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Missed parts of it. Not taking notes on CC's lately though to post anything worthwhile.
I thought it interesting we will get some 262+214 data! They did say so at beginning of year guess skeptics could say its a way to downplay bad 214 Melanoma data? No mention of PROPEL which was also mentioned as having data this year!
Think that cash figure is wrong its $2 Billion (counting short/long term notes)
VYGR:
So when cohort 3 is worse than cohort 2 you pool them :-?
Thanks that was my interpration from reading it but the Twitter poster suggested something else so I thought they were listening and the company said so. It appears not.
Here is a link to the webcast for anyone interested. I noticed its ongoing if I caught it right think they said they are using a voucher for the S1P1 (MS).
https://edge.media-server.com/m6/p/rke9cn38
I saw someone tweet out a 4-5m price tag for their (via AVXS) SMA Gene Therapy. Does someone know if that was actually said or is it (mis)reading slide 48?
QUite a lot of slides on Avexis!
Not just A1AT but no HAE IND and no clue what to bring forward... aav10 remains questionable. Surprised not a bigger AH drop just AMD which has its issues and early stage partnerships (which probably now in doubt if aav10) and cash of course
Is there any human data on the dMAbs specifically about presence/lack of ADA's? If you could point me to a paper/presentation I'd greatly appreciate it!
Do you make anything of a few companies now getting out of FoB's? Is it the FDA approval bar being too high, competition or something else?
Thanks! Every once in a while I speculate right
https://twitter.com/MauriceOnTW/status/1050472728770297856
Too bad it's not in what I buy/sell lately
I saw someone tweet something about Roches IL-2 showing activity in monotherapy. I haven't seen the data though.
Should add that NKTR has a collaboration with SNDX (where they test 214 in combo with entinostat not a development license). Again I haven't looked at SNDX to see if its related or not.
I've never done more than a cursory look at FCSC. Their cap structure is so scary it's not really investible to me plus now someone pointed out their ownership interest isn't 100% and it's not clear how much they do own!
It's worth noting what happened with the EB program Amicus acquired which failed in Phase 3
http://ir.amicusrx.com/news-releases/news-release-details/amicus-therapeutics-announces-top-line-phase-3-results-sd-101
I heard a presentation (granted by someone from Standford involved with the ABEO program) who emphasized poor selection of wounds and not an unsurprising result occurred.
FWIW there are actually some published data with the ABEO results
https://jamanetwork.com/journals/jama/fullarticle/2576610
Hi Jason,
I don't follow the company closely but I have my doubts. Its quite dubious to NOT take questions when there naturally are some.
Its worth noting they have a shelf that just came into effect... It sure smells of an offering to me but I'm certainly no expert.
Here is what I tweeted earlier
https://twitter.com/MauriceOnTW/status/1051881632360218625
ONCE:
I closed out my short position and sold my puts today. I don't think the reaction was strong enough but I am not good at predicting short-term moves. I think when they present the Hemophilia A data it could bump down a bit more as they gave limited data and spoke in generalities I suspect highlighting the positive aspects. Further, I would be concerned with a couple things:
1-Its not clear a different steroid protocol will stop the immune response on all patients or may lead to lower overall factor levels.
2-The president suspected they may be seeing this with higher doses and that is why didn't see it in their B program (when asked about earlier steroid use)... my reaction was what happens when in the B program when you go to a 100+ patients I suspect could see some similar issues (though perhaps at much lower scale).
Katherine High came across so bad that I decided not to consider a long position while she is at the company. Here are a couple things
https://twitter.com/MauriceOnTW/status/1026925366349324288
ONCE:
Today seemed like a good day to go short (my first ever who knows maybe my last ever too ). Just a small short position and I bought a few (very small) # of puts yesterday (spread is too big to get a decent amount).
There can be a few good things (from ONCE's perspective) from their Hemophilia A program (Higher factor levels, more consistent individual and within cohort #'s) but with the appreciation in stock price, I think a good part of that seems to be priced in. Probably if BMRN has a serious setback would be a bigger plus for their A program.
On Hemophilia B (which they only have a share of with PFE collaboration) they now seem to be lacking an advantage over Uniqure (time wise and potentially efficacy wise as well). Anyway, I don't intend to hold my short position that long. I see playing QURE vs ONCE Hemophilia B as an argument much further down the road.
The earlier stage pipeline is actually what I find most intriguing from a long investment standpoint (Pompe, CLN2 and Huntington's) and why I would considering going long at much, much lower prices since they are very early still.
GLPG / ABBV:
The company (GLPG) had been guiding to about 5 as a good sign of efficacy. I think analysts were putting the low at 8%. I certainly am disappointed with the results (and would have with anything in the 5% range too).
I have been suggesting for some time that the two companies are not seeing eye-to-eye on the development path. Clearly, GLPG is much more aggressive (I think they have more cash then know what to do with so prefer to push everything forward and see where the data leads).
While a setback they have a pretty big development program in CF. They said about trying to dose 2737 higher and have another C2 corrector (3221) a bit further behind so I highly doubt they want to stop pursuing CF.
I am guessing they did/will claim ABBV is in material breach by not pursuing the second triple. Its a bit hard to gain useful information from the amended agreement (perhaps someone more skilled then I can!).
Here is the link termination starts on page 102:
https://www.sec.gov/Archives/edgar/data/1421876/000119312516610049/d201818dex101.htm
Here is my tweet saying pretty much the same thing
https://twitter.com/MauriceOnTW/status/1012465709119623168
SRPT:
As important from Adam's STAT article (missed it if was mentioned on SRPT call) is they will modify the study and go for approval on the one trial (I speculated as such on twitter on more than one occasion)!
https://www.statnews.com/2018/06/19/sarepta-gene-therapy-duchenne-early-results/
SLDB:
I heard the call. I am long SRPT (no SLDB) but think that post is misleading. The patient was given only 3 doses of Soloris. Furthermore (as I'll describe below) I HIGHLY doubt they were given a full go ahead and FDA has several concerns!
That being said I don't know how their GT will turn out but I don't trust management at all! Notwithstanding the IPO disclosure fiasco, this call had numerous concerns that management is being less than forthright in their disclosure. If one reads between the lines of what was said (and more importantly what wasn't) there are clearly some regulatory limitations. I suspect they have some serious concerns go ahead (if they really don't know the cause) or are misleading investors. Just a few things:
1-New Steroid course may affect efficacy (their answer was deliberately misleading IMO)
2-Now dosing just ambulatory children (instead of older non-ambulatory). Why? didn't say but could it because the total amount of dose is a concern with FDA possible safety concern?
3-Moved up one of the biopsies to as early as 6 weeks. Didn't say but I suspect they may be thinking efficacy is impacted by the new steroid protocol.
4-Didn't know when could move to the higher dose....That has to BS I suspect they know its dependent on certain safety criteria but they, of course, didn't disclose that.
5-They said that it was clearly an immune reaction suffered by the initial patient but they did not believe it was manufacturing related. This is catch 22 IMO. Mfg is very important in GT and a problem here would be a major setback (delay) but I think a less serious one if it's fixable...The alternative something with the vector/promotor/transgene which would basically end the program.
There were numerous other things I didn't like about management on that call too! These were just top of my head.
ONCE:
From the slide I posted the lower green box indicates that patients 3 and 4 were put on tapering steroids after a drop in FVIII levels. I'd have to go back to the call to see if they gave any additional information.
ONCE Hemophilia A GT Data:
Spark's upcoming Hemophilia A data was brought to my attention and I got to thinking can anything good come of this? From the prior data cut
https://twitter.com/MauriceOnTW/status/1006259636876521472
there were a lot of questions/concerns. At the time I actually thought there is a good chance the program gets terminated (at some future date not based on just the 4 patients)
1-The expression levels have wide variations at different time points for the individuals and amongst the 4 patients presented.
2-No sign of dose-response at all!
3-Steriods were needed (not to control ALT elevations) but to stop loss of expression!
4-The competition (BMRN) has people concerned because mean/median expression level is 50% after 2-3 years yet Spark is nowhere near that in the presented data.
Curious what others think. I actually don't dislike the company and cheap enough (much much lower price) would consider a position.
Also, what would be the best way to play this if I wanted to limit risk and yet gain more potential exposure? I thought to buy puts (but they are a bit pricey) so I'd need to speculate on how low it could go 40's (if they terminate the program or becomes more apparent that will happen) or perhaps somewhere in the 50's/60's is more realistic. Maybe someone more experienced in options knows a better way and can comment? I'd appreciate any ideas!
NKTR / BMY / MRK NSCLC:
Thanks appreciate your thoughts!
Just finished listening to the Jefferies NKTR call. They added slides breaking down when the response occurred for the various indicates presented at ASCO (slides 19, 22 and 25) here is a link to the slides
http://www.nektar.com/application/files/9715/2830/9113/NKTR_Jefferies_2018_Presentation_FINAL.pdf
The webcast replay is now available at:
http://wsw.com/webcast/jeff113/nktr/
BMY / NKTR / MRK NSCLC:
NKTR / PD-1/PD-L1 / NSCLC
For what its worth a couple of the docs on the call said they didn't believe there was much difference between PD-1's (don't recall if it was a general question or specific indication) but one said he believed PD-1's are a little bit better then PD-L1.
I am curious to see the PROPEL results to see if there is any difference in combo with 214 its fewer indications then PIVOT but even with the cross trial caveats, it should still provide some interesting comp's. I just wonder how much Nektar will REVEAL (little joke lets see who gets it).
On NSCLC I disagree with Dew I think the strategy changed but they will still pursue some segments just with a different strategy. They had previously mentioned PIVOT being expanded to have a few triplets (to include Ipi) and one would be in NSCLC. I tweeted this link which points to recent changes to the clinicaltrials.gov record
https://twitter.com/MauriceOnTW/status/1003724103654756352 or go directly via
https://clinicaltrials.gov/ct2/history/NCT02983045?A=7&B=8&C=Side-by-Side#StudyPageTop
Miljenko argued about competing on price
Triplet (NSCLC) may not be right strategy, little added benefit while tox issue in rise. I would prefer: define population where double (214 + Nivo) work in synergy and argue with PRICE and TREATMENT COST! $NKTR
— Miljenko Zuanic (@Miljenkoz) June 4, 2018
NKTR/BMY:
Is there a risk that mean OS up till 2 years (or whatever point is selected) trends in favor of placebo? I vaguely recall in one HALO trial that seemed to be an issue when I was a shareholder (of HALO) I thought dosing may be tricky if for example weakening the extracellular matrix allowed the tumor to spread. How would regulators treat a drug that short-term may increase the risk of death but for a subset significantly increase survival?
NKTR:
I'm still trying to digest the slides/call. A couple of things
1) I think Brad Lancor put it best about the debate continuing
My take on the Nektar data is that it just became the biggest battleground in biotech. Either you believe 1) the new patients from the phase two expansions will turn into responses with time or 2) phase one was a low n mirage. We’ll only know with time.
— Brad Loncar (@bradloncar) June 3, 2018
Should note the preclinical poster (RCC and colon) is NOT in the indication of the Phase 2 (2nd line Melanoma)
Here is the preclinical poster from AACR
http://www.nektar.com/application/files/1415/2364/7401/2018_AACR_NKTR-214_ABS_123.pdf
When I first saw the poster I thought the synergy seemed intriguing but I'm far from an expert here.
NKTR:
Agree with what you said. IL-2 therapy (in Melanoma at least) on its own I believe is like 9-10 cycles (though most patients can't tolerate more than a couple even in ICU environment). I believe its one of the best (though still low) chances for a cure just few can get through enough cycles.
There also wasn't (in the abstract) a break down of patients from those newer to therapy vs. reported earlier to know the ORR of the same patients.
The other angle people are overlooking is 214/Nivo is not the only route forward. They are pursuing multiple combo's though most are earlier stage (there are a couple of posters at ASCO) it seems the new deal will have triplets (214, Nivo, Yervoy).
RGNX:
I don't think they changed the royalty rate from my notes when I was (very) close to buying I have mid-single to low double digit. The concern was how far out they would pay the royalty given the limited IP on AAV9. I thought in the new deal it clarified a bit about how long the royalty extends but not sure.
No position in RGNX but if the IP extends to the duration of it being on the market if one has any confidence in the asset/license I'd think RGNX should be up A LOT more just on the AVXS royalty not to mention additional premium for their vector/other licensees.
EDGE:
Heard the call (all 10 minutes of it, 1/4 is safe harbor ) said drug performed as expected... the dread control arm much better than any past study or any data that they're aware of.
88 million cash at end of December burn 4-5m per month. Doing controlled shut down of the program and already laying off staff. Only other program is preclinical. They have 17+m debt though and from the K looks like 30+m shares. Probably should be a reverse merger but not a big enough discount to cash (for me) to speculate especially since don't know if management will try to keep things (their jobs) going a bit longer.
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
FOR EVENTS/LINKS ALSO SEE:
https://www.troutcapital.com/conference-events/calendar
Edits: Removed obsolete entries, Updated April-May Events.
Roth Annual Growth Stock Conference
3/11-14
http://wsw.com/webcast/roth32/
Cowen & Co. Annual Health Care Conference
3/12-14
http://wsw.com/webcast/cowen46/
Barclays Capital Global Healthcare Conference
3/13-15
https://cc.talkpoint.com/barc002/031318a_as/
Oppenheimer's 28th Annual Healthcare Conference
3/20–21
https://www.opco.com/conferences/nyhealthcare18/index.aspx
Cowen 17th Annual Life Sciences Winter Meeting in Colorado
3/20-23
BioCentury Future Leaders in the Biotech Industry Conference
3/23
Needham Healthcare Conference
3/27-28
http://wsw.com/webcast/needham86/
Strongbridge Biopharma plc Investor and Analyst Day
4/5 8:00am
https://www.webcaster4.com/Webcast/Page/359/25047
H.C. Wainwright Global Life Sciences Conference
4/9–10
http://wsw.com/webcast/hcw2/
Mylan 2018 Investor Day
4/11 1:00pm
https://edge.media-server.com/m6/p/efbyioq5
ARM Annual Cell & Gene Investor Day
4/17
http://arminvestorday.com/
Revance to Host Investor Day on April 19, 2018
4/19 12:00pm
https://edge.media-server.com/m6/p/g9hwdgij
BioTrinity Conference
4/23-25
https://www.biotrinity.com/
Deutsche Bank Healthcare Conference
5/8-9
Bank of America Merrill Lynch Healthcare Conference
5/15-17
Citi Swiss Healthcare Day 2018
5/17
UBS Global Life Science Conference
5/21-23
https://www.ubs.com/microsites/ib-conferences/americas/global-healthcare-conference/en/overview.html
Goldman Sachs Global Healthcare Conference
6/12-14
--
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2. Make your additions or modifications, inserting new items in alphabetical or chronological order as the case may be.
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They are on retailroadshow now and show is Unum Therapeutics
https://www.retailroadshow.com/
Thanks for pointing out the valuation comp to Gene Editing companies (I don't really follow that area). Not sure how I see them since they are both. Seeing that they have over 100m in cash already the EV (at say $15 IPO) would be about 300m so not quite as bad but still, they are preclinical and I don't like their lead programs in both Gene Editing and Gene Therapy are in PKU (too competitive).
Oppenheimer Webcast links
Go to
https://www.opco.com/conferences/nyhealthcare18/index.aspx
for password enter: march18 (case sensitive)
Then select "Webcast" from "ADDITIONAL LINKS"
Thanks for the information!
Yes both are intended for both FDA and EMA filings.
QURE's isn't posted yet but here are (both) BMRN's (second is low dose and will have 3rd with patients with existing AAV5 antibodies
https://clinicaltrials.gov/ct2/show/study/NCT03370913
https://clinicaltrials.gov/ct2/show/study/NCT03392974
With how much factor level increases I can't imagine any of these (along with Sparks for that matter) not having a (highly) significant impact on bleeds.
Should add that BMRN is measuring baseline bleeds by patients historical records. Both companies are doing open label trials.
Phase 3 Different trial designs by companies
What does one think when two companies with similar programs have different Phase 3 plans particularly on the primary end point. Especially when the design of one would lead to a significantly faster timeline. Does it speak to the competence/relationship with regulators of one vs. the other? Are there legitimate reasons this could happen? Since its Phase 3 I doubt one is directly lying about agency guidance but perhaps stretching things a bit?
The question is general in nature but what prompted my question is thinking of the difference between QURE and BMRN (granted one is Hemophilia A the other is B).
QURE primary Bleeds (6 month run-in to measure bleeds)
BMRN primary change in Factor level
Note both have others primary as secondary though BMRN uses "well documented" history (no run in) to measure bleeds. Both also measuring amount of Factor replacement used amongst other things.
I don't know why some people keep saying that statin intolerance isn't a thing! I had a harrowing experience as well. I started on low dose Lipitor (I think 10mg) and for a few month didn't notice anything. Then I started to get cramping but didn't suspect Lipitor then on a vacation it started getting so bad I couldn't walk. Discontinuing the drug resolved symptoms in a couple days as I recall. The doctor had me try low dose pravastatin I had symptoms of tendon pain and this time I stopped quickly before risking a repeat scenario as with Lipitor. Fortunately (for me) my levels were not too bad that my doctor suggested just diet modifications and accept a little higher level (said guidelines changed for my "older" age anyway).
Thanks jbog appreciate the info!
I have till Jan '19 just the 1 year holding period is about up to be long term gain so was wondering what's best to do. If I exercise and hold do I only pay taxes when I sell the stock?