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NKTR:
Nektar is up again around ~4% so in 2 months and a week or so the stock is up ~50% on really no news (The Q2 report/call was basically uneventful).
While I can't be 100% certain I think its extremely likely related to NKTR-214. The study is open label and should be well into an efficacious dose. The combination portion should be starting relatively soon too. Since its open label some people should obviously know. The other thing of note is I've noticed Howard Robin likes to keep a sizeable amount of cash (above the level of their debt) and with the appreciation he's had ample opportunity to raise cash. I'd imagine 214 if the data is good is unlikely to pursued alone because of the broad application the question is how early would it be partnered or would the company just be acquired? If AZ had confidence in Movantik would seem like the ideal fit.
cUniQure (QURE):
I've been buying the stock of late. They have really been beaten down again since releasing Hemophilia B data in July, to the point that their EV is only ~40M.
1) The data to date in their Hemophilia B program has shown about 5% expression and some ALT elevations. Sparks has shown ~30% (21-42) and no ALT elevations which is perhaps why people are writing off QURE's Hemophilia B program. I find it interesting BMRN (which also uses AAV5) had more than a log increase in expression with a 3 fold increase in dose. Also BMRN's ALT elevations have been managed with prophylactic steroids (with apparently no loss of expression). The High dose in QURE's Hemophilia B program (4x the low dose) should have up to 6 months data by year end. The analysts notes I've seen have expectations in the 10-12% expression range. I would not be shocked for it to be substantially above that. One thing in QURE's favor is they in a sense have much longer data in Hemophilia B. The same Gene Cassette (though different vector) was used for the St. Jude trial (published in NEJM http://www.nejm.org/doi/full/10.1056/NEJMoa1407309?af=R&rss=currentIssue#t=article ), so they have now gone over 5 years if nothing else it demonstrates durability and safety.
2) The Sanfilippo B program may actually be their most advanced with encouraging one year data presented last September ( http://www.uniqure.com/news/267/182/Positive-Topline-Results-Announced-from-Phase-I-II-Trial-in-Sanfilippo-B-Syndrome-Patients-Using-uniQure-s-Novel-AAV5-Based-Gene-Therapy.html ) and 2.5 year data expected in Q1 '17. If the data continue to hold up they could start their pivotal program in '17.
3) The BMS partnership for up to 10 targets, primarily focused in cardio vascular area (S100A1 is the first target) is perhaps the most lucrative GT partnership to date with BioBucks up to 2.3B if all 10 targets are developed (140M to date). The animal data (pigs) had 1 year survival at 90% vs 10% in placebo. The company has said testing in man could start as early as 2017 or 2018. BMS has selected 4 targets. By target 6 they can up their stake to 14.9% and ultimately go to 19.9%
4) Other programs in/near human studies include parkinson's, Hemophilia A and Huntington's.
5) Though they use a much higher dose than Spark they do their own manufacturing with 2 facilities (insect viral line). They've said they have enough capacity to treat all the CV patients from one year of production (one reason sited for BMS choosing them).
6) They do in house research aside from trying to improve their vectors and payloads they've done work on retreatment which I found interesting.
- Biomarin also uses an insect viral line. The BMS partnership complicates things but I've wonder if at current market price QURE would be attractive to someone like them wanting to expand into GT and needing quite a lot of manufacturing capacity/expertise.
- An interesting comp is a transformed company Plasma Therapeutics who is now a Gene Therapy company with a new name of Abeona Therapeutics. There lead program in Sanfilippo Type A just announced dosing their third patient. No other GT program has dosed a patient. They do have some interesting areas they licensed programs (Sanfilippo B, Batten disease and a CRISPR program for Franconi Anemia). They appear to be a company which licenses programs and dose little (if anything) in house. Their EV is about 120M!
OCRX:
I'm currently traveling and limited access to things. Here is my summary bull case.
1) IV product (OCR-002) should have Phase 2 date Q1 '17
1a) Ravicti had positive P2 stopping future HE (Hyperion not pursuing since approved in much higher priced rare disorder). Comparable dose.
1b) April 2015 interim (safety, futility, overwhelming efficacy, sample size adjustment to maintain 80% power) resulted in sample size increase to 230 and company estimates that would indicate saw 1.5 - 2 day improvement.
2) Oral formulation of OCR-002 should have data Q4 '16 (single dose) and 1H '17 (multi dose)
3) Cash through 2017 (35M as of 6/30/16) have some debt though and active ATM
4) Estimated market opp. (per company) 600-700M IV, Oral 800-900M.
5) Have full worldwide rights
I see they have their August Presentation: http://files.shareholder.com/downloads/ABEA-5YQ58G/2761847900x0x904351/BE1982B0-982D-4689-9A33-5ECE37FB9B94/Ocera_Presentation_Aug_2016.pdf
Probably the reason for the spike in volume
https://www.fbo.gov/?s=opportunity&mode=form&id=0d7ad7887a21404946fcdb0d92817197&tab=core&_cview=0
h/t @TradeHawk
OCRX:
No just saw the high volume and placed a limit order. I was a fraction of the shares traded but I doubt I would have been able to execute this quantity on a normal trading day without the price moving a little bit.
OCRX:
A couple more very high volume days and no 13D filings. I was part of the high volume (2 days ago on the buy side about 40% increase in position). With the futility analysis/sample size readjustment indicating nearly 2 day benefit seems like a good risk reward here with EV in the ~25M range plus not to mention oral data not too far off (hopefully they have the formulation issues all figured out now).
HCV treatment in prisons:
I don't have an HCV investment at present but does anyone know what percentage of HCV prison population gets treated at the moment? It appears Pennsylvania only treats when there is evidence of liver damage and this article indicates other states have some type of rationing as well.
https://www.yahoo.com/news/abu-jamal-loses-suit-over-hepatitis-c-drug-163354769.html?ref=gs
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, Updated September Events, Added Bio-Techne Investor Day
Wedbush PacGrow Healthcare Conference
8/16-17
http://www.wedbush.com/services/cmg/equities-division/mgmt-access-events
Credit Suisse Antibody Day
https://cc.talkpoint.com/cred001/090716a_as/
9/7
Wells Fargo Securities Healthcare Conference
9/7-8
https://cc.talkpoint.com/well001/090716a_ae/
Citi's Annual Biotech Conference
9/7-8
http://citiconferences.com/
“Gene Therapy, A New Era” panel discussion https://www.veracast.com/webcasts/citigroup/biotech2016/23104167493.cfm
RW Baird Global Healthcare Conference
9/7-8
http://wsw.com/webcast/baird46/
Biocentury NewsMakers in the Biotech Industry
9/9
http://wsw.com/webcast/biocentury3/
Rodman and Renshaw 18th Annual Global Investment Conference
9/11-13
http://wsw.com/webcast/rrshq26
Morgan Stanley Global Healthcare Conference
9/12-14
https://cc.talkpoint.com/morg007/091216a_ae/
Bank of America Merrill Lynch Global Healthcare Conference
9/14-16
Bio-Techne 2016 Investor Day
9/15 9:00amET
http://public.viavid.com/index.php?id=121018
Aegis Capital Growth Conference
9/20-22
Ladenburg 2nd Annual Healthcare Conference
9/27
http://wsw.com/webcast/ladenburg2/
RBC Smallcap Conference
9/28
https://www.rbccm.com/about/cid-202541.html
Leerink Partners Rountable Series: Rare Disease & Immuno- Oncology
http://wsw.com/webcast/leerink27
9/28-29
Cowen 19th Annual Therapeutics Conference
10/5-6
http://www.cowen.com/conferences/upcoming-conferences/
BIO Investor Forum
10/18-19
https://www.bio.org/events/bio-investor-forum
Credit Suisse Healthcare Conference
11/7-9
Sanford C. Bernstein 4th Oncology Day
11/11
Stifel Healthcare Conference
11/15-16
Jefferies London Healthcare Conference
11/16-17
Canaccord Genuity MedTech & Diagnostics Forum
11/17
--
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.
CXRX:
That is quite a margin call, I wonder how leveraged he is (was) considering that is about a third of his shares of the company. I see he is very well paid too.
http://finance.yahoo.com/quote/CXRX/profile?p=CXRX
I think I would have a hard time investing in a company where the CEO seems more focused on (other) stocks rather than overseeing his own!
OCRX:
More high volume days. I still suspect some sort of prearranged trades whether it is via the ATM or not I wouldn't venture a guess but with a few of these days with the size of the volume perhaps we get at 13G in the near term?
Here are the big holders from the 6/14 DEF 14A (Page 10)
https://www.sec.gov/Archives/edgar/data/1274644/000104746916012477/a2228323zdef14a.htm
Domain Associates, LLC and affiliates(1)
One Palmer Square
Princeton, New Jersey 08542
2,956,648 13.88%
Thomas, McNerney & Partners, L.P. and affiliates(2)
One Stamford Plaza
263 Tresser Boulevard, Suite 1600
Stamford, Connecticut 06901
2,492,986 11.70%
Great Point Partners, LLC and affiliates(3)
165 Mason Street, 3rd Floor
Greenwich, Connecticut 06830
2,059,708 9.67%
InterWest Partners and affiliates(4)
2710 Sand Hill Road, Suite 200
Menlo Park, California 94025
1,360,779 6.39%
Venrock Healthcare Capital Partners, L.P.(5)
1,885,668 8.85%
QVT Financial LP(6)
1177 Avenue of the Americas, 9th Floor
New York, New York 10036
1,058,787 4.97%
Late-Stage Products Looking for a Home
I was googling for something and came across this list. I liked the idea but its interesting to see how many flops there were
Gene Therapy:
Just to add a bit more about some of the current thinking on retreatment a couple more takes from companies. Take it for what its worth perhaps some with the more modest expression are thinking retreatment and those with higher are not?
DMTX - Think retreatment will be needed but they are not working on it. Think in the next 5 years or so it will be clearer and they plan to be a "follower".
QURE - Think durability may be in the 10 year range. The gene cassette (but different AAV vector) they are using is actually the same as what was used in St. Jude's Hemophilia trial with published results out over 5 years (20l4 published results in NEJM)
http://www.nejm.org/doi/full/10.1056/NEJMoa1407309?af=R&rss=currentIssue#t=article
Uniqure is actually working on retreatment have some research underway and they have a couple posters on their website
http://www.uniqure.com/uploads/Publications/Readministration%20two-step%20AAV%20injection%20ASGCT%20poster2.pdf
http://www.uniqure.com/uploads/Publications/AAV1-AAV5%20readministration%20ASGCT%20poster.pdf
Maybe someone with a better science background could answer something for me. I was wondering if someone treated with one AAV vector would likely develop antibodies that would prevent a different AAV vector from working? If not wouldn't the retreatment option present a nice opportunity for "inferior" regimens of a different type?
GILD analysts opposing views
I thought this interesting. Basically Piper's Joshua Schimmer thinks spinning off HCV and/or HIV is value destructive while RBC's Michael Yee and Jefferies' Brian Abrahams think it could unlock value
http://thefly.com/landingPageNews.php?id=2420781&headline=GILD;BIIB;BMRN-Piper-says-splitting-Gilead-businesses-a-poor-idea
EpiPen Alternatives:
I was prescribed one after having a series of unknown allergic reactions. I would get new ones ever couple years (often keeping expired ones around) and with the changes in Health insurance over the years my payments have been going up. I am thinking about asking my doctor to just prescribe epinephrine and regular syringes. I've read a few articles saying some are doing that since its substantially cheaper (though not as convenient and doesn't last as long). My hope is that I never have to actually use it .
ITEK:
At the R&D day in December last year they talked a bit about the Phase 2 and have several slides. I don't know if its still on their website but I made a PDF of the slides if you PM me here or SI (same alias) I can email them to you. Here are a couple of the summary slides
The company said FDA has refused to approval Timolol/Latanoprost combo I don't know if that is formally or just because of the safety (drop in heart rate) concern.
The current registration study (first Phase 3 read out later this year) is monotherapy anyway though Timol is an active control. They are also testing a higher dose than in Phase 2. If the company is telling the truth that FDA will accept improvement over placebo (which they say they have in writing) the bar is pretty low. And as far as combo goes if FDA won't approve a prostaglandin and Timolol option this should be a nice additional agent available to glaucoma patients.
I am long too with the ubiquitous small position
I agree on all counts! Perhaps some of the extra delay is Wolgen "trying" for that uniform price. I remember Dan Welch describing the process as trying to get a price band across Europe with generally the richer countries paying more and the poor ones less. Here are notes I had (they are dated now and links don't works since Roche bought them):
Nice recap. I follow the sharescene board too and have translated some of the government links. I'd certainly recommend that board for anyone with a position there are some posters who are really good at finding relevant info (along with a lot of noise posts too but its worth weeding through those)
Do you happen to know when Italy will start reimbursing at the new price? I don't recall seeing a date and since its a commercial price if Wolgen said that Scenesse is available under the access scheme I'd assume its not the commercial price but the old price.
I am thrilled EPP may not need a US P3 if they get it approved in the US I agree uptake and commercialization would be much quicker and the opportunity (# patients+likely higher price) would be greater than the EU opportunity.
Vitiligo will be a sizeable undertaking in terms of trial size/costs it would certainly be big if they could go directly to Phase 3 though I am not comfortable with their Phase 2 results to feel they can design a trial (right patient population/protocol) to succeed.
BTW, I don't agree with many of valuations/comps that I've seen on sharescene and google groups. One needs to take consideration of the launch phase/size of opportunity/likely competition, etc. I owned UTHR which was basically a one product company and while they were growing 30-40% annually they were over 10x sales. The first couple years of the launch a multiple of sales didn't make sense because of small numbers. Gentium was another one product company (similar circumstances in getting approval actually) they were sold for about 1B when sales (actually it was right around commercialization they were being reimbursed under compassionate use like Clinuvel) were in the 30M range if I recall. And like Clinuvel they had not yet gained US approval plus Sigma Tau owned the majority of US rights (which Jazz later purchased from them).
Thanks for posting your quick!
Wolgen paints things a bit different then I would (process in Europe) The main update I noticed on first read are commercially available in austria and netherlands and still available under the access schemes in Italy and Switzerland (but no mention if its the commercial price so I'd guess its not)
I don't know if its deliberate or incompetence but they mention the EPP Workshop/conference FDA is holding October 24th as invitees only when it certainly appears to be open for the public http://www.fda.gov/Drugs/NewsEvents/ucm501389.htm
If you follow the link on the page you can register and then get an email with a link to the webcast of the event when its available.
I don't have a sub. Anything in the poster noteworthy not in abstract? Thanks.
FGEN:
For ITMN they used HRCT (for ASCEND) or biopsy to confirm IPF though I imagine its less involved then measuring the amount of fibrosis what FGEN is doing. For CAPACITY I think they may have used more biopsy proven. Not sure about what BI did. I think the patient populations are similar too, based on FVC/DLCO at least.
Here is the NEJM Nintedanib link for anyone interested (I forgot to include it in my prior post)
http://www.nejm.org/doi/full/10.1056/NEJMoa1402584#t=article
If it were just slowly enrolling here but they moved extremely slow from initial pilot results too. I remember asking about the medarex royalty around the time either right before/after they were acquired and thinking InterMune was 1-2 years ahead of them (As an ITMN long at the time I was too pessimistic I guess ). My hope is that they really have identified characteristics that will lead to optimal results.
On PC I asked about the 028 study and if you follow this thread with Peter and jq there are links to the ASCO presentations.
https://twitter.com/MauriceOnTW/status/760236678048985088
On last nights call too they talked about the data the link to the transcript and then a section I highlighted are here:
https://twitter.com/SudhanvaRaj/status/762843100385386497
FGEN:
Appreciate your notes and thoughts as always Peter!
One thing that I am perhaps (overly) critical of the company is just how slow they have moved in IPF. On the call Neff said they closed enrollment for the FG-3019 single agent portion in June with 103 patients. I went back to see how Pirfenidone (ASCEND) and nintedanib did (granted these were Phase 3 studies and had many more sites)
Pirfenidone - 555 patients July 2011 through January 2013 (~29 / month)
http://www.nejm.org/doi/full/10.1056/NEJMoa1402582#t=article
Nintedanib - 1006 patients, May 2011 and September 2012 (~60 / month)
pamrevlumab (FG-3019) - December 2013 - June 2016 (~3 / month)
http://investor.fibrogen.com/phoenix.zhtml?c=253783&p=irol-newsArticle&ID=1982808 (initiated enrollment PR)
With how small the study is I really hope they know what they are doing with patient selection its still a very difficult disease with lots of failures. Anecdotally I've seen patients say they were turned down for one reason or another.
I was actually thinking it may be (slightly) bullish as with how thin the stock is perhaps it was some sort of prearranged sell/buy of course it could be a larger shareholder doing the same too
The volume was pretty high wonder if the ATM was part of the volume today?
BMY IO Combo's:
Couldn't the case be made that BMY would be more likely to do IO combo deals that could be synergistic?
NKTR said they will be combining with a Checkpoint inhibitor later this year in their Phase 1/2 214 study but didn't say which one(s). Their preclinical data shows very good synergy.
I am long NKTR so I am likely very biased
EDIT: Here is a link to NKTR's 214 page with access to their posters
http://www.nektar.com/product_pipeline/oncology_nktr-214.html
PDLI:
I am not surprised to see the stock down in AH. Royalty companies generally attract some investors who seek out a dividend. Successful ones tend to have a small dividend and raise cash opportunistically. I passed on PDL a long time ago because of the whole patent issue with Genentech (I thought it too risky to call) and their dependence on it was too much. I never got a sense that management was particularly astute to do royalties more that they needed a new business model. Don't know if that was fair but that kept me out and haven't followed them since.
DMD / PTC / SRPT:
I didn't listen to the call but skimmed the transcript. I thought this interesting (can't disagree with them) criticizing FDA for not treating applicants fairly (I think FDA already opened themselves up to this and other criticisms in how they've handled DMD drugs)
NKTR:
The Q&A on the conference call (as has been common lately) focused around 214. The company did not disclose much new information. They did say there have been no reports of Vascular Leak Syndrome (I did a little searching and saw that is a common IL2 side effect http://www.ncbi.nlm.nih.gov/pubmed/9403331 ). They reiterated the plan to start combination studies later this year and to provide some top-line data as a single agent (though they expect some patients to be on drug). One analyst really tried to push to at least see where they were but no luck. Said even with initial dose would expect to see some activity but no mention of what cohort (they are planning 6) or how many patients are enrolled.
In other news... 181 (their pain drug) completed enrollment and top-line data still expected for Q1 '17. Human abuse trial to start Q4 with data in Q2 '17. IND for NKTR-358 (autoimmune diseases) planned for Q1 '17. Phase 3 readouts from Bayer programs Cipro inhale and Amikacin inhale still 2H '16/Q1 '17. Ophthotech’s Fovista Q4 '16 readout.
Transcript: http://seekingalpha.com/article/3995447-nektar-therapeutics-nktr-ceo-howard-robin-q2-2016-results-earnings-call-transcript?part=single
(Slight OT)
ADXS / Royalties
ITEK:
http://finance.yahoo.com/news/inotek-pharmaceuticals-announces-pricing-50-110100032.html
8.02 conversion price. Pretty bad IMO, I didn't calculate it but looks like the 30 day average price.
ITEK:
They'll certainly need more cash the burn will be up and go up more. I am long but didn't add I want to see the terms of the debt (conversion price, interest rate) and hear the Q2 call before I'd consider it. I posted this on SI
http://www.siliconinvestor.com/readmsg.aspx?msgid=30685552
FGEN:
Your probably right looking at the exclusion criteria it says 5 years I didn't see any specific mention of when this woman had breast cancer.
https://clinicaltrials.gov/ct2/show/NCT02210559?term=FG-3019&rank=9
EDIT: I see her ECOG is 2 (the current trial has 0 or 1 as entry criteria)
I guess this way they don't get randomized to placebo arm. I imagine they are getting close to stopping enrollment too. From the Goldman call (and I believe Q1 too) they said they hope to wrap up the study and have discussions with FDA on next steps by end of year.
FGEN-3019 Single patient compassionate use trial for PC. Sign of things to come?
https://clinicaltrials.gov/ct2/show/NCT02851381
ITEK -30% on convertible debt offering of $50m. The filing doesn't have terms yet https://www.sec.gov/Archives/edgar/data/1281895/000119312516665617/d204894d424b5.htm
don't recall seeing such a big drop with terms not yet defined [well some may have an idea i guess].
I am long.
NKTR:
Nektar was up around 15% (on significantly higher volume) the past few days on no news (that I saw) if someone else saw something would appreciate it. AZN reported this week but Movantik numbers were far from spectacular 17M in Q1 to 23M in Q2.
If I were to venture a guess it would be some 214 info leaked. The study is open label and they should be well into the single agent portion of the study probably on one of the higher doses. I believe they were targeting Q3 to start the combination portion of the study.
They report August 3rd (after market) if one else is curious.
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, Updated August - November Events
Cantor Fitzgerald’s 2nd Annual Healthcare Conference
7/12-13
http://www.meetmax.com/sched/event_37633/~public/conference_home.html?event_id=37633
Canaacord Genuity Growth Conference
8/10-11
http://wsw.com/webcast/canaccord23/
Wedbush PacGrow Healthcare Conference
8/16-17
http://www.wedbush.com/services/cmg/equities-division/mgmt-access-events
Wells Fargo Securities Healthcare Conference
9/7-8
https://elink.wellsfargo.com/iconference/Conferences/Custom/List/141?menuItemId=100
Citi's Annual Biotech Conference
9/7-8
http://citiconferences.com/
RW Baird Global Healthcare Conference
9/7-8
http://www.bairdconferences.com/conference2/Index/8
Biocentury NewsMakers in the Biotech Industry
9/9
http://www.biocentury.com/conferences/newsmakers/dates
Rodman and Renshaw 18th Annual Global Investment Conference
9/11-13
http://www.meetmax.com/sched/event_35153/~public/conference_home.html?event_id=35153
Morgan Stanley Global Healthcare Conference
9/12-14
Bank of America Merrill Lynch Global Healthcare Conference
9/14-16
Aegis Capital Growth Conference
9/20-22
Ladenburg Annual Healthcare Conference
9/27
http://www.ladenburg.com/about-us/news-events/events
RBC Smallcap Conference
9/28
https://www.rbccm.com/about/cid-202541.html
Cowen 19th Annual Therapeutics Conference
10/5-6
http://www.cowen.com/conferences/upcoming-conferences/
BIO Investor Forum
10/18-19
https://www.bio.org/events/bio-investor-forum
Credit Suisse Healthcare Conference
11/7-9
Sanford C. Bernstein 4th Oncology Day
11/11
Stifel Healthcare Conference
11/15-16
Jefferies London Healthcare Conference
11/16-17
Canaccord Genuity MedTech & Diagnostics Forum
11/17
--
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.
Is 2Q16 a typo or is there typically a delay in meeting the BsUFA?
Is it publicly known if there are other 40mg generic applicants?
BMRN / Hemophilia-A / Gene-Therapy:
I'll take a stab at some of your questions and raise some of my own
I think the rapid rise in Factor VIII levels being a concern is not as big a deal as some people are making it out to be. At first I thought that and then one must consider there is A LOT of room to dose down with the incredible levels they achieved. What it may say is they still need to work on dosing. Since the mid (and low) doses seems to produce much lower levels so it would not appear to be an issue that can't be addressed. People also need to keep in mind this is just an early 7 high (9 total) patients its an exploratory study not the final registration!
yes there were still bleeding episodes. Look at slide 16 I think
http://files.shareholder.com/downloads/ABEA-3W276N/2477655309x0x901460/C2800939-0F80-4E18-9B69-30EFF1569B04/BMN_270_Data_Update.pdf
The episodes are down to 2 at weeks 9-28 when the Factor VIII levels are much higher than earlier time points AND see the asterisk all but 1 bleed from week 2-28 was in a single patient (the lowest responder)!
I am long BMRN so probably biased but I put the results in the incredible category! The levels are so much better than would be needed to be considered an effective treatment. I don't follow a lot of other Gene Therapies for other Hemophilia's but the few I've seen have much lower levels of production of the deficient factor.
There is still a question of durability and longer term safety.
What I wonder still about is can Gene Therapy be a commodity. It still appears to me there is no shortage of academic researchers and licenses seem to happen frequently. That being said I've been tempted to play QURE and ABEO in that event.
EDIT:
The thrombocytopenia was discussed (briefly) on the call. It is a concern high levels do increase risk but not dramatically and nothing being done at present and again dose may be optimized.