008
http://www.siliconinvestor.com/readmsg.aspx?msgid=25050643
during the crisis just browsing it would seem most are gone though some may have been acquired. I did notice two I owned (not from quite that time but not too long after) GENT did remarkably well and NKTR did OK. I did also own some dogs (EPIX and especially ALTU popped out at me)
QURE / BMRN / GT Manufacturing:
On the call today BMRN was asked a fair amount about their GT manufacturing. They mentioned QURE in part of their reasoning to do their manufacturing using Baculovirus as they have an approved method. They said that they did also manufacture in mammalian cells and noticed no difference in potency. Insect viral manufacturer is much more scalable and the other reason they went that direction.
ON my thinking of QURE being a good acquisition target for BMRN that does not seem likely for two reasons:
1) They are well on their way in setting up their own manufacturing expected to be ready late next year. While they would not disclose costs they said its in their 150M annual budget for this year and next year on capital spending.
2) Nothing near term on acquisition horizon though continue to look for deals especially things more earlier stage for 2023-4 timeline.
OCRX:
I just bought more today and it executed right away.
Big drop today on moderate volume no news that I see so I take it is just the down bio market and people wanting out.
PRQR Is now basically flat!
I don't follow them closely but someone on twitter pointed out they took down their poster from their website. They do have a webcast scheduled for 9pm ET tonight
http://edge.media-server.com/m/p/tmnczi4s
Here is a link to the tweet with a JPEG of the poster that they took down
https://twitter.com/aschwartzphd/status/791609085003194369
h/t Dr. Tony Schwartz @aschwartzphd
ABEO has an ATM in place I am curious to see how much that was used prior to this offering.
I thought the (very preliminary) Sanfilippo data looked pretty good. I don't have any position in the company though.
QURE:
While I don't disagree especially when they named the CFO interim and said not currently planning on a CEO search it should be noted that BMY owns 9.9% and has options to acquire another 10% which may complicate any deal (unless of course they are the acquirer). There are specific terms see below
For shareholders who have been here a while the call can safely be missed.
The only tidbit (that I caught) was that they said payers indicated willingness to pay 1300/day for drug that would reduce hospitalization by 24 hours. I actually thought they could get a figure about 2x that.
No update on enrollment just reiterated expect to complete by EOY and data in Q1.
Thanks I read the changes backward!
Do you recall why they lowered it to 140?
At quick glance looks the same as september (I like the newer slide deck better than what they were using before)
The enrollment slide of it being changed from 200 to 230 bothered me so I went back to double check as I recalled initial enrollment being much lower
Here is the first change from 140 to 200
https://clinicaltrials.gov/archive/NCT01966419/2014_05_21/changes
I didn't recall that being done.
And then the second change on DSMB recommendation
https://clinicaltrials.gov/archive/NCT01966419/2016_01_14/changes
BIO CEO presentation in 30 minutes (8:30am PT) here is a direct link
which should be good for the replay too.
https://www.veracast.com/webcasts/bio/investorforum2016/93103255342.cfm?0.594393276077
And here is the October Slide Deck
http://files.shareholder.com/downloads/ABEA-5YQ58G/2822171477x0x911645/A6F4927F-D09B-479D-8DEE-9F5420754FA8/Ocera_Investor_Presentation_Oct_2016.pdf
NKTR:
Nektar Therapeutics Announces Public Offering of Shares of Common Stock
My guess is Howard was seeing partnership interest in 214 and when he didn't get terms he liked did the BMY collaboration and this was the long awaited financing (at least by me) since there was no partnership. Lets see if it works out better this time. Around the time I bought my first shares (before initiating P3) he had all but said NKTR-102 was about to be partnered but it would seem a deal fell through which resulted in them pushing it on their own (a move I wasn't a fan of).
http://finance.yahoo.com/news/nektar-therapeutics-announces-public-offering-200100922.html
SAN FRANCISCO, Oct. 17, 2016 /PRNewswire/ -- Nektar Therapeutics (NKTR) ("Nektar") today announced that it has commenced an underwritten public offering of $175 million of shares of its common stock. In connection with this offering, Nektar will also grant to the underwriters a 30-day option to purchase up to an additional $26.25 million of shares of common stock. J.P. Morgan is acting as the sole book-running manager in the offering. The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.
Nektar intends to use the net proceeds from this offering for general corporate purposes including research and development funding and working capital.
I don't have any hard numbers so can't quantify it. It seems to me the ratio of administrative people to physicians/pa has increased substantially in the past decade or two also there are more people in general involved in patient care then in the past (between nurses, aids, specialists, assistants, etc.). I would be curious if anyone has seen hard numbers.
ADVM:
I found the Annapuma assets more interesting then the Avalanche (I am long the stock) though I am obviously disappointed with the delay. All things considered its probably best that Salzman is now the CEO. They still have the partnership with Regeneron and claim their biofactory platform can be leveraged for many eye disease though I am not giving it and the earlier program with Editas much value at this point. They are well below cash and I was hoping some early human data would come while the balance sheet is still reasonable.
Here is a recent presentation from the Ophthalmology Innovation Summit
https://www.sec.gov/Archives/edgar/data/1501756/000119312516737669/d230538dex991.htm
BMRN Hemophilia A GT:
So BMRN is going to add another dose exactly between the mid and high dose (there was only 1 patient in the middle dose 2x10^13 before and 7 on the high dose 6x10^13). The other encouraging item is that they have removed the prophalactic use of steroids.
http://investors.bmrn.com/releasedetail.cfm?ReleaseID=993495
UK Regulatory Agency Approves Continued Enrollment in BioMarin Phase 1/2 Study of BMN 270 in Hemophilia A
Phase 1/2 Study Expected to Resume by End of 2016
Requirement for Prophylactic Corticosteroids Removed
SAN RAFAEL, Calif., Oct. 13, 2016 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that the Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom approved continued enrollment into the open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A. BioMarin had previously announced that after enrolling the first 9 patients in the study, that dosing of patients had been suspended due to observed increases in alanine aminotransferase (ALT) levels that exceeded a pre specified threshold set by the company. Following study suspension, the company reviewed safety and efficacy data on the 9 patients with the MHRA, and based on its review, the MHRA approved resumption of the study. The agency also approved the company's proposed amendments to the study, which included eliminating the requirement for prophylactic corticosteroids and increasing potential additional enrollment from up to three additional patients to up to six additional patients.
BioMarin intends to resume enrollment in the Phase I/2 study before the end of 2016. Based on protocol amendments, three patients will be enrolled at a dose of 4 x 1013 vg/kg, and an additional three may be enrolled at this dose or the previously tested high dose of 6 x 1013 vg/kg. In the up to six additional patients, the requirement for prophylactic corticosteroids has been removed and the threshold for starting therapeutic corticosteroids has been increased. Safety and efficacy data from these patients will inform the Phase 2b study planned to begin in the second half of 2017.
"We are pleased that MHRA has approved the resumption of enrollment of the BMN 270 study, as well as the study amendments. We believe that the amendments will allow us to optimize the design of a robust Phase 2b clinical trial, which potentially could support an accelerated approval by health authorities," said Hank Fuchs, M.D., Chief Medical Officer at BioMarin. "We are grateful to the patients who are participating in this current study, and are encouraged by the results so far for this Phase 1/2 trial."
Phase 1/2 Study Design
The current Phase 1/2 study is evaluating the safety and efficacy of BMN 270 gene therapy in up to 15 patients with severe hemophilia A defined by the WFH as less than 1% of blood clotting factor. The primary endpoints are to assess the safety of a single intravenous administration of a recombinant AAV vector coding for human-coagulation factor VIII and to determine the change from baseline of factor VIII expression level at 16 weeks after infusion. The kinetics, duration and magnitude of AAV-mediated factor VIII activity in individuals with hemophilia A will be determined and correlated to an appropriate BMN 270 dose.
This is a dose escalation study with the goal of observing an increase in factor VIII levels. Secondary endpoints include assessing the impact of BMN 270 on the frequency of factor VIII replacement therapy, the number of bleeding episodes requiring treatment and any potential immune responses. Patients will be monitored for safety and durability of effect for five years.
blue:
Here is a direct link to bluebird's GT Day webcast from earlier today. The replay is available now
http://edge.media-server.com/m/p/vvtozdsh/lan/en
OK found it here is the separate release for investors
http://www.asx.com.au/asxpdf/20161013/pdf/43bycpks11m0lv.pdf
The PR makes mention of a version for investors with more information. I didn't find it or else I read it again without realizing anything new in it. Did anyone happen to see a more detailed release or know what was different in them?
Slight OT PR to announce PR before CC:
Does anyone know the logic behind this. Why can't they just give the information out today and then have the CC tomorrow after people digest it and it saves them 1 PR ...
If I were to guess I would say the company doesn't want to give people too much time to digest the data before the CC which generally seems like a bad thing. I haven't done an analysis but wondering if others can think of when it was actually a positive situation.
AZRX:
I did a quick look at the company and think I'll pass for now a link to their slide presentation. Altus tried to get a recombinant pancreatic insufficiency drug and failed. As I recall their from Science Officer (and I think a founder), Alex Margolin bought the rights to the drug started a new company with it and sold it to someone who also failed.
http://www.azurrx.com/images/pdf/presentation/AzurRxBioPharma_Presentation_20160906_1300_FWP_as_filed.pdf
ITEK:
They (literally) fired their CSO friday night 8-k. Its obviously very concerning giving the data are near term. I went back over the R&D slides and it appears the CMO did the P3 presentation I didn't see mention of the CSO except in the opening management teams slides so I am not sure how involved he was.
https://www.sec.gov/Archives/edgar/data/1281895/000119312516733841/d235719d8k.htm
For anyone interested in the World Muscle Meeting (particularly SMA) I recommend following the tweets of
@AnnemiekeAarts2 (in particular) and
@VArechavala
AUPH:
I sold out at AUPH at a loss so my thoughts may not be worth much... The things that got me selling (originally before results and then the remaining for a loss shortly after):
1-The company claims its dose is not weight based and has lower tox because of a better pK. Looking at some older trials in other indications it appears that as the dose goes up the tox is more in line with other Calcineurin inhibitors. They broke out the results by male/female and interestingly men did better on higher dose and women on lower dose. I wonder if they did a weight based analysis and if part of the reason less deaths occurred in the higher dose was due to dose tapers or just overall lower compliance.
2-I was fairly sure FDA would require at least one (and likely 2) much longer trials (perhaps even 2 years as other lupus trials tended to be in that range)
While I am not certain obviously and the company did a good job at breaking down the deaths by site, I think there is something there also the costs to get to registration if my thinking is correct will be quite a lot of dilution lacking a partner (and diluting their ownership interest).
It seems counterintuitive that the FDA would OK a drug for use and a reimburser say no you have to use this one. (serious question) Does medicare limit use of one drug in favor of another? I imagine not since they can't negotiate price. If democrats gain more control I wouldn't be surprised if they get more scrutiny if for no other reason their unwillingness to play nice with Obamacare (not that I support it).