...
Register for free to join our community of investors and share your ideas. You will also get access to streaming quotes, interactive charts, trades, portfolio, live options flow and more tools.
Register for free to join our community of investors and share your ideas. You will also get access to streaming quotes, interactive charts, trades, portfolio, live options flow and more tools.
Appreciate the additional detail Peter.
"orangeone" previously pointed out Astellas has an anti CTGF but I am not familiar with their program.
http://investorshub.advfn.com/boards//read_msg.aspx?message_id=114249898
FGEN-3109
Do you know if they are actively working on follow on compound(s)? I ask for a couple reasons the IP can't be very good and Neff notably pointed out interest in Orphan indications. I am not a science guy so may be a dumb question but is it possible to PEGalyte or otherwise encapsulate an MAB to improve the bioavailability?
FGEN:
FGEN:
Quiz Answer:
Dew got the answer earlier. You have to be quick to beat him and genisi .
http://investorshub.advfn.com/boards/replies.aspx?msg=122513517
Basically it is reimbursement that is the main driver. What I found interesting was FGEN noted that in cities its better then rural areas.
Good job.
Government reimbursement is what FGEN said. They noted that reimbursement is higher in cities then rural areas so your comment about cities is especially relevant.
Correct its not the one I am looking for
Quiz (not open to anyone who heard FGEN CC):
What is the primary driver to increased dialysis use in China? 450k in 2015 15% annual growth from 2011
Hint:
Its sorta a trick question the key is how the question is worded
I looked over the paper but nothing overly convincing to me, I imagine it will be how good the sales effort will be.
From an ideological standpoint and (bad) personal experience I think agitation/aggression meds for Alzheimers are something pushed by nursing homes and the patient may truly benefit from better care. Thats not even to mention the side effects from some which IMO are far greater then the "problem" from a health standpoint. I recall reading an article with a nursing home where when the ratio of patients to aids was reduced so did the amount of agitation and aggression, go figure huh .
Thanks for the SA link!
I am still not sold on 786 but the Celgene partnership was one I over looked. It would probably be nice to see some of their data (if any is publicly available) and how benefit the deuteration is actually adding. Will have to look for some publications if have any links would appreciate it.
I've wondered why VRTX never (or couldn't if they thought it meaningful) deuterated ivacaftor
CNCE:
Thanks for the reply. The attraction was that their deuterated drugs could provide enough advantage to extend the IP of products there replacing and also offering a lower risk to approval. I'd have to be comfortable that the benefit is significant enough.
@bio_clouseau on twitter pointed out that their Pirfenidone interest was just patent related (and very small royalty) so its unlikely they'd get rights to the drug. I thought that may be the most interesting if the drug reduces abdominal AE's and could get QD dosing would be a nice way for a competitor to bring a combo to market.
Thanks for the info/reply.
I wasn't aware Novartis had a set back with their Potentiator. Not sure what you mean by "Bps"? I think it will be difficult for them to take just a potentiator to market by the time they get there VRTX / GLPG and perhaps others (think PFE in addition to Novartis had programs) may have triple combo's.
I never got overly impressed with 786 to think it could be a big drug.
I don't know why the stock dropped of late the deuterated Jakafi isn't something I would place much value in at this point (Isn't someone working on a topical JAK for this?)
Concert Pharma (CNCE):
Anyone long/following the company who can update me on a few things?
I was always more intrigued by their early stage assets now that the late stage are becoming a much lower part of the valuation I thought to take another look but I still have some questions that make it hard to invest.
1-2nd generation Xyrem. Have they been able to figure out a good formulation is the program advancing?
2-The Deuterium Pirfenidone program (now with Teva). Is it moving forward or can they get the rights back at some point if Teva isn't advancing it?
3-the deutrerated ivacaftor what is the plan going forward (how far will they take on own, develop other CF compounds?). I don't see a logical partner even with different mechanisms one would think its still best with correctors in the mix.
I really want to like the stock since it would seem to offer a bit lower development risk and its in the 2x cash range but I don't have a good comfort level with the path forward for the three above programs and unfortunately not much confidence in management. Anyone want to help me see that its worth a deeper look?
Thanks in advance!
Looking over the 10Q here are things I noticed:
1) Enrollment Figures From Q/K
As Of 9/30/15 110
as of 2/29/16 150
as of 4/29/16 165
So really no big step up in enrollment (7-8 avg) maybe a bit worse when figuring december/january likely slow and summer in EU things slow down so if pick up need it to be in Q2 otherwise maybe into Q1 to hit 230+.
2) Here is more info on the NIH study
DB Webcast Link May 4-5
http://edge.media-server.com/m/s/y55rmh2k/lan/en
BIND Therapeutics Initiates Voluntary Chapter 11 Bankruptcy Protection Proceeding
http://finance.yahoo.com/news/bind-therapeutics-initiates-voluntary-chapter-100000384.html
Company does not anticipate disruption to ongoing operations and financing activities
Business Wire BIND Therapeutics, Inc.
CAMBRIDGE, Mass.--(BUSINESS WIRE)--
BIND Therapeutics, Inc. (BIND), a biotechnology company developing targeted and programmable therapeutics called ACCURINS®, today announced that it has elected to file a voluntary petition under Chapter 11 of the Bankruptcy Code in the U.S. Bankruptcy Court for the District of Delaware.
"We believe this decision is in the best interests of the company and its stockholders,” said Andrew Hirsch, president and chief executive officer, BIND Therapeutics. “The protections afforded by Chapter 11 provide for an orderly process and additional time that enables us to pursue the strategic and financial alternatives that are in process. The filing minimizes the impact from the recent demand by our lender, Hercules Technology III, L.P, for accelerated repayment of our outstanding loan. Our current cash and assets exceed the loan amount, and we are current on our regularly scheduled repayment obligations. Through this process, we expect to be able to maintain ongoing financing activities and collaborator obligations while moving our R&D initiatives and pipeline forward.”
BIND intends to continue to manage and operate its business under the jurisdiction of the Bankruptcy Court and in accordance with the applicable provisions of the Bankruptcy Code and the orders of the Bankruptcy Court. The Company is working with an investment bank to review financial and strategic alternatives with the goal of maximizing stockholder value. Potential alternatives to be explored further and evaluated during the review process may include raising additional capital, a strategic collaboration with one or more parties, or the licensing, sale or divestiture of some, or all, of the Company’s proprietary technologies.
BIND plans to continue its development and collaboration activities in accordance with its current innovative medicines strategy throughout this process.
SRPT:
I took it as there is still a lot to know about the disease. While I would agree that sponsors tend to state things in their favor outside of trial issues the FDA arguments were more open ended (e.g. how much dystrophin is meaningful, what clinical endpoints may be most appropriate). While SRPT could be faulted for many things I thought in a disease with a lot of heterogeneity FDA wanting to do comparisons by age (vs. other base line characteristics) may not be the most accurate comparison.
The ITMN panel was the first one I saw at the time I took the FDA's doubt on FVC as indicative it was not a meaningful prognostic indicator on outcome. Added to that ITMN failed to get an SPA at the time I remember Dan Welch saying something to the effect that sometimes the agency is a little slow to catch up when a disease has no treatment. I actually didn't believe him at the time. In retrospect I think he may have been being quite fair in his assessment and certainly in with the ASCEND results they were able to correlate FVC decline with mortality.
OCRX: Ocera Therapeutics Reports First Quarter 2016 Financial Results and Pipeline Progress
http://ir.ocerainc.com/releasedetail.cfm?ReleaseID=968219
-- Company on target to complete enrollment in STOP-HE in the fourth quarter; approximately 165 patients enrolled to date --
-- Study with orally-available OCR-002 in patients with cirrhosis to begin in the third quarter with data expected by year-end --
PALO ALTO, Calif. and RESEARCH TRIANGLE PARK, N.C., April 29, 2016 (GLOBE NEWSWIRE) -- Ocera Therapeutics, Inc. (NASDAQ:OCRX), a clinical stage biopharmaceutical company focused on acute and chronic orphan liver diseases, today reported financial results for the quarter ended March 31, 2016, and provided updates to its clinical development programs of OCR-002 for the treatment of hepatic encephalopathy (HE), a debilitating liver disorder and significant burden on the healthcare system.
"We are very encouraged by the continued enrollment momentum in our STOP-HE study and thanks to the enthusiasm of our clinical trial sites, we now have approximately 165 patients enrolled to date," said Linda Grais, M.D., Chief Executive Officer of Ocera. "We remain on track to complete enrollment in the fourth quarter of 2016 with top-line results to be published soon thereafter."
"Following the promising pharmacokinetic (pK), safety and tolerability results of multiple orally bioavailable OCR-002 prototype formulations in a Phase 1 study with healthy volunteers, we have spent this quarter actively designing and preparing for an additional oral Phase 1 study, this time in patients with cirrhosis," said Stan Bukofzer, M.D., Ocera's Chief Medical Officer. "We expect that the study will be conducted in two parts: Part A will evaluate the pK and bioavailability of a single oral dose of OCR-002 in cirrhotic patients dosed under certain conditions (fasting, fed, with lactulose); Part B is expected to be a multi-dose study which will include the evaluation of steady state pK and the formation of phenylacetylglutamine (PAGN), the end-product responsible for clearing ammonia. We expect enrollment in Part A to begin in the third quarter of 2016 with data anticipated by year-end 2016."
Select First Quarter Financial Results
As of March 31, 2016, Ocera had cash, cash equivalents and investments of $39.9 million.
Net loss for the quarter was $7.5 million compared to a net loss of $6.7 million for the same period in 2015. Basic and diluted net loss per share were $0.36 for the quarter compared to basic and diluted net loss per share of $0.34 for the same period in 2015.
Research and development (R&D) expense for the quarter increased to $4.7 million, from $4.4 million for the same period in 2015 due primarily to an increase in personnel costs due to higher headcount and related expenses.
General and administrative (G&A) expense for the quarter increased to $2.6 million, from $2.3 million for the same period in 2015 due primarily to an increase in personnel costs due to higher headcount and related expenses.
Net interest income (expense) of $246,000 for the quarter was primarily attributable to interest and amortization associated with the debt facility which closed in July 2015.
Financial Guidance
Ocera reiterates its previous guidance and expects net use of cash for 2016 to be between $26 million and $30 million, and expects that it will have sufficient cash to fund operations into the second half of 2017 based on its current operating plan. If Ocera receives the second $10 million tranche of its debt facility, which is subject to the achievement of certain financial and clinical milestones, the Company expects that it will have cash to fund its operations into late 2017.
Wow this is a bit of a shocker.
http://espn.go.com/mlb/story/_/id/15425125/dee-gordon-miami-marlins-banned-80-games-peds-violation
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 May-June conferences
Bank of America Health Care Back-to-Basics Forum
4/5
OCUL Investor Day
4/6 12:00pm
http://investors.ocutx.com/phoenix.zhtml?c=253650&p=irol-calendar
Jefferies Immuno-Oncology Summit
4/7-8
Barclays Healthcare 101 Day
4/8
Goldman Sachs Alzheimer’s Symposium
4/11
15th Annual Needham Healthcare Conference
4/12-13/16
http://wsw.com/webcast/needham76/
Bloom Burton & Co. Healthcare Investor Conference
5/2-3
https://www.bloomburton.com/conference/
Deutsche Bank 41st Annual Healthcare Conference
5/4-5
https://conferences.db.com/americas/healthcare16/
Bank of America Health Care Conference
5/10-12
Piper Jaffray 3rd Annual GenomeRx Symposium
5/17-18
http://www.piperjaffray.com/2col.aspx?id=365
2016 Marcum MicroCap Conference
6/1-2
http://www.marcumllp.com/microcap
Goldman Sachs 37th Annual Global Healthcare Conference
6/7-9
Jefferies 2016 Healthcare Conference
6/7-10
http://www.jefferies.com/OurFirm/Conferences/325/267
William Blair 36th Annual Growth Stock Conference
6/14-16
https://www.williamblair.com/News-and-Events/Events/2016/June/14/36th-Annual-Growth-Stock-Conference.aspx
Citi European Healthcare Conference 2016
6/21-22
ROTH Capital Partners 3rd Annual Healthcare Day
6/22
J.P. Morgan European Healthcare Conference
6/23
Jefferies Alzheimer's Summit
6/28
--
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.
Opportunity is Everywhere if you know where to look. Get Started at E*TRADE.
SRPT:
I too thought the panel was more favorable then expected especially in light of how the questions were worded.
What was very interesting were the clarifying questions to the FDA (along with comments by panel members) before voting on Question 7. It seemed pretty clear if the portion inside the parenthesis were omitted several panel members would have voted the other way. When Janet Woodcock was asked about taking into consideration the patient voice in voting there was a deliberate hesitation and her response made me think she wanted the panel members to not consider that without coming out against the 2012 guidelines.
An interesting point was made by someone (I think with FDA) this was truely a unique situation basically every patient (or a family member) of the efficacy study was represented at the meeting.
I didn't read it yet (was actually looking at the XON story) when I noticed it. I don't check SA as much since Yahoo dropped them.
XON down presumably on this SA article. Even though he has been very successful in a few Bios I never got the sense Kirk was an especially astute Biotech entrepreneur.
Intrexon: The Public Markets' Theranos Part 1 - Zika Virus Hype Is Nonsensical
http://seekingalpha.com/article/3966892-intrexon-public-markets-theranos-part-1-zika-virus-hype-nonsensical
h/t @SashaDamouni
Does anyone know When was the speed of the ball was measured (i.e. initial release, when it reached the plate, etc.)
THey said he was a former pitcher (HS I believe) and could throw pretty hard. Maybe Chapman should throw balls home when he is shagging flies so we could compare
Ocera Therapeutics: A Speculative Investment
http://seekingalpha.com/article/3966219-ocera-therapeutics-speculative-investment
BMRN:
R&D Day Webcast: http://edge.media-server.com/m/p/794und98
R&D Day Slide Deck: http://investors.bmrn.com/common/download/download.cfm?companyid=ABEA-3W276N&fileid=886844&filekey=B10FD964-0DAB-431C-8729-BFBDB99462D6&filename=BioMarin_RD_Day_4_20_2016.pdf
Hemophilia A PR: http://investors.bmrn.com/releasedetail.cfm?ReleaseID=965945
Vosoritide (for Achondroplasia) PR: http://investors.bmrn.com/releasedetail.cfm?ReleaseID=966001
Only caught parts live (replay available now). The hemophilia data seems quite encourage I am a bit concerned about the ALT elevation which seemed to occur much after dosing (at least in one patient). Did respond to steroid course (missed answer to question on wether expect to continue on steroid or taper off). Vosoritide data also seems quite encouraging.
Company sounds more optimistic on PEG-PAL (then I am at least). One of the most promising and not widely valued/discussed by Analysts, CLN2 very encouraging as well. Think likely more patients then thought currently believe 1200-1600. Believe may have opportunity for even faster review (then priority) given BTD and severity/need.
PTCT:
NICE Recommends Translarna™ (ataluren) for the Treatment of Patients with Nonsense Mutation Duchenne Muscular Dystrophy in England
http://finance.yahoo.com/news/nice-recommends-translarna-ataluren-treatment-151600792.html
I wonder just how NICE (pun intended) a discount they got to get the recommendation. Still a surprise positive for PTC
NKTR / IL2
I would guess only a small part of current valuation is 214. The bulk being in its producing (and near term potential) royalties and some in its pain program.
IL2 is actually a fairly old approach in Melanoma its had very good success the problem is its so poorly tolerated it must be done in a specialized center and most patients can't tolerate many rounds of treatment. Nektar is going to explore a few different tumor types as single agent then try to combine with PD1/Checkpoint.
Here is a link to a recent paper on 214
http://clincancerres.aacrjournals.org/content/22/3/680.long
The bibliography may have some good references if your interested in IL2 as a target in general. I generally try a pubmed search when I am curious about how well studied a target is http://www.ncbi.nlm.nih.gov/pubmed sorry not specific references.
Nektar has 2 other programs a bit earlier too (IL5 and IDO). I asked ohad on his blog about his thoughts on 214 and he mentioned Roche is targeting IL2 too (I am not familiar with their program)
Depends on perspective... long NKTR
Actually the launch has been a bit of a disappointment the most impressive thing about it is AZ has inked two pretty good partnership deals. It may still turn out to be a big drug but certainly isn't happening quickly.
I've mentioned it in a couple places but NKTR's most valuable asset a year from now could end up being their first immunotherapy candidate NKTR-214 (they also have a couple other in the pipeline). First patient data should come later this year.
Some good Ricky stuff on Youtube
Jazz Defitelio cost:
If I did the math right came up with 156k for 21 day course of therapy (for 70kg person)
https://twitter.com/MauriceOnTW/status/715639283885948930/photo/1
JAZZ Defibrotide US Approval Call:
The webcast is starting shortly. The link can be found here:
http://investor.jazzpharma.com/phoenix.zhtml?c=210227&p=irol-eventDetails&EventId=5221334
Don't know if these will work without registering first, if so here are the slides:
http://event.lvl3.on24.com/event/11/56/11/4/rt/1/documents/resourceList1459448490417/defitelioinvestorupdate_033116_v2.pdf
And Label:
http://event.lvl3.on24.com/event/11/56/11/4/rt/1/documents/resourceList1459439835254/defiteliopi.pdf
JAZZ:
Here is the Jazz PR
http://finance.yahoo.com/news/jazz-pharmaceuticals-announces-fda-approval-183200040.html
They will have a conference call tomorrow at 4:30pm ET
Protalex (PRTX) and Protalix (PLX) are two of the worst offenders what makes it so (IMO) is an addition to the name confusion pulled their IPO this year PLx Pharma (PLXP). Maybe they should reverse merge instead of IPO and save a symbol
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 April-June conferences, Added OCUL Investor Day
Cowen & Co. 36th Annual Healthcare Conference
3/7-9
http://wsw.com/webcast/cowen30/
Future Leaders in the Biotech Industry
http://www.biocentury.com/conferences/futureleaders/dates
3/11
28th Annual Roth Conference
http://www.roth.com/main/Page.aspx?PageID=7270
3/13-16
ProQR R&D Day
http://www.proqr.com/rd-day/
3/14
28th Annual ROTH Conference
http://wsw.com/webcast/roth30/
3/14-16
Barclays Global Healthcare Conference
https://cc.talkpoint.com/barc002/031516a_ae/
3/15-17
Bank of America Health Care Back-to-Basics Forum
4/5
OCUL Investor Day
http://investors.ocutx.com/phoenix.zhtml?c=253650&p=irol-calendar
4/6 12:00pm
Jefferies Immuno-Oncology Summit
4/7-8
Barclays Healthcare 101 Day
4/8
Goldman Sachs Alzheimer’s Symposium
4/11
15th Annual Needham Healthcare Conference
http://wsw.com/webcast/needham76/
4/12-13/16
Bloom Burton & Co. Healthcare Investor Conference
5/2-3
https://www.bloomburton.com/conference/
Deutsche Bank 41st Annual Healthcare Conference
5/4-5
Bank of America Health Care Conference
5/10-12
Piper Jaffray 3rd Annual GenomeRx Symposium
5/17-18
Goldman Sachs 37th Annual Global Healthcare Conference
6/7-9
Jefferies 2016 Healthcare Conference
http://www.jefferies.com/OurFirm/Conferences/325/267
6/7-10
Citi European Healthcare Conference 2016
6/21-22
ROTH Capital Partners 3rd Annual Healthcare Day
6/22
J.P. Morgan European Healthcare Conference
6/23
Jefferies Alzheimer's Summit
6/28
--
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.
I looked over the report and while its long it repeats a lot! Seems to be a lot of the company line/presentation. Only thing I caught of interest which perhaps I missed OCRX disclosing at some point is this about the NIH study
There is a free research report available on the company available from
http://www.lifescicapital.com/equity-research/ocera/
You need to sign up so I don't think the direct link above will work. I haven't gone through it all yet just skimming through it is fairly detailed though. Not bad for free
The slingshot call that Adam referred to was interesting for those that didn't listen you can get a replay (I think) by signing up for the site. They have a free version which I am currently on though I would consider subscribing in the future. The site is founded with the objective of giving retail investors access to some of the doctors/experts such as those hosted by various investment firms.
http://slingshotinsights.com/projects
I've listened to a couple calls and found them worthwhile though nothing especially insightful. The Dr. Ratain call for example mentioned a lot of the things bought up on this board but it did add a couple of points I didn't give much thought to in the past.