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I'm wondering if this could backfire and there could be more scrutiny on insurance companies and pbm's. I didn't like it years ago when my insurer tried to change me from one PPI to another (that they likely had a deal with). I wonder if at some point there isn't legislation requiring insurers to reimburse all medicine prescribed for the indication it was indicated for and not favor one drug over another if they are not chemically equivalent... I could see it getting worse (for insurance companies) in Biosimilars if a patient is forced to switch and then has an anaphylactic reaction.
I don't know PW well (not at all actually) but my sense is in agreement with yours with the interviews I've read/seen of him. That being said I really like royalties ! Here are a few reasons why I'd like a royalty based partnership:
1) A royalty is more valuable then a percent interest since there are no expenses.
2) If you get a good partner (actually in Clinuvels case a so-so partner with Regulatory/commercial experience) they would advance the program/commercialize at a faster rate so there is a time/value associated with that. The track record of Clinuvel is extremely slow on developing, regulatory and now commercialization.
3) Without a partner they are either going to go extremely slow with Vitiligo and/or dilute a fair amount. My guess with chronic treatment in a fairly large disease indication they will need to well controlled studies with perhaps a thousand (or more) patients and then some descent long term safety. If you take an estimate of 50k/yr per patient in trial that could be 100m+ for 2 years of development.
IMO, I don't get the sense they'll get a top tier partner they need a better formulation and don't have a lot of data on Vitiligo to get someone overly excited at this point. The singapore study was another example of terrible execution of the company.
Some of the presenting companies at Cell & Gene Meeting today bit more followed:
Times are PT. Don't see replays for yesterday so suggest tuning in live if interested
http://www.meetingonthemesa.com/webcast/track1/
1:00pm – 1:15pm Sangamo BioSciences
1:15pm – 1:30pm bluebird bio
1:30pm – 1:45pm Editas Medicine
1:45pm – 2:00pm Intellia Therapeutics
2:00pm – 2:15pm Dimension Therapeutics
2:15pm – 2:30pm Voyager Therapeutics
2:30pm – 2:45pm Adverum Biotechnologies
4:15pm – 4:30pm MolMed
4:30pm – 4:45pm Argos Therapeutics
4:45pm – 5:00pm Kiadis Pharma
I think management is painting things very one sided in terms of redtape. First they wouldn't have quite as many restrictions if they had done proper trials and had sufficient safety data. If I recall correctly one of the documents seemed to suggest they didn't even have sufficient animal tox data for certain things. Secondly, they act as if they're the first company to have to maintain a registry, follow patients and deal with closed distribution. While the terms may vary there are quite a lot of prior therapies with these types of restrictions. I think they are just trying to do things with people not experienced doing these things and so as they learn on the job they're running into a lot of "unexpected" things that others have run into and dealt with more rapidly.
While I don't like the idea of stock holders bothering patients the Chairman's' rebuke of shareholders was pretty bad they should look themselves in the mirror and say why are shareholders going to extremes to find out some information.
As I am not a big fan of management (execution, communication, etc.) I am cautiously optimistic about one speeding things along. My negative opinion of management has me a little concerned that they do a poor deal (in particular too low a royalty) but hopefully their deliberate nature prevents that.
For Vitiligo they almost certainly would need one at some point. Not just to help with development costs but also commercialization/marketing a bigger company would put the necessary resources behind it. So it makes sense to do. I saw your post (SS or GG?) about expected terms I don't know that I could make a much better guess as there are a lot of factors (territories, compounds involved, up-front cash vs milestones and royalties, etc.). I'd imagine a partner would want rights to any follow on compounds/formulations for the indications they license. The bad thing is these are quite early so probably hard to get much at this point.
For EPP ordinarily I would prefer they don't partner such a niche indication that they could easily commercialize themselves especially in a country like the US but the experience in Europe leaves me to wonder. But it may be hard to separate the same product for two different indications in the same territory so partner may want both (though different dosing may help in getting someone to just take one indication).
Agree about timing of a buyout but there is always risk so a big enough premium and I wouldn't complain . I don't get the impression management is the type to sell out so easily so its not something I spend much time considering.
SMMT:
I don't follow them that closely so I could be wrong. I had the impression the most likely event was a capital raise, followed by partnering their antibiotic with this not even being on the horizon.
As far as telegraphing deals I know you owned a company (I briefly had a small position) where the CEO was very specific about doing a deal and then meeting that timeline! In fact I think he did that on a couple of occasions....ARRY sound familiar?
Anyway congratulations on SMMT! Guess its still a high reward, high risk play .
ITEK:
Yes, they've guided for data this year.
AERI vs. ITEK
I don't have a big position in ITEK but I am long. I don't see it as an either or especially at this stage of the game. If ITEK's current Phase 3 is successful they still need a 2nd and a long term safety study so AERI is certainly much further along. Also ITEK just started their Phase 2 dose ranging study combining with Latanoprost so it'll be a while before they get to a registration study. To me the valuation was cheap when I got in (after the bio collapse early this year) and saw opportunity for several glaucoma drugs (VRX has one too that had a setback recently). ITEK also said they would do some work with Trabodenoson on eye diseases. I thought we would have some data this year but I would guess it likely isn't going to happen till next year since the company hasn't mentioned it in a while.
NBC had a story last year
http://www.nbcnews.com/dateline/video/full-episode--out-of-the-shadows-469336131891
Nightline did as well
http://abcnews.go.com/Nightline/video/11-year-girl-allergic-sunlight-32199496
I guess most people don't pay much attention since its so rare. The FDA EPP Workshop later this month should hopefully help push the medical need.
https://www.eventbrite.com/e/scientific-workshop-for-erythropoietic-protoporphyria-epp-registration-24971245668
Cell & Gene Meeting
http://www.meetingonthemesa.com/webcast/
Don't know if there will be replays or not so may want to catch the live streams if interested in any of the companies (most are names I don't and some never heard of).
AERI:
Here is a direct link to the webcast
http://edge.media-server.com/m/p/9f3jhira
And the slides:
http://files.shareholder.com/downloads/AMDA-29CD43/2836956075x0x910637/C6A47881-D400-4D27-B13E-0164FDC129DC/AERI_Investor_Day_October_2016.pdf
If anyone listened/listens I'd appreciate any notes/comments as I doubt I will listen. Thanks in advance.
HCV treatment in prisons:
http://content.healthaffairs.org/content/35/10/1893.abstract
Prisoners bear much of the burden of the hepatitis C epidemic in the United States. Yet little is known about the scope and cost of treating hepatitis C in state prisons—particularly since the release of direct-acting antiviral medications. In the forty-one states whose departments of corrections reported data, 106,266 inmates (10 percent of their prisoners) were known to have hepatitis C on or about January 1, 2015. Only 949 (0.89 percent) of those inmates were being treated. Prices for a twelve-week course of direct-acting antivirals such as sofosbuvir and the combination drug ledipasvir/sofosbuvir varied widely as of September 30, 2015 ($43,418–$84,000 and $44,421–$94,500, respectively). Numerous corrections departments received smaller discounts than other government agencies did. To reduce the hepatitis C epidemic, state governments should increase funding for treating infected inmates. State departments of corrections should consider collaborating with other government agencies to negotiate discounts with pharmaceutical companies and with qualified health care facilities to provide medications through the federal 340B Drug Discount Program. Helping inmates transition to providers in the community upon release can enhance the gains achieved by treating hepatitis C in prison.
h/t @RebeccaDRobbins
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 October, November events; Added bluebird Bio, Eagle events
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
http://www.meetmax.com/sched/event_37061/~public/conference_presentations.html?event_id=37061&bank_access=0
BLRX Investor Day
9/22 9:00am
http://ir.biolinerx.com/phoenix.zhtml?c=208785&p=irol-EventDetails&EventId=5236931
ALKS Investors Event
9/25
http://phx.corporate-ir.net/phoenix.zhtml?c=92211&p=irol-calendar
ALDX R&D Day
9/26 8:00amET
https://event.on24.com/eventRegistration/EventLobbyServlet?target=reg20.jsp&referrer=http%3A%2F%2Fir.aldeyra.com%2F&eventid=1263617&sessionid=1&key=9CC241EF4734EA19F27264AEC6DBA7E7®Tag=&sourcepage=register
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
9/28-29
http://wsw.com/webcast/leerink27
Jefferies Cystic Fibrosis Summit
9/29
AERI R&D Day
10/5
http://investors.aeriepharma.com/events.cfm
Cowen 19th Annual Therapeutics Conference
10/5-6
http://www.cowen.com/conferences/upcoming-conferences/
Jefferies Gene Editing & Gene Therapy Summit
10/11
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
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
Jefferies London Healthcare Conference
11/16-17
Canaccord Genuity MedTech & Diagnostics Forum
11/17
Oppenheimer & Co. Inc. 2016 Life Sciences Summit
11/29
Piper Jaffray 28th Annual Healthcare Conference
11/29-30
--
Procedure for Updating Calendar
When adding or modifying entries, please follow these steps:
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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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SMA Drugs (BIIB/IONS, AXVS/RGNX)
I wasn't too familiar with either of the two programs and just started looking at them (mostly because of an interest in RGNX than any of the other companies).
It seems to me the drugs should in theory be very complimentary since AVXS-101 targets the SMN1 gene whereas Nusinersen targets SMN2. Assuming there are no overlapping toxicities of course it would make sense to give the gene therapy and follow up with regular Nusinersen. Since I don't have much of a science background and haven't followed the programs I'd be curious on opinions from others. Thanks in advance.
SMMT:
I heard the call a few things from memory:
- Gives them cash through end of 2018
- Don't look for another deal (at least in DMD)
- May be an opportunity for drug in Becker's MD
- Monotherapy is the current focus
- Said had others interested but by comments seemed only SRPT made an offer. Said something to the effect of interest before but once got approval more interested.
- Said have had partnership discussions on antibiotic
Here are @beauregard2 comments/notes
http://www.twitlonger.com/show/n_1sp5sm4
SMMT Webcast link (8am ET)
http://edge.media-server.com/m/p/88x5negc
SMMT:
Sarepta Therapeutics and Summit Enter Into Exclusive License and Collaboration Agreement for European Rights to Summit’s Utrophin Modulator Pipeline for the Treatment of Duchenne Muscular Dystrophy
https://finance.yahoo.com/news/sarepta-therapeutics-summit-enter-exclusive-110000517.html
OK you were right but the upfront still puts it in the size that I wouldn't consider it a significant deal
. Sarepta and Summit collaborate to advance the development of novel therapies for patients with Duchenne muscular dystrophy
. Summit receives $40 million upfront, with potential future ezutromid-related milestone payments totalling up to $522 million plus royalties
. Sarepta and Summit to share research and development costs
. Sarepta also receives option for Latin American rights
CAMBRIDGE, Mass. and OXFORD, United Kingdom, Oct. 04, 2016 (GLOBE NEWSWIRE) -- Sarepta Therapeutics (SRPT) and Summit Therapeutics plc (SMMT) (SUMM.L) today announced that they have entered into an exclusive license and collaboration agreement granting Sarepta rights in Europe, as well as in Turkey and the Commonwealth of Independent States (‘the licensed territory’), to Summit’s utrophin modulator pipeline, including its lead clinical candidate, ezutromid, for the treatment of Duchenne muscular dystrophy (‘DMD’). As part of the agreement, Sarepta also obtains an option to license Latin American rights to Summit’s utrophin modulator pipeline. Summit retains commercialization rights in all other countries.
Utrophin modulation is a potential disease-modifying treatment for all patients with the fatal muscle wasting disease DMD, regardless of their underlying dystrophin gene mutation. Ezutromid is currently in a Phase 2 proof of concept trial called PhaseOut DMD.
"This partnership with Summit Therapeutics furthers our commitment to invest in innovative approaches to treating Duchenne and supports our common goal of improving the lives of patients with DMD," said Edward Kaye, M.D., Sarepta’s Chief Executive Officer. "Summit’s utrophin modulation technology represents a potentially promising approach to treat DMD, which may complement our current approach of exon skipping therapy.”
“Sarepta Therapeutics has paved the way in the development of disease-modifying therapies for DMD with the first FDA-approved drug in this disease area, making them a strong strategic partner to support our utrophin modulator pipeline,” commented Glyn Edwards, Chief Executive Officer of Summit. “This agreement provides us with access to Sarepta’s development, regulatory and commercialisation expertise for the continued advancement of our promising utrophin modulator pipeline. We look forward to this partnership and working together to bring great advances to patients and families living with DMD.”
Under the terms of the agreement, Summit will receive an upfront fee of $40 million. In addition, Summit will be eligible for future ezutromid related development, regulatory and sales milestone payments totalling up to $522 million, including a $22 million milestone upon the first dosing of the last patient in Summit’s PhaseOut DMD trial, and escalating royalties ranging from a low to high teens percentage of net sales in the licensed territory. Summit will also be eligible to receive development and regulatory milestones related to its next-generation utrophin modulators. Sarepta and Summit will share specified utrophin modulator-related research and development costs at a 45%/55% split, respectively, beginning in 2018. If Sarepta elects to exercise its option for Latin American rights, Summit would be entitled to additional fees, milestones and royalties.
Sarepta and Summit will host an update call for the Duchenne community on Monday, October 10 at 12:00pm EDT. Details of the call can be accessed by visiting http://www.parentprojectmd.org/communitycall.
In addition to the PPMD call Summit will host a CC at 8am ET info can be found here
http://inpublic.globenewswire.com/releaseDetails.faces?rId=2046442
Orioles, Giants
Orioles, Red Sox
Dodgers, Cubs
Orioles, Cubs
Orioles in 6
34
AUPH:
I am a former long and didn't (yet) listen to the breakfast Friday but paging through the slides
http://www.auriniapharma.com/dnn/LinkClick.aspx?fileticket=DbugZOK0EOQ%3d&tabid=121&mid=723
a few interesting things popped up:
1) Though they claim don't need to dose to weight there certainly appears there may be a dose response (men did better in the high dose and women worse, slide 61).
2) I give them credit for breaking out the deaths in detail and the number of patients at each site particularly slide 110 where they show the randomization by country where deaths occurred. Though comparing total deaths to other studies is a very weak argument IMO.
That being said I doubt I'll consider reentering the stock their financial position is pretty weak and even if the deaths are explainable (I'm still unsure) I would have liked to see better efficacy.
SMMT:
Just to add to my skepticism on SMMT. It should be noted they are now on (at least) their third formulation of their Utrophin modulator. I think with their current market cap the street is sorta saying show me you have it figured out. I know that is one of the main things that keeps me from even a small position.
SRPT / DMD Deals:
I am not saying SRPT will not pursue combinations in fact I would be surprised if they don't AT SOME POINT. My thinking is they have limited resources at present with trying to get other exon's developed and then their next generation PPMO technology which I think they will priorities over spending a lot of capital (human and financial) on a big deal. To do these small preclinical collaborations costs them practically nothing in terms of resources so why not do them. To fully develop another technology in combination with exon skipping will take a lot more resources which I think they won't do for a couple years.
I think combination approaches in general make a lot of sense especially since the efficacy is limited. Once a company has an approved drug I think competitors are more interested in combining then the first to market (in general). Sarepta of course is a unique situation so we will see.
SRPT / DMD deals:
He was given options to buy at 4.
BMY / NKTR:
I heard the call it was (surprisingly) quite brief. They gave out some data on the ongoing MAD study and I think that is why the stock is down. I thought it especially unusual that they would not have slides or PR the data I'd have to go back and listen more closely to get what they did reveal. There were no RECIST responses thus far but I believe 9 (of 16?) continue on treatment. I believe they have enrolled 23 thus far. The safety seems to be a non-issue thus far with I believe one or two (grade 3) SAE's that were transient (responded to fluids) and I believe 1 discontinuation (injection site reaction but they said person also had same to an MAB).
On the BMS deal they have 2 year right of first negotiation. Then afterwards they have 10 days to match any offer. More info is in the 8-k on that.
Sorry all this is rough I didn't take notes.
GLPG / GILD:
2 are for Crohn's and 2 for UC each has a safety (long term extensions study). Yes the two main trials are listed as opening in October. Personally to me the most important thing is reading the trial design especially when the company hasn't disclosed it yet! I would say its worth a click and I have no compensation for the clicks
GLPG / GILD:
The company said they couldn't comment on P3 trial till Gilead goes ahead... and then these appear today
Filgotinib in the Induction and Maintenance of Remission in Adults With Moderately to Severely Active Ulcerative Colitis
https://clinicaltrials.gov/ct2/show/NCT02914522
Filgotinib in Long-Term Extension Study of Adults With Ulcerative Colitis
https://clinicaltrials.gov/ct2/show/NCT02914535
Filgotinib in the Induction and Maintenance of Remission in Adults With Moderately to Severely Active Crohn's Disease
https://clinicaltrials.gov/ct2/show/NCT02914561
Filgotinib in Long-Term Extension Study of Adults With Crohn's Disease
https://clinicaltrials.gov/ct2/show/NCT02914600
ALKS:
The webcast is today (14 minutes) not yesterday
The links for the webcast and separate slides PDF are available on their IR page:
http://phx.corporate-ir.net/phoenix.zhtml?c=92211&p=irol-calendar
Direct Link to webcast: https://event.webcasts.com/starthere.jsp?ei=1116082
Direct Link to Main Presentation: http://phx.corporate-ir.net/External.File?t=1&item=VHlwZT0yfFBhcmVudElEPTUyMzcyMjl8Q2hpbGRJRD02NDYyODQ=
PTIE:
GLPG / GILD:
The call was very short. No 20 week data available, will be presented later date. Only biopsy done at 10 week so at 20 weeks only CDAI
If it works here is the poster link:
https://cslide.ctimeetingtech.com/ueg2016/confcal/filgotinib#abstract-1563
GLPG / GILD:
The last two rows in the table weren't presented before. Actually the first row the 3/18/16 (don't know if its on website or not) slides have 48% (not 47%) for filgotinib. I don't know why their isn't a p value for the 50% improvement in SES-CD scores just a NS, numerically it looks like it would have been close.
There is a CC at 8amET today for anyone interested. Webcasts usually have link posted (near time of call) @ home page http://www.glpg.com/ (If not) Dial In Info is:
USA: +1 719 457 2086
UK: +44 330 336 9411
Netherlands: +31 20 703 8261
France: +33 1 76 77 22 57
Belgium: +32 2 400 6926
That is pretty good! Is it a one stock exception or a bull market in bio?
EDIT:
Here is the PR
http://finance.yahoo.com/news/sarepta-therapeutics-announces-pricing-300-220000748.html
QURE:
Not the best of thing to see after being CEO for less than a year. The quote they have from him seems genuinely a family problem and for his sake hope everything turns out well in his personal life! He did give the company a big vote of confidence by buying a 100k shares at I believe around 10 (via 105b) a little while after he was named and hasn't sold.
SRPT:
Its a one year old article and how close to an acquisitions are they?
They've been talking their own next generation PMO's from before I was a shareholder (am not now). I think that is really their priority after getting other Exon's that are in the pipeline out of course.
I think it sounds good and is LONG range plans. Perhaps they do a very early (cheap) deal but I wouldn't imagine anything over say $50m. They'll need a lot of that 500m in the next couple years (and I'd expect at least another raise before being profitable if I were a long trying to do a model too). Keep in mind they were running below 1 year cash which few Bio CEO's like to be at. The gamble paid off for them.
SRPT - DMD:
With their balance sheet I wouldn't bet on them doing a significant deal at this point. I think their priority would be to develop their next generation PMO's. Art Krieg (who should know) on twitter said they should be significantly more potent/better uptake.
PFizer has an interesting program in DMD though its hard to get a status update with them being so big. I'd imagine someone like that would be more likely as they have the resources to try to combine different MOA's.
SRPT:
On the call they said that they are expecting the patients getting treated will be earlier then in their trial. But they said estimating 25kg average weight, I imagine the average weight (and thus cost) will be significantly greater perhaps 300k sounds "friendlier" when compared to other ultra-orphans.
OCRX: Ocera Initiates Phase 1 Clinical Study of Orally Available OCR-002 in Patients with Cirrhosis for the Prevention of Hepatic Encephalopathy
Don't know if the price move friday was related to this (it was an expected event).
http://ir.ocerainc.com/releasedetail.cfm?ReleaseID=989875
--Phase 2b STOP-HE Enrollment Surpasses 205 Patients; On Track to Complete in Q4 2016--
PALO ALTO, Calif. and RESEARCH TRIANGLE PARK, N.C., Sept. 19, 2016 (GLOBE NEWSWIRE) -- Ocera Therapeutics, Inc. (NASDAQ:OCRX), a clinical stage biopharmaceutical company focused on acute and chronic orphan liver diseases, today announced the initiation of a Phase 1 clinical study of orally-available OCR-002 (ornithine phenylacetate) in patients with cirrhosis. OCR-002 is in development in both an intravenous (IV) formulation as a potential treatment for hospitalized patients with acute hepatic encephalopathy (HE), a neurocognitive disorder associated with serious liver disease, and in an oral formulation to potentially provide a chronic use option to maintain remission of HE in patients with cirrhosis.
"Based on the favorable results from last year's Proof of Principle study in healthy volunteers, we are keen to assess our oral drug candidate for the first time in patients with cirrhosis, the intended population for the drug's chronic use," said Stan Bukofzer, M.D., Chief Medical Officer at Ocera. "In healthy volunteers, oral OCR-002 demonstrated significant bioavailability, as measured by the mean plasma concentration of the drug's active ingredient phenylacetic acid (PAA), and exhibited substantial concentrations of plasma and urinary phenylacetylglutamine (PAGN), the end product by which PAA clears the neurotoxin Ammonia.
"OCR-002 is, to our knowledge, the only ammonia scavenger in clinical development for the treatment and now prevention of HE. Over 205 patients with acute HE have been enrolled in the STOP-HE Phase 2b trial of our IV formulation of OCR-002, and with the initiation of this Phase 1 trial for our oral formulation of OCR-002, we believe we are closer to realizing our vision of providing continuity of care to HE patients."
About the Phase 1 Orally-Available OCR-002 Study
The Phase 1 trial is a two-part, open-label, crossover study to understand the pharmacokinetics, PAGN formation and the absolute bioavailability of oral immediate-release doses of OCR-002 in 12 patients with varying degrees of cirrhosis. Part One will evaluate a single dose of an oral solution of OCR-002 administered under various fasted and fed conditions compared to a single dose of IV OCR-002 in patients with cirrhosis classifications of either Child-Pugh1 A or C, indicative of the degree of liver disease. We expect to complete and report top-line results of Part One of the trial by the end of 2016.
Part Two, also in patients with cirrhosis, will evaluate a multi-dose solid form regimen and resultant steady-state pharmacokinetics, and data is expected by the end of the first half of 2017.
We intend to initiate a Phase 2 development program for orally-available OCR-002 upon satisfactory results from both parts of the Phase 1 trial.
About Hepatic Encephalopathy
Hepatic encephalopathy is a debilitating and progressive complication of liver cirrhosis or liver failure, marked by mental changes including confusion, impaired motor skills, disorientation, and in its more severe form, stupor, coma and even death.
About Ocera
Ocera Therapeutics, Inc. is a clinical stage biopharmaceutical company focused on the development and commercialization of OCR-002 (ornithine phenylacetate) in both intravenous and oral formulations. OCR-002 is an ammonia scavenger and has been granted orphan drug designation and Fast Track status by the U.S. Food and Drug Administration (FDA) for the treatment of hyperammonemia and resultant hepatic encephalopathy (HE) in patients with acute liver failure and acute-on-chronic liver disease.
Ocera's HE clinical development efforts also include an ongoing Phase 2b clinical trial, STOP-HE, which is evaluating the safety and efficacy of intravenously-administered OCR-002 in resolving neurocognitive symptoms of acute HE in hospitalized patients with elevated ammonia. The Company expects to complete enrollment in the STOP-HE trial in the fourth quarter of 2016 with top-line data to be published soon thereafter. For additional information, please see www.ocerainc.com.
Forward-Looking Statements
This press release contains "forward-looking" statements, including, without limitation, all statements related to the OCR-002 clinical development program, including but not limited to the potential benefits of OCR-002 to help patients with hepatic encephalopathy, the timing of clinical and enrollment milestones, and the timing of our clinical development plans and release of study data. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believe," "expected," "hope," "plan," "potential," "will" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Ocera's current expectations. Forward-looking statements involve risks and uncertainties and Ocera's actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, including those risks and uncertainties discussed under the heading "Risk Factors" in Ocera's Annual Report on Form 10-K for the year ended December 31, 2015 and subsequent filings with the SEC. All information in this press release is as of the date of the release, and Ocera undertakes no duty to update this information unless required by law.
1Child-Pugh Scoring is a clinically relevant method of assessing the severity of liver impairment in patients with cirrhosis. A score, ranging from 5 (least severe) to 15 (most severe), is calculated by totaling the scores of five discrete variables: serum bilirubin, serum albumin, prothrombin time, ascites and encephalopathy. Scores of 5-6 are classified as Child-Pugh A (well compensated disease); scores of 10-15 are classified as Child-Pugh B (disease with significant functional compromise); and scores of 10-15 are classified as Child-Pugh C (decompensated liver disease).
Source: Ocera Therapeutics, Inc.
OCRX-G
Susan Sharpe
Ocera Therapeutics, Inc.
contact@ocerainc.com
919-328-1109
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 Investor Events for BLRX, ALKS, ALDX, AERI; Added Jefferies CF Summit
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
http://www.meetmax.com/sched/event_37061/~public/conference_presentations.html?event_id=37061&bank_access=0
BLRX Investor Day
9/22 9:00am
http://ir.biolinerx.com/phoenix.zhtml?c=208785&p=irol-EventDetails&EventId=5236931
ALKS Investors Event
9/25
http://phx.corporate-ir.net/phoenix.zhtml?c=92211&p=irol-calendar
ALDX R&D Day
9/26 8:00amET
https://event.on24.com/eventRegistration/EventLobbyServlet?target=reg20.jsp&referrer=http%3A%2F%2Fir.aldeyra.com%2F&eventid=1263617&sessionid=1&key=9CC241EF4734EA19F27264AEC6DBA7E7®Tag=&sourcepage=register
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
9/28-29
http://wsw.com/webcast/leerink27
Jefferies Cystic Fibrosis Summit
9/29
AERI R&D Day
10/5
http://investors.aeriepharma.com/events.cfm
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
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That is a pretty harsh (and IMO unfair) statement. If one is looking at it from strictly an investor standpoint perhaps you have a point. If however one is looking at it from the perspective of a parent of a child with a fatal/debilitating disease and NO treatment options its quite unfair. Their bar for efficacy in their case is much lower and your willingness to assume risk (that the efficacy is non-existent or even some level of safety risk) much greater. I would describe these people in other ways. Perhaps there is a sense of frustration and despair but I think its worth considering the situation these people are in before a generalized painting of them this way.
OCRX:
Now a big move with higher volume (but not as high as past days that didn't move the stock). No news that I see, can't be a deal with the CBO leaving
http://ih.advfn.com/stock-market/NASDAQ/ocera-therapeutics-inc-OCRX/trades
OCRX:
Not the type of management shake up I lose sleep over . I thought it curious they hired someone in the first place a company their size should have the CEO doing these things. I do find it a waste of money and a bit annoying like when companies hire Chief Commercial Officers when their furthest along program is in Phase 2 3-4 years from potentially reaching the market (and often they fail too).
EDIT: I guess its not as bad (costly) as hiring a full fledge sales force (and retaining them) after 2 CRL's... The CEO is now with ARWR