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RTRX:
I heard the call and am curious what longs think about them trying to do a deal to get a commercial product? It seems with running a couple trials now and possible payments for an autism drug the company may need to do a financing fairly soon. I wonder if the price decline is in part to people sensing that? I don't follow the company as close as some others so perhaps I'm missing something. I do like the PKAN program and Martin's focus on rare diseases with well understood mechanisms. I'm not sold on their FSGS program though.
BMRN Panel:
Lunch time now. I've missed parts but haven't heard anything of major concern. Seems the minor concerns are the limited efficacy and if the benefit plateau's and if patients in a certain age range have the most benefit. One panelist (didn't catch her name) seems to be obsessed (my opinion) about antibodies and analyzing the 5 patients no longer on drug to see if (longer) dose interruptions could provide benefit. Not questioning her theory just seems like something not practical with current data/at this time and would take years to try to optimize anyway.
David Miller is tweeting some of his takes for those interested @AlpineBV_Miller
NVIV:
Thanks for posting the article. Do you or anyone know if Astrue is a true interim and made it known he doesn't intend to stay on or is that an option still on the table?
BMRN:
In going through the documents I didn't read anything of concern (yet anyway). I did find this interesting.
BMRN:
Briefing docs our out
http://www.fda.gov/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/EndocrinologicandMetabolicDrugsAdvisoryCommittee/ucm331504.htm
There will be a free webcast too information in this link
http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/EndocrinologicandMetabolicDrugsAdvisoryCommittee/UCM375124.pdf
Direct Link:
https://collaboration.fda.gov/emdac1119/
Breakthrough Designation
There is an article in the NEJM that talks about it in more detail. Nothing new that I noticed it does have two nice tables though one with a list of those receiving the designation (through 9/30/13) and one with summary table of the differences between the various Expedited Programs from FDA
http://www.nejm.org/doi/full/10.1056/NEJMp1311439?query=TOC
LGND:
Here is a link to an 8-k with the slides for todays analyst day
http://www.sec.gov/Archives/edgar/data/886163/000088616313000110/analystdaypresentation11.htm
And a direct link to the webcast (10:30am Eastern)
http://www.investorcalendar.com/IC/CEPage.asp?ID=171865
Is the test hard to administer? I recall a question on it and if I recall correctly I think Hank said there were a couple ways to measure.
SRPT / BMRN:
That actually makes a lot of sense, surprised I didn't think of that ! Since it seems FDA will accept that endpoint in Pompe should be quite applicable to DMD too. With BMRN getting more involved with Pompe I wonder if their interest in DMD is elevated (I think they still have some research programs there). I highly suspect BMRN has talked/looked at SRPT and think they could probably help out a company like SRPT on the regulatory and probably commercially too, whether SRPT would be up for a deal with them or not though I don't know.
One could argue people thought AA was a given because of the CEO's comments (Matt Herper had a good article earlier today). I think masterlongevity had a good point too you probably see it coming even if FDA changed their line of thinking after the RNA and PTC failures.
SRPT / GSK / RNA:
SRPT:
Would an endpoint that has a measure like 6MWT be 0 when a patient becomes non-ambulatory? I recall some MPS studies had composite like 6MWT and FVC but thought they generally enrolled "healthier" patients to be able to complete the test. Do you have any ideas on what type of composite endpoint may be able to maximize the benefits of the drug?
Just to continue the discussion from twitter I'd prefer that someone else help them in their Phase 3. I haven't followed the company as close as you/others but Chris has never given me a comfortable sense of being able to do things in a manner suitable for regulators. GSK seems like the likely partner of choice given their involvement with RNA and the need to get access to the IP. Since I like a bigger partner commercializing on a global scale it would seem a good fit and its not like GSK hasn't done a similar deal before (FOLD) it may also help reduce the need to do a financing or two till the drug gets approved (I also like having access to RNA's data and getting them to have more input on the Phase 3).
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: Added Ligand Analyst Day, Updated Lazard and Jefferies links
BIO Europe
11/4-6
http://www.ebdgroup.com/bioeurope/index.php
Credit Suisse Healthcare Conference
11/11-14
http://cc.talkpoint.com/cred001/111313a_jw/
Canaccord Genuity Medical Technology & Diagnostics Forum
11/14
http://wsw.com/webcast/canaccord12/
Goldman Sachs US Emerging / SMID Cap Growth Conference
11/14
http://cc.talkpoint.com/gold006/111413a_gm/
Ligand Analyst Day
11/14 10:30am
http://www.ligand.com
Lazard 10th Annual Healthcare Conference
11/19-20
http://wsw.com/webcast/lz15/
Jefferies 2013 London Healthcare Conference
11/20-21
http://wsw.com/webcast/jeff80/
Brean Capital 2013 Life Sciences Summit
11/25
J.P. Morgan SMid Cap Conference
11/28-29
Piper Jaffray Annual Healthcare Conference
12/3-4
Oppenheimer Annual Healthcare Conference
12/10-11
Bank of America Merrill Lynch Healthcare One-on-one Conference
12/12
Biotech Showcase 2014
1/13-15/14
http://www.ebdgroup.com/bts/presenters/prs_comps.php
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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.
SRPT:
Matt Herper has a nice article (though I don't agree with all of it) on the delay and his opinion of management is inline with mine. I did/do think FDA should be more lenient especially when no options exist and no safety concerns have appeared (granted a small n).
I think Matt makes a good point about making sure the right trial is done I do have my doubts about current management.
http://www.forbes.com/sites/matthewherper/2013/11/12/what-the-delay-of-a-promising-muscular-dystrophy-drug-means-for-patients-investors-and-all-of-biotech/
DDXS:
Here are my notes on IP:
2014 Patent begin to expire (extend to 2016 Composition of Matter), Second family that if issued could extend to 2024 (Lp-PLA2 presence of an inhibitor like darapladib) Q3 ‘12 Call (Q&A), 5/21/12 Call (Q&A)
Not sure which expire in 2014 according to my notes COM goes to 2016.
SRPT:
The cash is 273.6M as of 9/13 according to the Q
http://www.sec.gov/Archives/edgar/data/873303/000119312513436840/d595042d10q.htm
Also the share count may be a bit higher than you are calculating so the figure may be somewhere between
DDXS:
The other issue (for me) of greater consequence is their IP is expiring and new IP is tied to use with an inhibitor (like darapladib) so it has the subgroup analysis has to be promising enough for GSK to go forward in new studies.
It looks like there will be a free webcast. Here is the link from the FDA sight it seems the link will be posted nearer to the date.
http://www.fda.gov/AdvisoryCommittees/Calendar/ucm371064.htm
Do you (or anyone) know if their will be a (free) live webcast of the panel? Any link would be appreciated TIA
With Ultragenix going public my dream would be BMRN gets bought out at a nice premium the IPO for Ultragenix isn't too pricey and Emil decides to move up to Chairman and hires JJ becomes the CEO .
Here is a link to their filing for those interested (hopefully not too many are )
http://www.sec.gov/cgi-bin/srch-edgar?text=Ultragenyx+&first=2013&last=2013
and the company website
http://www.ultragenyx.com/
Edit:
maybe it won't be so cheap
The number of shares of common stock to be outstanding after this offering is based on 11,607,173 shares of common stock outstanding as of September 30, 2013 and 61,431,574 additional shares of our common stock issuable upon conversion of all of our outstanding shares of preferred stock upon closing of this offering.
I did do a quick listen to the call and they said they are still open to other deals though it seems like it will be a while... I think an ERT company would have offered a lot of synergy too .
I'm not a long time follower of VPHM but the past year or two they seemed to be especially sensitive to competition questions maybe that is just addressing what the analysts were asking but before discontinuing it they were quite hopefully with the HALO sub-q. I think they did a good service to their shareholders and picked a good time to sell but time will tell
Shire / HAE Market:
IMO I thought they overpayed given its growth and potential competition from other delivery options and possibly even generics (though perhaps small risk here). I don't believe their pipeline is that good (as compared to say a BMRN which I would have thought a better deal for Shire).
Thanks I thought they lost exclusivity early (2012) but haven't kept up. I guess in 2016 PAH may not be an Orphan disease if they go by scripts . If patients had to go through a right heart catheterization probably would discourage a lot from a cheaper script .
NKTR:
Also guided on $50 million higher cash balance to end the year (partly because of delayed '181). One noteworthy thing is Robin softened his stance on partnering 102.
Isn't Revatio (the PAH indication for sildenafil) already generic? How difficult is it to maintain the price with a generic in another indication?
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: Added Goldman Sachs US Emerging / SMID Cap Growth Conference
BIO Europe
11/4-6
http://www.ebdgroup.com/bioeurope/index.php
Credit Suisse Healthcare Conference
11/11-14
http://cc.talkpoint.com/cred001/111313a_jw/
Canaccord Genuity Medical Technology & Diagnostics Forum
11/14
http://wsw.com/webcast/canaccord12/
Goldman Sachs US Emerging / SMID Cap Growth Conference
11/14
http://cc.talkpoint.com/gold006/111413a_gm/
Lazard 10th Annual Healthcare Conference
11/19-20
http://www.lazardcap.com/conferenceList.aspx?tn=4&ln=3&itm=4508&lid=3002
Jefferies 2013 London Healthcare Conference
11/20-21
http://www.jefferies.com/OurFirm/Conferences/325/108
Brean Capital 2013 Life Sciences Summit
11/25
J.P. Morgan SMid Cap Conference
11/28-29
Piper Jaffray Annual Healthcare Conference
12/3-4
Oppenheimer Annual Healthcare Conference
12/10-11
Bank of America Merrill Lynch Healthcare One-on-one Conference
12/12
Biotech Showcase 2014
1/13-15/14
http://www.ebdgroup.com/bts/presenters/prs_comps.php
--
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.
If the patents hold and SRPT's data continues to be good I'd imagine they agree on some sort of licensing deal. It seems there would be an outcry if the drug isn't made available there. Is that the value you see in RNA or do you think they'll be more successful with other candidates?
Thanks for pointing that out, I wasn't aware of that. It'll be interesting to see how results are with the deal. I may well turn out wrong but I thought margins would not be as good. The terrible terms were a big part of what made me sell.
10 Years ago Fred Price would have been CEO and the Orapred disaster only to be followed by (IMO) a worse interim CEO. I haven't seen SRPT do anything that bad .
It was before my time but I suspect it was Price who did the Aldurazyme deal. Even though Aldurazyme didn't turn out as big as some thought I'm sure getting it approved and available world-wide it helped having Genzyme as a partner. I'd still think especially if the technology proves applicable to other exon's a partner could help them and if they have x-US prospects they could benefit greatly there. Watching even a company the size of UTHR struggle getting approved much less market x-US makes me a bit cautious when a small company thinks they can do it alone OK.
SRPT:
Just to clarify I am NOT suspect management of fraud and haven't heard that before the article. Granted not from a view of someone who follows them closely I do I think they are not the most competent team.
I haven't heard anything specifically but I get the sense companies (like BMRN) have looked at them for possible partnerships of some sort and either passed or SRPT management wasn't willing to do a deal. I can't think of either scenario being bullish in the short-mid term for the company. My sense is management thinks too highly of themselves and perhaps that has hindered a deal?
SRPT:
Terrible in what way? I don't follow the stock too closely but haven't heard the fraud argument the author mentions. I don't know that I would buy the argument the FDA would not require muscle biopsies if it weren't going to approve it early either.
I do not have a position in the stock and while I have believed the drug is active I was skeptical on early approval. I still am but am a bit less so. I'd point to what other companies (BMRN is one I'm very familiar) have said about possible approval with 1 trial in conditions of extremely high unmet need (Batten's in BMRN's case).
I do wonder about the company management though I don't have reason to suspect fraud. I more wonder why they haven't been more aggressive in developing treatments for other exon's and getting manufacturing in place in a more timely fashion. I don't buy the argument they can to do it alone as fast as with a bigger partner and they clearly made at least some mistakes in designing their trial so I would be a bit leery on them executing well in the clinic (though if the drug is anywhere near as good as seems they may not have to).
Thanks researcher. I figured one of these times my Bio picks would pay off and this time the sector seems especially strong. DDXS I sold about half of my real time portfolio since they have a fairly big binary event coming up I thought to freeze the position. GENT was a stock I thought cheap and have owned it for some time they finally got the EU nod on their drug and analysts/investors are finally connecting the potential.
I had GENT in the microcapclub contest but couldn't keep up with MikeDD who did an outstanding job with 4 out 5 picks up over 100% (I think it was an 18 month contest).
I heard the call before I saw the FDA document and got a different picture from what Berger said to reading the document. When Berger was asked his first response was something like good question or good point. Then upon reading the document and hindsight at what he said made me wonder if the company won't exactly jump through hoops to make the drug available. I wonder if Berger is somewhat trying to leverage that as some sort of bargaining chip? Its certainly not fair to do for sick patients with alternatives but I am a *new* shareholder and don't know him that well.
Probably doesn't mean anything but just noticed that there were 5 CT Orders filed on the 30th. Usually I see one or two at a time don't think I've ever seen 5.
OK Thanks for the clarification noticed now the 7th is the date on the online version.
ARIA:
More data to come 11/7 in NEJM (thanks to @AndyBiotech for the link)
http://www.ft.com/intl/cms/s/2/c5d45314-3db3-11e3-9928-00144feab7de.html#axzz2jQWgWXKJ
Thanks should be an interesting read. Interesting timing and this was out today too
http://m.clincancerres.aacrjournals.org/content/19/21/5828.short
ISDR:
Nice jump today. I don't see any news. No surprise its gone on since the post I'm replying too, the people still in can thank me for selling .